Treatments, cures and clinical trials: an in-person update on CIRM’s progress

Patients and Patient Advocates are at the heart of everything we do at CIRM. That’s why we are holding three free public events in the next few months focused on updating you on the stem cell research we are funding, and our plans for the future.

Right now we have 33 projects that we have funded in clinical trials. Those range from heart disease and stroke, to cancer, diabetes, ALS (Lou Gehrig’s disease), two different forms of vision loss, spinal cord injury and HIV/AIDS. We have also helped cure dozens of children battling deadly immune disorders. But as far as we are concerned we are only just getting started.

Over the course of the next few years, we have a goal of adding dozens more clinical trials to that list, and creating a pipeline of promising therapies for a wide range of diseases and disorders.

That’s why we are holding these free public events – something we try and do every year. We want to let you know what we are doing, what we are funding, how that research is progressing, and to get your thoughts on how we can improve, what else we can do to help meet the needs of the Patient Advocate community. Your voice is important in helping shape everything we do.

The first event is at the Gladstone Institutes in San Francisco on Wednesday, September 6th from noon till 1pm. The doors open at 11am for registration and a light lunch.

Gladstone Institutes

Here’s a link to an Eventbrite page that has all the information about the event, including how you can RSVP to let us know you are coming.

We are fortunate to be joined by two great scientists, and speakers – as well as being CIRM grantees-  from the Gladstone Institutes, Dr. Deepak Srivastava and Dr. Steve Finkbeiner.

Dr. Srivastava is working on regenerating heart muscle after it has been damaged. This research could not only help people recover from a heart attack, but the same principles might also enable us to regenerate other organs damaged by disease. Dr. Finkbeiner is a pioneer in diseases of the brain and has done ground breaking work in both Alzheimer’s and Huntington’s disease.

We have two other free public events coming up in October. The first is at UC Davis in Sacramento on October 10th (noon till 1pm) and the second at Cedars-Sinai in Los Angeles on October 30th (noon till 1pm). We will have more details on these events in the coming weeks.

We look forward to seeing you at one of these events and please feel free to share this information with anyone you think might be interested in attending.

BIO International Panel Showed Stem Cell Science Poised to Make a Difference in Medical Practice Soon

When the biotechnology trade association began holding annual conferences in 1993, they drew 1,400 to the first event. This year BIO International expected nearly 20,000 here in San Diego. Among the dozens of concurrent sessions each day of this four-day scramble, stem cells got one track on one day this year. But listening to the progress being made by our presenters yesterday, our field is set to grow at the pace this meeting has—and could dominate the medical sessions here within the next decade.


After setting the scene with our opening panel yesterday, four subsequent panels confirmed the vast near-term potential painted by the opening speakers. They revealed a field maturing rapidly and starting to be a valued research tool of the bigger companies that have dominated the biotech industry, at the same time it is starting to deliver therapies to patients.

The second panel displayed the robust power of stem cells to model disease better than animal models ever could. These cells also let researchers dive much deeper into the genetic causes of disease, particularly diseases with multiple genes involved. Anne Bang from the Sanford-Burnham Institute mentioned her role in a consortium organized by the National Institutes of Health that is looking at the many genes involved in a type of heart weakening called left ventricular hypertrophy. Because different ethnicities tend to respond differently to drugs used for the condition, the consortium teams are creating iPS-type stem cell lines from 125 Caucasian patients and 125 African-American patients with various forms of the condition.

Their goal is to personalize and improve therapy across both patients groups. The way cells behave in the lab can tell the researchers much more relevant information than most animal models, so drugs developed based off their discoveries should have a better chance of success. All four panelists agreed that the field needs enough drugs developed with these tools to show that they do indeed have a better success rate. That track record should start to develop over the next few years.

The third panel talked about the shift in the medical mindset that will happen when genetically modified stem cells can change the care of chronic diseases from daily therapy to cures. Louis Bretton of Calimmune discussed how his company is trying to do this for HIV, which we blogged about yesterday when they announced promising first phase results from their first four patients. Faraz Ali of bluebird bio showed that his company has already made this life-changing shift for two patients with the blood disorder Beta Thalassemia. Like most patients with the disease they had been dependent on regular transfusions to survive, but when they received transplants of their own stem cells genetically modified to produce the correct version of a protein that is defective in the disease, they were able to live without transfusions.

