Charting a new course for stem cell research

What are the latest advances in stem cell research targeting cancer? Can stem cells help people battling COVID-19 or even help develop a vaccine to stop the virus? What are researchers and the scientific community doing to help address the unmet medical needs of underserved communities? Those are just a few of the topics being discussed at the Annual CIRM Alpha Stem Cell Clinic Network Symposium on Thursday, October 8th from 9am to 1.30pm PDT.

Like pretty nearly everything these days the symposium is going to be a virtual event, so you can watch it from the comfort of your own home on a phone or laptop. And it’s free.

The CIRM Alpha Clinics are a network of leading medical centers here in California. They specialize in delivering stem cell and gene therapies to patients. So, while many conferences look at the promise of stem cell therapies, here we deal with the reality; what’s in the clinic, what’s working, what do we need to do to help get these therapies to patients in need?

It’s a relatively short meeting, with short presentations, but that doesn’t mean it will be short on content. Some of the best stem cell researchers in the U.S. are taking part so you’ll learn an awful lot in a short time.

We’ll hear what’s being done to find therapies for

  • Rare diseases that affect children
  • Type 1 diabetes
  • HIV/AIDS
  • Glioblastoma
  • Multiple myeloma

We’ll discuss how to create a patient navigation system that can address social and economic determinants that impact patient participation? And we’ll look at ways that the Alpha Clinic Network can partner with community care givers around California to increase patient access to the latest therapies.

It’s going to be a fascinating day. And did I mention it’s free!

All you have to do is go to this Eventbrite page to register.

And feel free to share this with your family, friends or anyone you think might be interested.

We look forward to seeing you there.

Building a progressive pipeline

Dr. Kelly Shepard

By Dr. Kelly Shepard

One of our favorite things to do at CIRM is deliver exciting news about CIRM projects. This usually entails discussion of recent discoveries that made headlines, or announcing the launch of a new CIRM-funded clinical trial …. tangible signs of progress towards addressing unmet medical needs through advances in stem technology.

But there are equally exciting signs of progress that are not always so obvious to the untrained eye-  those that we are privileged to witness behind the scenes at CIRM. These efforts don’t always lead to a splashy news article or even to a scientific publication, but they nonetheless drive the evolution of new ideas and can help steer the field away from futile lines of investigation. Dozens of such projects are navigating uncharted waters by filling knowledge gaps, breaking down technical barriers, and working closely with regulatory agencies to define novel and safe paths to the clinic.

These efforts can remain “hidden” because they are in the intermediate stages of the long, arduous and expensive journey from “bench to beside”.  For the pioneering projects that CIRM funds, this journey is unique and untrod, and can be fraught with false starts. But CIRM has developed tools to track the momentum of these programs and provide continuous support for those with the most promise. In so doing, we have watched projects evolve as they wend their way to the clinic. We wanted to share a few examples of how we do this with our readers, but first… a little background for our friends who are unfamiliar with the nuts and bolts of inventing new medicines.

A common metaphor for bringing scientific discoveries to market is a pipeline, which begins in a laboratory where a discovery occurs, and ends with government approval to commercialize a new medicine, after it is proven to be safe and effective. In between discovery and approval is a stage called “Translation”, where investigators develop ways to transition their “research level” processes to “clinically compatible” ones, which only utilize substances that are of certified quality for human use. 

Investigators must also work out novel ways to manufacture the product at larger scale and transition the methods used for testing in animal models to those that can be implemented in human subjects.

A key milestone in Translation is the “preIND” (pre Investigational New Drug (IND) meeting, where an investigator presents data and plans to the US Food and Drug Administration (FDA) for feedback before next stage of development begins, the pivotal testing needed to show it is both safe and effective.

These “IND enabling studies” are rigorous but necessary to support an application for an IND and the initiation of clinical trials, beginning with phase 1 to assess safety in a small number of individuals, and phase 2, where an expanded group is evaluated to see if the therapy has any benefits for the patient. Phase 3 trials are studies of very large numbers of individuals to gain definitive evidence of safety and therapeutic effect, generally the last step before applying to the FDA for market approval. An image of the pipeline and the stages described are provided in our diagram below.

