There’s a large body of research that shows that many people learn better through visuals. Studies show that much of the sensory cortex in our brain is devoted to vision so our brains use images rather than text to make sense of things.
That’s why we think it just makes sense to use visuals, as much as we can, when trying to help people understand advances in stem cell research. That’s precisely what our colleagues at U.C. San Diego are doing with a new show called “Stem Cell Science with Alysson Muotri”.
Alysson is a CIRM grantee
who is doing some exciting work in developing a deeper understanding of autism.
He’s also a really good communicator who can distill complex ideas down into
easy to understand language.
The show features Alysson,
plus other scientists at UCSD who are working hard to move the most promising
research out of the lab and into clinical trials in people. Appropriately the
first show in the series follows that path, exploring
how discoveries made using tiny Zebrafish could hopefully lead to stem cell
therapies targeting blood diseases like leukemia. This first show also highlights
the important role that CIRM’s Alpha Stem Cell Clinic Network will play in
bringing those therapies to patients.
You can find a sneak preview of the show on YouTube. The series proper will be broadcast on California local cable via the UCTV channel at 8:00 pm on Thursdays starting July 8, 2019.
And if you really
have a lot of time on your hands you can check out the more
than 300 videos CIRM has produced on every aspect of stem cell research
from cures for fatal diseases to questions to ask before taking part in a
When governments cut funding for scientific research the consequences can be swift, and painful. In Canada last week for example, the government of Ontario cut $5 million in annual funding for stem cell research, effectively ending a project developing a therapy to heal the damaged lungs of premature babies.
Here in the US the federal government is already placing restrictions on support for fetal tissue research and there is speculation embryonic stem cell research could be next. That’s why agencies like CIRM are so important. We don’t rely on a government giving us money every year. Instead, thanks to the voters of California, we have had a steady supply of funds to enable us to plan long-term and support multi-year projects.
But those funds
are due to run out soon. We anticipate funding our last new awards this year
and while we have enough money to continue supporting all the projects our
Board has already approved, we won’t be able to take on any new projects. That’s
bad news for the scientists and, ultimately, really bad for the patients who
are in need of new treatments for currently incurable diseases.
We are going to talk about that in two upcoming events.
The first is a
patient advocate event at UC San Diego
on Tuesday, May 28th from 12.30pm to 1.30pm. It’s free, there is parking and snacks and
refreshments will be available.
feature UC San Diego’s Dr. Catriona
Jamieson, CIRM’s President and CEO Dr.
Maria Millan and CIRM Board member and Patient Advocate for Parkinson’s
Disease, David Higgins PhD. The
three will talk about the exciting progress being made at UC San Diego and other
programs around California, but also the uncertain future and the impact that
could have for the field as a whole.
For all of you
who don’t live in the San Diego Area – or who do but can’t make it to the event
– we are holding a similar discussion online on a special Facebook Live: Ask the Stem Cell Team About the Future of Stem Cell
Research event on Thursday, May 30th
from noon till 1pm PDT.
features Dr. Millan and Dr. Higgins, but it also features UC Davis stem cell
scientist, CIRM-grantee and renowned blogger Paul Knoepfler PhD.
their own experience, expertise and perspective on the field and will discuss the
impact that a reduction in funding for stem cell research would have, not just
in the short term but in the long run.
Because we all
have a stake in what happens, both events – whether it’s in person or online – include
time for questions from you, the audience.
You can find our
Facebook Live: Ask the Stem Cell Team
About the Future of Stem Cell Research on our Facebook
page at noon on May 30th PDT
When CIRM was created in 2004 the field of stem cell research was still very much in its infancy. Fast forward 15 years and it’s moving ahead at a rapid pace, probably faster than most scientists would have predicted. How fast? Find out for yourself at a free public event at UC San Diego on May 28th from 12.30 to 1.30p.
In the last 15 years CIRM has funded 53 clinical
trials in everything from heart disease and stroke, to spinal cord injury,
vision loss, sickle cell disease and HIV/AIDS.
Clearly progress is being made. But the field is also facing some challenges. Funding at the federal level for stem cell research is under threat, and CIRM is entering what could be its final phase. We have enough money left to fund new projects through this year (and these are multi-year projects so they will run into 2021 or 2022) but unless there is a new round of funding we will slowly disappear. And with us, may also disappear the hopes of some of the most promising projects underway.
If CIRM goes, then projects that we have supported and nurtured through different phases of research may struggle to make it into a clinical trial because they can’t get the necessary funding.
Clearly this is a pivotal time in the field.
We will discuss all this, the past, the present and the uncertain future of stem cell research at the meeting at UC San Diego on May 28th. The doors will open at noon for registration (snacks and light refreshments will also be available) and the program runs from 12.30p to 1.30p.
