Empowering and connecting California’s research ecosystem through shared labs

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A shared stem cell laboratory at UCLA

The California Institute for Regenerative Medicine (CIRM) has set ambitious goals in its new 5-year strategic plan. Made possible by renewed funding through Proposition 14, the plan lays out a roadmap for CIRM as the agency continues to advance world class science, deliver real world solutions, and provide opportunity for all.

In regenerative medicine (and many other fields), the lack of protocol standardization and lack of analytical toolkits make it difficult to access novel and reliable technology platforms.

CIRM recognizes these limitations, and as a response, the Agency has made it a goal in the “Prop 14 era” to develop next-generation competency hubs that empower and connect California’s research ecosystem.

One example of such competency hubs is the shared labs. The concept of shared labs isn’t new to CIRM. In fact, CIRM has awarded a total of 17 shared research laboratory grants to academic and nonprofit research institutions to provide lab space for innovative stem cell research and training.

CIRM will expand this shared lab model by creating networks of specialized competency hubs that offer knowledge and/or materials in cell and gene therapy development. These hubs will encourage collaborations and provide intra- and inter-institutional access to various competencies by sharing facilities, training, equipment, materials, protocols, and/or expertise.

As an example, a disease modeling competency hub would provide access to innovative models used to study diseases, collaborative researchers, shared facilities for conducting research, equipment and training programs for deriving or differentiating cell lines, etc. These collaborative environments would encourage researchers to work together with a team science approach, which would significantly accelerate discovery and therapy development.

Some of these hubs will also serve as a workforce training program for local and neighboring institutions. Most California state universities and community colleges have neither the financial nor experiential bandwidth for innovative research. They may, however, harbor a potentially diverse future workforce who could learn these techniques and use the technology platforms for small scale research. Training may also trigger the interest of the future workforce in pursuing the field of regenerative medicine.  

The competency hubs will also constitute part of the CIRM collaborative ecosystem making all their data available through the CIRM data infrastructure hub, also known as CIRM knowledge networks.

Finally, the network of competency hubs will greatly benefit the people of California by expanding geographic access to diverse communities and providing researchers with a unique opportunity of exposure to state-of-the-art platforms.

Visit this page to learn more about CIRM’s new 5-year Strategic Plan, and stay tuned as we share progress updates on our 5-year goals here on the Stem Cellar.

How two California researchers are advancing world class science to develop real life solutions

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In our recently launched 5-year Strategic Plan, the California Institute for Regenerative Medicine (CIRM) profiled two researchers who have leveraged CIRM funding to translate basic biological discoveries into potential real-world solutions for devastating diseases.

Dr. Joseph Wu is director of the Stanford Cardiovascular Institute and the recipient of several CIRM awards. Eleven of them to be exact! Over the past 10 years, Dr. Wu’s lab has extensively studied the application of induced pluripotent stem cells (iPSCs) for cardiovascular disease modeling, drug discovery, and regenerative medicine. 

Dr. Wu’s extensive studies and findings have even led to a cancer vaccine technology that is now being developed by Khloris Biosciences, a biotechnology company spun out by his lab. 

Through CIRM funding, Dr. Wu has developed a process to produce cardiomyocytes (cardiac muscle cells) derived from human embryonic stem cells for clinical use and in partnership with the agency. Dr. Wu is also the principal investigator in the first-in-US clinical trial for treating ischemic heart disease. His other CIRM-funded work has also led to the development of cardiomyocytes derived from human induced pluripotent stem cells for potential use as a patch.

Over at UCLA, Dr. Lili Yang and her lab team have generated invariant Natural Killer T cells (iNKT), a special kind of immune system cell with unique features that can more effectively attack tumor cells. 

More recently, using stem cells from donor cord-blood and peripheral blood samples, Dr. Yang and her team of researchers were able to produce up to 300,000 doses of hematopoietic stem cell-engineered iNKT (HSC–iNKT) cells. The hope is that this new therapy could dramatically reduce the cost of producing immune cell products in the future. 

Additionally, Dr. Yang and her team have used iNKT cells to develop both autologous (using the patient’s own cells), and off-the-shelf anti-cancer therapeutics (using donor cells), designed to target blood cell cancers.

The success of her work has led to the creation of a start-up company called Appia Bio. In collaboration with Kite Pharma, Appia Bio is planning on developing and commercializing the promising technology. 

CIRM has been an avid supporter of Dr. Yang and Dr. Wu’s research because they pave the way for development of next-generation therapies. Through our new Strategic Plan, CIRM will continue to fund innovative research like theirs to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and the world.

