UC Davis

Join the movement to fight rare diseases

/ Katie Sharify / 2 Comments

Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments.

Here at the California Institute for Regenerative Medicine (CIRM), we understand the importance of funding research that impacts not just the most common diseases. In fact, 50% of all the projects we fund target a rare disease or condition such as: Retinitis pigmentosa, Sickle cell disease, Huntington’s disease, and Duchenne Muscular Dystrophy.

Over the years, CIRM has invested millions of dollars in helping children born with severe combined immunodeficiency (SCID), including $12 million to test a newly designed therapy in a clinical trial at UC San Francisco.

Children born with SCID have no functioning immune system so even a simple infection can prove life-threatening or fatal. We recently shared an update from one of the young patients in the trial.

Additionally, last December, the CIRM governing Board awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.

Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of certain fats in the brain and nerve cells and causes progressive dysfunction.  

There are several forms of Tay-Sachs disease, including an infant, juvenile, and adult forms. Over a hundred mutations in the disease-causing Hex A gene have been identified that result in enzyme disfunction. There are currently no effective therapies or cures for Tay-Sachs. 

The irony of rare diseases is that a lot of people have them. The total number of Americans living with a rare disease is estimated at between 25-30 million. Two-thirds of these patients are children.

Right now, individual disease programs tend to try individual approaches to developing a treatment, which is time consuming and expensive. That’s why this past summer, CIRM signed a Memorandum of Understanding (MOU) with the Foundation for the National Institutes of Health (FNIH) to join the Bespoke Gene Therapy Consortium (BGTC).

BGTC is a public-private partnership, managed by FNIH, that brings together the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private sector organizations to streamline the development and delivery of gene therapies for rare diseases.

“At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as severe combined immunodeficiency,” says Dr. Maria T. Millan, the President and CEO of CIRM. “But even an agency with our resources can only do so much. This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases.”

CIRM is proud to fund and spread awareness of rare diseases and invites you to watch this video about how they affect families around the world.

Investing in CAR T-cell therapy to treat cancer

/ Katie Sharify / 1 Comment
Photo credit: UC Regents 

The California Institute for Regenerative Medicine (CIRM) is investing $4 million to support Dr. William Murphy and UC Davis researchers to develop and test a chimeric antigen receptor (CAR) T-cell therapy to treat various B-cell malignancies, ranging from lymphomas to leukemias. 

In this Q&A—courtesy of UC Davis Health—Dr. Murphy discusses the importance of T-cell therapy and its implications for developing cancer treatments. His work is a collaboration between CIRM, the nonprofit organization Caring Cross, and UC Davis Health. 

What are B-cell malignancies? 

B-cells are a type of white blood cells that make antibodies. They are key to the body’s immune system. When healthy B-cells change into fast-growing cancer cells that don’t die, they cause B-cell malignancies. 

This can affect people at different ages. They may show up in children as B-cell acute lymphoblastic leukemia (B-ALL), an aggressive blood and bone marrow cancer. In adults, they make up about 85% of non-Hodgkin lymphoma (NHL), a cancer that starts in B lymphocytes. In the elderly, B-cell malignancies may come as multiple myeloma, a cancer of the plasma cells. 

There are different lines of treatments for B-cell lymphoma and leukemia, including immunotherapy using chimeric antigen receptor (CAR) T cells. These cells have revolutionized cancer treatment since they have been shown to work, and cure, when nothing else can. 

What is chimeric antigen receptor (CAR) T-cell therapy? 

Chimeric antigen receptor (CAR) T-cell therapy uses the body’s own defenses to fight disease. It is a new and exciting form of immunotherapy that works by modifying the receptors of immune cells (T cells) involving antibodies to target specific cancers, such as leukemias and lymphomas. 

CAR T cells are being used to treat some blood cancers with long-term success. The U.S. Food and Drug Administration (FDA) first approved CAR T-cell therapy in 2017. Their use is growing rapidly and being applied to other tumor types. Yet, this therapy is extremely expensive, even with insurance. It’s also a very intensive procedure and it takes time to generate the CAR T cells from the patient. 

While it could be considered a game changer, one of the issues with this therapy is the case relapse rate. The big holy grail in cancer therapy is how to prevent tumors from evading or escaping the immune attack. Around 60% of patients who get CAR therapy see their cancer return. If we can get the relapse rate down to negligible, that would be a tremendous advance. 