The speakers are:
Jamieson, Director of the UC San Diego Health CIRM Alpha Stem Cell Clinic
and Sanford Stem Cell Clinical Center.
Millan, President and CEO of CIRM
Higgins, CIRM Board member and Patient Advocate for Parkinson’s Disease.
And of course, we want to hear from you, so we’ll leave
plenty of time for questions.
From Day One CIRM’s goal has been to advance stem cell research in California. We don’t do that just by funding the most promising research -though the 51 clinical trials we have funded to date clearly shows we do that rather well – but also by trying to bring the best minds in the field together to overcome problems.
Over the years we
have held conferences, workshops and symposiums on everything from Parkinson’s
palsy and tissue
engineering. Each one attracted the key players and stakeholders in the
field, brainstorming ideas to get past obstacles and to explore new ways of
developing therapies. It’s an attempt to get scientists, who would normally be
rivals or competitors, to collaborate and partner together in finding the best
It’s not easy to do,
and the results are not always obvious right away, but it is essential if we
hope to live up to our mission of accelerating stem cell therapies to patients
with unmet medical needs.
For example. This
past week we helped organize two big events and were participants in another.
The first event we
pulled together, in partnership with Cedars-Sinai Medical Center, was a
workshop called “Brainstorm Neurodegeneration”. It brought together leaders in stem
cell research, genomics, big data, patient advocacy and the Food and Drug
Administration (FDA) to tackle some of the issues that have hampered progress
in finding treatments for things like Parkinson’s, Alzheimer’s, ALS and
ambitiously subtitled the workshop “a cutting-edge meeting to disrupt the field”
and while the two days of discussions didn’t resolve all the problems facing us
it did produce some fascinating ideas and some tantalizing glimpses at ways to
advance the field.
Two days later we partnered with UC San Francisco to host the Fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium. This brought together the scientists who develop therapies, the doctors and nurses who deliver them, and the patients who are in need of them. The theme was “The Past, Present & Future of Regenerative Medicine” and included both a look at the initial discoveries in gene therapy that led us to where we are now as well as a look to the future when cellular therapies, we believe, will become a routine option for patients.
different groups together is important for us. We feel each has a key role to
play in moving these projects and out of the lab and into clinical trials and
that it is only by working together that they can succeed in producing the
treatments and cures patients so desperately need.
As always it was the patients who surprised us. One, Cierra Danielle Jackson, talked about what it was like to be cured of her sickle cell disease. I think it’s fair to say that most in the audience expected Cierra to talk about her delight at no longer having the crippling and life-threatening condition. And she did. But she also talked about how hard it was adjusting to this new reality.
Cierra said sickle
cell disease had been a part of her life for all her life, it shaped her daily
life and her relationships with her family and many others. So, to suddenly
have that no longer be a part of her caused a kind of identity crisis. Who was
she now that she was no longer someone with sickle cell disease?
She talked about how
people with most diseases were normal before they got sick, and will be normal
after they are cured. But for people with sickle cell, being sick is all they
have known. That was their normal. And now they have to adjust to a new normal.
It was a powerful
reminder to everyone that in developing new treatments we have to consider the
whole person, their psychological and emotional sides as well as the physical.
And so on to the third event we were part of, the Stanford Drug Discovery Symposium. This was a high level, invitation-only scientific meeting that included some heavy hitters – such as Nobel Prize winners Paul Berg and Randy Schekman, former FDA Commissioner Robert Califf. Over the course of two days they examined the role that philanthropy plays in advancing research, the increasingly important role of immunotherapy in battling diseases like cancer and how tools such as artificial intelligence and big data are shaping the future.
CIRM’s President and CEO, Dr. Maria Millan, was one of those invited to speak and she talked about how California’s investment in stem cell research is delivering Something Better than Hope – which by a happy coincidence is the title of our 2018 Annual Report. She highlighted some of the 51 clinical trials we have funded, and the lives that have been changed and saved by this research.
The presentations at
these conferences and workshops are important, but so too are the conversations
that happen outside the auditorium, over lunch or at coffee. Many great
collaborations have happened when scientists get a chance to share ideas, or
when researchers talk to patients about their ideas for a successful clinical
It’s amazing what happens when you bring people together who might otherwise never have met. The ideas they come up with can change the world.
Every day at CIRM we get emails and calls from people looking for a stem cell clinical trial to help them. Some have arthritis in the knee or hip and want to avoid surgery. Some have a child with autism and want something that will ease the symptoms. Some have cancer and conventional therapies no longer work for them. Many have run out of options. Some are running out of time.
It’s hard to tell
someone who is desperate that you don’t have anything that can help them, that
there are no stem cell clinical trials that would be appropriate for them. Many
often push back, saying they’ve seen ads online and visited websites for companies
that claim to have stem cell therapies that can help them. When I say those
therapies have not been approved by the Food and Drug Administration, or even
been shown to be safe let alone effective, I can hear the disappointment in
I know some will go on to try those therapies anyway, because they have nothing else. I don’t blame them. I might do the same myself.
