TOPICS IN HEMOPHILIA

• First Annual Fund Raising Dinner
• Second Annual New York Symposium
• Spencer Duggan - Michael O’Connor
  
• Eshton Hewitt Quality of Life Project Update
  
• Hemophilia Alliance Group Purchasing Org.
  
• NHF Walkathon - NYC Hemophilia Chapter Event
  
• Factor Nine Family Meeting - Inalex

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The Coalition for Hemophilia B First Annual Fundraising Dinner

Dr. David Clark, Chairman

The Coalition for Hemophilia B held its first annual fundraising dinner at the Millennium Broadway hotel in New York on Friday, March 7th, 2008. Attendees included the lotto families we flew in for the symposium, industry people and private donors. Monies raised will benefit our Educational Programs and the William N. Drohan Scholarship fund. Dinner began with a warm welcome to our guests by Dr. David Clark, Chairman of the Coalition for hemophilia B, followed by the acappella singers from ISMILE Productions.

Their first song was Sunrise-Sunset dedicated in memory of Dr. William N. Drohan (it was Bill’s favorite song). Renae Baker from ISMILE presented the Drohan family with a beautiful framed song sheet of Sunrise-Sunset personally addressed to each member of the Drohan Family and signed by Sheldon Harnick (he wrote Sunrise-Sunset). The Drohan family was very surprised and gave us a heartfelt thanks.

“When I wrote the lyrics for ‘Sunrise, Sunset’, I was
thinking of my own children. It’s always gratifying
to learn that one’s work has moved another person.
In this case, I’m deeply touched to find that the song
affected someone who was engaged in such admirable
humanitarian work as ‘The Coalition for Hemophilia
B’. That’s the best reward a songwriter could have!”
Best, Sheldon

The Droham Family
Maureen, Craig, Laura, Kathleen,
and Tommy Little

Afterward the singers went around to each table singing Broadway’s greatest tunes. Following dinner was our Casino night where everyone had a fun time, indeed. We thank all our contributors and hope to see you all next year!

The Coalition for Hemophilia B

Second Annual Symposium

Generously Funded by Wyeth Pharmaceuticals

The Coalition for Hemophilia B’s second annual symposium was held on Saturday, March 8, 2007 at the Millennium Broadway Hotel in New York. The symposium began with opening comments and welcome. Our first speaker was Dr. Christopher Walsh, head of the HTC program at Mt. Sinai hospital in New York City. Dr. Walsh spoke about current gene therapy research and advances being made for the treatment of hemophilia. His talk was followed by a multitude of questions from symposium participants. Second on the agenda, Mike Herbert and Dawn Feraci from Patient Services, Inc. (PSI). They spoke about the ongoing and current issues with insurance and how PSI can work with individuals in need of insurance assistance. Our next speaker was not only energetic and inspiring but he was also quite colorful. Michael O’Connor, a 17-year-old high school senior, and a person with severe hemophilia B, spoke to the audience about how swimming helped him both physically and mentally. Swimming helped strengthen his joints so he had fewer bleeding episodes, and how he felt like “just one of the guys” on the swim team. He spoke about swimming competition and also told a funny story about one of his coaches, which drew a good laugh from the audience. Michael was a true inspiration to parents and children alike, as he made them feel more comfortable about how a child can grow up living healthy productive lives with hemophilia. Our next speaker was another true inspiration to all as well. Ben Shuldiner, is the Founder and principal of the High School for Public Service in Brooklyn, NY. Ben also has severe hemophilia B. His speech was called the “Blood of Others.” It was a story about growing up and living with hemophilia. Ben’s talk
touched everyone’s hearts. He is a true role model for everyone living with hemophilia. After a wonderful lunch we continued the afternoon with a slide show presentation by Wayne Cook, about Joint Replacement and Continuous Infusion. The presentation was well received and the audience had plenty of questions afterward. Following was a wonderful new game we introduced called “Are You Smarter than your Hemophilia?” hosted by Lisa Weiderman. Each table was a team, and they had 30 seconds to answer the correct question by raising their cardboard letters A,B,C, or D. The team with the most points won. But everyone received a trophy, as you are all winners to us! The game was fun and educational. Finally the sessions ended with our traditional family gathering. The Coalition’s forum where everyone has a chance to talk about themselves, their families and just about any issues they may have or any insight they might want to share with the group. A big thank you to our sponsor, exhibitors and speakers for making our symposium a success!

Ben Shuldiner speaks about “The Blood of Others.”

[Show as slideshow]

Symposium Attendee Comments

“Just wanted to let you know how much I enjoyed the symposium. From a consumer viewpoint I thought you really gave the families what they want to know about: 1.) ongoing research/where we are/where we are going. I can’t tell you how many people have said they would like to see more of these sessions at other meetings. 2.) inspiration-wow two fantastic individuals and speakers. 3.) funding/financial support 4. vendors - what is out there/more education on what is available. Overall I honestly thought it was one of the best symposiums I have been to and I look forward to attending next year.” M.M., Massachusetts

“The symposium weekend was a great experience for me. I very much enjoyed the sessions; the speakers were great as well as the topics. Of course it was great to attend the Fundraiser Dinner and meet people all weekend; some new and some old. I especially enjoyed meeting the Drohan family. They are good people just like everyone else there. Thank you so much to you and the coalition for the weekend!” T.A., Arkansas

“The Symposium was really good! The speakers were all great. We really enjoyed the interaction of the game. It was really funny, but educational and the trophies were awesome. Having the vendors there was good too, and being smaller, was more personal and not so overwhelming. We picked up some info/vendor giveaways for a newly diagnosed family in Alabama. The Factor Nine Family meeting was great as usual, and thanks so much for the special book given to all the families. The kids had a great time with Kasie and Shannon. They did a great job! The kids had lots of fun with the games, activities, and the Magician. We cannot thank you enough for bringing us to New York. We had such a wonderful time. These are such memorable experiences for our family. The Coalition is such a blessing to us and all the Factor Nine families.” R.B.,Alabama

“It was excellent!! Will you please forward any pictures if possible? We were so busy enjoying the time we didn’t take many. I really liked the game. And Dr. Walsh was incredible! My son got the most out of listening to Michael O’Connor. We really got something out of every speaker. Wayne talked about continuous infusion… Nathanael has to have oral surgery before getting braces so I’d like to check into that for him!! We had the best time, although it went so quickly!” K.R.P., Florida

“I had a really great time meeting everyone and I learned a lot from the symposium on Saturday. I thought Michael O’Connor’s presentation was very informative and helpful. I had heard that swimming was good for people with Hemophilia but I never knew how helpful until I heard what he had to say. I also liked Wayne’s presentation on continuous infusion, it was very enlightening and I will keep it in mind as we continue on our journey with Hemophilia. The “Are You Smarter Than Your Hemophilia?” was so much fun. I really felt connected to everyone after the family meeting where I was able to share my experience with hemophilia.” C.L., Maryland

“It was a privilege for us to travel across the country to participate in such an event. We had a wonderful time socializing with other families, meeting representatives, and listening to the presentations. After talking and listening to other people, I think we both realized how little we know about Hemophilia B. We want you to know how grateful we are for the motivating experience. Because of this, we intend on being more involved and upto- date on current issues and information regarding our bleeding disorder. Overall, our experience was fun, informative, and invaluable. Thank you for making it possible.” E.G. and C.G, Alaska

“Again we had a wonderful time. We cannot thank you enough for providing this valuable educational experience. We got a lot out of all the speakers. Ben Shuldiner’s talk was truly an inspiration. PSI did a wonderful job educating us about insurance issues, something we all need to be up to date on. We enjoyed the game, as you made it fun, but we also learned a lot. We enjoyed the opportunity to visit the exhibit booths to obtain more information as well. We can’t wait to come back next year!” A.H., New York

Treble, Concert & Master Singers of the Fairfax Choral Society

Spencer Duggan will be performing at CarnegieHall in New York City on April 20, 2008. Spencer is a member of the Chamber Singers, an advanced chorus of his high school Choral Department. Last year he sang with his school chorus at the Kennedy Center and Strathmore Hall for their Christmas concert with the Washington Chorus and won the Outstanding Musician of the Year award. He has lettered in music and been chosen to be a member of the District XI Chorus three years in a row. He is also a member of the Westfield High School Guitar Ensemble and the Jazz Ensemble. He was inducted into Tri-M, the Music Honor Society, this year. Outside of school, he is a member of the Fairfax Choral Society. He will be traveling with this group to New York City to perform John Rutter’s Mass of the Children at Carnegie Hall. Spencer will be majoring in Music in college. He has auditioned at several universities and is awaiting their decisions. His plans include vocal performance, composition, and perhaps music education. We wish him good luck and hope some of you will be able to see him perform. If you would like to purchase tickets for his Carnegie Hall performance, please call (212) 239-4669.

Spencer Duggan

Michael O’Connor Speaks at Symposium

Michael O’Connor, 17, spoke at the Coalition for Hemophilia B meeting on March 8, 2008, to Hemophilia B patients and their families. Michael is currently the three-time Nassau County Champion in the 100 Breaststroke, and is ranked Eighth in New York State Public High Schools in that event. He has qualified for NCSA Junior Nationals, and will be competing in Orlando, Florida, March 19 -24, 2008 in that elite meet. At the family meeting, Michael spoke to patients and their parents about how he is able to compete on a “level field” as a swimmer despite having Hemophilia B, and how the sport of Competitive Swimming has actually protected him from joint problems for the last 6 years. He pointed out the many positive aspects of serious competitive swimming as a sport, as well as how rigorous it is, and what it takes to reach National competition. Michael has been recruited to a prestigious college in New England, and he hopes to help lead the Men’s Swim Team to an NCAA title during his next four years there. We wish Michael much success at Junior Nationals!

