Friday, August 26, 2011

Progress toward stem cell clinical trials?

The CIRM governing board meeting yesterday, held on the Stanford campus, included a number of important agenda items — planning awards worth $1.8 million and changes to the grants review process to name a few — and one seeming sleeper item titled "Presentation and discussion of CIRM Translational Grant Portfolio."

Yawn, right? But that's the item that generated the most excitement among board members, particularly the patient advocate members who are often asked to talk about progress CIRM is making in generating new therapies in their disease areas.

Many who read a major newspaper in California have probably read assertions about CIRM's apparent lack of progress. No new therapies in five years of funding? Must be a failure! (This, despite the twelve or more years it generally takes to go from a good idea in the lab to a new therapy in the clinic.)

The grant portfolio presentation by Patricia Olson, executive director of scientific activities, and Ellen Feigal, VP of research and development, should answer those complaints. The document was attached to the agenda and is available here. It looks about as dull as it sounds, and in its current state has more acronyms than complete words. However, the message is an exciting one: CIRM has 44 awards that are all in the late stages of therapy development, many with the goal of beginning clinical trials in the next few years. These include teams working on cancer, blindness, spinal cord injury, osteoarthritis, diabetes, and neurodegenerative disorders like Parkinson's disease, Alzheimer's disease and Huntington's disease.

We'll be putting this information online in a more digestible form over the next few weeks.

In addition to support from board members, the presentation prompted a humorous plea from new board chair Jonathan Thomas to members of the press to please, next time, look at this analysis before writing that we haven't accomplished much. All that research closing in on clinical trials likely wouldn't have occurred without us. That's pretty phenomenal.

We've written previously about the reasons for funding the full pipeline of research (Where are the cures?). Out of hundreds of basic science projects only a few even get to clinical trial, and of all clinical trials only a small number result in a new therapy. If we do want to see new therapies one day (and we do) then we need a lot of good ideas that will feed into these translational awards and eventual come out the end of the research pipeline as new therapies. With these 44 awards we should be seeing a number of new clinical trials over the next few years, and one day some of those are going to be therapies in a clinic near you.



  1. Are there any researchers at CIRM that could post their ideas on how this article will accelerate new therapies? A lot of us have a short time horizon.

    From skin cells to motor neurons

    The work by Eggan, a member of the Harvard Stem Cell Institute principal faculty and an associate professor in Harvard’s Department of Stem Cell and Regenerative Biology (SCRB), and his colleagues builds on and advances work by SCRB co-chair and Professor Doug Melton, who pioneered direct cellular reprogramming, and Marius Wernig of Stanford, who used direct reprogramming to produce generalized neurons.
    In a paper given “Immediate Early Publication” online by Cell Stem Cell, the Eggan team reports that the cells they are calling iMNs appear to be fully functional. “One of the most important things we’ve done is show that when you put them into the embryo they function normally like motor neurons,” Eggan said in an interview. “They move to the right place and function on their own.”
    When placed in the spinal cord of a chicken embryo, the iMNs settle into the cord and send out their projections to connect with muscles. “That’s a unique thing,” Eggan said. “We showed [that] they have contact with muscle cells and make synapses with them.”

  2. Hello,

    Thanks for providing these useful tips over here. Stem cells grown and transformed into specialized cells with characteristics consistent with cells of various tissues such as muscles or nerves through cell culture...

  3. As usual, nothing for lung disease, the number 3 killer in the U.S.