Confusing cancer to kill it

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Thomas Kipps, MD, PhD: Photo courtesy UC San Diego

Confusion is not a state of mind that we usually seek out. Being bewildered is bad enough when it happens naturally, so why would anyone actively pursue it? But now some researchers are doing just that, using confusion to not just block a deadly blood cancer, but to kill it.

Today the CIRM Board approved an investment of $18.29 million to Dr. Thomas Kipps and his team at UC San Diego to use a one-two combination approach that we hope will kill Chronic Lymphocytic Leukemia (CLL).

This approach combines two therapies, cirmtuzumab (a monoclonal antibody developed with CIRM funding, hence the name) and Ibrutinib, a drug that has already been approved by the US Food and Drug Administration (FDA) for patients with CLL.

As Dr. Maria Millan, our interim President and CEO, said in a news release, the need for a new treatment is great.

“Every year around 20,000 Americans are diagnosed with CLL. For those who have run out of treatment options, the only alternative is a bone marrow transplant. Since CLL afflicts individuals in their 70’s who often have additional medical problems, bone marrow transplantation carries a higher risk of life threatening complications. The combination approach of  cirmtuzumab and Ibrutinib seeks to offer a less invasive and more effective alternative for these patients.”

Ibrutinib blocks signaling pathways that leukemia cells need to survive. Disrupting these pathways confuses the leukemia cell, leading to its death. But even with this approach there are cancer stem cells that are able to evade Ibrutinib. These lie dormant during the therapy but come to life later, creating more leukemia cells and causing the cancer to spread and the patient to relapse. That’s where cirmtuzumab comes in. It works by blocking a protein on the surface of the cancer stem cells that the cancer needs to spread.

It’s hoped this one-two punch combination will kill all the cancer cells, increasing the number of patients who go into complete remission and improve their long-term cancer control.

In an interview with OncLive, a website focused on cancer professionals, Tom Kipps said Ibrutinib has another advantage for patients:

“The patients are responding well to treatment. It doesn’t seem like you have to worry about stopping therapy, because you’re not accumulating a lot of toxicity as you would with chemotherapy. If you administered chemotherapy on and on for months and months and years and years, chances are the patient wouldn’t tolerate that very well.”

The CIRM Board also approved $5 million for Angiocrine Bioscience Inc. to carry out a Phase 1 clinical trial testing a new way of using cord blood to help people battling deadly blood disorders.

The standard approach for this kind of problem is a bone marrow transplant from a matched donor, usually a family member. But many patients don’t have a potential donor and so they often have to rely on a cord blood transplant as an alternative, to help rebuild and repair their blood and immune systems. However, too often a single cord blood donation does not have enough cells to treat an adult patient.

Angiocrine has developed a product that could help get around that problem. AB-110 is made up of cord blood-derived hematopoietic stem cells (these give rise to all the other types of blood cell) and genetically engineered endothelial cells – the kind of cell that lines the insides of blood vessels.

This combination enables the researchers to take cord blood cells and greatly expand them in number. Expanding the number of cells could also expand the number of patients who could get these potentially life-saving cord blood transplants.

These two new projects now bring the number of clinical trials funded by CIRM to 35. You can read about the other 33 here.

 

 

 

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Stem cell agency funds Phase 3 clinical trial for Lou Gehrig’s disease

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At CIRM we don’t have a disease hierarchy list that we use to guide where our funding goes. We don’t rank a disease by how many people suffer from it, if it affects children or adults, or how painful it is. But if we did have that kind of hierarchy you can be sure that Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, would be high on that list.

ALS is a truly nasty disease. It attacks the neurons, the cells in our brain and spinal cord that tell our muscles what to do. As those cells are destroyed we lose our ability to walk, to swallow, to talk, and ultimately to breathe.

As Dr. Maria Millan, CIRM’s interim President and CEO, said in a news release, it’s a fast-moving disease:

“ALS is a devastating disease with an average life expectancy of less than five years, and individuals afflicted with this condition suffer an extreme loss in quality of life. CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurological condition for which there is currently no cure.”

