GUMC Parkinson’s Trial Shows Promise

GEORGETOWN UNIVERSITY Movement Disorders Program Director Fernando Pagan co-led the successful GUMC clinical trial for a drug to reverse symptoms of Parkinson’s disease.

Movement Disorders Program Director Fernando Pagan co-led the successful GUMC clinical trial for a drug to reverse symptoms of Parkinson’s disease.

Two Georgetown University Medical Center researchers have discovered a drug that could reverse symptoms of Parkinson’s disease, including cognition and motor function loss.

Georgetown’s Laboratory of Dementia and Parkinsonism Director Charbel Moussa and Movement Disorders Program Director Fernando Pagan led the clinical trial. Twelve patients with Parkinson’s disease received nilotinib, a drug normally used in the treatment of leukemia.

One initial participant had to drop out for unknown reasons, but all 11 patients who completed the trial saw improvements. After treatment, one wheelchair-bound patient could walk on his own and three patients could hold conversations again.

Moussa said the trial has potential to provide new insight into treating Parkinson’s disease. Currently, there is no approved treatment method that can reverse the condition’s symptoms.

“The success of this approach will provide a disease-modifying approach that no other current Parkinson’s medication provides,” Moussa wrote in an email to The Hoya. “All current therapies are for symptom management and do not alter or slow down disease progression, which is a critical goal.”

Pagan agreed and was optimistic about their findings.

“I’ve been treating Parkinson’s patients for over two decades now and I’ve never seen a Parkinson’s drug that improves both motor and non-motor symptoms at the same time the way this drug has, especially in the advanced patients that we were treating,” Pagan wrote in an email to The Hoya. “I’m extremely excited.”

According to Moussa, the drug must go through a series of further tests before it can be prescribed to treat Parkinson’s. The drug will go through two more trials, which will be larger, placebo-controlled, double-blind trials, meaning neither the patients nor the researchers will know if a patient is receiving nilotinib or a placebo. Once this process is complete, the drug can be prescribed for treatment in the next few years.

“If funding becomes available and early data hold out in larger clinical trials, this drug may be clinically available five years from now,” Moussa wrote.

Moussa originally got the idea to use nilotinib to treat Parkinson’s by looking for drugs that regulate a process called autophagy, or the process by which cells destroy themselves. The nilotinib drug normally uses this process to kill cancerous cells from leukemia.

“In neurologic diseases, the cells, called neurons, accumulate toxic proteins which kills the cell — and that’s not good,” Moussa wrote. “We thought if we could turn on the autophagy process for a short time by giving a lower dose of nilotinib, it would lead to the digestion of intracellular neurotoxic proteins and prevent neurodegeneration and disease progression without killing the cell.”

While the drug is normally used in 800-milligram doses to treat leukemia, it was given in 150 to 300 milligram doses in the clinical trial for Parkinson’s disease.

Moussa explained that the drug appears to reverse Parkinson’s by taking advantage of this autophagy process. The drug induces the degradation of brain toxic proteins that are associated with dementia and Parkinsonism, while increasing levels of brain neurotransmitters such as dopamine. Dopamine is known to underlie many brain functions, including movement and mental activity.

The results from the clinical trial appeared to follow this observation.

“The changes in these biomarkers — Tau, p-Tau, α-synuclein and Abeta-40 and 42 — in spinal fluid suggest to us that the drug is working by triggering cells to clean out the toxic proteins in the brain,” Moussa wrote.

For Alan Hoffman, a retired social sciences professor at Georgia State University who was diagnosed with Parkinson’s in 1997, the effect of the drug proved life-changing. Hoffman started noticing improvements three weeks after beginning the clinical trial.

“I didn’t notice it but everybody else did,” Hoffman said. “I found that it covers up my deficiencies in enunciation and fluency of words … and I thought, ‘Well gosh, the only thing that I’m doing different is taking those pills.’”

Hoffman continued to improve as the trial progressed. In a walking therapy activity, he was able to walk 20 feet twice as fast, going from taking 28 seconds before the trial started, to 14 seconds two months after the trial started. The most noticeable impact, however, was on everyday tasks.

“I find I was able to do a lot of things I would not normally attempt, like emptying the dishwasher, doing lawn and yard work,” Hoffman said. “I found that sometimes I did so much and felt so healthy, that the next day I’d be worn out. I have worked a normal day instead of just a Parkinson’s day.”



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  1. Jerry Tevrow says:

    This is an extremely exciting possible breakthrough!
    Should the upcoming clinical trials prove successful, all patients
    suffering from Parkinson’s, their families and caregivers would receive
    the ultimate gift, of obtaining quality of life.

  2. This is great news! Could you test it on MSA patients? Is there anyway the drug could be fast tracked? Five years is longer than some have to live and longer than most have to live a productive life.

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