The fourth panel provided proof that the field is maturing in that they discussed the many hurdles and pitfalls in taking those final steps to prepare a cell therapy to be a commercial product. The three big hurdles—financing, regulatory approval and reimbursement by insurers—all required creativity by the companies outlined in the two case studies. They are working through them but it is anything but a straightforward path. This is the area I hear the most hand wringing about in the halls of meetings in our field.

The last panel showed that one way around some of those end stage hurdles is to reach across borders. Four panelists discussed specific examples of ways international collaborations have accelerated their work toward developing therapies. CIRM has more than 20 collaborative agreements with funding agencies around the world, many of them painstakingly nurtured by our former president Alan Trounson. He gave the final presentation of the panel talking about one of his new projects, building an international stem cell bank with enough cell lines that almost everyone could get donor cells that were immunologically matched.

Our board chair, Jonathan Thomas, moderated the last panel and ended with a tribute to Alan noting that his build-out of our international program would be one of his many lasting legacies.
Don Gibbons

Innovative Stem Cell Therapy for HIV Passes Milestone

Milestones are useful things. They measure how far we have come on a journey, and give us a sense that we are on the right path. One of the projects we are helping fund just passed a big milestone, and it’s given the researchers the go-ahead to move on to the next, perhaps even more important stage.

Left to Right: CIRM President and CEO C. Randal Mills, Calimmune CEO Louis Breton, Calimmume Chief Scientific Officer Geoff Symonds at today's news conference in San Diego.

Left to Right: CIRM President and CEO C. Randal Mills, Calimmune CEO Louis Breton and Calimmume Chief Scientific Officer Geoff Symonds at today’s news conference in San Diego.

The project is Calimmune’s stem cell gene modification study, which takes blood stem cells from people who are HIV-positive, genetically modifies them so they carry a gene that blocks the AIDS virus from infecting cells, and then re-introduces the modified cells to the patient. The hope is that those stem cells will then create a new blood system that is resistant to HIV.

The milestone it passed is that the Data Safety Monitoring Board (DSMB) looked at the results from the first group of four patients treated with this approach, found that there were no serious adverse events or dangerous side effects from it, and gave Calimmune the go-ahead to start treating the next group of patients.

In a news release we put out jointly with Calimmune, their CEO Louis Breton said this is a big step forward for them:

“We are very excited and encouraged by this development. This recommendation from the DSMB is an important step in bringing this one-time therapy to the patients, and takes us closer to our ultimate goal of eradicating AIDS.”

It’s a pretty big deal for us too, as our President and CEO C. Randal Mills noted in the same release:

“The mission of CIRM is to efficiently accelerate the development of stem cell treatments for patients suffering from unmet medical conditions. While still early in clinical development this announcement demonstrates real progress towards this mission. The accomplishments of Calimmune’s team is a great example of how CIRM partnerships are working to impact patient’s lives today.”

Now, just treating four people might not seem particularly impressive, after all HIV/AIDS has killed more than 25 million people worldwide and has infected another 25 million more – around 1.1 million here in the U.S. But every treatment has to begin with a simple premise, that whatever you do is not going to hurt the patient. Getting the green light from the Data Safety Monitoring Board, an independent panel of experts who review data and advise the researchers doing clinical trials, shows this approach appears to be safe.

The next step is to repeat this same process in 3 or 4 more patients but to give those patients a preconditioning regimen, treating them with a medication before returning their modified stem cells to them, to try and make the therapy more effective. This could show that the therapeutic approach, called Cal-1, is not only safe but also is working to protect patients against HIV.

If the safety data from that second group also looks good, then Calimmune can move on to the next group of patients. Each step, no matter how small, moves us ever closer to our end goal of developing a cure for HIV/AIDS.

That’s still a very distant goal right now, but with each milestone we pass it shows that we are heading in the right direction.

Want to know more about Calimmune’s path towards clinical trial? Check out Calimmune CEO Louis Breton’s recent video describing their progress towards a cure for HIV.

Kevin McCormack