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:
Phase 1, Phase 1/2, Feasability Award Cap
Phase 2 Award Cap
Phase 3 Award Cap
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.
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.
This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two additional discovery research projects 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 15, including three clinical trials.
The Board awarded $249,999 to Dr. Evan Snyder at the Sanford Burnham Prebys Medical Discovery Institute. The study will use 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 treatment of the virus. The iPSCs and the subsequent lung organoids created will reflect diversity by including male and female patients from the Caucasian, African-American, and Latinx population.
This award is 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 Board also awarded $150,000 to Dr. Steven Dowdy at UC San Diego for development of another potential treatment for COVID-19.
Dr. Dowdy and his team are working on developing a new, and hopefully more effective, way of delivering a genetic medicine, called siRNA, into the lungs of infected patients. In the past trying to do this proved problematic as the siRNA did not reach the appropriate compartment in the cell to become effective. However, the team will use an iPSC lung model to help them identify ways past this barrier so the siRNA can attack the virus and stop it replicating and spreading throughout the lungs.
This award is part of CIRM’s Inception Awards Program (DISC1), which supports transformational ideas that require the generation of additional data.
A supplemental award of $250,000 was approved for Dr. John Zaia at City of Hope to continue support of a CIRM funded clinical study that is using convalescent plasma to treat COVID-19 patients. The team recently launched a website to enroll patients, recruit plasma donors, and help physicians enroll their patients.
“The use of induced pluripotent stem cells has expanded the potential for personalized medicine,” says Dr. Maria T. Millan, the President & CEO of CIRM. “Using patient derived cells has enabled researchers to develop lung organoids and lung specific cells to test numerous COVID-19 therapies.”
Sometimes it’s the smallest things that make the biggest difference. In the case of a clinical trial that CIRM is funding, all it takes to be part of it is four teaspoons of blood.
The clinical trial is being run by Dr. John Zaia and his team at the City of Hope in Duarte, near Los Angeles, in partnership with tgen and the CIRM Alpha Stem Cell Clinic Network. They are going to use blood plasma from people who have recovered from COVID-19 to treat people newly infected with the virus. The hope is that antibodies in the plasma, which can help fight infections, will reduce the severity or length of infection in others.
People who have had the virus and are interested in taking part are asked to give four teaspoons of blood, to see if they have enough antibodies. If they do they can then either donate plasma – to help newly infected people – or blood to help with research into COVID-19.
As a sign of how quickly Dr. Zaia and his team are working, while we only approved the award in late April, they already have their website up and running, promoting the trial and trying to recruit both recovered COVID-19 survivors and current patients.
The site does a great job of explaining what they are trying to do and why people should take part. Here’s one section from the site.
Why should I participate in your study?
By participating in our study, you will learn whether you have developed antibodies against SARS-CoV-2, the virus responsible for COVID-19. To do so, you just need to donate a small sample of blood (approximately 4 teaspoons).
If testing show you have enough antibodies, you will have the option of donating plasma that will be used to treat severely ill COVID-19 patients and may help save lives.
If you don’t want to donate plasma, you can still donate blood (approximately 3.5 tablespoons), which will be studied and help researchers learn more about COVID-19.
By donating blood or plasma, you will help us gain information that may be of significant value for patient management in future epidemic seasons.
You don’t even have to live close to one of the clinical trial sites because the team can send you a blood collection kit and information about a blood lab near you so you can donate there. They may even send a nurse to collect your blood.
The team is also trying to ensure they reach communities that are often overlooked in clinical trials. That’s why the website is also in Spanish and Vietnamese.
Finally, the site is also being used to help recruit treating physicians who can collect the blood samples and help infuse newly infected patients.
We often read about clinical trials in newspapers and online. Now you get a chance to not only see one working in real time, you can get to be part of it.
There is still a lot that we don’t understand about SARS-CoV-2 (COVID-19), the new coronavirus that has caused a worldwide pandemic. Some patients that contract the virus experiences heart problems, but the reasons are not entirely clear. Pre-existing heart conditions or inflammation and oxygen deprivation that result from COVID-19 have all been implicated but more evidence needs to be collected.