Having given several talks to ALS support groups around the state, I have had the privilege of meeting many people with ALS and their families. I have seen how quickly the disease works and the devastation it brings. I’m always left in awe by the courage and dignity with which people bear it.

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I thought of those people, those families, today, when our governing Board voted to invest $15.9 million in a Phase 3 clinical trial for ALS run by BrainStorm Cell Therapeutics. BrainStorm is using mesenchymal stem cells (MSCs) that are taken from the patient’s own bone marrow. This reduces the risk of the patient’s immune system fighting the therapy.

After being removed, the MSCs are then modified in the laboratory to  boost their production of neurotrophic factors, proteins which are known to help support and protect the cells destroyed by ALS. The therapy, called NurOwn, is then re-infused back into the patient.

In an earlier Phase 2 clinical trial, NurOwn showed that it was safe and well tolerated by patients. It also showed evidence that it can help stop, or even reverse  the progression of the disease over a six month period, compared to a placebo.

CIRM is already funding one clinical trial program focused on treating ALS – that’s the work of Dr. Clive Svendsen and his team at Cedars Sinai, you can read about that here. Being able to add a second project, one that is in a Phase 3 clinical trial – the last stage before, hopefully, getting approval from the Food and Drug Administration (FDA) for wider use – means we are one step closer to being able to offer people with ALS a treatment that can help them.

Diane Winokur, the CIRM Board Patient Advocate member for ALS, says this is something that has been a long time coming:

CIRM Board member and ALS Patient Advocate Diane Winokur

“I lost two sons to ALS.  When my youngest son was diagnosed, he was confident that I would find something to save him.  There was very little research being done for ALS and most of that was very limited in scope.  There was one drug that had been developed.  It was being released for compassionate use and was scheduled to be reviewed by the FDA in the near future.  I was able to get the drug for Douglas.  It didn’t really help him and it was ultimately not approved by the FDA.

When my older son was diagnosed five years later, he too was convinced I would find a therapy.  Again, I talked to everyone in the field, searched every related study, but could find nothing promising.

I am tenacious by nature, and after Hugh’s death, though tempted to give up, I renewed my search.  There were more people, labs, companies looking at neurodegenerative diseases.

These two trials that CIRM is now funding represent breakthrough moments for me and for everyone touched by ALS.  I feel that they are a promising beginning.  I wish it had happened sooner.  In a way, though, they have validated Douglas and Hugh’s faith in me.”

These therapies are not a cure for ALS. At least not yet. But what they will do is hopefully help buy people time, and give them a sense of hope. For a disease that leaves people desperately short of both time and hope, that would be a precious gift. And for people like Diane Winokur, who have fought so hard to find something to help their loved ones, it’s a vindication that those efforts have not been in vain.

CIRM-funded stem cell clinical trial for spinal cord injury expands patient recruitment

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It’s always great to start the week off with some good news. Today we learned that the Food and Drug Administration (FDA) has given Asterias Biotherapeutics approval to expand the number and type of people with spinal cord injuries that it treats in their CIRM-funded clinical trial.

Up till now, Asterias has been treating people who have injuries at the C5-C7 level, those are the lowest levels of the cervical spine, near the base of the neck. Now they will be able to treat people with injuries at the C4 level, that’s not only higher up the neck but it’s also the second most common form of spinal cord injury.

In a news release Dr. Ed Wirth, Asterias’ Chief Medical Officer, says this is a vote of confidence from the FDA in the company’s AST-OPC1 stem cell therapy:

“FDA’s decision to allow the company to enroll qualified patients with C-4 level injuries is the result of the data supporting the safety of both AST-OPC1 and the procedure to inject the cells and means that the second most common cervical spinal cord injury population can now be eligible to receive AST-OPC1. The overall changes to the study protocol will enhance our ability to enroll qualified patient candidates for our current SCiStar study and we also expect the changes to help enrollment rates in a future, larger clinical study.”

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Photo courtesy Shepherd Center, Atlanta

People who are injured at the C4 level are typically paralyzed from the neck down and need constant help, while people with C5-C7 injuries typically have some use of their hands and arms. Caring for someone with a C4 injury is expensive, with lifetime costs estimated around $5 million. Anything that could help people recover some movement would not only reduce those costs but would, more importantly, also increase the quality of life for people.

