Winter 2005 – Factor Nine News

TOPICS IN HEMOPHILIA

-Nosebleeds

-SCHIP Program

-Research – Velander

-Novo Nordisk -Ron Promer Retires

- ZLB Website – Wyeth CD/Booklet

-New site for Wives of men with hemophilia

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NOSEBLEEDS

It’s that time of year when many people experience an increased incidence of
nosebleeds. Bleeding usually comes from the rupture of small blood vessels inside
the nostrils. The piece of cartilage that divides the nostrils is called the septum and is the most likely spot for bleeding to occur. Nosebleeds can occur anytime but it is often caused as seasons change and one begins to breath very dry air. Sneezing and strong blowing of the nose as well as nose picking can also be the culprit to nosebleed episodes.

How to minimize nosebleeds:

• apply a small amount of Vaseline in each nostril to hold the moisture in during cold months. This will help prevent cracking, crust and bleeding
• use a vaporizer or humidifier to keep moisture in a room. Remember to clean unit daily to prevent germs and mold from growing in the water.

How to treat a nosebleed:

• Tilt your head forward so that the blood will not run down the back of the throat. By minimizing the amount of blood that is swallowed, there will be less likelihood of nausea. It is also easier to determine when the bleeding has stopped if you are leaning forward.
• One may see stringy, jelly-like clots of blood. These clots are weak and may actually prevent the strong/good clots from forming. These clots need to be gently blown from the nose.
• Press the sides of the nose firmly together. You may have to hold this pressure for up to twenty minutes to halt the bleeding. (Sometimes if pressure does not stop the bleeding, applying an ice pack over the bridge of the nose will help close the blood vessels in the nose).

When to call your Doctor or Treatment Center:

• if the nosebleed is very bad or lasts longer than usual or persists on and off over a period of several days, call your Doctor.
• if you think you need to take factor because the nosebleed is so bad or persistent and
• if the nosebleed was caused by an injury to the face or head.

Remember:

• in hemophilia, factor is not usually needed for nosebleeds that do not last long.
• gel foam packing is the preferred method for persistent nosebleeds instead of over cauterization and forceful packing.
• it is not uncommon for a person to throw up (vomit) after a nosebleed because the blood swallowed upset the stomach.
• stools may appear dark, tar-like in color if much blood has been swallowed during a nosebleed.~

Information courtesy of:
Markay Thrower, RN

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SCHIP PROGRAM

As part the Balanced Budget Act of 1997, Congress created the State Children’s Health Insurance program (SCHIP) to address the growing problem of children without health insurance. SCHIP was designed as a Federal/State partnership. similar to Medicaid, with the goal of expanding health insurance to children whose families earn too much money to be eligible for Medicaid, but not enough money to purchase private insurance. SCHIP is the single largest expansion of health insurance coverage for children since the initiation of Medicaid in the 1960’s.

The number of children covered nationwide in 1998 was 897,000 and grew rapidly to 3.95 million by mid 2003 before leveling off.

For more information on this program visit www.cms.hhs.gov/home/Schip.asp

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TRANSGENIC RESEARCH

Transgenic technology is the science of producing organisms that contain foreign genes, genes that the organism does not naturally contain. It has become a common tool in medical research: for instance, researchers might introduce a human cancer gene into mice to study how that gene behaves under different conditions. Gene therapy, which we have often written about as a potential future cure for hemophilia, is also a form of transgenic technology. In gene therapy for hemophilia B, a gene for factor IX would be introduced into a hemophilia patient (who does not have a factor IX gene, or at least one that works) so that his body would then produce factor IX.

Transgenic technology also has a potential use in producing human factor IX in animals as an alternative to collecting factor IX from human plasma. Researchers have been able to insert genes for human factor IX into the egg cells of animals in ways that an adult female animal that is born from that egg will produce factor IX in her milk. The factor IX can be purified from the milk and potentially made into a product for human use. This has been done experimentally with many common farm animals such as cows, sheep, goats and even pigs.

One of the leaders in this field is Prof. William Velander at the University of Nebraska. He recently received a large NIH grant to develop a transgenic factor IX for testing in humans. Prof. Velander’s

Dr. William Velander (photo taken at the Nebraska Chapter Conference held at Mahoney State Park, November 11, 2005 where he gave a speech on his transgenic research)

group makes transgenic factor IX in pig milk. Interestingly, although more common, milk animals like cows, sheep and goats can also produce transgenic factor IX, the mammary (milk-producing) cells in pigs produce a factor IX that is much closer in structure to human factor IX. In addition to the basic structure of a protein, which is determined by its gene, many proteins, including factor IX, have slight modifica-tions that are made by the mechanisms that the cell uses to manufacture the protein molecule. It turns out that the mechanismsin pig cells produce a factor IX protein molecule that is modified very similarly to that of human cells, while those of cows, sheep and goats are much more different.

Prof. Velander’s group now has two pigs that produce more than 3000 units of factor IX in each ounce of milk, about 100 times the amount in human plasma. At that level, they estimate that a herd of only 30-60 pigs could supply the entire U.S. requirements for factor IX, for prophylactic treatment. The factor IX has been purified and tested in hemophilic mice. It appears to have a specific activity equal or greater to than human plasma-derived factor IX. It also has a half-life twice that of plasma-derived
Factor IX. That is, it stays in the bloodstream twice as long.