To evaluate this, a joint study between Cedars-Sinai Board of Governors Regenerative Medicine Institute and the UCLA Broad Stem Cell Research Center used human induced pluripotent stem cells (iPSCs), a kind of stem cell that can become any kind of cell in the body and is usually made from skin cells. The iPSCS were converted into heart cells and infected with COVID-19 in order to study the effects of the virus.
The results of this study showed that the iPSC-derived heart cells are susceptible to COVID-19 infection and that the virus can quickly divide inside the heart cells. Furthermore, the infected heart cells showed changes in their ability to beat 72 hours after infection.
In a press release, Dr. Clive Svendsen, senior and co-corresponding author of the study and director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, elaborated on the results.
“This viral pandemic is predominately defined by respiratory symptoms, but there are also cardiac complications, including arrhythmias, heart failure and viral myocarditis. While this could be the result of massive inflammation in response to the virus, our data suggest that the heart could also be directly affected by the virus in COVID-19.”
Although this study does not perfectly replicate the conditions inside the human body, the iPSC heart cells may also help identify and screen new potential drugs that could alleviate viral infection of the heart.
The research team has already found that treatment with an antibody called ACE2 was able to decrease viral replication on the iPSC heart cells.
In the same press release Dr. Arun Sharma, first author and another co-corresponding author of the study and a research fellow at the Cedars-Sinai Board of Governors Regenerative Medicine Institute, had this to say about the ACE2 antibody.
“By blocking the ACE2 protein with an antibody, the virus is not as easily able to bind to the ACE2 protein, and thus cannot easily enter the cell. This not only helps us understand the mechanisms of how this virus functions, but also suggests therapeutic approaches that could be used as a potential treatment for SARS-CoV-2 infection.”
The study’s third co-corresponding author was Dr. Vaithilingaraja Arumugaswami, an associate professor of molecular and medical pharmacology at the David Geffen School of Medicine at UCLA and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.
The full results of this study were published in Cell Reports Medicine.
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $750,000 to Dr. Xiaokui Zhang at Celularity to conduct a clinical trial for the treatment of COVID-19. This brings the total number of CIRM clinical trials to 64, including three targeting the coronavirus.
This trial will use blood stem cells obtained from the placenta to generate natural killer (NK) cells, a type of white blood cell that is a vital part of the immune system, and administer them to patients with COVID-19. NK cells play an important role in defense against cancer and in fighting off viral infections. The goal is to administer these cells to locate the active sites of COVID-19 infection and destroy the virus-infected cells. These NK cells have been used in two other clinical trials for acute myeloid leukemia and multiple myeloma.
The Board also approved two additional awards for Discovery Stage Research (DISC2), which promote promising new technologies that could be translated to enable broad use and improve patient care.
One award for $100,000 was given to Dr. Albert Wong at Stanford. Dr. Wong has recently received an award from CIRM to develop a vaccine that produces a CD8+ T cell response to boost the body’s immune response to remove COVID-19 infected cells. The current award will enable him to expand on the initial approach to increase its potential to impact the Latinx and African American populations, two ethnicities that are disproportionately impacted by the virus in California.
The other award was for $249,996 and was given to Dr. Preet Chaudhary at the University of Southern California. Dr. Chaudary will use induced pluripotent stem cells (iPSCs) to generate natural killer cells (NK). These NK cells will express a chimeric antigen receptor (CAR), a synthetic receptor that will directly target the immune cells to kill cells infected with the virus. The ultimate goal is for these iPSC-NK-CAR cells to be used as a treatment for COVID-19.
“These programs address the role of the body’s immune T and NK cells in combatting viral infection and CIRM is fortunate enough to be able to assist these investigators in applying experience and knowledge gained elsewhere to find targeted treatments for COVID-19” says Dr. Maria T. Millan, the President & CEO of CIRM. “This type of critical thinking reflects the resourcefulness of researchers when evaluating their scientific tool kits. Projects like these align with CIRM’s track record of supporting research at different stages and for different diseases than the original target.”
The CIRM Board voted to endorse a new initiative to refund the agency and provide it with $5.5 billion to continue its work. The ‘California Stem Cell Research, Treatments and Cures Initiative of 2020 will appear on the November ballot.
