A new independent report says developing stem cell treatments and cures for some of the most common and deadly diseases could produce multi-billion dollar benefits for California in reduced healthcare costs and improved quality and quantity of life.
The report, by researchers at the University of Southern California’s Leonard D. Schaeffer Center for Health Policy & Economics, looked at the value of hypothetical future interventions to reduce or cure cancer, diabetes, stroke and blindness.
Predicting the future is always complicated and uncertain and many groups are looking at the best models to determine the value and economic impact of cell and gene therapy as the first products are just entering the market. This study provides some insights into the potential financial benefits of developing effective stem cell treatments for some of the most intractable diseases affecting California today.
The impact could affect millions of people. In
2018 for Californians over the age of 50:
Nearly half were
predicted to develop diabetes in their lifetime
More than one third
will experience a stroke
Between 5 and 8 percent will develop either breast, colorectal,
lung, or prostate cancer
The report says that a therapy that decreased
the incidence of diabetes by 50 percent in Californians over the age of 51
would translate into a gain for the state of $322 billion in social value
between now and 2050. Even just reducing diabetes 10% would lead to a gain of
$60 billion in social value over the same period.
For stroke a 50 percent reduction would generate an estimated $229 billion in social value. A 10 percent reduction would generate $47 billion
For breast cancer a 50 percent reduction would generate $56 billion in social value; for colorectal cancer it would be $72 billion; for lung cancer $151 billion; and prostate cancer $53 billion.
The impact of a cure for any one of those
diseases would be enormous. For example, a 51-year-old woman cured of lung
cancer could expect to gain a lifetime social value of almost half a million
dollars ($467,275). That’s a measure of years of healthy life gained, of years
spent enjoying time with family and friends and not wasting away or lying in a
hospital bed.
The researchers say: “Though advances in
scientific research defy easy predictions, investing in biomedical research is
important if we want to reduce the burden of common and costly diseases for
individuals, their families, and society. These findings show the value and
impact breakthrough treatments could have for California.”
“Put in this context, the CIRM investment
would be worthwhile if it increased our chances of success even modestly.
Against the billions of dollars in disease burden facing California, the
relatively small initial investment is already paying dividends as researchers
work to bring new therapies to patients.”
The researchers determined the “social value”
using a measure called a quality adjusted life-year (QALY). This is a way of
estimating the cost effectiveness and consequences of treating or not treating
a disease. For example, one QALY is equivalent to one year of perfect health for
an individual. In this study the value of that year was estimated at $150,000.
If someone is sick with, say, diabetes, their health would be estimated to be
0.5 QALY or $75,000. So, the better health a person enjoys and the longer they
enjoy it the higher QALY score they accumulate. In the case of a disease
affecting millions of people in that state or country that can obviously lead
to very large QALY scores representing potentially billions of dollars.
Proposition 71 is the state ballot initiative that created California’s Stem Cell Agency. This month, the Agency reached another milestone when the 71st clinical trial was initiated in the CIRM Alpha Stem Cell Clinics (ASCC) Network. The ASCC Network deploys specialized teams of doctors, nurses and laboratory technicians to conduct stem cell clinical trials at leading California Medical Centers.
These teams work with academic and industry partners to support patient-centered for over 40 distinct diseases including:
Amyotrophic Lateral Sclerosis (ALS)
Brain Injury & Stroke
Cancer at Multiple Sites
Diabetes Type 1
Eye Disease / Blindness Heart Failure
HIV / AIDS
Kidney Failure
Severe Combined Immunodeficiency (SCID)
Sickle Cell Anemia
Spinal Cord Injury
These clinical trials have treated over 400 patients and counting. The Alpha Stem Cell Clinics are part of CIRM’s Strategic Infrastructure. The Strategic Infrastructure program which was developed to support the growth of stem cell / regenerative medicine in California. A comprehensive update of CIRM’s Infrastructure Program was provided to our Board, the ICOC.
CIRM’s infrastructure catalyzes stem cell / regenerative medicine by providing resources to all qualified researchers and organizations requiring specialized expertise. For example, the Alpha Clinics Network is supporting clinical trials from around the world.