Visit this page to learn more about CIRM’s new 5-year Strategic Plan and stay tuned as we share updates on our 5-year goals here on The Stem Cellar.

Hitting our goals: Making good progress

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #5 was Advance.

A dictionary definition of progression is “The act of moving forward or proceeding in a course.” That’s precisely what we set out to do when we set one of the goals in our 2015 Strategic Plan. We wanted to do all that we could to make sure the work we were funding could advance to the next stage. The goal we set was:

Advance: Increase projects advancing to the next stage of development by 50%.

The first question we faced was what did we mean by progression and how were we going to measure it? The answer basically boiled down to this: when a CIRM award completes one stage of research and gets CIRM funding to move on to the next stage or to develop a second generation of the same device or therapy.

In the pre-2016 days we’d had some success, on average getting around nine progression events every year. But if we were going to increase that by 50 percent we knew we had to step up our game and offer some incentives so that the team behind a successful project had a reason, other than just scientific curiosity, to try and move their research to the next level.

So, we created a series of linkages between the different stages of research, so the product of each successful investment was the prerequisite for the next stage of development for the research or technology.

We changed the way we funded projects, going from offering awards on an irregular basis to having them happen according to a pre-defined schedule with each program type offered multiple times a year. This meant potential applicants knew when the next opportunity to apply would come, enabling them to prepare and file at the time that was best for them and not just because we said so. We also timed these schedules so that programs could progress from one stage to the next without interruption.

But that’s not all. We recognized that some people may be great scientists at one level but didn’t have the experience or expertise to carry their project forward. So, we created both an Accelerating Center and Translating Center to help them do that. The Translating Center helped projects do the work necessary to get ready to apply to the US Food and Drug Administration (FDA) for permission to start a clinical trial. The Accelerating Center helped the team prepare that application for the trial and then plan how that trial would be carried out.

Creating these two centers had an additional benefit; it meant the work that did progress did so faster and was of a higher quality than it might otherwise have been.

Putting all those new building blocks in place meant a lot of work for the CIRM team, on top of their normal duties. But, as always, the team rose to the challenge. By the end of December 2020, a total of 74 projects had advanced or progressed to the next level, an increase of 100 percent on our pre-2016 days.

When we were laying out the goals we said that “The full implementation of these programs will create the chassis of a machine that provides a continuous, predictable, and timely pathway for the discovery and development of promising stem cell treatments.” Thanks to the voter approved Proposition 14 we now have the fund to help those treatments realize that promise.

Hitting our Goals: Playing Matchmaker

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #3 was Partner.

In the musical “Fiddler on the Roof” two of the daughters sing about their hopes of finding a husband, through the services of a matchmaker:

Matchmaker, Matchmaker,
Make me a match,
Find me a find,
Catch me a catch

While CIRM isn’t in the business of finding husbands for young ladies, we have set up ourselves as matchmakers of a very different kind. Over the course of the last five years or more we have actively tried to find deep pocketed partners for some of the researchers we are funding. You could say we are changing the last line in that verse to “Catch me some cash.” And we do.

Our goal is to help these researchers have access to the kind of money they’re going to need to move their work into clinical trials and through the Food and Drug Administration (FDA) approval process, so they are available to people who need them. To do that we created what we call our Industry Alliance Program (IAP).

The goal of the IAP is simple, to be proactive in creating partnerships between industry and our grantees, helping develop direct opportunities for industry to partner with CIRM in accelerating the most promising stem cell, gene and regenerative medicine therapy programs to commercialization.

It takes a lot of money to move a promising idea out of the lab and into the arms, or other body parts, of patients; one recent estimate put that at around $1 billion. CIRM can help with providing the funding to get projects off the ground and into clinical trials, but as you get to larger clinical trials it gets a lot more expensive. The IAP brings in well-heeled investors to help cover those expense.

Back in 2015, when we were developing our Strategic Plan, we made these partnerships one of our Big 6 goals. And, as with everything we did in that plan, we set an ambitious target of “partnering 50% of unpartnered clinical projects with commercial partners.”

So, how did we go about trying to reach that goal? Our Business Development Team (Drs Shyam Patel and Sohel Talib) worked with large companies to help identify their strategic focus and then provided them with non-confidential information about projects we fund that might interest them. If they saw something they felt had promise we introduced them to the researchers behind that project. In essence, we played matchmaker.

