Stem cell research

#Take6ForPD: CIRM Board member David Higgins shares why Parkinson’s awareness matters

/ Katie Sharify / 1 Comment

April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease. 

In addition to highlighting our commitment to funding research for Parkinson’s Disease, we’re also giving patients and patient advocates the opportunity to share their stories and experiences.

In this blog, CIRM Board Member David Higgins shares why spreading awareness of Parkinson’s is important to him.

Dr. David R. Higgins grew up in West Virginia with four siblings. He earned his Ph.D. in molecular biology and genetics from the University of Rochester, followed by a postdoctoral fellowship at the National Cancer Institute before moving to San Diego in 1990 to work in biotech.

In December 2011, Higgins was diagnosed with Parkinson’s. Since then, he has become an advocate for people with Parkinson’s and their caregivers. He uses his personal experiences to guide this advocacy work, focused on improving quality of life issues through education, support, training, networking and promoting increased research funding to find a cure.

He currently serves as the Parkinson’s patient advocate member of CIRM’s governing board.

What is your personal connection to Parkinson’s Disease (PD)?

For me, Parkinson’s disease (PD) is a family legacy. My maternal grandmother suffered from PD and participated in some of the earliest Levodopa clinical trials held in the late 1960s.

In early 2014 my mother died with Lewy Body Dementia. My maternal uncle and great uncle also suffered from PD. In December 2011, I was diagnosed with PD completing the Parkinson’s Trifecta: care partner, scientist, and patient.

Additionally, in my biotech career, I worked for a company that is developing a gene therapy product to treat PD. This product currently is in human clinical trials in Europe.

What do you want people to know about PD?

PD is the second most common neurodegenerative disease, behind only Alzheimer’s disease in prevalence and with a rapidly growing incidence. Currently there are about 1 million people in the US diagnosed with PD.

PD is unique in that we know a lot about what the underlaying defect is. And there are likely several factors that influence whether you are going to develop PD. The most common model is a genetic predisposition to PD that exists in some people. If a person with a predisposition to PD is exposed to certain environmental factors, then development of the disease follows.

However, not every person who follows this pattern will develop PD. There is a familial form of PD that accounts for about 10% of the diagnosed cases. These tend to manifest earlier in life — in the patient’s 20s rather than the usual 60s.

There are a number of human genes known to be involved. However, identification of all of the so-called PD genes is not exhaustive. For example, there are about 80+ mutations that have been associated with the development of PD in humans. So clearly the situation is more complex.

Why is it important to you to raise awareness about PD? 

It is critical that the stake holders, PD patients, California citizens, understand the impact that new treatments and even a cure for PD could have:

  • Social — Quality of life, decrease pain and suffering
  • Medical — Decrease huge medical costs to society to support and care for chronic illnesses
  • Economic — Positive impact on the economy by maintaining and increasing productivity of a sector of society that is at its peak productivity
  • Diagnosis — Fast accurate differential diagnoses of PD and related movement disorders such as MSA and SMA.
Higgins on a recent scuba diving trip

Why is it important to continue investing in all PD research and treatments — such as those that improve certain symptoms — and not just research that aims at finding a cure?

The goal in PD research as well as any new therapy is that new and effective treatments are just as important—and likely more within reach—than the elusive Holy Grail cure. Effective treatments that slow progression or work to treat chronic symptoms are incredibly beneficial to patients and the kinds of treatments that patients ask about most.

Why is it important that an agency like CIRM continues to invest in regenerative medicine research and treatment for PD?

Basic research and clinical development both need funding to understand the disease better, as well as to design testing in humans. Developing new therapeutics for PD and the research effort that supports it will require a huge multi-discipline collaborative effort and the financial funding to enable it. CIRM has the resources to put together the collaborative effort needed and is uniquely positioned to build appropriate collaborations needed.

