Winter 2009 - Factor Nine News

TOPICS IN HEMOPHILIA 

• • Factor IX Family Meeting in Denver
  • Laureen Kelley Award Recognition
  • What are Clinical Trials?
  • Coalition B Fundraising Dinner
  • Coalition B New York Symposium
  • Grifols On Line PediGri® System
  • Matrix Health Releases Kid’s Activity Book
  • Our Newest Coalition Members!
  • Factor IX Family Meeting in Indianapolis
  • William N. Drohan Scholarship Fund Amended
  • Holiday Fund Update

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Factor IX Family Meeting - Denver, Colorado

Thank you to all the families that attended the Coalition for Hemophilia B Family Meeting in Denver,
Colorado, which was held in conjunction with the NHF’s 60th Annual Meeting last November. We were
very please to have 50 Coalition B members join us for breakfast. It was great to see you there!
Wayne Cook

Factor IX Family Meeting attendees

 3 year-old, Eli Roland of Arkansas pictured with Wayne Cook, President of The Coalition for Hemophilia B

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Laureen Kelley

Save One Life

Awarded for its Work with Impoverished Hemophilia Patients

Laureen Kelly (center) pictured with (left to right)
Tiki Barber, Al Leiter, Swizz Beatz, and actor Steve Guttenberg

The Children’s Cancer and Blood Foundation (CCBF) of New York held their Ninth Annual “Breakthrough Ball” at the newly renovated Plaza Hotel on October 28, 2008.  Laureen Kelly, President of LA Kelley Communications, Inc. of Georgetown, Massachusetts, was recognized for her achievements in helping the world’s poor with hemophilia.  Laureen is the mother of a child with hemophilia and the author of 11 books on hemophilia and related blood disorders.  She is also founder of Project SHARE, which donates millions of dollars of blood-clotting medicine annually to over 45 impoverished countries.  In addition, Laureen is the founder and President of Save One Life, Inc., a nonprofit company devoted to providing sponsorships for children with hemophilia in developing countries.  Save One Life currently supports 360 children in nine countries and is growing.

In her acceptance speech, Kelley said, “I accept this award, not for what I have done… but as a pledge for what I will do.  I promise you on this evening… I will enter the second half of my life completely dedicated to Save One Life, which will become the voice of impoverished patients with hemophilia.”
Kelley thanked gala patrons Bayer Corporation, Baxter BioScience, and Grifols.  She also invited people to come with her on her worldwide travels.  A number of people showed a keen interest to travel thoughout the country to help with this project.

Actor Charles Grodin acted as master of ceremony for the evening.  Grodin first aired a music video by Swizz Beatz, rapper/music producer (also honored at this event) highlighting his visit to New York-Presbyterian Hospital to cheer children with cancer.  Beatz composed a theme song, “One Day,” for the CCBF and was the first to receive an award.  He has worked with the United Nations to unite the entertainment community in its efforts to promote peace around the world.

Other honorees include actor Steve Guttenberg for his efforts to get 50,000 eyeglasses for low-income children, for volunteering 16-hour days to help Katrina victims, and for founding the “Guttenhouse,” a transitional home for foster children.

Former MLB pitcher Al Leiter was honored for having given more than $1.5 million since 1996 to various children-related charities in the New York area and in south Florida.  Leiter, who had 19 years in the Major League as a pitcher, has won nearly every philanthropic award MLB offers, including the 2000 Robert Clemente Award.  He is currently a baseball analyst for the YES Network.

Over 500 people attended this event including NFL great Tiki Barber, last year’s honoree and honorary co-chair.  The event raised over 1.5 million for the foundation.

For more information about the Children’s Cancer and Blood Foundation or to make a donation, please visit
www.childrenscbf.org

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What are Clinical Trials?
By: Dr. David Clark

Clinical research is research performed with human subjects.  Because the aim of research is to find out something that is unknown, there is always some risk involved.  Therefore, most developed countries, including the U.S., have evolved “ethical” approaches to minimize the risk to the subjects while maximizing the amount of information obtained.  The U.S. National Institutes of Health (NIH) have developed seven ethical requirements that must be met by any clinical research.  The research must have 1) social and scientific value, 2) scientific validity, 3) fair subject selection, 4) a favorable risk/benefit ratio, 5) independent review, 6) informed consent and 7) respect for potential and enrolled subjects.