But it wasn’t just about making introductions. We stayed involved as the two groups got to know each other, offering both scientific and legal advice, to help them overcome any reservations or obstacles they might encounter.

So how did we do? Pretty good I would have to say. By the end of 2020 we had partnered 63% of unpartnered clinical projects, 72 events altogether, generating almost $13 billion in additional investments in these projects. That money can help move these projects through the approvals process and ultimately, we hope, into the clinic.

But we’re not done. Not by a long shot. Now that we have achieved that goal we have our eyes set on even bigger things. We are now working on creating a new Strategic Plan that is considering bringing industry in to partner with projects at earlier stages or creating public-private partnerships to ensure there is enough manufacturing capacity for all the new therapies in the pipeline.

We have a lot of work to do. But thanks to the passage of Proposition 14 we now have the time and money we need to do that work. We’ve got a lot more matchmaking to do.

Hitting our Goals: Let’s start at the beginning shall we

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #3 was Discover.

When journalists write about science a lot of the attention is often focused on clinical trials. It’s not too surprising, that’s the stage where you see if treatments really work in people and not just in the lab. But long before you get to the clinical trial stage there’s a huge amount of work that has to be done. The starting point for that work is in the Discovery stage, if it works there it moves to the Translational stage, and only after that, assuming it’s still looking promising, does it start thinking about moving into the clinic.

The Discovery, or basic, stage of research is where ideas are tested to see if they have any promise and have the potential to lead to the development of a therapy or device that could ultimately help patients. In many ways the goal of Discovery research is to gain a better understanding of how, in our case, stem cells work, and how to harness that power to treat particular diseases or disorders.

Without a rigorous Discovery research program you can’t begin to create a pipeline of promising projects that you can advance towards patients. And of course having a strong Discovery program is not much use if you don’t have somewhere for those projects to advance to, namely Translational and ultimately clinical.

So, when we were laying out our Strategic Plan goals back in 2015 we wanted to create a pipeline for all three programs, moving the most promising ones forward. So we set an ambitious goal.

Introduce 50 new therapeutic or device candidates into development.

Now this doesn’t mean just fund 50 projects hoping to develop a new therapy or device. A lot of studies that are funded, particularly at the earliest stages, have a good idea that just doesn’t pan out. In fact one quite common definition of early research – in this case from Translational Medicine Communications – is “the earliest stage of research, conducted for the advancement of knowledge, often without any concern for its practical applications.

That’s not what we wanted. We aren’t in this to do research just for its own sake. We fund research because we want it to lead somewhere, we want it to have a practical application. We want to fund projects that actually ended up with something much more promising, a candidate that might actually work and was ready to move into the next level of research to test it further.

And we almost, almost made it to the 50-candidate goal. We got to 46 and almost certainly would have made it to 50 if we hadn’t run out of money. Even so, that’s pretty impressive. There are now 46 projects ready to move on, or are already moving on, to the next level of research.

Of course, there’s no guarantee that these will ultimately end up as an FDA-approved therapy or device. But if you don’t set goals, you’ll never score. And now, thanks to the passage of Proposition 14, we have a chance to support those projects as they move forward.

Budgeting for the future of the stem cell agency

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The CIRM Board discusses the future of the Stem Cell Agency

Budgets are very rarely exciting things; but they are important. For example, it’s useful for a family to know when they go shopping exactly how much money they have so they know how much they can afford to spend. Stem cell agencies face the same constraints; you can’t spend more than you have. Last week the CIRM Board looked at what we have in the bank, and set us on a course to be able to do as many of the things we want to, with the money we have left.

First some context. Last year CIRM spent a shade over $306 million on a wide range of research from Discovery, the earliest stage, through Translational and into Clinical trials. We estimate that is going to leave us with approximately $335 million to spend in the coming years.

A couple of years ago our Board approved a 5 year Strategic Plan that laid out some pretty ambitious goals for us to achieve – such as funding 50 new clinical trials. At the time, that many clinical trials definitely felt like a stretch and we questioned if it would be possible. We’re proving that it is. In just two years we have funded 26 new clinical trials, so we are halfway to our goal, which is terrific. But it also means we are in danger of using up all our money faster than anticipated, and not having the time to meet all our goals.

Doing the math

So, for the last couple of months our Leadership Team has been crunching the numbers and looking for ways to use the money in the most effective and efficient way. Last week they presented their plan to the Board.

It boiled down to a few options.

