Inspiring Video: UC Irvine Stem Cell Trial Gives Orange County Woman Hope in Her Fight Against ALS

Stephen Hawking

Last week, we lost one of our greatest, most influential scientific minds. Stephen Hawking, a famous British theoretical physicist and author of “A Brief History of Time: From the Big Bang to Black Holes”, passed away at the age of 76.

Hawking lived most of his adult life in a wheelchair because he suffered from amyotrophic lateral sclerosis (ALS). Also known as Lou Gehrig’s disease, ALS causes the degeneration of the nerve cells that control muscle movement.

When Hawking was diagnosed with ALS at the age of 21, he was told he only had three years to live. But Hawking defied the odds and went on to live a life that not only revolutionized our understanding of the cosmos, but also gave hope to other patients suffering from this devastating degenerative disease.

A Story of Hope

Speaking of hope, I’d like to share another story of an Orange County woman name Lisa Wittenberg who was recently diagnosed with ALS. Her story was featured this week on KTLA5 news and is also available on the UC Irvine Health website.

VIDEO: UCI Health stem cell trial helps Orange County woman fight neurodegenerative disease ALS. Click on image to view video in new window.

In this video, Lisa describes how quickly ALS changed her life. She was with her family sledding in the snow last winter, and only a year later, she is in a wheelchair unable to walk. Lisa got emotional when she talked about how painful it is for her to see her 13-year-old son watch her battle with this disease.

But there is hope for Lisa in the form of a stem cell clinical trial at the UC Irvine CIRM Alpha Stem Cell Clinic. Lisa enrolled in the Brainstorm study, a CIRM-funded phase 3 trial that’s testing a mesenchymal stem cell therapy called NurOwn. BrainStorm Cell Therapeutics, the company sponsoring this trial, is isolating mesenchymal stem cells from the patient’s own bone marrow. The stem cells are then cultured in the lab under conditions that convert them into biological factories secreting a variety of neurotrophic factors that help protect the nerve cells damaged by ALS. The modified stem cells are then transplanted back into the patient where they will hopefully slow the progression of the disease.

Dr. Namita Goyal, a neurologist at UC Irvine Health involved in the trial, explained in the KTLA5 video that they are hopeful this treatment will give patients more time, and optimistic that in some cases, it could improve some of their symptoms.

Don’t Give Up the Fight

The most powerful part of Lisa’s story to me was the end when she says,

“I think it’s amazing that I get to fight, but I want everybody to get to fight. Everybody with ALS should get to fight and should have hope.”

Not only is Lisa fighting by being in this ground-breaking trial, she is also participated in the Los Angeles marathon this past weekend, raising money for ALS research.

More patients like Lisa will get the chance to fight as more potential stem cell treatments and drugs enter clinical trials. Videos like the one in this blog are important for raising awareness about available clinical trials like the Brainstorm study, which, by the way, is still looking for more patients to enroll (contact information for this trial can be found on the website here). CIRM is also funding another stem cell trial for ALS at the Cedars-Sinai Medical Center. You can read more about this trial on our website.

Lisa’s powerful message of fighting ALS and having hope reminds me of one of Stephen Hawking’s most famous quotes, which I’ll leave you with:

“Remember to look up at the stars and not down at your feet. Try to make sense of what you see and wonder about what makes the Universe exist. Be curious. And however difficult life may seem, there is always something you can do and succeed at. It matters that you don’t just give up.”

Related Links:

Stem Cell Roundup: Improving muscle function in muscular dystrophy; Building a better brain; Boosting efficiency in making iPSC’s

Here are the stem cell stories that caught our eye this week.

Photos of the week

TGIF! We’re so excited that the weekend is here that we are sharing not one but TWO amazing stem cell photos of the week.

