San Francisco Business Times: Can Clinical Trials Save California's Stem Cell Agency?

Tippi McKenzie on front page of San Francisco Business Times

Long, difficult and expensive -- that's how the San Francisco Business Times describes stem cell therapy development in an article about the 33 clinical trials currently backed by the $3 billion California stem cell agency.

Will it all prove to be worthwhile? Reporter Ron Leuty addressed the matter in an Aug. 25, front-page piece that featured Tippi McKenzie of UC San Francisco. She recently received a $10.9 million award from the California Institute of Regenerative Medicine (CIRM), as the agency is formally known, to test her possible therapy on 10 pregnant women.

In what will be the first clinical trial of an in-utero blood stem cell transplant, McKenzie will transplant a mother's own stem cells into her fetus. Leuty wrote that it could be a precursor to pre-birth cures for other blood diseases, such as sickle cell disease.  He continued,

"It’s also the sort of high-profile medical breakthrough that could give the state stem cell funding research agency momentum as it prepares to (possibly) seek billions in fresh money to extend its life beyond 2020."

Leuty wrote,

"Still, any potential treatments are years into the future. Are trials such as MacKenzie’s too little, too late to help CIRM? "  

"It’s all an illustration of the long, difficult and expensive path for bringing any drug to market. Only four of every 10 late-stage trials succeed, according to a 2016 Biotechnology Innovation Organization study of clinical trials from 2006 to 2015. As a relatively new field, in which regulators and companies are seeking a new pathway to Food and Drug Administration approval, stem cell therapies take eight years to translate into clinical trials; non-stem cell therapies take 3.2 years, according to CIRM Chairman Jonathan Thomas. That’s a potentially awkward narrative as efforts ramp up to keep CIRM alive longer. "

Leuty said,

"CIRM can point to 33 projects that have reached clinical trials. For example, an $11.9 million grant to UCSF and St. Jude Children’s Research Hospital is paying for a mid-stage study in 'bubble boy disease.' The agency wants to fund another 40 clinical trials over the next four years, and it is pitching that scale and scope as a reason to keep CIRM going."

The article continued,

 "If anyone has a chance of showing CIRM’s value, it may be MacKenzie, an associate professor of surgery at UCSF’s pediatric surgery unit and head of its Fetal Treatment Center. Her lab since 2010 has been awarded $14.9 million in grants, including cash for basic research, translational research and now in the clinic. 

"CIRM is about more than funding for studies, MacKenzie said. Agency staff has provided advice about how to translate animal studies into work in humans, she said, as well as hiring an FDA consultant, writing an investigational new drug application and setting up a clinical protocol. 'I’m a clinician, but running a clinical trial is different,' MacKenzie said. 'CIRM’s been incredibly helpful in helping me navigate that.'"