Is Tasigna (Or Nilotinib) Worth Testing For Parkinson's? : Shots - Health News A leukemia drug seemed to help patients with Parkinson's disease. But critics say the results are equivocal and could raise false hopes.

A Cancer Drug For Parkinson's? New Study Raises Hope, Draws Criticism

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Researchers say a cancer drug has cleared another hurdle as a potential treatment for Parkinson's disease. If the drug succeeds, it would be the first to slow down Parkinson's rather than just reducing its symptoms. NPR's Jon Hamilton reports skeptics still have doubts about the unusual treatment.

JON HAMILTON, BYLINE: The drug is called nilotinib, and it's used for a type of leukemia. But a team at Georgetown University Medical Center has been studying nilotinib as a way to treat Parkinson's and other brain diseases. Dr. Fernando Pagan says it works like a garbage disposal to clear out toxic substances that build up in brain cells.

FERNANDO PAGAN: You turn on the garbage disposal daily, and you're able to get rid of that accumulation and hopefully see better function.

HAMILTON: Preliminary results looked encouraging, so the team launched a study of 75 Parkinson's patients. Some got relatively low doses of nilotinib, which is sold under the brand name Tasigna. Others got a placebo. Pagan says the results show that the drug is reasonably safe for these patients and may even be slowing down the disease.

PAGAN: This drug is still not ready for prime time. I think there's still a lot more work to be done, but we are seeing signals that this may be a potential treatment for our Parkinson's patients.

HAMILTON: Patients who got the drug had lower levels of two toxic substances in their spinal fluid. They also reported a better quality of life. And Georgetown's Charbel Moussa says the drug seemed to increase dopamine, the brain chemical that is lacking in people with Parkinson's.

CHARBEL MOUSSA: Nilotinib is increasing the availability of the stored dopamine in the brain. So the brain is now extracting its own endogenous stores of dopamine.

HAMILTON: The results appear in the journal JAMA Neurology, and they represent the latest exchange in an ugly scientific conflict over the use of nilotinib for Parkinson's. The Michael J. Fox Foundation for Parkinson's Research has publicly criticized the Georgetown research, even as it was conducting its own study of the drug. And earlier this month, the foundation announced that nilotinib didn't help Parkinson's patients in their study, even though the study results hadn't been published.

Moussa, who holds a patent on the use of nilotinib for treating brain diseases, says patients should ignore the scientific infighting.

MOUSSA: This may fail. It's OK. But I think the concept is very feasible. And if this drug doesn't work, another drug will work.

HAMILTON: But scientists, including Dr. Joel Perlmutter of Washington University in St. Louis, have doubts about the study.

JOEL PERLMUTTER: They really didn't find anything convincing.

HAMILTON: Perlmutter says some results look good at one time point, but not another. And he says it's not clear whether the dopamine changes were caused by nilotinib.

PERLMUTTER: It's possible these are really statistical aberrations and are not really convincing evidence of a change induced by the drug. But we don't know.

HAMILTON: Perlmutter says he's also concerned about side effects, which were more frequent in people who got the drug. He says it will take a much larger study to show whether nilotinib really can help people with Parkinson's.

Jon Hamilton, NPR News.

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