RMI IntestinalChip

Image caption: Cells of a human intestinal lining, after being placed in an Intestine-Chip, form intestinal folds as they do in the human body. (Photo credit: Cedars-Sinai Board of Governors Regenerative Medicine Institute)

Photo #1 is borrowed from a blog we wrote earlier this week about a new stem cell-based path to personalized medicine. Scientists at Cedars-Sinai are collaborating with a company called Emulate to create intestines-on-a-chip using human stem cells. Their goal is to create 3D-organoids that represent the human gut, grow them on chips, and use these gut-chips to screen for precision medicines that could help patients with intestinal diseases. You can read more about this gut-tastic research here.

Young mouse heart 800x533

Image caption: UCLA scientists used four different fluorescent-colored proteins to determine the origin of cardiomyocytes in mice. (Image credit: UCLA Broad Stem Cell Research Center/Nature Communications)

Photo #2 is another beautiful fluorescent image, this time of a cross-section of a mouse heart. CIRM-funded scientists from UCLA Broad Stem Cell Research Center are tracking the fate of stem cells in the developing mouse heart in hopes of finding new insights that could lead to stem cell-based therapies for heart attack victims. Their research was published this week in the journal Nature Communications and you can read more about it in a UCLA news release.

Stem cell injection improves muscle function in muscular dystrophy mice

Another study by CIRM-funded Cedars-Sinai scientists came out this week in Stem Cell Reports. They discovered that they could improve muscle function in mice with muscular dystrophy by injecting cardiac progenitor cells into their hearts. The injected cells not only improved heart function in these mice, but also improved muscle function throughout their bodies. The effects were due to the release of microscopic vesicles called exosomes by the injected cells. These cells are currently being used in a CIRM-funded clinical trial by Capricor therapeutics for patients with Duchenne muscular dystrophy.

How to build a better brain (blob)

For years stem cell researchers have been looking for ways to create “mini brains”, to better understand how our own brains work and develop new ways to repair damage. So far, the best they have done is to create blobs, clusters of cells that resemble some parts of the brain. But now researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have come up with a new method they think can advance the field.

Their approach is explained in a fascinating article in the journal Science News, where lead researcher Bennet Novitch says finding the right method is like being a chef:

“It’s like making a cake: You have many different ways in which you can do it. There are all sorts of little tricks that people have come up with to overcome some of the common challenges.”

Brain cake. Yum.

A more efficient way to make iPS cells


Shinya Yamanaka. (Image source: Ko Sasaki, New York Times)

In 2006 Shinya Yamanaka discovered a way to take ordinary adult cells and reprogram them into embryonic-like stem cells that have the ability to turn into any other cell in the body. He called these cells induced pluripotent stem cells or iPSC’s. Since then researchers have been using these iPSC’s to try and develop new treatments for deadly diseases.

There’s been a big problem, however. Making these cells is really tricky and current methods are really inefficient. Out of a batch of, say, 1,000 cells sometimes only one or two are turned into iPSCs. Obviously, this slows down the pace of research.

Now researchers in Colorado have found a way they say dramatically improves on that. The team says it has to do with controlling the precise levels of reprogramming factors and microRNA and…. Well, you can read how they did it in a news release on Eurekalert.




Stem cell-based gut-on-a-chip: a new path to personalized medicine

“Personalized medicine” is a trendy phrase these days, frequently used in TV ads for hospitals, newspaper articles about medicine’s future and even here in the Stem Cellar. The basic gist is that by analyzing a patient’s unique biology, a physician can use disease treatments that are most likely to work in that individual.


Emulate’s Organ-on-a-Chip device.
Image: Emulate, Inc.

This concept is pretty straight-forward but it’s not always clear to me how it would play out as a routine clinical service for patients. A recent publication in Cellular and Molecular Gastroenterology and Hepatology by scientists at Cedars-Sinai and Emulate, Inc. paints a clearer picture. The report describes a device, Emulate’s Intestine-Chip, that aims to personalize drug treatments for people suffering from gastrointestinal diseases like inflammatory bowel disease and Chrohn’s disease.

Intestine-Chip combines the cutting-edge technologies of induced pluripotent stem cells (iPSCs) and microfluidic engineering. For the iPSC part of the equation, skin or blood samples are collected from a patient and reprogrammed into stem cells that can mature into almost any cell type in the body. Grown under the right conditions in a lab dish, the iPSCs self-organize into 3D intestinal organoids, structures made up of a few thousand cells with many of the hallmarks of a bona fide intestine.