How do you intend to use CAR products to reduce cancer relapse? 

In CAR therapy, we take the immune T cells from a patient and use gene therapy to give a new receptor to signal and direct the T cell. The receptor usually has an antibody that recognizes a particular tumor antigen. Current FDA-approved CAR T therapies only target one tumor antigen. 

CARs have had tremendous success. However, there is significant patient relapse because the tumor adapts and may lose that one antigen that we are targeting, allowing it to escape the treatment. Our strategy is to target multiple antigens to reduce the potential for relapse since the tumor cannot adapt that quickly. 

We are also proposing a novel vector that will carry a CAR product, known as DuoCAR, that targets three antigens at the same time. As long as the tumor has one of the three antigens, then there’s little chance for the tumor to escape all three antibodies. This is similar to when you think about HIV treatment with the triple-drug therapy, where one alone is not sufficient. 

The hope is that the 60 to 70% of the population who would have relapsed if they had the original CAR T cell treatment, would have a home run with our kind of treatment or product. 

So, is this treatment for cancer patients who have relapsed? 

We see this product as a new frontline therapy and not just for patients who relapse. What the patient has to go through in order for CAR T therapy to work is very strenuous. So, yes, if there are relapsed patients, they can be given DuoCAR, but we’re also hoping this will become the new standard of care, replacing the other CARs in the future for everyone. 

To read the full Q&A, click here. 

Finding a treatment for Tay-Sachs disease

/ Esteban Cortez / 1 Comment

The California Institute for Regenerative Medicine (CIRM) has awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.  

Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of certain fats in the brain and nerve cells and causes progressive dysfunction.  

There are several forms of Tay-Sachs disease, including an infant, juvenile, and adult forms. Over a hundred mutations in the disease-causing Hex A gene have been identified that result in enzyme disfunction. There are currently no effective therapies or cures for Tay-Sachs. 

Illustration by Kateryna Kon

The UC Davis team will genetically modify the patient’s own blood stem cells to restore the Hex A enzyme that is missing in the disease.  

The goal is to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.  

“The successful development of this therapy will not only help patients with Tay-Sachs but will demonstrate the use case of this therapeutic approach for other monogenic neurodegenerative diseases,” the UC Davis team said. 

This work is a continuation of a CIRM grant that the team received. 

Tratando malformaciones congénitas antes del nacimiento 

/ Esteban Cortez / Leave a comment
El bebé, Tobi recibió un tratamiento de células madre, financiado por el CIRM, mientras aún estaba en el útero. To read this blog in English, click here.

Michelle y Jeff se llenaron de felicidad cuando se enteraron de que iban a tener un bebé.  

Luego, un examen de ultrasonido a las 20 semanas del embarazo reveló que el feto tenía espina bífida, una malformación congénita que ocurre cuando la columna vertebral y la médula espinal no se forman de manera adecuada. La espina bífida puede causar parálisis y otras complicaciones serias.   

Se derivó a la pareja a un ensayo clínico en la Universidad de California, Davis, que lleva a cabo la Dra. Diana Farmer, cirujana fetal y neonatal reconocida a nivel internacional, y su colega, el Dr. Aijun Wang.  

En este ensayo clínico, que se basó en una previa investigación financiada por el CIRM, se repara el defecto espinal aplicando células madre de una placenta donada, las cuales se insertan en una estructura sintética y se aplican al defecto de la médula espinal mientras el bebé se encuentra todavía en el útero.   

El hijo de Michelle y Jeff, Tobi, fue el segundo paciente que recibió este tratamiento. Michelle dijo que la cirugía fue difícil, pero el nacimiento de su bebé valió la pena.  

“Cuando lo abrazamos por primera vez dijimos, ‘No puedo creer que hayamos hecho esto. Lo logramos. Lo hicimos sin saber si funcionaría’.”   

A los tres meses, el progreso de Tobi parece promisorio. Jeff y Michelle saben que pueden surgir problemas más adelante, pero por ahora se sienten agradecidos de haber formado parte de este ensayo.

To read this blog in English, click here.

Making transplants easier for kids, and charting a new approach to fighting solid tumors.

/ Kevin McCormack / Leave a comment

Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but they also increase the risk of infection, gastrointestinal disease, pancreatitis and cancer.

