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California Institute of Regenerative Medicine awards Children's Hospital of Los Angeles $7.1 million grant

Grant will support development of new cellular therapy to treat digestive system nerve disorders, hospital says.

Beth Jones Sanborn, Managing Editor

The California Institute of Regenerative Medicine has awarded Tracy C. Grikscheit, MD, of The Saban Research Institute of Children's Hospital Los Angeles a $7.1 million grant to develop a cellular therapy for the treatment of digestive system nerve disorders, the hospital announced in a statement. The grant was awarded through CIRM's Translational Research Program.

Also called enteric neuropathies, these disorders can often only be treated by removing segments of the intestine that lack 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 Grikscheit, who is also a tenured associate professor of Surgery at the Keck School of Medicine of the University of Southern California.

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The new therapy would involve a type of cell, called human induced pluripotent cells, that can develop into many different kinds of cells making them useful in many ways. Dr. Grikscheit proposes to use these cells to generate nerve cells that are "HLA matched" to a large segment of the population. Because these cells are compatible with many patients, Grikscheit hopes to reduce or remove the need 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 disease. The work will include collaboration with scientists at Cedars Sinai Medical Center, University of Michigan, and Cincinnati Children's Hospital. Grant reviews made available publicly online praised the team as having "great expertise" and noted that "the team could perhaps be the best in the world for this indication," Children's Hospital said in a statement.

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Children's Hospital Los Angeles is home to The Saban Research Institute, one of the largest pediatric research facilities in the United States. Children's Hospital has also enjoyed a long-standing affiliation since 1932 with the Keck School of Medicine of the University of Southern California.

Twitter: @BethJSanborn