The CDCM bridges the divide between laboratory discovery and translation to patients in clinical trials
What is CDCM?
The Center for Definitive and Curative Medicine (CDCM) is the translational arm of the Institute for Stem Cell Biology and Regenerative Medicine. The CDCM was established as a joint initiative of the Stanford University School of Medicine, Stanford Health Care and Stanford Children’s Health. It is co-directed by Drs. Maria Grazia Roncarolo, Anthony Oro, and Matthew Porteus.
The CDCM provides the know-how, organizational and physical infrastructure to support investigator-initiated clinical translational studies on cell and gene therapy (CGT) from initial discovery through completion of clinical proof-of-concept trials. Stanford Medicine’s clinical enterprise provides an exemplary clinical environment in which to deploy cures. The CDCM supports the development of life-changing and curative treatments for patients who come to Stanford to receive the highest level of care.
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Why Stanford University?
A number of distinguishing features make Stanford particularly well-positioned to succeed
Pre-eminent, highly-funded cell and gene therapy program
Significant investment in translational infrastructure on campus
State-of-the-art GMP facility
Excellence in clinical care and access to patient populations
Dr. Rajni Agarwal-Hashmi Awarded the Fanconi Anemia Research Fund (FARF)'s Lifetime Achievement Award
October 1, 2023
Over the weekend of September 28 – October 1, Stanford’s Dr. Rajni Agarwal-Hashmi was awarded the Fanconi Anemia Research Fund (FARF) Lifetime Achievement Award at their 2023 Scientific Symposium. For more than 35 years of her career, she has been dedicated to the improvement of the care of patients with Fanconi Anemia (FA), including spearheading impactful and innovative clinical treatment strategies that have led to meaningful advances in the quality of life for FA patients and their families.
Dr. Agarwal-Hashmi’s work and diligence toward the care of FA is exemplified by the 70+ patients, that have come from all over the nation, treated by her at Stanford Children’s. Recognizing the extreme sensitivity that FA patients deal with regarding conventional pre-transplant conditioning strategies, she has pioneered efforts to advance non-toxic antibody-based conditioning which challenges the status quo. Specifically, her leadership in a first-in-human trial for utilizing non-toxic-antibody-based conditioning over genotoxic irradiation and busulfan is field-defining. This is the first-time this antibody is being combined with immunosuppression and alpha/beta T-cell depleted haploidentical grafts, the later providing greater access to donors as well as decreasing non-transplant related morbidities
Dr. Agarwal-Hashmi’s impact on the FA field is unequivocally vital toward the advancement and improvement of treatments and patient care. Her leadership and innovation in the field are reflected in her reception of this prestigious FARF honor.
South Korean Minister of Health Visits CDCM to Learn More About Stanford's Cell and Gene Therapies
July 7, 2023
Following a visit by the Samsung Medical Center’s Cell and Gene Therapy Institute (CGTI) in April, the CDCM hosted the South Korean Minister of Health and Welfare, Kyoo Hong Cho, on July 7, 2023. The purpose of the visit was to learn more about Stanford’s cell and gene therapy activities, including research, clinical care, and training.
During Minister Cho's visit, he met with Lloyd Minor, MD, Dean of the School of Medicine; David Entwistle, President and CEO of Stanford Health Care; Paul King, President and CEO of Stanford Medicine Children's Health; and Matthew Porteus, MD, PhD, Director of the CDCM. They discussed Stanford Medicine's advanced practices in various practices such as precision medicine through cell and gene therapy, the Medical Scientist Training Program (MSTP), and the Clinical and Translation Science Awards (CTSA). Opportunities for future collaborations between Stanford Medicine and research-focused hospitals in Korea were also explored.
In total, 17 people from the Korean Embassy, the Korea Health Industry Development Institute (KHIDI), and the CEOs of the Korea Drug Development Fund (KDDF) and Korea Medical Device Development Fund (KMDF) etc. came to the visit.
CIRM Awards Stanford University $2,000,ooo in their First Phase of Building California Cell & Gene Therapy Manufacturing Network
May 31, 2023
The California Institute for Regenerative Medicine (CIRM) awarded Stanford University $2,000,000 as part of their $10,000,000 Phase One Initiative to build a California Cell and Gene Therapy Manufacturing Network. Steven Feldman, PhD, was the recipient of this award with his work in Stanford's LCGM and his INFR5-14663 application. The full program title is the "Laboratory for Cell and Gene Medicine: A Partner in the California Cell and Gene Therapy Manufacturing Network".
You can read more here.
Stanford Medicine Children's Health Receives "Rare Disease Centers of Excellence" Designation
May 11, 2023
The National Organization for Rare Disease (NORD) has named Stanford Medicine Children's Health as one of its "Rare Disease Centers of Excellence", which are dedicated to diagnosing, treating, and researching all rare diseases. 40 hospitals nationwide make up this healthcare network that is at the forefront of cutting edge scientifc/medical breakthroughs and cares for thousands of rare disease patients.