Dr. Alice Bertaina and her team at Stanford University were awarded $11,998,188 to test an approach that uses combined blood stem cell (HSC) and kidney transplantation with the goal to improve outcomes with kidney transplantation in children. This approach seeks to improve on the blood stem cell preparation through an immune-based purification process.

In this approach, the donor HSC are transplanted into the patient in order to prepare for the acceptance of the donor kidney once transplanted. Donor HSC give rise to cells and conditions that re-train the immune system to accept the kidney. This creates a “tolerance” to the transplanted kidney providing the opportunity to avoid long-term need for medications that suppress the immune system.

Pre-clinical data support the idea that this approach could enable the patient to stop taking any immunosuppression medications within 90 days of the surgery.

Dr. Maria T. Millan, President and CEO of CIRM, a former pediatric transplant surgeon and tolerance researcher states that “developing a way to ensure long-term success of organ transplantation by averting immune rejection while avoiding the side-effects of life-long immunosuppression medications would greatly benefit these children.”

The CIRM Board also awarded $7,141,843 to Dr. Ivan King and Tachyon Therapeutics, Inc to test a drug showing promise in blocking the proliferation of cancer stem cells in solid tumors such as colorectal and gastrointestinal cancer.

Patients with late-stage colorectal cancer are typically given chemotherapy to help stop or slow down the progression of the disease. However, even with this intervention survival rates are low, usually not more than two years.

Tachyon’s medication, called TACH101, is intended to target colorectal cancer (CRC) stem cells as well as the bulk tumor by blocking an enzyme called KDM4, which cancer stem cells need to grow and proliferate.

In the first phase of this trial Dr. King and his team will recruit patients with advanced or metastatic solid tumors to assess the safety of TACH101, and determine what is the safest maximum dose. In the second phase of the trial, patients with gastrointestinal tumors and colorectal cancer will be treated using the dose determined in the first phase, to determine how well the tumors respond to treatment.  

The CIRM Board also awarded $5,999,919 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This is an inherited condition caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. As a result, undigested sugar molecules build up in the body, causing progressive damage to the brain, heart, and other organs. There is no effective treatment and life expectancy for many of these children is only around ten years.

Dr. Gomez-Ospina will use the patient’s own blood stem cells that have been genetically edited to restore the missing enzyme. The goal of this preclinical program is to show the team can manufacture the needed cells, to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.

Finally the Board awarded $20,401,260 to five programs as part of its Translational program. The goal of the Translational program is to support promising stem cell-based or gene projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Those can include therapeutic candidates, diagnostic methods  or devices and novel tools that address critical bottlenecks in research.

The successful applicants are:

APPLICATIONTITLEPRINCIPAL INVESTIGATOR – INSTITUTIONAMOUNT  
TRAN4-14124Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug DiscoveryNikesh Kotecha — Greenstone Biosciences  $1,350,000
TRAN1-14003Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1αJianhua Yu — Beckman Research Institute of City of Hope  $6,036,002  
TRAN1-13983CRISPR/Cas9-mediated gene editing of Hematopoietic
stem and progenitor cells for Friedreich’s ataxia		Stephanie Cherqui — University of California, San Diego  $4,846,579
TRAN1-13997Development of a Gene Therapy for the Treatment of
Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting		Allyson Berent — Mahzi Therapeutics  $4,000,000
TRAN1-13996Overcoming resistance to standard CD19-targeted CAR
T using a novel triple antigen targeted vector		William J Murphy — University of California, Davis  $4,168,679

CIRM Board Approves Funding for New Clinical Trial Targeting Brain Tumors

/ Kevin McCormack / Leave a comment

The governing Board of the California Institute for Regenerative Medicine (CIRM) has awarded almost $12 million to carry out a clinical trial targeting brain tumors.

This brings the total number of CIRM funded clinical trials to 83.  

$11,999,984 was awarded to Dr. Jana Portnow at the Beckman Research Institute of City of Hope. They are using Neural stem cells (NSCs) as a form of delivery vehicle to carry a cancer-killing virus that specifically targets brain tumor cells.

Glioblastoma is the most common malignant primary brain tumor in adults and each year about 12,000 Americans are diagnosed. The 5-year survival rate is only about 10%.

The current standard of care involves surgically removing the tumor followed by radiation, chemotherapy, and alternating electric field therapy. Despite these treatments, survival remains low.

