A CIRM-funded trial conducted by Oncternal Therapeutics in collaboration with UC San Diego released an interim clinical data update for patients with mantle cell lymphoma (MCL), a type of blood cancer.
The treatment being developed involves an antibody called cirmtuzumab (named after yours truly) being used with a cancer fighting drug called ibrutinib. The antibody recognizes and attaches to a protein on the surface of cancer stem cells. This attachment disables the protein, which slows the growth of the blood cancer and makes it more vulnerable to anti-cancer drugs.
Here are the highlights from the new interim clinical data:
Patients had received a median of two prior therapies before participating in this study including chemotherapy; autologous stem cell transplant (SCT); autologous SCT and CAR-T therapy; autologous SCT and allogeneic SCT; and ibrutinib with rituximab, a different type of antibody therapy.
6 of the 12 patients in the trial experienced a Complete Response (CR), which is defined as the disappearance of all signs of cancer in response to treatment.
All six CRs are ongoing, including one patient who has remained in CR for more than 21 months past treatment.
Four of the six patients achieved CRs within four months on the combination of cirmtuzumab and ibrutinib.
Of the remaining 6 patients, 4 experienced a Partial Response (PR), which is defined as a decrease in the extent of the cancer in the body.
The remaining two patients experienced Stable Disease (SD), which is defined as neither an increase or decrease in the extent of the cancer.
The full interim clinical data update can be viewed in the press release here.
Orphan drug designation is a special status given by the Food and Drug Administration (FDA) for potential treatments of rare diseases that affect fewer than 200,000 in the U.S. This type of status can significantly help advance treatments for rare diseases by providing financial incentives in the form of tax credits towards the cost of clinical trials and prescription drug user fee waivers.
Fortunately for us, a stem cell-gene therapy approach used in a CIRM-funded clinical trial for Cystinosis has just received orphan drug designation. The trial is being conducted by Dr. Stephanie Cherqui at UC San Diego, which is an academic collaborator for AVROBIO, Inc.
Cystinosis is a rare disease that primarily affects children and young adults, and leads to premature death, usually in early adulthood. Patients inherit defective copies of a gene called CTNS, which results in abnormal accumulation of an amino acid called cystine in all cells of the body. This buildup of cystine can lead to multi-organ failure, with some of earliest and most pronounced effects on the kidneys, eyes, thyroid, muscle, and pancreas. Many patients suffer end-stage kidney failure and severe vision defects in childhood, and as they get older, they are at increased risk for heart disease, diabetes, bone defects, and neuromuscular defects.
Dr. Cherqui’s clinical trial uses a gene therapy approach to modify a patient’s own blood stem cells with a functional version of the defective CTNS gene. The goal of this treatment is to reintroduce the corrected stem cells into the patient to give rise to blood cells that will reduce cystine buildup in affected tissues.
In an earlier blog, we shared a story by UCSD news that featured Jordan Janz, the first patient to participate in this trial, as well as the challenges promising approaches like this one face in terms of getting financial support. Our hope is that in addition to the funding we have provided, this special designation gives additional support to what appears to be a very promising treatment for a very rare disease.
You can read the official press release from AVROBIO, Inc. related to the orphan drug designation status here.
Glioblastoma (GBM) is an aggressive form of cancer that begins in the brain and results in tumors that can be very difficult to treat. This condition has claimed the lives of Beau Biden, former Vice President Joe Biden’s son, and John McCain, former Senator of Arizona. However, a new approach to combat this condition is being developed at City of Hope and has just received approval from the FDA to conduct clinical trials. The innovative approach involves using a combination of chimeric antigen receptor (CAR)-T cell therapy and specific components of scorpion venom!
Before we dive into how the scorpion venom is being used, what exactly is CAR-T cell therapy?
Diagram of CAR-T Cell Therapy Image Source: National Cancer Institute
This approach consists of using T cells, which are an immune system cell that can destroy foreign or abnormal cells, and modifying them with a protein called a chimeric antigen receptor (CAR). These newly designed CAR-T cells are able to identify and destroy cancer cells by detecting a specific protein on these cells. What makes CAR-T cell even more promising is that the specific protein detected can be set to virtually anything.
This is where the scorpion venom comes into play. One of the components of this venom is called chlorotoxin (CLTX), which has the ability to specifically bind to brain tumor cells.
