The Study
We went to Dr. Pagan and over a period of two years, I was involved in a study using Apokyn (a rescue drug) and I received botox in my feet for rigidity. I had always been eager to be involved in trials and had participated in many in the Atlanta area. When I saw information about a clinical trial using Nilotinib, a drug now used for Leukemia, I asked about participating. Dr. Pagan’s only concern was the number of trips we’d need to make to Georgetown for the nine month trial. We assured him we could do that easily.
I was tested first to see if I was a candidate. Nilotinib can potentially cause irregular heartbeat, so an EKG was called for. Bloodwork was also done. Parkinson’s mental and physical testing was completed and I was told that I qualified.
Over the next six months, we made multiple trips to Georgetown. Each time an EKG was done to ensure that the QT interval was appropriate. Bloodwork was done to ensure that I had sufficient minerals and vitamins in my system, and three lumbar punctures were administered.
The study was labeled as a safety study to see if Parkinson’s patients could tolerate low doses of Nilotinib. The hope was that it would not only be safe, but also that alpha-synuclein in the spinal fluid and tau in the blood stream would be reduced. These are plaque forming substances that cause brain cells to die. Patients were randomly assigned to either a 150mg or 300mg dose of Nilotinib per day. Strict guidelines for timing the doses were adhered to. Everyone took medication-there were no placebos given.
The results were significant. All 11 people who completed the study improved. We walked, we talked, we thought with greater ease and clarity.
Dr. Pagan videotaped me in the “get up and go” walking test at the beginning of the study and every visit afterward. Soon, I was walking steadily in half the time of my original walk. My speech was louder and clearer, I was no longer falling, I was less confused about things in general, and my life was considerably better. I had always been an avid reader, but had not read a book in a long time. I just couldn’t concentrate enough. I first read the David McCullough book on the Wright Brothers and then progressed to others. I was thrilled!
The study consisted of taking six months of medication. Three months after the medication ended, I returned to Georgetown for testing again. I had begun to regress without the medication. During the trial, the pharmaceutical company, Novartis, made a verbal commitment to continue to provide the medication for no charge to the trial participants. That did not happen and resulted in much frustration on the part of all of us. We held out, hoping that Novartis would come though.
The study was reported at a conference of Neuroscientists on October 17, 2015 in Chicago.
In preparation for that conference, information from the Georgetown communications person was released to the media. (See attachment 1). Immediately, our phone began to ring asking us to be interviewed. We were interviewed by NPR, NBC, Fox News, Univision, TV Globo-Brazil, and the Daily Mail in London.
News services picked up the story and our daughter, Jennifer, googled me to tell me that I was mentioned in thousands of articles.
https://www.youtube.com/watch?v=Rifa6LPy60A
Then the phone really began to ring. Parkinson’s patients and/or their caregivers looked up our phone number and began to call. There was a great deal of interest in the process and especially in the results. We spoke to people in twelve states and Canada. They wanted to know all about my symptoms, duration of the disease, how I was doing, where we got medication, etc. etc. etc. In short, they came looking for hope and found it. We began to expect return calls from people we had talked with who were in the process of getting the medication, were just beginning it, and wanted us to anticipate when positive changes would occur for them.
When Novartis didn’t come through with their promise of providing medication by the time of the conference, we began to investigate costs. While Nilotinib is an FDA approved drug for cancer, insurance will not pay for it to be used to treat Parkinson’s disease. We found it at a pharmacy in Canada for $5321 for three months’ medication. While the costs are exhorbitant, we were blessed that my younger brother helped to pay for it. We ordered it and I began taking the medication again on November 22, four months since my last dose during the trial. During those four months, I had regressed a lot. It took about two months of medication to see positive changes. I believe that I will continue to improve as time passes.
Millions of people worldwide have Parkinson’s and/or Lewy Bodies Disease. If, in fact, Nilotinib/Tasigna, can continue to decrease the plaque that causes brain cell death, there will still be millions of people who do not have access to the drug
because of cost.
During the time I was off Nilotinib, I wrote a few articles to express myself about pharmaceutical companies. I will be sharing them as a part of this blog.
I am beginning my 9th month of medication. I have many more good days than bad days. I am hopeful that this medication will continue to help me feel better and better.
We understand that there is research going on in Israel and England that is focusing on something similar to Nilotinib , phage, that has similar results. The researchers are hoping to find a cure for Parkinson’s, Alzheimers, ALS. We pray that they will. Hopefully, I will still be alive to reap the benefits of the research. If not, though, I am heartened to think that millions of people may be spared the debilitating effects of a degenerative neurological disease.
Whenever you have a degenerative, debilitating disease, you become a member of another “family”. All of us are in this together. I have learned so much during my 19 years with Parkinson’s about finding your purpose in life, about building relationships with people you love, about kindness, about looking everywhere and not being afraid to try medications and trials and surgery and anything else that might help you or others who share the same burdens, about making your life count for SOMETHING. To paraphrase the educational psychologist Louis Raths, “to make a difference that you lived at all.”
I continued to grow. I was trying to leave no stone unturned. I remembered an audiotape of a speech by the late Leo Buscaglia in which he describes an old man with no teeth and dirty clothes sitting along the river in St. Louis. He was the kind of guy most people would pass by without noticing, but Leo, being Leo, was bored with speech after speech at the convention he was attending, so he sat down and engaged this man in conversation. Finally, Leo asked the man for the one important thing he’d learned in his life. The man said, “The secret to life is to keep your mind full and your bladder empty.” Buscaglia said that was the best advice he had received during the entire conference because…”you see, most of us do the exact opposite; we keep our bladders full and our minds empty.” (Source unknown)
I was awake for the first one- drilling a hole in my skull, using a camera to pinpoint where the contacts needed to be placed in my brain and clumping the wires at my hairline. The surgery didn’t begin until Dr. Okun came in. He was so excited to share with me that his 6 month old son had recognized himself in the mirror. He said, ‘that’s smart, isn’t it, Alan?’ I was afraid he might go back home to see more. HA! When Dr. Okun came out to tell my family that I had done well and they were almost finished and would be sending me to the recovery room, he reminded them that I might act “goofy”. Our middle daughter, Allison, said “how will we know?” After some days of recuperation at the Laurel Oak Inn (our home away from home that is owned and operated by the wonderful Monta and Peggy Burt), we went home to Atlanta to wait for a month for my brain to shrink (it swells when it is tampered with).
