UNMC RESEARCHERS IDENTIFY BETTER-TOLERATED THERAPY FOR PARKINSON'S DISEASE

OMAHA- A team of University of Nebraska Medical Center researchers has shown that a smaller dose of a drug repurposed as a Parkinson’s disease treatment produced fewer side effects than a larger dose while keeping disease signs and symptoms stable.

Now, the researchers are “incredibly well-positioned” to seek approval from federal regulators for a larger study, said Dr. Howard Gendelman, chairman of UNMC’s pharmacology and experimental neurosciences department and one of the lead researchers.

The study builds off a 2016 trial in humans in which the researchers used the drug to shift a population of white blood cells from a destructive mode to a protective state that can help defend parts of the brain from injury.

For the new trial, the researchers, working with drugmaker Partner Therapeutics of Lexington, Massachusetts, halved the original dose and followed five patients for a year. The drug, called sargramostim, or Leukine, sometimes is used in patients receiving chemotherapy.

The patients tolerated the drug and took it safely for a year. In addition, signs and symptoms of the disease, rather than progressing — as usually is the case with Parkinson’s — remained stable.

The results of the study were published recently in EBioMedicine, an online Lancet journal. Because the data proved promising, the study was extended to 24 months and enrolled additional patients.

Currently, drugs and other therapies can be used to fight the symptoms of Parkinson’s, but they don’t treat the disease itself.

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