New Video: Spinal Cord Injury and a CIRM-Funded Stem Cell-Based Trial

Just 31 years old, Richard Lajara thought he was going to die.


Richard Lajara, the 4th participant in Geron’s stem cell-based clinical trial for spinal cord injury.

On September 9, 2011 he slipped on some rocks at a popular swimming hole and was swept down a waterfall headfirst into a shallow, rocky pool of water. Though he survived, the fall left him paralyzed from the waist down due to a severed spinal cord.

Patient Number Four
At that same time period, Geron Inc. had launched a clinical trial CIRM helped fund testing the safety of a stem cell-based therapy for spinal cord injury (SCI). It was the world’s first trial using cells derived from human embryonic stem cells and Lajara was an eligible candidate. Speaking to CIRM’s governing Board this past summer for a Spotlight on Disease seminar, he recalled his decision to participate:

“When I participated with the Geron study, I was honored to be a part of it. It was groundbreaking and the decision was pretty easy. I understood that it was very early on and I wasn’t looking for any improvement but laying the foundation [for future trials].”

A few months after his treatment, Geron discontinued the trial for business reasons. Lajara was devastated and felt let down. But this year the therapy got back on track with the announcement in June by Asterias Biotherapeutics that they had treated their first spinal cord injury patient after having purchased the stem cell assets of Geron.

Getting Hope Back on Track
Dr. Jane Lebkowski, Asterias’ President of R&D and Chief Scientific Officer, also spoke at the Spotlight on Disease seminar to provide an overview and update on the company’s clinical trial. A video recording of Lebkowski’s and Lajara’s presentations is now available on our web site and posted here:

As Dr. Lebkowski explains in the video, Asterias didn’t have to start from scratch. The Geron study data showed the therapy was well tolerated and didn’t cause any severe safety issues. In that trial, five people (including Richard Lajara) with injuries in their back received an injection of two million stem cell-derived oligodendrocyte progenitor cells into the site of spinal cord damage. The two million-cell dose was not expected to show any effect but was focused on ensuring the therapy was safe.

Oligodendrocyte Precursors: Spinal Cord Healers
As the former Chief Scientific Officer at Geron, Lebkowski spoke first hand about why the oligodendrocyte precursor was the cell of choice for the clinical trial. Previous animal studies showed that oligodendrocyte progenitors, a cell type normally found in the spinal cord, have several properties that make them ideal cells for treating SCI: first, they help stimulate the growth of damaged neurons, the cell type responsible for transmitting electrical signals from the brain to the limbs.

Second, the oligodendrocytes produce myelin, a protein that acts as an insulator of neurons, very much like the plastic covering on a wire. In many spinal cord injuries, the nerves are still intact but lose their myelin insulation and their ability to send signals. Third, the oligodendrocytes release other proteins that help reduce the size of cysts that often form at the injury site and damage neurons. In preclinical experiments, these properties of oligodendrocyte progenitors improved limb movement in spinal cord-severed rodents.

Together, the preclinical animal studies and the safety data from the Geron clinical trial helped Asterias win approval from the Food and Drug Administration (FDA) to start their current trial, also funded by CIRM, this time treating patients with neck injuries instead of back injuries.

The Asterias trial is a dose escalation study with the first group of three patients again receiving two million cells. The trial was designed such that if this dose shows a good safety profile in the neck, as it did in the Geron trial in the back, then the next cohort of five patients will receive 10 million cells. In fact, Asterias reported in August that the lower dose was not only safe but also showed some encouraging results in one of the patients. And just two days ago Asterias announced their data monitoring committee recommended to begin enrolling patients for the 10 million cell dose.  If all continues to go well with safety, the dose will be escalated to 20 million cells in the third cohort of five patients. While two million cells was a very low safety dose, Asterias anticipates seeing some benefit from the 10 and 20 million cell doses.

Changing Lives by Increasing Independence
Does Lebkowski’s team expect the patients to stand up out of their wheelchairs post-treatment? No, but they do hope to see a level of improvement that could dramatically increase quality of life and decrease the level of care needed. Specifically, they are looking to see a so-called “two motor level improvement.” In her talk Lebkowski explained this quantitative measure with the chart below:

“If a patient is a C4 [meaning their abilities are consistent with someone with a spinal cord injury at the fourth cervical, or neck, bone] they will need anywhere from 18 to 24 hours of attendant care for daily living. If we could improve their motor activity such that they become a C6, that is just two motor levels, what you can see is independence tremendously increases and we go from 18 to 24 hour attendant care to having attendant care for about four hours of housework.”

