CIRM funded study uses drug development in a dish for treatment of heart arrhythmias

Image Credit: Center for Disease Control and Prevention (CDC)

Cardiac (heart) arrhythmias occur when electrical impulses that coordinate your heartbeats don’t work properly, causing your heart to beat too fast, too slow, or in an irregular manner. In the U.S. alone, almost one million individuals are hospitalized every year for heart arrhythmias. Close to 300,000 individuals die of sudden arrhythmic death syndrome every year, which occurs when there is a sudden loss of blood flow resulting from the failure of the heart to pump effectively. Unfortunately, drugs to treat arrhythmias have liabilities and several drugs have been pulled from the market due to serious side effects. Mexiletine is one potential drug for heart arrhythmias that has liabilities and potential side effects.

That is why a CIRM funded study ($6.3 million) conducted by John Cashman, Ph.D. at the Human BioMolecular Research Institute in San Diego looked at re-engineering mexiletine in a way that the drug could still produce a desired result and not be as toxic.

The study used induced pluripotent stem cells (iPSCs), a type of stem cell “reprogrammed” from the skin or blood of patients that can be used to make virtually any kind of cell. iPSCs obtained for the study were from a healthy patient and from one with a type of heart arrhythmia. The healthy and arrhythmia iPSCs were then converted into cardiomyocytes, a type of cell that makes up the heart muscle.

By using their newly created healthy cardiomyocytes and those with the arrhythmia defect, Cashman and his team were able to carry out drug development in a dish. This enabled them to attempt to lessen drug toxicity while still potentially treating heart arrhythmias. The team was able to modify mexiletine such that is was less toxic and found that it could potentially decrease a patient’s risk of developing ventricular tachycardia (a fast, abnormal heart rate) and ventricular fibrillation (an abnormal heart rhythm), both of which are types of heart arrhythmias.

“The new compounds may lead to treatment applications in a whole host of cardiovascular conditions that may prove efficacious in clinical trials,” said Cashman in a press release. “As antiarrhythmic drug candidate drug development progresses, we expect the new analogs to be less toxic than current therapeutics for arrhythmia in congenital heart disease, and patients will benefit from improved safety, less side effects and possibly with significant cost-savings.”

The team hopes that their study can pave the way for future research in which cells in a dish can be used to lessen the toxicity of a potential drug candidate while still producing a desired result for different diseases and conditions.

The full study was published in ACS Publications.

Identifying the visually impaired patients most likely to benefit from jCyte’s stem cell therapy

We have written about jCyte many times on The Stem Cellar. For one reason, they are showing really encouraging results in their treatment for retinitis pigmentosa (RP). And now they have taken an even deeper dive into those results and identified which patients may be most likely to benefit from the therapy.

RP is a rare genetic disorder that slowly destroys the rods and cones, the light sensing cells in the back of the eye. If you look at the image below the one on the left shows normal vision, the one on the right shows what happens with RP. At first you start to lose night vision, then other parts of your vision are slowly eroded until you are legally blind.

RP starts early, often people are diagnosed in their teens and are legally blind by middle age. There is no treatment, no cure. It’s estimated that as many as 100,000 people in the US have RP, as many as two million worldwide.

That’s where jCyte comes in. They developed jCell, a therapy using adult stem cells that have been changed into human retinal progenitor cells (hRPCs). These are injected into the back of the eye where they secrete small proteins called neurotrophic factors.

Dr. Henry Klassen, one of the founders of jCyte, says jCell works by preserving the remaining photoreceptors in the eye, and helping them bounce back.

“Typically, people think about the disease as a narrowing of this peripheral vision in a very nice granular way, but that’s actually not what happens. What happens in the disease is that patients lose like islands of vision. So, what we’re doing in our tests is actually measuring […] islands that the patients have at baseline, and then what we’re seeing after treatment is that the islands are expanding. It’s similar to the way that one would track, let’s say a tumor, in oncology of course we’re looking for the opposite effect. We’re looking for the islands of vision to expand.”

And in patients treated with jCell those islands of vision did expand. The team followed patients for one-year post treatment and found that patients given the highest dose, six million cells, experienced the biggest improvement and were able to read, on average, 16 more letters on a standard eye chart than they had been before treatment. In comparison people given a sham or placebo treatment only had an improvement of less than two letters.

This group also experienced improvements in their peripheral vision, their ability to distinguish objects in the foreground from the background and were better able to get around in low light.