Eshton Hewitt Quality of Life Update

In 2003, The Coalition for Hemophilia B formed an umbrella project called “The Eshton Hewitt Quality of Life Project” (see winter 2004 newsletter and alsounder Programs on our website). It began to help Eshton Hewitt who has hemophilia B with inhibitors and also Ehlers Danlos Syndrome (EDS), a connective tissue disorder that wreaked havoc on Eshton’s body causing severe joint damage and making it difficult to control the bleeding episodes that inevitably follow a subluxation, or dislocation.

After a great deal of research and struggle to find adequate long-term care for Eshton, his parents decided that his best opportunity to slow the progression of disease in the joints was to strengthen the muscles supporting them through aquatic exercise, which has basically no impact on the joints. Eshton was not able to swim in a public swimming pool due to the combination of his fragile skin and having a port, which makes him susceptible to infection. There is no way to regularly test a public pool for bacteria and the Health Department does not require that logs be kept showing their testing. This puts him at a very high risk.

The Hewitt’s started out with an outdoor above ground pool. When Eshton was swimming, his bleeding episodes decreased and they had to infuse him less. When Eshton has a bleed, he has to be infused every 3-6 hours (depending on the dosage) for 1-4 days until he has full range of motion due to the EDS. As published in the Michigan Flint Journal (Title: Pool to Help Boy Be Like Any Other Kid; Businesses come through for mom; July 26, 2003) “When Eshton is in water, everything is normal for him.” Joyce Hewitt explained. “One time he was swimming and said he was so happy we had the pool and that when I’m in here, it’s like I’m like any other kid!”
The difference was enough that Joyce and Jay wanted to give him a better quality of life year-round and that meant building an indoor swimming pool. Joyce and Jay Hewitt mailed out many letters and made numerous phone calls to prospective donors for this project. Soon after, donations for building supplies, pools, roofing material, windows and doors, a boiler and plumbing supplies came in. Individuals, companies and The Coalition for Hemophilia B donated monetarily as well. The Hewitt’s were completely overwhelmed and never expected an out-pouring of such magnitude. Joyce Hewitt said “Without the support we would never have been able to make this dream a reality for our son.”

As of 2008, The Coalition for Hemophilia B “Eshton Hewitt Quality of Life Project” is still going strong and continues to help fund the Hewitts on a yearly basis. The fund has also helped many families who have hemophilia, inhibitors, and also dual diagnosis to have a better quality of life. Following is an update on the Hewitts from Joyce Hewitt:

“First and foremost my family and I want to thank everyone who has contributed to the Eshton Hewitt fund. Thank you for your support both financially and in your hearts and prayers. We are keenly aware that without all of you we would not be able to provide these things that my son has desperately needed. And for that I will never be able to find enough words to adequately convey my gratitude to you all.
It’s now been almost five years since my husband and I first started working on the handicapped bathroom and pool. We’ve come a long way: Eshton’s able to use the pool and the bathroom unassisted and the boys are in their expanded room, but there is still a great deal that needs to be done.
We have at this point gotten the pool placed and enclosed; the boiler to heat the building, pool and handicap bathroom is placed and working. The handicapped bathroom is finished, new back doors have been installed and their bedroom has been enlarged for handicap accessibility. (And promptly turned into the Great Divide-MSU on Tekoa’s side and U of M on Eshton’s!)
Eshton has had few ankle bleeds since he has been able to use the pool, but continues to suffer from easily dislocating joints in his extremities. The muscle strengthening, joint stability, and conditioning he’s been able to gain has come only as a result of aquatic therapy. Before the pool, Eshton was bleeding into each ankle 6-9 times a year. He’s had less than 6 in the past 4 years! Although Esh bleeds less in his ankles, he still continues to bleed in his lower extremities on a very regular basis. He is using his wheelchair more and more. He was also just diagnosed with scoliosis.

There is still so much more that needs to be done. We are doing all the labor in our home with further renovations to create a home that will be handicap accessible for Eshton. A home that will serve him, throughout his lifetime. We have replaced doors, put in a medical closet, removed obstructions, and created new entry ways for wheelchair accessibility. Our next step is to remodel the kitchen for handicap accessibility. Our twin boys are now 13 and at an age where independence and dependence are often at war. They want to make their own decisions, learn who they are, and challenge what we know as elders. It’s the way it’s supposed to be, for young people as well as old alike, to make the break from each other. There’s no difference here except that in order for Eshton, and to a smaller extent, Tekoa, to learn and grow from those “learning” experiences, they need some adaptations that most of us don’t. Their ability to become independent, selfsufficient people that we all want for our children simply requires from us a little bit more than most. We consider it a privilege and an honor to be blessed with such amazing children. Currently, we need about $50,000 to complete the project. Donations of appliances, flooring, light fixtures, kitchen cabinets, counter tops, windows, dry wall, lumber. I’ve taken some pictures of where we are now. Just remember: we’ve been living in the construction phase for almost five years now! Joyce Hewitt.

If you would like to make a donation to the Eshton
Hewitt Quality of Life Project please send your
donation to:
The Eshton Hewitt Quality of Life Project
The Coalition for Hemophilia B
825 Third Avenue, Suite 226
New York, NY 10022

All donations and materials will go directly to the
Hewitt family.

The Eshton Hewitt Quality of Life Project is
funded by Novo Nordisk

Hemophilia Alliance Group Purchasing Organization

(HAGPO)

Two years ago, a group of Hemophilia Treatment Centers, formed a group purchasing organization called Hemophilia Alliance Group Purchasing Organization (HAGPO). The group is headed by Mark Plencner, RPH, President. Mark is the managing pharmacist of the hemophilia program at Roger Maris, Cancer Center in Fargo, North Dakota. Joe Pugliese is the Executive Vice President. Joe has been involved in the hemophilia community for the last 30 years. Currently there are 26 participating treatment centers enrolled. It is a federal requirement that each member of HAGPO sign a participation agreement.

The objectives of this organization are to:
1. Secure a “guaranteed” supply of product in the event of shortage to help care for the needs of their patients.
2. Negotiate better prices and terms to effectively manage their business, which in turn will help patients in managing a very expensive disease, in the most economical manner possible.
3. Create a pool of unencumbered dollars that would be reinvested in the hemophilia
community.

In January, 2008, HAGPO made its first reinvestment to the community by giving a restricted grant in the amount of $25,000 to the National Hemophilia Foundation to fund nurses, social workers, and physical therapists from each region across the country to attend MASAC working group and educational programs. Any remaining support will be used toward chapter educational activities.

HAGPO is currently in the process of developing a foundation, headed by Joe Pugliese, which will be independent of HAGPO. The foundation board will make the decision as to the distribution of funds that HAGPO gives to the foundation to reinvest in the community’s national and local advocacy organizations and HTC’s. Joe Pugliese stated, “The goal is to support operational excellence, helping provide the resources necessary to fulfill the recipient’s mission.” The plan is to utilize staff members from other member organizations for administrative positions, thereby lowering overhead cost. Guidelines will be available soon.

We will keep you posted as to further developments with this organization. For more information please contact Joe Pugliese at (215) 439-7173 or email joe_Pugliese@hotmail.com

National Hemophilia Foundation Walk

Please join the Factor IX Team as we walk on Sunday, June 1, 2008 with the National Hemophilia Foundation to raise monies for research. We will meet at Pier 1 at 70th Street, 8:30 am. It is a casual fun walk; you can walk 1 mile or 5 miles. Bring your dog, your friends, neighbors, and grandparents, and have a great time walking! Each person should get sponsors for the walk and checks should be made payable to: Hemophilia Walk, The National Hemophilia Foundation 116 West 32nd Street, 11th Floor New York, New York 10001 When sending checks to the National Hemophilia Foundation, please make a note that funds should go under the Factor IX Team and person’s name you are sponsoring. You can also register and donate online at www.hemophilia.org/walk (our team is listed under Factor IX). Or go directly to website address: www.active.com/donate/hemophiliaWalkNY/FactorIXTeam.
For more information please contact Kim Phelan at hemob@ix.netcom.com.

New York City Hemophilia Chapter Event

New York City Hemophilia Chapter, headed by Melissa Penn, mother of a son with Factor VIII, is launching its first event on Sunday, April 13, 2008 at the American Museum of Natural History. The event is for all people with bleeding disorders and their families. It is free of charge and includes museum admission, private tour and lunch. Meet at 10 am, private tour of the dinosaurs, whale room, and dioramas at 11 am, lunch is at noon.

Pre-registration is required. You can also register online at www.nyhemophilia.org. Please call Melissa at 212-877-0280 if you have any questions.

New Inalex DVD in the “A Bright Future” Series Now Available

In 2005, Inalex Communications founder and president, Joe Caronna, conceptualized a series of topic specific DVD’s that would offer the bleeding disorder community “Hope, Information and Inspiration”. To realize his vision, Joe partnered with a multiple Emmy and Peabody award winning film crew to create Inalex Productions. The result was the first DVD of the “A Bright Future” series, titled “The Hemophilia Diagnosis for the Parent”. This DVD made its premiere at the 2006 National Hemophilia Foundation’s annual meeting in Philadelphia, and was extremely well received and touted as “A must see for all new parents!” and “Will give others hope that they too can master their new life.”

