One man’s story points to hope against a deadly skin cancer

One of the great privileges and pleasures of working at the stem cell agency is the chance to meet and work with some remarkable people, such as my colleagues here at CIRM and the researchers we support. But for me the most humbling, and by far the most rewarding experience, is having a chance to get to know the people we work for, the patients and patient advocates.

Norm Beegun, got stem cell therapy for metastatic melanoma

Norm Beegun, got stem cell therapy for metastatic melanoma

At our May Board meeting I got to meet a gentleman who exemplifies everything that I truly admire about the patients and patient advocates. His name is Norm Beegun. And this is his story.

Norm lives in Los Angeles. In 2002 he went to see his regular doctor, an old high school friend, who suggested that since it had been almost ten years since he’d had a chest x-ray it might be a good idea to get one. At first Norm was reluctant. He felt fine, was having no health problems and didn’t see the need. But his friend persisted and so Norm agreed. It was a decision that changed, and ultimately saved, his life.

The x-ray showed a spot on his lung. More tests were done. They confirmed it was cancer; stage IV melanoma. They did a range of other examinations to see if they could spot any signs of the cancer on his skin, any potential warnings signs that they had missed. They found nothing.

Norm underwent surgery to remove the tumor. He also tried several other approaches to destroy the cancer. None of them worked; each time the cancer returned; each time to a different location.

Then a nurse who was working with him on these treatments suggested he see someone named Dr. Robert Dillman, who was working on a new approach to treating metastatic melanoma, one involving cancer stem cells.

Norm got in touch with Dr. Dillman and learned what the treatment involved; he was intrigued and signed up. They took some cells from Norm’s tumor and processed them, turning them into a vaccine, a kind of personalized therapy that would hopefully work with Norm’s own immune system to destroy the cancer.

That was in 2004. Once a month for the next six months he was given injections of the vaccine. Unlike the other therapies he had tried this one had no side effects, no discomfort, no pain or problems. All it did was get rid of the cancer. Regular scans since then have shown no sign that the melanoma has returned. Theoretically that could be because the new therapy destroyed the standard tumor cells as well as the cancer stem cells that lead to recurrence.

Norm says when you are diagnosed with an incurable life-threatening disease, one with a 5-year survival rate of only around 15%, you will try anything; so he said it wasn’t a hard decision to take part in the clinical trial, he felt he had nothing to lose.

“I didn’t know if it would help me. I didn’t think I’d be cured. But I wanted to be a guinea pig and perhaps help others.”

When he was diagnosed his son had just won a scholarship to play football at the University of California, Berkeley. Norm says he feared he would never be able to see his son play. But thanks to cleverly scheduling surgery during the off-season and having a stem cell therapy that worked he not only saw his son play, he never missed a game.

Norm returned to Berkeley on May 21st, 2015. He came to address the CIRM Board in support of an application by a company called NeoStem (which has just changed its name to Caladrius Biosciences). This was the company that had developed the cell therapy for metastatic melanoma that Norm took.

“Talking about this is still very emotional. When I got up to talk to the CIRM Board about this therapy, and ask them to support it, I wanted to let them know my story, the story of someone who had their life saved by this treatment. Because of this I am here today. Because of this I was able to see my son play. But just talking about it left me close to tears.”

It left many others in the room close to tears as well. The CIRM Board voted to fund the NeoStem application, investing $17.7 million to help the company carry out a Phase 3 clinical trial, the last hurdle it needs to clear to prove to the Food and Drug Administration that this should be approved for use in metastatic melanoma.

Norm says he is so grateful for the extra years he has had, and he is always willing to try and support others going through what he did:

“I counsel other people diagnosed with metastatic melanoma. I feel that I want to help others, to give them a sense of hope. It is such a wonderful feeling, being able to show other people that you can survive this disease.”

When you get to meet people like Norm, how could you not love this job.

Faster, better, more efficient. Challenging? That too. An update on CIRM 2.0.

