The Top Five Questions and Answers about Nilotinib (a cancer drug) for Treatment of Parkinson's disease
Recently there was a rush of Parkinson’s patients getting in line to try a cancer drug (Nilotinib/Tasigna) in an attempt to delay or to treat their Parkinson’s disease symptoms. We have been receiving a ton of questions on this topic and we have complied these into the 5 things you need to know about this drug for Parkinson’s disease.
Nilotinib is used to treat Leukemia. What does it mean to “repurpose” this drug for Parkinson’s disease?
There are many drugs which are already approved by a regulatory agency, such as the U.S. FDA. In many cases, we do not know if these drugs (e.g. cancer or bowel disease) could be repurposed to treat Parkinson’s disease. Researchers in many prominent laboratories including at Georgetown and John’s Hopkins have been interested in a pathway in the brain called c-abl. These researchers believe that there is a possibility that through inhibition of this c-abl pathway it may lead to improvement in Parkinson’s disease symptoms or even slow its progression. Most of the early successes with the approach were in animal models. There are notably many c-abl inhibitors and Nilotinib is just one.
2. How/Why do c-abl inhibitors possibly work for Parkinson’s?
c-Abl also known as ABL1 and Abelson tyrosine kinase is a member of Abl family of non-receptor tyrosine kinases. Higher levels of the c-Abl protein have been shown to be activated in Parkinson's disease.
There are many theories about how and why a c-abl inhibitor strategy may work in Parkinson’s. Here are just a few:
Cell signaling adaptors to kinases and phosphatases
Regulation of cell growth and survival
Cell migration
Synapse formation
Neurite outgrowth
Neurogenesis
Reduce the loss of PARKIN function (an important protein in Parkinson’s disease)
Reduce apoptotic neuronal death
3. There were two major trials recently completed on Nilotinib and Parkinson’s disease. What were the results?
The first trial was published in JAMA Neurology. The authors commented they thought the drug was reasonably safe, but that there were more side effects for those taking Nilotinib than in the placebo group. There was not evidence for a strong clinical effect. This was a small single center trial.
A second large trial (25 sites) was recently performed and a press release from the Parkinson Study Group suggested the study was ended early because it showed a lack of effect. Nilotinib was safe and tolerable but did not “exert a clinically meaningful benefit or biological effect to benefit those with Parkinson's disease.”
A full summary is on the Parkinson’s Foundation website.
4. If I have Parkinson’s disease should I take Nilotinib to prevent progression or to treat my Parkinson’s disease?
Based on what information we have available from animal and human studies we do not recommend patients with Parkinson’s disease take Nilotinib (Tasigna).
5. Is it possible that other c-abl inhibitors may be repurposed for Parkinson’s disease?
Yes, there is still ongoing research at many top laboratories and as we learn more results, we will keep the Parkinson’s community updated.
To read more books and articles by Michael S. Okun MD check Twitter @MichaelOkun and these websites with blogs and information on his books and http://parkinsonsecrets.com/ #EndingPD and https://www.tourettetreatment.com/