Inalex Productions is now excited to announce the release of the second DVD in the series titled, “The Hemophilia Diagnosis for the Extended Family”. The DVD was premiered at the 2007 National Hemophilia Foundation’s annual meeting in Orlando, and once again, was extremely well received by the bleeding disorder community. Some of the comments were, “This DVD is a professional, sensitive, intelligent, and needed education tool for all family members”, and “Inalex has done it again! This DVD points out the importance and the benefits of the extended family involvement…well done !”

Inalex Productions is currently in pre-production on their third volume in the series, which offers education and support to educators of school aged children with hemophilia. If you would like to receive a complimentary copy of either “The Hemophilia Diagnosis for the Parents” and/or “The Hemophilia Diagnosis for the Extended Family”, please go to www.inalex.com, click on the “Inalex Productions” button, fill out the form, and submit for immediate delivery. This DVD series is sponsored by Baxter BioScience.

Inalex Communications

38 East Ridgewood Avenue, #244
Ridgewood, NJ 07450
201 493-1399
www.inalex.com

FYI: Hemophilia B Online Group

Website address for the Hemophilia B online group:

http://health.groups.yahoo.com/group/Factor9/

Factor Nine Family Meeting in May

Sponsored by CSL Behring

The Coalition for Hemophilia B

Factor Nine Family Meeting

Friday, May 2, 2008
5:45 pm - Manning Room
in the Peabody Hotel
Come and eat dinner, meet
your peers, share stories
and information.
New - fun educational game
show!
We look forward to seeing you!

Held in conjunction with the

Hemophilia Federation of
America
Annual Symposium
May 1-4, 2008
Peabody Hotel
200 West Markham Hotel
Little Rock, Arkansas 72201
(501) 906-4000

For back issues of Factor Nine Newsletter or for more information on research, please call or write to:
Kim Phelan; 825 Third Avenue, Suite 226; New York, New York 10022; Telephone (212) 520-8272
Telefax (212)520-8501; E-mail: hemob@ix.netcom.com Website: www.coalitionforhemophiliab.org

Funding for this newsletter is provided by a grant from Wyeth Pharmaceuticals

- Factor Nine Family Meeting – Orlando, Florida
- NHF – Coalition Award
- Factor Nine Products in Development
- Coalition for Hemophilia B 2008 Fundraising Event & Symposium

FACTOR NINE FAMILY MEETING, ORLANDO FLORIDA

We had a wonderful turnout at our Factor Nine Family meeting held in Orlando Florida in conjunction with the National Hemophilia Foundation Conference in November 2007. Over 65 people attended this meeting. We will keep you informed of upcoming meetings in 2008 in our spring edition of Factor Nine News !

Wayne Cook, Ray Stanhope, David Clark

On Thursday, November 1, 2007, The Coalition for Hemophilia B attended the Celebration of Giving cocktail party hosted by The National Hemophilia Foundation (NHF). Ray Stanhope, Chairman of the Board of NHF presented Wayne Cook and David Clark from The Coalition for Hemophilia B with an award in recognition of our support of the National Hemophilia Foundation in the past year. Areas of support include ongoing scientific research and education.

FACTOR NINE PRODUCTS IN DEVELOPMENT

After being relatively quiet for a few years, there is currently a flurry of activity by several companies in developing new factor IX products and therapies.

Inspiration Biopharmaceuticals filed an Investigational New Drug (IND) application in November 2007 to begin testing a new recombinant factor IX concentrate in patients. An IND is the document describing all of the laboratory and safety testing of a new drug that the FDA uses to determine whether the drug appears safe enough to test in humans. Inspiration plans to test their product for both on-demand and prophylactic use, as well as for use in surgery.

Inspiration has proprietary manufacturing technology that can produce recombinant proteins more efficiently and with higher yields. They have also been doing research to increase the recovery of recombinant factor IX after infusion. Their goal is to be able to produce factor IX and other factor products at much lower cost to make them available to larger numbers of patients all over the world. Lower cost factor IX also makes it possible to explore non-intravenous delivery methods, which potentially require larger amounts of factor IX.

Baxter Healthcare developed the first factor IX product in the U.S. in 1969 but has been largely out of the factor IX market for several years. Now they have announced that they are developing a recombinant factor IX concentrate and also starting research on a longer-acting factor IX. A longer-acting factor IX would require less frequent infusions and potentially make prophylactic treatment easier. One of the methods Baxter is reportedly exploring is PEGylation, attaching polyethylene glycol (PEG) polymer molecules to the factor IX molecule. Attaching PEG to a protein tends to protect the protein from being broken down and cleared from the bloodstream. PEGylation has already been used successfully to increase the half-life of several other protein drugs.

GTC Biotherapeutics is developing a recombinant factor IX made in the milk of transgenic animals. Currently, recombinant products are made by genetically engineered cells that are grown in large tanks. GTC is genetically engineering cells in the mammary glands of pigs so that they produce factor IX in their milk. The milk is collected and processed to purify the factor IX. Because huge amounts of protein can be made this way, it is another source of potentially low-cost and abundant factor IX. GTC previously developed a transgenic recombinant antithrombin, which was licensed in Europe in 2005; the first transgenic product ever approved anywhere. Antithrombin is an anti-clotting protein. People with antithrombin deficiencies clot too easily.

Nastech Pharmaceutical Company is also looking at the possibility of developing a non-intravenous delivery method for factor IX. Very few details are available, but one of their specialties is nasal delivery of drugs. They are working with one of the major factor IX manufacturers, so far unidentified.

THE COALITION FOR HEMOPHILIA B
2ND ANNUAL
NEW YORK SYMPOSIUM
SATURDAY, MARCH 8, 2008

MILLENNIUM BROADWAY HOTEL
145 WEST 44TH STREET
NEW YORK, NEW YORK

THE COALITION FOR HEMOPHILIA B
1ST ANNUAL
FUNDRAISING DINNER
FRIDAY, MARCH 7, 2008

MILLENNUM BROADWAY HOTEL
145 WEST 44TH STREET
NEW YORK, NEW YORK

TOPICS IN HEMOPHILIA

- School In-service – Two parents perspectives

The summer is over and school is here! We thought you would like to read about in-service at schools, two perspectives. If you would like to get in touch with these parents please send us an email and we will be happy to forward it to them.

JOYCE H. - MICHIGAN

Ten years ago my twin boys started pre-school like so many other three-year-olds do, with one very large exception; one of my boys, Eshton, has Severe Hemophilia B with high inhibitor and anaphylaxis to FIX products. Ten years ago, and it remains the same today, the only factor product that he could receive was NovoSeven. Because NovoSeven wasn’t licensed at that point making the occurrence of a bleed extremely life threatening, it took weeks of frustrating phone calls before we could find an appropriate school willing to take Eshton. We made several visits to prepare the staff on how to treat this child with the rare bleeding disorder. I’ve learned a lot since then, but something that hasn’t changed is the need for in-servicing the staff and students when appropriate.Thankfully, by the time Esh was ready for first grade NovoSeven became available on the market and we were able to enroll the boys at our local public school. Our district had just hired a new elementary principal. He had been on the job about three weeks. He arrived at our first IEP (Individualized Education Plan) meeting at the special services department, dressed very professionally: shirt, tie, and suit jacket. Within the first fifteen minutes he had taken off his jacket and loosened his tie. By the end of the meeting he was wiping his brow with visible sweat rings under his arms. A year later, at our next IEP, I asked him about his obvious anxiety, (by then we’d seen each other many times as I traveled to the school to infuse Esh that first year). He said he was so scared when he left that first meeting that he went home and told his wife he wasn’t sure he wanted to keep the job anymore! He said, “I feel sort of foolish now. I’m not sure why I was so overwhelmed by it, because by the time we finished the staff in-service and the year started, I knew we’d be all right. It just required some accommodations.” He may not know why he was so rattled by Esh’s condition, but I believe it was because he lacked information. He feared the unknown. We’ve all experienced that, and if you’ve ever had to live with an inhibitor, you know that fear all too well.

Talking to our principal made me realize the commonality he and I shared. We had both been thrown into an area that neither of us had any prior knowledge of or interest in. We now found ourselves struggling to find out how to best handle the situation we were in. It also made me realize that educators want knowledge. (That may seem obvious, but there are a lot of people we have to deal with every day who really don’t want to know anything.) So I developed an in-service for schools to help the staff with the transition of caring for and educating someone with a bleeding disorder, and especially someone with an inhibitor.

When Esh started public school, I asked him if he wanted me to talk to the kids in his class. He said yes and I arranged with his teacher a time on the first day of school to talk with his class. I explained to the children how Esh’s blood doesn’t work exactly like theirs, but that in pretty much every other way he was just like them. We watched a video of Esh having his port accessed and I showed them the needle we use to poke his port. I also assured them that they couldn’t catch hemophilia; you have to be born with it. The children’s response was wonderful. They were interested and curious. Some of the children actually cried because they felt so bad that he had to be poked like that. Every year we would do this and every year the children would respond the same way.

By the time 5th grade rolled around, I asked Esh the standard question and his response was the same, minus the video. He felt he was too old to show it anymore, so I eliminated it in my “first day of the year talk”. When we got to the question and answer portion that day, Josh, a boy that had been in Esh’s class almost every year, raised his hand and wanted to know where the video was. It had been a part of their education on hemophilia for so long that when it was removed he felt like there was something missing!