The pipeline can be notoriously long and tricky, with plenty of twists, turns, and unexpected obstacles along the way. Many more projects enter than emerge from this gauntlet, but as we see from these examples of ‘works in progress”, there is a lot of momentum building.

Caption for Graphic: This graphic shows the number of CIRM-funded projects and the stages they have progressed through multiple rounds of CIRM funding. For example, the topmost arrow shows that are about 19 projects at the translational stage of the pipeline that received earlier support through one of CIRM’s Discovery stage programs. Many of these efforts came out of our pre-2016 funding initiatives such as Early Translation, Basic Biology and New Faculty Awards. In another example, you can see that about 15 awards that were first funded by CIRM at the IND enabling stage have since progressed into a phase 1 or phase 2 clinical trials. While most of these efforts also originated in some of CIRM’s pre-2016 initiatives such as the Disease Team Awards, others have already progressed from CIRM’s newer programs that were launched as part of the “2.0” overhaul in 2016 (CLIN1).

The number of CIRM projects that have evolved and made their way down the pipeline with CIRM support is impressive, but it is clearly an under-representation, as there are other projects that have progressed outside of CIRM’s purview, which can make things trickier to verify.

We also track projects that have spun off or been licensed to commercial organizations, another very exciting form of “progression”. Perhaps those will contribute to another blog for another day! In the meantime, here are a just a few examples of some of the progressors that are depicted on the graphic.

Project: stem cell therapy to enhance bone healing in the elderly

– Currently funded stage: IND enabling development, CLIN1-11256 (Dr. Zhu, Ankasa Regenerative Therapeutics)

– Preceded by preIND-enabling studies, TRAN1-09270 (Dr. Zhu, Ankasa Regenerative Therapeutics)

– Preceded by discovery stage research grant TR1-01249 (Dr. Longaker and Dr. Helm, Stanford)

Project: embryonic stem cell derived neural cell therapy for Huntington Disease

– Currently funded stage: IND enabling development, CLIN1-10953 (Dr. Thompson, UC Irvine)

– Preceded by preIND-enabling studies, PC1-08117 (Dr. Thompson, UC Irvine)

– Preceded by discovery stage research grant (TR2-01841) (Dr. Thompson, UC Irvine)

Project: gene-modified hematopoietic stem cells for Artemis Deficient severe combined immunodeficiency (SCID)

– Currently funded stage: Phase 1 clinical trial CLIN2-10830 (Dr. Cowan, UC San Francisco)

– Preceded by IND enabling development, CLIN1-08363 (Dr. Puck, UC San Francisco)

– Preceded by discovery stage research grant, TR3-05535  (Dr. Cowan, UC San Francisco)

Project: retinal progenitor cell therapy for retinitis pigmentosa

– Currently funded stage: Phase 2 and 2b clinical trials, CLIN2-11472, CLIN2-09698 (Dr. Klassen, JCyte, Inc.)

– Preceded by IND enabling development, DR2A-05739 (Dr. Klassen, UC Irvine)

– Preceded by discovery stage research grant, TR2-01794 (Dr. Klassen, UC Irvine)

Precision guided therapy from a patient’s own cells

Dr. Wesley McKeithan, Stanford

Imagine having a tool you could use to quickly test lots of different drugs against a disease to see which one works best. That’s been a goal of stem cell researchers for many years but turning that idea into a reality hasn’t been easy. That may be about to change.

A team of CIRM-funded researchers at the Stanford Cardiovascular Institute and the Human BioMolecular Research Institute in San Diego found a way to use stem cells from patients with a life-threatening heart disease, to refine an existing therapy to make it more effective, with fewer side effects.

The disease in question is called long QT syndrome (LQTS). This is a heart rhythm condition that can cause fast, chaotic heartbeats. Some people with the condition have seizures. In some severe cases, particularly in younger people, LQTS can cause sudden death.

There are a number of medications that can help keep LQTS under control. One of these is mexiletine. It’s effective at stabilizing the heart’s rhythm, but it also comes with some side effects such as stomach pain, chest discomfort, drowsiness, headache, and nausea.