Many of these trials are sponsored by commercial companies that have no CIRM funding. To date, the ASCC Network has over $27 million in contracts with outside sponsors. These contracts serve to leverage CIRMs investment and provide the Network’s medical centers with a diverse portfolio of clinical trials to address patients’’ unmet medical needs.
Alpha Clinics – Key Performance Metrics
70+ Clinical Trials
400+ Patients Treated
40+ Disease Indications
Over $27 million in contracts with commercial sponsors
The CIRM Alpha Stem Cell Clinics and broader Infrastructure Programs are supporting stem cell research and regenerative medicine at every level, from laboratory research to product manufacturing to delivery to patients. This infrastructure has emerged to make California the world leader in regenerative medicine. It all started because California’s residents supported a ballot measure and today we have 71 clinical trials for 71.
For the past few years the Signals blog site – which offers an insiders’ perspectives on the world of regenerative medicine and stem cell research – has hosted what it calls a “Blog Carnival”. This is an event where bloggers from across the stem cell field are invited to submit a piece based on a common theme. This year’s theme is “Has Regenerative Medicine Come of Age?” Here’s my take on that question:
Many cultures have different traditions to mark when a child comes of age. A bar mitzvah is a Jewish custom marking a boy reaching his 13th birthday when he is considered accountable for his own actions. Among Latinos in the US a quinceañera is the name given to the coming-of-age celebration on a girl’s 15th birthday.
Regenerative Medicine (RM) doesn’t have anything quite so simple or obvious, and yet the signs are strong that if RM hasn’t quite come of age, it’s not far off.
For example, look at our experience at the California Institute for Regenerative Medicine (CIRM). When we were created by the voters of California in 2004 the world of stem cell research was still at a relatively immature phase. In fact, CIRM was created just six years after scientists first discovered a way to derive stem cells from human embryos and develop those cells in the laboratory. No surprise then that in the first few years of our existence we devoted a lot of funding to building world class research facilities and investing in basic research, to gain a deeper understanding of stem cells, what they could do and how we could use them to develop therapies.
Fast forward 14 years and we now have funded 49 projects in clinical trials – everything from stroke and cancer to spinal cord injury and HIV/AIDS – and our early funding also helped another 11 projects get into clinical trials. Clearly the field has advanced dramatically.
In addition the FDA last year approved the first two CAR-T therapies – Kymriah and Yescarta – another indication that progress is being made at many levels.
But there is still a lot of work to do. Many of the trials we are funding at the Stem Cell Agency are either Phase 1 or 2 trials. We have only a few Phase 3 trials on our books, a pattern reflected in the wider RM field. For some projects the results are very encouraging – Dr. Gary Steinberg’s work at Stanford treating people recovering from a stroke is tremendously promising. For others, the results are disappointing. We have cancelled some projects because it was clear they were not going to meet their goals. That is to be expected. These clinical trials are experiments that are testing, often for the first time ever in people, a whole new way of treating disease. Failure comes with the territory.
As the number of projects moving out of the lab and into clinical trials increases so too are the other signs of progress in RM. We recently held a workshop bringing together researchers and regulators from all over the world to explore the biggest problems in manufacturing, including how you go from making a small batch of stem cells for a few patients in an early phase clinical trial to mass producing them for thousands, if not millions of patients. We are also working with the National Institutes of Health and other stakeholders in discussing the idea of reimbursement, figuring out who pays for these therapies so they are available to the patients who need them.
And as the field advances so too do the issues we have to deal with. The discovery of the gene-editing tool CRISPR has opened up all sorts of possible new ways of developing treatments for deadly diseases. But it has also opened up a Pandora’s box of ethical issues that the field as a whole is working hard to respond to.
These are clear signs of a maturing field. Five years ago, we dreamed of having these kinds of conversations. Now they are a regular feature of any RM conference.
The simple fact that we can pose a question asking if RM has come of age is a sign all by itself that we are on the way.
Like little kids sitting in the back of a car, anxious to get to their destination, we are asking “Are we there yet?” And as every parent in the front seat of their car responds, “Not yet. But soon.”