Natalia Gomez-Ospina, MD, PhD will serve as the director for Stanford Children's NORD Center of Excellence for Rare Disorders. You can read more here.
CIRM Awards Stanford University $11,813,964 for their CLIN2-14315 Program headed by Matthew Porteus, MD, PhD
April 27, 2023
Principal investigator Matthew Porteus, MD, PhD, along with fellow investigators Rajni Agarwal, MD and Agnieszka Czechowicz, MD, PhD were awarded $11,813,964 by The California Institute for Regenerative Medicine (CIRM) for their CLIN2-14315 program. The program's full title is "Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients". You can read more about the awards here.
Other active CIRM grants:
- Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy - $6,056,713 (TRAN1-14609)
- Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome) - $5,444,353 (CLIN1-13988)
- Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant - $11,998,188 (CLIN2-14024)
- Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome - $11,999,179 (CLIN2-13259)
- Phase 1/1b study of T-allo10 infusion after HLA-partially matched abdepleted-HSCT in children and young adults with hematologic malignancies - $10,563,822 (CLIN2-12563)
Natalia Gomez-Ospina, MD, PhD Awarded $5,444,353 for Late-Stage Preclinical Program by CIRM
January 20, 2023
Stanford University's Natalia Gomez-Ospina, MD, PhD and her team were awarded $5,444,353 by The California Institute for Regenerative Medicine (CIRM) for their late-stage preclinical trial targeting Hurler Syndrome. Hurler Syndrome, caused by a faulty gene, is an inherited condition that results in the lack of an enzyme needed to digest sugar. Undigested sugar then builds up in the body and causes progessive damage to the brain, heart, and other organs.
The goal of this preclinical program is to showcase the team's capability of manufacturing the necessary stem cells to restore the missing enzyme while completing safety studies and applying clinical trial authorization from the FDA. You can read more here.
Joseph C. Wu, MD, PhD Named a Fellow of National Academy of Inventors
December 13, 2022
Stanford University's Joseph C. Wu, MD, PhD was among three faculty that were named Fellows of the National Academy of Inventors. This honor is the highest professional distinction awarded to academic inventors. Dr. Joseph C. Wu was recognized for his lab's work "on biological mechanisms of patient-specific and disease-specific induced pluripotent stem cells." You can read more here.
The National Academy of Inventors states that the 2022 Fellow class is comprised of 110 research universities and governmental and nonprofit research institutions globally. As a whole, the 2022 class holds over 5,000 U.S. patents.
Stanford-born Therapy Reaches Clinical Milestone
November 3, 2022, New York and Cleveland
Abeona Therapeutics has announced positive data in its Phase 3 VIITAL study, which assesses the safety and efficacy of EB-101 for treatment of recessive dystrophic epidermolysis bullosa (RDEB). The study met its two co-efficacy endpoints showing statistically significant, clinically meaningful improvements in healing wounds and reducing pain for large chronic RDEB wounds.
The Phase 3 VIITAL study evaluated the safety, tolerability, and efficacy of EB-101 in 43 large wound pairs across 11 RDEB subjects. At six months, EB-101 significantly improved wound healing and pain reduction when compared to the control.
EB-101 was also shown to be tolerated well by the subjects, with no serious treatment-related adverse events observed. Stanford University’s Jean Tang, M.D., Ph.D, the Principal Investigator of the study, states that while EB-101 has been shown to significantly heal and reduce pain, the Phase 1/2a study will continue to monitor its clinical benefits with up to eight years of follow-up.
Following the positive study results, Abeona plans to submit a Biologics License Application (BLA) for EB-101 to the FDA for approval.
Join us at the 7th Annual CDCM Symposium to hear more about this therapy’s journey from bench to bedside!
Registration for the CDCM 7th Annual Symposium is now Open!
We look forward to welcoming everyone back to an in-person format after 2 years of virtual conferences!
Anthony Oro, MD, PhD Elected to the National Academy of Medicine
October 24, 2022
Stanford University's Anthony Oro, MD, PhD was among 100 members newly elected to the National Academy of Medicine for "solidifying the first link between Hedgehog signaling and human cancer and building chromatin maps identifying how environmental factors drive tumor epigenetic plasticity and drug-resistance."
Dr. Anthony Oro was among 6 professors at Stanford University to be elected to the National Academy of Medicine, which recognized individuals that have "made major contributions to the advancement of the medical sciences, health care and public health." You can find more information here.
Natalia Gomez-Ospina, MD, PhD Named as a Grantee in the Science Diversity Leadership Program
October 20, 2022
Stanford University's Natalia Gomez-Ospina, MD, PhD has been awarded a grant from the Science Diversity Leadership program for her work in Targeting Macrophage/Microglia Niches with Engineered Stem Cells to Treat Disease. Her work "aims to develop novel conditioning tools that support the safe, efficient, and rapid repopulation of macrophage/microglia niches by bone marrow-derived cells and use them to treat metabolic diseases."