Miniature versions of a human intestinal lining, known as organoids, derived from induced pluripotent stem cells (iPSCs).
Image: Cedars-Sinai Board of Governors Regenerative Medicine Institute

These iPSC-derived organoids have been described in previous studies and represent a breakthrough for studying human intestinal diseases. Yet, they vary a lot in shape and size, making it difficult to capture consistent results. And because the intestinal organoids form into hollow tubes, it’s a challenge to get drugs inside the organoid, a necessary step to systematically test the effects of various drugs on the intestine.

The Intestine-Chip remedies these drawbacks. About the size of a double A battery, the Chip is made up of specialized plastic engineered with tiny tunnels, or micro-channels. The research team placed the iPSC-derived intestinal organoid cells into the micro-channels and showed that passing fluids with a defined set of ingredients through the device can prod the cells to mimic the human intestine.

RMI IntestinalChip

Cells of a human intestinal lining, after being placed in an Intestine-Chip, form intestinal folds as they do in the human body. Image: Cedars-Sinai Board of Governors Regenerative Medicine Institute

The Intestine-Chip not only looks like a human intestine but acts like one too. A protein known to be at high levels in inflammatory bowel disease was passed through the microchannel and the impact on the intestinal cells matched what is seen in patients. Clive Svendsen, Ph.D., a co-author on the study and director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, explained the exciting applications that the Intestine-Chip opens up for patients:


Clive Svendsen

“This pairing of biology and engineering allows us to re-create an intestinal lining that matches that of a patient with a specific intestinal disease—without performing invasive surgery to obtain a tissue sample,” he said in a press release. “We can produce an unlimited number of copies of this tissue and use them to evaluate potential therapies. This is an important advance in personalized medicine.”

Emulate’s sights are not just set on the human intestine but for the many other organs affected by disease. And because disease rarely impacts only one organ, a series of Organs-on-Chips for a particular patient could be examined together. Geraldine A. Hamilton, Ph.D., president and chief scientific officer of Emulate, Inc. summed up this point in a companion press release:


Geraldine Hamilton

“By creating a personalized Patient-on-a-Chip, we can really begin to understand how diseases, medicines, chemicals and foods affect an individual’s health.”



Using heart stem cells to help boys battling a deadly disorder



Caleb Sizemore, a young man with DMD, speaks to the CIRM Board about his treatment in the Capricor clinical trial.

It’s hard to imagine how missing just one tiny protein can have such a devastating impact on a person. But with Duchenne Muscular Dystrophy (DMD) the lack of a single protein called dystrophin has deadly consequences. Now a new study is offering hope we may be able to help people with this rare genetic disorder.

DMD is a muscle wasting condition that steadily destroys the muscles in the arms and legs, heart and respiratory system. It affects mostly boys and it starts early in life, sometimes as young as 3 years old, and never lets up. By early teens many boys are unable to walk and are in a wheelchair. Their heart and breathing are also affected. In the past most people with DMD didn’t survive their teens. Now it’s more common for them to live into their 20’s and 30’s, but not much beyond that.

Results from a clinical trial being run by Capricor Therapeutics – and funded by CIRM – suggest we may be able to halt, and even reverse, some of the impacts of DMD.

Capricor has developed a therapy called CAP-1002 using cells derived from heart stem cells, called cardiospheres. Boys and young men with DMD who were treated with CAP-1002 experienced what Capricor calls “significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function.”

In a news release Dr. Ronald Victor, a researcher at Cedars-Sinai Heart Institute and the lead investigator for the trial, said they followed these patients for 12 months after treatment and the results are encouraging:

“Because Duchenne muscular dystrophy is a devastating, muscle-wasting disease that causes physical debilitation and eventually heart failure, the improvements in heart and skeletal muscle in those treated with a single dose of CAP-1002 are very promising and show that a subsequent trial is warranted. These early results provide hope for the Duchenne community, which is in urgent need of a major therapeutic breakthrough.”