But before making an informed decision about any therapy it is important for people to have all the facts in front of them.
That’s why we are
holding a special Facebook Live “Ask the
Stem Cell Team About Clinical Trials” event on Thursday, April 25th from noon till 1pm PDT.
We are bringing
together three experts who will help us all understand what’s a good clinical
trial, and what’s a bogus one. They will talk about:
Red flags that a stem cell “clinic” might be
more interested in making money than making you better
Key things to look for to choose a bona fide
stem cell clinical trial
What are the questions you need to ask before
signing up for any clinical
What are good sources of information to turn
to for guidance
The Stem Cell Team
will talk about CIRM’s Alpha Stem Cell Clinics Network, contrasting the time
and resources they devote to offering patients stem cell clinical trials that
are endorsed by the FDA, with clinics that promise people their own fat or
blood cells can fix everything from bad knees to multiple sclerosis.
Our experts include
a doctor and a nurse from the Alpha Clinics Network with years of experience in
running and managing clinical trials, plus our own Geoff Lomax who helps
support the entire network.
It will be an eye
opening, informative and engaging hour and we want you to be part of it. You can either join us on the day and post
questions for the panel to answer, or you can email
them directly to us beforehand at email@example.com.
For years we have talked about the “promise” and the “potential” of stem cells to cure patients. But more and more we are seeing firsthand how stem cells can change a patient’s life, even saving it in some cases. That’s the theme of the 4th Annual CIRM Alpha Stem Cell Clinics Network Symposium.
It’s not your usual
symposium because this brings together all
the key players in the field – the scientists who do the research, the nurses
and doctors who deliver the therapies, and the patients who get or need those
therapies. And, of course, we’ll be there; because without CIRM’s funding to
support that research and therapies none of this happens.
We are going to look
at some of the exciting progress being made, and what is on the horizon. But
along the way we’ll also tackle many of the questions that people pose to us
every day. Questions such as:
How can you distinguish between a good
clinical trial offering legitimate treatments vs a stem cell clinic offering sham
What about the Right to Try, can’t I just
demand I get access to stem cell therapies?
How do I sign up for a clinical trial, and how
much will it cost me?
What is the experience of patients that have
participated in a stem cell clinical trial?
researchers will also talk about the real possibility of curing diseases like
sickle cell disease on a national scale, which affect around 100,000 Americans,
mostly African Americans and Hispanics. They’ll discuss the use of gene editing
to battle hereditary diseases like Huntington’s. And they’ll highlight how they
can engineer a patient’s own immune system cells to battle deadly cancers.
So, join us for what
promises to be a fascinating day. It’s the cutting edge of science. And it’s
To Mend: (verb used with object) to make (something broken, worn, torn or otherwise damaged) whole, sound or usable by repairing.
It’s remarkable to believe, but today doctors literally have the tools to repair damaged cells. These tools are being used to treat people with diseases that were once incurable. The field of regenerative medicine has made tremendous progress in the last 15 years, but how did these tools come about and what is the experience of patients being treated with them?
These questions, and hopefully yours too, are going to be answered at the fourth annual CIRM Alpha Stem Cell Clinics Symposium on April 18, 2019 at the University of California at San Francisco.
The symposium is free, and the program is designed with patients and the public in mind, so don’t be shy and put your scientific thinking caps on! A complete agenda may be found here
Perhaps one of the most remarkable discoveries in the past decade are new tools that enable doctors to “edit” or correct a patient’s own DNA. DNA correction tools came about because of a remarkable string of scientific breakthroughs. The symposium will dive into this history and discuss how these tools are being used today to treat patients.
One specific example of the promise that DNA editing holds is for those with sickle cell disease (SCD), a condition where patients’ blood forming stem cells contain a genetic error that causes the disease. The symposium will describe how the CIRM Alpha Stem Cell Clinics Network, a series of medical centers across California whose focus is on stem cell clinical trials, are supporting work aimed at mending blood cells to cure debilitating diseases like SCD.
Doctors, nurses and patients involved with these trials will be telling their stories and describing their experiences. One important focus will be how Alpha Clinic teams are partnering with community members to ensure that patients, interested in new treatments, are informed about the availability of clinical trials and receive sufficient information to make the best treatment choices.
The fourth annual CIRM Alpha Stem Cell Clinics Symposium is an opportunity for patients, their families and the public to meet the pioneers who are literally mending a patients own stem cells to cure their disease.
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
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.
Every day in the U.S. four children are born with spina bifida. It is the most common cause of lifelong paralysis and also frequently leads to other serious health problems affecting the bowel and bladder. The impact on families is enormous. A new approach to repairing the defect that causes spina bifida was today awarded $5.66 million by the Board of the California Institute for Regenerative Medicine (CIRM).
