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.

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 joins forces with US Department of Defense to fight COVID

Photo courtesy of Gabrielle Lurie / San Francisco Chronicle / Polaris

Small state agencies like CIRM don’t normally get to partner with a behemoth like the Department of Defense (DOD), but these are not normal times. Far from it. That’s why we are both joining forces with the National Institutes of Health to fund a clinical trial that hopes to help patients on a ventilator battling a sometime fatal condition that attacks their lungs.

The study is being run by Dr. Michael Matthay from U.C. San Francisco. CIRM awarded Dr. Matthay $750,000 to help expand an existing trial and to partner with U.C. Davis to bring in more patients, particularly from underserved communities.

This approach uses mesenchymal stem cells (MSCs) taken from bone marrow to help patients with a condition called acute respiratory distress syndrome (ARDS). This occurs when the virus attacks the lungs.

In an article in UCSF News, Dr. Matthay says the impact can be devastating.

“Tiny air spaces in the lungs fill up with fluid and prevent normal oxygen uptake in the lungs. That’s why the patient has respiratory failure. Usually these patients have to be intubated and treated with a mechanical ventilator.”

Many patients don’t survive. Dr. Matthay estimates that as many as 60 percent of COVID-19 patients who get ARDS die.

This is a Phase 2 double blind clinical trial which means that half the 120 patients who are enrolled will get MSCs (which come from young, health donors) and the other half will get a placebo. Neither the patients getting treated nor the doctors and nurses treating them will know who gets what.

Interestingly this trial did not get started as a response to COVID-19. In fact, it’s the result of years of work by Dr. Matthay and his team hoping to see if MSC’s could help people who have ARDs as a result of trauma, bacterial or other infection. They first started treating patients earlier this year when most people still considered the coronavirus a distant threat.

“We started the study in January 2020, and then COVID-19 hit, so we have been enrolling patients over the last eight months. Most of the patients we’ve enrolled in the trial have ended up having severe viral pneumonia from COVID.”

So far CIRM has funded 17 different projects targeting COVID-19. You can read about those in our Press Release section.

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

Driving Innovation While Addressing Health Disparities Among People of Color

Image courtesy of Science Photo Library

One of the wonders of regenerative medicine is its broad applicability, which provides us with the opportunity to build upon existing knowledge and concepts.  In the midst of a global pandemic, researchers have responded to the needs of patients severely afflicted with COVID-19 by repurposing existing therapies being developed to treat patients.  The California Institute for Regenerative Medicine (CIRM) responded immediately to the pandemic and to researchers wanting to help by providing $5 million in emergency funding for COVID-19 related projects.  In a short time span, this funding has driven innovation in the form of 17 new projects targeting COVID-19, many of which are based on previously developed concepts being repurposed to deal with the novel coronavirus.

One such example is a clinical trial funded by CIRM that uses natural killer (NK) cells, a type of white blood cell that is a vital part of the immune system, which are administered to patients with COVID-19. NK cells play an important role in defense against cancer and in fighting off viral infections.  In fact, this exact same therapy was previously used in a clinical trial for patients with Acute Myeloid Leukemia, a type of blood cancer.

Another clinical trial funded by CIRM uses mesenchymal stromal cells (MSCs), a type of stem cell, to treat acute respiratory distress syndrome (ARDS), a life-threatening lung injury that occurs when fluid leaks into the lungs.  As a result of ARDS, oxygen cannot get into the body and patients have difficulty breathing.  ARDS is one of the most serious and lethal consequences of COVID-19, which is why this trial was expanded after the coronavirus pandemic to include COVID-19 positive patients.   

Despite these great strides in driving innovation of therapies, one challenge that still needs to be tackled is providing patients access to these therapies, particularly people from underrepresented and underserved communities.  In California alone, there have been over 621,000 positive cases as of August 2020, with more cases every day.  However, the impact of the pandemic is disproportionately affecting the Latinx and African American communities more than others. An analysis by the Los Angeles Times found that the Latinx and African American communities have double the mortality rate from the coronavirus in Los Angeles County.  Additionally, a surge in cases is being seen in poorer communities in comparison to wealthier ones.

Until a vaccine can be successfully developed and implemented to obtain herd immunity, the number of cases will continue to climb.  There is also the challenge of the long term health effects of COVID-19, which can consist of neurological, breathing, and heart problems according to an article in Science.  Unfortunately, a study published in the New England Journal of Medicine found that despite disproportionately higher rates of COVID-19 infection, hospitalization and death among people of color, they are significantly underrepresented in COVID-19 clinical trials.