The Chan Zuckerberg Initiative partnered with the National Academies of Science, Engineering, and Medicine to launch the Science Diversity Leadership Program. This program is a funding initiative that recognizes biomedical researchers and awards them grants to further their leadership in "promoting diversity, equity and inclusion in their scientific fields." You can click here for more information.
Registration for the CDCM's 7th Annual Symposium will be Opening Soon
September 13, 2022
Please visit the event's webpage for updates/more information!
Ginger Exley Receives the Donna Schurr Spirit Award
June 30, 2022
The CDCM would like to congratulate and recognize Ginger Exley for receiving the Donna Schurr Spirit Award last Friday. This staff honor is awarded to individuals based on faculty and staff nominations. Everyday, Ginger works to support the CDCM in many administrative facets, which help build the foundation for our work. As such, Ginger is a vital component of the CDCM and instrumental in ensuring that the department operates as smoothly as possible. Thank you Ginger for tirelessly supporting the CDCM and all of us! We greatly appreciate all of your contributions!
Jasper Therapeutics Announces Treatment of First Patient in JSP191 Conditioning Study
May 17, 2022
The CDCM's collaboration with Jasper Therapeutics has resulted in the successful launch of an investigator-initiated clinical trial treating patients with Fanconi Anemia. Read the latest news here.
CDCM 6th Annual Symposium Recap
March 29, 2022
Every year, the Center for Definitive and Curative Medicine (CDCM) hosts a symposium gathering experts from gene and cell therapy fields to discuss their new discoveries, clinical development, and challenges in bringing treatments to patients with unmet needs. This year’s symposium “Delivering on Curative Promises” featured 10 distinguished speakers and drew 600+ registrants, cementing another successful event from the CDCM. We received an overwhelming amount of positive feedback from attendees, Stanford University leadership, industry partners, hospital leadership, and our sponsors for arranging a day that was educational, scientifically cutting edge, versatile, entertaining, and for most attendees, deeply meaningful.
This year’s keynote speakers were:
- Marina Cavazzana, MD, Director of Biotherapy Department - Hôpital Necker Enfants Malades - Paris – France
- Irv Weissman, MD, Director, Institute of Stem Cell Biology and Regenerative Medicine, Stanford University
We would like to thank all participants, speakers, and sponsors for their continued support of the CDCM, the goal of translating Stanford University discoveries to cures for patients with both rare and common diseases, and the development of a unique infrastructure to achieve this goal.
We look forward to the 7th annual symposia and hope you will be able to join us again.
The symposium is an annual conference put on by the CDCM (http://med.stanford.edu/cdcm.html). The Center provides the organizational and physical infrastructure to support investigator-initiated clinical translational studies in stem cell and gene therapy from initial discovery through completion of clinical proof-of-concept studies.
Rosa Bacchetta, MD Receives $12 Million in CIRM Funding for IPEX clinical trial
Oakland, February 24, 2022
The California Institute for Regenerative Medicine (CIRM) has invested approximately $12 million in a therapy being tested in a clinical trial to help patients that are affected by IPEX Syndrome. IPEX Syndrome is a rare autoimmune condition where an individual’s body cannot control their immune responses; this results in immune cells attacking their own healthy tissue. It is more commonly diagnosed in boys during the first year of life and is often fatal.
Dr. Rosa Bacchetta and her team have developed a therapy that utilizes a patient’s own CD4 T cells, which have been genetically modified to properly express the FOXP3 gene. Because the cells originate from the patient, there are less concerns regarding potential rejection and other fatal transplant complications. Dr. Bacchetta has received approval from the FDA to test this therapy in a Phase 1 clinical trial for affected patients (NCT05241444).
IND Clearance Received for Sickle Cell Disease Gene Editing Clinical Trial
December 14, 2020
FDA Investigational New Drug (IND) clearance has been received to launch a phase I/II clinical trial of gene correction for sickle cell disease.
California Votes Yes on Proposition 14: California Stem Cell Research, Treatments and Cures Initiative of 2020
November 13, 2020
California voters vote Yes on Prop 14! This proposition provides needed funding to continue investing in the research and discovery of stem cell treatments and cures for chronic illnesses and conditions.
Dr. Porteus' article featured in a Nature Communications Editors’ Highlights webpage
May 18, 2020
The editors at Nature Communications have put together an Editors’ Highlights webpage of recent research on Therapeutics and have chosen to feature Dr. Porteus' article, entitled “Cas9-AAV6-Engineered Human Mesenchymal Stromal Cells Improved Cutaneous Wound Healing in Diabetic Mice”.