For hemophilia, the most common type of clinical study is done to test a new clotting factor product.  To license a new clotting factor, a company must prove to the FDA that the new product is safe and efficacious, that is, that it works.  This usually involves a lot of laboratory studies, called pre-clinical studies, followed by an actual test in patients, a clinical study.  The pre-clinical phase can last for years and includes determining how to manufacture the drug reliably, how to package it, determining its shelf life and laboratory and animal studies showing the product is safe and likely to work as intended.  When a company has accumulated enough pre-clinical data that it thinks it can satisfy both itself and the FDA that the product is safe and has a good chance of actually working, it files an Investigational New Drug (IND) application.

The IND contains all of the preclinical data, as well as the detailed plans for the clinical study.  The clinical study plans have to be very specific.  A company can’t just say they want to give a product to ten patients and see what happens.  The company has to decide ahead of time exactly how it’s going to administer the product, what data it’s going to collect, how the data will be analyzed and interpreted, and what the results have to be to say that the study was successful.  All of these things make up the study protocol, the detailed plan for how the study will be conducted.

The company or other organization that is setting up the clinical study is called the Sponsor.  However, to minimize bias, clinical studies are usually performed by independent physicians and institutions, who are called the Investigators.  Often there are one or two investigators overseeing the whole study who are called the Principle Investigators.  The investigators ideally have no stake in the outcome, one way or the other, and therefore give the product a fair test.

Clinical studies are always done in volunteers, and the methods are carefully designed to make sure that subjects do not feel coerced into participating.  One of the most important parts of the IND package is the “informed consent” document.  The name “informed consent” comes from the idea that all subjects in the study must be completely informed about the risks and benefits of participating in the study and must voluntarily consent to being in the study.  The informed consent document puts this in writing and must be signed by all study subjects.
Before a clinical study can begin, the proposed study protocol and the informed consent document must be approved by the Institutional Review Board (IRB) at the facility(ies) where the study will be performed.  An IRB is a panel of at least five people with varying backgrounds and expertise who review all proposed human clinical studies to make sure they are ethical, have sufficient protection for the participants and are likely to produce the intended results.

INDs are not approved by the FDA; they are a notice to the FDA that someone plans to start a study in 30 days.  If the FDA has a problem with the study, it will tell the Sponsor of the IND not to start the study.  However, if the FDA does not respond, the study can proceed.  INDs can also be amended as more is learned about the new product during the clinical studies.

Clinical studies are often done as comparisons between a new drug or method of treatment and what is called the “current standard of care.”  The gold standard for clinical studies is the randomized clinical trial.  This is the kind of study done, for instance, with a new heart drug in which hundreds, or even thousands, of patients are enrolled.  Patients are randomly assigned to get either a new drug or the old “current standard of care” drug.  Where possible, such a study is performed “double blind” so that neither the physician nor the patient knows which drug is being used until the end of the study.  That helps to eliminate bias.

However, because hemophilia, especially hemophilia B, is such a rare disease, large randomized clinical trials are rarely performed – there are not enough patients.  Fortunately, hemophilia B is fairly well understood.  It is known that finding sufficient factor IX clotting activity in the blood after infusing a new product is good evidence that a product works.  Factor IX studies are often done without comparison to another product.  However, if it is desired to do a direct comparison, a “crossover” study design is often used.  In a crossover study each patient receives both the new and old products in random order, usually with a “washout” period of a few weeks between products.

In the U.S., clinical studies of new products usually go through three distinct phases, which build on each other.  Phase 1, primarily concerned with safety, is the first introduction of a new product into humans, so it proceeds very carefully.  (Note that the government does not seem to have standardized on a numbering system for the phases.  You will see Phases 1, 2 and 3 as well as Phases I, II and III.)  Often in Phase 1, the dose starts out very low in the first patients and then is gradually increased as the study proceeds.  In many non-hemophilia studies, Phase 1 is done in normal healthy subjects to minimize the risk to patients who might already have compromised health.  However, for hemophilia, all phases are usually done in patients with hemophilia.

Phase 2 is usually done in patients with the given disease or condition and starts to look at efficacy, how the new product works and should be dosed, as well as side effects of the product.  For products and diseases that are thought to be fairly well understood, Phase 1 and Phase 2 are often combined as a Phase 1/2 study.  Phases 1 and 2 provide information that is used to determine the dosage regimen that will be used in the final Phase 3 study.