The challenge of underrepresentation in clinical trials and research needs to be addressed by creating a more diverse population of study participants, so as to better generalize results to the U.S. population as a whole.  CIRM Board Member Ysabel Duron, a leading figure in cancer education in the Latinx community, has advocated for more inclusion and outreach efforts directed towards underserved and underrepresented communities.  By communicating with patients in underserved and underrepresented communities, building relationships established on a foundation of trust, and connecting patients with potential trial matches, underrepresentation can be alleviated.

To help in addressing these disparities, CIRM has taken action by changing the requirements for its discovery stage research projects, which promote promising new technologies that could be translated to enable broad use and improve patient care, and clinical trial stage projects.

For clinical trials, all proposals must include a written plan in the application for outreach and study participation by underserved and disproportionately affected populations. Priority will be given to projects with the highest quality plans in this regard. For discovery projects, all proposals must provide a statement describing how their overall study plan and design has considered the influence of race, ethnicity, sex and gender diversity.  Additionally, all proposals should discuss the limitations, advantages, and/or challenges in developing a product or tools that addresses the unmet medical needs of California’s diverse population, including underserved communities.  There is still much more work that needs to be done to address health disparities, but steps such as these can help steer progress in the right direction.

Driving innovation while addressing health disparities among people of color is just one of many opportunities and challenges of regenerative medicine in a post pandemic world.  This blog post is part of Signal’s fifth annual blog carnival. Please click here to read what other bloggers think about this topic.

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.

Therapy developed with CIRM award used in new clinical trial for COVID-19

Dr. Joshua Rhein, Assistant Professor of Medicine in the University of Minnesota Medical School’s Division of Infectious Diseases and International Medicine
Image Credit: University of Minnesota

While doctors are still trying to better understand how to treat some of the most severe cases of COVID-19, researchers are looking at their current scientific “toolkit” to see if any potential therapies for other diseases could also help treat patients with COVID-19. One example of this is a treatment developed by Fate Therapeutics called FT516, which received support in its early stages from a Late Stage Preclinical grant awarded by CIRM.

FT516 uses induced pluripotent stem cells (iPSCs), which are a kind of stem cell made from reprogrammed skin or blood cells. These newly made stem cells have the potential to become any kind of cell in the body. For FT516, iPSCs are transformed into natural killer (NK) cells, which are a type of white blood cell that are a vital part of the immune system and play a role in fighting off viral infections.

Prior to the coronavirus pandemic, FT516 was used in a clinical trial to treat patients with acute myeloid leukemia (AML) and B-cell lymphoma, which are two different kinds of blood cancer.

Due to the natural ability of NK cells to fight off viruses, it is believed that FT516 may also help play a role in diminishing viral replication of the novel coronavirus in COVID-19 patients. In fact, Fate Therapeutics, in partnership with the University of Minnesota, has treated their first COVID-19 patient with FT516 in a new clinical trial.

In a news release, Dr. Joshua Rhein, Physician at the University of Minnesota running the trial site, elaborates on how FT516 could help COVID-19 patients.

“The medical research community has been mobilized to meet the unique challenges that COVID-19 presents. There are limited treatment options for COVID-19, and we have been inundated daily with reports of varying quality describing the potential of numerous therapies. We know that NK cells play an important role in responding to SARS-CoV-2, the virus responsible for COVID-19, and that these cells often become depleted in infected patients. Our intent is to replenish NK cells in order to restore a functional immune system and directly target the virus.”

In its own response to the coronavirus pandemic, CIRM has funded three clinical trials as part of $5 million in emergency funding for COVID-19 related projects. They include the following: a convalescent plasma study conducted by Dr. John Zaia at City of Hope, a treatment for acute respiratory distress syndrome (a serious and lethal consequence of COVID-19) conducted by Dr. Michael Matthay at UCSF, and a study that also uses NK cells to treat COVID-19 patients conducted by Dr. Xiaokui Zhang at Celularity Inc.  Visit our dashboard page to learn more about these clinical projects.

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.

CIRM Board Approves Two New Discovery Research Projects for COVID-19

Dr. Karen Christman (left) and Dr. Lili Yang (right)

This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two new discovery research project as part of the $5 million in emergency funding for COVID-19 related projects.  This brings the number of COVID-19 projects CIRM is supporting to 17, including three clinical trials.