Under the NIH grant, Prof. Velander’s group plans to produce enough transgenic factor IX to test in hemophilic dogs and then, if that works to produce material to test in human volunteers. The University of Nebraska has a laboratory equipped to produce drugs with the purity needed to satisfy FDA requirements for use in humans. The work is expected to take about five years. That might seem like a long time, but it can easily take that long to do all of the research and testing required to produce a drug that appears safe and effective enough to try in humans.

You might ask, why couldn’t I just drink the milk to get my factor IX? That’s not as far fetched an idea as it sounds, although the milk itself might not be the best liquid form to use. Prof. Velander is also working on oral delivery of factor IX. He has found that giving mice a daily drink of factor IX solution can produce a reasonable level of factor in their bloodstream. Only about ½ to 1% of the factor IX they ingest shows up in their bloodstream, but Prof. Velander thinks they can increase that by modifying the mixture of components in the factor IX solution. However, because such a large amount of factor IX can be made in pig milk, even if only 1% is absorbed, enough could be made to make that a reasonable treatment method.

Because of the large amounts that can be produced, transgenic factor IX should be relatively inexpensive. Cow milk at the store is $1-2 per quart. Transgenic pig milk will be more expensive than that. Even $10-20 per quart is probably a conservative estimate, and that quart would contain 100,000 units or more of factor IX. In fact, a number of less-developed countries have been very interested in Prof. Velander’s work because it represents a cheap, safe, and plentiful source of clotting
factor for their hemophiliac populations.

Transgenic factor IX should be as safe as recombinant factor IX. In fact, transgenic factor IX is a recombinant product.The current recombinant product is made in hamster cells in stainless steel bioreactors, while transgenic factor IX is made in pig cells in walking bioreactors. Just like the hamster cells, the pigs would be screened for diseases and would be protected from exposure to any outside source of disease. Also, just in case, the purification process for transgenic factor IX will contain steps to inactivate or remove viruses, as is the case for the recombinant factor IX product.

Transgenic factor IX, especially with oral delivery, has the potential to revolutionize treatment of hemophilia B. Gene therapy will hopefully one day give us a cure, but until then, it’s not too hard to imagine every hemophilia B patient in the world getting prophylactic treatment by just drinking a glass of Factor-ade for breakfast.

Article by:
Dr. David Clark

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Ron Promer retires from Novo Nordisk

After 35+ years at Novo Nordisk, Ron Promer is retiring. Ron and his wife, Nancy are looking forward to their “new life” together doing some traveling, enjoying their grandchildren and spending time with friends and family.

Ron was such an instrumental part of Novo Nordisk and a man who truly cared about
people with hemophilia and their quality of life. We will truly miss you and wish you the best always.

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New Comprehensive Website

www.HemophiliaB.com is a new website that is a valuable resource. There is a broad range of topics with up-to-date information on hemophilia B, links to helpful web resources and free educational materials.

HemophiliaB.com educational information is produced on behalf of ZLB Behring. ~

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Josh Valence looking to form support group for men in Florida

For those in the Flordia area who are interested in attending please contact Josh Valence at (321) 259-5421 ~

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Coming soon by mail…..

A new booklet out called B to B, stamped with the approval of the consumer advisory board (made up of people with hemophilia B or a loved one with hemophilia B) will be coming directly from The Coaliltion for Hemophilia B to you shortly. The booklet was produced by Wyeth Pharmaceuticals.~

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Visit inalex.com for the Latest information on Seminars by Joe Caronna

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Living with Hemophilia Choices Throughout a Lifetime

A DVD on this interesting conference held at the NHF this past November will be coming to you in the mail directly from us as well. On this DVD you will hear various stories from people with hemophilia, some older, teenagers, college students, mothers, etc. Each person shares their view on hemophilia and how they have dealt with it in their lives. We found this to be an extremely valuable tool for people, support and education wise. This conference and DVD material is supported by Wyeth Pharmaceuticals. The moderators are Maureen Cook and Yolanda Garcia. The site is bilingual and will be up and running by February 1. To join please visit our website: www.coalitionforhemophiliab.org to see information posted. If you have any questions please direct them to Kim at hemob@ix.netcom.com

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The Coalition for Hemophilia B introduces a new email support site for wives of men with Hemophilia

The support site is founded for the unique purpose of helping wives gain the support they need from women who have already gone through the emotional roller coaster
of dealing with the financial issues of hemophilia, need support in learning how to navigate insurance issues, learn what to expect if your husband is starting Hepatitis C treatment, or has another dual diagnosis. And addresses many other issues as well.

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REMINDER

THE HEMOPHILIA FEDERATION OF AMERICA (HFA) SYMPOSIUM IS IN ST. LOUIS, MO – MARCH 31-APRIL2 THE COALITION FOR HEMOPHILIA B WILL HAVE A BOOTH AND ALSO HOLD A FACTOR IX FAMILY MEETING

DETAILS COMING SOON!

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