A few months ago, I wrote this piece trying to talk Novartis into paying for Nilotinib for the clinical trial participants. We were initially told that after they had agreed to allow us to continue on the drug under "Compassionate care" that the drug would be provided to the participants. As you may now know, that did not happen. Since I wrote this, one of the participants has passed away. We continue to wait for Novartis to do the right thing by all of us who participated in the trial.
WHERE IS THE KNIGHT ON THE WHITE HORSE?
Alan J. Hoffman, Ph.D
Do you remember the joke that went around some years ago about a divorce? The husband in the couple said that his former wife had “gotten the gold mine” and he had “gotten the shaft.” That’s the way I and eleven other patients in a clinical trial feel.
Twelve of us participated in a clinical trial at Georgetown University which tested the drug Tasigna (Nilotinib) which is used to treat leukemia to see if it was safe for patients with advanced Parkinson’s Disease. The idea was formulated by Dr. Charbel Moussa at Georgetown. He believed that substances from the bloodstream and spinal fluid which cause plaque that increases brain cell death could be reduced by Tasigna. This would slow down the progress of Parkinson’s Disease. There was pre-trial testing to make sure the patients would be able to take the medication without side effects of heart disease. The twelve who were accepted into the program began to take a daily dose of either 150mg or 300 mg capsules.
After about a month, wonderful things began to happen. The quality of my life began to improve. I no longer had to have my wife translate what I was saying to our friends. My articulation and volume improved. I began to do things around the house that I had not done in years-emptying the trash, unloading the dishwasher, setting the table, etc. My attendance at church and at activities increased. We began to participate in social activities as well. My wife says that it was life-changing for her and for my children and grandchildren. Moreover, my cognitive skills improved. I could carry on an intelligent conversation without losing track of the point. I understand that another patient who was bilingual in Spanish and English and had completely forgotten English began to speak it again after taking Nilotinib. This made a huge difference for his English-speaking wife.
Then came the rub. The medication ended for me on August 5. We dreaded that day because we feared regression without it. Just then the knight on the white horse appeared and promised Dr. Fernando Pagan, my neurologist, that Novartis, the drug manufacturer, would continue the medication for the twelve trial participants for free. Excitement was rampant. We were thrilled beyond measure. I could continue to take Nilotinib and hopefully would get better and better and at the least, be stable.
Alas, the knight on the white horse has either vanished or is delayed. Since Nilotinib is not yet approved for treatment of Parkinson’s Disease, insurance won’t pay for it. The cost is astronomical for us- $5000 in Canada for 112 days worth and over $16000 in the US for the same amount- very serious decisions for non-wealthy people.
I am now on the medication again. $5321 later, I began the medication on November 22. Today is December 15. I am beginning to get better already. One other individual in the trial is also taking the medication again. We worry about the 10 others who don’t have a choice.
Dr. Pagan has secured private funding to conduct more trials to see if Nilotinib makes the kind of difference for others that it has made for me. Years from now, the FDA may approve the medication for Parkinson’s Disease and insurance may pay for it. I believe that Nilotinib offers the next hope for patients with Parkinson’s Disease. In the meantime, the twelve of us wait for the knight on the white horse. When we agreed to be “guinea pigs”, we took the risk that we wouldn’t be harmed by the treatment. Since the medication helped all of us, wouldn’t it seem fair for the pharmaceutical company, Novartis, that manufactures it and is the richest drug company in the world to pay for the 12 original “guinea pigs” whose quality of life was so poor that they were willing to try anything? Where is the promised compassionate care?
Where is the knight on the white horse?
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