Fall 2003 - Factor Nine News

The Coalition for Hemophilia B

Topics In Hemophilia

• Gene Therapy - John Taylor, Jr.
• Mononine 500 IU Now Available
• 2003 Lawrence Madeiros Scholarships
• Wyeth Benefix Production Expansion
• Eric Frey - Highschool Football
• Hemophilia B Family Forum - November 8

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Gene Therapy
John Taylor, Jr.

Many of us in the Hemophilia community believe that gene therapy will be “the cure” as the NHF puts it. But when is this coming?

Way back in 1992, I was among a small group of patients, advocates and doctors who were invited to discuss the recombinant factor IX project at Genetics Institute (GI). The company was worried that gene therapy would make their new product worthless.

The scientists at GI were rushing the product - Benefix - to the market as fast as they could, but they were afraid that the $100 million that they would have to spend would be thrown away. What if no one used Benefix? Some experts had forecast that gene therapy would replace all factor replacement products within ten years - that is by 2002.

Every one of us told GI that we wanted the product, and we are lucky that they listened. Because Genetics Institute took a chance spending money and developing Benefix, we owe them a great deal of thanks for the risks they took.

Even GI thought that Benefix would only have a few years of marketing success before gene therapy pushed it aside. They were wrong.

What happened to gene therapy? What slowed it down? Basically, there were two problems. First, it was much more difficult to bring to perfection than it was to demonstrate the concept, and it must be perfect to be safe and dependable. Second, there were some major mistakes made in early trials for other conditions and the FDA reacted by tightening the standards for the other candidates, including Factor IX, to very high levels.

The Company working to get the Factor IX gene therapy product through the FDA’s trials is Avigen, and it has bet its survival (by my estimate) on the success of this product. It has assembled an outstanding team of scientists, all extremely knowledgeable and well known in hemophilia circles.

About a year ago, Dr. Glenn Pierce joined Drs. Mark Kay and Kathy High to push the product through. One could hardly imagine a stronger team. However, the road has not been smooth, and the steps to approval have been very slow in coming. They are still in Phase I and earlier this year the trial was halted for a while as one of the patients had “a temporary elevation in the levels of two liver enzymes,” something that had never occurred before. You can read all about it on Avigen’s website (www.avigen.com) ~

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Mononine 500 IU Now Available

Exciting news for people who rely on Mononine Coagulation Factor IX (Human) Monoclonal Antibody Purified. In an effort to ensure greater dosing flexibility and more convenience, as well as to help families manage the costs associated with treatment. Mononine is now available in an assay size of 500 IU per vial.

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2003 Lawrence Madeiros Scholarships Winners Announced

Elise L. Anderesen of Schenectady, NY will be studying psychology, at Utica College, in the fall of 2003. She aspires to become a child life specialists, teaching sick children through medical play to help them understand their illnesses and the procedures they are about to undergo. She feels she can provide a unique perspective to kids since she knows first hand how it feels to be a patient. Elise has undergone two brain surgeries necessitated from a genetic disorder, Arnold Chiari Malformation 1 of the brain. “Good timber does not grow with ease. The stronger the wind, the stronger the trees” is one of the philosophies she believes.

Samuel W. Doughty of Salisbury, MD is planning a course of study in the recording industry at Middle Tennessee State University in the fall of 2003. “Living with a bleeding disorder has provided me with many opportunities and affects my life in many positive ways. I have met many inspiring people, who have inspired me to do my best in whatever I do so that I can be an example to other youth growing up with a bleeding disorder. “I love the art of making music because it shows the whole spirit of a person when they can express themselves through verse or song.”

The Lawrence Madeiros Scholarship was formed in 2001 in memory of Larry Madeiros. Through extensive fundraising with the Adirondack Spintacular, for the first time, we are proud to announce the selection of 4 inspiring recipients.

Joseph R. Pileri of Newbury Park, CA has been accepted at UCLA in September 2003. “For me, the greatest challenge that I have with hemophilia is maintaining a normal life.” Hopefully I will be able to do something that will allow me to touch others’ lives and I do not see hemophilia as hindering my future.”

Neil G. Swanson of Virginia Beach, VA has been accepted in the Intelligence Analyst Research Program at Mercyhurst College in Eerie, PA, and plans to major in history. My career goals are to have a job with a federal government agency and contribute to society by helping others. “My father has been a powerful role model for me and I can cope effectively with my bleeding disorder.”~

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Wyeth Benefix Production Expansion

As part of Wyeth’s ongoing efforts to ensure the supply of effective recombinant factor products with enhanced viral safety for the hemophilia community, we have recently filed an application with the U.S. Food an Drug Administration (FDA) to expand our production capacity for BeneFIX Coagulation Factor IX (Recombinant).

BeneFIX is currently produced in Andover, Massachusetts. Pending approval by the FDA, the additional manufacturing facility, located in Conshohocken, Pennsylvania, will increase Wyeth’s capacity to produce BeneFIX by more than 50 percent.

The filing initiates the FDA’s formal review of Wyeth’s application. It does not in any way anticipate the outcome of that review. The FDA can approve, reject, or see more information with respect to the application.

We will continue to keep you updated on the outcome. For more information, please contact Wyeth’s Hemophilia Hotline at 1-888-999-2349~

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Eric Frey - Highschool Football

Friday night at Centerville Football Stadium, Eric Frey did something he could only dream about before.

It was the night of firsts for the Knights senior as he connected on his first career field-goal attempt. The 32-yard shot proved to be the difference as the Knights edged Columbus Independence 24-21.

Frey who has hemophilia B, was only recently cleared by his doctor to play the game. “If someone had told me a year ago that I’d be playing football, I wouldn’t have believed them,” the Knights’ placekicker said. “But all I kept thinking when I was getting ready to kick was that this was exactly what I wanted to do. I wanted to have the chance to kick the game-winner.”

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Hemophilia B Family Forum - November 8

AVENTIS BEHRING INDUSTRY SPONSORED SYMPOSIUM
in conjunction with
THE COALITION FOR HEMOPHLIA B FAMILY FORUM MEETING
Saturday, November 8 from 7:00 am - 9:00 am
Grand American Hotel, Imperial Ballroom D (Located on Lobby Level )
TOPIC: THE ROAD TO RECOVERY: CHOICES FOR HEMOPHILIA B

Speaker: Dr. Jorge DiPaula, Assistant Professor of Pediatrics and Director, Hemophilia Treatment Center, University of Iowa

Program Description: The program will provide an overview and discussion of the various treatment options available for Hemophilia B. The history, manufacturing processes, and pros and cons of each FIX product on the market will be discussed. Q & A/open forum will follow the presentation.

Babysitting will be provided by the Youth and Adolescent program beginning a few minutes before 7 am. The location of the Y&A program/babysitting will be meeting rooms on the 3rd floor of the Grand America Hotel). Children must be pre-registered for the Y&A program in order to be eligible for babysitting services. If you have small infants and want to bring them to the meeting please do. If you have any questions please call Kim at (212) 628-3445

We look forward to seeing you!