Parkinson's patients show improvements in clinical trial of new drug

- Georgetown University researchers say results from a small trial testing of a Food and Drug Administration-approved leukemia drug on patients with Parkinson’s disease are dramatic.

They say for the first time, they have found a drug that not only improves Parkinson’s symptoms, but it actually appears to reverse progression of the disease as well.

Before 88-year-old Mary Leigh started a clinical trial at Georgetown University this year to test the new drug to treat her advanced Parkinson’s disease, she struggled to speak, feed herself and control her arms, legs and hands.

After taking part in the six-month trial of the leukemia drug known as nilotinib or Tasigna, the McLean, Virginia woman is now able to feed herself.

“That’s big for her life because before that, she couldn’t do that,” said Leigh’s daughter, Elizabeth. “So she's engaging with the world. That’s really big.

“Everything kind of perked up. Her focus got better, her speaking ability got better, her motor skills got better. Communicating with more clarity where before she couldn’t really communicate at all.”

Georgetown University researchers say all eleven patients who completed the trial of the drug saw meaningful improvements in their Parkinson’s disease. Many were able to walk, talk and grip objects again -- skills they had lost long ago.

“All of a sudden this clear sentence came out with a very pertinent question and then she talked about minimum wage and I was like I have my old mom back,” said Elizabeth. “That's pretty amazing.”

The results were life-changing for Alan Hoffman of Dumfries, Virginia. His cognition and speech improved, and in tests at the hospital, his walking speed doubled.

Doctors say the drug clears out toxic proteins around brain cells. They caution more research is needed for this disease and others like Alzheimer’s, but the findings are exciting.

“We hypothesize that this may slow down progression of disease and we hypothesize that maybe we could potentially stop it in humans if we give it very early,” said Dr. Fernando Pagan, associate professor in the Department of Neurology at Georgetown University Hospital and co-director of the Movement Disorders Program.

Elizabeth Leigh said the results are dramatic, but not always consistent. She said her mother still has good and bad days, but the medication makes her hopeful.

“It's exciting because you are able to reverse the progression so it's incredibly helpful and I feel very optimistic,” she said.

Elizabeth added, "I don’t know what miracles are. I'm just very happy this drug exists. But it’s really important and it's significant.”

Hoffman and other patients in the clinical trial can stay on the drug thanks to an expanded access study. Researchers say they are now planning larger clinical trials for early next year in the hopes the drug could be available to the public in as little as three to five years.

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