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New therapeutic strategies for protecting the nervous system

Conquest - Summer 2013

Neurodegenerative Consortium

By Hilary Graham

Phillip Jones, Ph.D., Giulio Draetta, M.D., 
Ph.D., and Ming-Kuei Jang, Ph.D. (from 
left to right), discuss research directions 
and drug discovery strategies that will 
lead to the development of novel neural 
protective therapeutics.
Photo: F. Carter Smith

In 2011, MD Anderson received what seemed an unusual gift — $25 million — from the Belfer Family Foundation to establish the Neurodegeneration Consortium. As a challenge gift, the donation must be matched to be realized.

The aim is to better understand the underlying biology of Alzheimer’s disease and translate this knowledge into therapeutic interventions that can effectively alleviate symptoms by delaying, reversing and/or eliminating the pathology of the disease.

The multidisciplinary team includes researchers from Baylor College of Medicine, the Massachusetts Institute of Technology and MD Anderson’s Institute for Applied Cancer Science.

So, one might ask, why is a cancer center an appropriate place for this kind of scientific research? Here is why.

Alzheimer’s is a neurodegenerative disorder of the central nervous system associated with progressive and severe brain atrophy and damage to the neurons. To date, researchers do not know exactly what causes the disease or when onset will occur.

In contrast, chemotherapy-induced neurodegeneration occurs in the peripheral nervous system and the onset is predictable.

While chemotherapy effectively kills cancer cells, it also inflicts collateral damage on normal cells, including neurons. Long after chemotherapy is completed patients can be left with neuropathy, the debilitating side effect that can cause tingling, numbness and pain.

“The large number of MD Anderson patients who suffer from this side effect provide an opportunity for collaboration between their physicians and researchers to better understand the underlying biology associated with neuropathy,” says Ronald DePinho, M.D., MD Anderson’s president. “Such a discovery could lead to the development of prevention and treatment strategies.”

What can be discovered through those suffering from chemotherapy-induced peripheral neuropathy (CIPN) could also hold significant answers for Alzheimer’s.

Innovative patient-centered research 

After extensive analyses, the consortium developed a program that focuses on helping the body better protect itself by engaging innate protective mechanisms — so that the brain and neurons can resist toxic insults. Therapeutics derived from this approach would benefit patients with Alzheimer’s, other neurodegenerative diseases such as ALS, Huntington’s and Parkinson’s, as well as cancer patients on chemotherapy.

After extensive preclinical studies are completed, the effectiveness of new therapies will be evaluated in Alzheimer’s and CIPN patients. Because the disease mechanisms and onset are known for CIPN, successful trials in this patient population would provide proof of principle for the neuroprotective strategy.

The consortium hopes to demonstrate that neuroprotective compounds can provide therapeutic and protective effects, which would significantly improve the quality of life for MD Anderson’s patients, as well as for those with Alzheimer’s.

Neuroprotection is an example of the innovative patient-centered research and drug discovery programs that the consortium supports. The consortium will pursue unbiased programs that provide maximum benefit for those with neurodengenerative disorders.

See MD Anderson’s online Newsroom and Cancer Frontline for more information on these and other basic, translational and clinical research findings at
MD Anderson.

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