Disease Team Planning
ICOC Funds Committed:
In the United States, 250,000-450,000 people are living with spinal cord injuries (SCI). Their numbers grow by 11,000 to 15,000 each year. In addition to devastating personal loss and severe limitations on personal freedom, costs associated with SCI are staggering, to both the individual and society. When considering the age of onset, expected lifespan, unemployment rate, associated acute and chronic medical costs, and psychosocial ramifications, the impact of SCI is tremendous. Transplantation of cells lost to disease or trauma represents a viable treatment strategy for many conditions of the injured human central nervous system. The success of cell replacement strategies for the treatment of central nervous system disorders has been demonstrated in several human conditions including Parkinson`s disease and Huntington`s disease, and in rodent models of spinal cord injury. The transplant populations used in all of these instances has been fetal cells. Despite the encouraging results of fetal cell transplantation studies, the widespread clinical use of harvested fetal cells is limited by the availability of donor tissue, concerns regarding the purity and viability of the transplant population, as well as ethical and political controversy. These issues currently limit the development of this sector of the biotechnology industry in California and abroad. Human embryonic stem cells (hESCs) represent a viable solution, as they can be amplified almost indefinitely with out genetic mutation. hESCs have recently been made to become one cell type that is lost following SCI, and transplantation has been shown to be safe and cause improvement of walking ability. To create a viable therapy significantly more research and development is required. These needs will be addressed by a planning approach led by a well-regarded and practicing scientist that is centrally involved in the project, who has extensive experience in developing basic discoveries for application to humans. The planning approach will involve the establishment and integration of 4 components, an Executive Committee to provide overall guidance for the planning process and identify critical milestones, multiple Focus Groups each of which will discuss a particular element of the translational path, an External Advisory Committee to provide world-wide expert guidance, and an Independent Consultant to guide the process with respect to the FDA.
Statement of Benefit to California:
The proposed research will benefit California by preserving and strengthening the State’s position as a leader in the field of stem cell therapeutics. Through the passage of Proposition 71 and subsequent establishment of the California Institute for Regenerative Medicine, the voters of California have identified stem cell research as a key area of focus for the state, with anticipated positive impacts including the creation of biotechnology jobs, attraction of leading researchers to California universities, creation of valuable intellectual property, and advancement of therapeutics beneficial to California residents. No other hESC-based treatment is as developed as the hESC-OPC approach described herein, and remyelination is an approach widely accepted to benefit SCI. Thus, this approach may be the first to validate the State’s support of stem cell research and its application. This program has already led to job creation, tax revenue and other financial and reputation benefits; its advancement will lead to more. Additionally, over a thousand California spinal cord injury patients annually stand to benefit from this product if its development is successful.