­​​​​​​​Metabolite Treatments for Metabolic Dysfunction and Neurological Disease Caused by Mutations in Mitochondrial Enzyme GPT2



The mitochondrial enzyme glutamate pyruvate transaminase 2 (GPT2) is necessary for metabolic mechanisms central to neuronal survival and axonal growth. Having demonstrated that loss-of-function mutations in GPT2 cause neuronal vulnerabilities involved in a variety of brain diseases and areas of brain function, we have developed a metabolite treatment for GPT2 disease and related clinical problems.

Market Opportunity

Diseases caused by loss-of-function mutations in GPT2 include GPT2 disease and motor diseases such as amyotrophic lateral sclerosis (ALS), while areas of brain function affected include those damaged in Alzheimer’s disease and diminished in states of sleep deprivation. There is currently no treatment for GPT2 disease, and few effective treatments for other diseases involving GPT2-mediated mechanisms, such as ALS and Alzheimer’s disease.

Innovation and Meaningful Advantages

We have developed a metabolite treatment for GPT2 disease and related clinical problems. Our dietary supplementation consists of combinations of alanine and triheptanoin and/or other anaplerotic supplements. This treatment may also be useful for delaying disease onset and progression in Alzheimer’s disease. In addition, it may be effective in enhancing attention and wakefulness in shift workers and others who need to remain alert and attentive over extended hours, such as health care providers, military personnel, and pilots. We plan to develop biomarkers based on a combination of plasma metabolite tests and pupillometry.

Collaboration Opportunity

We are interested in exploring 1) startup opportunities with investors; 2) research collaborations with leading pharmaceutical companies; and 3) licensing opportunities with companies.

Principal Investigator

Eric M. Morrow, MD, PhD

Mencoff Family Professor of Biology, Professor of Neuroscience, Professor of Psychiatry and Human Behavior

Brown University



IP Information

US Utility Filed, Priority Date: May 15, 2020


Baytas O, Davidson SM, DeBerardinis, and RJ, Morrow EM. Mitochondrial enzyme GPT2 regulates metabolic mechanisms required for neuron growth and motor function in vivo. Human Molecular Genetics. 2022 Feb 15;31(4):587-603. doi.org/10.1093/hmg/ddab269.

Ouyang Q, Nakayama T, Baytas O, et al. Mutations in mitochondrial enzyme GPT2 cause metabolic dysfunction and neurological disease with developmental and progressive features. Proc Natl Acad Sci U S A. 2016;113(38):E5598-E5607. doi:10.1073/pnas.1609221113


Andrew Bond, PhD

Senior Director of Business Development,


Brown Tech ID 3040

Patent Information:
For Information, Contact:
Brown Technology Innovations
350 Eddy Street - Box 1949
Providence, RI 02903
Eric Morrow
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