This year we moved into our district’s Junior/Senior High School which meant a whole new in-service was necessary for the staff and there would be no “first day talk”. Because speaking with the principal did not bring the desired response, I made an appointment with the District Superintendent and explained to him why it was necessary for his entire staff, including office, cafeteria, janitorial, maintenance, and support staff, to be present at this in-service prior to the first day of school. After pointing out the liability issues the school could face, I gently reminded him that the federal law, as well as our own school board, requires that every child be provided a safe place to learn. He agreed and we scheduled a time for me to in-service the staff during their required Professional Development days prior to the beginning of school.

Thus, “Hemophilia 101” was created. I provided lunch and presented a power point as I spoke. Of course, due to the inhibitor and anaphylaxis issue we have, it took about two and a half hours to present the information and then answer their questions. I’ve learned the most productive way to get the staff involved has been to expose them to the problems that Esh’s transition into their building will bring and ask them to help problem solve the issues with me. The staff feels more empowered having some say in how we can work together not to disrupt the way their classes are run and still provide the accommodations that Esh requires.
The response has been overwhelming. Every person who was there and has seen me since has thanked me for the in-service. They all say the same thing, “We heard he was coming and we weren’t sure what we were supposed to do with him. I feel so much better knowing what’s going on with him and what to do if he needs me. Its’ really not that big of a deal, is it?”

In my humble opinion it all comes back down to knowledge. Truth will eliminate, and illuminate, fear. We as a community have faced fear before many times. We only have to think of Ryan White to know how much ignorance fuels fear and to remember how courage and love triumph. It is my “job” to continue to teach not only the professionals, but also my children, that knowledge is power.

JILL L. - ILLINOIS

As I planned our yearly in-service – a fancy word for educational meeting – with the boys’ teachers and staff I tried to look at things from the teacher’s perspective, a parent’s perspective and my kid’s point of view.My boys’ teachers have anywhere from 20-30 students in their classroom. Of those students, many have learning issues that require the teacher to make educational accommodations to each lesson. One or two may have behavioral issues that the teacher will have to monitor and report home on. There’s probably one with severe food allergies, one with asthma, a couple who wear glasses, one with diabetes, one allergic to bee stings, a couple with personal and family issues that the teacher may need to be documenting, a bully, a few underachievers, a few perfectionists…. I think you get the idea.

My goal is to make the teacher’s life easier, not more complicated. I want her to know that I do not expect her to give my child special treatment. I don’t expect her to diagnose bleeds or guess if my son is faking a bleed to get out of dodge ball. In fact, I would prefer that the teacher have very little to do with my son’s hemophilia. Because of this, I emphasize over and over that the teacher should call me if she/he has questions or concerns.

Now that the boys are older and beginning to manage their own care, I am also insisting that the teachers give the boys responsibilities in managing their care. I encourage the teachers, school nurse, principal, and secretary to have the boys themselves call me. I would like to speak to the boys myself to assess the situation. It sure is easier to talk directly to Nat and ask him “is it a bad bonk or will ice be ok?” This isn’t a question that I want the teacher asking my son. I know my kids; I can read a lot into their answers. There’s a difference between a firm “No, I’m fine” and “well, no, I think I’m ok.” If they seem wishy-washy or uncertain on the phone, I will take that as a sign that they need an infusion. I can also take the time to remind them when they have had their last infusion. If he’s tripped up the stairs, I can say to him, “you had an infusion yesterday morning; do I need to bring your supplies over or can you wait?”

These aren’t conversations that school staff can or should have with my kids. But this is the type of internal dialog they need to have when they have an injury. I’m hoping that by having this dialog WITH them, it will help them better handle the situations when they are on their own.

When we have our in-service, we do a mini-lesson with the school about Hemophilia 101. I let the boys do this now; they are old enough and had better be able to tell someone else what hemophilia is! We explain that any emergency with the boys is to be handled just the same as an emergency with a child who doesn’t have a bleeding disorder: call 911, apply ice, keep them immobile, apply pressure; whatever the emergency calls for, that’s what should be done. Again, I let the boys lead this discussion as I add details along the way. This gives the boys a great sense of pride and ownership and also lets the school staff know that the boys are assertive and in control of their own hemophilia.

The last thing I emphasize is to LISTEN TO MY CHILD. I explain that the boys have very good body awareness. We tell them that if the boys are having a bleed, it might not be noticeable to anyone else. The boys know if they have been injured; if they say that something doesn’t feel right, believe them. I have told school staff that, so far, neither of the boys has “faked” a bleed. I tell them that I know it probably will happen someday, but I don’t what the school to make that decision. If the child really is having a bleed and school thinks they are faking, really dangerous things can happen. On the other hand, if one of the boys FAKES a bleed and my husband and I find out? More really dangerous things will happen – to them when they get home!

Our approach with the school has always been to be open and honest. As the boys have advanced through each grade, we give them more responsibility. Just as we want the boys to manage their hemophilia here at home by doing their own infusions, we want them to transition to managing their hemophilia while they are away from home.

8 March 2008 @ Broadway Millennium Hotel - New York City

Below are the Registration Information/Form and the Agenda for the event.

Registration Information and Form

Symposium Agenda

7 March 2008 @ Broadway Millennium Hotel - New York City

Below are the Dinner Invitation and the Reservation Card for the event.

Dinner Invitation

Reservation Card

Lower-cost manufacturing of the drug may have worldwide impact. “Our cost-effective manufacturing platform for factor IX positions Inspiration to expand the global supply of i.v. rFIX and to develop next generation products to facilitate prophylactic care of the hemophilia B patients worldwide,” said Michael Griffith, PhD, President and CEO of Inspiration Biopharmaceuticals. “Our ultimate goal is to leverage our low-cost manufacturing technology to find the means for noninvasive delivery of factor IX, which we believe may represent the equivalent of a therapeutic cure for patients suffering from this disease.”

Inspiration plans to follow successful PK studies with multiple clinical trials to measure the efficacy and safety of on-demand treatment, prophylactic therapy and surgery.

In a related development, Cook Pharmica, the contract biopharmaceutical manufacturing and development services division of Cook Medical in Indiana, announced that it signed an agreement with Inspiration to conduct development work for IB1001. Its Bloomington-based development laboratory will eventually manufacture the product.

“We are pleased to be working with Cook Pharmica,” added Griffith. “We look forward to our partnership as we prepare for large-scale manufacturing of our factor IX product for the treatment of hemophilia B.”

Source: Inspiration Biopharmaceuticals news release dated November 13, 2007 and The News & Observer, November 19, 2007

-CHB New York Symposium
-Dr. William N. Drohan
-FundFusion, Arizona
-Spintacular Walkathon, NY (upcoming! 8/25)
-HFA Conference, New Mexico - Industry News
-Factor Nine Family Meeting, Indianapolis,
Indiana (upcoming! 8/25)

The Coalition for Hemophilia B

(New York Symposium)

Wayne Cook, John R. Taylor and David Clark

On Saturday, March 31st, The Coalition for Hemophilia B held a Symposium at the Millennium Broadway Hotel in New York City. The day was filled with educational speakers and special awards. We began at 9:30 am with opening remarks from Dr. David Clark, Chairman of The Coalition for Hemophilia B. Dave welcomed everyone and presented the William N. Drohan PhD scholarship plaque to the Drohan family. Sadly, Bill passed away several days earlier. Bill’s son Craig accepted the plaque on behalf of his family.

Mario “the Magician” and Tyler Blair

Wayne Cook presented an award to John Taylor for his many years of dedication and service to the hemophilia B community. John & Joyce Taylor founded the Coalition in the early 1990’s. Bill Drohan was one of the Coalition’s first boardmembers. Their dedication and perseverance has made the Coalition what it is today and given so many people with hemophilia B a better quality of life through research, education and support.

John Taylor spoke of the many trials and tribulations the Coalition has endured and of the future. He talked about his relationship with his dear friend Bill Drohan and how he will miss him.
When John finished we had a moment of silence and played Bill’s favorite song “Sunrise, Sunset”.

Our first educational speaker was Dr. Zale Bernstein from the State University of New York at Buffalo School of Medicine. Dr. Bernstein had the audience gripped with his knowledge and wit.
He spoke about all aspects of treating hemophilia
from prophylaxis to how to become a good
advocate in the Emergency Room.

Families gathered to board the Circle Line boat tour to see the Statue of Liberty

Our next speaker was Michael O’Connor, a 16 year old with hemophilia B from Cold Springs Harbor High School in Long Island. Michael is on the varsity swimming team. He talked about youth athletics, how to stay in shape and how he infuses
in order to be a competitive swimmer. Michael fielded many questions from parents of children with hemophilia. We ended the morning session with Dana Kuhn from PSI (Patient Services Inc.), a non-profit organization that helps individuals with bleeding disorders, with insurance issues. Dana spoke about the many issues that are plaguing the hemophilia community, from sole source providers to lifetime caps and what to do when you reach your limit.

After lunch, the afternoon program was held by Joe Caronna, founder of Inalex Communications, a non-profit organization dedicated to serving the needs of those with a bleeding disorder. The speaker for the session was Ron Potter-Efron PhD from Wisconsin. Dr. Potter-Efron spoke of issues ranging from finding a space for anger to advice for parents who worry too much. He motivated people to look at what lessons we can learn by
developing a positive attitude.

