One of the rejected applicants for CIRM's $263 million in lab construction grants has asked the agency's directors to reverse the decision and fund an effort that has an "immediate and broad application" in the treatment of sickle cell anemia.
Bertram Lubin, president of the Children's Hospital Oakland Research Institute, wrote members of the CIRM Oversight Committee on Dec. 28 seeking to overturn the negative recommendation by the CIRM Grants Working Group.
Lubin's letter follows CIRM's unusual reversal last month of its longstanding policy of secrecy concerning the names of grant applicants. In the case of the lab grants, CIRM identified 12 applicants that its Grants Working Group decided were worthy of funding by the Oversight Committee. The agency said public disclosure of the names would help the 12 institutions raise matching funds for the grant proposals. Grants with larger amounts of matching funds will have an edge in the competition for the CIRM money. However, the agency has refused to disclose the names of rejected applicants, a policy that has met objections from the Foundation for Taxpayer and Consumers Rights and the California Stem Cell Report.
Lubin (see photo) said his organization's proposal would expand a program "focused on clinical and preclinical research into the use of stem cells to cure inherited disorders of hemoglobin (sickle cell anemia and thalassemia), which affect a disproportionate number of children in California."
"In contrast to some approved proposals that may have only distant and uncertain prospects of actually curing disease, our proposed facility would support the development of curative therapy with a new type of stem cell, with immediate and broad application to very large numbers of individuals."He said the research involves umbilical cord blood stem cells and "will have a "direct and near-term impact on clinical practice."
"The cost of medical care for sickle cell disease averages over $50,000/year over a life expectancy of 30-50 years. In the course of our research, individuals with inherited blood diseases will receive transplants, and our extensive experience indicates these will be curative in the large majority of cases. The enhanced and extended lives of these individuals will represent a direct benefit; the savings to the health care system as a consequence of their cure is less direct but will benefit all California citizens. The knowledge gained from this research will enable improved treatment worldwide, with consequent saving of lives and resources."Lubin also cited language in Prop.71 that states that CIRM should address the medical needs of the ethnically diverse population in California. Sickle cell anemia mainly affects African Americans in the United States.
The appeal from the Children's Hospital is the first such to surface publicly at the agency in any of its grant programs. We are querying CIRM about whether other applicants have appealed in this round or in the past.
The Oversight Committee will meet publicly next week to consider the decisions of the working group. Only grants that receive the go-ahead next week will go on to the next stage of the competition, which is the largest round of grants in CIRM history.
A copy of Lubin's letter was made available to the California Stem Cell Report by a source that asked not to be identified. The copy did not come from Children's Hospital. The full text of the letter is carried below.