The award to Dr. Portnow will fund a clinical trial to assess the safety and effectiveness of this stem cell-based treatment for Glioblastoma.

The Board also awarded $3,111,467 to Dr. Boris Minev of Calidi Biotherapeutics. This award is in the form of a CLIN1 grant, with the goal of completing the testing needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial in people.

This project uses donor fat-derived mesenchymal stem cells that have been loaded with oncolytic virus to target metastatic melanoma, triple negative breast cancer, and advanced head & neck squamous cell carcinoma.

“There are few options for patients with advanced solid tumor cancers such as glioblastoma, melanoma, breast cancer, and head & neck cancer,” says Maria T. Millan, M.D., President and CEO of CIRM. “Surgical resection, chemotherapy and radiation are largely  ineffective in advanced cases and survival typically is measured in months. These new awards will support novel approaches to address the unmet medical needs of patients with these devastating cancers.”

The CIRM Board also voted to approve awarding $71,949,539 to expand the CIRM Alpha Clinics Network. The current network consists of six sites and the Board approved continued funding for those and added an additional three sites. The funding is to last five years.

The goal of the Alpha Clinics award is to expand existing capacities for delivering stem cell, gene therapies and other advanced treatment to patients. They also serve as a competency hub for regenerative medicine training, clinical research, and the delivery of approved treatments.

Each applicant was required to submit a plan for Diversity, Equity and Inclusion to support and facilitate outreach and study participation by underserved and disproportionately affected populations in the clinical trials they serve.

The successful applicants are:

ApplicationProgram TitleInstitution/Principal InvestigatorAmount awarded
INFR4-13579The Stanford Alpha Stem Cell ClinicStanford University – Matthew Porteus  $7,997,246  
INFR4-13581UCSF Alpha Stem Cell ClinicU.C. San Francisco – Mark Walters  $7,994,347  
INFR4-13586A comprehensive stem cell and gene therapy clinic to
advance new therapies for a diverse patient
population in California  		Cedars-Sinai Medical Center – Michael Lewis  $7,957,966    
INFR4-13587The City of Hope Alpha Clinic: A roadmap for equitable and inclusive access to regenerative medicine therapies for all Californians  City of Hope – Leo Wang  $8,000,000
INFR4-13596Alpha Stem Cell Clinic for Northern and Central California  U.C. Davis – Mehrdad Abedi  $7,999,997  
INFR4-13685Expansion of the Alpha Stem Cell and Gene Therapy Clinic at UCLA  U.C. Los Angeles – Noah Federman  $8,000,000
INFR4-13878Alpha Clinic Network Expansion for Cell and Gene Therapies  University of Southern California – Thomas Buchanan  $7,999,983  
INFR4-13952A hub and spoke community model to equitably deliver regenerative medicine therapies to diverse populations across four California counties  U.C. Irvine – Daniela Bota  $8,000,000
INFR4-13597UC San Diego Health CIRM Alpha Stem Cell Clinic  U.C. San Diego – Catriona Jamieson  $8,000,000

The Board also unanimously, and enthusiastically, approved the election of Maria Gonzalez Bonneville to be the next Vice Chair of the Board. Ms. Bonneville, the current Vice President of Public Outreach and Board Governance at CIRM, was nominated by all four constitutional officers: the Governor, the Lieutenant Governor, the Treasurer and the Controller.

In supporting the nomination, Board member Ysabel Duron said: “I don’t think we could do better than taking on Maria Gonzalez Bonneville as the Vice Chair. She is well educated as far as CIRM goes. She has a great track record; she is empathetic and caring and will be a good steward for the taxpayers to ensure the work we do serves them well.”

In her letter to the Board applying for the position, Ms. Bonneville said: “CIRM is a unique agency with a large board and a long history. With my institutional knowledge and my understanding of CIRM’s internal workings and processes, I can serve as a resource for the new Chair. I have worked hand-in-hand with both the Chair and Vice Chair in setting agendas, prioritizing work, driving policy, and advising accordingly.  I have worked hard to build trusted relationships with all of you so that I could learn and understand what areas were of the most interest and where I could help shed light on those particular programs or initiatives. I have also worked closely with Maria Millan for the last decade, and greatly enjoy our working relationship. In short, I believe I provide a level of continuity and expertise that benefits the board and helps in times of transition.”

