Stem cell agency’s response to questions about its performance

Ed’s Note: Here is the full text of the response from the California stem cell agency to a query about the five most important things that it thinks Californians should know about the $3 billion effort. The information was provided by Kevin McCormack, senior director for communications at the agency.

“It’s hard to limit it to 5 because we have done so many different things that, in their own way, are pioneering and ground-breaking. It’s like asking someone to choose their favorite child. We love them all equally.

“1)   Research we have supported has cured more than 30 children of a fatal rare immune disorder. That same approach has also cured a young man of another rare immune disorder and is now being used to help find a cure for sickle cell anemia – a condition that affects more than 100,000 people in the US, most of them African Americans

“2)   We have funded 29 projects in clinical trials for a variety of diseases including heart disease, cancer, diabetes, HIV/AIDS, spinal cord injuries and the leading cause of vision loss and blindness in the US

“3)   We are focused on adding another 40 new clinical trials by 2020, including rare diseases and those affecting children

“4)   We place patients at the heart of everything we do and every decision we make. Nothing gets done at CIRM, including what we fund and how clinical trials are designed, without the input of patients and patient advocates. They know best what needs to be done and their voices are essential in making decisions about treatments that could change their lives, even save their lives

“5)   CIRM has made California a world leaders in stem cell research, attracting some of the most talented scientists to the state to develop new treatments and funding world-class research facilities in which the research is conducted free from federal restrictions

“6)   We have created the CIRM Alpha Stem Cell Clinic Network and the Stem Cell Center, two unique institutions that combined can improve the ability of researchers to get their products approved for clinical trials, and to increase the chances those clinical trials will be successful. These are already attracting both CIRM and non-CIRM funded projects to California, and will be an enduring contribution to the development of stem cell projects of all kinds.

“In addition we created the iPSC Bank which is developing a repository of more than 3,000 different cell types for research into the causes, and potential treatments for diseases as varied as Alzheimer’s and autism, heart disease and vision loss.

“We are helping create new jobs and generate new taxes. A 2014 economic impact report showed that by that year alone we had generated $284 million in new taxes for the state and helped create 38,000 new job years (that’s economist speak for 38,000 jobs that last one year, 19,000 that last two, etc.)

We changed the way our Board votes to ensure there would be no more concerns about the perception of conflict of interest. By preventing heads of institutions who could receive stem cell funds from voting on any funding issue, we took that off the table so that we can focus on our main goal, helping patients.

“We have created leadership and trainee programs – such as our Bridges program – that are helping train the next generation of stem cell scientists.

“We played a key role in advancing the 21^st Century Cures Act and that two of the first projects approved for accelerated review and approval are CIRM-funded clinical trials. Humacyte was the very first project granted Regenerative Medicine Advanced Therapy and last week jCyte was granted RMAT designation for its work in treating retinitis pigmentosa (here’s a fabulous blog about that).

“Don’t forget, we have around $650 million left to spend, that’s far more than any other state has available (New York, for example has $50m a year, Connecticut just $10m ) and more than many countries, and we intend to use that money to continue to accelerate the research that will provide new treatments and cures.”