The prospect that some still-unapproved drug will provide a significant advantage for a serious condition that is not responding well to current treatments is understandably appealing. Cancer patients are most susceptible to this kind of unproven promise, but those with nervous system disorders such as Alzheimer’s disease, multiple sclerosis or Parkinson’s disease are also anxious to find better treatments than those that are currently not doing much for them. This reader is interested in a possible treatment for Parkinson’s disease.
A New Treatment for Parkinson’s Disease?
Q. I have Parkinson’s disease (PD) and it is getting progressively worse. My hands shake so much that it is almost impossible to hold a cup without spilling its contents.
Given the recent study using reduced doses of the FDA approved anti-leukemia drug nilotinib to successfully treat PD, should I ask my neurologist to prescribe it off-label?
A. Nilotinib (Tasigna) is a leukemia medicine that has shown promise in a pilot study of 12 people with Parkinson’s disease. Some demonstrated impressive improvement, but the research is still in its early days.
Off-Label Prescribing:
Your neurologist could legally prescribe Tasigna for Parkinson’s disease, but most physicians are cautious about prescribing unproven medications. Given the very limited experience with this drug for Parkinson’s disease, we think that caution is justified.
It would be worth a conversation, though. Perhaps your doctor could refer you for another clinical trial, or maybe he or she is aware of other treatment options that could possibly offer you some benefit.
What Would Tasigna Cost?
Although the dose for Parkinson’s disease was much lower than the dose for leukemia, Tasigna would still be expensive. Cancer treatment with Tasigna costs over $10,000 a month. Insurance rarely covers off-label prescriptions.
Potential Side Effects:
One reason a doctor might be reluctant to prescribe such a medication off-label is the possibility of serious side effects. We don’t know whether the people in the pilot study experienced serious complications from taking low-dose Tasigna for six months. Used in higher doses for cancer, Tasigna can cause rash, elevated blood sugar, headache, blood disorders, digestive upset, fatigue, high cholesterol, fever and painful joints, along with other side effects.