In accepting the position Ms. Bonneville said: “I am truly honored to be elected as the Vice Chair for the CIRM Board. I have been a part of CIRM for 11 years and am deeply committed to the mission and this new role gives me an opportunity to help support and advance that work at an exciting time in the Agency’s life. There are many challenges ahead of us but knowing the Board and the CIRM team I feel confident we will be able to meet them, and I look forward to helping us reach our goals.”

Ms. Bonneville will officially take office in January 2023.

The vote for the new Chair of CIRM will take place at the Board meeting on December 15th.

Pioneering a new approach to HIV/AIDS

/ Kevin McCormack / Leave a comment
Dr. Steven Deeks. Photo courtesy UCSF

I’ve always been impressed by the willingness of individuals to step forward and volunteer for a clinical trial. Even more so when they are the first person ever to test a first-in-human therapy. They really are pioneers in helping advance a whole new approach to treating disease. 

That’s certainly the case for the first individual treated in a CIRM-funded clinical trial to develop a functional cure for HIV/AIDS. Caring Cross announced recently that they have dosed the first patient in the trial testing their anti-HIV duoCAR-T cell therapy.  

The trial is being led by UC San Francisco’s Dr. Steven Deeks and UC Davis’ Dr. Mehrdad Abedi. Their approach involves taking a patient’s own blood and extracting T cells, a type of immune cell.  The T cells are then genetically modified to express two different chimeric antigen receptors (CAR), which enable the newly created duoCAR-T cells to recognize and destroy HIV infected cells.  The modified T cells are then reintroduced back into the patient. 

The goal of this one-time therapy is to act as a long-term control of HIV with patients no longer needing to take anti-HIV medications. If it is successful it would be, in effect, a form of functional HIV cure.   

This first phase involves giving different patients different levels of the duoCAR-T therapy to determine the best dose, and to make sure it is safe and doesn’t cause any negative side effects.  

This is obviously just the first step in a long process, but it’s an important first step and certainly one worth marking. As Dr. Deeks said in the news release, “We have reached an important milestone with the dosing of the first participant in the Phase 1/2a clinical trial evaluating a potentially groundbreaking anti-HIV duoCAR-T cell therapy. Our primary goal for this clinical trial is to establish the safety of this promising therapeutic approach.” 

Dr. Abedi, echoed that saying. “The first participant was dosed with anti-HIV duoCAR-T cells at the UC Davis medical center in mid-August. There were no adverse events observed that were related to the product and the participant is doing fine.” 

This approach carries a lot of significance not just for people with HIV in the US, but also globally. If successful it could help address the needs of people who are not able to access antiretroviral therapies or for whom those medications are no longer effective.  

Today there are an estimated 38 million people living with HIV around the world. Every year some 650,000 people die from the disease.  

CIRM-funding supports world’s first stem cell treatment for spina bifida delivered during fetal surgery

/ California Institute for Regenerative Medicine (CIRM) / 2 Comments
Dr. Diana Farmer (right) meets with Emily Lofton and her baby Robbie who had stem cell treatment for spina bifida in the womb. Photo: UC Davis Health

The California Institute for Regenerative Medicine (CIRM) recently shared some encouraging news on The Stem Cellar about a CIRM-funded stem cell clinical trial for spina bifida at UC Davis Health. 
 
Spina bifida is a birth defect that occurs when the spine and spinal cord don’t form properly and can result in life-long walking and mobility problems for the child, even paralysis. 
 
Now, UC Davis has released more details about the clinical trial and the babies born after receiving the world’s first spina bifida treatment combining surgery with stem cells. The story was featured in BBC News and The Sacramento Bee.  
 
The first phase of the trial is funded by a $9 million grant from the California Institute for Regenerative Medicine. 
 
The one-of-a-kind treatment, delivered while a fetus is still developing in the mother’s womb, could improve outcomes for children with this birth defect. 

A Decade’s Work

“I’ve been working toward this day for almost 25 years now,” said Dr. Diana Farmer, the world’s first woman fetal surgeon, professor and chair of surgery at UC Davis Health and principal investigator on the study.  

In previous clinical trial, Farmer had helped to prove that fetal surgery reduced neurological deficits from spina bifida. Many children in that study showed improvement but still required wheelchairs or leg braces.  

