Nilotinib, a blood cancer medicine, has long been considered for its potential to help Parkinson’s patients. Lab and animal studies suggested that the drug could protect dopamine-producing cells in people. But its toxicity made researchers nervous. Georgetown University lead a small safety trial using a small dosage of Nilotinib to test safety on PD patients. The results exceeded all expectations.
Nilotinib appeared to double patients’ dopamine production, reduce the death of dopamine-producing nerve cells, and clear from those cells a toxic protein associated with Parkinson’s and a related disorder, dementia with Lewy bodies. Relief from symptoms seemed equally striking among the 11 patients who completed the trial (conducted at Georgetown).
Phase II trials of the most promising new treatment for Parkinson’s disease in decades might be delayed because of a feud between a key scientist and the influential Michael J. Fox Foundation, which donates more money to Parkinsons than all other donators combined.
The trial offers Moussa, a little-known scientist, a chance to vault into the top ranks of researchers, while Fox and its collaborators could reinforce their standing and tap into a bonanza of donations from wealthy philanthropists and worried patients.
The episode also highlights the tension between scientists and foundations that are no longer content simply to dole out money. Following the lead of the colossal Bill & Melinda Gates Foundation, major medical research philanthropies increasingly seek to coordinate or manage studies, or control details of how they are done — vexing many grant recipients.
This is an important development for PD and I am hoping that we get to Phase II Trials quickly.