The Board also approved a resolution honoring Ken Burtis, PhD., for his long service on the Board. Dr. Burtis was honored for his almost four decades of service at UC Davis as a student, professor and administrator and for his 11 years on the CIRM Board as both a member and alternate member. In the resolution marking his retirement the Board praised him, saying “his experience, commitment, knowledge, and leadership, contributed greatly to the momentum of discovery and the future therapies which will be the ultimate outcome of the dedicated work of the researchers receiving CIRM funding.”
Jonathan Thomas, the Chair of the Board, said “Ken has been invaluable and I’ve always found him to have tremendous insight. He has served as a great source of advice and inspiration to me and to the ICOC in dealing with all the topics we have had to face.”
Lauren Miller Rogen thanked Dr. Burtis, saying “I sat next to you at my first meeting and was feeling so extraordinarily overwhelmed and you went out of your way to explain all these big science words to me. You were always a source of help and support, and you explained things to me in a way that I always appreciated with my normal brain.”
Dr. Burtis said it has been a real honor and privilege to be on the Board. “I’ve been amazed and astounded at the passion and dedication that the Board and CIRM staff have brought to this work. Every meeting over the years there has been a moment of drama and then resolution and this Board always manages to reach agreement and serve the people of California.”
In late March the CIRM Board approved $5 million in emergency funding for COVID-19 research. The idea was to support great ideas from California’s researchers, some of which had already been tested for different conditions, and see if they could help in finding treatments or a vaccine for the coronavirus.
Less than a month later we were funding a clinical trial and two other projects, one that targeted a special kind of immune system cell that has the potential to fight the virus.
Researchers use stem cells to model the immune response to COVID-19
By Tiare Dunlap
Cities across the United States are opening back up, but we’re still a long way from making the COVID-19 pandemic history. To truly accomplish that, we need to have a vaccine that can stop the spread of infection.
But to develop an effective vaccine, we need to understand how the immune system responds to SARS-CoV-2, the virus that causes COVID-19.
Vaccines work by imitating infection. They expose a person’s immune system to a weakened version or component of the virus they are intended to protect against. This essentially prepares the immune system to fight the virus ahead of time, so that if a person is exposed to the real virus, their immune system can quickly recognize the enemy and fight the infection. Vaccines need to contain the right parts of the virus to provoke a strong immune response and create long-term protection.
Most of the vaccines in development for SARS CoV-2 are using part of the virus to provoke the immune system to produce proteins called antibodies that neutralize the virus. Another way a vaccine could create protection against the virus is by activating the T cells of the immune system.
T cells specifically “recognize” virus-infected cells, and these kinds of responses may be especially important for providing long-term protection against the virus. One challenge for researchers is that they have only had a few months to study how the immune system protects against SARS CoV-2, and in particular, which parts of the virus provoke the best T-cell responses.
For years, they have been perfecting an innovative technology that uses blood-forming stem cells — which can give rise to all types of blood and immune cells — to produce a rare and powerful subset of immune cells called type 1 dendritic cells. Type 1 dendritic cells play an essential role in the immune response by devouring foreign proteins, termed antigens, from virus-infected cells and then chopping them into fragments. Dendritic cells then use these protein fragments to trigger T cells to mount an immune response.
Using this technology, Crooks and Seet are working to pinpoint which specific parts of the SARS-CoV-2 virus provoke the strongest T-cell responses.
Building long-lasting immunity
“We know from a lot of research into other viral infections and also in cancer immunotherapy, that T-cell responses are really important for long-lasting immunity,” said Seet, an assistant professor of hematology-oncology at the David Geffen School of Medicine at UCLA. “And so this approach will allow us to better characterize the T-cell response to SARS-CoV-2 and focus vaccine and therapeutic development on those parts of the virus that induce strong T-cell immunity.”
Crooks’ and Seet’s project uses blood-forming stem cells taken from healthy donors and infected with a virus containing antigens from SARS-CoV-2. They then direct these stem cells to produce large numbers of type 1 dendritic cells using a new method developed by Seet and Suwen Li, a graduate student in Crooks’ lab. Both Seet and Li are graduates of the UCLA Broad Stem Cell Research Center’s training program.
“The dendritic cells we are able to make using this process are really good at chopping up viral antigens and eliciting strong immune responses from T cells,” said Crooks, a professor of pathology and laboratory medicine and of pediatrics at the medical school and co-director of the UCLA Broad Stem Cell Research Center.