Michael Barish, Ph.D. (Left), Christine Brown, Ph.D. (Center), Dongrui Wang (Right) Photo Credit: Business Wire
For this study, Dr. Christine Brown, Dr. Michael Barish, and a team of researchers at City of Hope designed CAR-T cells using chlorotoxin in order to specifically detect and destory brain tumor cells. Now referred to as CLTX-CAR-T cells, they found that these newly engineered cells were highly effective at selectively killing brain tumor cells in animal models. What’s more remarkable is that the CLTX-CAR-T cells ignored non-tumor cells in the brain and other organs.
In a press release, Dr. Barish describes the CLTX-CAR-T cell approach in more detail.
“Much like a scorpion uses toxin components of its venom to target and kill its prey, we’re using chlorotoxin to direct the T cells to target the tumor cells with the added advantage that the CLTX-CAR T cells are mobile and actively surveilling the brain looking for appropriate target. We are not actually injecting a toxin, but exploiting CLTX’s binding properties in the design of the CAR. The idea was to develop a CAR that would target T cells to a wider variety of GBM tumor cells than the other antibody-based CARs.”
In the same press release, Dr. Brown talks about the promise of this newly developed therapy.
“Our chlorotoxin-incorporating CAR expands the populations of solid tumors potentially targeted by CAR T cell therapy, which is particularly needed for patients with cancers that are difficult to treat such as glioblastoma. This is a completely new targeting strategy for CAR T therapy with CARs incorporating a recognition structure different from other CARs.”
Do an online search for “autism stem cells” and you quickly come up with numerous websites offering stem cell therapies for autism. They offer encouraging phrases like “new and effective approach” and “a real, lasting treatment.” They even include dense scientific videos featuring people like Dr. Arnold Caplan, a professor at Case Western Reserve University who is known as the “father of the mesenchymal stem” (it would be interesting to know if Dr. Caplan knows he is being used as a marketing tool?)
The problem with these sites is that they are offering “therapies” that have never been proven to be safe, let alone effective. They are also very expensive and are not covered by insurance. Essentially they are preying on hope, the hope that any parent of a child with autism spectrum disorder (ASD) will do anything and everything they can to help their child.
But there is encouraging news about stem cells and autism, about their genuine potential to help children with ASD. That’s why we are holding a special Facebook Live “Ask the Stem Cell Team” about Autism on Thursday, March 19th at noon (PDT).
The event features Dr. Alysson Muotri from UC San Diego. We have written about his work with stem cells for autism in the past. And CIRM’s own Associate Director for Discovery and Translation, Dr. Kelly Shephard.
We’ll take a look at Dr. Muotri’s work and also discuss the work of other researchers in the field, such as Dr. Joanne Kurtzberg’s work at Duke University.
But we also want you to be a part of this as well. So, join us online for the event. You can post comments and questions during the event, and we’ll do our best to answer them. Or you can send us in questions ahead of time to info@cirm.ca.gov.
If you can’t tune in while we are live, not to worry, you’ll be able to watch it again afterwards when it gets posted to our CIRM Facebook page.
It promises to be a lively, engaging and fascinating event and we look forward to seeing you there.
It’s not every day that a company and a concept that you helped support from the very beginning gets snapped up for $4.9 billion. But that’s what is happening with Forty Seven Inc. and their anti-cancer therapies. Gilead, another California company by the way, has announced it is buying Forty Seven Inc. for almost $5 billion.
The deal gives Gilead access to Forty Seven’s lead antibody therapy, magrolimab, which switches off CD47, a kind of “do not eat me” signal that cancer cells use to evade the immune system.
CIRM has supported this program from its very earliest stages, back in 2013, when it was a promising idea in need of funding. Last year we blogged about the progress it has made from a hopeful concept to an exciting therapy.
When Forty Seven Inc. went public in 2018, Dr. Irv Weissman, one of the founders of the company, attributed a lot of their success to CIRM’s support.
Dr. Irv Weissman
“The story of the funding of this work all of the way to its commercialization and the clinical trials reported in the New England Journal of Medicine is simply this: CIRM funding of a competitive grant took a mouse discovery of the CD47 ‘don’t eat me’ signal through all preclinical work to and through a phase 1 IND with the FDA. Our National Institutes of Health (NIH) did not fund any part of the clinical trial or preclinical run up to the trial, so it is fortunate for those patients and those that will follow, if the treatment continues its success in larger trials, that California voters took the state’s right action to fund research not funded by the federal government.”
