Jonathan Thomas, California Institute of Regenerative Medicine (CIRM)

Thanking the organizers, admiring the agenda, hoping to add to it in a good way . . . starting off by telling the story of a couple of recent meetings that have to do with the general field of regenerative medicine.

A month or so ago, Mike Milken hosted a group designed to move research faster — Faster Cures — at this moment in DC they’re talking about cutting everything.  The threat of sequestration is real and would mean 8% cut at NIH, which would be a very big deal.

Milken wanted to talk about organizing an event on the doorstep of the Capitol.  The Celebration of Science was 1000 people for 3 days, pulling together leaders of gov’t interested in health care, university presidents, philanthropists, last few heads of NIH and the FDA . . . on the middle day of this even they were at NIH, and there were a host of speakers talking about what NIH is up to, what kind of cutting edge work was being done.  There was a satellite feed with scientists talking live about their work, including SCI and regeneration.

The most striking thing to me was that regenerative medicine seems to mean stem cell research and genomics (DNA sequencing) . . . these two things combine to become what will be personalized medicine.

On the theme of personalized medicine, there’s a family in San Diego called the Beery family, who had twins, a boy and a girl.  They were diagnosed at age 2 with cerebral palsy.  The mother noticed that the condition of the children was deteriorating, which isn’t usual for CP . . . she went looking for anything she could find that the doctors had missed.  She found an article about something called Dopa Responsive Dystopia and brought it to her doctor.

He said it was worth a shot.  There’s a treatment for that, and the kids got it.  Picture the parents standing on the stage at NIH . . . with images of the kids being healthy and normal through grade school.  But at about age 12 they started to deterioriate for reasons nobody understood.  Back to the internet goes Mom.  She gets a doctor at Baylor U. to do a full genome sequencing on the kids, and there it was, a gene that caused serotonin deficiency in their bodies.

They got the treatment, and today are track athletes in high school in San Diego . . . and at that point in the talk the kids came bounding onto the stage.  The point of all this is that this kind of personalized medicine is what’s coming.

The second story has to do with a conference sponsored by CIRM, which we called Meeting on the Mesa — an aggregation of companies involved in stem cell research.  It’s a very interesting annual event, where each org. gets 15 minutes to describe what they’re working on.  One of them was the Stem Cells Inc presentation, which you’re about to hear.

There were others, too.  One is called RINO Site, which is about to ask for human clinical trials, along with others working on remyelinating nerves .  . . . a number of them that are making real progress.  We know that things are happening, & we’ve already funded a great many of them, as have others.  You don’t always hear about these things in the press, which tends to cover ethical issues and not progress.

We’re very happy to be able to fund stem cell research, thanks to Prop 71, which 8 years ago passed by 57% of the populace.  It was a grand experiment . . . $3 billion to fund companies in the state of CA doing stem cell research.  What that has done is turn CA into the epicenter of stem cell research in the USA.  We have a continuing stream of scientists from around the world moving here just to work in this environment.  At last count 135 of them, along with their staffs . . . it’s a tremendous pool of talent, which has led to a tremendous acceleration in medical research.

To date we’ve funded $1.8B, aimed at the most prevalent incurable conditions, one of which is spinal cord injury.  We’re aiming very high.  We’re looking for cures.  One of the problems we have with the press is that their stories go like this:  Gee.  We gave you all that money and where are the cures?  You were supposed to get this done in a month or two weeks or whatever.

When I sit down with reporters trying to figure out what we actually do, they can be a little intimidated . . . I sit down with them and they ask me why there are no cures, and I say: if your grandfather had been sitting with Jonas Salk in 1954 and asking him how come money raised since 1938 was just going down the hole, what could Salk have said?

The very next year he found the vaccine for polio, and that was 18 years after the effort started.  And that has arguably saved millions of lives.

That’s the level of difference we plan to make, and specifically in SCI.  We’ve funded 38 to 40 million so far, from basic research to clinical trials.  We funded Geron when they were working on SCI, and were very badly disappointed when they chose to discontinue.  At this point Mike West and Tom Okarma have bought the SCI portion of Geron’s portfolio, which all of us find very interesting and are watching closely.

As you know, six patients were treated with the Geron cells in a Phase I safety trial; they’re still being carefully monitored and have shown no ill effects from the cells.  I met patient #6, a young woman in northern CA, who was tremendously enthusiastic about the trials and full of hope that the trials would eventually deliver recovery for her and for the others.  She and the other 5 are eager to see those trials go forward.  Geron was the first company in the world to get FDA approval for using human stem cells in a product.

They did us all a considerable service by working through the FDA to break this ground; the FDA is very deliberate and slow-moving when it comes to new therapies.  Geron hung in long enough to get that part of it done.

At CIRM, we have some projects that we look to see heading toward successful conclusions.  One is about macular degeneration, another is Type 1 diabetes, HIV, heart disease . . .

All that said, nothing is closer to our hearts than SCI; Christopher Reeve and Roman Reed were hugely important in the effort to pass Prop 71, and we’re determined to carry on.

(Marilyn gets back up to say that CIRM funded the video-taping of this conference, and to introduce our friend, the amazing Donna Sullivan.)

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One Comment on “Jonathan Thomas, California Institute of Regenerative Medicine (CIRM)”

  1. […]  Just being in the same room with the head of the California Institute of Regenerative Medicine (CIRM), which still has control of more than a billion dollars to spend on stem cell-based research, […]

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