Researchers at Johns Hopkins say they have gleaned two important new clues in the fight against Parkinson's disease: that blocking an enzyme called c-Abl prevents the disease in specially bred mice, and that a chemical tag on a second protein may signal the disorder's presence and progression. Their work, described online June 27 in The Journal of Clinical Investigation, suggests both a promising target for drug research and a tool that could speed Parkinson's disease research more broadly, they say.

There were indications that c-Abl activity leads to Parkinson's disease, and our experiments show there is indeed a connection," says Ted Dawson, M.D., Ph.D., professor of neurology and director of the Institute for Cell Engineering at the Johns Hopkins University School of Medicine. "There is already a Food and Drug Administration-approved c-Abl inhibiting drug in use for leukemia," he adds, "so we're interested in whether it could be used safely against Parkinson's disease or as a starting point to develop other treatments.

.....Dawson and Ko caution that the use of the anti-leukemia drug nilotinib is not yet indicated for Parkinson's disease patients and that further studies are needed before their results can be applied to clinical care.

Blocking key enzyme halts Parkinson's disease symptoms in mice

I find it very frustrating that there is an approved drug on the market, which has been shown to be effective for treating PD, yet researchers can't get funding to conduct the required clinical trials. Yes, we all are aware of the many limitations associated with the research conducted at Georgetown. But, isn't this the way research is supposed to proceed. A small, proof of concept study shows some efficacy, which leads to, controlled trials. I can't understand why it's been so difficult to get funding. But, I intend to find out.