Asterias is not only expanding the patient population they are working with, they are also expanding the window for treating the injury. Currently patients have to be enrolled from 14 to 30 days post injury. In this new C4 group that window has been extended to 21 to 42 days post injury.

The reason for that change is that because C4 is higher up in the neck, newly injured people often need to be placed on a ventilator to help stabilize them. These patients take a little more time to recover from the initial trauma before they are ready to be treated.

We have blogged several times (here, here and here) about the encouraging news from the Asterias trial and how it appears to be helping people with injuries at the C5-C7 level recover some movement in their arms and hands. In some cases, such as with Kris Boesen for example, the improvement has been quite dramatic. Now the hope is that this new patient population will see similar benefits.

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Kris Boesen, CIRM spinal cord injury clinical trial patient.

The study is being conducted at six centers in the U.S., including some here in California,  and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment.

Emotions and gratitude at changing of the guard at Stem Cell Agency

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Randy Mills and his family

Randy, as regular readers of this blog know, is, or rather was, the President and CEO of CIRM. James Harrison is less well known to the outside world but his imprint on CIRM, as our General Counsel and one of the key figures behind Proposition 71, is even bigger than that of Randy’s.

Randy came to the stem cell agency a little over three years ago and in pretty quick order completely refashioned us. Under his guidance CIRM 2.0 became a sleek, streamlined funding machine, turning what had been an almost two-year process from application to funding into one that took just 120 days. He revamped the frequency with which we offered specific programs, making it more predictable and so easier for researchers to know when the next round was coming up. He helped usher in a new Strategic Plan that is a blueprint for us until 2020.

But the changes he implemented were not just about the way we worked, it was also about how we worked and particularly how we worked together. He turned the agency into a true team, one where everyone felt they not only had a role to play but that what they did was important in determining the success of the agency.

Not surprisingly there was no shortage of people ready to praise him. CIRM Board Chair Jonathan Thomas (JT) thanked Randy for turning the agency around, transforming it into an organization that even the National Institutes of Health (NIH) now looks to as a model (more on that in a subsequent blog). Vice Chair Art Torres thanked Randy for his leadership and for his compassion toward patients, always putting them first in everything that he and the agency did. Board member Sherry Lansing called Randy “a genius and visionary”.

But perhaps the most moving tributes came from patients advocates.

Don Reed said; “When I first met Randy I didn’t like him. I thought CIRM was one of the best, if not the best, organization out there and who was this person to say they were going to come in and make it better. Well, you did Randy and we are all so very grateful to you for that.”

Adrienne Shapiro from Axis Advocacy, an organization dedicated to finding a cure for sickle cell disease, presented Randy with the “Heart of a Mother” award, thanking him for his tireless support of patients and their families.

Jake Javier, a participant in the Asterias spinal cord injury trial, wrote a note saying: “You positively affect so many through your amazing funding efforts for life changing research, and should be very proud of that. But something I will always remember is how personal and genuine you were while doing it. I hope you got the chance to meet as many of the people you helped as possible because I know they would remember the same.”

Randy – who is leaving to become President/CEO of the National Marrow Donor/Be The Match program – was clearly deeply moved by the tributes, but reminded everyone that he was leaving us in good hands. The Board named Dr. Maria Millan as the interim President and CEO, pending a meeting of a search committee to determine the steps for appointing a permanent replacement.

Randy praised Maria for her intelligence, compassion and vision:

“Maria Millan has been a great partner in all that we have achieved at CIRM. She was a key part of developing the Strategic Plan; she  understands it inside out and has been responsible for administering it. She is a wonderful leader and is going to be absolutely phenomenal.”

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James Harrison (left) with CIRM Board members Jonathan Thomas and Bert Lubin

The tributes for James Harrison were ever bit as moving. James has been a part of CIRM since before there was a CIRM. He helped draft Proposition 71, the ballot initiative that created the stem cell agency, and has played a key role since as General Counsel.

