In a shocking article, Crohn's patient accuses the FDA of killing him. Gideon J. Sofer is a Crohn's sufferer who has taken part in the Osiris Therapeutics Phase II trials of their new adult stem cell therapy Prochymal. Because Osiris has stated that all of the patients that received the actual drug in the trials reported positive results, Mr, Sofer understands that he must have received the placebo. He has been in and out of the hospital and his disease is progressing. His doctors have asked Osiris to give them information on whether he received the real drug or the placebo in the double blind tests, but they will not tell them.
Gideon Sofer wants the real drug, but even though it is on Fast Track for approval by the FDA, it might be years before it is available. Read this quote for the most shocking part of the issue:
I am of two minds on this issue. One the one hand, I do understand the value of Double-Blind scientific testing of new medications. Otherwise we will not properly know if the product really works. Also, they need to study the long term effects of the drug. Only look at the new black box warnings on Humira and Remicade to understand that what seems like a miracle drug can sometimes have unintended longer term side effects that need to be studied as well as the immdiate results.
On the other hand, if it is within the scientific protocols to allow a patient who received the placebo in a trial to receive the actual medication after the trial period is over, why will Osiris not allow this to occur in this case. In a case where the disease can lead to a huge reduction in quality of life, extreme pain, and even the possibility of death, can't they bend?
I am personally really looking forward to see the long term effects of Prochymal. I beleive that stem cell therapies are a beacon on hope for myself and others with a wide range of diseases. However, I am also a bit concerned about long term effects and would like to know that a product is safe. I am avoiding taking Humira, although it is the planned next step in my treatment plan, because of two reasons. One, I want to save the "big guns" of biologic treatments as long as possible so as not to run out of options. Two, I am afraid of the possible increased cancer risk that may be associated with the biologic treatments.
Although my "mild" Crohn's causes me pain, fatigue, and has hospitalized me, it is not threatening my life at this time. In fact, Crohn's is not generally considered to be a killer disease, which may be part of why Osiris doesn't want to bend their study rules and provide the medication to their placebo subjects. However, Crohn's can cause death, usually in terms of complications from surgery.
I really feel for Gideon Sofer's plight, and I hope that Osiris changes it's mind. I realize that they have a potential multi-million dollar market for Prochymal and they want the trials to be scientifically sound. But please, people at Osiris/Genzyme - reconsider and help your study subjects.
And two other requests: to the FDA I plead that you are able to properly balance the need for testing and study with the need for fast tracking drugs that have the potential to save lives for people that simply cannot wait. And to the President, please create funding opportunities to allow all appropriate uses for stem cells to save lives. G.W. Bush and his opposition of stem cell reaseach are about to be out the door. Please let's have a government environment that allows scientific research to thrive. We need it!