  • Keep funding at the current rate and run out of money by 2019
  • Limit funding just to clinical trials, which would mean we could hit our 50 clinical trial goal by 2020 but would not have enough to fund Discovery and Translational level research
  • Place caps on how much we fund each clinical trial, enabling us to fund more clinical trials while having enough left over for Discovery and Translational awards

The Board went for the third option for some good reasons. The plan is consistent with the goals laid out in our Strategic Plan and it supports Discovery and Translational research, which are important elements in our drive to develop new therapies for patients.

Finding the right size cap

Here’s a look at the size of the caps on clinical trial funding. You’ll see that in the case of late stage pre-clinical work and Phase 1 clinical trials, the caps are still larger than the average amount we funded those stages last year. For Phase 2 the cap is almost the same as the average. For Phase 3 the cap is half the amount from last year, but we think at this stage Phase 3 trials should be better able to attract funding from other sources, such as industry or private investors.

cap awards

Another important reason why the Board chose option three – and here you’ll have to forgive me for being rather selfish – is that it means the Administration Budget (which pays the salaries of the CIRM team, including yours truly) will be enough to cover the cost of running this research plan until 2020.

The bottom line is that for 2018 we’ll be able to spend $130 million on clinical stage research, $30 million for Translational stage, and $10 million for Discovery. The impact the new funding caps will have on clinical stage projects is likely to be small (you can see the whole presentation and details of our plan here) but the freedom it gives us to support the broad range of our work is huge.

And here is where to go if you are interested in seeing the different funding opportunities at CIRM.

Raising awareness about Rare Disease Day

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One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases. Since then we have funded 13 new clinical trials including four targeting rare diseases (retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s disease, and Duchenne’s Muscular Dystrophy). It’s a good start but clearly, with almost 7,000 rare diseases, this is just the tip of the iceberg. There is still so much work to do.

And all around the world people are doing that work. Today we have asked Emily Walsh, the Community Outreach Director at the Mesothelioma Cancer Alliance,  to write about the efforts underway to raise awareness about rare diseases, and to raise funds for research to develop new treatments for them.

“February 28th marks the annual worldwide event for Rare Disease Day. This is a day dedicated to raising awareness for rare diseases that affect people all over the world. The campaign works to target the general public as well as policy makers in hopes of bringing attention to diseases that receive little attention and funding. For the year 2017 it was decided that the focus would fall on “research,” with the slogan, “With research, possibilities are limitless.”

Getting involved for Rare Disease Day means taking this message and spreading it far and wide. Awareness for rare diseases is extremely important, especially among researchers, universities, students, companies, policy makers, and clinicians. It has long been known that the best advocates for rare diseases are the patients themselves. They use their specific perspectives to raise their voice, share their story, and shed light on the areas where additional funding and research are most necessary.

To see how you can help support the Rare Disease Day efforts this year, click here.

Groups like the Mesothelioma Cancer Alliance and the Mesothelioma Group are adding their voices to the cause to raise awareness about mesothelioma cancer, a rare form of cancer caused by exposure and inhalation of airborne asbestos fibers

Rare diseases affect 300 million people worldwide, but only 5% of them have an FDA approved treatment or cure. Malignant mesothelioma is among the 95 percent that doesn’t have a treatment or cure.

Asbestos has been used throughout history in building materials because of its fire retardant properties. Having a home with asbestos insulation, ceiling tiles, and roof shingles meant that the house was safer. However, it was found that once asbestos crumbled and became powder-like, the tiny fibers could become airborne and be inhaled and lodge themselves in lung tissue causing mesothelioma. The late stage discovery of mesothelioma is often what causes it to have such a high mortality rate. Symptoms can have a very sudden onset, even though the person may have been exposed decades prior.

Right now, treatment for mesothelioma includes the usual combination of chemotherapy, radiation, and surgery, but researchers are looking at other approaches to see if they can be more effective or can help in conjunction with the standard methods. For example one drug, Defactinib, has shown some promise in inhibiting the growth and spread of cancer stem cells – these are stem cells that can evade chemotherapy and cause patients to relapse.”

Some people might ask why spend limited resources on something that affects so few people. But the lessons we learn in developing treatments for a rare disease can often lead us to treatments for diseases that affect many millions of people.

But numbers aside, there is no hierarchy of need, no scale to say the suffering of people with Huntington’s disease is any greater or less than that of people with Alzheimer’s. We are not in the business of making value judgements about who has the greatest need. We are in the business of accelerating treatments to patients with unmet medical needs. And those suffering from rare disease are very clearly  people in need.

 


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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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