JT: “James has been a part of literally every decision and move that CIRM has made in its entire history. He’s been integral in everything. When I first came to CIRM, I was told by Bob Klein (JT’s predecessor as Chair) ‘Don’t brush your teeth without checking with James first’ suggesting a level of knowledge and expertise that was admirable.”

Jeff Sheehy “We would not be here without James. He organized the defense when we were sued by our opponents in the early days, through the various leadership challenges we had, all of the legal difficulties we had James was there to guide us and it’s been nothing short of extraordinary. Your brilliance and steadiness is amazing. While we are screaming and pulling our hair out there was James. Just saying his name makes me feel more relaxed.”

Sherry Lansing: “One thing I never worried about was our ethics, because you protected us at all times. You have such strong ethical values, you are always calm and rational and no matter what was going on you were always the rock who could explain things to everyone and deal with it with integrity.”

James is leaving to take a more active role in the law firm Remcho, Johansen & Purcell, where he is partner. Succeeding him as General Counsel is Scott Tocher, who has been at CIRM almost as long as James.

Randy; “To have someone like Scott come in and replace someone who wrote Proposition 71 speaks for the bench strength of the agency and how we are in very good hands.”

Art Torres joked “Scott has been waiting as long as Prince Charles has to take over the reins and we’re delighted to be able to work with him.”

We wish Randy and James great good luck in their next adventures.

 

Bridging the divide: stem cell students helping families with rare diseases become partners in research

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CIRM’s Bridges students and Rare Science’s families with rare diseases

Sometimes it’s the simplest things that make the biggest impact. For example, introducing a scientist to a patient can help them drive stem cell research forward faster than either one could do on their own.

Want proof? This year, students in CIRM’s Bridges to Stem Cell Research and Therapy program at California State University (CSU) San Marcos teamed up with parents of children with rare diseases, and the partnerships had a profound impact on all of them, one we hope might produce some long-term benefits.

Christina Waters, who helped create the partnerships, calls it “science with love.”

“We wanted to change the conversation and have researchers and families communicate, making families equal stakeholders in the research. The students bonded with the families and I truly feel that we made a difference in the lives of future researchers, in knowing how much their work can make a life changing impact on the lives of patients’ families who now have hope.”

The CIRM Bridges program helps prepare California’s undergraduate and master’s graduate students for highly productive careers in stem cell research. Students get a paid internship where they get hands-on training and education in stem cell research. They also work with patients and take part in outreach activities so they get an understanding of research that extends beyond the lab.

That’s where Christina Waters comes in. Christina is the founder of Rare Science, a non-profit group focused on rare diseases in children – we blogged about her work here – and she teamed up with CSU San Marcos to partner their Bridges students with five patient families with different rare diseases.

Cutting edge science

One of those families was Aaron Harding’s. Aaron’s son Jaxon has SYNGAP, a genetic disorder that can cause seizures, mental retardation, speech problems and autistic-like behavior. Two of the Bridges students who were doing their internship at ThermoFisher Scientific, Uju Nwizu and Emily Asbury, were given the task of using the gene-editing tool CRISPR Cas9 to help develop a deeper understanding of SYNGAP.

The students say it was an amazing experience:

Uju: “It had a huge impact on me. Every time I thought about SYNGAP I saw Jaxon’s face. This motivated me a lot.”

Emily: “People who work in labs everyday are most often working out the minutiae of research. They don’t often get a chance to see how their research can change or save the lives of real people. Meeting patients is so motivating because afterwards you aren’t just studying a mechanism, you now have a friend with the disease, so you can’t help but be personally invested in the search for a treatment.”

Emily and Uju are working to create iPSCs (induced pluripotent stem cells) that have the SYNGAP mutation. They hope these can be used to study the disease in greater depth and, maybe one day, lead to treatments for some of the symptoms.

Aaron says for families like his, knowing there are scientists working on his child’s disorder is a source of comfort, and hope:

“Personalizing diseases by connecting scientists with those they seek to impact is so important. Emily and Uju took this opportunity and ran with it, and that says a lot about them, and the team at ThermoFisher, taking on an exploring the unknown. That attitude is the heart of a scientist.”