Slide13 cropped

Small improvements in movement abilities can be life changing for people with spinal cord injuries.

It’s so exciting the field is at a point in time that scientists like Dr. Lebkowski are discussing real stem cell-based clinical trials that are underway in real patients who could achieve real improvements in their lives that otherwise would not be possible.

And we have people like Richard Lajara to thank. I think Dr. Oswald Stewart, the Board’s spinal cord injury patient advocate, summed it up well when speaking to Lajara at the meeting:

“Science and discovery and translation [into therapies] doesn’t happen without people like you who are willing to put yourselves on the line to move things forward. Thank you for being in that first round of people testing this new therapy.”

December ICOC Board Meeting to Begin Soon

The December ICOC Board Meeting begins this morning in Berkeley, CA.

The complete agenda can be found here. Dude to inclement weather our Spotlight on Disease has been canceled.

For those not able to attend, you are welcome to dial in:

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[Members of the Public will be invited to provide testimony before or during consideration of each item. Makers of public comments are asked to limit their testimony to three (3) minutes.]

What everybody needs to know about CIRM: where has the money gone

It’s been almost ten years since the voters of California created the Stem Cell Agency when they overwhelmingly approved Proposition 71, providing us $3 billion to help fund stem cell research.

In the last ten years we have made great progress – we will have ten projects that we are funding in or approved to begin clinical trials by the end of this year, a really quite remarkable achievement – but clearly we still have a long way to go. However, it’s appropriate as we approach our tenth anniversary to take a look at how we have spent the money, and how much we have left.

Of the $3 billion Prop 71 generates around $2.75 billion was set aside to be awarded to research, build laboratories etc. The rest was earmarked for things such as staff and administration to help oversee the funding and awards.

Of the research pool here’s how the numbers break down so far:

  • $1.9B awarded
  • $1.4B spent
  • $873M not awarded

So what’s the difference between awarded and spent? Well, unlike some funding agencies when we make an award we don’t hand the researcher all the cash at once and say “let us know what you find.” Instead we set a series of targets or milestones that they have to reach and they only get the next installment of the award as they meet each milestone. The idea is to fund research that is on track to meet its goals. If it stops meetings its goals, we stop funding it.

Right now our Board has awarded $1.9B to different institutions, companies and researchers but only $1.4B of that has gone out. And of the remainder we estimate that we will get around $100M back either from cost savings as the projects progress or from programs that are cancelled because they failed to meet their goals.

So we have approximately $1B for our Board to award to new research, which means at our current rate of spending we’ll have enough money to be able to continue funding new projects until around 2020. Because these are multi-year projects we will continue funding them till around 2023 when those projects end and, theoretically at least, we run out of money.

But we are already working hard to try and ensure that the well doesn’t run dry, and that we are able to develop other sources of funding so we can continue to support this work. Without us many of these projects are at risk of dying. Having worked so hard to get these projects to the point where they are ready to move out of the laboratory and into clinical trials in people we don’t want to see them fall by the wayside for lack of support.

Of the $1.9B we have awarded, that has gone to 668 awards spread out over five different categories:

CIRM spending Oct 2014

Increasingly our focus is on moving projects out of the lab and into people, and in those categories – called ‘translational’ and ‘clinical’ – we have awarded almost $630M in funding for more than 80 active programs.


Under our new CIRM 2.0 plan we hope to speed up the number of projects moving into clinical trials. You can read more about how we plan on doing there in this blog.

It took Jonas Salk almost 15 years to develop a vaccine for polio but those years of hard work ended up saving millions of lives. We are working hard to try and achieve similar results on dozens of different fronts, with dozens of different diseases. That’s why, in the words of our President & CEO Randy Mills, we come to work every day as if lives depend on us, because lives depend on us.

October ICOC Board Meeting to Begin Soon

The October ICOC Board Meeting begins this morning in Los Angeles, CA.

The complete agenda can be found here, including a special Spotlight on Disease focusing on Retinitis Pigmentosa.

For those not able to attend, you are welcome to dial in!