Products may still change and evolve during Phases 1 and 2 as more experience is gained with them.  However, by the start of Phase 3, the product should be in its final state and the recommended dosage should be known.  Phase 3 tests the actual product that is intended to be marketed in the way it is intended to be used.  Any significant changes after the start of Phase 3 could require repeating some or all of the previous studies.  Phase 3 is usually performed in a larger number of patients and provides the primary information on efficacy and adverse effects on which licensure will be based.

For Factor IX products, pharmacokinetic studies are often performed during the clinical studies on some or all patients.  Back when all products were plasma-derived, differences in the recovery or half-life compared to those of normal factor IX were often evidence of damage to the factor IX molecules during processing.  Recombinant factor IX molecules are already slightly different, so the pharmacokinetic behavior doesn’t necessarily give the same kind of information on the molecules themselves, but it is important in determining dosage amount and frequency, especially for use in prophylaxis.

There may also be a Phase 4 study, which is being required more often nowadays.  The information obtained during Phases 1 through 3 is usually what FDA needs to license a new product.  However, if there are still questions about a product, for instance, about any possible long-term effects, FDA can require a company to conduct a Phase 4 study after a product is licensed.  Another reason for a Phase 4 study is that under certain circumstances FDA can license a new drug based on a limited amount of clinical data, if it is the only potential treatment for a life-threatening condition.  To be safe and make sure that nothing was overlooked, they will then require the sponsor to continue doing studies after licensure.

Until recently, clinical studies of new or modified products have been the major type conducted for hemophilia B.  However, in the last few years more studies are being conducted or proposed that do not focus on a specific product.  Instead they are used to determine optimum treatment methods such as for prophylaxis or inhibitor treatment or to test new approaches such as gene therapy.  Such studies involve a number of other considerations and will be covered in a future article.

All clinical studies in the U.S. that require government notification or approval are listed on the web site www.clinicaltrials.gov.  Several clinical studies of products in development for hemophilia B are currently in progress.  Descriptions of the studies and contact information for the sites where these studies are being performed can be found on the web site by searching on “hemophilia B.”  If you are interested in possibly participating in a study, ask your doctor about it, or you can contact the clinical study sites directly.  Because of patient confidentiality restrictions, sponsors and clinical study sites have to be careful about how they approach potential participants, which can be a problem with small patient populations like ours.  If you are wondering about a clinical trial, proactively contacting a study site is the best way to learn more.

If you are interested in participating in a clinical trial, some of the things you will want to understand are:
What is the objective of this particular study?
What is the study medication and how is it expected to differ (if at all) from your current therapy?
What are the risks involved in the study?  These are covered thoroughly in the informed consent document and should not be taken lightly.  If you don’t understand any of it, ask questions and don’t stop asking until you are satisfied.
How is the study designed and what will be your commitment?  For instance, how many treatments will be included, what laboratory testing is required, what are the follow-up requirements and what is the overall length of the study?
How will the study affect your current and future treatment?  For instance, if you are on prophylaxis, you may not want to participate in a study that does not include prophylactic treatment.
Do you qualify for the study?  Because clinical studies are highly controlled, numerous criteria must be met in order for a subject to be eligible.  These may include age, general health and inhibitor status among other criteria.
Cost is generally not a factor.  The cost of treatment and other expenses associated with a study are usually borne by the sponsor.  In fact, depending on the study, subjects receive free treatment for a period of time.

People volunteer to participate in clinical trials for a variety of reasons.  The hemophilia B community, although small, has many members who are very committed to helping improve the health and well-being of their blood brothers.  Many are also interested in gaining access to a new or improved therapy that is not yet available to the public.  (However, just because someone is trying something new, remember that there is no guarantee that it works.  On the other hand, even if something doesn’t immediately benefit you, the knowledge gained from poor results often leads to future success.)

Participating in any research study can be inconvenient and time-consuming.  However, without the participation of willing individuals the ability to bring a promising new product to market for the benefit of everyone affected is not possible.

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March 20, 2009

The Coalition for Hemophilia B

2nd Annual Fundraising Dinner

Honoring Dr. Christopher Walsh
of Mt. Sinai Hospital, New York

Join us for a Night at the Races!
Millennium Broadway Hotel; New York City

Information is now available on our website www.coalitionforhemophiliab.org
Invitations will be mailed out February 1, 2009

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March 21, 2009

The Coalition for Hemophilia B 3rd Annual Symposium

Millennium Broadway Hotel; New York City

Sponsored by Wyeth Pharmaceuticals.

Agenda and registration forms are now available online at: www.coalitionforhemophiliab.org
Hard copies will be mailed out on February 1, 2009.

