CHOC Stem Cell Production Facility to Accelerate Research into Rare Neurological Diseases

StemCellLabpicCHOC Children’s new stem cell production facility, slated to open late this summer, will allow CHOC researchers to produce patient-specific cells for immune-matching therapies that could positively impact fatal neurological diseases in children – all at a fraction of the cost of building a larger, more complex laboratory.

Within the state-the-art softwall clean room, CHOC researchers will study a stem cell-based therapy for the treatment for mucopolysaccharidosis (MPS-1), a rare and progressive neurodegenerative disease that typically claims patients before they reach the age of 10.

“Based on the results of animal trials we’ve conducted so far, we have a high degree of confidence that stem cell-based therapy will work to treat MPS-1,” said Philip Schwartz, Ph.D., senior scientist at the CHOC Children’s Research Institute and managing director of the National Human Neural Stem Cell Resource.

“If our research is successful, the approach could be used to treat a number of other immune-based diseases that damage the nervous system, like multiple sclerosis,” Dr. Schwartz said.

The approach involves using umbilical cord blood to replace a patient’s immune system, then implanting neural cells derived from the same blood into the brain to repair and prevent brain damage.

While implanting cells directly into the brain isn’t new, current treatment protocols require that patients take immunosuppressant drugs to reduce the risk of rejection, which leaves them vulnerable to a host of infections. Standard procedures for replacing the immune system, like bone marrow transplants, aren’t effective for patients with brain disorders caused by their underlying disease because the transplanted cells don’t cross the blood-brain barrier and therefore don’t slow the progression of brain disease.

The new facility will be one of less than a dozen in the nation and the only one that is focused on immune matching rather than immune suppression.

Dr. Schwartz estimated that it would require about five years of work to establish a program before approaching the U.S. Food and Drug Administration for approval to begin Phase I clinical trials. The current research project is supported by a $4.27 million grant from the California Institute for Regenerative Medicine.

Dr Mary Zupanc and Dr Sharief Taraman Talk About Infantile Spasms

Taraman and Zupanc

Dr. Mary Zupanc, chief of neurology and the director of CHOC’s comprehensive epilepsy program, and Dr. Sharief Taraman, who leads the CHOC Concussion Program, recently stopped by CHOC Radio to talk about an upcoming conference no pediatrician should miss.

From sleep disorders to epilepsy, there are a wide range of neurological conditions that can easily escape diagnosis by a general practitioner. Sadly, the delay in diagnosis and proper treatment can negatively impact a patient’s outcome.

Dr. Zupanc and Dr. Taraman share a recent case study involving infantile spasms. These can present in a subtle way and yet are very serious. Knowing what to ask parents and what to look for during an examination are key factors that can lead to the right diagnosis.

Listen in to this segment of CHOC Radio for more about this case and the conference, “Neurology Problems Pediatricians Shouldn’t Miss.”

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Dr. Sharief Taraman addresses symptoms that mimic brain tumors

Pseudotumor cerebri is a condition in which patients have symptoms that mimic those of a brain tumor, Dr. Sharief Taraman, a neurologist at the CHOC Children’s Neuroscience Institute, tells “American Health Journal.”

The condition can affect all people, but it’s most typically found in adolescent girls and older women, and usually in those who are obese, Dr. Taraman says.

Learn more about this condition in “American Health Journal,” a television program that airs on PBS and other national network affiliates that reach more than 30 million households.

Each 30-minute episode features six segments with a diverse range of medical specialists discussing a full spectrum of health topics. For more information, visit www.discoverhealth.tv.

Sharief Taraman, M.D., completed his medical education at Wayne State University School of Medicine, and completed residency training in pediatrics and pediatric neurology at the Children’s Hospital of Michigan.

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