Digital Impact Report (Philanthropy)

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From Possibility
to Reality

20 YEARS OF STEM CELL RESEARCH AT UCLA

Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA

Director's photo

Message from the Director

Dear friends,

As we celebrate our center’s 20th anniversary, I am enormously proud of all we have achieved and excited for what lies ahead. From paradigm-shifting technologies to innovative therapies, we are transforming the field of regenerative medicine from a realm of possibility into one of reality, bringing hope and healing to patients facing the most difficult health challenges.

A key area in which this transformation is having profound impacts is the science of aging. Stem cells will play a critical role in treating age related diseases as well as combating the effects of aging itself. In my own research, I have explored the mechanisms of aging stem cells to better understand how we can restore youthful function to aging cells and tissues. This work holds the promise of improving health span — enabling people to live longer, healthier lives.

Since joining as the center’s director in 2021, I have witnessed firsthand the vital role that philanthropy plays in propelling this kind of research forward. Your support allows us to push the boundaries of medicine, developing novel therapies to combat age-related conditions including Alzheimer’s disease, cancer, type 2 diabetes, and heart disease, which impact millions of people worldwide.

Philanthropy bridges the gap between inspiration and clinical application, turning bold ideas into real-world treatments. From funding early-stage research to building critical infrastructure, your investment is essential to our progress. None of this would be possible without visionary donors and partners like you who share our belief in the future we are creating.

As we look ahead, I invite you to consider expanding your support of our mission to create a future in which the most devastating diseases can be prevented, treated, and even cured. Together we can translate today’s discoveries into tomorrow’s life-saving therapies.

Thank you for being part of our journey.

Dr. Thomas A. Rando

Director, UCLA Broad Stem Cell Research Center

THE TRANSFORMATIVE POWER OF STEM CELL RESEARCH

Stem cell research and regenerative medicine are revolutionizing healthcare, creating unprecedented opportunities to repair, regenerate and heal the body.With cutting-edge technologies like artificial intelligence, gene and base editing, and 3D bioprinting driving progress, the potential for transformativetherapies is expanding like never before.

Stem cells, with their ability to self-renew indefinitely and give rise to any cell type, enable us to model complex disorders with extraordinary precision andtest new therapies, accelerating advancements in all areas of medicine. From correcting the causes of genetic diseases to rejuvenating aging tissues, theseinnovations are bringing once-distant possibilities within reach.

Together, we’re not just advancing medicine — we’re redefining it, turning discoveries into therapies that will reshape the future of human health.

Three Siblings, One Life-Saving Therapy
A Hub for Cross-Disciplinary Discovery
A Hub for Cross-Disciplinary Discovery
01 / 03

Our Center

The UCLA Broad Stem Cell Research Center was born from a bold idea: to bring together the brightest minds to transform the future of medicine throughstem cell science. Today, we’re realizing that vision by developing groundbreaking therapies that can prevent, treat and even cure diseases once thoughtincurable.

A Hub for Cross-Disciplinary Discovery

Established as a campus-wide initiative in 2005, our center transcends traditional academic barriers to support technology-driven, cross-disciplinary researchfrom fundamental discoveries to clinical applications. Our more than 250 faculty members pool their diverse expertise to develop innovative solutions totackle our most pressing health challenges — from extremely rare diseases to the age-related decline we will all experience.

Fueling Breakthroughs With Basic Science

At the heart of our mission is a deep commitment to basic science research. By studying how stem cells give rise to and repair our tissues throughout life, ourscientists are uncovering the intricate mechanisms that contribute to health and disease. This knowledge is serving as a foundation for next-generationtreatments for conditions including cancer, heart disease, infertility and autoimmune diseases.

Investing in Ideas, Innovators and the Next Generation

Our center has made extraordinary progress by investing in innovative ideas and infrastructure, focusing on training and education, recruiting preeminentfaculty and making an overarching commitment to collaboration. Every day, we’re finding new ways to remove roadblocks and ensure that breakthroughsmove swiftly from the lab to the patients who need them most.

