For the last few weeks CIRM has been doing a lot of introspection as we develop a new Strategic Plan, pushing stem cell research forward.

Patients Advocates

But we haven’t done this alone. We traveled to San Diego, Los Angeles, and San Francisco to meet with Patient Advocates in each city and hear their thoughts on what we should prioritize.

Why Patient Advocates?

Because they have the most at stake. They are the reason we do this work, so it’s essential that they help shape how we do it.

As Chris Stiehl, a Patient Advocate for type 1 diabetes, said in San Diego: “Let the patient be in the room, let them be part of the conversation about these therapies. They are the ones in need, so let them help make decisions about them right from the start, not at the end.”

A strategic plan

A Strategic Plan is, on the surface, a pretty straightforward thing to put together. You look at where you are, identify where you want to go, and figure out the best way to get from here to there. But as with many things, what seems simple on the surface often turns out to be a lot more complicated when looked at in more depth.

The second bit, figuring out where you want to go, is easy. We want to live up to our mission of accelerating the development of stem cells therapies to patients with unmet medical needs. We don’t want to be good at this. We want to be great at this.

The first part—seeing where you are—is harder. It requires what our President and CEO, Dr. Randy Mills, calls “confronting some brutal facts,” being honest about your current position, because without that honesty you can’t make progress.

So where are we as an agency? We have nearly one billion dollars left, 12 projects in clinical trials with more coming, a role in advancing stem cell research from a fledgling field to one on the verge of delivering remarkable treatments, and a strong team ready to push the field even further.

But how do we get from where we are to where we want to be? CIRM 2.0 is part of the answer—a faster, simpler, more responsive process for funding research and supporting the scientists and companies developing new therapies. But it’s only part of the solution.

Leading with science

Some Patient Advocates asked whether we should focus solely on a few diseases—such as the ten leading causes of death in the U.S.—and direct all remaining resources toward solving them. And the answer is yes, we looked at every single option. But we quickly decided against that because, as Randy Mills said:

“This is not a popularity contest, you can’t judge need by numbers, deciding the worth of something by how many people have it. We are disease agnostic. What we do is find the best science, and fund it.”

Another necessary element is developing better ways to attract greater investment from big pharmaceutical companies and venture capital to really help move the most promising projects through clinical trials and into patients. That is starting to happen, not as fast as we would like, but as our blog yesterday shows things are moving in this direction.

The regulatory process

The third piece is getting the Food and Drug Administration (FDA) to approve therapies for clinical trials. And this last piece clearly hit a nerve.

Many Patient Advocates voiced frustration with how slowly the FDA approves new therapies. Often feels like the agency keeps adding obstacles, they said.

Dr. Mills noted that the FDA is caught between a rock and a hard place. The agency is criticized for moving too slowly. But if a treatment later proves unsafe, it is chastised for moving too quickly. He agreed that changes are needed:

“The regulatory framework works well for things like drugs and small molecules,” Mills said. ” that can be taken in pills, “But it doesn’t work well for cellular therapies like stem cells. It needs to do better at that.”

One Advocate suggested creating a Boot Camp to train researchers in the skills needed to navigate the FDA approval process. Others proposed using political pressure from pateint groups to push for change.

There are no easy answers. However, the meeting raised important questions that are helping shape our thinking about the Strategic Plan.

Randy reminded everyone that the stem cell agency “is in the time business. What we do is time sensitive.” For too many people, that time is already running out. We have to do everything we can to change that.