Budgeting for the future of the stem cell agency

ICOC_DEC17-24

The CIRM Board discusses the future of the Stem Cell Agency

Budgets are very rarely exciting things; but they are important. For example, it’s useful for a family to know when they go shopping exactly how much money they have so they know how much they can afford to spend. Stem cell agencies face the same constraints; you can’t spend more than you have. Last week the CIRM Board looked at what we have in the bank, and set us on a course to be able to do as many of the things we want to, with the money we have left.

First some context. Last year CIRM spent a shade over $306 million on a wide range of research from Discovery, the earliest stage, through Translational and into Clinical trials. We estimate that is going to leave us with approximately $335 million to spend in the coming years.

A couple of years ago our Board approved a 5 year Strategic Plan that laid out some pretty ambitious goals for us to achieve – such as funding 50 new clinical trials. At the time, that many clinical trials definitely felt like a stretch and we questioned if it would be possible. We’re proving that it is. In just two years we have funded 26 new clinical trials, so we are halfway to our goal, which is terrific. But it also means we are in danger of using up all our money faster than anticipated, and not having the time to meet all our goals.

Doing the math

So, for the last couple of months our Leadership Team has been crunching the numbers and looking for ways to use the money in the most effective and efficient way. Last week they presented their plan to the Board.

It boiled down to a few options.

  • Keep funding at the current rate and run out of money by 2019
  • Limit funding just to clinical trials, which would mean we could hit our 50 clinical trial goal by 2020 but would not have enough to fund Discovery and Translational level research
  • Place caps on how much we fund each clinical trial, enabling us to fund more clinical trials while having enough left over for Discovery and Translational awards

The Board went for the third option for some good reasons. The plan is consistent with the goals laid out in our Strategic Plan and it supports Discovery and Translational research, which are important elements in our drive to develop new therapies for patients.

Finding the right size cap

Here’s a look at the size of the caps on clinical trial funding. You’ll see that in the case of late stage pre-clinical work and Phase 1 clinical trials, the caps are still larger than the average amount we funded those stages last year. For Phase 2 the cap is almost the same as the average. For Phase 3 the cap is half the amount from last year, but we think at this stage Phase 3 trials should be better able to attract funding from other sources, such as industry or private investors.

cap awards

Another important reason why the Board chose option three – and here you’ll have to forgive me for being rather selfish – is that it means the Administration Budget (which pays the salaries of the CIRM team, including yours truly) will be enough to cover the cost of running this research plan until 2020.

The bottom line is that for 2018 we’ll be able to spend $130 million on clinical stage research, $30 million for Translational stage, and $10 million for Discovery. The impact the new funding caps will have on clinical stage projects is likely to be small (you can see the whole presentation and details of our plan here) but the freedom it gives us to support the broad range of our work is huge.

And here is where to go if you are interested in seeing the different funding opportunities at CIRM.

New Stem Cell Agency President Says; “It’s all about the Patients”

This week we held two events to introduce our new President, C. Randal Mills, to our biggest supporters, the researchers we fund and the patient advocates who are looking to stem cell science for treatments and cures. The events were at our offices in San Francisco and at the University of Southern California campus in Los Angeles (we are holding a third event in San Diego on June 24th as part of the BIO2014 conference)

Randy began by saying that he has a simple guiding principle; that everything we do at the agency should be “all about the patients”. In fleshing that out he identified four criteria that he will use in making any decision:
1. Will it speed up the development of treatments for patients
2. Will it increase the likelihood of developing a successful treatment for patients
3. Will it meet an unmet medical need
4. Is it efficient

Stem cell agency President Randy Mills talking at USC

Stem cell agency President Randy Mills talking at USC

He says those criteria will help make sure that everything we do at the stem cell agency is in alignment with our goals; that we aren’t funding work that could easily attract funding from other agencies or even the pharmaceutical industry.

Randy says he wants to come in every day knowing that everyone at the agency is working as hard as they can, “working as if people’s lives depended on it; because people’s lives do depend on us. There is a real urgency to what we do. The work we do can help save lives. We can’t afford to let people down.”