Hearing stories like this is very gratifying, not just for the students and families involved, but for everyone here at CIRM. When we created the Bridges program our goal was to help students get the skills and experience needed to pursue a career in science. Thanks to the people at CSU San Marcos and Rare Science these students got a whole lot more.

Christina Waters: “We learned, we shared hope, we celebrated the courage of our families and the commitment of the students. It takes a village, and it is all of us working together that will make great changes for kids with rare diseases.”

For Uju and Emily, their experience in the Bridges program has made them doubly certain they want to pursue a career in science.

Uju: “I love stem cells and the promise they hold. After this program I hope to be part of a team that is committed to accelerating new stem cell therapies for rare and chronic diseases.”

Emily: “I’ve learned that I love research. After I finish my bachelor’s degree at CSU San Marcos I plan to pursue a graduate degree in molecular or cellular biology.”

 

Stem Cells Profile in Courage: Pat Furlong, Patient Advocate

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Pat Furlong: Photo by Colin McGuire – http://www.colinmcguire.com

One of the true joys for me in helping put together this year’s Annual Report was getting to know the patients and patient advocates that we profiled in the report. These are some extraordinary individuals and the short profiles we posted only touch the surface of just how extraordinary.

So, over the next few weeks we are going to feature four of these people at greater length, allowing them, in their own words, to talk about what makes them tic, and how they keep going in the face of what is often heartbreak and tragedy.

We begin with Pat Furlong, a Patient Advocate and the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (DMD).

DMD is the most common fatal, genetic childhood disorder, which affects approximately 1 out of every 3,500 boys each year worldwide. It’s a progressive muscle disorder that leads to loss of muscle function, meaning you lose your ability to walk, to use your arms, and ultimately to breathe. And because the heart is a muscle, that is often seriously affected. There is no cure, and treatment options are limited. At the time her sons were diagnosed life expectancy was in the teens.

Pat’s story:

“When my sons, Chris and Pat were diagnosed with DMD, at the ages of 4 and 6, there was nothing available for them. Doctors cared about them but they didn’t have the tools they needed, or the National Institutes of Health the money it needed to do research.

Doctors were faced with diagnosing a disease and saying “there’s nothing we can do”. And then parents like me, coming to them hearing there was nothing they could do, no hope, no help. When your son is diagnosed with something like this you are told go home and love them.

When I asked questions, I was often ignored or dismissed by some doctors.

When my sons were diagnosed with DMD I would drop them off at school and go walking and that would help me deal with the anger.

For me staying in this is to be able to say to Chris and Pat in the universe, when you were here I tried my very best and when you were gone I continued to try my best so that others would have advantages that you didn’t receive.

I haven’t stood back and said I can’t go on.

The family is all scarred, we all suffered this loss. It’s much more apparent when we are together, there are empty chairs, emptiness. If we go to a family gathering we wish Chris and Pat were here, could be married. Now there’s my husband and our two daughters. We have a granddaughter, who is wonderful, but still we are incomplete and we will live with that forever.

I am trained as a nurse and I find DMD equal parts fascinating disease, heartbreaking and painful. I try to emphasize the fascinating so I can keep going. There are frustrations; lack of money, the slow process of regulatory approval, but I have an incredible team of very smart people and we are passionate about change so that helps keep us going.

Your only interest can’t be DMD, it can’t be. For me it’s certainly a priority, but it’s not my only interest. I love to go to an art museum and see how creative people work. I love Cirque du Soleil because they do things with their muscles I can’t imagine. Going outside and seeing these things makes the world better.

I am interested in the expression of art, to see how people dress, to see how people are creative, I love creativity, I think the human spirit is pretty amazing and the creativity around it. I think we are all pretty amazing but sometimes we don’t say it enough.

I recently saw a woman on the subway with a pair of tennis shoes that said “you are beautiful” and people around her were looking at her shoes and smiling, just because of those shoes. We forget to interact, and that was such a simple way of doing that.

