fusion

Long, Long Time to Come

Laurie Kelley February 13, 2012

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.

So Long! Extended Haf-Life Factor Sets the Stage

Laurie Kelley January 23, 2012
You’ve probably heard about Biogen Idec’s clinical studies of factor with extended half-lives. They aren’t the only ones investigating. CSL Behring just announced that they are starting a global phase II/III, multi-center study to assess the safety, efficacy and pharmacokinetics of recombinant fusion protein.

This recombinant fusion protein links coagulation factor IX with recombinant albumin, called “rIX-FP.” rIX-FP is indicated for the prophylaxis and treatment of bleeding episodes in patients with factor IX deficiency.

Of course, extended half-life factor means potentially fewer injections for patients, and may enable or enhance prophylactic treatment, improving quality of life for patients.

So many patients worldwide have been hoping for gene therapy–a “cure”–but it seems that the next great thing that will come along just might be extended half-life factor. We’re not sure yet which company will deliver first, but keep watching. These are exciting times and the long acting factor race is on!

Book I Just Read
The Hunger Games by Suzanne Collins

This best selling novel takes place in post-apocalyptic America, now called–for no reason disclosed– Panem. In fact, there are a lot of things that happen for no apparent reason in this book. Quick synopsis: the powers that be dictate that one teen from every district must compete to the death in the Hunger Games, in retribution for an uprising that happened a long time ago. To the victor goes fame and food to their district. The whole event is televised–reality TV where kids get killed. The heroine narrates the story, and has a pretty flat tone throughout. Think “The Truman Show” meets “Mad Max Beyond Thunderdome” meets The Olympics meets “Rollerball” and you get a great idea what the book is about. It’s not terribly original; the writing is mediocre and the story pretty predictable, characters shallow with no depth of thought or emotion. After killing her first kid, arrow through the throat, the 16-year-old narrator with the flat affect seems more concerned about whether the boy she is with loves her. Someone recommended this to me, but neglected to tell me it’s teen romance fiction, with a lot of killing. Read it if you want to kill time; it’s a few steps up from a comic book. If you want fantasy with substance and depth, try Tolkein. The book seems to be meant for a movie, and reads like a screenplay; I somehow think the movie will be better as a result, coming in March. Two stars/five.

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