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MCL 3180.0: Treatment and Diagnosis of Diseases Characterized by Mitochondrial Dysfunction

Anne Cataldo, Ph.D., et al.

Background and Description

Many heritable diseases are caused by mutations in mitochondrial genes or nuclear genes encoding mitochondrial proteins. In addition, environmentally-induced mutations in mitochondrial DNA have been implicated in many common acquired disorders. Among the disorders involved in mitochondrial dysfunction are neuropsychiatric diseases such as bipolar disorder, depression, and schizophrenia; and neurodegenerative diseases like Alzheimer's disease, Parkinson's disease, Friedreich's ataxia; amyotrophic lateral sclerosis, and Huntington's disease. Treatment of such diseases has been attempted using transplanted tissues or cells, but such attempts can be complicated by the necessity of replacing diseased tissue rather than improving the function of existing cells and tissue. The present invention provides a therapeutic approach to overcoming mitochondrial dysfunction through mitochondrial replacement, without the replacement of existing cells. The viability of the method has been demonstrated in vitro, in experiments where mitochondria from adult stem cells were successfully transplanted into cells from bipolar disorder patients and were capable of normalizing the abnormal mitochondrial structure that is common in bipolar disorder.

Potential Commercial Uses

The invention features methods and kits for treatment of disorders associated with mitochondrial dysfunction, in which healthy mitochondria (e.g. from stem cells or other sources) are transplanted into diseased cells in the brain. The invention provides potentially powerful cell therapy strategies to treat brain disorders in situ without the replacement of existing cells, and offers the possibility that preparations of purified mitochondria could someday become an off-the-shelf product for therapeutic use. The invention also features methods of diagnosing neuropsychiatric and neurodegenerative disorders based on mitochondrial structural abnormalities.

Publication and Patent Status

McLean Hospital is the owner of a pending U.S. patent application claiming this invention. Aspects of this research have been published as Cataldo, et al. Am. J. Pathol. 177(2):575-585 (2010).

Licenses Available

McLean Hospital is offering a worldwide exclusive license to this technology.

For more information, please contact:

David J. Glass, Ph.D.
Senior Associate Director, Technology Transfer
McLean Hospital Research Administration
115 Mill Street
Belmont, MA 02478-9106
(617) 855-3825 - tel
(617) 855-3745 - fax