The team wanted to find a way to test different forms of that medication to see if they could find one that worked better and was safer to take. So they used induced pluripotent stem cells (iPSCs) from patients with LQTS to do just that.

iPSCs are cells that are made from human tissue – usually skin – that can then be turned into any other cell in the body. In this case, they took tissue from people with LQTS and then turned them into heart cells called cardiomyocytes, the kind affected by the disease. The beauty of this technique is that even though these cells came from another source, they now look and act like cardiomyocytes affected by LQTS.

Dr. Mark Mercola, Stanford

In a news release Stanford’s Dr. Mark Mercola, the senior author of the study, said using these kinds of cells gave them a powerful tool.

“Drugs for heart disease are typically developed using overly simplified models, like tumor cells engineered in a specific way to mimic a biochemical event. Consequently, drugs like this one, mexiletine, have undesirable properties of concern in treating patients. Here, we used cells from a patient to generate that person’s heart muscle cells in a dish so we could visualize both the good and bad effects of the drug.”

The researchers then used these man-made cardiomyocytes to test various drugs that were very similar in structure to mexiletine. They were looking for ones that could help stabilize the heart arrhythmia but didn’t produce the unpleasant side effects. And they found some promising candidates.

Study first author, Dr. Wesley McKeithan, says the bigger impact of the study is that they were able to show how this kind of cell from patients with a particular disease can be used to “guide drug development and identify better drug improvement and optimization in a large-scale manner.”

 “Our approach shows the feasibility of introducing human disease models early in the drug development pipeline and opens the door for precision drug design to improve therapies for patients.”

The study is published in the journal Cell Stem Cell.

Meet the people who are changing the future

Kristin MacDonald

Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day.

I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial to treat retinitis pigmentosa (RP). The disease had destroyed Kristin’s vision and she was hoping the therapy, pioneered by jCyte, would help her. Kristin, being a bit of a pioneer herself, was the first person to test the therapy in the U.S.

Anyway, Kristin was doing a Zoom presentation and wanted to look her best so she asked a friend to come over and do her hair and makeup. The woman she asked, was Rosie Barrero, another patient in that RP clinical trial. Not so very long ago Rosie was legally blind. Now, here she was helping do her friend’s hair and makeup. And doing it beautifully too.

That’s when you know the treatment works. At least for Rosie.

There are many other stories to be heard – from patients and patient advocates, from researchers who develop therapies to the doctors who deliver them. – at our CIRM 2020 Grantee Meeting on next Monday September 14th Tuesday & September 15th.

It’s two full days of presentations and discussions on everything from heart disease and cancer, to COVID-19, Alzheimer’s, Parkinson’s and spina bifida. Here’s a link to the Eventbrite page where you can find out more about the event and also register to be part of it.

Like pretty much everything these days it’s a virtual event so you’ll be able to join in from the comfort of your kitchen, living room, even the backyard.

And it’s free!

You can join us for all two days or just one session on one day. The choice is yours. And feel free to tell your friends or anyone else you think might be interested.

We hope to see you there.

CIRM & CZI & MOU for COVID-19

Too many acronyms? Not to worry. It is all perfectly clear in the news release we just sent out about this.

A new collaboration between the California Institute for Regenerative Medicine (CIRM) and the Chan Zuckerberg Initiative (CZI) will advance scientific efforts to respond to the COVID-19 pandemic by collaborating on disseminating single-cell research that scientists can use to better understand the SARS-CoV-2 virus and help develop treatments and cures.

CIRM and CZI have signed a Memorandum of Understanding (MOU) that will combine CIRM’s infrastructure and data collection and analysis tools with CZI’s technology expertise. It will enable CIRM researchers studying COVID-19 to easily share their data with the broader research community via CZI’s cellxgene tool, which allows scientists to explore and visualize measurements of how the virus impacts cell function at a single-cell level. CZI recently launched a new version of cellxgene and is supporting the single-cell biology community by sharing COVID-19 data, compiled by the global Human Cell Atlas effort and other related efforts, in an interactive and scalable way.