Wayne Cook closed the Symposium by thanking everyone for coming. We went overtime as usual and afterward many people continued talking in the hotel lobby. The children had a good time in our babysitting room filled with games, crafts and videos. Mario “the Magician” did a wonderful job entertaining them. Our goal is to have another symposium next March. We will keep you informed of the future date. We thank all our volunteers, speakers and sponsors of this event and hope to see many of our members attend the next one!

Dr. William N. Drohan

Bill and youngest daughter, Laura at Redskins Game

I am sure many of you in the hemophilia community have had the pleasure of meeting Bill Drohan over the years. If you did, you would not forget him, he was one of the nicest people I have ever known. On March 25th, Bill lost his battle with metastic lung cancer. He was 60 years old.

Bill was a well-known microbiologist and educator who will be remembered for his many contributions to science. He was a pioneer in using molecular biology to produce recombinant proteins and a visionary scientist who dedicated his life to improving the safety of blood and blood products. Other important contributions include investigating mad cow disease in the blood supply, and his development of novel ways to treat traumatic injuries including bandages to stem hemorrhage. Bill had a great sense of humor and carried that into his work. I remember seeing a scientific slide show presentation on transgenic proteins and Bill was in the slideshow dressed in a pig costume!

Bill was equally dedicated to scientific research and to mentoring students and young scientists.
His career included important positions with the National Cancer Institute, The American Red Cross, and private companies dedicated to treating bloodborne disorders, most recently as Chief Scientific Officer of STB, Ltd., as well as Chief Scientific Officer at Inspiration Biopharmaceuticals, Inc. and previously Chief Scientific Officer of Clearant Inc. He was also a very involved Board Member of The Coalition for Hemophilia B, his passion; drive and vision were instrumental in the formation of the Coalition when it first began in the early 1990’s. Bill served as Professor in the Graduate Program of the Department of Genetics of George Washington University and formerly as Adjunct Professor in the Department of Chemical and Biochemical Engineering of the University of Maryland. Dr. Drohan published more than 145 scientific papers and holds 30 U.S. patents. He served on the editorial boards of several scientific journals, and was a member of the Scientific Steering Committee for Blood Products at the Walter Reed Army Institute of Research and the Chairman for the Panel on Biotechnology of the National Research Council.

Bill’s professional and personal enthusiasm was always a great motivation for many fortunate to have worked with him. He will be remembered by many younger scientists for his generous mentoring role. In addition to his passion for science and business, Bill was an avid sports fan whose next-day summaries of the Virginia Tech Hokies’ games or Washington Redskins football games were eagerly anticipated by all. Bill asked that his friends, family and professional colleagues celebrate the life that was whenever they think of his passing, rather than grieve his loss. He leaves to all of us a legacy of scientific achievement and inspirational leadership that was accompanied by
an extraordinary level of kindness and generosity.

John Taylor, Kim Phelan and Bill Drohan, exhibiting at annual NHF Conference in 1996

On March 31st, Bill’s wife Marian, and their 5 children, Colleen, Maureen, Kathleen, Laura and Craig attended the Coalition for Hemophilia B Symposium in New York where we presented the family with a plaque that establishes a scholarship fund in Bill’s name. The scholarship fund is for children of scientists, working in the hemophilia area, who have a need for funds, especially those who have lost their father or mother. Anyone who wishes to make a contribution can send a check payable to: The Dr. William N. Drohan Scholarship Fund and mail to: The Coalition for Hemophilia B, 825 Third Avenue, Suite 226, New York, NY 10022, Attn: Kim Phelan

When somebody dies, a cloud turns into an angel
and flies up to tell God to put another flower
on a pillow. A bird givse the message back to
the world and sings a silent prayer that makes
the rain cry…

People disappear, but they never really go away.
The spirits up there put the sun to bed, wake up
the grass, and spin the earth in dizzy circles.
Sometimes you can see them dancing in a cloud
during the daytime when they’re supposed to be
sleeping.

They paint the rainbows and also the sunsets
and make waves splash and tug at the tide.
They toss shooting stars and listen to wishes.
And when they sing windsongs, they whisper to
us, “Don’t miss me too much. The view is nice,
and I’m doing just fine.”

FUNDFUSION

Tempe, Arizona, February 18-20, 2007

We received a generous invitation from the Hemophilia Association of Arizona to attend a FUNDFUSION event at the Mission Palms Hotel. Some 100 people gathered from over 35 chapters and various organizations within the hemophilia community. The purpose of the conference was to educate chapters and organizations on fundraising strategies from academic and fundraising professionals, to network and promote ongoing
dialog among chapters and organizations.

The keynote speaker was an extraordinary woman named Carol Weissman, President of Boardbuilders.com. Carol also served on the board of NHF in the 1970’s and has served on over 29 boards since then. She can captivate an audience with her wit and humor. Carol makes learning fun. David Sternberg, another speaker from the Center of Philanthropy at Indiana University made us feel as if all we thought we knew was wrong, so we basically had to empty our teacup to allow new ideas to flow in. He forced us to look at new ways of seeing our missions and to revive our statement purpose. The Ethics session by Bill Harrison of Southwest Fundraising Institute, had us each form groups where we had to take a real life case scenario and come to a conclusion as to how we would handle it. Each group was faced with a certain dilemma. This was a wonderful energetic time for everyone and we all worked together with excitement. We were given a certain amount of time and then one person from each group would speak on the outcome that was decided. Later Bill discussed how it turned out in real life.

“This was a wonderful energetic time for everyone and we all worked together with excitement.”

The conference was filled with a wealth of invaluable information needed for the success of any organization. We saw what organizations are doing on an international basis. We learned about bequests, that is when an individual leaves the organization of their choice in their will so when they pass on, the organization receives monies. The discussion of members and donors came up and Carol made the comment that if you are a board member, you should contribute to the organization which you belong. She told us a story about a time when she was married with a young child and they were on a budget. She wanted to make a donation to her organization so she and her husband spent one month eating in, not going to the movies, no babysitter and at the end of the month they were able to make a donation of $250.00. That is what she would call a sacrificial donation.

Many other topics were covered by a number of speakers ranging from internet fundraising, specials events, annual campaigns using direct mail and telemarketing, making a case for support and so much more.

We learned much and I am sure we will all delve into the booklets we were given quite often.
Special thanks to Cindy Komar of the Hemophilia Association of Arizona and her team. I am sure we all look forward to making wonderful strides!

Adirondack Spintacular

Saturday, August 25th, 2007

The 6th annual “Adirondack Spintacular will be held at 10:00 am in Mayfield, New York. Rain or shine you can run, cycle or walk for hemophilia and to raise organ donor awareness.

The Spintacular is a community based volunteer driven 5K, 1, 6 or 12 mile cycle, run or walk that is family oriented, promotes physical fitness, recreation and a healthy way to spend time.
After the ride there is a BBQ, Silent Auction, Children’s activities and Bounce House. Awards are presented to the top overall finishers in Running, Cycling and Walking (not the 1 mile) events and, to the top 2 male and female winners in each event by age divisions.

Historically, over 400 people, ranging in age from 1-88 years young have participated in the Spintacular. Many people from the surrounding areas attend as well as persons from outside the New York state.

Proceeds from this event benefit the Lawrence Madeiros Scholarship founded in 2001, in memory of Larry Madeiros, who had severe hemophilia B, HIV and Hepatitis C. In 2001, at the age of 38, Larry passed away awaiting a liver transplant.
He was a passionate, positive, caring, loving husband, father and friend. He touched and inspired many people’s lives and always asked “What is your passion?” Larry firmly believed that every child should have the opportunity to further his/her education, and overcome any illness, disorder or adversity and realize their passions, goals and dreams. Scholarships are awarded to the inspiring young people who live daily with tremendous chronic disorders and still excel scholastically and contribute to their communities. For more information on how to attend or contribute please contact Lisa or Carol
at (518) 661-6005, website:
www.adirondackspintacular.com

Cory is one of the 2006 scholarship winners!

Carol Madeiros and Cory Kruger

2007 Lawrence Madeiros Scholarship Winners

Thomas J. Kim, Milpitas, CA and will attend UC Berkeley in the fall, majoring in Pre-Med.

Michael T. Mayers, Crystal Lake, IL, will attend the University of Iowa to pursue a degree in Business Administration

Lindsay N. Norville, Albany, NY attending Emerson College in Boston, Mass. Majoring in Writing and Publishing.

Derick J. Stace-Naughton, Madison, WI will attend Georgetown Univ. and study for degrees in Psychology and Physics.

Hannah L. Taylor, Charlotte, NC plans to attend University of North Carolina, Chapel Hill study in Journalism and Int’l relations

Sarah A. Woodcock, Johnstown, NU will attend Fulton Montgomery Community College, working toward a degree in Nursing.

Congratulations to you all!

HFA Conference

The Hemophilia Federation of America’s (HFA) 2007 Educational Symposium took place in Albuquerque, New Mexico March 1-4th. Over 600 people attended. The weekend was filled with great speakers and presentations. Adults heard about the most current issues affecting the bleeding disorders community from leading medical, government, advocacy and industry experts. Children from infants to 18 made new friends in their own programs. Teens and preteens visited Albuquerque museums while the younger children learned Native American dances.

We received the news of Jan Hamilton’s upcoming retirement in November. The HFA gang put on a funny skit for her and played videos of her family.
They shared wonderful stories of past travels with Jan. At the end Jan was given a lovely Pink riding helmet. Upcoming issues will share more about
Jan. We will all miss her but I am sure many have her home phone number!

