Nobel Prize-winning Science is Key to Georgetown Neurotherapeutic Research

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Karen Teber

WASHINGTON (Oct. 3, 2016) — Today, the 2016 Nobel Prize in the category of medicine or physiology was awarded to Yoshinori Ohsumi “for his discoveries of mechanisms for autophagy,” a fundamental process for degrading and recycling cellular components.

When autophagy (a process also described as cellular “self eating”) doesn’t work properly, cells cannot empty its trash. 

In describing autophagy, the prize committee says, “Mutations in autophagy genes can cause disease, and the autophagic process is involved in several conditions including cancer and neurological disease,” and it says, “Intense research is now ongoing to develop drugs that can target autophagy in various diseases.”

Some of that work is well underway at Georgetown University Medical Center.

Researchers led by Charbel Moussa, MBBS, PhD, scientific and clinical research director for Georgetown’s Translational Neurotherapeutics Program, are examining autophagy and developing therapeutics to jump start the process when its failure causes neurodegenerative diseases including Alzheimer’s and Parkinson’s diseases.

Preclinical studies, led by Moussa, showed that nilotinib, a tyrosine kinase inhibitor, effectively penetrates the blood-brain barrier and destroys toxic proteins that build up in Parkinson’s disease and dementia by turning on autophagy inside neurons.

Based on that work, Moussa and his colleague Fernando Pagan, MD, conducted a pilot study in people with Parkinson’s disease that provided molecular evidence that nilotinib, an FDA-approved drug for leukemia, significantly increased brain dopamine and reduced toxic proteins linked to disease progression in patients with Parkinson’s disease or dementia with Lewy bodies.

This work continues.  Georgetown has announced the FDA has completed its review of an investigational new drug application (IND) for the use of nilotinib in a phase II clinical trial for patients with mild to moderate Alzheimer’s disease.  A phase II study in Parkinson’s is also being planned. 

Moussa describes the selection of Yoshinori Ohsumi for his work in autophagy as “Amazing,” noting that the Nobel prize committee acknowledged the role of autophagy in neurodegenerative diseases and its mention of autophagy as a way of treatment. 

Moussa is an inventor on an allowed US patent and other pending US and foreign patent applications for use of nilotinib and other tyrosine kinase inhibitors for the treatment of neurodegenerative disease.

Read more about Moussa’s work in autophagy and neurotherapeutics here.