THE BLOG
07/21/2014 06:59 pm ET | Updated Sep 20, 2014

A Talk With C. Randal Mills, New Head of the California Institute for Regenerative Medicine

In April 2014, C. Randal Mills was named President and CEO of CIRM, the California Institute for Regenerative Medicine, a San Francisco-based agency created in 2004 to allocate over $3 billion in funding for stem cell research for California. As the former CEO of Osiris Therapeutics Inc., the first company to commercialize a stem cell treatment, Randal's rise to the top of CIRM has significant implications for the U.S. cell therapy market, which is poised to grow to $5.7 billion by 2020.

I recently caught up with Randy to discuss his new role at CIRM and the road ahead.

RS: What inspired you to leave a successful public company that you built to join the public sector and take on CIRM?

RM: Well, in reality it was a two-step process. In December of 2013, I made the decision to step down as CEO of Osiris after an amazing, decade-long journey. Osiris had grown up. With three cell therapy products on the market growing at over 200 percent, having a market value in excess of a half a billion dollars, and being profitable, Osiris had turned the corner that many biotech companies never get the chance to. And so, with the company solidly positioned, I started discussing with my family what might be next, including taking a break to spend more time with them. However, fate had other plans.

In February I received a call from CIRM asking if I was interested in the President and CEO position. Having spent the past five years as a grant reviewer for CIRM, I was already quite familiar with the stem cell agency. If you believe in the potential of regenerative medicine and cell therapy as I do, there is no place in the world you could go to have a bigger impact. No company, no other state, not even a country can have the impact California can have in bringing these treatments to patients. And so with that, I accepted the challenge.

RS: At Osiris, you made history by commercializing Prochymal, the first-ever approved stem cell therapy, used for the treatment of acute graft-vs.-host disease in children, a lethal complication of a bone marrow transplant where the transplanted cells start to attack the patient's body. How will that experience shape your work at CIRM?

RM: First off I need to correct you on one point. "We" made history, not "I." Gaining approval for Prochymal was one of the greatest experiences of my life, but it would never have happened without the efforts of an extraordinary team.

Now to your point. One word: urgency. A child with severe GvHD has a median survival of just 87 days. Months, even days, equal lives. Watching a child die of an incredibly painful disease drives home in the most personal and powerful of ways that there is not a second to lose in this game.

At Osiris, we adopted a saying to help us make decisions. I even wrote it into one of our annual reports so investors would know what motivated us. It was, "If it were your child, could it wait?"

That question made for a lot of long nights and weekends but also kept the gravity of our work squarely in everyone's mind. The same is true at CIRM.

RS: What are your top priorities for CIRM in the year ahead?

RM: Accelerating stem cell treatments to patients in need. That is our primary goal. Everything else is subordinate. We have developed a four-part test which the team and I use as a compass.

First, will what we are doing speed up the development of stem cell treatments for patients? Second, will it increase the likelihood of a successful treatment reaching patients? Third, is it for an unmet medical need? And lastly is it efficient?

For CIRM to achieve its full potential, I firmly believe we need to remain focused on bringing treatments to patients, fast. Everyone loves that word, "focus." However, what they may not love is living with its reciprocal, which is everything else we don't do. Without focus, you never have to have the hard conversation. You never have to say "no." However, without focus, you also tend to not get things done. I came to CIRM to get stem cell treatments to patients in need, and that means focus.

RS: What do you feel are CIRM's greatest successes to date? And what will change under your leadership?

RM: After five years working for CIRM reviewing grants, the thing I love is how they are always evolving to better serve their mission, being confident enough to say, "We don't have it all figured out yet," and open-minded enough to implement new ideas.

As for me, I am not here to change anything for the sake of change. I am focused on whatever accelerates stem cell treatments to patients with unmet medical needs. So to that end, I will listen to the team, to our stakeholders, to anyone with a good idea, and change anything that improves our ability to achieve the mission. We already have a few good ideas in the pipeline.

RS: What diseases and conditions are your top targets?

RM: My only consideration with respect to the disease is that it be an unmet medical need. That means a disease that does not have a treatment or the treatment is inadequate or intolerable. It also targets those indications or approaches that would not be attempted if not for CIRM.

RS: In the past, there was some criticism of CIRM for investing so heavily in the deep waters of cellular R&D -- for not supporting more viable commercial plays that can deliver here-and-now therapies. Do you feel any of that criticism was warranted?

RM: You have to remember that in the early days of the agency, the field was still relatively young, and there were lots of questions that needed to be answered at the more basic level before we could begin funding more advanced programs.

CIRM exists under the principle of "If not for us...." California stepped up when, and most importantly because, others would not. Funding something here-and-now that will otherwise get done without CIRM is not consistent with what the people of California wanted to accomplish with Proposition 71. It also violates my third rule, because it wouldn't really be an unmet need if it were going to happen in any event.

We are here to help get stem cell treatments that, if not for us, would take longer to reach patients or might not happen at all. If you look at the projects we support that are now heading into clinical trials, many would never have even gotten off the ground if it hadn't been for us.

RS: iPS cells, or induced pluripotent stem cells, have been a total game changer for our industry, and CIRM has been adding them to its research portfolio. Do you plan to expand CIRM's work in iPS?

RM: If doing so meets the four criteria. Again, for me, it is all about getting stem cell solutions to patients. I am technology and cell-type agnostic. I do want to make sure that while we are working on the next greatest thing, we are also pragmatic enough to not let "perfect" get in the way of "better." In California we have some of the world's best scientists working on cutting-edge technology. We also need to recognize that when something can meaningfully help a patient, it should be advanced, even if it is not perfect. Every great thing has a good first generation.

RS: It has now been 10 years since the passage of Proposition 71, the voter-approved initiative that created CIRM. Of the initial $3 billion in bond funding for stem cell research, the agency has allocated roughly $1.7 billion in grants. How do you plan to refill the coffers?

RM: That remains to be seen, but there are several options people are working on. My job, however, is to make sure CIRM performs as well as possible with the money we have, which is still a lot. One thing is certain: Our goal, for however long we are around, is to come to work every day as if people's lives depend on us. Because people's lives depend on us.