Dr. Maria Millan, CIRM’s President & CEO, says the deal is a perfect example of CIRM’s value to the field of regenerative medicine and our ability to work with our grantees to make them as successful as possible.
“To say this is incredible would be an understatement! Words cannot describe how excited we are that this novel approach to battling currently untreatable malignancies has the prospect of making it to patients in need and this is a major step. Speaking on behalf of CIRM, we are very honored to have been a partner with Forty Seven Inc. from the very beginning.
CIRM Senior Science Officer, Dr. Ingrid Caras, was part of the team that helped a group of academic scientists take their work out of the lab and into the real world.
“I had the pleasure of working with and helping the Stanford team since CIRM provided the initial funding to translate the idea of developing CD47 blockade as a therapeutic approach. This was a team of superb scientists who we were fortunate to work closely with them to navigate the Regulatory environment and develop a therapeutic product. We were able to provide guidance as well as funding and assist in the ultimate success of this project.”
Forty Seven Inc. is far from the only example of this kind of support and collaboration. We have always seen ourselves as far more than just a funding agency. Money is important, absolutely. But so too is bringing the experience and expertise of our team to help academic scientists take a promising idea and turn it into a successful therapy.
After all that’s what our mission is, doing all we can to accelerate stem cell therapies to patients with unmet medical needs. And after a deal like this, Forty Seven Inc. is definitely accelerating its work.
Each year, around 24,000 women in the US lose a pregnancy. One reason for this unfortunate occurrence are metabolic disorders, one of which is known as Sly syndrome and is caused by a single genetic mutation. In Sly syndrome, the body’s cells lack an enzyme necessary for proper cell function. Many fetuses with this condition die before birth but those that survive are treated with regular injections of the lacking enzyme. Unfortunately, patients can eventually develop an immune response to these injections and it cannot enter the brain after birth.
However, a team of researchers at UCSF are looking at exploring a potential treatment that could be delivered in-utero. In a CIRM supported study, Dr. Tippi Mackenzie and Dr. Quoc-Hung Nguyen transplanted blood-forming stem cells from normal mice into fetal mice carrying the genetic mutation for Sly syndrome. The researchers were most interested to see whether these cells could reach the brain, and whether they would change into cells called microglia, immune cells that originate from blood-forming stem cells. In a normally developing fetus, once matured, microglia produce and store the necessary enzyme, as well as regulate the immune environment of the brain.
Stem cells transplanted in utero (green) engrafted into fetal mouse brain tissue. Image credit: Q-H Nguyen/MacKenzie lab.
The researchers found that the stem cells were able to engraft in the brain, liver, kidney, and other organs. Furthermore, these stem cells were able to eventually turn into the appropriate cell type needed to produce the enzyme in each of the organs.
In a press release, Dr. Mackenzie talks about the impact that this potential treatment could have.
“This group of vulnerable patients has been relatively ignored in the fetal surgery world. We know these patients could potentially benefit from a number of medical therapies. So this is our first foray into treating one of those diseases.”
In the same press release, Dr. Nguyen talks about the impact of the results from this study.
“These exciting findings are just the tip of the iceberg. They open up a whole new approach to treating a range of diseases. At the same time, there’s also a lot of work to do to optimize the treatment for humans.”
The next step for Dr. Mackenzie is to apply to the U.S. Food and Drug Administration to launch a clinical trial of enzyme replacement therapy that will ultimately enroll patients with Sly syndrome and related metabolic disorders.
This approach is similar to a CIRM funded trial conducted by Dr. Mackenzie that involves a blood stem cell transplant in utero.
The full results to this study were published in Science Translational Medicine.
Logan Lacy, a child with AADC Deficiency: Photo courtesy Chambersburg Public Opinion
Tomorrow, the last day in February, is Rare Disease Day. It’s a day dedicated to raising awareness about rare diseases and the impact they have on patients and their families.
But the truth is rare diseases are not so rare. There are around 7,000 diseases that affect fewer than 200,000 Americans at any given time. In fact, it’s estimated that around one in 20 people will live with a rare disease at some point in their lives. Many may die from it.
This blog is about one man’s work to find a cure for one of those rare diseases, and how that could lead to a therapy for something that affects many millions of people around the world.
