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MCL 3182.0: Stem Cell Method for Treating Neurodegenerative Diseases

Anne M. Cataldo, Ph.D.

Background and Description

The remediation of neurodegeneration and cognitive decline in Alzheimer's Disease and other neurodegenerative disorders remains a challenge. Among methods that have been tried, with little success to date, are various methods of cell therapy, wherein cells would be implanted into the brain of affected patients in an attempt to overcome loss of functional neurons. Evidence in the literature suggests that adult bone marrow stem cells can transdifferentiate into different neuronal phenotypes. However, therapeutic methods involving marrow-derived multipotent adult progenitor cells (MAPCs) have not yet been worked out. The inventors have shown that the α-secretase-cleaved fragment of the amyloid precursor protein (sAPPα), a potent neurotrophic factor, can potentiate the nerve growth factor/retinoic acid induced transdifferentiation of MAPCs into neural progenitor cells. Specifically, MAPCs engineered to express sAPPα, when administered intravenously, cross the blood brain barrier and terminally differentiate into cholinergic neurons. MAPCs differentiated in the presence of sAPPα and NGF/RA exhibited a 40-75% increase in expression of neuronal markers compared to MAPCs differentiated by NGF/RA alone.

Potential Commercial Uses

The invention therefore comprises methods of treating neurodegenerative diseases by using MAPCs as vehicles of trophic factor delivery. Specifically, recombinant MAPCs, engineered to express sAPPα, can be administered to patients so that the MAPCs can differentiate into cholinergic neurons in the brain. The method is potentially useful for a variety of neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, and other diseases characterized by loss of neuronal function.

Publication and Patent Status

McLean Hospital is the owner of a pending U.S patent application claiming this invention. The research underlying the invention has been published in Chen et al. (2006) Curr. Alzheimer Res. 3(1):63-70.

Licenses Available

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

For more information, please contact:

Anne Ritter, Licensing Manager
Partners Research Ventures and Licensing
(617) 954-9529