The World Federation of Hemophilia Congress was a wonderful time, and very informative. Representatives from all over the world came to Buenos Aires, Argentina, all last week, to share news, present studies and network.
One of the most talked about subjects was not gene therapy, but longer acting factor. As we mentioned in the February issue of PEN (https://www.kelleycom.com/newsletter.html) it looks like extended half-life factor will be next on the horizon for new products. Of all the companies presenting, Biogen Idec perhaps stole the show. The company reported positive results from a Phase I/IIa safety and pharmacokinetic study of its recombinant factor IX protein, called “rFIXFc,” in hemophilia B patients. The primary objective of the study was to assess safety, and the product was well tolerated (albeit in a single-dose) with no signs of inhibitor development or antibodies.
Chief medical officer of hemophilia products at Biogen Idec, is none other than Glenn Pierce, twice president of the National Hemophilia Foundation. As president of NHF a long time ago, Glenn pushed hard for gene therapy and was hopeful that it would be found in ten years, and then dampened that hope to in our lifetime. For now, it seems we may have found the next best thing: long acting factor.
The Congress gave me a chance to meet with a variety of colleagues from around the world, including those from Pakistan, Honduras, Tanzania, Ghana, India, Belize and New Zealand, just to name a few! Nice break for me: instead of traveling to meet them all in their countries over several years, I was able to meet with them all in one week!
Great Book I Just Read
The Man Who Tried to Save the World by Scott Anderson
This book gets better each time I read it. The enthralling story of larger-than-life Texan Fred Cuny, the “Master of Disaster,” who disappeared in Chechnya in 1995. Anderson actually travels there during wartime (dubbed one of the scariest places on earth) to learn what happened to Cuny. Cuny was a visionary, who turned the international disaster relief world on its head with his revolutionary–and effective–ways of approaching disaster relief. Largely self-taught, his compassion, passion and “bedrock practicality” led him to devise better and cheaper ways to save lives–not always a welcome thing to the comfy and bloated big international nonprofits. He was drawn to danger and this book reads like a spy thriller and a fascinating biography of a fascinating American. He is a true American hero. Harrison Ford bought the rights to the movie in 2002: come on Ford, where is the movie? Three stars.