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I relax by doing yoga, 90-minute hot yoga, as often as I can. I’ve also done a number of half marathons, but I’m more a walker than a runner. I find getting outside or hot yoga makes me concentrate on what I’m doing so that I can’t think of anything else. I can put it down and think about nothing and whisper prayers to my sons and say am I doing the right thing, is there something I should be doing differently? It’s my time to think about them and meditate about what they think would be important.

You need to give your mind time to cope, so it’s putting your phone down and your computer away. It’s getting rid of those interruptions. To put the phone, the computer down and get in a hot room and do yoga, or run around outside, to look at a tree and think about the changing season, the universe, the sun. It’s an incredible break for the brain to be able to rest.

I think the disease has made us kinder people and more thoughtful. When Chris died, we found a notebook he kept. In it was written “the meaning of life is a life of meaning”. I think that’s where we have all landed, what we all strive for, a life of meaning.

 

 

 

Cured by Stem Cells

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To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual Report for 2016, now online, is our way of checking that we are still on the right path.

I think, without wishing to boast, that it’s safe to say not only are we on target, but we might even be a little bit ahead of schedule.

The Annual Report is chock full of facts and figures but at the heart of it are the stories of the people who are the focus of all that we do, the patients. We profile six patients and one patient advocate, each of whom has an extraordinary story to tell, and each of whom exemplifies the importance of the work we support.

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Brenden Whittaker: Cured

Two stand out for one simple reason, they were both cured of life-threatening conditions. Now, cured is not a word we use lightly. The stem cell field has been rife with hyperbole over the years so we are always very cautious in the way we talk about the impact of treatments. But in these two cases there is no need to hold back: Evangelina Padilla Vaccaro and Brenden Whittaker have been cured.

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Evangelina: Cured

 

In the coming weeks we’ll feature our conversations with all those profiled in the Annual Report, giving you a better idea of the impact the stem cell treatments have had on their lives and the lives of their family. But today we just wanted to give a broad overview of the Annual Report.

The Strategic Plan was very specific in the goals it laid out for us. As an agency we had six big goals, but each Team within the agency, and each individual within those teams had their own goals. They were our own mini-maps if you like, to help us keep track of where we were individually, knowing that every time an individual met a goal they helped the Team get closer to meeting its goals.

As you read through the report you’ll see we did a pretty good job of meeting our targets. In fact, we missed only one and we’re hoping to make up for that early in 2017.

But good as 2016 was, we know that to truly fulfill our mission of accelerating treatments to patients with unmet medical needs we are going to have do equally well, if not even better, in 2017.

That work starts today.

 

California’s stem cell agency rounds up the year with two more big hits

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CIRM Board meeting with  Jake Javier, CIRM Chair Jonathan Thomas, Vice Chair Sen. Art Torres (Ret.) and President/CEO Randy Mills

It’s traditional to end the year with a look back at what you hoped to accomplish and an assessment of what you did. By that standard 2016 has been a pretty good year for us at CIRM.

Yesterday our governing Board approved funding for two new clinical trials, one to help kidney transplant patients, the second to help people battling a disease that destroys vision. By itself that is a no small achievement. Anytime you can support potentially transformative research you are helping advance the field. But getting these two clinical trials over the start line means that CIRM has also met one of its big goals for the year; funding ten new clinical trials.

If you had asked us back in the summer, when we had funded only two clinical trials in 2016, we would have said that the chances of us reaching ten trials by the end of the year were about as good as a real estate developer winning the White House. And yet……..

Helping kidney transplant recipients

The Board awarded $6.65 million to researchers at Stanford University who are using a deceptively simple approach to help people who get a kidney transplant. Currently people who get a transplant have to take anti-rejection medications for the rest of their life to prevent their body rejecting the new organ. These powerful immunosuppressive medications are essential but also come with a cost; they increase the risk of cancer, infection and heart disease.

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CIRM President/CEO Randy Mills addresses the CIRM Board

The Stanford team will see if it can help transplant patients bypass the need for those drugs by injecting blood stem cells and T cells (which play an important role in the immune system) from the kidney donor into the kidney recipient. The hope is by using cells from the donor, you can help the recipient’s body more readily adjust to the new organ and reduce the likelihood the body’s immune system will attack it.

