Dr. C. Randal Mills

Partnering with Big Pharma to benefit patients

/ Kevin McCormack / 1 Comment

Our mission at CIRM is to accelerate the development of stem cell therapies for patients with unmet medical needs. One way we have been doing that is funding promising research to help it get through what’s called the “Valley of Death.” This is the time between a product or project showing promise and the time it shows that it actually works.

Many times the big pharmaceutical companies or deep pocketed investors, whose support is needed to cover the cost of clinical trials, don’t want to get involved until they see solid proof that this approach works. However, without that support the researchers can’t do the early stage clinical trials to get that proof.

The stem cell agency has been helping get these projects through this Catch 22 of medical research, giving them the support they need to get through the Valley of Death and emerge on the other side where Big Pharma is waiting, ready to take them from there.

We saw more evidence that Big Pharma is increasingly happy doing that this week with the news that the University of California, San Diego, is teaming up with GSK to develop a new approach to treating blood cancers.

Dr. Catriona Jamieson: Photo courtesy Moores Cancer Center, UCSD

Dr. Catriona Jamieson:
Photo courtesy Moores Cancer Center, UCSD

Dr. Catriona Jamieson is leading the UCSD team through her research that aims at killing the cancer stem cells that help tumors survive chemotherapy and other therapies, and then spread throughout the body again. This is work that we have helped fund.

In a story in The San Diego Union Tribune, reporter Brad Fikes says this is a big step forward:

“London-based GSK’s involvement marks a maturation of this aspect of Jamieson’s research from basic science to the early stages of discovering a drug candidate. Accelerating such research is a core purpose of CIRM, founded in 2004 to advance stem cell technology into disease therapies and diagnostics.”

The stem cell agency’s President and CEO, Dr. C. Randal Mills, is also quoted in the piece saying:

“This is great news for Dr. Jamieson and UCSD, but most importantly it is great news for patients. Academic-industry partnerships such as this bring to bear the considerable resources necessary to meaningfully confront healthcare’s biggest challenges. We have been strong supporters of Dr. Jamieson’s work for many years and I think this partnership not only reflects the progress that she has made, but just as importantly it reflects how the field as a whole has progressed.”

As the piece points out, academic researchers are very good at the science but are not always as good at turning the results of the research into a marketable product. That’s where having an industry partner helps. The companies have the experience turning promising therapies into approved treatments.

As Scott Lippman, director of the Moores Cancer Center at UCSD, said of the partnership:

“This is a wonderful example of academia-industry collaboration to accelerate drug development and clinical impact… and opens the door for cancer stem cell targeting from a completely new angle.”

With the cost of carrying out medical research and clinical trials rising it’s hard for scientists with limited funding to go it alone. That’s why these partnerships, with CIRM and industry, are so important. Working together we make it possible to speed up the development and testing of therapies, and get them to patients as quickly as possible.

Faster, better, more efficient. Challenging? That too. An update on CIRM 2.0.

/ Kevin McCormack / Leave a comment

Changing direction is never easy. The greater the change the greater the likelihood you’ll have to make adjustments and do some fine-tuning along the way to make sure you get it right.

On January 1st of this year we made a big change, launching CIRM 2.0. Our President and CEO Dr. C. Randal Mills called it “a radical overhaul of the way the Agency does business.” This new approach puts the emphasis on patients, partnerships and speed and cuts down the time from application to funding of clinical-stage projects from around two years to just 120 days.

You can read more about 2.0 here.

So, several months into the program how are we doing?

Clinical stage of CIRM 2.0 has three programs

Clinical stage of CIRM 2.0 has three programs

Well, since January 1st we have had three application tracks under 2.0 that reflect our goal of accelerating therapies to patients with unmet medical needs. These focus on late stage work to either get a promising therapy into a clinical trial, to carry out a clinical trial, or to help a promising project move even faster.

Under those three programs we have had 12 applications for funding, for a total request of $111 million. With application deadlines the last business day of each month two of those were in January, two in February, three more in March and five in April.