According to the 12-month results:

  • 89 percent of patients treated with CAP-1002 showed sustained or improved muscle function compared to untreated patients
  • The CAP-1002 group had improved heart muscle function compared to the untreated group
  • The CAP-1002 group had reduced scarring on their heart compared to the untreated group.

Now, these results are still very early stage and there’s a danger in reading too much into them. However, the fact that they are sustained over one year is a promising sign. Also, none of the treated patients experienced any serious side effects from the therapy.

The team at Capricor now plans to go back to the US Food and Drug Administration (FDA) to get clearance to launch an even larger study in 2018.

For a condition like DMD, that has no cure and where treatments can simply slow down the progression of the disorder, this is a hopeful start.

Caleb Sizemore is one of the people treated in this trial. You can read his story and listen to him describing the impact of the treatment on his life.

Can Stem Cell Therapies Help ALS Patients?

A scientist’s fifteen-year journey to develop a stem cell-based therapy that could one day help ALS patients.

Jan Kaufman

Photo of Clive Svendsen (top left) and Jan & Jeff Kaufman

“Can stem cells help me Clive?”

The sentence appeared slowly on a computer screen, each character separated by a pause while its author searched for the next character using a device controlled by his eye muscle.

The person asking the question was Jeff Kaufman, a Wisconsin man in his 40s completely paralyzed by amyotrophic lateral sclerosis (ALS). On the receiving end was Clive Svendsen, PhD, then a scientist at the University of Wisconsin-Madison, determined to understand how stem cells could help patients like Jeff.

Also known as Lou Gehrig’s disease, ALS is a rapid, aggressive neurodegenerative disease with a two to four-year life expectancy. ALS destroys the nerve cells that send signals from the brain and spinal cord to the muscles that control movement. Denervation, or loss of nerves, causes muscle weakness and atrophy, leaving patients unable to control their own bodies. Currently there are two FDA-approved ALS drugs in the US – riluzole and a new drug called edaravone (Radicava). However, they only slow disease progression in some ALS patients by a few months and there are no effective treatments that stop or cure the disease.

Given this poor prognosis, making ALS the focus of his research career was an easy decision. However, developing a therapeutic strategy was challenging to Svendsen. “The problem with ALS is we don’t know the cause,” he said. “Around 10% of ALS cases are genetic, and we know some of the genes involved, but 90% of cases are sporadic.” He explained that this black box makes it difficult for scientists to know where to start when trying to develop treatments for sporadic ALS cases that have no drug targets.

From Parkinson’s disease to ALS

Svendsen, who moved to Cedars-Sinai in Los Angeles to head the Cedars-Sinai Board of Governors Regenerative Medicine Institute in 2010, has worked on ALS for the past 15 years. Before that, he studied Parkinson’s disease, a long-term neurodegenerative disorder that affects movement, balance and speech. Unlike ALS, Parkinson’s patients have a longer life expectancy and more treatment options that alleviate symptoms of the disease, making their quality of life far better than ALS patients.

Clive Svendsen, PhD, Director, Regenerative Medicine Institute. (Image courtesy of Cedars-Sinai)

“I chose to work on ALS mainly because of the effects it has on ALS families,” explained Svendsen. “Being normal one day, and then becoming rapidly paralyzed was hard to see.”

The transition from Parkinson’s to ALS was not without a scientific reason however. Svendsen was studying how an important growth factor in the brain called Glial Cell Line-Derived Neurotrophic Factor or GDNF could be used to protect dopamine neurons in order to treat Parkinson’s patients. However other research suggested that GDNF was even more effective at protecting motor neurons, the nerve cells destroyed by ALS.

Armed with the knowledge of GDNF’s ability to protect motor neurons, Svendsen and his team developed an experimental stem cell-based therapy that they hoped would treat patients with the sporadic form of ALS. Instead of using stem cells to replace the motor neurons lost to ALS, Svendsen placed his bets on making another cell type in the brain, the astrocyte.