But that’s not all. Dr. Sunil Srivastava, with the Cleveland Clinic Cole Eye Institute, did a detailed analysis of patients treated in the trial and identified central foveal thickness (CFT- the part of the eye located in the center of the retina) as an important marker for who would be most likely to benefit from jCell. People who started out with a higher CFT score were most likely to get the biggest benefits.

In a news release, jCyte CEO Dr. Shannon Blalock said the findings are really encouraging: “We look forward to working closely with our scientific advisory board and principal investigators to apply these key learnings to our upcoming pivotal study of jCell to optimize its probability of success in an effort to advance the clinical development program of our RMAT designated therapy for RP patients who currently have no treatment options.”

Remembering Eli Broad, philanthropist and stem cell champion

Eli Broad, Photo by Nancy Pastor

The world of stem cell research lost a good friend this weekend. Eli Broad, a generous supporter of science, education and the arts, passed away at the age of 87.

Eli came from humble origins, born in the Bronx to an immigrant father who worked as a house painter and a mother who was a seamstress. He went to Michigan State University, working a number of jobs to pay his way, including selling women’s shoes, working as a door-to-door salesman for garbage disposal units, and delivering rolls of film to be developed. He graduated in three years and then became the youngest person ever to pass the CPA exam in Michigan.

He started out as an accountant but quickly switched to housing and development and was a millionaire by the time he was 30. As his wealth grew so did his interest in using that money to support causes dear to him and his wife Edythe.

With the passage of Proposition 71 in 2004 Broad put up money to help create the Broad Stem Cell Centers at UCLA, UC San Francisco and the University of Southern California. Those three institutions became powerhouses in stem cell research and the work they do is a lasting legacy to the generosity of the Broads.

Rosa Dilani, histology core manager at the Eli and Edythe Broad CIRM Center, explains the lab’s function to Eli Broad after the Oct. 29 ribbon cutting of the new building. In the background are U.S. Rep. Lucille Roybal-Allard (in purple) and Bob Klein in gray suit.

“Science has lost one of its greatest philanthropic supporters,” says Jonathan Thomas, PhD, JD, Chair of the CIRM Board. ” Eli and Edye Broad set the table for decades of transformative work in stem cell and gene therapy through their enthusiastic support for Proposition 71 and funding at a critical time in the early days of regenerative medicine. Their recent additional generous contributions to USC, UCLA and UCSF helped to further advance that work.  Eli and Edye understood the critical role of science in making the world a better place.  Through these gifts and their enabling support of the Broad Institute with Harvard and MIT, they have left a lasting legacy in the advancement of medicine that cannot be overstated.”

Through the Broad Foundation he helped fund groundbreaking work not just in science but also education and the arts. Gerun Riley, President of the Broad Foundation says Eli was always interested in improving the lives of others.

“As a businessman Eli saw around corners, as a philanthropist he saw the problems in the world and tried to fix them, as a citizen he saw the possibility in our shared community, and as a husband, father, mentor and friend he saw the potential in each of us.”

Eli and Edythe Broad

New technique maps out diversity and location of cells in tissue or tumor

Image Description: Alex Marson is part of a team of researchers who developed a new technique to map the specialized diversity and spatial location of individual cells within a tissue or tumor. Photo Credit: Anastasiia Sapon

All the cells in your body work together and each can have a different role. Their individual function not only depends on cell type, but can also depend on their specific location and surroundings.

A CIRM supported and collaborative study at the Gladstone Institutes, UC San Francisco (UCSF), and UC Berkeley has developed a more efficient method than ever before to simultaneously map the specialized diversity and spatial location of individual cells within a tissue or a tumor.

The technique is named XYZeq and involves segmenting a tissue into microscopic regions. Within each of these microscopic grids, each cell’s genetic information is analyzed in order to better understand how each particular cell functions relative to its spacial location.

For this study, the team obtained tissue from mice with liver and spleen tumors. A slice of tissue was then placed on a slide that divides the tissue into hundreds of “microwells” the size of a grain of salt. Each cell in the tissue gets tagged with a unique “molecular barcode” that represents the microwell it’s contained in, much like a zip code. The cells are then mixed up and assigned a second barcode to ensure that each cell within a given square can be individually identified, similar to a street address within a zip code. Finally, the genetic information in the form of RNA from each cell is analyzed. Once the results are obtained, both barcodes tell the researchers exactly where in the tissue it came from.

The team found that some cell types located near the liver tumor were not evenly spaced out. They also found immune cells and specific types of stem cells clustered in certain regions of the tumor. Additionally, certain stem cells had different levels of some RNA molecules depending on how far they resided from the tumor.