"
The vision for the center was to create a hub where collaboration and innovation would drive breakthroughs in human health. Today, that vision continues toinspire as we advance stem cell research to deliver real-world solutions that improve lives and redefine the future of medicine.
"

Dr. Thomas A. Rando

OUR IMPACT BY THE NUMBERS

TRAINING THE NEXT GENERATION

0

young scientists including:

0

UCLA graduate students, postdocs and clinical fellows

0

UCLA undergraduate students

0

student interns from Cal State Northridge

CATALYZING RESEARCH

$0M+

distributed by our center to support UCLA research

0

new faculty recruited to UCLA

FACULTY OUTPUTS

0+

faculty members

0

clinical trials

0

patents for new therapies and technologies

0K+

publications

INNOVATION ECOSYSTEM

0

startups founded by our members

0K+

jobs created by these startups

$0.0B+

raised by these startups to support continued therapy development

Timeline

2005
Research impact
MARCH 2005
The center is founded under Dr. Owen Witte’s leadership, bringing together scientific, ethical, legal and policy experts from across UCLA to advance stem cell research.
2006
Research impact
SEPTEMBER 2006
Our Stem Cell Training Program welcomes its first cohort of graduate students, postdoctoral scholars and clinical fellows.
2007
Research impact
SEPTEMBER 2007
A $20 million donation from The Broad Foundation fuels cutting-edge research at the newly renamed Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.
2008
iPSC Creation
FEBRUARY 2008
Four recently recruited center faculty make history as the first in California to create human induced pluripotent stem cells.
2009
Research impact
FEBRUARY 2009
Dr. Donald Kohn begins a UCLA clinical trial of a stem cell gene therapy for the deadly immune deficiency ADA-SCID, commonly known as “bubble baby disease.”
2014
Research impact
OCTOBER 2014
The UCLA Health Alpha Clinic is founded to accelerate the delivery of cell and gene therapy clinical trials to patients.
2016
Research impact
SEPTEMBER 2016
Dr. Brigitte Gomperts uses stem cells to grow mini 3D lung organoids, revolutionizing the study of complex lung diseases.
2017
APRIL 2017
A discovery from the lab of Dr. Samantha Butler paves the way for methods to regenerate nerves in patients with spinal cord injuries.
Research impact
AUGUST 2017
Drs. Heather Christofk and William Lowry identify a way to activate hair follicle stem cells to stimulate hair growth.
2018
Research impact
OCTOBER 2018
A team led by Dr. Owen Witte discovers that aggressive prostate and lung cancers are driven by common mechanisms, opening a new avenue for treating deadly small cell neuroendocrine carcinomas.
2019
JANUARY 2019
UCLA scientists led by Dr. Gay Crooks develop an artificial thymic organoid platform that creates a renewable source of cancer-fighting T cells from blood stem cells.
OCTOBER 2019
Drs. Yvonne Chen and Sarah Larson launch a clinical trial of a groundbreaking dual-target CAR-T cell immunotherapy for lymphoma and leukemia.
2020
APRIL 2020
Center researchers combine their specialized expertise to advance COVID-19 diagnostic tests, treatments and vaccines.
2021
MAY 2021
Forty-eight of 50 babies treated for ADA-SCID in the trial are cured.
Research impact
JULY 2021
Dr. Thomas Rando, a renowned neurologist and stem cell biologist, is appointed director of the center.
2023
FEBRUARY 2023
Scientists led by Dr. Thomas Rando identify a method to rejuvenate old stem cells, pointing toward strategies to enhance the body's ability to recover from injuries as we age.
MAY 2023
Ten of 11 patients with lymphoma enrolled in the trial respond to treatment, with eight entering remission.
2024
Research impact
JUNE 2024
Dr. Hanna Mikkola identifies a protein that enables blood stem cells to self-renew in a lab dish, a critical step toward making life-saving blood stem cell transplants more accessible.
2025

The iPSC Revolution

In 2008, a landmark achievement put our young center on the global scientific map. Four newly recruited faculty members became the first in California —and among the first in the world — to create induced pluripotent stem cells (iPSCs) from human skin cells.

These cells have transformed biomedical research by allowing scientists to reprogram a patient’s skin or blood cells back to a pluripotent state and thenguide them to develop into any cell type. This breakthrough opened the door to an unlimited source of patient-specific cells for disease modeling, drugdiscovery and personalized cell therapies.

Scientists at our center and beyond are working to develop therapies that use iPSC-derived cells to replace cells and tissues — like those of the heart, brainand liver — that have been lost to injury, aging or disease.

Learn More

Collaborating for Cures

Gene Therapy Meets Nanotechnology

Cystic fibrosis (CF) is a life-threatening genetic disorder where thick mucus builds up in the lungs, causing infections and severe organ damage. Despitemedical advances, many patients face respiratory failure by their 30s or 40s.

Because CF results from mutations in a single gene, it’s a prime candidate for gene therapies designed to precisely repair faulty DNA. However, deliveringthese therapies deep into lung stem cells has long been a challenge — until now.