Rooting for the underdog

Astrocytes are the underdog cells of the brain, often overshadowed by neurons that send and receive information from the central nervous system to our bodies. Astrocytes have many important roles, one of the most critical being to support the functions of neurons. In ALS, astrocytes are also affected but in a different way than motor neurons. Instead of dying, ALS astrocytes become dysfunctional and thereby create a toxic environment inhospitable to the motors neurons they are supposed to assist.

Fluorescent microscopy of astrocytes (red) and cell nuclei (blue). Image: Wikipedia.

“While the motor neurons clearly die in ALS, the astrocytes surrounding the motor neurons are also sick,” said Svendsen. “It’s a huge challenge to replace a motor neuron and make it grow a cable all the way to the muscle in an adult human. We couldn’t even get this to work in mice. So, I knew a more realistic strategy would be to replace the sick astrocytes in an ALS patients with fresh, healthy astrocytes. This potentially would have a regenerative effect on the environment around the existing motor neurons.”

The big idea was to combine both GDNF and astrocyte replacement. Svendsen set out to make healthy astrocytes from human brain stem cells that also produce therapeutic doses of GDNF and transplant these cells into the ALS patient spinal cord. Simply giving patients GDNF via pill wouldn’t work because the growth factor is unable to enter the brain or spinal cord tissue where it is needed. The hope, instead, was that the astrocytes would secrete the protective factor that would keep the patients’ motor neurons healthy and alive.

With critical funding from a CIRM Disease Team grant, Svendsen and his colleagues at Cedars-Sinai tested the feasibility of transplanting human brain stem cells (also referred to as neural progenitor cells) that secreted GDNF into a rat model of ALS. Their results were encouraging – the neural progenitor cells successfully developed into astrocytes and secreted GDNF, which collectively protected the rat motor neurons.

Svendsen describes the strategy as “a double whammy”: adding both healthy astrocytes and GDNF secretion to protect the motor neurons. “Replacing astrocytes has the potential to rejuvenate the niche where the motor neurons are. I think that’s a very powerful experimental approach to ALS.”

A fifteen year journey from bench to bedside

With promising preclinical data under his belt, Svendsen and his colleagues, including Robert Baloh, MD, PhD, director of neuromuscular medicine at the Cedars-Sinai Department of Neurology, and neurosurgeon J. Patrick Johnson, MD, designed a clinical trial that would test this experimental therapy in ALS patients. In October 2016, CIRM approved funding for a Phase I/IIa clinical trial assessing the safety of this novel human neural progenitor cell and gene therapy.

Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, and Robert Baloh, MD, PhD, director of neuromuscular medicine in the Cedars-Sinai Department of Neurology, in the lab. Svendsen is the sponsor of a current ALS clinical trial at Cedars-Sinai and the overall director of the program. Baloh is the principal investigator for the clinical trial. (Image courtesy of Cedars-Sinai)

This is a first-in-human study, and as such, the U.S. Food and Drug Administration (FDA) required the team to transplant the cells into only one side of the lumbar spinal cord, which effectively means that only one of the patient’s legs will get the treatment. This will allow for a comparison of the function and progression of ALS in the leg on the treated side of the spinal cord compared with the leg on the untreated side.

The trial was approved to treat a total of 18 patients and started in May 2017.

 Svendsen, who first started working on ALS back in 2002, describes his path to the clinic as a “very long and windy road.” He emphasized that this journey wouldn’t be possible without the hard work of his team, Cedars-Sinai and financial support from CIRM.

“It took ten years of preclinical studies and an enormous amount of work from many different people. Just producing the cells that we’re going to use took three years and a lot of trials and tribulations to make it a clinically viable product. It was really thanks to CIRM’s funding and the support of Cedars-Sinai that we got through it all. Without that kind of infrastructure, I can safely say we wouldn’t be here today.”

This “behind-the-scenes” view of how much time and effort it takes to translate a stem cell therapy from basic research into the clinic isn’t something that the public is often exposed to or aware of. Just as “Rome wasn’t built in a day,” Svendsen stressed that good quality stem cell trials take time, and that it’s important for people know how complicated these trials are.