When we launched our Facebook Live “Ask the Expert” series earlier this year we wanted to create an opportunity for people to hear from and question experts about specific diseases or disorders. The experts we turned to were medical ones, neurologists and neuroscientists in the case of the first two Facebook Live events, stroke and ALS.
Then we learned about a blog post on the ALS Advocacy website questioning our use of the word “expert”. The author, Cathy Collet, points out that doctors or scientists are far from the only experts about these conditions, that there are many people who, by necessity, have become experts on a lot of issues relating to ALS and any other disease.
Here’s Cathy’s blog. After you read it please let us know what you think: should we come up with a different title for the series, if so what would you suggest?
“Over the years I’ve experienced many “Ask the Experts” sessions related to ALS. It’s always a panel of neuroscientists who talk a lot about ALS research and then take a few questions.
The “Expert” crown defaults to them. They speak from the dais. We get to listen a lot and ask. They are by default “The Experts” in the fight against ALS.
But wait, there are all kinds of people with superb and valuable knowledge related to ALS –
There are people who know a lot about insurance.
There are people who know a lot about communication technology.
There are people who know a lot about low-tech hacks.
There are people who know a lot about suction machines.
There are people who know a lot about breathing.
There are people who know a lot about the FDA.
There are people who know a lot about moving a person on and off a commode.
There are people who know a lot about taxes.
There are people who know a lot about drugs.
There are people who know a lot about data.
There are people who know a lot about choking.
There are people who know a lot about financing research.
There are people who know a lot about stem cells.
There are people who know a lot about feeding tubes and nutrition.
There are people who know a lot about what’s important in living with the beast ALS.
There are people who know a lot about primary care in ALS.
There are people who know a lot about constipation.
Our default implication for the word experts being neuroscientists is revealing.There are many people in the fight against ALS, including those living with it, who know a lot. We still live in a hierarchy where people with ALS and caregivers are at the bottom.
Words matter. “Expert” is not a royal title to be owned by anyone by default.
It’s time for simple changes to some traditions. “Ask the Neuroscientists,” anyone?
By the way, our next Facebook Live “Ask the ?” feature is targeting Sickle Cell Disease. It will be from noon till 1pm on Tuesday August 28th. More details, and maybe even a new name, to follow.
We recently held our first ever Facebook Live event. It was focused on the use of stem cells and recovery from a stroke and featured three great guests: Dr. Gary Steinberg, chief of Neurosurgery at Stanford, Sonia Coontz, a patient of Dr. Steinberg’s, and CIRM’s own Science Officer Dr. Lila Collins.
We had an amazing response from people during the event and in the days since then with some 6,750 people watching the video and almost 1,000 people reacting by posting a comment or sharing it with friends. It was one of the most successful things we have ever done on Facebook so it’s not surprising that we plan on doing many more Facebook Live ‘Ask the Expert’ events in the future. We will post more details of that as we finalize them.
We tried to cover as many topics as possible during the hour but there were simply too many questions for us to get to all of them. So here is a recap of the key issues we covered, and a few we didn’t have a chance to answer.
Let’s start with Dr. Steinberg’s explanation of the research that led to his current clinical trial:
Dr. Steinberg: “I got interested in this about 18 years ago when I took human cells and transplanted them into rodent models of stroke. What we found was that when we transplanted those cells into the stroke region, the core of the stroke, they didn’t survive very well but when we moved them a few millimeters away from the stroke they not only survived but they migrated to the stroke.
The reason they migrate is that the stem cells have receptors on them that interact with chemicals given off by the stroke environment and that’s why they migrate to the stroke site. And when they get to the site they can turn into different kinds of cells. Very importantly we found these mice and rats that had behavioral problems – walking, moving – as a result of the stroke, we found we could improve their neurological outcomes with the stem cells.
With the help of CIRM, which has been very generous, we were fortunate enough to receive about $24 million in funding over the last 8 years, from 2010, to move this therapy into the clinic to understand the basic mechanisms of the recovery and to start clinical trials
One of the surprising things was that our initial notion was that the cells we transplanted into the brains would initially turn into the cells in the brain affected by the stroke and reconstitute those circuits. We were shocked to find that that was not what was happening, that only a few of the transplanted cells turned into neurons. The way they were recovering function was by secreting very powerful growth factors and molecules and proteins that enhanced native recovery or the ability of the normal brain to recover itself. Some of these processes included outgrowth of neurons, new connections, new synapses, not from the stem cells but from the native cells already in the brain.