$249,974 was awarded to Dr. Karen Christman at UC San Diego to develop a treatment for Acute Respiratory Distress Syndrome (ARDS), a life-threatening lung injury that occurs when fluid leaks into the lungs and is prevalent in COVID-19 patients.  Dr. Christman and her team will develop extracellular matrix (ECM) hydrogels, a kind of structure that provides support to surrounding cells.  The goal is to develop a treatment that can be delivered directly to site of injury, where the ECM would recruit stem cells, treat lung inflammation, and promote lung healing.

$250,000 was awarded to Dr. Lili Yang at UCLA to develop a treatment for COVID-19.  Dr. Yang and her team will use blood stem cells to create invariant natural killer T (iNKT) cells, a powerful kind of immune cell with the potential to clear virus infection and mitigate harmful inflammation.  The goal is to develop these iNKT cells as an off the shelf therapy to treat patients with COVID-19.

These awards are part of CIRM’s Quest Awards Program (DISC2), which promotes promising new technologies that could be translated to enable broad use and improve patient care.

“The harmful lung inflammation caused by COVID-19 can be dangerous and life threatening,” says Maria T. Millan, M.D., the President and CEO of CIRM. “Early stage discovery projects like the ones approved today are vital in developing treatments for patients severely affected by the novel coronavirus.”

Earlier in the week the Board also approved changes to both DISC2 and clinical trial stage projects (CLIN2). These were in recognition of the Agency’s remaining budget and operational timeline and the need to launch the awards as quickly as possible.

For DISC2 awards the changes include:

  • Award limit of $250,000
  • Maximum award duration of 12 months
  • Initiate projects within 30 days of approval
  • All proposals must provide a statement describing how their overall study plan and design has considered the influence of race, ethnicity, sex and gender diversity.
  • All proposals should discuss the limitations, advantages, and/or challenges in developing a product or tools that addresses the unmet medical needs of California’s diverse population, including underserved communities.

Under the CLIN2 awards, to help projects carry out a clinical trial, the changes include:

  • Adjust award limit to the following:
Applicant typePhase 1, Phase 1/2, Feasability Award CapPhase 2 Award CapPhase 3 Award Cap
Non-profit$9M$11.25M$7.5M
For-profit$6M$11.25M$7.5M
  • Adjust the award duration to not exceed 3 years with award completion no later than November 2023
  • Initiate projects within 30 days of approval
  • All proposals must include a written plan in the application for outreach and study participation by underserved and disproportionately affected populations. Priority will be given to projects with the highest quality plans in this regard.

The changes outlined above for CLIN2 awards do not apply to sickle cell disease projects expected to be funded under the CIRM/NHLBI Cure Sickle Cell Disease joint Initiative.

Using mini lungs to test potential COVID-19 therapies

Dr. Evan Snyder

If someone told you they were working on lungs in a dish you might be forgiven for thinking that’s the worst idea for a new recipe you have ever heard of. But in the case of Dr. Evan Snyder and his team at Sanford Burnham Prebys Medical Discovery Institute it could be a recipe for a powerful new tool against COVID-19. 

Earlier this month the CIRM Board approved almost $250,000 for Dr. Snyder and his team to use human induced pluripotent stem cells (hiPSCs), a type of stem cell that can be created by reprogramming skin or blood cells, to create any other cell in the body, including lung cells.

These cells will then be engineered to become 3D lung organoids or “mini lungs in a dish”. The importance of this is that these cells resemble human lungs in a way animal models do not. They have the same kinds of cells, structures and even blood vessels that lungs do.

These cells will then be infected with the coronavirus and then be used to test two drugs to see if those drugs are effective against the virus.

In a news release Dr. Snyder says these cells have some big advantages over animal models, the normal method for early stage testing of new therapies.

“Mini lungs will also help us answer why some people with COVID-19 fare worse than others. Because they are made from hiPSCs, which come from patients and retain most of the characteristics of those patients, we can make ‘patient-specific’ mini lungs. We can compare the drug responses of mini lungs created from Caucasian, African American, and Latino men and women, as well as patients with a reduced capacity to fight infection to make sure that therapies work effectively in all patients. If not, we can adjust the dose or drug regime to help make the treatment more effective.

“We can also use the mini lungs experimentally to evaluate the effects of environmental toxins that come from cigarette smoking or vaping to make sure the drugs are still effective; and emulate the microenvironmental conditions in the lungs of patients with co-morbidities such as diabetes, and heart or kidney disease.”

To date CIRM has funded 15 projects targeting COVID-19, including three that are in clinical trials.