“We are pleased to be able to enter into this partnership with CZI,” said Dr. Maria T. Millan, CIRM’s President & CEO. “This MOU will allow us to leverage our respective investments in genomics science in the fight against COVID-19. CIRM has a long-standing commitment to generation and sharing of sequencing and genomic data from a wide variety of projects. That’s why we created the CIRM genomics award and invested in the Stem Cell Hub at the University of California, Santa Cruz, which will process the large complex datasets in this collaboration.”  

“Quickly sharing scientific data about COVID-19 is vital for researchers to build on each other’s work and accelerate progress towards understanding and treating a complex disease,” said CZI Single-Cell Biology Program Officer Jonah Cool. “We’re excited to partner with CIRM to help more researchers efficiently share and analyze single-cell data through CZI’s cellxgene platform.”

In March 2020, the CIRM Board approved $5 million in emergency funding to target COVID-19. To date, CIRM has funded 17 projects, some of which are studying how the SARS-CoV-2 virus impacts cell function at the single-cell level.

Three of CIRM’s early-stage COVID-19 research projects will plan to participate in this collaborative partnership by sharing data and analysis on cellxgene.   

  • Dr. Evan Snyder and his team at Sanford Burnham Prebys Medical Discovery Institute are using induced pluripotent stem cells (iPSCs), a type of stem cell that can be created by reprogramming skin or blood cells, to create lung organoids. These lung organoids will then be infected with the novel coronavirus in order to test two drug candidates for treating the virus.
  • Dr. Brigitte Gomperts at UCLA is studying a lung organoid model made from human stem cells in order to identify drugs that can reduce the number of infected cells and prevent damage in the lungs of patients with COVID-19.
  • Dr. Justin Ichida at the University of Southern California is trying to determine if a drug called a kinase inhibitor can protect stem cells in the lungs and other organs, which the novel coronavirus selectively infects and kills.

“Cumulative data into how SARS-CoV-2 affects people is so powerful to fight the COVID-19 pandemic,” said Stephen Lin, PhD, the Senior CIRM Science Officer who helped develop the MOU. “We are grateful that the researchers are committed to sharing their genomic data with other researchers to help advance the field and improve our understanding of the virus.”

CZI also supports five distinct projects studying how COVID-19 progresses in patients at the level of individual cells and tissues. This work will generate some of the first single-cell biology datasets from donors infected by SARS-CoV-2 and provide critical insights into how the virus infects humans, which cell types are involved, and how the disease progresses. All data generated by these grants will quickly be made available to the scientific community via open access datasets and portals, including CZI’s cellxgene tool.

CIRM partners with UCLA scientists to take on COVID-19

Don’t you love it when someone does your job for you and does it so well you have no need to add anything to it! Doesn’t happen very often – sad to say – but this week our friends at UCLA wrote a great article describing the work they are doing to target COVID-19. Best of all, all the work described is funded by CIRM. So read, and enjoy.

Two scientists in a lab at the UCLA Broad Stem Cell Research Center

By Tiare Dunlap, UCLA

As the COVID-19 pandemic rages on, UCLA researchers are rising to the occasion by channeling their specialized expertise to seek new and creative ways to reduce the spread of the virus and save lives. Using years’ — or even decades’ — worth of knowledge they’ve acquired studying other diseases and biological processes, many of them have shifted their focus to the novel coronavirus, and they’re collaborating across disciplines as they work toward new diagnostic tests, treatments and vaccines.

At UCLA, more than 230 research projects, including several being led by members of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, are contributing to that mission.

Dr. Brititte Gomperts, Photo courtesy UCLA

“As a result of the pandemic, everyone on campus is committed to finding ways that their unique expertise can help out,” said Dr. Brigitte Gomperts, professor and vice chair of research in pediatric hematology-oncology and pulmonary medicine at the David Geffen School of Medicine at UCLA and a member of the UCLA Children’s Discovery and Innovation Institute. “So many of my colleagues have repurposed their labs to work on the virus. It’s very seldom that you have one thing that everybody’s working on, and it has been truly inspiring to see how everyone has come together to try and solve this.”

Here’s a look at five projects in which UCLA scientists are using stem cells — which can self-replicate and give rise to all cell types — to take on COVID-19.