When type 1 dendritic cells chop up viral antigens into fragments, they present these fragments on their cell surfaces to T cells. Our bodies produce millions and millions of T cells each day, each with its own unique antigen receptor, however only a few will have a receptor capable of recognizing a specific antigen from a virus.
When a T cell with the right receptor recognizes a viral antigen on a dendritic cell as foreign and dangerous, it sets off a chain of events that activates multiple parts of the immune system to attack cells infected with the virus. This includes clonal expansion, the process by which each responding T cell produces a large number of identical cells, called clones, which are all capable of recognizing the antigen.
“Most of those T cells will go off and fight the infection by killing cells infected with the virus,” said Seet, who, like Crooks, is also a member of the UCLA Jonsson Comprehensive Cancer Center. “However, a small subset of those cells become memory T cells — long-lived T cells that remain in the body for years and protect from future infection by rapidly generating a robust T-cell response if the virus returns. It’s immune memory.”
Producing extremely rare immune cells
This process has historically been particularly challenging to model in the lab, because type 1 dendritic cells are extremely rare — they make up less than 0.1% of cells found in the blood. Now, with this new stem cell technology, Crooks and Seet can produce large numbers of these dendritic cells from blood stem cells donated by healthy people, introduce them to parts of the virus, then see how T cells taken from the blood can respond in the lab. This process can be repeated over and over using cells taken from a wide range of healthy people.
“The benefit is we can do this very quickly without the need for an actual vaccine trial, so we can very rapidly figure out in the lab which parts of the virus induce the best T-cell responses across many individuals,” Seet said.
The resulting data could be used to inform the development of new vaccines for COVID-19 that improve T-cell responses. And the data about which viral antigens are most important to the T cells could also be used to monitor the effectiveness of existing vaccine candidates, and an individual’s immune status to the virus.
“There are dozens of vaccine candidates in development right now, with three or four of them already in clinical trials,” Seet said. “We all hope one or more will be effective at producing immediate and long-lasting immunity. But as there is so much we don’t know about this new virus, we’re still going to need to really dig in to understand how our immune systems can best protect us from infection.”
Supporting basic research into our body’s own processes that can inform new strategies to fight disease is central to the mission of the Broad Stem Cell Research Center.
“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. “And it was only because we already had these tools in place that we could spring into action so fast.”
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two additional projects as part of the $5 million in emergency funding for COVID-19 related projects. This brings the number of projects CIRM is supporting to 11, including two clinical trials.
The Board awarded $349,999 to Dr. Vaithilingaraja Arumugaswami at UCLA. The focus of this project will be to study Berzosertib, a therapy targeting viral replication and damage in lung stem cells. The ultimate goal would be to use this agent as a therapy to prevent COVID-19 viral replication in the lungs, thereby reducing lung injury, inflammation, and subsequent lung disease caused by the virus.
This award is part of CIRM’s Translational Stage Research Program (TRAN1), which promotes the activities necessary for advancement to clinical study of a potential therapy.
The Board also awarded $149,916 to Dr. Song Li at UCLA. This project will focus on developing an injectable biomaterial that can induce the formation of T memory stem cells (TMSCs), an important type of stem cell that plays a critical role in generating an immune response to combat viruses. In vaccine development, there is a major challenge that the elderly may not be able to mount a strong enough immunity. This innovative approach seeks to address this challenge by increasing TMSCs in order to boost the immune response to vaccines against COVID-19.
This award is under CIRM’s Discovery Stage Research Program (DISC2), which promotes promising new technologies that could be translated to enable broad use and improve patient care.
“CIRM continues to support novel COVID-19 projects that build on previous knowledge acquired,” says Dr. Maria T. Millan, the President & CEO of CIRM. “These two projects represent the much-needed multi-pronged approach to the COVID-19 crisis, one addressing the need for effective vaccines to prevent disease and the other to treat the severe illness resulting from infection.”
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) expanded efforts related to the $5 million in emergency funding for the CIRM COVID-19 program.
