|Factor Nine News|
|The Coalition for Hemophilia B|
|Topics In Hemophilia|
In this issue we are going to discuss two research studies promising results with Factor IX. The first looks at the potential for subcutaneous administration of Factor IX. Factor IX currently must be infused by the intravenous (IV) route, that is directly into a vein. Subcutaneous (SC) injection, meaning injection under the skin, is much simpler and is done with a smaller, shorter needle, like that used for many types of vaccinations.
SC injection could be a real benefit for prophylactic treatment in which Factor IX is administered every few days or even every day to keep a patient's factor level high enough to maintain normal clotability. Previous studies reported in issues of Factor IX News, have shown that when prophylactic treatment is started at an early age, patients suffer far fewer joint problems and have a much higher overall quality of life. One of the major problems with prophylactic treatment is maintaining access to veins for IV injections, especially in very young boys. The Catheters and other devices that are currently used have relatively high rates of infection and other complications that would be eliminated with SC injection.
Another problem is the difficulty of IV injection in all instances, an issue that increases in importance as boys become young men and take responsibility for their own care.
Research has shown that Factor IX administered by SC injection does find its way into the bloodstream, but the problem has been that the volume of factor needed is much larger than can be easily administered by the SC route. However, researchers from Wyeth, the distributor of BeneFix recombinant Factor IX, and from the University of North Carolina at Chapel Hill (UNC) have recently shown that a concentrated BeneFix, which was produced specifically for this test, is quite suitable for SC injection. With an injection of 1ml. to 2ml., significant circulating levels of Factor IX were obtained.
In the study, the concentrated material was given by SC injection to dogs and monkeys with very promising results. UNC has a colony of dogs with hemophilia B that has been an extremely valuable tool used by many researchers over the years. When the dogs were given the concentrated product by the SC route, about 50% of it showed up in their bloodstream with full activity. They were able to keep the dogs' Factor IX levels above 5% of normal for 5 days with a single SC injection. However there is still much work to be done. The biggest concern is that SC injection could lead to higher rates of inhibitor formation. The work with hemophiliac dogs suggeststhat this might not be a problem but the issue needs to be explored much further.
SC injection is also not suitable for immediate treatment of bleeding episodes since there is a lag of 6 - 48 hours between the time of the injection and when the maximum factor concentration shows up in the bloodstream. Of course, commercial highly concentrated Factor IX products would also have to be developed, and approved by the FDA, a process that will take several years.
The second study we would like to report on is the human clinical trial of gene therapy in hemophilia B patients. Gene therapy involves introducing the gene for Factor IX into the body to give the body the ability to make its own Factor IX. This technique has great promise as the long-sought cure for hemophilia. As usual, gene therapy has turned out to not be as easy as scientists had initially hoped, but significant advances are being made as several dedicated scientists have given their lives to this effort.
Researchers led by Dr. Kathryn High and Dr. Mark Kay working with Avigen, a biotech company specializing in gene therapy, recently reported good results from an early clinical study in six hemophilia B patients.
Avigen has developed an AAV vector which is a modified virus in which some of the viral genes have been replaced with the Factor IX gene. When this virus infects cells in the body, instead of introducing genes that produce more viruses and thus cause disease, it "infects" the cells with the Factor IX gene enabling them to produce Factor IX.
So far, six severe hemophilia B patients have received an injection of the AAV vector which Avigen calls Coagulin-B. The first four patients received low doses as part of a safety study. They tolerated the procedure well with no side effects or signs of toxicity. Although they initially achieved Factor IX levels between 1% and 2% of normal, those levels have since diminished.
Patient #5 received a higher dose and has produced factor levels above 10% of normal. Although at the time of the report, it had only been six weeks since the injection, he has shown significant therapeutic benefit. He normally needs treatment with recombinant Factor IX up to six times a month, but has not needed any factor since receiving Coagulin-B despite several minor injuries. He remains in good health including being active in competitive long-distance swimming. Patient #6 who received the same higher dose has also shown an increase in factor levels, but since he had only been treated three weeks prior to the report it was still too early to report confirmed data.
The results from both studies are very promising for improved treatment and one is a big step toward a potential cure for hemophilia B. We are fortunate to be living in a time of great medical advances - even if it seems very slow to us. It has only been 50 years since hemophilia B was recognized as a separate form of hemophilia, and less than 40 years since the first crude Factor IX products became available. Since then factor products and treatment options have improved markedly and many dedicated individuals are working toward improving life with this condition and even toward an eventual cure.
Soozie Courter Sharing A Brighter Tomorrow Hemophilia Scholarship Program
This scholarship is awarding:
12 $5,000 undergraduate scholarships
2 $5,000 graduate scholarships
3 $1,000 vocational scholarships
To be eligible for scholarships, students much have either hemophilia A or B and be a high school senior or have a graduate equivalency degree (GED) orcurrently be enrolled in an accredited junior college, college (undergraduate or graduate) or vocational school.
Scholarship applicants are required toprovide a personal essay or statement,information on their academic achieve-ments, and letters of recommendation.
To receive an application for this 2003-2004 academic year program please call Wyeth Pharmaceutical at 888-322-6010
Footnote: Soozie Courter was one of the most caringand wonderfully exciting people ever to do scientific research on Factor IX. Anyone who met her would never forget her as she lit up the room. Soozie talked with every one - over a beer, at conferences, and any time day or night. She died way before her time and we all miss her. Our cheers go to Wyeth for starting this Scholarship program. It could not be named after a more deserving person.
John and Kim
Arthur B Kane Memorial Scholarships
Application deadline: March 15,2003
This scholarship program awards at least four scholarships of up to $25,000 each (up to $6,250 per school year based on tuition, fees, books and other educational expenses)
This is available to Aventis Behring Choice Members diagnosed with a bleeding disorder who are applying to, accepted at, or currently enrolled in an accredited 2-4 year college, university, or vocational/trade school in the United States.
For more information:
Hemophilia Federation of America Symposium
Marriott Houston International Airport (on the airport property adjacent to C Terminal)
It is important to make your flight arrangements for the Inter-continental Airport. The Houston Hobby Airport is an hour away and shuttle service is expensive. For more information contact Kay Simmons 1-800-683-9882.
Deadline to register for symposium is March 12, 2003. For more information on registration or scholarship availability please call Susan Swindle at 1-800-230-9797.
Please make your hotel reservations directly with the Marriott Houston Intercontinental Airport Hotel (mention the HFA Block) Telephone number: (281) 443-2310 or 1 800-228-9290.
Factor IX Family Meeting
Saturday,March 29, 20037:30 am, Austin Room
The meeting is for people with hemophilia B and their families to support each other and share your concerns so we may support you better. Contact: Kim Phelan (212) 213-0547 or email Hemob@ix.netcom.com.
If you would like to attend, please call or email Kim or you can let her know at the booth exhibit. You may also come to the breakfast meeting without registration. T-shirts and raffle drawing.
We look forward to seeing you!
|For back issues of Factor Nine Newsletter of for more information on research please call or write to
Kim Phelan, 225 West 34th Street, Suite 710, New York, New York 10122, Telephone (212)2130547
Telefax (212)554-6900, WebSite: http://www.coalitionforhemophiliab.org/ E-mail: firstname.lastname@example.org