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We will be providing a summary of today’s highlights after the meeting—so stay tuned!

New Videos: Living with Crohn’s Disease and Working Towards a Stem Cell Therapy

Note: the two videos below are also available on our website

She doesn’t want your sympathy. She doesn’t want your admiration. She just wants your understanding.

Rachel Bonner, a sixteen-year-old high school student and founder of the Hope for Crohn’s charity, spoke to the CIRM governing Board on September 10th about what it’s like living with Crohn’s disease. In the eight years since her diagnosis, Rachel has come a long way in talking publicly about her condition:

“I never thought I’d stand up here and admit to wearing a diaper while being in middle school. But Crohn’s turns from a secret struggle to something I want to share with other people. And ultimately have others understand the life of a Crohn’s patient just a bit more. “

Crohn’s disease is a type of inflammatory bowel disease (IBD) in which the intestines are chronically inflamed. Symptoms of Crohn’s include a frequent need to pass bowel movements, constant diarrhea, rectal bleeding, fatigue and loss of appetite.

In a healthy individual, the friendly bacteria living in the gut are ignored by the immune system. But in the case of IBD, the immune cells attack these bacteria as foreign invaders, causing an inflammatory response. The sustained inflammation eventually damages the gut wall causing the symptoms of IBD.

Current therapies for IBD focus solely on treating the inflammation. Dr. Ophir Klein, a CIRM grantee and UCSF researcher, also spoke to the governing Board and described another treatment avenue:

“There’s another component that’s been under-explored and potentially has a lot of impact therapeutically which is the regenerative aspects of the condition because after the inflammation occurs in the gut, the gut needs to heal, and that healing comes from stem cells. “

In his presentation to the Board, Dr. Klein detailed his lab’s work to understand how stem cells regulate the healing of the intestine and to eventually find cures for IBD.

Although Rachel and her doctors have found a treatment sweet spot, which has kept her Crohn’s at bay, she still holds out hope that a cure, perhaps from a stem-cell based therapy, is not too far away:

“Everyday I go to sleep hoping that this treatment sweet spot will work until they find a cure”

CIRM 2.0: How to Build a Better Stem Cell Agency and Speed up Treatments to Patients

Change is never easy. We all get used to doing things in a certain way and it can sometimes be difficult to realize that the way we have chosen, while it may have worked well at one time is perhaps not the best way to achieve our goals at this time. Well, change is coming to the stem cell agency.


It’s not surprising that our new President & CEO, C. Randal Mills, Ph.D., would want to introduce some of his own ideas about how best to run the agency in the current moment of stem cell science. After all, it’s those ideas that landed him the job in the first place. Now Randy wants us to develop a clearer focus, one that is more aligned with his 4-point criteria for assessing everything we do.

  1. Will it speed up treatments to patients
  2. Will it increase the likelihood of successful treatments for patients
  3. Does it target an unmet medical need
  4. Is it efficient.

That new focus begins with re-imagining how we can be most effective in the way we fund research. Right now we put out what’s called an RFA or Request for Application, telling people who have promising projects in a particular area of stem cell research to submit an application and if they are successful they’ll get up to $20 million, depending on the kind of project.

The problem is, we often have long gaps between each round of funding and so a company or institution with a promising therapy will sometimes have to wait as much as a couple of years before they can apply again. If they do wait and are successful in their application it could still be another year or two before they are able to gain actual funding and begin a clinical trial. But when lives are at stake, you can’t afford to wait that long. So we’re looking at ways of speeding things up, making it easier for the best science to get the funds needed when they are needed.

At our Board meeting yesterday Randy outlined some broad concepts about what he wants to do and how it can be done. It’s part of his vision for the agency, a new focus that he is calling CIRM 2.0 (with acknowledgments to Dr. Paul Knoepfler who coined the term earlier this year)

As with any simple idea it’s really complicated. We need to achieve greater speed, to streamline the way we do things, without sacrificing the quality of the review process because we need to ensure that we only fund the best science.

In the months to come, as the precise details about these proposed changes are fine tuned, the Board will hear in greater detail how this will work and, as always, it will be up to them to decide if they think it’s a good idea.

Either way it will start a conversation about how we can become more efficient and more effective at living up to our mission, of accelerating therapies that target patients with unmet medical needs. And that always has to be a good thing.