The new guidelines mean that inception discovery projects (DISC1), whose goal is developing new and transformational ideas, will now be eligible for CIRM COVID-19 funding. These projects can receive up to $150,000 and must have data to confirm or reject their hypothesis within 6 months. In addition to this, quest discovery projects (DISC2), which promote the discovery of new technologies that could be translated to enable broad use, can now receive up to $250,000 in funding.
The Board approved using $1 million from the program in supplemental support for CIRM-funded COVID-19 clinical trials. Under the change an existing clinical trial can receive up to $250,000 in additional funding but must demonstrate sufficient progress and specific activities in order to be eligible. The Board will also require that all clinical trial projects include a plan for outreach and study participation by underserved and disproportionately affected populations.
The Board also strongly encouraged those that meet the stem cell component for vaccine development for COVID-19 to apply for funding.
“We continue to receive large amounts of inquiries and applications to the COVID-19 program announcement,” said Maria T. Millan, M.D., President and CEO of CIRM. “The amendments passed by our Board today will provide additional opportunities for CIRM to support novel vaccine development, fundamental discoveries and the acceleration of clinical programs.”
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved new clinical trials for COVID-19 and sickle cell disease (SCD) and two earlier stage projects to develop therapies for COVID-19.
Dr. Michael Mathay, of the University of California at San Francisco, was awarded $750,000 for a clinical trial testing the use of Mesenchymal Stromal Cells for respiratory failure from Acute Respiratory Distress Syndrome (ARDS). In ARDS, patients’ lungs fill up with fluid and are unable to supply their body with adequate amounts of oxygen. It is a life-threatening condition and a major cause of acute respiratory failure. This will be a double-blind, randomized, placebo-controlled trial with an emphasis on treating patients from under-served communities.
This award will allow Dr. Matthay to expand his current Phase 2 trial to additional underserved communities through the UC Davis site.
“Dr. Matthay indicated in his public comments that 12 patients with COVID-related ARDS have already been enrolled in San Francisco and this funding will allow him to enroll more patients suffering from COVID- associated severe lung injury,” says Dr. Maria T. Millan, CIRM’s President & CEO. “CIRM, in addition to the NIH and the Department of Defense, has supported Dr. Matthay’s work in ARDS and this additional funding will allow him to enroll more COVID-19 patients into this Phase 2 blinded randomized controlled trial and expand the trial to 120 patients.”
The Board also approved two early stage research projects targeting COVID-19.
Dr. Stuart Lipton at Scripps Research Institute was awarded $150,000 to develop a drug that is both anti-viral and protects the brain against coronavirus-related damage.
Justin Ichida at the University of Southern California was also awarded $150,00 to determine if a drug called a kinase inhibitor can protect stem cells in the lungs, which are selectively infected and killed by the novel coronavirus.
“COVID-19 attacks so many parts of the body, including the lungs and the brain, that it is important for us to develop approaches that help protect and repair these vital organs,” says Dr. Millan. “These teams are extremely experienced and highly renowned, and we are hopeful the work they do will provide answers that will help patients battling the virus.”
The Board also awarded Dr. Pierre Caudrelier from ExcellThera $2 million to conduct a clinical trial to treat sickle cell disease patients
SCD is an inherited blood disorder caused by a single gene mutation that results in the production of “sickle” shaped red blood cells. It affects an estimated 100,000 people, mostly African American, in the US and can lead to multiple organ damage as well as reduced quality of life and life expectancy. Although blood stem cell transplantation can cure SCD fewer than 20% of patients have access to this option due to issues with donor matching and availability.
Dr. Caudrelier is using umbilical cord stem cells from healthy donors, which could help solve the issue of matching and availability. In order to generate enough blood stem cells for transplantation, Dr. Caudrelier will be using a small molecule to expand these blood stem cells. These cells would then be transplanted into twelve children and young adults with SCD and the treatment would be monitored for safety and to see if it is helping the patients.
“CIRM is committed to finding a cure for sickle cell disease, the most common inherited blood disorder in the U.S. that results in unpredictable pain crisis, end organ damage, shortened life expectancy and financial hardship for our often-underserved black community” says Dr. Millan. “That’s why we have committed tens of millions of dollars to fund scientifically sound, innovative approaches to treat sickle cell disease. We are pleased to be able to support this cell therapy program in addition to the gene therapy approaches we are supporting in partnership with the National Heart, Lung and Blood Institute of the NIH.”