Cure Sanfilippo Foundation Funds New Stem Cell Research

October 22, 2018

Cure Sanfilippo Foundation is pleased to support the work of Dr. Jan Nolta’s lab in collaboration with biotech partner Neuralstem. This project aims to develop an off-the-shelf neural stem cell therapy for patients with Sanfilippo Syndrome (MPS IIIA).

As the Director of the UC Davis Stem Cell Program and Institute for Regenerative Cures, Dr. Nolta brings a wealth of experience and expertise to this project. Prior research conducted by her team has focused on the use of stem cell therapies for Huntington’s Disease, 20+ years of work on lysosomal storage diseases, and involvement in prior clinical trials.

University of California, Davis, conducting research on Sanfilippo Syndrome, MPS III

Dr. Nolta commented, “We are very grateful to the Cure Sanfilippo Foundation and excited to work with Neuralstem to develop a gene-modified neural stem cell therapy for MPSIIIA. The data for retention of the Neuralstem product is compelling and gives us hope that an impact could potentially be made on this heartbreaking disease.”

Neuralstem is a clinical stage biopharmaceutical company developing treatment for neurologic diseases via small molecule and stem cell therapies. Our joint collaboration will enable the gene modification of Neuralstem’s proprietary neural stem cell line and testing of these cells in the immunodeficient MPSIIIA mouse model which was created in the Nolta lab as a part of Cure Sanfilippo Foundation’s previous support.

Neural stem cells (NSCs) are the cells that give rise to neurons and supporting cells(glia) of the central nervous system. The application of NSCs in neurodegenerative disorders such as Sanfilippo syndrome is particularly exciting. Gene modified (to express large amounts of the
deficient enzyme) neural stem cells could address the primary disease mechanism. However, NSCs are special in that they also offer the possibility to restore and support healthy brain tissue in patients.

“The opportunity to address multiple critical needs in this disease with one therapy would be an amazing thing for the children. Since we do not yet have newborn screening or an approved therapy for Sanfilippo syndrome, 99% of those diagnosed are already symptomatic and
experience ongoing brain damage. The ability to positively impact disease progression even after a child is symptomatic is of key importance to families today,” says Dr. Cara O’Neill, Chief
Scientific Officer at Cure Sanfilippo Foundation.

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Cure Sanfilippo Foundation is a 501(c)(3) not-for-profit organization dedicated to advocating for and funding research directed towards a cure and treatment options for patients with Sanfilippo Syndrome. Sanfilippo Syndrome, also called MPS III, is an inherited disease of
metabolism that means the body cannot properly break down long chains of sugar molecules called mucopolysaccharides or glycosaminoglycans (i.e., GAGs). A genetic defect passed on
from each parent results in missing or poorly functioning enzymes needed for cells to work normally. Without these enzymes, cells are unable to break down and recycle cellular waste.

Over time, this waste builds up causing cells to act abnormally and then to die. Children with this genetic disease face a progressively debilitating and rapid decline in physical and intellectual abilities, leading to an early death.

For more information on the Cure Sanfilippo Foundation and Sanfilippo Syndrome, please visit Contact Cure Sanfilippo Foundation at

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