As Dr. Mills told our governing Board when they met last week, that number is more than we were expecting:

 “When we started the program we calculated there’d be around one or two applications a month, not five. I don’t think having five applications a month is sustainable, but that’s probably just the backlog, the pent up demand for funding, working its way through the system. For now we can cope with that volume.”

Interestingly eight of those applications were for funding for clinical trials:

  • Two for Phase 1
  • One for Phase 2
  • Five for Phase 3

Last week our Board approved one of those Phase 3 trials (the last big hurdle to clear before the Food and Drug Administration will consider approving it for wider use), investing almost $18 million in NeoStem’s therapy for one of the deadliest forms of skin cancer, metastatic melanoma.

This is the first time we have ever funded a Phase 3 trial. So, quite a milestone for us. But it may well not be the last one. The Board also approved a project to conduct the late preclinical work needed to apply to conduct a trial in retinitis pigmentosa.

Dr. Mills said there are two clear patterns so far:

“We are getting a more mature portfolio of clinical stage programs for adjudication. We are also starting to see requests for accelerating activities, where we have made previous awards to researchers who now have identified new ways to accelerate that work and they are turning to us for help in doing that.”

Of the 12 applications received we have screened all of them within the 7-day target window to make sure they meet funding criteria. Some have been ruled out for not being within the scope of the award program. The accepted applications have all had budget reviews and been sent on for expert analysis within the slated time frames.

We had a couple of hiccups with our first review but that resulted from on-line technology and getting everyone comfortable with the new rules we were bringing in. The second review resulted in the first two awards by our Board last week, and the third review occurred yesterday.

“The bottom line is things are moving through and things are being weeded out. In March we had two clinical stage applications and one add-on funding application but that one add-on failed in screening. So, in general CIRM 2.0 is being well utilized. There’s no question we are significantly reducing application time from application to funding, attracting later stage applications. Clearly this has not been without its challenges but the team is doing a great job of managing everything.”

And remember this is only the first part of CIRM 2.0. We have two other programs, for Discovery or basic research and Translational research, that are being developed and we plan on rolling those out later this summer.

Stay tuned for more details on those programs.

CIRM 2.0: A New Year, a new start, a new way to advance research

/ Kevin McCormack / Leave a comment

It’s tradition to begin the New Year by making a resolution. Wikipedia has a wonderful description of what this involves saying it is where “a person makes a promise to do an act of self-improvement or something slightly nice, such as opening doors for people beginning from New Year’s Day.”

CIRM2.0_Logo

Well, by that criteria, CIRM 2.0 is a perfect way for us to start 2015 because it is both an act of self-improvement and something “slightly nice” (love that phrase).

2.0, for those of you who haven’t been following us, is a rather dramatic overhaul of the way we do business. It’s about streamlining the way we work in a way that places added emphasis on speed, partnerships and patients.

CIRM 2.0 makes it easier for both companies and academic researchers with promising projects to partner with CIRM to get the support they need when they need it, reducing the time from application to funding from around two years to just 120 days – that’s the “self-improvement”.

In a news release marking the launch of 2.0, our President and CEO Randy Mills summed up the reason why we are making these changes:

“Our mission is to accelerate the development of stem cell therapies for patients with unmet medical needs. Today, in officially launching the first three programs under CIRM 2.0, we have boldly reaffirmed our commitment to continuously seek new and innovative ways to better serve that mission.”

Simply put, we hope that by improving the way we work we can help speed up the development of treatments for patients in need. I would say that more than qualifies as being “slightly nice.”

You can hear Randy talking about CIRM 2.0 here

This is just the first phase of our new look. In December our governing Board gave us $50 million to get this up and running for clinical stage work over the next six months (you can find links to the Program Announcements for that work on our news release). Later this year we are going to expand 2.0 to include both discovery – or basic – research and translational research.

We are now in our 11th year as an agency funding stem cell research. Last year was a big year for us with 8 projects we are funding approved for clinical trials. But as we see every New Year, getting a little older shouldn’t stop you from wanting to improve or making the next year or years even better. Or from just doing something “slightly nice” for others.