For the scientists Randy said his goal was to give them as much support as they needed, particularly in the areas where they may not be very experienced – such as moving products into clinical trials, getting approval from regulatory agencies such as the Food and Drug Administration (FDA) and commercializing a potential therapy.

“I’m a great believer in not trying to teach fish to fly. I want the fish, our researchers, to swim as fast as they can, to do the best research they can, and we can help with all the other elements involved in moving a product out of the lab and into clinical trials and ultimately into patients.”

Before being appointed as President at the end of April Randy worked as President and CEO at Osiris Therapeutics, a company that specialized in the use of mesenchymal stem cells (MSCs), a kind of stem cell found in bone marrow that helps form bones, cartilage and fat among other things. Asked if that experience with MSCs would influence his time at the agency he said no:

“When it comes to stem cells, when it comes to technology, I am completely agnostic. I don’t care about particular cell types; I care about the people that we are here to help. So I want to take whatever is the best cell for the job, whatever is the best, most effective technology for the job and use those, support those, to speed up the development of therapies for patients.”

While he has been a member of our Grants Working Group review panel for five years and has a good grasp of the different kinds of research that we fund Randy said this is still very much a learning period for him; a time to talk to, and listen to as many people and listen to as many people as possible as he settles in to his new position.

As he looks forward to where the agency is going he says he is very positive and excited at being part of something truly unique:

“One of the ways I think about decision making is asking; “if this were our child what would we do?” And that motivates me every day. If we do this really well, if we succeed, we are going to have such a profoundly transformative effect on people’s lives.”

New Stem Cell Agency President Says; “It’s all about the Patients”

This week we held two events to introduce our new President, C. Randal Mills, to our biggest supporters, the researchers we fund and the patient advocates who are looking to stem cell science for treatments and cures. The events were at our offices in San Francisco and at the University of Southern California campus in Los Angeles (we are holding a third event in San Diego on June 24th as part of the BIO2014 conference)

Randy began by saying that he has a simple guiding principle; that everything we do at the agency should be “all about the patients”. In fleshing that out he identified four criteria that he will use in making any decision:
1. Will it speed up the development of treatments for patients
2. Will it increase the likelihood of developing a successful treatment for patients
3. Will it meet an unmet medical need
4. Is it efficient

Stem cell agency President Randy Mills talking at USC

Stem cell agency President Randy Mills talking at USC

He says those criteria will help make sure that everything we do at the stem cell agency is in alignment with our goals; that we aren’t funding work that could easily attract funding from other agencies or even the pharmaceutical industry.

Randy says he wants to come in every day knowing that everyone at the agency is working as hard as they can, “working as if people’s lives depended on it; because people’s lives do depend on us. There is a real urgency to what we do. The work we do can help save lives. We can’t afford to let people down.”

For the scientists Randy said his goal was to give them as much support as they needed, particularly in the areas where they may not be very experienced – such as moving products into clinical trials, getting approval from regulatory agencies such as the Food and Drug Administration (FDA) and commercializing a potential therapy.

“I’m a great believer in not trying to teach fish to fly. I want the fish, our researchers, to swim as fast as they can, to do the best research they can, and we can help with all the other elements involved in moving a product out of the lab and into clinical trials and ultimately into patients.”

Before being appointed as President at the end of April Randy worked as President and CEO at Osiris Therapeutics, a company that specialized in the use of mesenchymal stem cells (MSCs), a kind of stem cell found in bone marrow that helps form bones, cartilage and fat among other things. Asked if that experience with MSCs would influence his time at the agency he said no:

“When it comes to stem cells, when it comes to technology, I am completely agnostic. I don’t care about particular cell types; I care about the people that we are here to help. So I want to take whatever is the best cell for the job, whatever is the best, most effective technology for the job and use those, support those, to speed up the development of therapies for patients.”

While he has been a member of our Grants Working Group review panel for five years and has a good grasp of the different kinds of research that we fund Randy said this is still very much a learning period for him; a time to talk to, and listen to as many people and listen to as many people as possible as he settles in to his new position.

As he looks forward to where the agency is going he says he is very positive and excited at being part of something truly unique:

“One of the ways I think about decision making is asking; “if this were our child what would we do?” And that motivates me every day. If we do this really well, if we succeed, we are going to have such a profoundly transformative effect on people’s lives.”