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Brain, Brain Heal Thyself


by Joe Harding and Leen Kawas, Washington State University

By 2034, there will be an effective treatment for diseases like Parkinson’s and Alzheimer’s.   This will represent a new class of regenerative drugs that restore lost function and prevent further deterioration – a significant departure from current treatments that aim simply to slow disease progression, not fix what’s broken.

Why do we think this enormous leap forward in medical science is possible when it’s only been 20 years since the concept of “regenerative medicine” was introduced as a targeted branch of medical science? Recent laboratory research has revealed a regenerative therapeutic pathway that holds the potential to reverse the course of neurodegenerative diseases.

The implications are enormous. Millions of Americans currently live with these debilitating and deadly diseases. The emotional and financial tolls are staggering. The medical costs associated with Alzheimer’s disease alone are estimated to be more than $200 billion a year.

The key to unlocking this medical future lies in the ability to control growth factor proteins in the body that orchestrate the development of the nervous system, guide changes in the brain associated with learning and memory formation, and, most critically, support repair and recovery when the nervous system is injured. The activity of most growth factors is governed by a “switch,” which when turned on, initiates cellular regeneration and, when turned off, inhibits cellular growth and induces cell death. These proteins just need to be revved up to accelerate and enhance their inherent regenerative capacity.

Research conducted at Washington State University and being commercialized by M3 Biotechnology has resulted in a flexible technology that guides the design of inexpensive small molecule drugs that can either activate or inhibit growth factor function. Proof-of-concept studies using this approach have enabled our team to activate a growth factor in the brains of rodent models of Parkinson’s disease and dementia, resulting in a dramatic restoration of motor and cognitive function.

The most compelling aspect of this new technology is that this switch can be turned on and off by M3’s small molecule drugs, which in the case of neurodegenerative disease results in the activation of critical growth factors and the stimulation of regenerative activity. On the flip side, small molecule growth factor inhibitors have proven to be potent anti-cancer agents where growth factor over-activation is nearly universal.

Given time and continued investment in its development, this technology has the potential to revolutionize treatment for an array of diseases.  Alzheimer’s and Parkinson’s are just the beginning.

Joseph W. Harding, PhD, is a professor in the Department of Integrative Physiology and Neuroscience at Washington State University and co-founder of M3 Biotechnology, Inc. He is a pharmacologist and peptide chemist working on growth factor targeted drugs for the treatment of various neurodegenerative diseases and cancers. His work has been supported by the National Institutes of Health, National Science Foundation, Michael J Fox Foundation, Alzheimer’s Drug Discovery Foundation, Life Sciences Discovery Fund from the state of Washington, Murdock Foundation, American Heart Association, National Multiple Sclerosis Society, Department of Defense, and other funding sources.

Leen H. Kawas, Ph.D, is president and CEO of M3 Biotechnology Inc. as well as co- inventor of the M3’s drug MM-201. Kawas also is an adjunct Associate Researcher in the Department of Integrative Physiology and Neuroscience at Washington State University. Kawas is a pharmacist and a pharmacologist with a focus on translational sciences specifically growth-factor therapeutics for the treatment of various neurodegenerative diseases and cancers. Her work has been supported by the National Institutes of Health, Michael J Fox Foundation, Alzheimer’s Drug Discovery Foundation, Washington State’s Life Sciences Discovery Fund and other funding sources.

If you want to make sure that we realize the benefits from this and other scientific advances, let your members of Congress know that federal funding for science matters and should be made a higher national priority. The Science Coalition’s Science Matters toolkit can help you get started.
Categories The Brain