Dr. Diana Farmer and Dr. Aijun Wang. Photo courtesy UC Davis Health

Farmer recruited bioengineer Dr. Aijun Wang to help take that work to the next level. Together, they researched and tested ways to use stem cells and bioengineering to advance the effectiveness and outcomes of the surgery.  

Farmer, Wang and their research team have been working on their novel approach using stem cells in fetal surgery for more than 10 years. Over that time, animal modeling has shown it is capable of preventing the paralysis associated with spina bifida. 

Preliminary work by Farmer and Wang proved that prenatal surgery combined with human placenta-derived mesenchymal stromal cells, held in place with a biomaterial scaffold to form a “patch,” helped lambs with spina bifida walk without noticeable disability. When the team refined their surgery and stem cells technique for canines, the treatment also improved the mobility of dogs with naturally occurring spina bifida. 

The CuRe Trial

When Emily and her husband Harry learned that they would be first-time parents, they never expected any pregnancy complications. But the day that Emily learned that her developing child had spina bifida was also the day she first heard about the CuRe trial, as the clinical trial is known.  

Participating in the trial would mean that she would need to temporarily move to Sacramento for the fetal surgery and then for weekly follow-up visits during her pregnancy.  

After screenings, MRI scans and interviews, Emily received the news that she was accepted into the trial. Her fetal surgery was scheduled for July 12, 2021, at 25 weeks and five days gestation.  

Farmer and Wang’s team manufactured clinical grade stem cells—mesenchymal stem cells—from placental tissue in the UC Davis Health’s CIRM-funded Institute for Regenerative Cures. The lab is a Good Manufacturing Practice (GMP) Laboratory for safe use in humans. It is here that they made the stem cell patch for Emily’s fetal surgery. 

The Procedure

During Emily’s historic procedure, a small opening was made in her uterus and they floated the fetus up to that incision point so they could expose its spine and the spina bifida defect. 

Credit: UC Davis Health

Then, the stem cell patch was placed directly over the exposed spinal cord of the fetus. The fetal surgeons then closed the incision to allow the tissue to regenerate. The team declared the first-of-its-kind surgery a success. 

On Sept. 20, 2021, at 35 weeks and five days gestation, Robbie was born at 5 pounds, 10 ounces, 19 inches long via C-section. 

For Farmer, this day is what she had long hoped for, and it came with surprises. If Robbie had remained untreated, she was expected to be born with leg paralysis. 

Baby Robbie underwent treatment for spina bifida while in the womb. Photo credit: UC David Health

“It was very clear the minute she was born that she was kicking her legs and I remember very clearly saying, ‘Oh my God, I think she’s wiggling her toes!’” said Farmer. “It was amazing. We kept saying, ‘Am I seeing that? Is that real?’” 

Both mom and baby are at home and in good health. Robbie just celebrated her first birthday. 

Emily Lofton and her baby daughter Robbie who underwent treatment for spina bifida while in the womb.

The CuRe team is cautious about drawing conclusions and says a lot is still to be learned during this safety phase of the trial. The team will continue to monitor Robbie and the other babies in the trial until they are 6 years old, with a key checkup happening at 30 months to see if they are walking and potty training. 

“This experience has been larger than life and has exceeded every expectation. I hope this trial will enhance the quality of life for so many patients to come,” Emily said. “We are honored to be part of history in the making.” 

Read the official release from UC Davis Health here.  

Stem Cell Agency Invests $46 Million in New Education Program

/ Kevin McCormack / Leave a comment
CIRM Bridges students 2022. The CIRM Board approved funding for a program to help even more students advance a career in science.

The governing Board of the California Institute for Regenerative Medicine (CIRM) has approved $46,076,430 to invest in its newest education pillar- the COMPASS (Creating Opportunities through Mentorship and Partnership Across Stem cell Science) training program.

Education is at the core of CIRM’s mission of accelerating world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world. And funding these additional programs is an important step in ensuring that California has a well-trained stem cell workforce.

The objective of COMPASS is to prepare a diverse cadre of undergraduate students for careers in regenerative medicine through combining hands-on research opportunities with strategic and structured mentorship experiences.

“Education and infrastructure are two funding pillars critical for creating the next generation of researchers and conducting stem cell based clinical trials,” says Jonathan Thomas, Ph.D., J.D., Chair of the CIRM Board. “The importance of these programs was acknowledged in Proposition 14 and we expect that they will continue to be important components of CIRM’s programs and strategic direction in the years to come.”