Dr. Krystof Bankiewicz; Photo courtesy Ohio State Medical Center
Dr. Krystof Bankiewicz is a brain surgeon at U.C. San Francisco and The Ohio State University. He is also the President and CEO at Brain Neurotherapy Bio and a world expert in delivering gene and other therapies to the brain. More than 20 years ago, he began trying to develop a treatment for Parkinson’s disease by looking at a gene responsible for AADC enzyme production, which plays an important role in the brain and central nervous system. AADC is critical for the formation of serotonin and dopamine, chemicals that transmit signals between nerve cells, the latter of which plays a role in the development of Parkinson’s disease.
While studying the AADC enzyme, Dr. Bankiewicz learned of an extremely rare disorder where children lack the AADC enzyme that is critical for their development. This condition significantly inhibits communication between the brain and the rest of the body, leading to extremely limited mobility, muscle spasms, and problems with overall bodily functions. As a result of this, AADC deficient children require lifelong care, and particularly severe cases can lead to death in the first ten years of life.
“These children can’t speak. They have no muscle control, so they can’t do fundamental things such as walking, supporting their neck or lifting their arms,” says Dr. Bankiewicz. “They have involuntary movements, experience tremendously painful spasms almost like epileptic seizures. They can’t feed themselves and have to be fed through a tube in their stomach.”
So, Dr. Bankiewicz, building on his understanding of the gene that encodes AADC, developed an experimental approach to deliver a normal copy, injected directly into the midbrain, the area responsible for dopamine production. The DDC gene was inserted into a virus that acted as a kind of transport, carrying the gene into neurons, the brain cells affected by the condition. It was hoped that once inside, the gene would allow the body to produce the AADC enzyme and, in turn, enable it to produce its own dopamine .
And that’s exactly what happened.
“It’s unbelievable. In the first treated patients their motor system is dramatically improved, they are able to better control their movements, they can eat, they can sleep well. These are tremendous benefits. We have been following these children for almost three years post-treatment, and the progression we see doesn’t stop, it keeps going and we see these children keep on improving. Now they are able to get physical therapy to help them. Some are even able to go to school.”
For Dr. Bankiewicz this has been decades in the making, but that only makes it all the more gratifying: “This doesn’t happen very often in your lifetime, to be able to use all your professional experience and education to help people and see the impact it has on people’s lives.”
So far he has treated 20 patients from the US, UK and all over the world.
But he is far from finished.
Already the therapy has been given Orphan Drug Designation and Regenerative Medicine Advanced Therapy designation by the US Food and Drug Administration. The former is a kind of financial incentive to companies to develop drugs for rare diseases. The latter gives therapies that are proving to be both safe and effective, an accelerated path to approval for wider use. Dr. Bankiewicz hopes that will help them raise the funds needed to treat children with this rare condition. “We want to make this affordable for families. We are not in this to make a profit; we want to get foundations and maybe even pharmaceutical companies to help us treat the kids, so they don’t have to cover the full costs themselves.”
CIRM has not funded any of this work, but the data and results from this research were important factors in our Board awarding Dr. Bankiewicz more than $5.5 million to begin a clinical trial for Parkinson’s disease. Dr. Bankiewicz is using a similar approach in that work to the one he has shown can help children with AADC deficiency.
While AADC deficiency may only affect a few hundred children worldwide, it’s estimated that Parkinson’s affects more than ten million people; one million of those in the US alone. Developing this gene therapy technique in a rare disease, therefore, may ultimately benefit large populations of patients.
So, on this Rare Disease Day, we celebrate Dr. Bankiewicz and others whose compassion and commitment to finding treatments to help those battling rare conditions are helping change the world, one patient at a time.
Today, we here at CIRM wanted to provide an update on the fascinating world of hematopoietic (blood) stem cell-based therapies. What is the current status of this promising field and what are some of the challenges that need to be overcome? Dr. Kelly Shepard, Associate Director of Discovery and Translation here at CIRM, answers these questions and many more in the blog entry below.
