May 2007

News from Scotland

I am so often focused on the problems with hemophilia in the developing world, it often still comes as a surprise to hear of problems with patients in developed countries, where hemophilia treatment is abundant and considered the world’s standard. Canada, Western Europe, Australia and New Zealand enjoy not only a high level of hemophilia care, but most have socialized medicine– which means everyone gets factor. But not all is perfect. While in the US we are battling tightened insurance coverage for the product and delivery company of our choice, in some countries patients are also battling compensation for blood infections from 30 years ago! Lately Scotland has been making the news, and just secured a landmark decision. Read below to see what is happening there.

The irony for the developing world is this: as most did not have access to factor concentrates during the 1970s and 80s, most were not infected with HIV or hep C. However, HIV is on the rise in developing countries through other means, not concentrates, and as patients often rely primarily on plasma for treatment, HIV and hep C rates there could potentially soar. This is why so many of us work to get factor concentrates to as many people as possible in the developing world. Later this year, I’ll be traveling to Africa, which has among the highest rates of HIV in the world, to assess care in several countries. Whether we live in developed or developing countries, our problems are not exclusive: we are all fighting for better care, greater compensation, and justice. In most cases, for simply the right to live.

Executive about-turn

A PUBLIC inquiry examining how patients [with hemophilia] were infected with hepatitis through contaminated blood products is to be held, it was revealed yesterday. Hundreds of people in Scotland, including haemophilia sufferers and other patients, were given contaminated blood in the 1970s and 1980s. But the previous administration had resisted calls from victims and their families for a public inquiry.

The revised decision comes two months after an independent inquiry concerning the deaths of nearly 2,000 haemophilia patients who were exposed to HIV and or hepatitis C through contaminated NHS blood and blood products began in London.

Former Solicitor General Lord Archer of Sandwell, who is heading the public inquiry, said its purpose was to investigate the circumstances surrounding the supply of contaminated NHS blood and blood products to patients, the consequences for the haemophilia community and others afflicted.

At the time that inquiry was announced, former health minister Andy Kerr said there were no grounds for such an investigation in Scotland – although he added he would reconsider if fresh evidence came to light.

He said in February that there would be “no further practical lessons” to be learned from a government-led inquiry, arguing there had already been several probes into different aspects of the “unfortunate events”.

The full remit and timing of the inquiry have still to be decided by ministers. An Executive spokesman said: “The Scottish government believes in a more accountable health service, and a public inquiry in Scotland to find out why people were infected with hepatitis through NHS treatment is the best way forward.”

New Inhibitor Book in Progress!

Well, I was hoping to be blogging from Nashville this weekend, as I was planning to attend the Inhibitor Summit meeting sponsored by Novo Nordisk.

Did anyone reading this go to the Summit? If so, let us know how it was! These are really fantastic events that bring the inhibitor community in touch and closer, and I think by now we all recognize that this has been a neglected part of our community. Also, this is a great time to mention that I am now researching and writing the world’s first inhibitor book for parents and patients. If you have inhibitors or a child with inhibitors, please let me know. We’d love to interview you and hear what you have to say. If you know of anyone with inhibitors who might like to participate, please have them contact us.

I just finished watching a nice video called “A Bright Future,” geared for parents of children newly-diagnosed. It consists mainly of various testimonials by families of all types. It’s warm and fuzzy, beautifully shot, with a soft, reassuring tone. It’s sparse on information about hemophilia, so if you are looking for facts and figures, you might supplement this with some other materials. But I think if you know of new families, or if you are one, this will be a nice beginning. Typically, when there is a new diagnosis, most families don’t usually want facts and figures anyway–they want reassurance. This was created by my friend Joe Caronna at Inalex Communications, sponsored by Baxter BioScience, and features a lot of people I know! That made it very fun to watch. Order it at

Have a great week!

Factor From Goats?

A persistent question we are always asked is “What ever happened to gene therapy?” We used to write extensively about this subject when gene therapy was hot, but since the deaths of two young people in gene therapy trials unrelated to hemophilia, all gene therapy seems to have taken a slow and extremely cautious route. There instead seems to be great interest in creating new and less expensive ways to manufacture factor. We recently heard of a new biotech company in California, founded by a parent of a child with hemophilia, that is developing all therapies, which hints that these products one day will be lower in cost.

And the newswires announced this last week: GTC Biotherapeutics, a Massachusetts-based biotech company, held a webcast this morning from Monaco; the webcast will be available tomorrow through the company’s website. This company “develops, produces, and commercializes therapeutic proteins through transgenic animal technology.” This means literally milking animals for the desired protein that is developed through gene therapy and then expressed through the animal’s milk.

Like many, I feel a bit skeptical but according to GTC, in August 2006 its recombinant form of human antithrombin was approved by the European Commission for use in patients with hereditary antithrombin deficiency undergoing surgical procedures. This was the first approval anywhere in the world of a therapeutic protein produced from a transgenic animal. GTC has developed goats that have the human antithrombin gene linked to a milk-protein promoting gene so that they express this protein in their milk.

In 2006, GTC was granted a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal. And GTC has established a strategic collaboration with LFB Biotechnologies of France to jointly develop recombinant human factor VIIa as a potential treatment for hemophilia inhibitor hemophilia patients. This would be a direct competitor to NovoSeven, apparently. Although the article doesn’t directly state this, it implies that this recombinant FVIIa would be expressed by animals?

It bears watching, and we hope to bring more information about this through PEN and through HemaBlog. Watch the webcast yourself tomorrow at the GTC web site,

Would You Rather Fight Than Switch?

The ongoing insurance cost-cutting has impacted hemophilia deeply, particularly since we are flashing beacon on payers’ radar. One of the most prominent impacts has been forced home care switching. Reactions by families include irritation at being switched without forewarning; anger at being switched without any say in the matter; and fear at being switched to a company that has no experience with hemophilia.

Read the current issue of PEN, which delves deeply into what is happening, why, and how this could impact you. We interview 17 patients and opinion leaders to see how forced switching is impacting lives. What we do know is this: forced switching is not something that can easily be reversed, and it is definitely a cost-cutting tactic that is here to stay. Tims are changing–read PEN to learn about all the changes and how to adapt while best protecting your loved ones with bleeding disorders. If you are not on our mailing list, please contact us to sign up! It’s free to patients, HTCs and nonprofits.

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