The researchers aren’t entirely sure what this pattern means, but they believe that it’s possible that signals generated by or near the tumor affect what nearby cells do.

In a press release, Alex Marson, M.D., Ph.D., a senior author of the study, elaborates on what the XYZeq technology could mean for disease modeling.

“I think we’re actually taking a step toward this being the way tissues are analyzed to diagnose, characterize, or study disease; this is the pathology of the future.”

The full results of the study were published in Science Advances.

Hitting our Goals: Accelerating to the finish line

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 #6 was Accelerate.

Ever wonder how long it takes for a drug or therapy to go from basic research to approval by the US Food and Drug Administration (FDA)? Around 12 years on average is the answer. That’s a long time. And it can take even longer for stem cell therapies to go that same distance.

There are a lot of reasons why it takes so long (safety being a hugely important element) but when we were sitting down in 2015 to put together our Strategic Plan we wanted to find a way to speed up that process, to go faster, without in any way reducing the focus on safety.

So, we set a goal of reducing the time it takes from identifying a stem cell therapy candidate to getting an Investigational New Drug (IND) approval from the FDA, which means it can be tested in a clinical trial. At the time it was taking us around eight years, so we decided to go big and try to reduce that time in half, to four years.

Then the question was how were we going to do that? Well, before we set the goal we did a tour of the major biomedical research institutions in California – you know, University of California Los Angeles (UCLA) UC San Francisco, Stanford etc. – and asked the researchers what would help them most. Almost without exception said “a clearing house”, a way to pair early stage investigators with later stage partners who possess the appropriate expertise and interest to advance the project to the next stage of development, e.g., helping a successful basic science investigator find a qualified partner for the project’s translational research phase.

So we set out to do that. But we didn’t stop there. We also created what we called Clinical Advisory Panels or CAPs. These consisted of a CIRM Science Officer with expertise on a particular area of research, an expert on the kind of research being done, and a Patient Representative. The idea was that CAPs would help guide and advise the research team, helping them overcome specific obstacles and get ready for a clinical trial. The Patient Representative could help the researchers understand what the needs of the patient community was, so that a trial could take those into account and be more likely to succeed. For us it wasn’t enough just to fund promising research, we were determined to do all we could to support the team behind the project to advance their work.

How did we do. Pretty good I would have to say. For our Translational stage projects, the average amount of time it took for them to move to the CLIN1 stage, the last stage before a clinical trial, was 4.18 years. For our CLIN1 programs, 73 percent of those achieved their IND within 2 years, meaning they were then ready to actually start an FDA-sanctioned clinical trial.

Of course moving fast doesn’t guarantee that the therapy will ultimately prove effective. But for an agency whose mission is “to accelerate stem cell therapies to patients with unmet medical needs”, going slow is not an option.

CIRM funded stem cell therapy could one day help stroke and dementia patients

Image Description: Microscope images showing brain tissue that has been damaged by white matter stroke (left) and then repaired by the new glial cell therapy (right). Myelin (seen in red), is a substance that protects the connections between neurons and is lost due to white matter stroke. As seen at right, the glial cell therapy (green) restores lost myelin and improves connections in the brain. | Credit: UCLA Broad Stem Cell Research Center/Science Translational Medicine

Dementia is a general term that describes problems with memory, attention, communication, and physical coordination. One of the major causes of dementia is white matter strokes, which occurs when multiple strokes (i.e. a lack of blood supply to the brain) gradually damages the connecting areas of the brain (i.e. white matter).

Currently, there are no therapies capable of stopping the progression of white matter strokes or enhancing the brain’s limited ability to repair itself after they occur.

However, a CIRM-funded study ($2.09 million) conducted by S. Thomas Carmichael, M.D., Ph.D. and his team at UCLA showed that a one-time injection of an experimental stem cell therapy can repair brain damage and improve memory function in mice with conditions that mimic human strokes and dementia.

The therapy consists of glial cells, which are a special type of cell present in the central nervous system that surround and protect neurons. The glial cells are derived from induced pluripotent stem cells (iPSCS), stem cells that are derived from skin or blood cells through the process of reprogramming and have the ability to become virtually any type of cell.

Dr. Carmichael and his team injected the newly developed glial cells into the brains of mice that had damage similar to humans in the early to middle stages of dementia. The team found that the cell therapy traveled to the damaged areas of the brain and secreted chemicals that stimulated the brain’s own stem cells to start repairing the damage. This not only limited the progression of damage, but also enhanced the formation of new neural connections and increased the production of myelin, a fatty substance that covers and protects neurons.