In spina bifida the spinal cord doesn’t form properly, in many cases leaving a section of it open, exposing tissues and nerves. The current standard of care is surgery, but even this leaves almost 60% of children unable to walk independently. Diana Farmer MD, and Aijun Wang PhD at U.C. Davis will use mesenchymal stem cells, taken from a donor placenta, and place them on a form of synthetic scaffold over the injury site in the womb. Tests in animals show this approach was able to repair the defect and prevent paralysis.
“Spina bifida is a devastating condition for babies born with this disorder and the families who care for them,” says Maria T. Millan, MD, President & CEO of CIRM. “CIRM has funded this important work from its earliest stages and we are committed to working with Dr. Farmer’s team to moving this work to the stage where it can be tested in patients.”
The CLIN1 award will provide funding to enable the UC Davis team to do the final testing and preparations needed to apply to the FDA for permission to start a clinical trial.
Dr. Farmer says she and Dr. Wang, have been working on this approach for more than ten years and are excited about being able to take the next step.
“There were many times of frustration, many times when cell types we explored and worked with didn’t work,” says Dr. Farmer. “But it’s the patients, seeing them, talking to them and working with them, that keeps me motivated to do the science, to keep persevering.”
If this therapy is successful it will have a huge economic impact on California, and on the rest of the world. Because spina bifida is a lifelong condition involving many operations, many stays in the hospital and, in some cases, lifelong use of a wheelchair this has a huge financial, and psychological, burden on the family.
“It affects them in so many ways; parents having to miss work or take time off work to care for their child, other children in the family feeling neglected because their brother or sister needs so much attention,” says Dr. Farmer. “That’s why we are so grateful to CIRM. Because this is a rare disease and finding funding for those is hard. CIRM has been a perfect partner in helping bring this approach, blending stem cell therapy and tissue engineering, together to help these families.”
This video shows English bulldogs treated with this approach who are now able to walk:
The following article is an Op Ed that appeared in today’s print version of the San Francisco Chronicle
Biotechnology was born in California in the 1970s based on the discovery out of one of its universities and California is responsible for an industry that has impacted the lives of billions of people worldwide. In 2004, the voters of California approved Proposition 71, creating the California Institute for Regenerative Medicine and setting the state on the path to becoming a global leader in stem cell research. Today the therapies resulting from the institute’s work are not just changing lives, they are already saving lives.
Lives like Evie Vaccaro, who is alive today because of a treatment CIRM is funding. Vaccaro was born with SCID, also known as “bubble baby disease,” an immune disorder that often kills babies in their first two years. Vaccaro and dozens of other babies were given stem cell treatments thanks to the institute. All are showing improvement; some are now several years past treatment and considered cured.
An accident left Jake Javier from Danville paralyzed from the chest down on the eve of his high school graduation. Javier was treated in a CIRM-funded clinical trial. Today he has regained the use of his arms and hands, is driving a car and is a sophomore at Cal Poly San Luis Obispo. Five other patients treated at the same time as Javier have all experienced improvements meaning that instead of needing round-the-clock care, they can lead independent lives.
A study by the Tufts Center for the Study of Drug Development estimated it takes at least 10 years and $2.6 billion to develop one successful drug. In 14 years, and with just $3 billion, CIRM has funded 1,000 different projects, enrolled 900 patients, and supported 49 different clinical trials targeting diseases such as cancer, kidney failure and leukemia. Four of these programs have received an expedited designation by the U.S. Food and Drug Administration, meaning they could get faster approval to help more patients
We have created a network of world class medical clinics that have expertise in delivering treatments to patients. The CIRM Alpha Clinics offer treatments based on solid science, unlike the unlicensed clinics sprouting up around California that peddle unproven and potentially harmful therapies that cost patients thousands of dollars.
Supported the creation of 12 stem-cell research facilities in California
Attracted hundreds of top-tier researchers to California
Trained a new generation of stem-cell scientists
Brought clinical trials to California — for example, one targeting ALS or Lou Gehrig’s disease
Deployed rigorous scientific standards and support so our programs have a “seal of approval” to attract $2.7 billion in additional investments from industry and other sources.
We recently have partnered with the National Institutes of Health to break down barriers and speed up the approval process to bring curative treatments to patients with Sickle Cell Disease.
Have we achieved all we wanted to? Of course not. The first decade of CIRM’s life was laying the groundwork, developing the knowledge and expertise and refining processes so that we can truly accelerate progress. As a leader in this burgeoning field of regenerative medicine, CIRM needs to continue its mission of accelerating stem-cell treatments to patients with unmet medical needs.
Dr. Maria T. Millan is President and CEO and Jonathan Thomas, JD, PhD, is the Board Chairman of the California Institute of Regenerative Medicine.