Using lung organoids as models to test possible treatments 

Dr. Brigitte Gomperts

Gomperts has spent years perfecting methods for creating stem cell–derived three-dimensional lung organoids. Now, she’s using those organoids to study how SARS-CoV-2, the virus that causes COVID-19, affects lung tissue and to rapidly screen thousands of prospective treatments. Because the organoids are grown from human cells and reflect the cell types and architecture of the lungs, they can offer unprecedented insights into how the virus infects and damages the organ.  

Gomperts is collaborating with UCLA colleagues Vaithilingaraja Arumugaswami, a virologist, and Robert Damoiseaux, an expert in molecular screening. Their goal is to find an existing therapy that could be used to reduce the spread of infection and associated damage in the lungs.

“We’re starting with drugs that have already been tested in humans because our goal is to find a therapy that can treat patients with COVID-19 as soon as possible,” Gomperts said. Read more.

Repurposing a cancer therapy

Dr. Vaithi Arumugaswami: Photo courtesy UCLA

Vaithilingaraja Arumugaswami, associate professor of molecular and medical pharmacology at the Geffen School of Medicine

In addition to collaborating with Gomperts, Arumugaswami and Damoiseaux identified the cancer drug Berzosertib as a possible treatment for COVID-19 after screening 430 drug candidates. The drug, which is currently being tested in clinical trials for cancer, works by blocking a DNA repair process that is exploited by solid cancers and the SARS-CoV-2 virus, and the UCLA scientists found that it is very effective at limiting viral replication and cell death. 

“Clinical trials have shown that Berzosertib blocks the DNA repair pathway in cancer cells, but has no effects on normal, healthy cells,” Arumugaswami said.

Now, Arumugaswami and Gustavo Garcia Jr., a staff research associate, are testing Berzosertib and additional drug combinations on lung organoids developed in Gomperts’ lab and stem cell–derived heart cells infected with SARS-CoV-2. They suspect that if the drug is administered soon after diagnosis, it could limit the spread of infection and prevent complications. Read more.

Studying the immune response to the virus

Dr. Gay Crooks

Dr. Gay Crooks, professor of pathology and laboratory medicine and of pediatrics at the Geffen School of Medicine, and co-director of the Broad Stem Cell Research Center; and Dr. Christopher Seet,  

assistant professor of hematology-oncology at the Geffen School of Medicine

Crooks and Seet are using stem cells to model how immune cells recognize and fight the virus in a lab dish. To do that, they’re infecting blood-forming stem cells — which can give rise to all blood and immune cells — from healthy donors with parts of the SARS-CoV-2 virus and then coaxing the stem cells to produce immune cells called dendritic cells. Dendritic cells devour viral proteins, chop them up into pieces and then present those pieces to other immune cells called T cells to provoke a response.

By studying that process, Crooks and Seet hope to identify which parts of the virus provoke the strongest T-cell responses. Developing an effective vaccine for SARS-CoV-2 will require a deep understanding of how the immune system responds to the virus, and this work could be an important step in that direction, giving researchers and clinicians a way to gauge the effectiveness of possible vaccines.

“When we started developing this project some years ago, we had no idea it would be so useful for studying a viral infection — any viral infection,” Crooks said. “It was only because we already had these tools in place that we could spring into action so fast.” Read more.

Developing a booster that could help a vaccine last longer

Song Li, chair and professor of bioengineering at the UCLA Samueli School of Engineering

A COVID-19 vaccine will need to provide long-term protection from infection. But how long a vaccine protects from infection isn’t solely dependent on the vaccine.

The human body relies on long-living immune cells called T memory stem cells that guard against pathogens such as viruses and bacteria that the body has encountered before. Unfortunately, the body’s capacity to form T memory stem cells decreases with age. So no matter how well designed a vaccine is, older adults who don’t have enough of a response from T memory stem cells will not be protected long-term.

To address that issue, Li is developing an injectable biomaterial vaccine booster that will stimulate the formation of T memory stem cells. The booster is made up of engineered materials that release chemical messengers to stimulate the production of T memory stem cells. When combined with an eventual SARS-CoV-2 vaccine, they would prompt the body to produce immune cells primed to recognize and eliminate the virus over the long term.