For more details about the other big events at yesterday’s Board meeting, including awarding $16 million to ViaCyte to help it advance its promising therapy for type 1 diabetes, you can read the news release posted on our website.

September ICOC Boarding Meeting Begins Soon

The September ICOC Boarding Meeting begins this morning in Berkeley, CA.

The complete agenda can be found here, including a special Spotlight on Disease focusing on Inflammatory Bowel Disease.

For those not able to attend, feel free to dial in:

Dial in Infomation:

United States: (800) 230-1093
Access Code: 334835

* Meeting Number(s): 5114686455

1. Go to…
2. Click “Join Now”.

We will be providing a summary of the meeting’s highlights after the meeting—so stay tuned!

A life long battle with Parkinson’s disease earns Dr. David Higgins a place on our Board

David Higgins, Ph.D., the new member of the stem cell agency Board as the Patient Advocate for Parkinson's disease

David Higgins, Ph.D., the new member of the stem cell agency Board as the Patient Advocate for Parkinson’s disease

As a child David Higgins imagined that the reason his grandmother wouldn’t talk to him was because of something he did, some fault of his. In time he came to realize that the reason was because his grandmother had Parkinson’s disease and she had trouble communicating with, not just him, but with everyone. It was the start of his life-long relationship with the disease, one that led to his own diagnosis with Parkinson’s a few years ago, and that has now led to him becoming the newest member of our governing Board, the Independent Citizens Oversight Committee.

David, now Dr. Higgins, was appointed by State Controller John Chiang. In his official letter announcing the news – which you can also find in our news release – he said:

“Diagnosed with Parkinson’s in 2011, his experience with the disease and its impact on a personal level brings a distinct perspective to the ICOC. Dr. Higgins also brings a broad range of experience from the biotechnology field. As a trained molecular biologist, his involvement in drug development and business operations places him in a unique position, understanding both science and process.”

David says he learned many important lessons from his grandmother, including the power of medical research:

“She was in one of the first L-dopa trials (this is a drug that converts into dopamine in the brain, to help counter the disease). I saw how that therapy helped change her life and it gave me my first lesson in how science and scientific research could change someone’s life.”

David’s mother was also diagnosed with, and later died from, a Parkinson’s-like form of dementia. His own diagnosis only served to further reinforce his commitment to doing something to fight the disease.

“I probably had symptoms that I was suspicious of for about two years before I was diagnosed. It was a mixed bag because as a scientist I finally had a label for what was going on and I thought at least now I can start fighting it. But the other side of me said “Oh my God I know exactly where this is going.”

As a research scientist he has held positions at several biotech companies, most recently as Head of US Operations for BioMedica, Inc., a UK-based gene therapy company. He also became a highly active Patient Advocate, and is now the California Assistant State Director with the Parkinson’s Action Network, as well as the new President of the Board of Directors of the Parkinson’s Association in San Diego.

David says he was completely stunned when he was asked if he would like to be on the CIRM Board. But the Chair of our Board, Jonathan Thomas, Ph.D., J.D., says it’s clear that he will be a terrific addition to the team:

“He is a powerful and passionate advocate not just for people with Parkinson’s disease but for science in general. He understands on a personal level how important it is for the public to support research trying to find new treatments and cures for currently incurable diseases.”

David says his personal experience with the disease has clearly influenced his life but does not mean he will focus on that alone:

“One thing I feel strongly about is that, yes I’m the Parkinson’s Patient Advocate on the Board, but first and foremost I’m an Advocate for everyone and I want to make sure that we spend our money wisely, and that we use our resources to identify and nurture the most promising stem cell projects across all target diseases.”

He says he takes up his new role with mixed emotions:

“I am thrilled to be a part of the CIRM governing Board, but I am also humbled to follow in the footsteps of Joan Samuelson, who has been CIRM’s Parkinson’s Patient Advocate from the beginning. She is a hard act to follow.”

Dr. Thomas echoed those sentiments saying:

“Joan set the gold standard for patient advocacy. She was a part of the agency from the very first and her passion, dedication and commitment to helping others was an inspiration to all of us. Joan was always willing to ask tough questions and push us all to do more to speed up the development of new therapies. We are all better for her service, and she will be missed.”

We are delighted to have David join us and we’re looking forward to working with him in the years to come.

kevin mccormack