To date, CIRM has invested more than $60 million in helping research for Parkinson’s progress from a basic or Discovery level through clinical trials. To learn more about CIRM’s investments in finding treatments for the disease, visit this page on our website.

CIRM commits $60 million for Parkinson’s research, joins #Take6ForPD awareness campaign 

/ Esteban Cortez / 1 Comment

April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease. 

What is Parkinson’s Disease? 

Parkinson’s disease (PD) is a neurodegenerative disease that affects nearly 1 million people in the United States and more than 10 million people around the world. In California, more than 106,000 people live with PD. 

Symptoms of PD include tremors, slow movement, muscle rigidity, balance issues and lack of facial expressions. Parkinson’s disease occurs when the neurons or nerve cells in the portion of the brain that controls movement die off. These neurons send signals by releasing a chemical called dopamine and are referred to as dopaminergic neurons.  

Parkinson’s is the second-most common neurodegenerative disease after Alzheimer’s disease. No cure exists for the disease. 

CIRM’s Commitment to Funding Parkinson’s Research

CIRM remains committed to funding regenerative medicine research—including stem cell and gene therapy research—for PD treatments to improve quality of life for patients.  

To date, CIRM has invested more than $60 million in helping research for PD progress from a basic or Discovery level through clinical trials. 

This year, CIRM awarded $4 million to a late-stage preclinical project by Ryne Bio aiming to improve treatment for idiopathic PD. In the proposed stem cell therapy, investigators at Ryne Bio are aiming to deliver dopamine-producing cells to replace the lost neurons to the brain of PD patients to restore or improve motor function. 

“Receiving a Parkinson’s diagnosis is a difficult and life-changing event,” says Ryne Bio CEO Nick Manusos. “This Parkinson’s Awareness Month, we honor patients and their loved ones by reflecting on the clinical progress made so far, and looking forward to a brighter, healthier future.” 

Other regenerative medicine approaches CIRM has funded to target PD involve understanding the disease and looking for new drugs to treat it.  

CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, turning them into the kind of stem cell that can be transformed into any other cell in the body, then coaxing those cells to become dopaminergic neurons that are lost to the disease.  

Those cells showed signs of the disease in the lab dish and were distinctly different from the same cells created from people without PD.  

Another approach involves using CRISPR gene editing technology to reduce the levels of a toxic protein called alpha synuclein, which builds up in the dopaminergic brain cells affected by PD. 

Overall, CIRM has awarded more than 30 grants aiming to better understand the disease and find regenerative medicine-based treatments.  

“CIRM’s commitment to funding research for Parkinson’s is unwavering,” says CIRM President and CEO Maria Millan. “Our goal is to fund the most promising regenerative medicine research to support finding the best treatments to fight this devastating disease that affects millions.” 

CIRM’s Task Force on Neuroscience & Medicine 

As part of its Strategic Plan, CIRM has also launched a Task Force on Neuroscience and Medicine to set a plan for the $1.5 billion allocated for the support of research and the development of treatments for diseases and conditions of the brain and central nervous system. These include Parkinson’s, Alzheimer’s, stroke, dementia, epilepsy and other diseases and conditions. 

The Task Force will host public meetings to gather community input to identify potentially high-impact opportunities in basic neuroscience, neurodegenerative disease, neuropsychiatric disease, neurodevelopment, and normal brain aging.   

The goal of the task force is to provide final recommendations to CIRM and its governing board within six months of inception. 

To see a listing of upcoming and past Task Force meetings, visit our website. 

Meet CIRM’s growing world class team 

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The California Institute for Regenerative Medicine (CIRM) continues to build its world class team to deliver the full potential of regenerative medicine to the people of California and around the world. CIRM is rebuilding and expanding its team to meet new challenges and advance the mission of the Agency. 

Rafael Aguirre-Sacasa and Koren Temple-Perry join the growing ranks of new team members that CIRM has hired since the passage of Proposition 14 in November 2020. We recently shared an announcement welcoming them to the team here.