Changing direction is never easy. The greater the change the greater the likelihood you’ll have to make adjustments and do some fine-tuning along the way to make sure you get it right.

On January 1st of this year we made a big change, launching CIRM 2.0. Our President and CEO Dr. C. Randal Mills called it “a radical overhaul of the way the Agency does business.” This new approach puts the emphasis on patients, partnerships and speed and cuts down the time from application to funding of clinical-stage projects from around two years to just 120 days.

You can read more about 2.0 here.

So, several months into the program how are we doing?

Clinical stage of CIRM 2.0 has three programs

Clinical stage of CIRM 2.0 has three programs

Well, since January 1st we have had three application tracks under 2.0 that reflect our goal of accelerating therapies to patients with unmet medical needs. These focus on late stage work to either get a promising therapy into a clinical trial, to carry out a clinical trial, or to help a promising project move even faster.

Under those three programs we have had 12 applications for funding, for a total request of $111 million. With application deadlines the last business day of each month two of those were in January, two in February, three more in March and five in April.

As Dr. Mills told our governing Board when they met last week, that number is more than we were expecting:

 “When we started the program we calculated there’d be around one or two applications a month, not five. I don’t think having five applications a month is sustainable, but that’s probably just the backlog, the pent up demand for funding, working its way through the system. For now we can cope with that volume.”

Interestingly eight of those applications were for funding for clinical trials:

  • Two for Phase 1
  • One for Phase 2
  • Five for Phase 3

Last week our Board approved one of those Phase 3 trials (the last big hurdle to clear before the Food and Drug Administration will consider approving it for wider use), investing almost $18 million in NeoStem’s therapy for one of the deadliest forms of skin cancer, metastatic melanoma.

This is the first time we have ever funded a Phase 3 trial. So, quite a milestone for us. But it may well not be the last one. The Board also approved a project to conduct the late preclinical work needed to apply to conduct a trial in retinitis pigmentosa.

Dr. Mills said there are two clear patterns so far:

“We are getting a more mature portfolio of clinical stage programs for adjudication. We are also starting to see requests for accelerating activities, where we have made previous awards to researchers who now have identified new ways to accelerate that work and they are turning to us for help in doing that.”

Of the 12 applications received we have screened all of them within the 7-day target window to make sure they meet funding criteria. Some have been ruled out for not being within the scope of the award program. The accepted applications have all had budget reviews and been sent on for expert analysis within the slated time frames.

We had a couple of hiccups with our first review but that resulted from on-line technology and getting everyone comfortable with the new rules we were bringing in. The second review resulted in the first two awards by our Board last week, and the third review occurred yesterday.

“The bottom line is things are moving through and things are being weeded out. In March we had two clinical stage applications and one add-on funding application but that one add-on failed in screening. So, in general CIRM 2.0 is being well utilized. There’s no question we are significantly reducing application time from application to funding, attracting later stage applications. Clearly this has not been without its challenges but the team is doing a great job of managing everything.”

And remember this is only the first part of CIRM 2.0. We have two other programs, for Discovery or basic research and Translational research, that are being developed and we plan on rolling those out later this summer.

Stay tuned for more details on those programs.

Getting the right tools for the right job

Imagine a device that sits outside the body and works like a form of dialysis for a damaged liver, filtering out the toxins and giving the liver a chance to regenerate, and the patient a chance to avoid the need for a transplant.

Or imagine a method of enhancing the number of stem cells we can harvest or generate from umbilical cord blood, enabling us to use those stem cells and offer life-saving bone marrow transplants to all the patients who don’t have a matched donor.

Well, you may not have to imagine for too long. Yesterday, our governing Board approved almost $30 million in funding for our Tools and Technology Awards and two of the successful applications are for researchers hoping to turn those two ideas into reality.

The Tools n Tech awards may not have the glamor or cache of the big money awards that are developing treatments heading towards clinical trials, but they are nonetheless an essential part of what we do.

