I am getting emails and requests from both pharmaceutical companies and now HTCs to help recruit patients for clinical trials. It’s tough: we have great products out there. What’s the incentive to try something experimental?
Some patients do it for free factor. Others do it to try to help advance science. Chris Bombardier, from Denver, I just learned summited Mt. Aconcagua in Argentina (22,000+ feet!) and he is on an experimental long-lasting factor. He chose to do it to advance science and see how the product works. He’s proving it works the hard way!
There has probably not been such a clogged pipeline for new products coming to market (we hope) in maybe 20 years. Patents have expired, which protected products from competition, and now manufacturers are all competing to get new products into the biggest hemophilia market on earth: the United States of America.
Here’s one I just read about:
CSL Behring has announced that the first patient has been enrolled in the pivotal pediatric phase III study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) in previously treated children (up to age 11 years). The study site for this first enrollment is the Czech Republic.
CSL Behring, in collaboration with its parent company, CSL Limited, is developing rIX-FP through the PROLONG-9FP clinical trial program for the prophylaxis and treatment of bleeding episodes, including control and prevention of bleeding in surgical settings
in patients with factor IX deficiency.
Results of a Phase I study evaluating recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with severe hemophilia B were publicly presented earlier this year and published in BLOOD 2012 showing that rIX-FP achieved a 91.57 hours terminal half- life, incremental recovery of 1.376 (IU/dL) / (IU/kg), and clearance of 0.75 mL/h/kg. This was an extension in half-life of 5.3 times that of the current recombinant FIX therapy.
We all know that an extended half-life could potentially reduce the number of injections needed in
patients receiving prophylaxis from two or three injections per week to once weekly or significantly less frequently. Several companies and HTCs involved in research, are now in need patient to volunteer to participate.
If you’re interested, go to www.clinicaltrials.gov, where you can read about clinical trials for hemophilia.
Great Book I Just Read
Why is It Always About You? by Sandy Hotchkiss
You know those people who hog the whole conversation, grab the limelight [often from you], make you their personal audience, and don’t listen or even seem to care about what’s happening with you? They could have Narcissistic Personality Disorder. More than just being talkers, they may latch on to powerful or attractive people to make themselves look or feel better; slowly chip away at another’s self-esteem to build themselves up; and go blank even when the person in front of them is in severe emotional pain. They lack empathy. It’s all about them, their agenda, their needs and their ego. We call them emotional vampires because they can leave you depleted and drained. And yet, they are smart and charming typically, and can draw you in, and back again. Run for the hills when you discover someone has this insidious personality, or get professional help to support you through a relationship with them. This excellent book will help you recognize a narcissist, and teach you how to protect your own self against their manipulations. Three/five stars.