This would be no small feat. Every year around 17,000 kidney transplants take place in the US, and many people who get a donor kidney experience fevers, infections and other side effects as a result of taking the anti-rejection medications. This clinical trial is a potentially transformative approach that could help protect the integrity of the transplanted organ, and improve the quality of life for the kidney recipient.

Fighting blindness

The second trial approved for funding is one we are already very familiar with; Dr. Henry Klassen and jCyte’s work in treating retinitis pigmentosa (RP). This is a devastating disease that typically strikes before age 30 and slowly destroys a person’s vision. We’ve blogged about it here and here.

Dr. Klassen, a researcher at UC Irvine, has developed a method of injecting what are called retinal progenitor cells into the back of the eye. The hope is that these cells will repair and replace the cells damaged by RP. In a CIRM-funded Phase 1 clinical trial the method proved safe with no serious side effects, and some of the patients also reported improvements in their vision. This raised hopes that a Phase 2 clinical trial using a larger number of cells in a larger number of patients could really see if this therapy is as promising as we hope. The Board approved almost $8.3 million to support that work.

Seeing is believing

How promising? Well, I recently talked to Rosie Barrero, who took part in the first phase clinical trial. She told me that she was surprised how quickly she started to notice improvements in her vision:

“There’s more definition, more colors. I am seeing colors I haven’t seen in years. We have different cups in our house but I couldn’t really make out the different colors. One morning I woke up and realized ‘Oh my gosh, one of them is purple and one blue’. I was by myself, in tears, and it felt amazing, unbelievable.”

Amazing was a phrase that came up a lot yesterday when we introduced four people to our Board. Each of the four had taken part in a stem cell clinical trial that changed their lives, even saved their lives. It was a very emotional scene as they got a chance to thank the group that made those trials, those treatments possible.

We’ll have more on that in a future blog.

 

 

 

 

With an eye toward 2020, CIRM looks at clinical milestones achieved in 2016

strategy-wideOne year ago, CIRM announced its strategic plan for the next five years. It’s a bold vision to maximize our impact in stem cell research by accelerating stem cell treatments to patients with unmet medical needs.

Our strategic plan, which can be found on our website, details how CIRM will invest in five main program areas including infrastructure, education, discovery, translation and clinical research. While CIRM has invested in these areas in the past, we are doing so now with a renewed focus to make sure our efforts have a lasting impact in California and more importantly for patients.

Now that a year has passed, it’s time to review our progress and look ahead to the next four years.

Our Progress

2016 was a very productive year. On the infrastructure side, CIRM successfully launched the Translating and Accelerating Centers, awarding both grants to QuintilesIMS. The Translating Center supports preclinical research that’s ready to advance to clinical trials but still needs approval by the US Food and Drug Administration (FDA). The Accelerating Center picks up where the Translating Center leaves off and offers support and management services for clinical trial projects to ensure that they succeed. Collectively called The Stem Cell Center, the goal of this new infrastructure is to increase efficiency and shorten the time it takes to get human stem cell trials up and running.

On the research side in 2016, CIRM funded over 70 promising stem cell projects ranging from education to discovery, translational and clinical projects. While of these areas are important to invest in, CIRM has shifted its focus to funding clinical trials in hopes that one or more of these trials will develop into an approved therapy for patients. So far, we’ve funded 25 trials, 22 of which are currently active since CIRM was established.

In our strategic plan, we gave ourselves the aggressive goal of funding 50 new clinical trials by 2020, which equates to 10 new trials per year. So far in 2016, we’ve funded eight clinical trials and tomorrow at our December ICOC meeting, our Governing Board will determine whether we meet our yearly clinical milestone of 10 trials by considering two more for funding.

The first trial is testing a stem cell treatment that could improve the outcome of kidney transplants. For normal kidney transplants, the recipient is required to take immunosuppressive drugs to prevent their body from rejecting the donated organ. This clinical trial aims to bypass the need for these drugs, which carry an increased risk of cancer, infection and heart disease, by injecting blood stem cells and other immune cells from the kidney donor into the patient receiving the kidney. You can read more about this proposed trial here.

