The answer right now is an absolute NO. I do not recommend starting Tasigna (Nilotinib) off-label at this time, unless the administering medical team included a neurologist, oncologist and cardiologist. This is a drug used for the treatment of CML cancer. It is not one to be used haphazardly. The good news is that the drug has been used for many years now as a cancer treatment, so we have very good safety/side effect data, and there is no reason to believe that the side effects would be worse at a lower dosage in PD patients. However, the most significant side effect, for which the FDA required Novartis to issue a Black Box warning, is possible life-threatening heart problems that may lead to an irregular heartbeat and possible sudden death. This problem is particularly prevalent for people who have QT prolongation, a condition involving heart wave intervals and rhythms. You need to be properly monitored before and during use. There are also possible interactions with other drugs, including MAO-b inhibitors, as well as with low blood potassium or magnesium. My point is that this is not a drug to take casually. It is much more serious a consideration than one may have, for example, in using Isradipine off-label.

That all being said, I believe the Georgetown phase I proof-of-concept study certainly indicates the need for further research. Although the study was small, poorly designed and open label, there was more than enough positive results to suggest the drug has symptomatic benefits, and possibly interventional disease modifying benefits. This is particularly true with the biomarker data, which you wouldn't think would be influenced to any great extent by a placebo effect. The results on the 6 month change in total UPDRS scores were significant, although certainly somewhat erratic. Even to John's point regarding what he quoted as a relatively small increase in UPDRS score was important. The reference he is using about needing a five point change in score to have practical significance for a patient is one that is used for symptomatic treatments, and wouldn't necessarily be the case for a longer term study of an interventional treatment. Keep in mind, the scores for these patients would have been anticipated to INCREASE by approximately 5 points over the 6 months (based on comparable results from the PPMI study for mid to late stage patients). In fact, it would be significant information even if there is no change in UPDRS over a long time period, which would imply the slowing or stopping of progression.

So, in the whole, the study results were certainly strong enough to support a PWP making the decision to go into the next phase of the research, if not actually take the drug unmonitored and off-label. What i would suggest at this time is wait for the larger, multi-centered trial, funded by MJFF/CPT/Van Andel, to get underway next year. If you qualify under the inclusion criteria, then you can get the drug for free with proper monitoring, while helping the whole PD community by being a trial volunteer.