It’s all about the patients

So, what motivates Svendsen to continue this long and harrowing journey to develop a treatment for ALS? He said the answer is easy. “I’m doing it for the patients,” he explained. “I’m not doing this for the money or glory. I just want to develop something that works for ALS, so we can help these patients.”

Svendsen revisited his story about Jeff Kaufman, a man he befriended at the Wisconsin ALS Chapter in 2003. Jeff had three daughters and a son, a wonderful wife, and was a successful lawyer when he was diagnosed with ALS.

“Jeff had basically everything, and then he was stricken with ALS. I still remember going to his house and he could only move his eyes at that point. He tapped out the words ‘Can stem cells help me Clive?’ on his computer screen. And my heart sank because I knew how much and how long it was going to take. I was very realistic so I said, ‘Yes Jeff, but it’s going to take time and money. And even then, it’s a long shot.’ And he told me to go for it, and that stuck in my brain.”

It’s people like Jeff that make Svendsen get out of bed every morning and doggedly pursue a treatment for ALS. Sadly, Jeff passed away due to complications from ALS in 2010. Svendsen says what Jeff and other patients go through is tragic and unfair.

“There’s a gene that goes along with ALS and it’s called the ‘nice person gene,’” he said. “People with ALS are nice. I can’t explain it, but neurologists would say the same thing. You feel like it’s just not fair that it happens to those people.”

The future of stem cell therapies for ALS

It’s clear from speaking with Svendsen, that he is optimistic about the future of stem cell-based therapies for ALS. Scientists still need to unravel the actual causes of ALS. But the experimental stem cell treatments currently in development, including Svendsen’s, will hopefully prove effective at delaying disease progression and give ALS patients more quality years to live.

In the meantime, what concerns Svendsen is how vulnerable ALS patients are to being misled by unapproved stem cell clinics that claim to have cures. “Unfortunately, there are a lot of charlatans out there, and there are a lot of false claims being made. People feed off the desperation that you have in ALS. It’s not fair, and it’s completely wrong. They’ll mislead patients by saying ‘For $40,000 you can get a cure!’”

Compelling stories of patients cured of knee pain or diseases like ALS with injections of their own adult stem cells pop up in the news daily. Many of these stories refer to unapproved treatments from clinics that don’t provide scientific evidence that these treatments are safe and effective. Svendsen said there are reasonable, research-backed trials that are attempting to use adult stem cells to treat ALS. He commented, “I think it’s hard for the public to wade through all of these options and understand what’s real and what’s not real.”

Svendsen’s advice for ALS patients interested in enrolling in a stem cell trial or trying a new stem cell treatment is to be cautious. If a therapy sounds too good to be true, it probably is, and if it costs a lot of money, it probably isn’t legitimate, he explained.

He also wants patients to understand the reality of the current state of ALS stem cell trials. The approved stem cell trials he is aware of are not at the treatment stage yet.

“If you’re enrolled in a stem cell trial that is funded and reputable, then they will tell you honestly that it’s not a treatment. There is currently no approved treatment using stem cells for ALS,” Svendsen said.

This might seem like discouraging news to patients who don’t have time to wait for these trials to develop into treatments, but Svendsen pointed out that the when he started his research 15 years ago, the field of stem cell research was still in its infancy. A lot has been accomplished in the past decade-and-a-half and with talented scientists dedicated to ALS research like Svendsen, the next 15 years will likely offer new insights into ALS and hopefully stem cell-based treatments for a devastating disease that has no cure.

Svendsen hopes that one day, when someone like Jeff Kaufman asks him “Can stem cells help me Clive?” He’ll be able to say, yes they can, yes they can.

CIRM-Funded Clinical Trials Targeting the Heart, Pancreas, and Kidneys

This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs.

This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Today we are featuring trials in our organ systems portfolio, specifically focusing on diseases of the heart/vasculature system, the pancreas and the kidneys.

CIRM has funded a total of nine trials targeting these disease areas, and eight of these trials are currently active. Check out the infographic below for a list of our currently active trials.