The second clinical trial is a stem cell derived therapy to improve vision in patients with a degenerative eye disease called retinitis pigmentosa. This disease destroys the light sensing cells at the back of the eye and has no cure. The trial hopes that by transplanting stem cell derived retinal progenitor cells into the back of the eye, these injected cells will secrete factors that will keep the cells in the eye healthy and possibly improve a patient’s vision. You can read more about this proposed trial here.

Our Future

No matter the outcome at tomorrow’s Board meeting, I think our agency should be proud of its accomplishments since launching our strategic plan. The eight clinical trials we’ve funded this year are testing stem cell therapies for diseases including muscular dystrophy, kidney disease, primary immune diseases, and multiple types of cancer and blood disorders.

At this pace, it seems likely that we will achieve many of the goals in our strategic plan including our big goal of 50 new clinical trials. But pride and a sense of accomplishment are not what CIRM is ultimately striving for. Our mission and the reason why we exist are to help people and improve their lives. I’ll leave you with a quote from our President and CEO Randy Mills:

CIRM CEO and President, Randy Mills.

Randy Mills

“In everything we do there is a real sense of urgency, because lives are at stake. Our Board’s support for these programs highlights how every member of the CIRM team shares that commitment to moving the most promising research out of the lab and into patients as quickly as we can.”


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Translating great stem cell ideas into effective therapies

alzheimers

CIRM funds research trying to solve the Alzheimer’s puzzle

In science, there are a lot of terms that could easily mystify people without a research background; “translational” is not one of them. Translational research simply means to take findings from basic research and advance them into something that is ready to be tested in people in a clinical trial.

Yesterday our Governing Board approved $15 million in funding for four projects as part of our Translational Awards program, giving them the funding and support that we hope will ultimately result in them being tested in people.

Those projects use a variety of different approaches in tackling some very different diseases. For example, researchers at the Gladstone Institutes in San Francisco received $5.9 million to develop a new way to help the more than five million Americans battling Alzheimer’s disease. They want to generate brain cells to replace those damaged by Alzheimer’s, using induced pluripotent stem cells (iPSCs) – an adult cell that has been changed or reprogrammed so that it can then be changed into virtually any other cell in the body.

CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and Alzheimer’s – which has no cure and no effective long-term treatments – clearly represents an unmet medical need.

Another project approved by the Board is run by a team at Children’s Hospital Oakland Research Institute (CHORI). They got almost $4.5 million for their research helping people with sickle cell anemia, an inherited blood disorder that causes intense pain, and can result in strokes and organ damage. Sickle cell affects around 100,000 people in the US, mostly African Americans.

The CHORI team wants to use a new gene-editing tool called CRISPR-Cas9 to develop a method of editing the defective gene that causes Sickle Cell, creating a healthy, sickle-free blood supply for patients.

Right now, the only effective long-term treatment for sickle cell disease is a bone marrow transplant, but that requires a patient to have a matched donor – something that is hard to find. Even with a perfect donor the procedure can be risky, carrying with it potentially life-threatening complications. Using the patient’s own blood stem cells to create a therapy would remove those complications and even make it possible to talk about curing the disease.

While damaged cartilage isn’t life-threatening it does have huge quality of life implications for millions of people. Untreated cartilage damage can, over time lead to the degeneration of the joint, arthritis and chronic pain. Researchers at the University of Southern California (USC) were awarded $2.5 million to develop an off-the-shelf stem cell product that could be used to repair the damage.

The fourth and final award ($2.09 million) went to Ankasa Regenerative Therapeutics, which hopes to create a stem cell therapy for osteonecrosis. This is a painful, progressive disease caused by insufficient blood flow to the bones. Eventually the bones start to rot and die.

As Jonathan Thomas, Chair of the CIRM Board, said in a news release, we are hoping this is just the next step for these programs on their way to helping patients:

“These Translational Awards highlight our goal of creating a pipeline of projects, moving through different stages of research with an ultimate goal of a successful treatment. We are hopeful these projects will be able to use our newly created Stem Cell Center to speed up their progress and pave the way for approval by the FDA for a clinical trial in the next few years.”