Among the new hires is Emily Reyes, who joins CIRM as a project manager in the Medical Affairs and Policy team. She earned her MPH from California State University, Fullerton and her BS degree from Oregon State University. Emily comes to CIRM from UCSF, where she was a clinical research coordinator overseeing several research studies with responsibilities including patient coordination, data collection and manuscript preparation. 

Marivel De La Torre also joins the Medical Affairs and Policy team as a project manager and is responsible for overseeing critical activities as CIRM continues to build and execute the medical affairs strategic plan. Marivel comes to CIRM from Sacramento, where she has over 18 years of State service managing numerous projects within the Department of Transportation, Department of Healthcare Services, Department of Corrections and Rehabilitation, and California Air Resources Board. 

Ben Chau is CIRM’s new Associate Director of IT. He has worked in management for over 13 years and brings a wide range of skills and leadership to this role. Before joining CIRM, he was the IT Director for Asian Health Services where he put forward IT strategic plans to ensure systems were up to date with technology and security compliance.  

James (Jim) Campanelli, PhD joins CIRM as a Senior Science Officer in Therapeutics Development. Prior to CIRM, Jim was employed at Q Therapeutics where he directed research and development efforts for past 17 years.Jim received a PhD in Biological Sciences from Stanford and was an assistant Professor of Biochemistry at University of Illinois at Urbana-Champaign. 

Elizabeth (Liz) Noblin, PhD joins the Review team as a Senior Science Officer. Before joining us at CIRM, Liz was a program and project manager at 23andMe, where she supported both the Research and Therapeutics divisions. Liz received a PhD in Genetics from Yale and completed her postdoc with Dr. Anne Brunet at Stanford.  

Charlie Shaw, PhD is a Senior Science Officer and the newest member of the Business Development & Alliance Management team. Charlie joins CIRM after more than 12 years’ experience in academic technology licensing offices. His most recent role was at the Moffitt Cancer Center where he was the Associate Director of Licensing. Charlie has a PhD in Molecular Genetics and Microbiology from Duke University, post-doctoral training in genetic medicine from University of Chicago, BS in Biology from Davidson College and is currently obtaining his MBA from East Carolina University. 

Scott Tocher, CIRM’s former General Counsel, returned to the agency last November as a retired annuitant to help fill CIRM’s workload gap in the legal division, and rejoined as a permanent employee as Senior Director of Board Governance in February. Scott originally came to CIRM’s legal department in 2005, assisting in the development of CIRM’s policies, programs and regulations through 2019.  

Janie Byrum, PhD, joins the Scientific Programs team as a Science Officer. In her most recent role as an R&D engineer at CZ Biohub, she led and built imaging pipeline projects to interrogate the impacts of viral infection on cell function as part of the Infected Cell and Quantitative Cell Science departments. Janie has a PhD in Biomedical Sciences from the University of New Mexico Health Sciences Center and a post-doctoral training in immunology from Columbia University Irving Medical Center. 

Chan Lek Tan, PhD, a Science Officer in CIRM’s Scientific Programs team, is an experienced and inquisitive scientist with a background in neurodevelopmental and neurodegenerative diseases. In his most recent roles as Principal Scientist at Genentech and BridgeBio, Chan furthered his expertise in pre-IND development of small molecule and RNA-based therapeutics for neuro-based indications. Chan has a PhD in Molecular and Cellular Neuroscience from The Rockefeller University, New York where he studied under Dr. Paul Greengard.  

Gemma Domingo joins CIRM as Sr. Finance Officer.  She attended the University of the East in Manila and earned a Bachelor of Science degree in accounting. In 1990, she immigrated to the U.S. to join her family and joined state service in 1998. Throughout her time with state service, Gemma previously worked at the Department of Parks and Recreation, Department of Child Support Services, and California Office of Emergency Services. 

