New Hemophilia Product

Long, Long Time to Come

I just wrote a couple of weeks ago about long acting factor: Biogen Idec’s clinical studies have been in the works for a while, and CSL Behring just announced that they are starting a global phase II/III, multi-center study.

On January 5, Baxter announced that they are launching a Phase I clinical study of “BAX 855,” a longer-acting (PEGylated) form of a full-length recombinant factor VIII (rFVIII) protein, based on Advate’s manufacturing process. BAX 855 leverages Nektar Therapeutics’ proprietary PEGylation technology, which is designed to extend the duration of activity of proteins and larger molecules.

This means, if the trials go as hoped, longer acting factor in the bloodstream, requiring fewer infusions to get the job done.

So that’s three companies in clinical trials for longer-acting factor! More great news for our children’s future.

Great Book I Just Read
Born to Run by Christopher McDougall (Kindle version)
Stymied by a nagging injury, McDougall questions his doctors, and sets out to find a way to continue running, which leads him to the Tarahumara Indians in Mexico’s deadly Copper Canyons. These people living in near isolation, are able to run hundreds of miles without rest or injury. McDougall shares his adventure of traveling to meet them, interspersing chapters with the history of ultra running and introduces all its quirky characters, the history of the Tarahumara, and making a good case for running barefoot! The book has stirred some controversy–has he done a disservice to the Tarahumara, ruined Nike’s reputation and exalted barefoot running without citing his studies? Whatever he has done, it is a joy to read due to his crackling style, which is witty, colorful, funny and has great cultural references–this guy knows how to write! I’m not sure I buy everything he’s selling, but I did go running today, and loved it. Three star/four.

Strange Ad about A New Hemophilia Product

Sewer rat genes… That’s the science behind a new clotting gel (TT-103MH) made by a company called Thrombotargets Corporation of Spain, a gel which apparently will be able to be used by people with hemophilia. I read about this in an ad in a recent BusinessWeek, as I flew to my meeting last week in New Orleans. Flipping through the pages, the word “hemophilia” caught my eye in a headline. You don’t often see hemophilia anywhere outside of our community, so I immediately read the ad.

The ad declares the FDA has given orphan drug status to this product. And the ad makes many amazing declarations that will raise eyebrows.

The ad is an amazing essay of misinformation. Apparently it is intended to attract investors who want to sink money into something revolutionary and biotechnical–but that doesn’t know its factor VIII from its elbow.

According to the ad, there has been “little scientific innovation in the area of blood coagulation in the past 40 years.” (I guess antihemophilic factor wasn’t anything revolutionary, right?) According to Thrombotargets’ president Dr. Pedreno, “there is no medicine for treating hemorrhages.”  And by using this product, a gel, a hemophilic child can “go play in the park, and if he falls down the hemorrhage can be stopped without having to go to the hospital for a transfusion.” Are they claiming the gel somehow stop internal joint bleeds? Head bleeds? Nothing is said about these bleeds.

And what about topical thrombin products, which have been around a long time and are used for circumcisions, dental work and topical wounds? Maybe I lost something in the Spanish to English translation.

Thrombotargets promises this product will change the lives of those with hemophilia, much like insulin did for diabetics. As we in hemophilia community already know, that was already done, many times over, by antihemophilic factor, then monoclonal AHF, then recombinant AHF–generations 1, 2, 3. If Thrombotargets is going to come to our markets, it had better know its customers. It will be amazed at hemophilia customers–but they don’t like misinformation or grandiose statements. They’ve suffered much too much to be hoodwinked by fast talk and false promises.

Thrombotargets predicts that they can make about $2.1 billion from this product–but I don’t think they will get much of that from the hemophilia community. We’re smart, educated, and dedicated to ever-safer, more convenient and technologically advanced clotting factor. I’ve spent the last 20 years educating hemophilia families, and am proud at how savvy and smart they are when it comes to choices about blood clotting medicine.

Thrombotargets is looking to license the drug with a US pharmaceutical company… let’s hope potential buyers are better at studying their markets than Thrombotargets.


Great Book I Just Read: Mayflower by Nathaniel Philbrick. This is a fascinating and amazing true story about the landing of the Pilgrims, and their hardships in Massachusetts. More than that, it reveals how the native Americans helped them, describes the first true Thanksgiving, and later, how descendants of both the Indians and Pilgrims led the communities to war, which devastated the Indian tribes and shaped the future of New England. Most surprising to learn was the complex, ever-changing relationship between Indians and Pilgrims; there are no bad guys here, just complex people and communities. As a New Englander, I learned so much about how our country was founded, and especially how my state was founded. Anyone interested in American history must read this. Philbrook is a gifted storyteller, and really did his homework. This is a New York Times bestseller and has won many awards. Enjoy it in time for your Thanksgiving!

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