Board gives stem cell institute marching orders, and a road map

The poet T. S. Eliot once wrote: “If you aren’t in over your head, how do you know how tall you are?” Well, everyone at CIRM, California’s stem cell institute, is about to find out how tall we are.

Strategic Plan coverYesterday our governing Board approved a new Strategic Plan. To call it ambitious might be considered an understatement. Among the goals it commits us to achieving are:

  • Funding 50 new clinical trials in 5 years including 10 for rare or orphan disorders and 5 in conditions affecting children
  • Fostering enactment of a new, more efficient federal regulatory approval process for stem cell treatments
  • Introducing 50 new therapeutic candidates or devices into the development pipeline
  • Reducing the time it takes to move a stem cell treatment from the earliest Discovery stage into a clinical trial by 50%
  • Increasing the number of projects moving to the next stage of development by 50%

No easy task

Each goal by itself might be considered challenging. Taken together they are likely to stretch us all. And yet that’s why we joined CIRM, why we feel fortunate to be part of this mission. We have a chance to be part of a movement that could change the face of medicine as we know it. We knew it wouldn’t be easy. But now we know what we have to do to help achieve that.

As Randy Mills, our President and CEO, said in a news release, the goal in developing this Strategic Plan was to create a clear vision for the next five years of the Institute:

”We have around $900 million left to work with and we wanted a plan that used that money to the best possible effect, maximizing our chances of pushing as many new treatments to patients as possible. We didn’t want something ‘good enough’, we wanted something ‘great’. This plan is extremely ambitious, but also realistic in the goals it sets out and the way those goals can be met.”

The Strategic Plan – you can read it in full here – doesn’t just lay out goals, it also creates a road map on how to meet those goals. They include engaging industry more, being more creative in how we move the most promising projects from one stage of research to the next, and finding ways to change the regulatory approval process to help remove obstacles and speed up the progress of these therapies into clinical trials.

Aiming high

We know we may not achieve all our goals. As Randy Mills said at our Board meeting: “This is a difficult plan. These goals are not easy to achieve.” There are always risks in pursuing something so big and ambitious but no one ever achieved anything truly worthwhile by playing it safe. We are not interested in playing it safe.

We may start out by being, as T. S. Eliot put it “in over our heads”. But we’re confident we’ll be able to grow tall enough to make this plan work.

As Randy Mills told the Board: “If we are all in this together then the probability of success is high, and if we are successful then all this would have been worthwhile.”

Call to Action by FDA at World Stem Cell Summit

Califf

FDA Deputy Commissioner Dr. Robert Califf talking at the World Stem Cell Summit

The World Stem Cell Summit annual conference in Atlanta kicked off today with a clarion call from Dr. Robert Califf, the Deputy Commissioner for the Food and Drug Administration. He told the audience:

“We want you to accelerate translation to produce safe and effective therapies that can be delivered reliably”

It was a message that everyone in the room, scientists and patient advocates, would love to be able to comply with. The question of course is how do you do that in a way that puts the emphasis on both speed, to get the therapies to patients who need them, and safety, so you don’t put those patients at risk.

That’s quite a challenge considering that, as panel moderator Julie Allickson of Wake Forest Institute for Regenerative Medicine said:

“the estimate now is it costs $2.4 billion and up to ten years to take something to the clinic.”

Even if that dollar amount is higher than many think it would take to bring a stem cell therapy to a clinical trial it is an indication of the challenge the field faces.

Califf, who has only been at the FDA for 8 months, says that regenerative medicine is:

“not the only field exploding with scientific knowledge and seeing a future that’s very different from what we see today so it’s exciting but also an enormous challenge for the FDA. One of the real eye openers for me is to be at the FDA and hear about drugs that have been on the market for 45 years and we’re still learning about them.”

He says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. “That’s an issue we struggle with every day.”

Califf was optimistic that the balance can be struck and progress can be made, but said that this can only truly be done if the patient is at the table as an active participant.

“Our national clinical research system is well intention but flawed. We need to have a new system that shares information right across the system and where patients are at the center. Patients should be driving the national research infrastructure. They are an essential part of change. It’s happening in Congress because they are hearing from constituents that this is what they want, a voice in the research being done that affects them.”

For the patients and patient advocates in the audience it was a welcome message. For years they have been calling for a louder voice in the research that affects them and their loved ones. Knowing they have a sympathetic ear in the FDA could be an encouraging sign that their voices are finally being heard.

We will be writing more as the conference unfolds so stay tuned!