Leyla Najmi, a seasoned Senior Human Resources (HR) leader and business partner, joins CIRM as the Director of HR. Leyla has used her strong HR acumen and a blend of experience in various positions within private and public sectors to affect positive change in the organizations where she has worked.  Effectively balancing big picture thinking and strategic planning with hands-on execution has allowed Leyla to combine decisiveness, strong business acumen and formal education to achieve objectives both professionally and personally. 

“We are delighted to have these talented individuals join the CIRM team,” says CIRM President and CEO Dr. Maria Millan. “They all bring unique qualifications and critical skills to support our strategic plan and advancement of CIRM’s mission to accelerate world class science for California and the world. We look forward to working with all of them.” 

CIRM welcomes two additions to its leadership team

/ Katie Sharify / 1 Comment
Left to right: Rafael Aguirre-Sacasa, General Counsel and Koren Temple-Perry, Senior Director of Marketing Communications

The California Institute for Regenerative Medicine (CIRM) continues to build its world class team to deliver the full potential of regenerative medicine to the people of California and around the world. 

CIRM is pleased to welcome two new members to its leadership team, including Rafael Aguirre-Sacasa as General Counsel and Koren Temple-Perry as Senior Director of Marketing & Communications. 

Rafael has over 25 years of experience in corporate law and joins CIRM after seven years at Standard BioTools (formerly Fluidigm Corp). There, he provided worldwide commercial, strategic and transactional legal support for all functions of the NASDAQ-listed life science company.  
 
Prior to this role, he held a variety of management positions and provided legal support for a wide range of commercial, intellectual property and corporate matters at Teradici Corporation, PMC-Sierra, Inc., Autodesk, Hyperion solutions, Grupo Financiero and Xilinx Inc. 
 
Rafael received his bachelor’s degree in history and government from Dartmouth College and law degree from the University of California-Hastings. As CIRM’s new General Counsel, Rafael will support the President, Board (ICOC), management and working groups on all legal matters affecting the agency. 
 
“Joining the Institute as General Counsel is not just a job to me,” says Raphael. “It’s an opportunity to be part of an organization that is changing lives. I am enthusiastic about helping to implement CIRM’s strategic plan and contributing to their mission of accelerating stem cell treatments to patients in need, especially those in underserved and underrepresented communities.” 
 
The agency also welcomes Koren Temple-Perry as Senior Director of Marketing Communications.  
 
Koren joins CIRM after spending the last 15 years translating complex scientific information into compelling content for diverse audiences.  
 
As a former journalist, she has worked extensively in the areas of patient and community health education, health advocacy, and scientific communications for hospitals, research institutes, and healthcare organizations.  
 
Prior to joining CIRM, she founded Temple Communications LLC, which specializes in scientific copywriting and marketing communications for public health organizations.  
 
She served as consulting communications director for March for Science, where she led the media and communications campaign for the global marches. She spent many years as a communications writer and digital marketing manager at academic medical centers throughout New York.  
 
Koren earned her master’s degree in journalism from the S.I. Newhouse School of Public Communications at Syracuse University and a bachelor’s degree in communications from Santa Clara University. Her passion for improving health equity led to her appointment as District 5 Commissioner to the Alameda County Public Health Commission and to the Board of Directors for Jamal’s Helping Hands, a non-profit dedicated to helping families of color navigate rare diseases.  
 
“I am truly honored to lead CIRM’s marketing communications team as the Agency aims to bring promising stem cell and gene therapy science to diverse communities,” says Koren. “I see incredible potential in building meaningful partnerships and elevating our outreach strategies to amplify cutting-edge treatments so that all Californians have equitable access to them. I am eager to make a positive impact in my new role and contribute to CIRM’s overall success.”   
 
“We are delighted to have these talented individuals join the CIRM team,” says CIRM President and CEO Dr. Maria Millan. “They all bring unique qualifications and critical skills to support our strategic plan and advancement of CIRM’s mission to accelerate world class science for California and the world. We look forward to working with all of them.”