To close out the weekend, everyone attended an authentic barn dance/southwestern barbeque with entertainment by Native American and Hispanic dancers in costume. New and old friends of all ages left New Mexico armed with new information and fond memories.

The Coalition for Hemophilia B held a Factor Nine Family breakfast meeting there as well.
We had a wonderful turnout and were delighted to meet so many new families. We realize the
importance of support and how much more empowered new families become when they
meet our members. Many old and new members gathered later on that evening to continue earlier discussions, exchange phone numbers, and form friendships.

Industry News

Wyeth Parmaceuticals has received FDA approval for the new convenience enhancements for BeneFIX® A 2000 IU vial, a needle-less reconstitution device, a prefilled diluent syringe and reduced diluent volume for the 1000 IU dosage strength.

The new BeneFIX features allow patients to use a lower volume of diluent to administer the product for the most commonly used dosage size (1000 IU), leading to a lower total volume of infused product.

The new needle-less preparation process will eliminate the risk of needlesticks during reconstitution.

Wyeth began shipping BeneFIX with new convenience enhancements to hemophilia treatment centers, home healthcare companies and hospitals beginning July 9, 2007.

It is recommended that you use your current supply of original BeneFIX before beginning to use BeneFIX with the new R2 Kit. Also, do not mix original BeneFIX and BeneFIX with new enhancements because the diluents are different. BeneFIX should only reconstituted with the diluent provided with the dosage vial.

For more information you can call the Wyeth Hemophilia Hotline at 1-888-999-2349.

Grifols: As part of Grifols safety program each vial of Grifols coagulation products is laser inscribed with the lot number identifying it as an authentic Grifols plasma derivative, to help deter tampering. The laser etched lot number into the glass is permanent and legible even if the label is missing, allowing the traceability of each vial.

Grifols also offers a PatientCare Program which is a patient assistance program designed to help provide eligible patients with access to their coagulation products, specifically, Alphanate®, Alphanine® SD, and Profilinine® SD. The PatientCare program consists of two distinct parts:

Grifols Assurance for Patients or GAP Program
Available to patients in the event they experience temporary lapse in insurance coverage. Eligible patients have been treated with Grifols coagulation product for three consecutive months prior to lapse
in coverage through non-government (State or Federal) insurance plan. Applicants must have
US Citizenship or legal resident status.

Grifols Patient Assistance or GPA Program
Available to patients without insurance coverage
and in need of temporary assistance obtaining
Hemophilia clotting factor. Applicants much not
be eligible for State or Federal funding health
plans. You must have US Citizenship or legal
resident status and meet financial eligibility
criteria of 250% of the Federal poverty level.

Patients are encouraged to coordinate enrollment through their factor provider. Enrollment forms for both assistance programs can be downloaded from www.grifolspatientcare.com or by calling 888-Grifols (press option 3).

Grifols Sample Program
Grifols AlphaNine® SD sample program is available for patients who are not currently using this product and/or have not sampled it in the past. Available in vial sizes 500 IU, 1,000 IU or 1500 IU with 10 ml diluent.

You can obtain the sample program through your factor provider or by calling 888-325-8579.

NHF 59th Annual Meeting Florida
Grifols will be sponsoring a breakfast symposium on Factor IX Pharmacokinetics: difference between plasma derived vs. recombinant and their clinical implications. Key speakers: Dr. Cindy Leissinger, Dr. Larry Logan, Bobby, Wiseman, Becky Berkowitz, RN.
Syntonix Pharmaceuticals, a recently acquired subsidiary of Biogen Idec, is pursuing the development of two products that could offer exciting new treatment options for patients with hemophilia. The first, FIXFc, is a long-acting recombinant factor IX product to treat hemophilia B. The product is being developed jointly with Biovitrum of Sweden. The companies intend to file an investigational new drug (IND) application with the FDA by the end 2007. The second product is a long acting recombinant factor VIII for hemophilia A, FVIIIFc, which is in the early stages of development.

Both FIXFc and FVIIIFc utilize the company’s proprietary Fc fusion technology. The products consist of factor molecule fused to the Fc portion of an antibody. Antibodies are naturally produced by the body to help fend off infection. The Fc region is the part of the antibody that allows antibodies to remain in the bloodstream for long periods of time. The FVIII and FIX fusion proteins are able to harness the body’s natural pathway for protecting antibodies against premature destruction and may optimize the properties of the drug by extending the amount of time that the Factor is effective in the body. This could allow for Factor to be injected less frequently.

CSL Behring, change of name from ZLB Behring to CSL Behring. The renaming program, announced by CSL Behring’s parent organization, CSL Ltd., will present a consistent and compelling view of the company’s operating in new and established markets around the world. “CSL has a tradition of innovation with important vaccines and medicines that began 90 years ago and continues to grow,” Said Peter Turner, President of CSL Behring.

“The Behring part of our name originate with Emil von Behring, an innovator with serum therapies won the first noble prize in physiology and medicine. We have changed our name but remain strongly committed to the value of innovation, safety and passion for our customers that we are known for.”

SAVE THE DATE

On Saturday, August 25 at the Hemophilia Foundation of Indianapolis Conference, The Coalition for Hemophilia B will hold a Factor Nine Family Meeting from 2:00 – 3:00 pm, Monaco Room B, located on the second floor of the Holiday Inn Select North Hotel, 3850 DePauw Blvd. Indianapolis Indiana. We look forward to seeing our members from Indiana!

SPECIAL EDITION

-A Letter from John R. Taylor, Jr.
-Industry Update
-Coalition 2006 Meeting Pictures
- Future Meeting Information

A Letter from John R. Taylor, Jr.

Dear all members,

It is with a mixture of sadness and elation that I write this letter to you all. My wife Joyce and I had the idea to start the Coalition several months after our son John was born. We wanted to provide information and support for parents like us, men, women and children with hemophilia and their families. Although filled with much anxiety, we realized the more we learned, the stronger we became to deal with the problems we faced. We set out to empower others so they would not have to deal with the same issues we faced, alone. Thus the birth of The Coalition for Hemophilia B in 1990.

While serving as Chairman for the past 16 years, we have seen many changes in the treatment and the lives of those who have been able to enjoy the progress that science has made. First with the introduction of Alphanine, which was the first product just for us, and then Mononine, the first monoclonal factor, we began to get the feeling of what life could be. The Coalition was very happy to have played a part in the FDA approval of Mononine, but we pushed hard for BeneFIX as soon as Mononine hit the market. When BeneFIX was approved in 1996, we all thought this was great, but then immediately began wondering how we could make things better again. Everyone involved in the Coalition has always desired to go the next mile, take the next step up the ladder, and eventually reach some unknown goal.

With that in mind, the Coalition has pushed prophylaxis before it was common and before MASAC said that this was the approved treatment. But we worried about the cost of this treatment. This pushed us into research, and the Coalition championed transgenic research to make factor much cheaper. It also spent money to support the search for alternate delivery methods – pills, nasal sprays, skin patches and anything else that would eliminate the dreaded i.v. needle.

Anyone who has spoken with me knows that I think that more should be done, and after many years of talking about improving products and delivering them by something better than i.v., I have taken a big step. I have started a company, Inspiration Biopharmaceuticals, Inc., with a partner and fellow Coalition member, Scott Martin from Houston, Texas. Inspiration’s first product will be a recombinant Factor IX and we hope the second product will be an orally delivered Factor IX. Our goal is to revolutionize the treatment of hemophilia, and it will take a lot of work to reach that goal – but we are on our way.

Because of my new role, I can no longer be associated with the Coalition. Upon my resignation I have passed the baton to two individuals who express the same desires and hopes that the Coalition values so highly: Dr. David Clark as Chairman. Dave has been writing the scientific end of our newsletter for many years.
He has a strong interest in hemophilia B. And Wayne Cook, who has hemophilia B and has been a long-term advocate for the cause. The Coalition has moved its offices and is now located at 825 Third Avenue here in Manhattan.

All of the Coalition’s functions – advocating for new research, serving as a resource for patients and their families, and spreading new treatment ideas – will remain as they were.

This is a very exciting time for our community as the efforts of the Coalition have led not only to my new company Inspiration, but also to new product developments at Wyeth and a new entrant in our market – Biogen. Whatever the outcome, I am sure that our community will be better served in the years ahead – and that’s the whole idea.

My experience with the Coalition has been one of the highlights of my life. It certainly helped us. It has been a pleasure serving you all.

Warm regards,

John R. Taylor, Jr.

Industry Update

Grifols’ Alphanate approved by FDA

Grifols, a plasma fractionation company in Los Angeles, produces Alphanate, a plasma-derived factor VIII clotting factor concentrate that also contains significant amounts of von Willebrand factor (vWF) protein. Physicians know this and have used Alphanate off-label for years to treat Von Willebrand disease (vWD). However, Grifols has recently completed a clinical study of 76 patients, including children that allowed the FDA to formally license Alphanate for treatment of vWD. vWD is a disease that has a lot of similarities to hemophilia A and B. vWF is a protein that protects factor VIII and carries it around in the bloodstream. FDA approval of an indication for treatment of vWD allows Grifols to market Alphanate for treatment of vWD, and it is sometimes important in getting insurance companies to pay for the treatment. An “indication” is an approved use for a drug. Therefore, this potentially is a significant development for vWD patients. ~

Biogen Idec has announced that it is acquiring Syntonix Pharmaceuticals, a biotechnology company based in Waltham, Massachusetts. Syntonix has been developing a long-acting Factor IX product for the treatment of Hemophilia B. Their SynFusion technology links a factor IX molecule to an antibody molecule. Antibodies last longer in the bloodstream than may other proteins, so this helps the factor IX stay in the bloodstream longer. Antibodies also can move across cell barriers such as those in the lungs. Thus the new product may be able to be delivered by inhalation rather than injection.