In a press release from UCLA, Francesca Bosetti, Ph.D., Pharm.D., Program Director at the National Institute of Neurological Disorders and Strokes, was optimistic about what these findings could mean for patients with strokes or dementia.

“These preliminary results suggest that glial cell-based therapies may one day help combat the white matter damage that many stroke and vascular dementia patients suffer every year.”

Another interesting finding from this study is that even if the injected cells were eliminated a few months after they had been transplanted, the mice’s recovery was unaffected. The researchers believe that this indicates that the therapy primarily serves as a way to stimulate the brain’s own repair process.

In the same press release, Dr. Carmichael elaborates on this concept.

“Because the cell therapy is not directly repairing the brain, you don’t need to rely on the transplanted cells to persist in order for the treatment to be successful.”

The team is now conducting the additional studies necessary to apply to the Food and Drug Administration (FDA) for permission to test the therapy in a clinical trial in humans. If the therapy is shown to be safe and effective through clinical trials in humans, the team envisions that it could be used at hospitals as a one-time treatment for people with early signs of white matter stroke.

The full results of this study were published in Science Translational Medicine.

Regulated, Reputable and Reliable: FDA’s Taking Additional Steps to Advance Safe and Effective Regenerative Medicine Products

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research

In February 2020, CIRM presented a series of benchmarks for the responsible delivery of stem cell and regenerative medicine products. These benchmarks are outlined in the publication Regulated, reliable and reputable: Protect patients with uniform standards for stem cell treatments. In a nutshell, CIRM advocates for the delivery of regenerative medicine products in a context where:

  • The product is authorized by the Food and Drug Administration (FDA) and is overseen by an IRB or ethics board,
  • The treatment is delivered by qualified doctors, nurses, and technicians,
  • Treatment occurs at a clinical treatment center with expertise in regenerative medicine, and
  • There is ongoing monitoring and follow-up of patients.

On April 21 of 2021, Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research, indicated the FDA’s intent to ensure new regenerative medicine products are FDA-authorized. Specifically, the FDA will require product developers to obtain an Investigational New Drug or IND authorization. In his news release Dr. Marks says the agency is willing to exercise more enforcement of these rules should clinics or therapy producers fail to follow these guidelines.

“These regenerative medicine products are not without risk and are often marketed by clinics as being safe and effective for the treatment of a wide range of diseases or conditions, even though they haven’t been adequately studied in clinical trials. We’ve said previously and want to reiterate here – there is no room for manufacturers, clinics, or health care practitioners to place patients at risk through products that violate the law, including by not having an IND in effect or an approved biologics license. We will continue to take action regarding unlawfully marketed products.”

IND authorization is particularly important as the agency pays close attention to how the product is produced and whether there is a scientific rationale and potential clinical evidence that it may be effective against the specific disease condition. All CIRM-funded clinical trials and all trials conducted in the CIRM Alpha Stem Cell Clinics Network must have IND authorization.

Regenerative medicine products are generally created from human cells or tissues. These products are frequently referred to as “living medicines.” The “living” nature of these products is what contributes to their remarkable potential to relieve, stop or reverse disease in a durable or sustainable manner.

The risk with unregulated products is that there is no assurance that they have been  produced in a quality controlled process or manner  where all components of the  injected material have been well characterized and studied for safety and efficacy for a given disease as well as a specific site in the body. In addition, there is no way to ensure that unregulated products meet standards or quality specifications such as ensuring that they have the active and beneficial component while making sure that they do not include harmful contaminants..  There have been documented examples of patients being severely injured by unregulated and inadequately characterized products. For example, in 2017 three Florida women were blinded by an unauthorized product.  Dr. George Daley, a stem cell expert and the Dean of Harvard Medical School, described the clinic operators as “charlatans peddling the modern equivalent of snake oil.”

To receive FDA authorization, detailed scientific data and well controlled clinical data are required to ensure safety and a demonstration that  the product is safe has the potential to improve or resolve the patient’s disease condition.

While it seems both important and self-evident that stem cell products be safe and effective and supported by evidence they can impact the patient’s disease condition, that doesn’t always happen. Unfortunately, too many patients have experienced unnecessary medical risks and financial harm from unauthorized treatments. CIRM applauds the FDA for taking additional steps to advance regenerative medicine products where the clinical benefits of such therapies outweigh any potential harms.

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.

Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease

Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease that prevents patients from combating infections. This leads to recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations, and can be fatal. It is caused by a mutation in a specific gene that causes low levels of a protein called CD18. The low levels of CD18 affect the immune system’s ability to work efficiently and reduces the body’s ability to combat infections.