CIRM invests $2.7 million in research to develop stem cell-based tendon tissue for shoulder injuries

/ California Institute for Regenerative Medicine (CIRM) / 1 Comment

The California Institute for Regenerative Medicine (CIRM) has awarded a $2.7 million research grant to Scripps Health to investigate the use of stem cells to engineer rotator cuff tendons in the lab that can then be used to repair common shoulder injuries.  

The rotator cuff is a group of four muscles and their attached tendons, which hold the upper arm in place in the shoulder socket. The rotator cuff attaches the humerus to the shoulder blade and helps to lift and rotate the arm.  

Most rotator cuff tears result from natural degeneration of the tendon over time, frequently causing pain and possibly limited use of the arm. As the condition progresses and worsens, the shoulder can become weaker, more painful, and less useful. 

Image credit: Cedars-Sinai

The use of pluripotent stem cells to develop lab-grown tendon tissue to repair rotator cuffs could benefit older patients with significant tears due to age-related degeneration.  

“For this group of patients, the failure rate for conventional repair surgery is about 40%,” said Dr. Darryl D’Lima, director of orthopedic research for the Shiley Center for Orthopaedic Research and Education (SCORE) at Scripps Clinic and the initiative’s lead investigator. “This is a common injury, and as our population ages, there’s a pressing need to find new solutions.” 

Annually, over 100,000 Californians sustain tendon injuries, the majority of which require surgical repair. Failure rates for shoulder rotator cuff tendon repairs vary between 20% and 90%. Failed rotator cuff tendons lead to early development of osteoarthritis, for which the only effective treatment is total joint replacement. Preventing disability offers significant socioeconomic benefits and reductions in healthcare costs also are likely to be significant. 

Image credit: Scripps

The funding from CIRM will support the initiative’s three-year discovery phase at Shiley Center for Orthopaedic Research and Education (SCORE) on Torrey Pines Mesa. Researchers plan to develop a series of lab tests called assays to assess and measure the makeup of the tendon cells and develop the tendon material in the lab.

SCORE researchers intend to explore the possibility of transforming pluripotent stem cells into tendon-like cells, which would be embedded into a scaffold structure of “electrospun” fibers and grown into tendon tissue using bioreactors.  

The lab-engineered tendon tissue also would be stretched to help stimulate the cells to produce more tissue, and to help align the electrospun fibers so they mimic the intricate alignment of fibers in native tissue. Lining up the tendon fibers in the same direction that stretching occurs is needed to resist tensile force, or the stretching force experienced during motion. 

To learn more about the California Institute for Regenerative Medicine, visit our website.

Meshing a passion for medicine with stem cell research

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Kevin Brown is an adventurer at heart. He was a pre-med student when he found a unique way to combine his passion for medicine in neuroscience with his natural inclination to explore and discover through scientific research.

A 2022 graduate of the California Institute for Regenerative Medicine’s (CIRM) Bridges to Stem Cell Research and Therapy Program, Kevin says the experience was a great opportunity to mesh his passion for medicine with his instinct for research in the stem cell/regenerative medicine field.

“I recognized the vital importance of having basic scientific training and thinking skills in relationship to being a good physician,” Kevin said, noting that these factors inspired him to apply to the Bridges program.

Started in 2009, the CIRM Bridges program provides paid regenerative medicine and stem cell research internships to students at universities and colleges that don’t have major stem cell research programs. Each Bridges internship includes thorough hands-on training and education in regenerative medicine and stem cell research, and direct patient engagement and outreach activities that engage California’s diverse communities. 

A Growing Field with Many Opportunities

Kevin recognizes that regenerative medicine is a growing field that offers great opportunity for exploration and discovery. It also is likely to have a strong impact on patient care in the field of neuroscience and many other areas of human health. Participating in the program has helped Kevin find a career path that excites both his passions for exploration and for patient care.

