BrainStorm Advances International Patent Application for Stem Cell Procedure

BrainStorm Cell Therapeutics has announced that it has advanced its patent application from a provisional to an international patent application for a procedure to derive "neuronal-supporting cells" from adult bone marrow with the US Patent and Trademark Office.

The patent protects a procedure for inducing bone marrow stem cells to differentiate into astrocytes, which are brain cells that naturally support neurons in the brain.

The induced cells display astrocyte-like morphology, express typical astrocyte proteins and most importantly, have the capacity to synthesize and secret neurotrophic factors, including glial-derived neurotrophic factor (GDNF).

Transplanting these neuronal-supporting astrocyte cells-acting on their own or in combination with dopamine-producing cells-holds promise for the replacement of and/or preservation of neurons in Parkinson's and other neurodegenerative diseases.

The invention involves inducing adult bone marrow stem cells to differentiate into neuronal supporting cells using the proprietary stem cell technology developed at Tel Aviv University. 

The latest patent application was filed by the technology transfer company of Tel Aviv University, Ramot, on the basis of research funded by Brainstorm.

Worldwide rights to the development and commercialization of the technology are exclusively licensed to BrainStorm.

"Developing the capability to induce adult stem cells to differentiate into cells that secrete a battery of neurotrophic factors is a major step forward because of the important role that these factors are believed to have in brain cell survival and growth," said Yoram Drucker, Principal Executive Officer of BrainStorm.

"Neurotrophic factors have the capacity to protect neurons and induce neural sprouting, and hold great promise for the treatment of many neurodegenerative diseases, including Parkinson's disease, Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Spinal Cord Injury (SCI) and even Alzheimer's disease," he added.

BrainStorm has previously announced observing a significant beneficial effect of transplanting GDNF-producing cells-derived from human bone marrow stem cells utilizing the propriety technology-into animal models of Parkinson's disease.

The scientific team transplanted the GDNF-producing cells into rats with Parkinson's disease, generated by specifically damaging their dopaminergic cells.

Within just two weeks of cell transplantation, the team observed significant improvement in the rats' characteristic disease behavior, including 50% reduction in rotational movements and enhancement in their paw reaching capacity. The beneficial effect was maintained for over four months.

Other routes of delivery of GDNF to the disease site have proven difficult to achieve.

GDNF is a protein and, as such, has limited stability and ability to penetrate the brain. Attempts made to deliver the protein directly into the brain have met with limited success.

An alternative approach, to deliver GDNF by genetic therapy, suffers the limitations and risks of using viral vectors.

Other cell therapeutic approaches using either genetically engineered or differentiated embryonic and neural stem cells are limited by issues of graft rejection and potential tumorogenic risk.

"BrainStorm's approach of using patient-derived differentiated stem cells holds the promise to overcome the above pitfalls," said Dr. Daniel Offen, Chief Scientist.

"Recognized as the patient's own cells, there should be no graft rejection. Moreover, unlike embryonic cells, the bone marrow derived-cells are not known to be tumorogenic."

"Thus, the newly transplanted cells are expected to survive and integrate, releasing the therapeutic GDNF in a physiological manner."