Rocket Pharmaceuticals is conducting a CIRM-funded ($6.56 M) clinical trial that is testing a treatment that uses a gene therapy called RP-L201. The therapy uses a patient’s own blood stem cells and inserts a corrected version of the mutated gene.  These modified stem cells are then reintroduced back into the patient. The goal is to establish functional immune cells, enabling the body to combat infections. Previous studies have indicated that an increase in CD18 expression to 4-10 percent is associated with survival into adulthood. 

Rocket presented promising results from four patients enrolled in the trial at the Clinical Immunology Society 2021 Annual Meeting.

Patient 1001 was 9 years-of-age at enrollment and had been followed for 18-months after treatment. Patient 1004 was 3 years-of-age at enrollment and had been followed for 9-months. Patients 2006 and 2005 were 7 months- and 2 years-of-age at enrollment and had been followed for 3-months.

Key findings from trial include the following:

  • RP-L201 was well tolerated and no safety issues reported with infusion or treatment.
  • Patient 1001 demonstrated CD18 expression of about 40 percent and resolution of skin lesions with no new lesions reported 18-months post-treatment.
  • Patient 1004 demonstrated CD18 expression of about 28 percent 9-months post-treatment.
  • Patient 2006 demonstrated CD18 expression of about 70 percent 3-months post-treatment.
  • Patient 2005 demonstrated CD18 expression of about 51 percent 3-months post-treatment.

In a news release, Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket expressed optimism for these findings.

“Today’s positive updates on our LAD-I program add to the growing body of encouraging evidence that RP-L201 may provide durable clinical benefit for patients with severe LAD-I who face recurrent, life-threatening infections from birth.”

To access the poster used for this presentation, visit Rocket’s website linked here.

Saying thanks and farewell to a friend

Tom Howing

In this job you get to meet a lot of remarkable people, none more so than the patients who volunteer to take part in what are giant experiments. They are courageous pioneers, willing to be among the first people to ever try a new therapy, knowing that it may not help them and, potentially, might even harm them.

Tom Howing was one such person. I got to know Tom when we were putting together our 2017 Annual Report. Back in 2015 Tom was diagnosed with Stage 4 cancer that had spread throughout his body. He underwent surgery and chemotherapy. That worked for a while, but then the cancer returned. So, Tom had more surgery and chemotherapy. Again, it worked for a while but when the cancer returned again Tom was running out of options.

That’s when he learned about a clinical trial with a company called Forty Seven Inc. that was testing a new anti-cancer therapy that CIRM was supporting. Tom says he didn’t hesitate.

“When I was diagnosed with cancer I knew I had battle ahead of me. After the cancer came back again they recommended I try this CD47 clinical trial. I said absolutely, let’s give it a spin. I guess one is always a bit concerned whenever you put the adjective “experimental” in front of anything. But I’ve always been a very optimistic and positive person and have great trust and faith in my caregivers.”

Optimistic and positive are great ways to describe Tom. Happily, his optimism was rewarded. The therapy worked.

“Scans and blood tests came back showing that the cancer appears to be held in check. My energy level is fantastic. The treatment that I had is so much less aggressive than chemo, my quality of life is just outstanding.”

But after a year or so Tom had to drop out of the trial. He tried other therapies and they kept the cancer at bay. For a while. But it kept coming back. And eventually Tom ran out of options. And last week, he ran out of time.

Tom was a truly fine man. He was kind, caring, funny, gracious and always grateful for what he had. He talked often about his family and how the stem cell therapy helped him spend not just more time with them, but quality time.

He knew when he signed up for the therapy that there were no guarantees, but he wanted to try, saying that even if it didn’t help him that the researchers might learn something to help others down the line.

“The most important thing I would say is, I want people to know there is always hope and to stay positive.”

Tom ultimately lost his battle with cancer. But he never lost his spirit, his delight in his family and his desire to keep going as long as he could. In typical Tom fashion he preferred to put his concerns aside and cheer others along.

“To all those people who are putting in all the hours at the bench and microscope, it’s important for them to know that they are making a huge impact on the lives of real people and they should celebrate it and revel in it and take great pride in it.”

We consider ourselves fortunate to have known Tom and to have been with him on part of his journey. He touched our lives, as he touched the lives of so many others. Our thoughts and wishes go out to his family and friends. He will be remembered, because we never forget our friends.

A few years ago Tom came and talked to the CIRM Board. Here is the video of that event.