At our center, a team of pediatric physician-scientists is tackling this problem head-on. Lung disease expert Dr. Brigitte Gomperts, gene therapy pioneerDr. Donald Kohn and nanotechnologist Dr. Steven Jonas are developing a strategy using nanoparticle “containers” to deliver gene editing systems like CRISPRdirectly to hard-to-reach lung stem cells. The team hopes this therapy, delivered as an inhalable mist, will serve as a one-time curative treatment addressingthe root cause of CF.

As they propel this experimental treatment toward clinical trials, they’re also adapting their nanoparticle platform into a tool other stem cell scientists can useto reach target cells in conditions ranging from cancer to sickle cell disease.

Read More

Immunotherapy for Everyone

Each year, over 300,000 women worldwide are diagnosed with ovarian cancer. Even after surgery and chemotherapy, the disease often returns in a moreaggressive form, leaving patients in urgent need of new treatments.

CAR-T cell therapy, which engineers a patient’s immune cells to fight cancer, holds promise but must be customized for each patient, causing delays andincreasing costs.

To address this, immunotherapy expert Dr. Lili Yang developed an innovative approach to produce large quantities of CAR-equipped invariant natural killer T(CAR-NKT) cells from blood stem cells. Known as the “special forces” of the immune system, NKT cells are highly effective at fighting cancer and, crucially,compatible with any patient’s immune system.

Yang’s ultimate goal is for these CAR-NKT cells to be mass-produced and shipped to hospitals around the world, ensuring this therapy is readily available topatients as soon as they need it.

Now, Yang is teaming up with gynecologic surgeon Dr. Sanaz Memarzadeh, bioinformatician Dr. Matteo Pellegrini and biostatistician Dr. Jin Zhou to completethe extensive studies necessary to bring this therapy to a clinical trial for ovarian cancer.

Read More
Microscopy image of a mouse brain highlighting a biomarker for vascular dementia in green. Credit: Dr. Min Tian/Carmichael Lab (Note: this image has been animated for artistic effect)
Microscopy image of a mouse brain highlighting a biomarker for vascular dementia in green. Credit: Dr. Min Tian/Carmichael Lab (Note: this image has been animated for artistic effect)

Regenerating the Aging Brain

By 2050, dementia cases are expected to skyrocket to 139 million worldwide. Most people with dementia have a combination of Alzheimer’s disease andvascular dementia, which is caused by decreased blood flow to the brain.

Vascular dementia is one of the most significant unmet needs because it's so common and increasing in prevalence. After age 80, all of us will havethe brain lesions that are a hallmark of this condition, no matter how healthy we've lived.

Dr. S. Thomas Carmichael

Dr. S. Thomas Carmichael and his team have uncovered a gene system that could be the key to unlocking the brain’s natural ability to repair its owndamaged tissue. In studies using aged mice with vascular dementia, targeting this system with an experimental drug led to significant improvements inmemory and mobility. Carmichael is now working to bring this regenerative therapeutic strategy to a clinical trial in humans.

This breakthrough was made possible by a generous gift from Dave and Diane Steffy, whose support fueled innovative, interdisciplinaryresearch into brain aging. “No one has really explored regenerative medicine for vascular dementia,” Carmichael said. “The Steffys’ philanthropy enabled usto pursue this hugely ambitious goal.”

Where Discoveries Become Cures

Bringing breakthrough discoveries to patients requires more than just innovative ideas. It demands access to cutting-edge technology, highly skilled technicalteams and advanced manufacturing facilities — resources that individual labs can’t sustain on their own.

That’s where our center’s shared research cores and clinical translation infrastructure come in.

From advanced DNA sequencers to cleanroom facilities that meet stringent FDA standards for cell and gene therapy manufacturing, we provide the tools andexpertise to streamline research and eliminate roadblocks. These resources promote collaboration, increase efficiency and accelerate the development ofnew therapies.

Thanks to this infrastructure, groundbreaking therapies can be discovered, manufactured and tested in clinical trials — all right here on the UCLA campus.

Our Human Gene and Cell Therapy Facility has manufactured more than 300 cell and gene therapy products for patients in 25 clinical trials.

BRINGING THERAPIES TO PATIENTS

For 20 years, our scientists have been turning visionary research into real-world therapies that save lives.

From leading the first FDA-approved clinical trial using embryonic stem cell-derived cells to treat patients with macular degeneration, to testing apersonalized vaccine that boosts the effects of immunotherapy in people with lung cancer, we're finding new ways to treat the untreatable.

To reduce the time it takes for innovative treatments to reach patients, our members partner with industry, launch startups and leverage the UCLA HealthAlpha Clinic, which specializes in bringing cell and gene therapies to the clinic equitably.