As our Board Chair Jonathan Thomas said in a news release they focus on developing new approaches or creating new ways of overcoming some of the biggest obstacles in stem cell research.

“Sometimes even the most promising therapy can be derailed by a tiny problem. These awards are designed to help find ways to overcome those problems, to bridge the gaps in our knowledge and ensure that the best research is able to keep progressing and move out of the lab and into clinical trials in patients.”

Altogether 20 awards were funded for a wide variety of different ideas and projects. Some focus on improving our ability to manufacture the kinds of cells we need for transplanting into patients. Another one plans to use a new class of genetic engineering tools to re-engineer the kind of stem cells found in bone marrow, making them resistant to HIV/AIDS. They also hope this method could ultimately be used to directly target the stem cells while they are inside the body, rather than taking the cells out and performing the same procedure in a lab and later transplanting them back.

Dr. Kent Leach, UC Davis School of Engineering

Dr. Kent Leach, UC Davis School of Engineering

One of the winners was Dr. Kent Leach from the University of California, Davis School of Engineering. He’s looking to make a new kind of imaging probe, one that uses light and sound to measure the strength and durability of bone and cartilage created by stem cells. This could eliminate the need for biopsies to make the same measurements, which is good news for patients and might also help reduce healthcare costs.

We featured Dr. Leach in one of our Spotlight videos where he talks about using stem cells to help repair broken bones that no longer respond to traditional methods.

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.


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

A Second Chance for a Spinal Cord Injury Trial, and a Powerful Reminder from Patient Advocates

Yesterday’s meeting of our governing Board was important for a number of reasons. First, the Board voted to invest some $32 million to try and get two promising projects into clinical trials – more on that in a minute – and also to try and attract some world-class researchers to California through our Research Leadership awards. It was also the first Board meeting for our new President, C. Randal Mills, Ph.D.

However, for me one of the most important parts of it was that it offered patient advocates a chance to come and talk to the Board directly, to share with them their hopes for stem cell research, and their needs in battling disabling conditions.

Yesterday a mother, Silvia Michelazzi, who suffered preeclampsia during her pregnancy and almost lost her child talked about the need for research to find better ways of preventing this deadly condition. Silvia’s daughter was born at 29 weeks and spent the first couple of months of life in a hospital neonatal intensive care unit.

One of the researchers we are funding, Dr. Mana Parast of UC San Diego, is doing some fascinating work in using iPS cells to better understand how preeclampsia works, and hopefully to find better ways of preventing it or treating it when it’s detected. We’ll be posting video of both talks in the next few weeks.

Earlier a group of individuals who have Parkinson’s disease talked to the Board about what it is like to live with that disease, to slowly lose control over their bodies and know that it was only going to get worse. They made a strong plea for more funding for stem cell research into this area.

To hear people like this speak is a powerful reminder of why we do this work; it puts a human face on the need for more research into so many areas, and why we need to do all that we can to accelerate that research, to find new treatments and cures.

Too often patients are left out of the discussion when it comes to funding research. At the stem cell agency we invite them into the room and welcome hearing from them. It’s not always easy to listen to what they have to say, particularly as we know some research is at an early stage of development and we won’t always be able to do what they want us to. But those voices are an important part of what this agency is all about. We were created by the people of California, so it’s important that the people feel they can come and talk to us any time they want.

From a business perspective yesterday’s meeting was very productive. The Board voted to invest $14.3 million in Asterias Biotherapeutics to move a stem cell therapy for spinal cord injury into clinical trials. This is the second time this approach will have been tried. The first was with Geron in 2010 and that trial, even though it ended earlier than expected because of financial reasons, showed the approach appears to be safe. Asterias is going to take it to the next level.

The other big award was $5.6 million to John Zaia at the City of Hope near Los Angeles to move his work in finding a treatment for HIV/AIDS into clinical trials.

Both are part of our Strategic Partnership program that requires them to provide matching funds for this work.

You can read all about those awards and the Research Leadership ones too in a news release we issued after the meeting.

kevin mccormack