FTCR: Rejected Lab Grant Applicants Should Appeal in Public

A CIRM watchdog organization today urged five institutions "rejected" for multimillion dollar state lab construction grants to appear publicly next week and make their case for funding from the California stem cell agency.

John M. Simpson, stem cell project director of the Foundation for Taxpayer and Consumers Rights, said the review process for the $263 million lab grant program "has been flawed and smacks of favoritism that can only be cured by transparency." He said,

"CIRM management decided to reveal (last month) the 12 institutions that will be recommended for an invitation to seek funding so that they could use the information in year-end fundraising efforts. How is that fair to the five who were not anointed in secret by the closed scientific brotherhood?"

The California stem cell agency has declined to say whether any other lab grant applicants – besides Childrens Hospital Oakland Research Institute – are attempting to overturn negative decisions on their grant applications.

Ellen Rose, a spokeswoman for the agency, said,

"Appeals are allowable only if there is demonstrable evidence of a financial or scientific conflict of interest. Differences of scientific opinion among PIs and reviewers are not grounds for appeal. In the past, applicants who have raised questions about their grant applications or sought clarification, which is common in any granting exercise, have been informed of this policy. Thus far we have had no formal appeals to our grant-making process."

Simpson said CIRM's statement was "Orwellian double-speak." He said,

"I strongly urge representatives of all five rejected institutions to show up at next week's ICOC meeting and make their case in public directly to the board. It is, they claim, the decision making authority. What's happened so far were merely "recommendations.'"

By law, the Oversight Committee is the final authority on grant approval. It can accept or reject – for virtually any reason -- decisions by the working groups. That is one reason CIRM says it is not necessary for grant reviewers to publicly disclose their financial interests.

In response to queries from the California Stem Cell Report last month, two other institutions disclosed that their applications have been rejected. They are the University of California at Riverside and Cedars-Sinai in Los Angeles. However, they have not responded to queries on Thursday about whether they are asking Oversight Committee to reconsider the working group action.

The lab grant program is the first in which the names of applicants have been disclosed by CIRM, which previously said the names of all applicants were confidential even when the applicants themselves disclosed their own identity. The decision to assist the applicants with fundraising is important because CIRM will look more favorably during the two-step approval process on applicants with large matching funds.

The Oversight Committee is scheduled to take up the grants next Wednesday during the scientific portion of the approval process. Only those approved next week will move on to the next step.

FTCR on Childrens Hospital Appeal: Fairness v. Secrecy

John M. Simpson, stem cell project director of the Foundation for Tazpayer and Consumers Rights, offered the following comment on the item below dealing with Childrens Hospital Oakland appeal of what amounts to a grant denial.

"CIRM management's now inconsistent penchant for secrecy coupled with news of the Children's Hospital letter of appeal once again demonstrates the importance of complete transparency.

"When institutions ask for public money they should be publicly identified. When they are rejected -- even if it's by members of a closed scientific brotherhood -- they should be identified and the reasons for the rejection should be spelled out.

"In this case CIRM management touted the 'successful' applicants and remained mum about what may, in fact, be excellent programs.

"Anything less than full disclosure leaves CIRM management justifiably open to charges of favoritism. In the long run that inevitably undercuts public faith in an agency whose dedicated staff in fact is attempting to serve the public interest by fostering important scientific research."

Rejected CIRM Grant Applicant Seeks Reversal of Action

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."

Lubin wrote:

"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."

Lubin continued,

"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.

Text of Lab Grant Appeal Letter

Here is the text of the grant appeal letter from the Childrens Hospital Oakland Research Institute.

December 28, 2007

Dear Members of the ICOC,

We are requesting that the ICOC reverse the decision of the Grants Working Group and recommend CHORI’s application for a Major Facilities Grant. We think it is very much in the interest of the citizens of California, and of CIRM itself, that this proposal be funded. We have proposed a facility that will expand and further develop 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. These disorders cause severe anemia with onset in infancy, but also widespread damage to vital organs such as brain, lung, liver and kidney. Our track record in this work is, we are confident, world-class.

Our request to the ICOC rests on the following points:

Our program has pioneered use of umbilical cord blood stem cells to cure genetic and malignant diseases in children; of particular significance is our demonstration that cord blood stem cells can be used to cure sickle cell disease.

CHORI laboratories have identified a new type of blood stem cell present in the placenta. These cells promise to provide a rich source of stem cells that will make curative therapy available to individuals who currently need it but do not have suitable stem cells available. Fulfillment of this promise will however require a program of rigorous preclinical and clinical research. The facility we have proposed is designed to house this research program. Our track record in curative stem cell therapy, and our discovery of these cells, make us the most suitable center to carry out the research.

The RFA for the Major Facilities Grant did not restrict the work to human ES cells and their derivatives. In fact, our application seems to fit precisely with the Program Objectives listed in the RFA. It is unusual only because we are proposing to carry out research that will have direct and near-term impact on clinical practice.

In framing our proposal, we had in mind the goals of Proposition 71 and of CIRM to serve the diverse population of California by encouraging ethnic diversity and participation in clinical trials focused upon the applicability of stem cell therapy. We have pioneered, and continue to develop, curative stem cell-based therapies for sickle cell anemia and thalassemia, which affect individuals from underserved minority populations in California.

Our proposal addresses another key goal of Proposition 71, which is to improve the California health care system and reduce the long-term health care cost burden on California through the development of therapies that treat disease and injuries with the ultimate goal to cure them. 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.

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.

No resources to support construction of this facility are likely to be provided by the federal or state governments in the foreseeable future. We and our colleagues would welcome the opportunity to discuss this matter with you in detail.

Respectfully,
Bertram H. Lubin, MD
President, Director of Medical Research


Mark Walters, MD
Director, Blood and Marrow Transplant Program
Children's Hospital & Research Center, Oakland