Wyeth Pharmaceuticals and Nautilus Biotech, a French company, have agreed to work together to develop a recombinant factor IX that has a longer half-life. The half-life is the amount of time it takes for half of the factor IX to disappear from the bloodstream. The longer the half-life, the less frequently an infusion is needed. This is especially important for prophylactic treatment where it could lengthen the time between infusions.

Nautilus has proprietary technology that can make changes in the amino acid sequence of a recombinant protein like factor IX. Amino acids are small molecules that are strung together into long chains to create proteins. There are 21 common amino acids that the human body uses to make all of its proteins. The type and order in which the amino acids are strung together determine which protein is produced.

Small changes in the type of amino acid at a specific place in the protein molecule, which might not significantly affect the activity of the protein, can make a big difference in how long a protein lasts in the bloodstream.

Wyeth and Nautilus will look at various amino acid changes to see if a recombinant factor IX molecule can be produced that has a longer half-life without sacrificing activity.
Wyeth has also signed a research agreement to work with MediVas, a biotechnology company in San Diego to develop improved treatments for hemophilia. Although the exact nature of the research was not announced, MediVas has developed unique drug delivery systems for recombinant proteins that can improve patient convenience by giving the protein a longer half-life. MediVas technology can potentially be used for oral, inhaled or subcutaneous injection. (injection with a short needle directly under the skin). This can make the administration of the recombinant protein more convenient, more effective and safer, potentially leading to higher patient compliance and better overall health.

The news from Wyeth and Biogen is very dramatic and it is a very positive change for those of us who want Factor IX products to get better and better. Two cheers for these companies, we wish them success. Although starting a new research project to develop new kinds of factor is
a long way from that factor being available, it is
a very important first step. We are very glad that they have chosen to take that step. Hopefully, some time in the future we will have long lasting Factor IX, from either Biogen or Wyeth, and we will have orally delivered Factor IX from either Wyeth or Inspiration. We are keeping our fingers crossed.~

Highlight pictures from a gathering and our Breakfast meeting at the NHF, October 11, 2006

Many Factor Nine families gathered at an evening event held at the Franklin Institute

Our Factor Nine family meeting on Saturday morning had a very good turnout!

Please join us at the HFA in New Mexico for a breakfast meeting on Saturday, March 3, 2007

And our New York Symposium on Saturday, March 31, 2007 which is posted on our website!

-Factor Nine Family Meeting, October 14, 2006
-HFA, WFH, Binghamton Chapter Meetings
-FIX Replacement Therapy
-Prophylaxsis Update
-Grifols Donates 1.4 Million in Clotting Therapies

Your Invited!

Factor Nine Family Breakfast Meeting

In conjunction with the NHF Annual Meeting

******************************

Saturday, October 14, 2006
8:30 – 9:30 am
Marriott Courtyard Hotel
(located across the street from the Downtown Marriott)
Philadelphia, PA
Salon Room 3
(level 1 right off elevators)

A great place for people with factor IX and their families to get together, gain support,
share stories and make friends.

We learn from each other. New families can gain valuable knowledge from those who have been through the ropes.

Knowledge ~ Resources ~ Peer Support

Empower Yourself and your loved ones!

Hemophilia Federation of America Conference

March 31 – April 2, 2006

St. Louis, MOHFA’s annual meeting was a great success this year. Enthusiasm and optimism were very high. Speakers presentations were better attended than ever before. Of particular interest was the Reimbursement Issues Panel facilitated by Jan
Hamilton, HFA Advocacy Director. Speakers
were Patrick Collins, Sr. Manager, Public Affairs, ZLB Behring, Dana Kuhn, PhD, President, Patient Services Inc. (PSI), and Kim Bernstein, Esq, Director, ACCESS Program, Hemophilia Health Services, (HHS). Patrick discussed Medicare and Medicaid Issues, Dana’s topic was on Insurance Trends and Kim informed the audience
about Social Security Issues that affect many in the community. HFA has many organizations working together to keep you updated. If you have any questions, please call Kim Phelan at (212) 554-6898, hemob@ix.netcom.comOur meeting had a good turnout with people from all ages and places! We heard interesting stories about experiences with hemophilia. The group shared their views with each other on treatment options. Friends were made, phone numbers exchanged. The bridge of support keeps growing.

Bleeding Disorders of the Southern Tier (BDAST) Conference, Binghamton, NY

April 29, 2006

A good turn out at this conference. Speakers included John Williams, Hemophilia Health Services, Jill Williams, Critical Care Systems, Paul Brayshaw, Rep, HFA, Tom Vaclavik, HFA, Meredith Zerbe, Baxter, John Davis and Wayne Cook as well as representatives from Wyeth, Bayer, Baxter and Grifols. The sessions were Dads in Action, Adult Men’s program. Factor information from the manufacturers, Insurance and Legislation updates. Wives of men with bleeding disorders and Woman with bleeding disorders. On Sunday, a movie was shown “The Unexpected Life”. Also a session on how to smile and have a positive outlook on life. For Saturday Nights event, the chapter held a Hawaiin Luau in the pool area of the hotel.

World Federation of Hemophilia

Congress May 21-25, 2006

The meeting was held in the spectacular waterfront setting of the Vancouver Exhibition. Ceremony
audience was treated to a colourful performance by a 30 member pipe band and a first Nations drum group.Sessions were well attended and topics ranged from the latest developments in areas ranging from inhibitors, prophylaxis, to pain management, quality of life, research and family issues. Other sessions also focused on bleeding disorders such as von Willebrands, as well as the challenges for patients in countries with limited economic resources.

Close to 4,000 people from more than 100 countries gathered in Vancouver, CanadaThe record number of participants included people with hemophilia, medical professionals, national hemophilia organizations, industry and regulators.

Wayne Cook and Helmut Gast, Germany Representatives for The Coalition for Hemophilia B included Kim Phelan, Wayne and Maureen Cook. We truly enjoyed meeting people all over the world and to finally meet in person, people we have been in correspondence with for years. We had many requests to hold educational workshops worldwide. It was also nice to see people from the community back home in the USA.

Wayne and Maureen Cook Exhibitors showcased the latest hemophilia treatment products, services and publications. Hemophilia associations from various countries held booths at the WFH. A poster display featured numerous contributions from individuals and organizations.~

Factor IX Replacement in Surgery

“A pharmacokinetic study tells the physician how much factor IX shows up in the bloodstream (the recovery) and how long it lasts (the half-life

Surgery presents specials issues for hemophilia treatment. Before the advent of modern Factor IX Concentrates, only the most necessary surgical procedures were performed on hemophilia B patients. Elective surgery was rarely considered. For instance, older hemophilia B patients with severe joint damage often had to just live with the pain and disability because joint replacement surgery was too risky. Prior to the introduction of more highly purified factor IX Concentrates in the late 1980s, patients were treated with Factor IX Complex Concentrates, which have the dangerous side effect of inducing thrombosis, unwanted clotting. When used in large doses patients would have successful surgeries, without excessive bleeding, only to end up with serious, even fatal thrombotic complications during their recovery.

It is still not certain why Factor IX Complex cause thrombosis, but todays more highly purified factor IX concentrates seem to be largely free of this side effect. Now hemophilia B patients can even consider elective (non-essential) surgery. An article from Northwestern University discusses a number of issues involved in factor IX replacement, to control bleeding during surgery.

Even though a blood factor level of one to two percent of normal is usually enough to prevent bleeding in everyday life, that is far too little for surgery. Patients are usually brought up to blood levels of 80-100% of normal for surgery. This takes a lot more factor concentrate and more precise dosing. The Northwestern article (Factor IX Replacement in Surgery & Prophylaxis by Anaadriana Zakariji) highly recommends performing a pharmacokinetic study on a patient before surgery, while he is not bleeding.A pharmacokinetic study tells the physician how much of the factor IX infused shows up in the patient’s bloodstream (the recovery) and how long it lasts (the half-life). This helps the physician determine the correct dosage of factor IX to prescribe.

In the past, pharmacokinetic studies were done less frequently and physicians often used general guide-lines in seting the dose for the “average” patient.
However, it is now known that few patients are actually average. Another study of 43 hemophilia B patients found recoveries of factor IX after infusion ranging from 57% to 143% of the average recovery. Looking at the individual patient data, there was not a large group of patients clustered around the average recovery with a few extreme cases. The patients’ recoveries were spread over the whole range, so most of the patients, in fact, were not average. Just going by an average guideline could result in a patient with a low recovery getting too little factor IX to prevent bleeding, while a patient with a high recovery would receive too much, a waste of the expensive product.

Patients will usually stay with the factor IX product they already use successfully for prophylaxis or routine treatment, whether plasma-derived or recombinant. It is very important to do the pharmacokinetic study with the product that will be used for surgery because patients can respond quite differently to different products.