Most undergraduate research training programs, including those targeting students from underserved communities, target individuals with predefined academic credentials as well as a stated commitment towards graduate school, medical school, or faculty positions in academia. COMPASS will support the development and implementation of novel strategies to recognize and foster untapped talent that can lead to new and valuable perspectives that are specific to the challenges of regenerative medicine, and that will create new paths to a spectrum of careers that are not always apparent to students in the academic, undergraduate environment.

COMPASS will complement but not compete with CIRM’s Bridges program, a subset of which serve a different, but equally important population of undergraduate trainees; similarly, the program is unlikely to compete for the same pools of students that would be most likely to receive support through the major NIH Training Programs such as MARC and RISE.

Here are the 16 successful applicants.

Application numberTitlePrincipal InvestigatorAmount
EDUC5-13840  The COMPASS Scholars Program – Developing Today’s Untapped Talent into Tomorrow’s STEM Cell Researchers    John Matsui, University of California, Berkeley    $2,908,950
EDUC5-13634  COMPASS Undergraduate Program  Alice F Tarantal, University of California, Davis    $2,909,950  
EDUC5-13637  Research Mentorship Program in Regenerative Medicine Careers for a Diverse Undergraduate Student Body    Brian J. Cummings, University of California, Irvine    $2,729,900
EDUC5-13665  CIRM COMPASS Training Program (N-COMPASS)  Cindy S Malone, The University Corporation at California State University, Northridge    $2,909,700  
EDUC5-13817  COMPASS: Accelerating Stem Cell Research by Educating and Empowering New Stem Cell Researchers  Tracy L Johnson, University of California, Los Angeles    $2,910,000  
EDUC5-13744  Training and mentorship program in stem cell biology and engineering: A COMPASS for the future  Dennis Clegg, University of California, Santa Barbara    $2,746,000  
EDUC5-13636  Research Training and Mentorship Program to Inspire Diverse Undergraduates toward Regenerative Medicine
Careers (RAMP)		  Huinan Hannah Liu, The Regents of the University of California on behalf of its Riverside Campus    $2,910,000  
EDUC5-13679  Inclusive Pathways for a Stem Cell Scholar (iPSCs) Undergraduate Training Program    Lily Chen, San Francisco State University    $2,894,500
EDUC5-13733  A COMPASS to guide the growth of a diverse regenerative medicine workforce that represents California and benefits
the world		  Kristen OHalloran Cardinal, Cal Poly Corporation, an Auxiliary of California Polytechnic State University, San Luis Obispo    $2,887,939  
EDUC5-13619  Increase Diversity, Equity, and Advancement in Cell Based Manufacturing Sciences (IDEA-CBMS)  Michael Fino, MiraCosta College    $2,894,500  
EDUC5-13667  COMPASS Program for Southern California Hispanic Serving Institution  Bianca Romina Mothé, California State University San Marcos Corporation    $2,877,200  
EDUC5-13653  Student Pluripotency: Realizing Untapped Undergraduate Potential in Regenerative Medicine  Daniel Nickerson, California State University, San Bernardino    $2,909,853  
EDUC5-13647  COMPASS: an inclusive Pipeline for Research and Other Stem cell-based Professions in Regenerative medicine
(iPROSPR)  		  Alison Miyamoto, CSU Fullerton Auxiliary Services Corporation    $2,883,440
EDUC5-13686  Training Undergraduates in Stem Cell Engineering and Biology (TUSCEB)    Kara E McCloskey, University of California, Merced    $2,909,999
EDUC5-13853  COMPASS: Guiding Undergraduates to Careers in Regenerative Medicine    Senta Georgia, University of Southern California    $2,899,999
EDUC5-13910  IDEA-CBMS – Increase Diversity, Equity, and Advancement in Cell Based Manufacturing Sciences    James Dekloe, Solano Community College    $2,894,500

Life lessons learned in the CIRM summer intern program

/ Kevin McCormack / Leave a comment
SPARK poster session; Photo by Esteban Cortez

When I was in high school I spent my summers working in a shoe shop and playing soccer with my mates. It never occurred to me that I could do something really  worthwhile with that time. So, when I meet the high school students who took part in the California Institute for Regenerative Medicine’s SPARK program I realized I had wasted a lot of time.

