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.

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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

WEB MEETING ACCESS INFORMATION:
——————————-
* https://www.webmeeting.att.com
* Meeting Number(s): 5114686455
* PARTICIPANT CODE: 313650

WEBEX LINK:
1. Go to https://cirm.webex.com/cirm/onstage/g.php?MTID=ef6aa60e45eb581e0e24ea4d2…
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

Spiderman Sets the Tone for Stem Cell Agency Board Meeting

I don’t often think about Spiderman at meetings of our governing Board – no, really I don’t – but yesterday was an exception. Not that I was daydreaming, rather I was listening to our new President & CEO C. Randal Mills, Ph.D., talk about his determination to set a very specific tone in leading the agency.

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Randy had just explained to the Board that he had asked the agency’s General Counsel to draw up an agreement stating he – Randy, not the lawyer – will not accept a job with any company funded by CIRM for at least one year following his departure from the agency. In addition he will also refuse to accept gifts or travel payments from any company, institution or individual who receives agency funding.

In a news release we issued following the Board meeting he explained his reasons for making this commitment:

“I want the people of California to know that my sole interest in being at CIRM is to help advance stem cell treatments to patients who are in need. I will do so with a full commitment to transparency and by never compromising the integrity of our mission nor our trust to the taxpayers of California.”

And that’s where Spiderman comes in. As any fan of the movie or comic books can tell you one of the things Spiderman says a lot is “With great power comes great responsibility.”

In making his commitment Randy wanted to send a very clear and very strong message that he understands what his role as the President involves, and that it’s important for him to demonstrate that through his actions.

Board member and patient advocate, Sherry Lansing, echoed that saying:

“We take even the possibility of a perception of a conflict of interest very seriously and are determined to do whatever is necessary to ensure that we protect the reputation of the agency and the work that we do. We fully support Dr. Mills in the way he is handling this issue.”

Randy decided to make that commitment after his predecessor, Dr. Alan Trounson joined the Board of Stem Cells Inc., a company that we awarded more than $19 million to develop a therapy for Alzheimer’s disease. While there is nothing illegal about Dr. Trounson’s actions the news did cause a bit of a stir with a few commentators saying this was a dark mark against the agency – even though there is nothing we could have done to stop it because we did not know it was happening.

Randy is not asking anyone else to make the same commitment he has made, but he says it was important for him to do so. His role as President & CEO carries great responsibility and he says he wants to show that he takes it very seriously and will lead by example.

I rather think Spiderman would approve.

Kevin McCormack

July ICOC Board Meeting Now Beginning

The July ICOC Boarding Meeting is now beginning in San Francisco.

The complete agenda can be found here.

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

Dial In (800) 230-1085
Confirmation Number: 331407

Audio Cast:
Web Meeting Address: https://www.webmeeting.att.com
* Meeting Number(s): (511)468-6455
* HOST CODE: 697745
* PARTICIPANT CODE: 249234

WebEx Link:
Go to https://cirm.webex.com/cirm/onstage/g.php?MTID=eb948a1127913f44c7cdffcedc0fa510a
Click “Join Now”.

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