It is important to continue to maintain adequate factor IX levels to minimize bleeding after surgery, while the patient heals. Excessive bleeding internally at the surgical site can interfere with proper healing and affect the long-term outcome of the procedure. For instance, with a total joint replacement, excessive bleeding could cause the body to try to reject the implanted artificial joint. This sometimes happens by the body growing a tough fibrous material around the implant to try to wall it off from the neighboring tissue. The fibrous material can interfere with the proper functioning of the artificial joint. In other types of surgery, excessive post-surgical bleeding can cause excessive scar tissue to form, which could interfere with proper functioning in what-ever part of the body is involved. None of this should discourage the hemophilia B patient from considering surgery when needed. All of these issues have relatively simple solutions today. Physicians and surgeons have performed a large number of surgeries on hemophilia patients with success in the vast majority of cases. The patient and his family should discuss the risks and benefits for his individual case with his physician in order to make an informed, confident decision about surgery.~

Prophylaxis Update

Dr. David Clark

Prophylaxis or prophylactic treatment is considered to be the best method of treatment for hemophilia B. Prophylaxis, when started at an early age, is the only known treatment that can prevent the joint deterioration that is a major consequence of hemophilia and the major hindrance to quality of life for the hemophilia patient. Prophylaxis is regular administration of clotting factor, often two or three times a week, with the aim of keeping the blood level of factor IX high enough to prevent bleeding into the joints.However, even though prophylaxis is considered to be the best treatment, the best method for performing prophylaxis is still in question. Physicians and researchers are still trying to find the optimum dose and frequency of infusion to give the best results. A recent study from the hemophilia treatment center in Malmo, Sweden, one of the pioneers in developing prophylactic treatment, now shows that there may be no single method that works for all patients. The best results are probably obtained by tailoring the dose and frequency to each patient based on their individual results.

The Malmo group followed a large number of Hemophilia A and B patients on prophylaxis for six years and found some interesting results. They looked at how various treatment regimens (dose and frequency of infusion) affected factor levels in the blood and how the factor level in the blood affected the incidence of bleeding. They looked at the effects of age and level of existing joint damage. One goal was to determine whether the amount of existing joint damage affected the factor level needed to prevent new joint bleeds and further damage.

The study of the distribution of any drug in the body is called pharmacokinetics. (“Pharmaco”) refers to drugs, as in “pharmaceuticals”, and “kinetics” is movement, so pharmacokinetics is the study of the movement of drugs throughout the body. When factor IX is injected into a vein, it quickly distributes itself throughout the bloodstream. If you were measuring the amount of factor IX in the blood, you would see an immediate sharp increase in the factor IX level. Then the factor IX level starts to fall off. Some of it is used up in clotting and some of it naturally degrades and is removed by the body. When the next infusion is given, the level jumps up again and then falls off, repeating the process. In prophylaxis, the factor IX level over time looks like a sawtooth pattern with peaks and valleys; the lowest point, the valleys, are called the
trough levels. The main goal in prophylaxis is to keep the trough levels high enough to minimize bleeding into the joints and joint damage. Besides the trough level, the two main pharaco-kinetic parameters are the recovery and half-life. For factor IX, the recovery is the amount of factor IX that can be found in the blood immediately
after infusion. Interestingly, the average recovery for factor IX is only 50%, that is, only about half of the factor IX infused shows up in the blood. The rest of the factor IX immediately goes to other parts of the body. The half-life is a measure of how long the factor IX stays in the bloodstream; it is the amount of time it takes for half of the factor IX to disappear (not including the half that never shows up in the first place because of the 50% recovery). Note that physicians also use a number of other parameters as indicators of pharmacokinetics, but all of those parameters are related to the recovery and half-life.

This study reinforced the findings of other studies that the pharmacokinetics of clotting factors varies widely from patient to patient. Therefore, it is best to tailor the dose and frequency to the individual patient. Although it is expensive and time-consuming to do a pharmacokinetic study on each patient, it is probably worthwhile in the long run.
It can even pay for itself if it is found, for instance, that a patient has a higher recovery or longer half-life. Then that patient might need lower or less frequent doses and thus use less factor concentrate. Conversely, a patient with a lower recovery or shorter half-life might need larger or more frequent doses to be treated effectively.

It has been known for a long time that patients with moderate hemophilia, who have more than 1/% of normal factor levels in their blood, bleed only infrequently and rarely experience severe joint damage. Therefore, one of the main ideas behind prophylaxis has been that if you change severe hemophilia into moderate hemophilia by keeping the trough levels above 1%, you will generally prevent joint damage. The most interesting findings of the Malmo study was that the amount of factor IX in the bloodstream is not strongly related to the reduction in joint bleeding.
The study found patients who experienced no bleeding problems even when their factor levels were below 1% and others who still experienced bleeds even with levels above 3%. They also did not find any relationship between the amount of existing joint damage and the amount of factor IX needed to prevent new bleeds. Therefore, not only does the pharmacokinetics vary from patient to patient, but each patient may require a different trough level for optimum effectiveness of the treatment.

The bottom line is that prophylactic dosing should be tailored to the individual and should be based on clinical outcome, that is, actual reduction in bleeding, not just on measured blood levels of factor IX. Rather than the ideal of having a single dosage that works for everyone, the patient and physician need to work together to determine the patient’s pharmacokinetic profile and his required trough level. That requires more work up front, but should give better results over the long run. ~

Grifols Donates 1.4 Million

In Hemophilia Blood Clotting Therapies to World Federation of Hemophilia

As part of its ongoing commitment to the global disorders community, Grifols has donated plasma-derived blood clotting therapies to be used to treat patients in developing countries. The donated products include those used to treat both hemophilia A and B and will be targeted for patients in developing countries where access to adequate treatment is often lacking. It is anticipated that the donation of hemophilia therapies will be used to treat hundreds of patients. The WFH will direct the distribution of the donated products to areas of greatest need.

An estimated 400,000 people around the world have hemophilia, yet, only 25% received adequate
treatment. According to WFH President, Mark Skinner their new vision is “Treatment for all”.
Mr. Skinner added “The Grifols donation moves us up one step closer to making our vision a reality; but we have a long way to go.”
“Through the donation we hope our therapies will reach the most needy patients. The mission of WFH is noble, and is consistent with the ethical value of our company.” said Victor Grifols, President and CEO of the company.

Thank you all for your participation in our Survey. The results are attached with the newsletter.

The information really helps us to continue to provide you with the quality of information that you would like to see in upcoming newsletters. We hear you and we hope you find the information useful as well.

We hope to see you at the NHF Conference in Philadelphia!

Reminder: SAVE THE DATE:

October 12-14, 2006

Product

Benefix 80%
Mononine 12 %
Alphanine 7%
Other 1%

Age

1-12 yrs 40%
13-19 30%
25-49 25%
50+ 5%

Family History 70%
No Family History 30%

Severe 70%
Moderate 20%
Mild 10%

50% of men 45+ had or have Hepatitis C

Home Healthcare 60%
Hemophilia Treatment Center 25%
Retail/Specialty Pharmacy 15%
Other 1%

Treatment

On Demand 60%
Prevention Prophy 10%
Prophylaxsis 30%

Boys with Hemophilia 98%
Girls with Hemophilia 2%

Would you change product during an operation?

NO 90%
YES 10%

Reason: If Doctor recommends it
Another product might work better

Activities

Archery, Baseball, Boating, Ballet, Bicycle Riding, Basketball, Boogie-boarding, Boy Scouts, Bowling, Camping, Canoeing, Cub Scouts, Dodgeball, Dancing, Football, Fishing, Fencing, Golf, Guitar, Gymnastics, Gardening, Hiking, Horseback Riding, Hunting, Jogging, Jump Rope, King Fu, Kickball, Kyaking, Life Guard, Marching Band, Piano, Photography, Raquetball, Rafting, Rollarblading, Rodeo, Restoring & Showing Antique Farm Gas Engines, Swimming, Sailing, Soccer, Skateboarding, Softball, Singing, Soccer, Skiing, Ski Racing, Showing Cattle,Tee-Ball, Tae Kwon Do,Tennis, Track Cross Country, Walking, Weight Lifting, Yoga

Reasons for switching product?

Product Purity 83%
Safe, Effective

Doctor Recommendation: 15%

Works better for me
Recovery good 1%

Allergy/Inhibitor
Availability, Costs 1%

Emergency Room Experience
Funny after 12 years we only have 1% of people with hemophilia having a good experience

Emergency Room Experience

Takes too long
Inconvenient, Torturous, Dangerous
They don’t listen
Nightmare, Arduous, Harrowing
Lack of knowledge on Hemophilia
Traumatic
We stopped going
Slower than paint drying
Horrible, we infuse first and then go (if we think something is sprained or broken)
Difficult

Funny Bloopers from Doctors

“When did he contract Hemophilia?’
“Your overdosing him”

Funny answers When asked to describe your experience or your childs with receiving treatment for Hemophilia in an Emergency Room

“Unlikely to happen - they don’t have it or know what to do with it (regarding hemophilia)”

“They don’t care, my hospital is so little they don’t have time for me”

“ER Treatment is a course of treatment I try to avoid at all costs”

“A circus -they still look at me like a bearded lady!”

Funny Parent’s comments on type of info needed for ER physicians and nursing staff

“Anything they can get because obviously thar are not getting any”

Information that ER’s should have:

Much information about Hemophilia and different types, also severity
Have factor on hand ASAP
Know that people with hemophilia also carry factor with them
LISTEN to the Parent or Patient
Bleeds need to be treated IMMEDIATELY - Then examine
LET Parent or Patient mix factor if you do not know how
TRUST Parent or Patients Knowledge
KNOW bruising is not child abuse
Know each case is different based on type, severity, inhibitors, etc.
Teach them how to reconstitute factor
Classes on hemophilia
Dosing chart for As and Bs