For those not familiar with SPARK, it stands for Summer Program to Accelerate Regenerative Medicine Knowledge. It’s a summer program offering high school students a chance to work in a world-class stem cell and gene therapy research facility. The quality of the work they do is truly remarkable. By the end of the summer they are doing projects that many full-time researchers would be proud of.

As part of that program the students also must write blogs and post photos and videos to Instagram to chart their progress. The quality of that work is equally impressive. Last week we posted items about the two best blogs from the students. But there were so many other fine entries that we thought it would be worthwhile to highlight elements of those.

For instance, Ricardo Rodriguez at Charles R. Drew University had some interesting observations on life, even when it’s not always working out the way you planned:

Ricardo Rodriguez: Photo by Esteban Cortez

“Cancer is not life going wrong so much as it is life changing. If mutation is random, then so is life. That beautiful randomness that drives evolution and extinction, change and stagnation, life and death, and for you to think that that part of your body could be simple in any way, whether it be simply evil, simply inconvenient, simply structured, is simply hilarious. There is beauty in your body’s complexity, adaptability, and resilience, and these attributes are not barred from any part of your life.”

Mindy Rodriguez at Beckman City of Hope says she learned valuable lessons from working with mice, creatures she previously considered scary, dirty and vicious, but later came to like:

“The CIRM SPARK program reinforced the value of facing my fears by exploring the unknown and most importantly taught me to be comfortable with the uncomfortable. In both cases, I found that it is our response to fear that shapes who we are. We can either run away from the thing that scares us or take each moment as a learning opportunity, embracing change over comfort.”

Manvi Ketireddy at work at UC Davis

Manvi Ketireddy at UC Davis had a similar experience, learning to accept things not working out.

“A researcher must be persistent and have the ability to endure lots of failures. I think that is what I love about research: the slight possibility of discovery and answers amid constant defeat is one of the greatest challenges to exist. And boy, do I love challenges.”

Ameera Ali in the lab (fish not included)

Ameera Ali at Sanford Burnham Prebys says she had struggled for years to decide on a career direction, but the internship gave her a fresh perspective on it all.

“Growing up, I never really knew what I wanted to do for a living, and I think that’s because I wanted to do everything. In kindergarten I wanted to be a paleontologist. In 5th grade I wanted to be the CEO of The San Diego Union Tribune, and in 9th grade I wanted to be a physicist at NASA. By 10th grade I was having an existential crisis about what to do with my life, and so began the search for my purpose at the ripe old age of 15.

So now, writing this blog, I never thought I’d end up spending so much of my time in a room filled floor to ceiling with fish tanks. You might be wondering, how does one end up going from physicist to fish farmer? Well, I’m not completely sure to be honest, but it’s been a very fun and interesting experience nonetheless.”

She says by the end she says what initially felt like mundane chores were actually moments worth celebrating.

“These aquatic friends have taught me a lot of valuable life lessons, like being appreciative of the little things in life, caring for others and see things from a different perspective, and realizing that

working in a biology lab allows me to explore my passions, be creative, and be a mother to hundreds of fish children on the side.”

SPARK attracts students from all over California, and it’s that diversity that makes it so important.

Alexa Gastelum

My name is Alexa Gastelum and I am from a small border town called Calexico. It is located in the Imperial Valley around two hours away from San Diego. I found out about this Internship from my Math teacher and Mesa Coordinator. They discussed what it was about, and I immediately knew that I wanted to apply. I have always been interested in doing labs and researching so I knew that it would be the perfect opportunity for me. It is not normal to be presented with an opportunity like this from where I’m from because it is a small and low-income town. When I told my family about this internship they were very supportive. They agreed that I needed to apply for it since it was an extremely good opportunity. Even though I would need to spend my summer away from my hometown, they were okay with it because they knew that I could not miss out on the opportunity. I decided to write my personal statement on a disease that hit close to home with my family which was Alzheimer’s. It is a disease that runs in my family and my uncle passed from it. I believe that this is what sparked my interest because I wanted to understand how it worked and how it affects the brain.

At the SPARK event Alexa told me her grandmother was so proud of her for being accepted at the program that she was going around town telling everyone about it. Her grandmother, and all the other grandmothers and mothers and fathers, had every reason to be proud of these students. They are remarkable young people and we look forward to following their careers in the years to come.