“I consider it my responsibility as a scientist and an engineer to translate scientific findings into applications to help people and the community,” Li said. Read more.

Creating an off-the-shelf cell therapy

Lili Yang, associate professor of microbiology, immunology and molecular genetics in the UCLA College

Invariant natural killer T cells, or iNKT cells, are the special forces of the immune system. They’re extremely powerful and can immediately recognize and respond to many different intruders, from infections to cancer.

Yang is testing whether iNKT cells would make a particularly effective treatment for COVID-19 because they have the capacity to kill virally infected cells, offer protection from reinfection and rein in the excessive inflammation caused by a hyperactive immune response to the virus, which is thought to be a major cause of tissue damage and death in people with the disease.

One catch, though, is that iNKT cells are incredibly scarce: One drop of human blood contains around 10 million blood cells but only around 10 iNKT cells. That’s where Yang’s research comes in. Over the past several years, she has developed a method for generating large numbers of iNKT cells from blood-forming stem cells. While that work was aimed at creating a treatment for cancer, Yang’s lab has adapted its work over the past few months to test how effective stem cell–derived iNKT cells could be in fighting COVID-19. With her colleagues, she has been studying how the cells work in fighting the disease in models of SARS-CoV-2 infection that are grown from human kidney and lung cells.

“My lab has been developing an iNKT cell therapy for cancer for years,” Yang said. “This means a big part of the work is already done. We are repurposing a potential therapy that is very far along in development to treat COVID-19.” Read more.

“Our center is proud to join CIRM in supporting these researchers as they adapt projects that have spent years in development to meet the urgent need for therapies and vaccines for COVID-19,” said Dr. Owen Witte, founding director of the UCLA Broad Stem Cell Research Center. “This moment highlights the importance of funding scientific research so that we may have the foundational knowledge to meet new challenges as they arise.” Crooks, Gomperts, Seet and Yang are all members of the UCLA Jonsson Comprehensive Cancer Center. Damoiseaux is a professor of molecular and medical pharmacology and director of the Molecular Shared Resource Center at the California NanoSystems Institute at UCLA

Scientists develop immune evading pancreas organoids to treat type 1 diabetes

By Stephen Lin, PhD., CIRM Senior Science Officer

A diabetic child is checking her blood sugar level (self glycaemia).

Type 1 diabetes affects millions of people.  It is a disease where beta islet cells in the pancreas are targeted by the body’s own immune system, destroying the ability to produce insulin.  Without insulin, the body cannot break down sugars from the bloodstream that produce energy for organs and that can lead to many significant health problems including damage to the eyes, nerves, and kidneys.  It is a life-long condition, most commonly triggered in children and teenagers.  However, type 1 diabetes can manifest at any time.  I have a family member who developed type 1 diabetes well into adulthood and had to dramatically alter his lifestyle to live with it. 

Fortunately most people can now live with the disease.  There was a time, dating back to ancient civilizations when getting type 1 diabetes meant early death.  Thankfully, over the past hundred years, treatments have been developed to address the disease.  The first widespread treatment developed in the 1920s was injections of animal insulin isolated from pancreatic islets in cattle and pigs.  Over 50 years later the first genetically engineered human insulin was produced using E. coli bacteria, and variations of this are still used today. However, the disease is still very challenging to manage.  My family member constantly monitors his blood sugar and gives himself injections of insulin to regulate his blood sugar. 

A therapy that can self-regulate blood sugar levels for diabetes would greatly improve the lives of millions of people that deal with the disease.  Pancreatic islet cells transplanted into patients can act as a natural rheostat to continually control blood sugar levels.  Pancreas organ transplantation and islet cell transplantation are treatment options that will accomplish this.  Both options are limited in supply and patients must be kept on life-long immunosuppression so the body does not reject the transplant.  Pancreatic beta cells are also being developed from pluripotent stem cells (these are cells that have the ability to be turned into almost any other kind of cell in the body). 