The path from the laboratory to the clinic isn't unidirectional. Clinical trials yield insights that feed back into the lab, sparking new research questions andunlocking even greater possibilities.

Take Dr. Donald Kohn's groundbreaking work — he has spent more than 35 years perfecting a stem cell gene therapy for the rare immune system deficiencyknown as "bubble baby disease," giving more than 50 children healthy, functioning immune systems. Now, he's applying the insights gained from this work todevelop similar treatments for a host of rare blood disorders and collaborating on a CRISPR therapy for sickle cell disease.

With each trial, we're not just advancing science — we're creating a brighter future for human health.

Key:
Pre-clinicalClinical Trial Phase 1Clinical Trial Phase 2Clinical Trial Phase 3Applied for FDA Approval

DRUG THERAPIES

Drug to facilitate repair and prevent heart failure following heart attackPre-clinical

GENE THERAPIES

Blood stem cell gene therapy for ADA-SCIDClinical Trial Phase 3

Blood stem cell gene therapy for LAD-1Applied for FDA Approval

CRISPR for sickle cell diseaseClinical Trial Phase 1

Gene therapy for ornithine transcarbamylase deficiencyClinical Trial Phase 3

CELL THERAPIES

Ipsc-derived cell therapy for blinding retinal conditionsPre-clinical

Stem cell therapy for limbal stem cell deficiencyClinical Trial Phase 1

IMMUNOTHERAPIES

Dual-target CAR-T cell therapy for non-hodgkins B cell lymphoma or chronic lymphocytic leukemiaClinical Trial Phase 1

Dual-target CAR-T cell therapy for multiple myelomaPre-clinical

Training the Next Generation

Our center is committed to fostering the next generation of leaders in regenerative medicine through immersive, cross-disciplinary stem cell trainingprograms.

We offer a continuum of opportunities for UCLA undergraduates, graduate students, postdoctoral scholars and clinical fellows, as well as year-long paidinternships for California State University, Northridge students.

Trainees advance research under the mentorship of our world-class scientists and gain expertise in clinical translation, community outreach, ethics andscience communications.

The collaborative nature of our center ensures trainees emerge with the wide-ranging knowledge they’ll need to lead the future of regenerative medicine,driving life-changing innovations while helping build a more diverse and inclusive scientific community.

Explore More

From Trainee to Trailblazer

Dr. Ranmal Samarasinghe’s epilepsy research is driven by the patients he sees in the clinic, many of whom have no effective treatment options. “Epilepsy isnot a curable disease,” he explained. “Thirty percent of patients don’t respond to medications, and that hasn’t changed in 100 years.”

This lack of progress is largely due to the challenge of studying disease processes directly in living human brain tissue. Samarasinghe is addressing thisobstacle head-on through his work developing brain organoids, tiny 3D brain-like structures grown from human stem cells.

Three confocal images of brain organoids derived from human iPSCs. Credit: Samarasinghe Lab

When generated from the cells of patients, organoids offer unprecedented visibility into how neural networks form, function and go awry, which is critical for understanding how epilepsy develops and affects brain function. They also allow scientists to model various forms of the disease and can serve as a platform for testing personalized therapies.

Samarasinghe first learned to develop and refine these critical models while working in the lab of neurobiologist Dr. Bennett Novitch as a clinical fellow in ourStem Cell Training Program. These models have since become the backbone of his research program.

With Novitch’s support, Samarasinghe built a network of collaborators at UCLA and beyond who were eager to help him refine organoids to be more valuableand accurate tools in the study of epilepsy’s complex disease processes. The knowledge he gained and the connections he forged as a trainee laid thegroundwork for his own lab at UCLA.

A human iPSC-derived assembloid (composed of multiple brain region organoids fused together) showing deep layer neurons in red and green and astrocytesin green. Credit: Samarasinghe Lab

Today, he remains dedicated to making discoveries that will drive the development of real treatments for the millions of people living with epilepsy around theworld.

"
Resources like the training program attract future scientific leaders to UCLA and retain the interest of graduate students and postdocs in stem cell research.The center’s uniquely collaborative spirit then helps those young researchers make an impact — if you take the initiative, others are always willing to sharetheir expertise and resources.
"

Dr. Ranmal Samarasinghe

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01 / 07

Igniting a Passion for STEM

Since 2023, our Outreach Program has been forging connections between LA-area high school students and leading scientists at our center. Throughhands-on classroom visits and immersive field trips to UCLA, we aim to inspire students from all backgrounds to envision a future in STEM.

