Stem cells are today recognised for the vast potential they hold in curing diseases and healing injuries. With successes on various fronts, a great deal of research still needs to be undertaken to fully understand stem cells, which are, essentially, the building blocks of life.
With their ability to multiply rapidly and to develop into a variety of cell types they provide doctors with huge supplies from which to draw from to initiate cures for various illnesses and healing for diverse injuries.
Knowing very well that stem cells might hold the answers to diseases that have hitherto defied science, Californians voted for 3 billion US$ to be used in stem cell research in 2004. Considered as one of California’s biggest investments in Science, the whole project faces the gloomy prospect of dying still-born as the money is fast running out. With treatments yet to emerge from the researches the voters of Californian now face a stark choice: call off the project and count their losses or vote in more billions that may turn the tide and produce concrete results in cutting edge stem cell therapies.
Out of the initial $3 billion, slightly over half a billion ($650 million) remains. This amount is expected to run out in 2020 and already lobbying to raise more money from the private sector is under way. Jonathan Thomas, chair of the California Institute for Regenerative Medicine (CIRM) governing board acknowledges the predicament they find themselves in. CIRM, itself a creation of the vote (Proposition 71) that established the US $3 billion research fund, has funded over 750 projects and reports promising results from various clinical trials. One outstanding example will suffice. In a clinical study that CIRM co-founded, and that involved 10 children with congenital severe combined immunodeficiency, the results obtained were emphatic enough to warrant the continuation of the various studies under way. 9 out of the 10 children in the study were reported to be doing well 8 years after treatment. The children were able to go to school, play outside and overcome the inevitable infections, all without the aid of injections.
CIRM has also played a pivotal role in enabling research on ageing and regenerative medicine. With the construction of a dozen facilities dedicated to this work many early-career academics have benefitted from these grants dispensed by CIRM. Early federal restrictions in this line of scientific study (following President George W. Bush’s order in 2001) plus the prohibitive cost of stem cell research had locked out many of these academics from this field.
To put to maximum use the remaining $650 million CIRM is now working with Quintiles IMS of North Carolina (a contract-research organisation) to carry out further clinical trials in the hope that by so doing 40 new stem cell therapies will transition to the clinical trial stage by 2020. Americans for Cures and advocacy group aims at getting an additional US $ 5 billion to continue research into stem cells and intends to poll voters on this matter. With the current US administration proposing cuts to the National Institutes of Health (NIF), Bob Klein, instrumental in putting Proposition 71 on the ballot and who also leads Americans for Cures, expresses high hopes that Californians will once again step up and vote for science.
While Bob Klein and his associates make every effort to raise more funds (even soliciting funds from wealthy individuals and philanthropic organisations), others are not so sure that this is the way to go. John Simpson, attached to the advocacy group Consumer Watchdog, and who directs stem cell oversight work in this group, is particularly disenchanted with the idea of securing research funding through a popular vote and he is opposed to any efforts to extend CIRM. The disbursement of funds through CIRM has previously attracted its share of controversy. From some of the scientists vetting grants having vested interests to the perception that CIRM had not been astute enough in directing research, many have felt that CIRM displayed a lack of probity that held back the progress of research and, by extension, facilitated wastage of the resources.
The entry of Randy Mills as CEO of CIRM did a lot to change how CIRM is perceived. Although he is now leaving to head the National Marrow Donor Program, he has left behind a leaner, stronger agency that can now boast of an accelerated funding process. He has also been instrumental in shortening the period between research and clinical trials and is credited with cleaning up an agency that was on the verge of losing all credibility, especially with proven cases of conflict of interest coming to light. Crucially, Mills has made it easier for the Food and Drug Administration (FDA) to approve stem cell treatments.
Researchers point out that expecting treatments to be rolled out after only a decade of research is unrealistic. The monolithic nature and painfully slow pace of drug development (from allocation of funding to unveiling of treatments) would appear to bear out the researchers’ concerns. Ultimately it will be up to the people of California to weigh the issues raised and decide whether further funding will be forthcoming or whether the researchers will have to seek funding elsewhere to complete their projects.