This is not cell replacement but enhancing native recovery and, in a simple sense, what the cells are doing, we believe, is to change the adult brain, which has a hard time recovering from a stroke, into an infant brain and infants recover very well after a stroke.”
All this work was focused on ischemic strokes, where a blockage cuts off blood flow to the brain. But people like Cheryl Ward wanted to know: “Will this work for hemorrhagic stroke?” That’s where a blood vessel in the brain leaks or ruptures.
Dr. Steinberg: “I suspect we will be generalizing this therapy into hemorrhagic patients very, very soon and there’s no reason why it shouldn’t work there. The reason we didn’t start there is that 85% of strokes are ischemic and only 15% are hemorrhagic so it’s a smaller population but a very, very important population because when patients have a hemorrhage from a stroke they are often more seriously disabled than from ischemic.”
Dr. Lila Collins: “I would like to highlight one trial for hemorrhagic stroke with the Mayo Clinic and that’s using mesenchymal stem cells (normally found in bone marrow or blood). It’s an early stage, Phase 1 safety study in patients with recent cerebral hemorrhage. They are looking at improvements in neurological function and patients have to be treated within 72 hours after the stroke.”
Dr. Steinberg explained that because it’s more difficult to enroll patients within 72 hours of a stroke that we may end up offering a combination of therapies spread out over months or even years.
Dr. Steinberg: “It may be that and we may figure this out in the next 5 to 10 years, that you might want to treat patients acutely (right away) with an intravenous therapy in the first 72 hours and then you might want to come in again sub-acutely within a few months, injecting the cells into the brain near the stroke, and then maybe come in chronically a few years later if there are still problems and place the cells directly in the brain. So, lots of ways to think about how to use this in the future.”
James Russell suffered a stroke in 2014 and wrote:
“My left side was affected. My vision was also impacted. Are any stroke patients being given stem cells seeing possible improvement in visual neglect?”
Dr. Steinberg: “We don’t know the answer to that yet, it’s quite possible. It’s true these vision circuits are not dead and could be resurrected. We have not targeted visual pathways in our work, we have targeted motor functions, but I would also be optimistic that we could target patients who have vision problems from stroke. It’s a very important area.
A number of people wondered if stem cells can help people recovering from a stroke can they also help people with other neurological conditions.
Hanifa Gaphoor asked “What about Parkinson’s disease?” and Ginnievive Patch wondered “Do you feel hopeful for neurological illnesses like Huntington’s disease and ALS? Dr. Steinberg was cautiously optimistic.
Dr. Steinberg: “We’ve extended this kind of treatment not just for ischemic stroke but into traumatic brain injury (TBI) and we just completed a trial for patients with chronic TBI or who have suffered a trauma to the brain. Many other indications may be possible. In fact, now that we know these circuits are not dead or irreversibly injured, we believe we could even extend this to neurodegenerative diseases like ALS, Parkinson’s, maybe even to Alzheimer’s disease in the future. So, lots of hope but we don’t want to oversell this, and we want to make sure this is done in a rigorous fashion.”
Several people had questions about using their own adipose, or fat stem cells, in therapies being offered at clinics around the US and in other countries. Cheri Hicks asked: “I’m curious if adipose stem cell being used at clinics at various places is helpful or beneficial?”
Dr. Steinberg: “I get emails or calls from patients every week saying should I go to Russia, India or Mexico and get stem cell transplants which are done not as part of a rigorous trial and I discourage patients from getting stem cells that are not being given in a controlled fashion. For one thing, patients have been getting hurt by these treatments in these clinics; they have developed tumors and infections and other problems. In many cases we don’t even know what the cells are, there’s not published information and the patients pay cash for this, of course.”
At CIRM we also worry about people going to clinics, in the US and in other countries, where they are getting therapies that have not been approved by the US Food and Drug Administration (FDA) or other appropriate regulatory bodies. That’s why we have created this page on our website to help people who want a stem cell therapy but don’t know what to look for in a clinical trial or what questions to ask to make sure it’s a legitimate trial, one that’s been given the go-ahead by the FDA.