During these interactions, students learn how stem cells are used in life-saving therapies and see revolutionary technologies in action. They also receivepractical guidance on pursuing a science degree while gaining firsthand insight into the diverse opportunities a research career offers.

500+ HIGH SCHOOL STUDENTS REACHED THROUGH OUR OUTREACH PROGRAMS

"
Our students generally come from low-income families, so it's difficult for them to imagine themselves in certain professions because they're focused onsurviving instead of thriving. Having young people from backgrounds like theirs come talk to them during this pivotal time can open their eyes to all theopportunities available to them.
"

Sonia Perez, 10th grade biology teacher, East Los Angeles Renaissance Academy

Where Science Meets Life: Patient Stories

A Second Act: Hirotaka's Cancer Journey

In 2018, actor Hirotaka Matsunaga was preparing for his big break — a leading role in a play. Just weeks from opening night, however, he receiveddevastating news: his non-Hodgkin’s lymphoma had returned. Once again, the 39-year-old’s career — and life — was suddenly placed on hold.

After four years of unsuccessful treatments and recurrences, Matsunaga took a leap of faith and joined a UCLA clinical trial for a cutting-edge CAR-T celltherapy.

The novel therapy was a success, putting Matsunaga and seven other clinical trial participants into remission.

Study leaders Drs. Yvonne Chen and Sarah Larson are now adapting this dual-target CAR-T cell therapy to combat multiple myeloma and developingstrategies to ensure these innovative treatments are more accessible and affordable for patients.

Now free of symptoms, Matsunaga has returned to acting and is able to embrace new roles without the constant worry of relapse.

“I feel like myself again,” he said. “I hope my story offers comfort to others by letting them know you can live a fulfilling, exciting life after cancer.”

The Langenhop siblings attend a wedding

The Langenhop siblings attend a wedding

Ava Langenhop in the hospital for her stem cell transplant in May 2020

Ava Langenhop in the hospital for her stem cell transplant in May 2020

Landon Langenhop in the hospital for his transplant in September 2020

Landon Langenhop in the hospital for his transplant in September 2020

Olivia Langenhop in the hospital for her transplant in November 2020

Olivia Langenhop in the hospital for her transplant in November 2020

The Langenhop family in the UCLA sculpture garden in October 2020

The Langenhop family in the UCLA sculpture garden in October 2020

Olivia, Landon and Ava during a virtual meeting with the UCLA Bruins football team in December 2020

Olivia, Landon and Ava during a virtual meeting with the UCLA Bruins football team in December 2020

The Langenhop siblings visiting with Dr. Donald Kohn in 2024

The Langenhop siblings visiting with Dr. Donald Kohn in 2024

The Langenhop siblings dressed up for Halloween

The Langenhop siblings dressed up for Halloween

The Langenhops celebrating the holidays

The Langenhops celebrating the holidays

The Langenhop family at UCLA in 2024

The Langenhop family at UCLA in 2024

01 / 10

Three Siblings, One Life-Saving Therapy

When Jon and Alicia Langenhop learned that their three young children had the rare and deadly genetic disorder leukocyte adhesion deficiency-I (LAD-I), theirworld turned upside down.

The disorder — which affects about 1 in 1 million children worldwide — cripples the immune system, making even minor infections life-threatening. Withouttreatment, it’s often fatal by age two.

While the children battled constant infections, the Langenhops searched for bone marrow donors. A news story about the family’s plight caught the attentionof Dr. Donald Kohn, who was leading a clinical trial of a stem cell gene therapy for LAD-I.

In 2020, the siblings received the treatment, which replaced a faulty gene in their blood stem cells with a healthy copy. Since receiving this therapy, Ava, 9,Olivia, 7, and Landon, 5, have full immune system function and are able to do things that were once impossible, like attending school and playing outside.

The Langenhops feel immense gratitude for the medical breakthroughs that saved their children’s lives. “We’re so lucky to live in a time where science is atthis point,” Alicia shared.

A NEW ERA

Twenty years of tireless discovery have led us to this moment — where the horizon of possibility meets the reality of transformation.

We stand at the forefront of a new era of biologic medicine in which cell and gene therapies aren’t just promises, but real tools reshaping how we prevent,treat and cure disease.

This revolution is built on hard-won insights into the most foundational processes of life — from embryonic development to age-related decline.

By integrating artificial intelligence and machine learning, we’re accelerating discovery, uncovering hidden patterns of disease risk and unlocking newtherapies to enhance resilience.

With these advances in hand, we’re poised to deliver the right treatments to the right patients at the right time — fulfilling the promise of longer, healthierlives for all.

Join us on our journey