During his time in the Bridges program, Kevin attended California State University, San Marcos and he completed his program training at the Dorris Neuroscience Center at The Scripps Research Institute in San Diego.

“Medicine was all that was on my mind but as I got deeper into my research I fell more and more in love with the process of science and began seeing myself as a leader in this field in the future,” Kevin said. “I am now considering taking the MD-PhD path to satisfy both my yearning to leave a positive impact on the world through healthcare and continue venturing through the unknowns to hopefully create meaningful breakthroughs in science in the future.”

He credits his mentors—graduate student Anna Verduzco and principal investigator Dr. Hollis Cline—for helping him discover his future career path. The program and his mentors, he said, have made him a better student, a better scientist, and a better person overall.

“They helped mold the way that I approach scientific and life problems—curiosity at the forefront followed by openness to try something new.”

Overcoming Challenges

Kevin said one challenge of the internship was learning how to think, write, and communicate scientifically.

“The conventional way of learning and thinking in school is vastly different than the scientific way of thought,” Kevin said. “However, my lab partners were extremely helpful in helping me cultivate these skills by having me consistently talking through my experiments with them, do periodic write-ups on my progress, and give lab meeting presentations.”

Kevin clearly was an apt student of these lessons because at the conclusion of his internship, he offered a poster presentation at the Annual Biomedical Research Conference for Minoritized Scientists and won the 2022 Outstanding Presentation Award.

Paying It Forward

Today, Kevin is a full-time student at CSU San Marcos completing his Bachelor of Science in Biology with a concentration in Physiology. He is still a part-time research intern at The Scripps Research Institute in the Cline lab working toward understanding how stem cell derived brain cells can be used to study the intricacies of Alzheimer’s Disease in a genetic and cell-signaling context.

And he’s paying it forward for other students. “I am building a mentoring network to provide CIRM’s SPARK high school program interns with the necessary help and tools to transition into their college career feeling empowered and confident in their ability to succeed.”

Image courtesy The Cline Lab

He offers important advice regarding the field of stem cell and regenerative medicine research, and strong words for future explorers everywhere: be courageous, adaptable, and resilient.

“A lot of the work being done, especially within the context of regenerative medicine and neuroscience, has never been done before,” he said, noting that young pioneers in this space should be creative in their approach and not easily dissuaded by failure.

Kevin urges more people to become pioneers.

He adds, “Stem cell research is vital to the development of understanding how we can address the vast amount of diseases and conditions that impact humans.”

About the Bridges Program

The Bridges program is proud to claim 1,735 Bridges alumni, and more Bridges trainees are completing their internships in 2023. CIRM has 15 active Bridges programs throughout California, each with its own eligibility criteria and application process.

If you are interested in applying, please visit this web page for more details about each program. If you have questions about the Bridges program, please email the CIRM Bridges director, Dr. Kelly Shepard at education@cirm.ca.gov. 

Investing in a stem cell treatment for Hurler syndrome

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The California Institute for Regenerative Medicine (CIRM) awarded $5,444,353 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 are no signs or symptoms of the condition at birth, although some have a soft out-pouching around the belly-button or lower abdomen. Those with severe MPS I generally begin to show other signs and symptoms of the disorder within the first year of life. 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.

“The funding will pave the way for trials in people to realize a more effective therapy for this devastating disease,” Gomez-Ospina said. “We will also generate safety and toxicity data that could facilitate the application of our genome editing platform to other genetic disorders for which a significant unmet need still exists.”

Advancing cutting-edge treatment to improve kidney transplantation in children

/ Katie Sharify / 1 Comment

Stanford physician-scientist Alice Bertaina, MD, PhD, associate professor of pediatrics has received about $18 million from the California Institute for Regenerative Medicine (CIRM) for a clinical trial to allow kidney transplantation without the need for long-term immunosuppression.

Dr. 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 which have significant long-term risks.

