Disease Team Planning
ICOC Funds Committed:
The goal of this proposal is to create and prepare a team of California-based researchers and clinicians to write a Disease Team Research Award application aimed at curing corneal vision loss through the culture and implantation of stem-cell-derived full or partial thickness human corneal grafts. Corneal vision loss is a growing malady that affects millions of persons worldwide. The efficiency of regulatory approval and the significant benefit to a very large patient population worldwide will validate and promote California’s efforts to become the world leader in stem-cell-based regenerative medicine. The research concept is to optimize existing technologies to differentiate human stem cells into sterile, standardized, functional, FDA-approved human corneas and corneal tissue under cGMP conditions. Manufacturing methods will be created to produce standardized corneal tissue that will be quality tested and shipped to corneal transplant surgeons for implantation using existing techniques. A disease team research approach is well suited to conduct the basic and transitional research necessary to bring this therapeutic concept to the clinic. The goal of this proposal requires knowledge and experience in a broad set of scientific and clinical disciplines, including the fields of histology, corneal structure and function, molecular and genetic biology, immunology, corneal transplantation, cGMP manufacturing and clinical trial design and implementation. The planning award will allow the assembly of such a team. Corneal vision loss as a disease target is very well suited as the focus for a Disease Team Award. The corneal constructs have been created and proof of principle is established. Regulatory approval of the implantation of stem-cell derived corneal tissue should be minimal. Clinical trial design is facilitated by the fact that subjective analysis of improved vision is available from patients as well as objective analytical data from clinicians. Intellectual property is likely to be generated through this work, resulting in increased valuation for California research organizations involved. This will generate further growth and investment and result in increased employment for Californians and increased tax revenues for California. Planning will use a strategic planning process. This process will consist of a set of direct meetings with internal and external experts to formulate objectives aimed at finding gaps in human and material resources. These gaps will be filled and follow-on meetings will take place to find and fill remaining weaknesses in the team. Once a team is formed, objectives will be formed. Management tactics will consist of implementation plans containing measurable goals, assignment of responsibilities and timelines, and periodic meetings to evaluate the success or failure the objectives.
Statement of Benefit to California:
Large economic and health benefits will accrue to California and critical validation of stem-cell-based regenerative medicine can be established through the creation and implantation of human corneas and corneal tissue derived from human stem cells. A growing billion dollar market for corneal tissue currently exists. The rationale and justification of funding stem-cell-based regenerative medicine can be expediently established since proof of principle is established and the regulatory pathway is less demanding. According to the Eye Bank Association of America’s 2006 Eye Banking Statistical Report, there are more than 34,000 corneal transplants performed annually in the US. An additional 150,000 transplants are performed in the rest of the world. There are eight million corneal blind patients in developing countries (Sinha et al., 2005) who would benefit from corneal replacement were it not for the lack of established eye banking operations and religious/ethical issues. Concern over donor-to-recipient disease transmission and the increasing use of LASIK treatment has reduced the availability of donated corneas and increased costs. Demand for corneal tissue is growing based on advances in corneal transplant techniques (Goins et al., 2007). Even considering the fact that fees would be less in developing countries, the existing corneal transplant market is billions of dollars in size and is growing. Intellectual property is likely to be generated through this work, resulting in increased valuation for California research organizations involved. This will generate further growth and investment and result in increased employment for Californians and increased tax revenues for California. Regulatory approval of the implantation of stem-cell derived corneal tissue should be minimal in comparison to other stem cell based therapies. The front of the eye is an immune privileged site (Cursiefen et al., 2007). Regulatory approval for corneal transplantation techniques already exists. Clinical trial design is facilitated since subjective analysis of improved vision is available from patients as well as objective analytical data from clinicians. Stem cells can be used to generate sufficient corneal tissue to meet the worldwide demand to treat corneal blind patients and provide significant income and employment to California. The tissue source will not be subject to donor-related medical issues or to ethical or religious resistance associated with donor tissue transplantation. The efficiency of regulatory approval and the significant benefit to a very large patient population worldwide will validate and promote California’s efforts to become the world leader in stem-cell-based regenerative medicine.