In this interview, Maria Millan, the new President and CEO of the California Institute for Regenerative Medicine, reflects on CIRM’s successes so far.
In this interview, Maria Millan, the new President and CEO of the California Institute for Regenerative Medicine, reflects on CIRM’s successes so far and makes her predictions for the future of regenerative medicine.
Maria Millan is the President and CEO of CIRM. Beginning her career as an academic transplant surgeon and researcher, Maria trained at Harvard Medical School- Beth Israel Deaconess Medical Center and Stanford University School of Medicine, and served as Associate Professor of Surgery and Director of the Pediatric Organ Transplant Program at Stanford. Joining CIRM in 2012, Maria was the Medical Officer for two of CIRM’s early first- in-human clinical trials and led the development of CIRM’s strategic clinical and translational infrastructure programs. Before serving as President, Maria was Vice President of CIRM’s Therapeutics group.
Please introduce yourself and your career before joining CIRM?
I am Maria Millan, MD, the new President and CEO of the California Institute for Regenerative Medicine (CIRM; USA), which is the state’s stem cell agency. Before joining CIRM I began my career as an organ transplant surgeon. I was trained in general surgery and obtained an immunology postdoctoral fellowship at Harvard Medical School-Beth Israel Deaconess Medical Center (MA, USA) before going to Stanford University School of Medicine (CA, USA).
“[CIRM] look[s] for stem cell and regenerative medicine projects that are based on solid science”
At Stanford, I completed specialty training in transplantation surgery prior to being recruited to join the faculty. I served as Associate Professor of Surgery and Director of the Pediatric Organ Transplant Program at Stanford & the Lucile Packard Children’s Hospital. Through basic and clinical research, I was involved in driving innovative approaches to improving transplant outcomes and was on the team that developed a first-in-human protocol to achieve “immune tolerance” to the transplanted organs. After Stanford, I became the Vice President and acting Chief Medical Officer for StemCells, Inc. (CA, USA) where I oversaw the launch of the company’s first clinical trial for a rare and fatal pediatric CNS condition and served as the head of the company’s liver program.
You were previously Vice President of CIRM’s Therapeutics group. Can you tell us about a project from this that you found particularly interesting?
By its very nature, CIRM’s therapeutics portfolio is extremely compelling in that these are innovative stem cell and regenerative medicine approaches to addressing unmet medical needs. The potential for these novel technology platforms to impact human health is extremely exciting. What I really enjoyed about leading the Therapeutics team is that we had a chance not only to bring in such high impact projects that are based on solid science but to truly partner with these programs to accelerate the development process and to increase the probability of success that the projects could continue along the path to reaching patients. We have set out bold goals to partner 50 new clinical trials in 5 years and, in 2 years’ time, we will be halfway there.
What did you look for when identifying promising clinical stage stem cell projects to fund?
We look for stem cell and regenerative medicine projects that are based on solid science and that have the potential to meaningfully impact human health by addressing an unmet medical need.
We strive to make the requirements for our funding extremely clear; you can read about those here. In addition to being “within scope” as a stem cell and regenerative medicine project, we make sure that the applicants are at the appropriate stage to come in for our funding and have the necessary regulatory input from the FDA for the given stage of program. For instance, applicants coming in for a clinical trial award (CLIN2) are required to have an active “IND” in place, the permission from the FDA to proceed with a clinical trial. There is also a financial and budget review that takes place before the application is evaluated by a panel of independent scientific experts, our “Grants Working Group.” This group evaluates the proposals and then makes recommendations to our governing Board, which has the final say in which ones are funded.
“CIRM has played an essential role in growing the stem cell/regenerative medicine field”
It may seem like a long process but we have refined it to the point that an application can go from submission to being funded in just 130 days. That’s really very fast by funding agency and investor funding metrics.
What will happen once CIRM has given out all US$3 billion allocated to it by Proposition 71?
We are working with our Board to pursue sustainability options to ensure that the products of the Proposition 71 investment have the best chance of reaching the patients.
CIRM has played an essential role in growing the stem cell/regenerative medicine field by bringing in the critical mass of resources, top notch researchers, rigorous basic and translational research and building the most robust late development and clinical stage portfolio to accelerate novel treatments to patients with unmet medical needs. With the CIRM 2.0 processes that drove operational excellence and acceleration, there is incredible potential to build upon the recent early successes that led to projects that are nearing FDA marketing approval and to drive more of these successes to the benefit of healthcare and patients.
What are your plans for CIRM over the next few years?
We are fully committed and motivated to executing on the 5 year strategic plan that we launched in January 2016. When we proposed these strategic goals, they were “stretch” goals and now it looks like we will achieve these goals. This strategic plan is driven by our mission “to accelerate stem cell treatments to patients with unmet medical needs.”
What are your predictions for the future of stem cell/regenerative medicine?
I believe that we are on an upward trajectory in the field.
Just in the past two years, we are scheduled to double the number of stem cell/regenerative medicine clinical trials in our portfolio from the previous number accumulated over a 10 year period. This is an indication of two things: (1) the field is maturing, and (2) CIRM is actively attracting these programs and, through active partnership, has the opportunity to shape the field.
“We have refined it to the point that an application can go from submission to being funded in just 130 days”
In the recent months, two first-in-class gene modified cell therapy projects for blood malignancies have received marketing approval from the FDA. CIRM is currently clinical trial-testing gene modified stem cell and regenerative medicine products for elusive diseases such as ALS (Lou Gehrig’s Disease), gene modified TSCM (a memory stem cell giving rise to T cells) for solid malignancies in the brain and spinal cord, various forms of fatal immune deficiencies such as ADA SCID (the trial is a “pivotal trial” that could obtain FDA approval upon review of the trial results), X-linked SCID, Artemis SCID and blood forming diseases such as Sickle Cell Disease. These are just a few of our programs. For more information on CIRM’s clinical portfolio, please explore our clinical dashboard.