Now in an advance using pluripotent stem cells, Dr. Ronald Evans and his team at the Salk Institute have created cell clusters called organoids that mimic several properties of the pancreas.  Previously, in work supported by CIRM, the team discovered that a genetic switch called ERR-gamma caused the cells to both produce insulin and be functional to respond to sugar levels in the bloodstream.  They incorporated these findings to create their functional islet clusters that they term “human islet-like islet organoids” (HILOs).  Knowing that the immune system is a major barrier for long term cell replacement therapy, Dr. Evans’ team engineered the HILOs, in work also funded by CIRM, to be resistant to immune cells by expressing the checkpoint protein PD-L1.   PD-L1 is a major target for immunotherapies whose discovery led to a Nobel Prize in 2018.  Expressing PD-L1 acts as an immune blocker.  

When the PD-L1 engineered HILOs were transplanted into diabetic mice with functioning immune systems, they were able to sustain blood glucose control for time periods up to 50 days.  The researchers also saw significantly less mobilization of immune cells after transplantation.  The hope is that these engineered HILOs can eventually be developed as a long term therapy for type 1 diabetes patients without the need for lifelong immunosuppression. 

In a press release, the Salk researchers acknowledge that more research needs to be done before this system can be advanced to clinical trials.  For example, the transplanted organoids need to be tested in mice for longer periods of time to confirm that their effects are long-lasting. More work needs to be done to ensure they would be safe to use in humans, as well. However, the proof of concept has now been established to move forward with these efforts.  Concludes Dr. Evan’s in the announcement, “We now have a product that could potentially be used in patients without requiring any kind of device.”

The full study was published in Nature.

Don’t believe everything you read

(PRNewsfoto/Deseret News)

The Deseret News is Utah’s oldest continuously published daily newspaper. It has a big readership too, with the largest Sunday circulation in the state and the second largest daily circulation. That’s why when they publish paid advertisements that look like serious news articles it can be misleading, even worse.

This week the Deseret News (that’s not a misspelling by the way, the name is taken from the word for honeybee in the Book of Mormon) ran an advertisement written by the East West Health Clinic. The advertisement  is about regenerative medicine and its ability to help repair damaged knee, hip and shoulder joints. It quotes from some well-regarded scientific sources such as WebMD and the National Health Interview Survey.

They also quote CIRM. Here’s what they say:

“In theory, there’s no limit to the types of diseases that could be treated with stem cell research,” the California Institute for Regenerative Medicine (CIRM) explains. CIRM posits that stem cell therapy could be used to “replace virtually any tissue or organ that is injured or diseased.”

That’s from a page on our website that talks about the potential of stem cell research. And it’s all true. But then the advertisement switches quickly, and rather subtly, to talking about what the clinic is doing. And that’s where things get murky.

East West Health offers therapies using umbilical and cord blood that they claim can treat a wide range of diseases and disorders from tendonitis to arthritis and suggest they might even help people with Alzheimer’s and dementia. But none of these have been proven in an FDA-sanctioned clinical trial or approved by the FDA. In fact, if you scroll down to the bottom of the website you find this statement.

*These statements have not been evaluated by the FDA*

And they also say that “Individual results may vary”.

I bet they do.

There are many clinics around the US that claim that stem cells have almost magical powers to heal. They don’t.

What stem cells do have is enormous potential. That’s why we invest in solid, scientifically rigorous research to try and harness that potential and bring it to patients in need. But that takes years of work, meticulous testing in the lab long before it ever is tried in people. It takes working with the FDA to get their support in starting a clinical trial to show that the therapy is both safe and effective.

CIRM has long promoted the importance of the Three R’s, making sure research is regulated, reliable and reputable. We want to help advance promising regenerative medicine therapies and products while protecting patients from the risks posed by unproven interventions.

That’s why we have a commitment to only funding the best science, work that has undergone rigorous peer review. That’s why we collaborate with expert advisors, ensure all projects we fund are in alignment with FDA rules and regulations and that meet the highest standards set by the organizations like the National Institutes of Health.

There are no short cuts. No easy ways to just stick cells in someone and tell them they are good to go.