For more details about all CIRM-funded clinical trials, visit our clinical trials page and read our clinical trials brochure which provides brief overviews of each trial.

CIRM-Funded Clinical Trials Targeting Brain and Eye Disorders

This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs.

 This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Our Agency has funded a total of 40 trials since its inception. 23 of these trials were funded after the launch of our Strategic Plan in 2016, bringing us close to the half way point of our goal to fund 50 new clinical trials by 2020.

Today we are featuring CIRM-funded trials in our neurological and eye disorders portfolio.  CIRM has funded a total of nine trials targeting these disease areas, and seven of these trials are currently active. Check out the infographic below for a list of our currently active trials.

For more details about all CIRM-funded clinical trials, visit our clinical trials page and read our clinical trials brochure which provides brief overviews of each trial.

Treatments, cures and clinical trials: an in-person update on CIRM’s progress

Patients and Patient Advocates are at the heart of everything we do at CIRM. That’s why we are holding three free public events in the next few months focused on updating you on the stem cell research we are funding, and our plans for the future.

Right now we have 33 projects that we have funded in clinical trials. Those range from heart disease and stroke, to cancer, diabetes, ALS (Lou Gehrig’s disease), two different forms of vision loss, spinal cord injury and HIV/AIDS. We have also helped cure dozens of children battling deadly immune disorders. But as far as we are concerned we are only just getting started.

Over the course of the next few years, we have a goal of adding dozens more clinical trials to that list, and creating a pipeline of promising therapies for a wide range of diseases and disorders.

That’s why we are holding these free public events – something we try and do every year. We want to let you know what we are doing, what we are funding, how that research is progressing, and to get your thoughts on how we can improve, what else we can do to help meet the needs of the Patient Advocate community. Your voice is important in helping shape everything we do.

The first event is at the Gladstone Institutes in San Francisco on Wednesday, September 6th from noon till 1pm. The doors open at 11am for registration and a light lunch.

Gladstone Institutes

Here’s a link to an Eventbrite page that has all the information about the event, including how you can RSVP to let us know you are coming.

We are fortunate to be joined by two great scientists, and speakers – as well as being CIRM grantees-  from the Gladstone Institutes, Dr. Deepak Srivastava and Dr. Steve Finkbeiner.

Dr. Srivastava is working on regenerating heart muscle after it has been damaged. This research could not only help people recover from a heart attack, but the same principles might also enable us to regenerate other organs damaged by disease. Dr. Finkbeiner is a pioneer in diseases of the brain and has done ground breaking work in both Alzheimer’s and Huntington’s disease.

We have two other free public events coming up in October. The first is at UC Davis in Sacramento on October 10th (noon till 1pm) and the second at Cedars-Sinai in Los Angeles on October 30th (noon till 1pm). We will have more details on these events in the coming weeks.

We look forward to seeing you at one of these events and please feel free to share this information with anyone you think might be interested in attending.

Family, faith and funding from CIRM inspire one patient to plan for his future

Caleb Sizemore speaks to the CIRM Board at the June 2017 ICOC meeting.

Having been to many conferences and meetings over the years I have found there is a really simple way to gauge if someone is a good speaker, if they have the attention of people in the room. You just look around and see how many people are on their phones or laptops, checking their email or the latest sports scores.

By that standard Caleb Sizemore is a spellbinding speaker.

Last month Caleb spoke to the CIRM Board about his experiences in a CIRM-funded clinical trial for Duchenne Muscular Dystrophy. As he talked no one in the room was on their phone. Laptops were closed. All eyes and ears were on him.

To say his talk was both deeply moving and inspiring is an understatement. I could go into more detail but it’s so much more powerful to hear it from  Caleb himself. His words are a reminder to everyone at CIRM why we do this work, and why we have to continue to do all that we can to live up to our mission statement and accelerate stem cell treatments to patients with unmet medical needs.

Video produced by Todd Dubnicoff/CIRM

Related Links:

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


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

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

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

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

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


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

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

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

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

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

CIRM Board member and ALS Patient Advocate Diane Winokur

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

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

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

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

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