Bret Ryan asked: “What becomes of the implanted cells?”
Dr. Steinberg: We found after transplanting the cells, one week after the transplant, we see a new abnormality in the premotor cortex, the area of the brain that controls motor function. We saw a new abnormality there or a new signal that disappears after a month and never comes back. But the size of that temporary abnormality after one week correlates very closely with the degree of recovery after six months, one year and two years.
One of the interesting things is that it doesn’t seem to be necessary for the cells to survive long term to have beneficial effects. The cells we used in the SanBio trial don’t survive more than a month and yet they seem to aid recovery function in our pilot studies which is sustained for years.”
And of course, many people, such as Karen Smart, wanted to know how they could get the therapy. Right now, the clinical trial is fully enrolled but Stanford is putting together a waiting list for future trials. If you are interested and would like more information, please email: stemcellstudy@stanford.edu.
Sonia Coontz, the patient who was also a key part of the Facebook Live event, has an amazing story to tell. She was left devastated, physically and emotionally, after having a stroke. But then she heard about Dr. Steinberg’s clinical trial and it changed her life. Here’s her story.
We were thrilled to receive all of your comments and questions during our first Facebook Live event. It’s this kind of dialogue between scientists, patients and the public that will be critical for the continued support of our mission to accelerate stem cell treatments to patients with unmet medical needs.
Due to the response, we plan to regularly schedule these “Ask the Expert” events. What disease area would you like us to focus on next time? Leave us a comment or email info@cirm.ca.gov
Stroke is one of the leading causes of death in the US and the leading cause of serious, long-term disability. But could stem cell therapies change that and help people who’ve had a brain attack? Could stem cells help repair the damage caused by a stroke and restore a person’s ability to speak normally, to be able to walk without a limp or regain strength in their hands and arms?
To find out the answers to these and other questions joins us for “Ask the Expert”, a special Facebook Live event this Thursday, May 31, from noon till 1pmPDT
The event will feature Dr. Gary Steinberg, the Chair of Neurosurgery at Stanford University. Dr. Steinberg is currently running a CIRM-funded clinical trial targeting stroke.
We will also be joined by CIRM Senior Science Officer Lila Collins, PhD who can talk about the broad range of other projects using stem cells to help people recover from a stroke.
We are also delighted to welcome Sonia Coontz, who suffered a devastating stroke several years ago and made a remarkable recovery after getting a stem cell therapy.
To join us for the event, all you have to do is go to our Facebook page on Thursday at noon (PDT) and you should see a video playing, which you can watch on mobile or desktop. Click the video to enter viewing mode.
Also, make sure to “like” our page before the event to receive a notification that we’ve gone live.
And we want to hear from you, so you will be able to post questions for the experts to answer or, you can email them directly to us at info@cirm.ca.gov
It’s not often you get a chance to ask a world class stem cell expert a question about their work, and how it might help you or someone you love. But on Thursday, May 31 you can do just that.
CIRM is hosting a special ‘Ask the Expert’ event on Facebook Live. The topic is Strokes and Stem Cells. Just head over to our Facebook Page on May 31st from noon till 1pm PST to experience it live. You can also re-watch the event any time after the broadcast has ended from our Facebook videos page.
We will be joined by Dr. Gary Steinberg, chair of neurosurgery at Stanford University, who will talk to us about his work in helping reverse the damage caused by a stroke, even for people who experienced a brain attack several years ago.
CIRM Senior Science Officer, Dr. Lila Collins, will talk about other stem cell research targeting stroke, its promise and some of the problems that still need to be overcome.
You will have a chance to ask questions of both our experts, either live on the day or by sending us questions in advance at info@cirm.ca.gov.
We’ll post reminders on Facebook so make sure to follow us. But for now, mark the date and time on your diary and please feel free to share this information with anyone you think might be interested.
I am embarrassed to admit that I have never been to the Inland Empire in California, the area that extends from San Bernardino to Riverside counties. That’s about to change. On Monday, April 16th CIRM is taking a road trip to UC Riverside, and we’re inviting you to join us.