That’s why when we see advertisements like the one that ran in The Deseret News it concerns us, because people will see our name and think we support the work being done by the people who wrote the piece. We don’t. Quite the opposite.

If you would like to learn more about the kinds of questions you need to ask before signing up for a clinical trial or therapy of any kind just go to our website. And if you want to see the list of clinical trials we do support, you can go here.

Stem Cell All-Stars, All For You

goldstein-larry

Dr. Larry Goldstein, UC San Diego

It’s not often you get a chance to hear some of the brightest minds around talk about their stem cell research and what it could mean for you, me and everyone else. That’s why we’re delighted to be bringing some of the sharpest tools in the stem cell shed together in one – virtual – place for our CIRM 2020 Grantee Meeting.

The event is Monday September 14th and Tuesday September 15th. It’s open to anyone who wants to attend and, of course, it’s all being held online so you can watch from the comfort of your own living room, or garden, or wherever you like. And, of course, it’s free.

BotaDaniela2261

Dr. Daniela Bota, UC Irvine

The list of speakers is a Who’s Who of researchers that CIRM has funded and who also happen to be among the leaders in the field. Not surprising as California is a global center for regenerative medicine. And you will of course be able to post questions for them to answer.

srivastava-deepak

Dr. Deepak Srivastava, Gladstone Institutes

The key speakers include:

Larry Goldstein: the founder and director of the UCSD Stem Cell Program talking about Alzheimer’s research

Irv Weissman: Stanford University talking about anti-cancer therapies

Daniela Bota: UC Irvine talking about COVID-19 research

Deepak Srivastava: Gladsone Institutes, talking about heart stem cells

Other topics include the latest stem cell approaches to COVID-19, spinal cord injury, blindness, Parkinson’s disease, immune disorders, spina bifida and other pediatric disorders.

You can choose one topic or come both days for all the sessions. To see the agenda for each day click here. Just one side note, this is still a work in progress so some of the sessions have not been finalized yet.

And when you are ready to register go to our Eventbrite page. It’s simple, it’s fast and it will guarantee you’ll be able to be part of this event.

We look forward to seeing you there.

Perseverance: from theory to therapy. Our story over the last year – and a half

Some of the stars of our Annual Report

It’s been a long time coming. Eighteen months to be precise. Which is a peculiarly long time for an Annual Report. The world is certainly a very different place today than when we started, and yet our core mission hasn’t changed at all, except to spring into action to make our own contribution to fighting the coronavirus.

This latest CIRM Annual Reportcovers 2019 through June 30, 2020. Why? Well, as you probably know we are running out of money and could be funding our last new awards by the end of this year. So, we wanted to produce as complete a picture of our achievements as we could – keeping in mind that we might not be around to produce a report next year.

Dr. Catriona Jamieson, UC San Diego physician and researcher

It’s a pretty jam-packed report. It covers everything from the 14 new clinical trials we have funded this year, including three specifically focused on COVID-19. It looks at the extraordinary researchers that we fund and the progress they have made, and the billions of additional dollars our funding has helped leverage for California. But at the heart of it, and at the heart of everything we do, are the patients. They’re the reason we are here. They are the reason we do what we do.

Byron Jenkins, former Naval fighter pilot who battled back from his own fight with multiple myeloma

There are stories of people like Byron Jenkins who almost died from multiple myeloma but is now back leading a full, active life with his family thanks to a CIRM-funded therapy with Poseida. There is Jordan Janz, a young man who once depended on taking 56 pills a day to keep his rare disease, cystinosis, under control but is now hoping a stem cell therapy developed by Dr. Stephanie Cherqui and her team at UC San Diego will make that something of the past.

Jordan Janz and Dr. Stephanie Cherqui

These individuals are remarkable on so many levels, not the least because they were willing to be among the first people ever to try these therapies. They are pioneers in every sense of the word.

Sneha Santosh, former CIRM Bridges student and now a researcher with Novo Nordisk

There is a lot of information in the report, charting the work we have done over the last 18 months. But it’s also a celebration of everyone who made it possible, and our way of saying thank you to the people of California who gave us this incredible honor and opportunity to do this work.

We hope you enjoy it.