Children’s Hospital Los Angeles Awarded $7.1 Million by CIRM

Grant will fund development of cellular therapy to treat digestive
nerve disorders

LOS ANGELES–(BUSINESS WIRE)–Tracy C. Grikscheit, MD, of The Saban Research Institute of Children’s
Hospital Los Angeles, has 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 Grikscheit,
who is also a tenured associate professor of Surgery at the Keck School
of Medicine of the University of Southern California.

Working with human induced pluripotent cells (iPS) that have the ability
to develop into many human cell types, 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, 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 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.”

“The goal of our Translation program is to support the most promising
stem cell-based projects and to help them accelerate that research out
of the lab and into the real world, such as a clinical trial where they
can be tested in people,” says Jonathan Thomas, PhD, JD, and Chair of
the CIRM Board. “The projects that our Board approved today are a great
example of work that take innovative approaches to developing new
therapies for a wide variety of diseases.”

About Children’s Hospital Los Angeles

Children’s Hospital Los Angeles has been named the best children’s
hospital in California and among the top 10 in the nation for clinical
excellence with its selection to the prestigious U.S. News & World
Report Honor Roll. Children’s Hospital is home to The Saban Research
Institute, one of the largest and most productive pediatric research
facilities in the United States. Children’s Hospital is also one of
America’s premier teaching hospitals through its affiliation since 1932
with the Keck School of Medicine of the University of Southern
California. For more information, visit
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Children’s Hospital Los Angeles
Ellin Kavanagh
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