We are holding a special, free, public event at UC Riverside to talk about the work that CIRM does and to highlight the progress being made in stem cell research. We have funded 45 clinical trials in a wide range of conditions from stroke and cancer, leukemia, lymphoma, vision loss, diabetes and sickle cell disease to name just a few. And will talk about how we plan on funding many more clinical trials in the years to come.
We’ll be joined by colleagues from both UC Riverside, and City of Hope, talking about the research they are doing from developing new imaging techniques to see what is happening inside the brain with diseases like Alzheimer’s, to using a patient’s own cells and immune system to attack deadly brain cancers.
It promises to be a fascinating event and of course we want to hear from you, our supporters, friends and patient advocates. We are leaving plenty of time for questions, so we can hear what’s on your mind.
So, join us at UC Riverside on Monday, April 16th from 12.30pm to 2pm. The doors open at 11am so you can enjoy a poster session (highlighting some of the research at UCR) and a light lunch before the event. Parking will be available on site.
Visit the Eventbrite page we have created for all the information you’ll need about the event, including a chance to RSVP and book your place.
The event is free so feel free to share this with anyone and everyone you think might be interested in joining us.
With the Thanksgiving holiday behind us, we’re back to the grind at CIRM. Here are two exciting CIRM-funded stem cell stories that happened while you were away.
Stanford Scientists Are Treating Stroke Patients with Stem Cells
Smithsonian Magazine featured the work of a CIRM-funded scientist in their December Magazine issue. The article, “A Neurosurgeon’s Remarkable Plan to Treat Stroke Victims with Stem Cells”, features Dr. Gary Steinberg, who is the Chair of Neurosurgery at Stanford Medical Center and the founder of the Stanford Stroke Center.
Gary Steinberg (Photo by Jonathan Sprague)
The brain and its 100 billion cells need blood, which carries oxygen and nutrients, to function. When that blood supply is cut off, brain cells start to die and patients experience a stroke. Stroke can happen in one of two ways: either by blood clots that block the arteries and blood vessels that send blood to the brain or by blood vessels that burst within the brain itself. Symptoms experienced by stroke victims vary based on the severity of the stroke, but often patients report experiencing numbness or paralysis in their limbs or face, difficulty walking, talking and understanding.
Steinberg and his team at Stanford are developing a stem cell treatment to help stroke patients. Steinberg believes that not all brain cells die during a stroke, but rather some brain cells become “dormant” and stop functioning instead. By transplanting stem cells derived from donated bone marrow into the brains of stroke patients, Steinberg thinks he can wake up these dormant cells much like how the prince wakens Sleeping Beauty from her century of enchanted sleep.
Basically, the transplanted cells act like a defibrillator for the dormant cells in the stroke-damaged area of the brain. Steinberg thinks that the transplanted cells secrete proteins that signal dormant brain cells to wake up and start functioning normally again, and that they also trigger a “helpful immune response” that prompts the brain to repair itself.
Sonia has seen first hand how a stroke can rob you of even your most basic abilities.
Steinberg tested this stem cell treatment in a small clinical trial back in 2013. 18 patients were treated and many of them showed improvements in their symptoms. The Smithsonian piece mentions a particular patient who had a remarkable response to the treatment. Sonia Olea Coontz, at age 32, suffered a stroke that robbed her of most of her speech and her ability to use her right arm and leg. After receiving Steinberg’s stem cell treatment, Sonia rapidly improved and was able to raise her arm above her head and gained most of her speech back. You can read more about her experience in our Stories of Hope.
In collaboration with a company called SanBio, Steinberg’s team is now testing this stem cell therapy in 156 stroke patients in a CIRM-funded phase 2 clinical trial. The trial will help answer the question of whether this treatment is safe and also effective in a larger group of patients.
The Smithsonian article, which I highly recommend reading, shared Steinberg’s future aspirations to pursue stem cell therapies for traumatic brain and spinal cord injuries as well as neurodegenerative diseases like Alzheimer’s, Parkinson’s and ALS.
Capricor Approved to Launch New Clinical Trial for Duchenne Muscular Dystrophy
On Wednesday, Capricor Therapeutics achieved an exciting milestone for its leading candidate CAP-1002 – a stem cell-based therapy developed to treat boys and young men with a muscle-wasting disease called Duchenne muscular dystrophy (DMD).
