Team Led by McLean Hospital Researcher Uncovers Potential Parkinson’s Treatment

July 22, 2015

Kwang-Soo Kim, PhD, director of McLean Hospital’s Molecular Neurobiology Laboratory and professor of psychiatry at Harvard Medical School, along with collaborative teams from Brandeis University and Nanyang Technological University (NTU) in Singapore, recently announced the findings of a multi-year research project that shows that existing anti-malaria drugs may have the ability to treat Parkinson’s disease. This study was published in a recent issue of Proceedings of the National Academy of Sciences of the United States of America (PNAS) online.

Parkinson’s disease (PD) is the most prevalent movement disorder, affecting more than five million people worldwide. At present there are no available treatments that can stop or slow down the progress of the disease, and while currently available treatments can help relieve symptoms, there is no cure.

Kwang-Soo Kim, PhD
Dr. Kim in his lab at McLean Hospital

After screening more than 1,000 drugs approved by the U.S. Food and Drug Administration (FDA), the researchers discovered that two common anti-malaria treatments—chloroquine and amodiaquine—could bind and activate a protein in the brain vital to fight Parkinson’s. This protein, called Nurr1, is essential for development and maintenance of dopamine neurons, which are essential to the body’s ability to move muscles. Parkinson’s disease disrupts the production of dopamine neurons and progressively causes the loss of motor control.

“Backed by various lines of scientific evidence, Nurr1 is known to be a potential drug target to treat Parkinson’s. Despite great efforts from pharmaceutical companies and academia, no one has managed to find a molecule which can directly bind to it and activate it, except for us,” said Kim, senior author of the study.

In laboratory tests, the researchers found that by activating Nurr1, rats with Parkinson’s disease appeared to improve and showed no signs of side effects.

According to Kim, current treatment for PD is aimed at replenishing dopamine levels via pharmacological or surgical treatment, but while these methods can improve mobility functions and treat symptoms in the early stages, they are unable to slow or halt the disease.

And Kim has seen firsthand how desperate Parkinson’s sufferers, and those who love them, are for a cure. “I never imagined that I would receive so many calls and emails from patients, family members and reporters,” he said. “I fully understand the excitement around this finding, but we need to be cautious and conduct more research to determine the efficacy of these medications.”

Both chloroquine and amodiaquine are approved by the FDA and have been used to treat malaria infections. Amodiaquine was used in the late 1940s to early 1950s, until the malaria parasite grew resistant and its significant liver toxicity was known while chloroquine is still being used in Africa today.

The research team still plans to look into additional drug combinations that might halt or slow the progression of PD and noted their intent to modify the existing formulations of chloroquine and amodiaquine for human clinical trials, which they hope to begin soon.

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