There have been a number of exciting advances in regenerative medicine over the past few years, especially in the use of gene therapy and hematopoietic (blood) stem cell transplantation to treat and even cure various diseases of the blood and immune system. These studies built off groundbreaking research by Till and McCulloch in the 1950-60’s, who identified a rare and special stem cell in the bone marrow of mice that gives rise to all cells of the blood and immune system for the lifetime of the animal, the “hematopoietic stem cell”, or HSC. It wasn’t long before scientists and doctors realized the therapeutic implications of this discovery, and the journey to identify the human counterpart began. Fast forward to the present, and HSC transplantation (HSCT) has become a standard medical procedure for treating various cancers and genetic disorders of the blood. The basic premise is this: a patient with a diseased or defective blood/immune system receives an infusion of healthy HSCs, which are typically procured from donated bone marrow or umbilical cords, but in certain situations, might come from the patient him/herself. Once established in the recipient, these healthy cells will divide and regenerate a new blood and immune system over the course of the patient’s lifetime.
For HSCT to be successful, the donor cells must “engraft”, or take up permanent residence in their new environment. This usually necessitates “conditioning” the recipient with some form of chemotherapy or radiation, which eliminates some of the patient’s own cells to create room for the new arrivals. Unfortunately, conditioning creates a situation where the patient is extremely vulnerable to infections and other complications during the period of recovery, as it will take weeks for his/her blood and immune systems to be reestablished. These inherent risks mean HSC transplants can only be offered to patients with life threatening diseases such as leukemia, or to those with significant blood/immune disorders who are sufficiently healthy to tolerate the toxic conditioning regimen and to weather the extended period of recovery.
A second major issue preventing a more widespread use of HSCT is the shortage of healthy donor HSCs that are available for transplant, which must be immune matched to the recipient to prevent rejection. Immune matching is also critical to avoid a dangerous complication called graft vs. host disease, where the transplanted cells or their progeny launch an immune attack against the recipient’s organs, often leading to chronic disease and sometimes, death. Unfortunately, there are many people who have no compatible donors and for whom the risk of even a partially matched transplant is unacceptable.
Scientists and clinicians have long sought means to overcome the technical challenges of HSCT in order to “unleash” its true potential to cure and treat a wider variety of diseases, and to make it feasible (and affordable) for a much larger number of patients. CIRM has endeavored to support novel approaches that could hopefully produce game changing advances for the field. Some of these approaches were recently highlighted in a Perspective article, published in Stem Cells Translational Medicine in early 2020, along with a discussion of other important advances in related areas, listed below. More information can be found in that article or referring to our website to learn more about the individual projects.
Approaches that could increase the availability of healthy HSCs for transplant include development of non-toxic conditioning regimens to facilitate a patient’s acceptance and recovery from the transplant procedure; novel technologies for expanding HSCs for transplant; and gene modification technologies to correct inherited mutations in HSCs. Illustration Credit: Dr. Kelly Shepard, CIRM
Developing New Sources of Healthy and Immune Compatible HSCs for transplant
Exploring ways to produce HSCs from pluripotent stem cells in the lab
Expanding populations of HSCs that are already present in donated tissues such as cord blood
Using genetic engineering to “repair” defects in the DNA of HSCs from patients with inherited blood and/or immune disorders
Using genetic engineering to create “immune invisible” or “universal donor” HSCs that will not be rejected after transplantation
Developing Safer and More Tolerable Conditioning Regimens
Exploring reduced intensity forms of conditioning with drugs or radiation
Using antibodies rather than chemicals to free up space in the bone marrow for incoming, donor HSCs
Using dietary methods to free up space in the bone marrow for incoming, donor HSCs
Accelerating Reovery of Immune Function Lost Through Conditioning
Adding back key populations of immune cells to protect the host during regeneration of their immune system
Discovering new drugs and treatments to accelerate the pace of regeneration after transplant, or to prevent the death of HSCs that survived conditioning
Overcoming these scientific and technical challenges could create a paradigm shift in the way HSCT is applied and used and consequently, reduce the costs and risks associated with the procedure. In this way, the true potential of HSCT could be unleashed for the greatest good.
Smoking is one of the leading causes of preventable death not just in the US, but worldwide. According to the US Centers for Disease Control and Prevention tobacco causes an estimated seven million deaths around the world, every single year. And for every person who dies, another 30 live with a serious smoking-related illness. Clearly quitting is a good idea. Now a new study adds even more incentive to do just that.