The Los Angeles-based company announced that it received approval from the US Food and Drug Administration (FDA) for their investigational new drug (IND) application to launch a new clinical trial called HOPE II that’s testing repeated doses of CAP-1002 cells in DMD patients. The cells are derived from donated heart tissue and are believed to release regenerative factors that strengthen heart and other muscle function in DMD patients.
Earlier this year, the company shared encouraging, positive results from the HOPE-1 trial suggesting that the therapy was improving some heart function and upper limb movement six months after treatment and was well-tolerated in patients. The goal of the new trial will be to determine whether giving patients repeated doses of the cell therapy over time will extend the benefits in upper limb movement in DMD patients.
In a news release, Capricor President and CEO Dr. Linda Marbán shared her company’s excitement for the launch of their new trial and what this treatment could mean for DMD patients,
Linda Marban, CEO of Capricor Therapeutics
“The FDA’s clearance of this IND upon its initial submission is a significant step forward in our development of CAP-1002. While there are many clinical initiatives in Duchenne muscular dystrophy, this is one of the very few to focus on non-ambulant patients. These boys and young men are looking to maintain what function they have in their arms and hands and, based on our previous study, we think CAP-1002 may be able to do exactly that.”
The Salk team. From left: Krishna Vadodaria, Lynne Moore, Carol Marchetto, Arianna Mei, Fred H. Gage, Callie Fredlender, Ruth Keithley, Ana Diniz Mendes. Photo courtesy Salk Institute
Astrocytes are some of the most common cells in the brain and central nervous system but they often get overlooked because they play a supporting role to the more glamorous neurons (even though they outnumber them around 50 to 1). But a new way of growing those astrocytes outside the brain could help pave the way for improved treatments for stroke, Alzheimer’s and other neurological problems.
Astrocytes – which get their name because of their star shape (Astron – Greek for “star” and “kyttaron” meaning cell) – have a number of key functions in the brain. They provide physical and metabolic support for neurons; they help supply energy and fuel to neurons; and they help with detoxification and injury repair, particularly in terms of reducing inflammation.
Studying these astrocytes in the lab has not been easy, however, because existing methods of producing them have been slow, cumbersome and not altogether effective at replicating their many functions.
Finding a better way
Now a team at the Salk Institute, led by CIRM-funded Professor Fred “Rusty” Gage, has developed a way of using stem cells to create astrocytes that is faster and more effective.
“This work represents a big leap forward in our ability to model neurological disorders in a dish. Because inflammation is the common denominator in many brain disorders, better understanding astrocytes and their interactions with other cell types in the brain could provide important clues into what goes wrong in disease.”
Stylized microscopy image of an astrocyte (red) and neuron (green). (Salk Institute)
In a step by step process the Salk team used a series of chemicals, called growth factors, to help coax stem cells into becoming, first, generic brain cells, and ultimately astrocytes. These astrocytes not only behaved like the ones in our brain do, but they also have a particularly sensitive response to inflammation. This gives the team a powerful tool in helping develop new treatment to disorders of the brain.
But wait, there’s more!
As if that wasn’t enough, the researchers then used the same technique to create astrocytes from induced pluripotent stem cells (iPSCs) – adult cells, such as skin, that have been re-engineered to have the ability to turn into any other kind of cell in the body. Those man-made astrocytes also showed the same characteristics as natural ones do.
Krishna Vadodaria, one of the lead authors on the paper, says having these iPSC-created astrocytes gives them a completely new tool to help explore brain development and disease, and hopefully develop new treatments for those diseases.
“The exciting thing about using iPSCs is that if we get tissue samples from people with diseases like multiple sclerosis, Alzheimer’s or depression, we will be able to study how their astrocytes behave, and how they interact with neurons.”
![AskExpertsMAY2018[1]](https://aholdencirm.files.wordpress.com/2018/05/askexpertsmay20181.png)
It’s not often you get a chance to ask a world class stem cell expert a question about their work, and how it might help you or someone you love. But on Thursday, May 31 you can do just that.
The Stem Cellar 