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|Grant Enables Digestive Disorder Research|
Grant Enables Digestive Disorder Research
Tracy Grikscheit, MD, of the Saban Research Institute of Children’s Hospital Los Angeles, recently received a $7.1 million grant from the California Institute of Regenerative Medicine Translational Research program to develop a cellular therapy for the treatment of nerve disorders of the digestive system. These disorders, called enteric neuropathies, include degenerative neuromuscular conditions as well as those caused by a deficient or missing nerve supply to the intestines. Currently, in many cases the only treatment is removing segments of intestine that do not have a properly formed nervous system.
“Our goal is to develop an ‘off the shelf’ cellular therapy to treat enteric neuropathies before patients require surgery or to rescue patients who still have symptoms following surgery,” said Dr. Grikscheit.
Working with human induced pluripotent cells (iPS) that have the ability to develop into many human cell types, Dr. Grikscheit proposes to generate nerve cells from “superdonor” iPS cell lines that are HLA-matched to a large portion of the population. Because these cells would match many patients, Dr. Grikscheit hopes to reduce or remove the requirement for immunosuppressive drugs that are often required for transplantation.
This cellular treatment, called advanced superdonor cellular enteric neuropathy therapy (ASCENT), could replace absent or diseased components of the enteric nervous system—the cause of medical conditions such as Hirschsprung’s disease. The work will include collaboration with scientists at Cedars Sinai Medical Center, University of Michigan, and Cincinnati Children’s Hospital.
Some of Dr. Grikscheit’s earlier research is featured in an article in the January/February 2015 LifelineLetter (see “Tissue-engineered Small Intestine: A Proposed Future Treatment for Short Bowel Syndrome”).
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