Funding development of a vaccine for acute myelogenous leukemia (AML)

/ Esteban Cortez / 2 Comments
Dr. Karin Gaensler. Photo credit: Steve Babuljak/UCSF

Adult acute myelogenous leukemia—also known as acute myeloid leukemia (AML)—is a blood cancer in which the bone marrow makes a large number of abnormal blood cells. 

About 20,000 new cases of AML are diagnosed each year in the US with a 5-year survival rate of around 29%. In 2022, there were nearly 12,000 deaths from AML. Many AML patients—a majority of which are over 60 years old—relapse after treatment. Blood stem cell transplant can be curative, but many older patients do not qualify, showing that there is a significant unmet medical need in treating AML. 

That’s why the California Institute for Regenerative Medicine (CIRM) awarded $6,000,000 to Dr. Karin Gaensler at the University of California, San Francisco (UCSF) to support development of a safe and effective vaccine for the blood cancer AML to improve relapse-free survival. 

To develop the cancer vaccine, Dr. Gaensler and her team will engineer the patient’s blood stem cells to maximize stimulation of leukemia-specific killing activity and reintroduce engineered cells back to the patient to target and kill residual leukemia stem cells.  

This approach holds the potential for long-term effectiveness as it targets both AML blasts and leukemic stem cells that are often the source of relapse.  

This award is a continuation of a previous CIRM grant that will support the manufacture of the vaccine and the completion of late-stage testing and preparation needed to apply to the US Food and Drug Administration (FDA) for permission to begin a clinical trial. 

The Most Read Stem Cellar Blog Posts of 2022

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This year was a momentous one for the California Institute for Regenerative Medicine (CIRM). You can read some of our achievements in our 2021-2022 annual report.  

As always, we shared our most exciting updates and newsworthy stories—topics ranging from stem cell research to diversity in science—right here on The Stem Cellar. More than 100,000 blog visitors followed along throughout the year!  

In case you missed them, here’s a recap of our most popular blogs of 2022. We look forward to covering even more topics in 2023 and send a sincere thank you to our wonderful Stem Cellar readers for tuning in!   

In Memory of Kevin McCormack 

We cannot close out the year without honoring our dear friend and colleague Kevin McCormack, who passed away suddenly in December. Kevin was CIRM’s Director of Patient Advocacy and loved writing for The Stem Cellar. He did a wonderful job in translating complex science for the general public and was a great mentor to the CIRM team. Many of his closest friends and colleagues wrote memories about him, and we compiled them in this blog post honoring his life and dedication to CIRM and patients everywhere.  

How stem cells helped Veronica fight retinitis pigmentosa and regain her vision 

We shared the story of Veronica McDougall, who thought everyone saw the world the way she did: blurry, slightly out-of-focus and with tunnel vision. When she was 24, she went to see a specialist who told her she had retinitis pigmentosa, a rare degenerative condition that would eventually leave her legally blind. Click through to read about her experience participating in a CIRM-funded clinical trial with a company called jCyte. 

Smoking marijuana could be bad for your heart, but there is an unusual remedy 

Millions of Americans use marijuana for medical reasons, such as reducing anxiety or helping ease the side effects of cancer therapy. Millions more turn to it for recreational reasons, saying it helps them relax. Now a new study says those who smoke marijuana regularly might be putting themselves at increased risk of heart disease and heart attack. Check out this blog to learn how a team at Stanford Medicine used the iPSC method to create human endothelial cells and, in the lab, found that THC appeared to promote inflammation in the cells. 

A pioneering couple uproot their lives to help their baby 

This year, we shared some encouraging news 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. This blog told the story of parents Michelle Johnson and Jeff Maginnis, who learned 20 weeks into the pregnancy that the fetus had spina bifida. Read the whole story to learn about their experience and the status of their baby Tobi.  

And that wraps up The Stem Cellar’s top blog posts of 2022! If you’re looking for more ways to get the latest updates from The Stem Cellar and CIRM, follow us on social media on Facebook, Twitter, LinkedIn, and Instagram.