Scientists at the Welcome Trust Sanger Institute and University College London in the UK, found that quitting smoking did more than just stop further damage to the lungs. They found that cells in the lining of the lungs that were able to avoid being damaged, were able to regrow and repopulate the lung, helping repair damaged areas.
In an article in Science Daily Dr Peter Campbell, a joint senior author of the study, said: “People who have smoked heavily for 30, 40 or more years often say to me that it’s too late to stop smoking — the damage is already done. What is so exciting about our study is that it shows that it’s never too late to quit — some of the people in our study had smoked more than 15,000 packs of cigarettes over their life, but within a few years of quitting many of the cells lining their airways showed no evidence of damage from tobacco.”
Researchers at UCLA have also made a discovery that could help people with lung disease.
They examined the lungs of people with cancer and compared them to the lungs of healthy people. They were able to identify a group of molecules, called the Wnt/beta-catenin signaling pathway, that appear to influence the activity of stem cells that are key to maintaining healthy lungs. Too much activity can tilt the balance away from healthy lungs to ones with mutations that are more prone to developing tumors.
In a news release Dr. Brigitte Gomperts, the lead author of the study, says although this work has only been done in mice so far it has tremendous potential: “We think this could help us develop a new therapy that promotes airway health. This could not only inform the treatment of lung cancer, but help prevent its progression in the first place.”
The study is published in the journal Cell Reports.
CIRM has funded some of Dr. Gomperts earlier work in this area.
And there’s encouraging news for people trying to recover from a stroke. Results from ReNeuron’s Phase 2 clinical trial show the therapy appears to help people who have experienced some level of disability following a stroke.
ReNeuron says its CTX therapy – made from neural stem cells – was given to 23 people who had moderate to severe disability resulting from an ischemic stroke. The patients were, on average, seven months post stroke.
In the study, published in the Journal of Neurology, Neurosurgery & Psychiatry, researchers used the Modified Rankin Scale (mRS), a measure of disability and dependence to assess the impact of the therapy. The biggest improvements were seen in a group of 14 patients who had limited movement of one arm.
38.5% experienced at least a one-point improvement on mRS six months after being treated.
50% experienced a one-point improvement 12 months after being treated.
If that doesn’t seem like a big improvement, then consider this. Moving from an mRS 3 to 2 means that a person with a stroke regains their ability to live independently.
Image of a blood stem cell in its natural environment: Photo courtesy UC Merced
When it comes to using stem cells for therapy you don’t just need to understand what kinds of cell to use, you also need to understand the environment that is best for them. Trying to get stem cells to grow in the wrong environment would be like trying to breed sheep in a pond. It won’t end well.
But for years scientists struggled to understand how to create the right environment, or niche, for these cells. The niche provides a very specific micro-environment for stem cells, protecting them and enabling them to self-renew over long periods of time, helping repair damaged tissues and organs in the body.
But different stem cells need different niches, and those involve both physical and chemical properties, and getting that mixture right has been challenging. That in turn has slowed down our ability to use those cells to develop new therapies.
UC Merced’s Joel Spencer in the lab: Photo courtesy UC Merced
Now UC Merced’s Professor Joel Spencer and his team have developed a way of capturing an image of hematopoietic or blood stem cells (HSCs), inside their niche in the bone marrow. In an article on UC Merced News, he says this could be a big step forward.
“Everyone knew black holes existed, but it took until last year to directly capture an image of one due to the complexity of their environment. It’s analogous with stem cells in the bone marrow. Until now, our understanding of HSCs has been limited by the inability to directly visualize them in their native environment.
“This work brings an advancement that will open doors to understanding how these cells work which may lead to better therapeutics for hematologic disorders including cancer.”
In the past, studying HSCs involved transplanting them into a mouse or other animal that had undergone radiation to kill off its own bone marrow cells. It enabled researchers to track the HSCs but clearly the new environment was very different than the original, natural one. So, Spencer and his team developed new microscopes and imaging techniques to study cells and tissues in their natural environment.
In the study, published in the journal Nature, Spencer says all this is only possible because of recent technological breakthroughs.
“My lab is seeking to answer biological questions that were impossible until the advancements in technology we have seen in the past couple decades. You need to be able to peer inside an organ, inside a live animal and see what’s happening as it happens.”
Being able to see how these cells behave in their natural environment may help researchers learn how to recreate that environment in the lab, and help them develop new and more effective ways of using those cells to repair damaged tissues and organs.
