WE WERE disappointed that the tone of the Stat article “The patients wait” further spread the unwarranted hype about nilotinib. For example, nilotinib is referred to as “the most promising new treatment for Parkinson’s disease in decades,” and in the online version, videos are shown of a patient before and after treatment with this drug.
The published study involved only 12 patients, without a placebo group. The placebo effect is strong in Parkinson’s disease, and so no conclusions regarding symptomatic benefits can be made from the doctors’ and patients’ reports of clinical benefits. Parkinson’s disease patients have many reasons for hope, but exaggerating the significance of early clinical data is counterproductive. These words are not without consequences. Some doctors are now prescribing nilotinib to Parkinson’s disease patients despite a lack of sufficient data regarding efficacy or long-term safety in these patients.
On the other hand, nilotinib is indeed worthy of further study. The focus should be on how to move forward with the best-designed, scientifically rigorous placebo-controlled study as a step toward more definitively determining whether or not nilotinib will be of benefit for Parkinson’s disease patients.
The writers are members of the Linked Clinical Trials scientific committee established by the Cure Parkinson’s Trust, have received funding from the Michael J. Fox Foundation, and conduct clinical trials for Parkinson’s disease.
https://www.bostonglobe.com/opinion/letters/2016/09/04/hype-about-parkinson-drug-unwarranted/oCRWpsaqYzDaHIWI3Y0rQL/story.html
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