Early Translational I
ICOC Funds Committed:
Many injuries to the brain and spinal cord do not heal. Spinal cord injury can leave a patient confined to a wheelchair for life. After suffering a stroke a patient is often left unable to move parts of their body or have impaired brain function such as losing the ability to speak. With both stroke and spinal cord injury the window for spontaneous healing is typically about three months. After that time has passed, conventional therapeutic interventions do not significantly help. Multiple sclerosis is another type of injury to the brain and spinal cord. The immune system in these patients attacks the insulation (called myelin) that surrounds nerve fibers. The body attempts to repair this damage but cannot keep up with the progression of the disease. The nerves of the injured brain and spinal cord do not regenerate for several reasons. In stroke and spinal cord injury a scar forms (the glial scar), physically preventing the regrowth of nerve fibers. In multiple sclerosis the progressive loss of the myelin insulation causes the nerves to malfunction and die. One possible way to overcome both of these problems is to add a type of cell with potential to destroy or prevent the formation of the scar, encourage the growth of new nerve fibers, and re-establish the myelin insulation. A scientist in Japan has attempted to create a cell able to do some of these potentials from an a from bone marrow adult stem cell. Treatment of these cells with a collection of reagents and growth factors caused them to resemble in certain ways the cells that form part of the myelin insulation. This has inspired several researchers to test these cells in animal models of nerve injury. The cells, now called SB618 cells, were very effective in restoring nerve function. Exactly how SB618 cells restore nerve function is not understood. This project will optimize the production of SB618 cells so that additional experiments can be performed. Effects on models of stroke, spinal cord injury and multiple sclerosis will be examined. Tests to help determine the mechanism of action will be performed in the laboratory. If this three year project is successful, it will trigger the production of clinical grade SB618 cells and the safety studies required by the FDA for the filing of an Investigational New Drug application (an IND). SB618 cells will be ready for human clinical a little more than a year later.
Statement of Benefit to California:
This proposed research is for the translation of experimental observations made in academia to the point where a product will be ready for pre-clinical and clinical development. If all goes well, the product will be ready to initiate human clinical trials four years from the beginning of the project and be ready for commercialization six years after that. During the 10 years between project initiation and commercialization, the company will need to add skills and manpower. This will bring high paying jobs to California, adding to the tax base for the benefit of all Californians. This will be part of the movement, already underway, making California the leading center for stem cell research and development in the world. If the project is commercially successful, there is an agreement with CIRM that revenue would be shared with the State of California. The amount to be shared is up to three times the amount of CIRM grant money awarded. If the therapy achieves blockbuster status the amount paid to California could be many millions of dollars more than that. There is also an agreement with CIRM that a therapy resulting from CIRM funds will be made available to uninsured Californians through the California Discount Prescription Drug Program. Finally, and most importantly, this project aims to provide relief from stroke, spinal cord injury and multiple sclerosis. These devastating conditions have huge impacts on the quality of life and the productivity of people everywhere. Californians do not benefit disproportionately from this except in taking pride in the fact that the voters had the wisdom to put this in motion.