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Grifols Launches PediGri® On Line for its US Plasma Therapies

September 10, 2008, Grifols launched in the US its proprietary PediGri® On Line system that gives healthcare providers access to quality and safety information about the plasma used in the production of Grifols’ therapies.  Through a web portal at www.pedigrionline.net, registered healthcare providers (including physicians, nurses, and pharmacists) can access lot specific information about the individual plasma sources that contributed to each product vial.  Grifols USA is the US sales and marketing division of Grifols, SA, a global healthcare company based in Barcelona, Spain that specializes in the production of life-saving plasma therapies.

The Grifols PediGri® system provides full traceability from donation to final product.  “Today, it is more important than ever that healthcare providers have confidence in the source of medications they prescribe and administer,” commented Bill Stopher, President of Grifols USA.  “Grifols PediGri®  provides an unparalleled level of information about the source and quality of the plasma used to produce Grifols’ therapies,” continued Mr. Stopher. “By simply entering the lot number found on the product vial, healthcare providers can view specific quality and safety information about each plasma donation used in the production of that vial, the complete certificate of analysis for the lot, and the package insert.”  To use the system, healthcare providers must register online at www.pedigrionline.net and obtain a confidential user name and password prior to accessing the PediGri® On Line web portal.

Dr. Laurence Logan, Medical Director for Grifols USA stated.  “As a physician who prescribes and administers plasma therapies, I believe healthcare providers will embrace PediGri® On Line.  This level of transparency underscores Grifols’ commitment of quality and safety to the healthcare community.”

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Matrix Health Releases Activity Book for Kids

Ft. Lauderdale, Florida - January 2009 – Matrix Health Group is pleased to announce the release of a new children’s activity book.  The book aims to educate children up to the pre-teen years about the many facets of life with a bleeding disorder.  Featuring numerous activities in a colorful and fun format, both children and adults will enjoy testing their knowledge.

Copies are available at Matrix Health booths at local and national events, by contacting any Matrix Regional Care Coordinator, by visiting our website, or by calling (toll free) 1-877-337-3002.

Matrix Health Group is a specialty pharmacy that primarily services the bleeding disorders community.  With a full range of factor and ancillary supplies, in addition to unwavering dedication and unique experience, Matrix Health Group provides an unprecedented level
of service for clients and the bleeding disorders community.

Please visit us online at: www.matrixhealthgroup.com

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Welcome Our Newest Members!

Join us in welcoming 15-month old twins,
Max Edward (left) and Eli Sloan,
sons of Lisa and Bradley Schoenfeld
of New York.

As an obligatory carrier, Lisa knew to take precautions.  Lisa and Bradley visited the treatment center prior to, and were essentially prepared for the birth of these beautiful little boys born September 30, 2007.

Welcome to The Coalition for Hemophilia B!

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The Coalition for Hemophilia B

March 2009  Factor Nine Family Meeting

Indianapolis, Indiana
Saturday, March 14, 2009    11:00 a.m. - 12:30 p.m.

The next Factor Nine Family Meeting will be held in conjunction with the 2009 Hemophilia Federation of America Educational Symposium in Indianapolis, Indiana Marriott East Indianapolis Indiana  -  Grand Ballroom - Salon

Please join our Hemophilia B forum to discuss current events, share and support, and play our newly updated game, “Are you Smarter than Your Hemophilia,” which will truly test your knowledge!

Lunch will be served.  We look forward to seeing you!

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William N. Drohan Scholarship Fund Amended. . .

Please be advised the William N. Drohan Scholarship fund has been amended to read:
“This fund was created for children of scientists, doctors, nurses, pharmacists and bleeding
disorder healthcare professionals who are part of the comprehensive care team.”
Forms are available on our website at www.coalitionforhemophiliab.org under scholarships.

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With Much Thanks . . .

We want to extend our sincere appreciation to our members who donated to the holiday fund this past December.  Your donations along with those of  The Coalition for Hemophilia B, we, as a team, were able to purchase holiday gifts for over 72 children; we purchased food baskets and bought coats, shoes, clothing, and toys for so many.

It reaffirms our strong belief that a small group of people can
indeed have a strong impact on the lives of others!

  “Never doubt that a small group of thoughtful,
committed citizens can change the world;
indeed, it’s the only thing that ever does.”
Margaret Mead
From Everyone at The Coalition for Hemophilia B, we wish each and every one of you a very healthy and Happy New Year!