Experience a day in the life of a BSCRC trainee

Dr. Thomas A. Rando
Director, UCLA Broad Stem Cell Research Center
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.
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.
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.
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.
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.
Dr. Thomas A. Rando
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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.
Years later, the same four scientists who combined their diverse expertise to make this discovery continue to leverage iPSCs in their labs — leading majoradvances that are shaping their fields.
focuses on the biology of human reproduction, aiming to develop treatments for infertility and other reproductive disorders.
studies how healthy muscle forms to create gene and cell therapies for Duchenne muscular dystrophy and other neuromuscular diseases.
explores the epigenetic mechanisms that regulate stem cell fate, which could enhance these cells’ utility in research and medicine.
investigates the molecular processes behind cell fate decisions in development and cancer, aiming to develop therapies for a broad range of conditions.
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.
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.
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.”
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.
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.
Drug to facilitate repair and prevent heart failure following heart attackPre-clinical
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
Ipsc-derived cell therapy for blinding retinal conditionsPre-clinical
Stem cell therapy for limbal stem cell deficiencyClinical Trial Phase 1
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
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.
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
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.
Dr. Ranmal Samarasinghe
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
Sonia Perez, 10th grade biology teacher, East Los Angeles Renaissance Academy
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.”
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.
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.