Training Scholars in Regenerative Medicine and Stem Cell Research

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Grant Award Details

Grant Type:

Research Training Grant

Grant Number:

EDUC4-12811

Investigator(s):

Name:

Peter G. Schultz

Institution:

Scripps Research Institute

Type:

Award Value:

$5,304,759

Status:

Active

Progress Reports

Reporting Period:

Year 1

Nominations for the training grant were solicited October 2021. There was a high degree of interest, and the quality of pre- and postdoctoral applicants was very high across a diversity of scientific disciplines. Six pre-doctoral students and six postdoctoral students were appointed from the applicant pool. The trainees engaged in the rigorous educational elements of the program, including a multi-institutional team-taught Core Course that provided current information in stem cell biology, medicine, and ethics. The trainees participated in the core course, building scholarly interactions across institutions. They also participated in and presented at a multi-institutional retreat in December 2022 that encourages scholarly interaction between colleagues within the Sanford Consortium for Regenerative Medicine (a CIRM major facility). Trainees also engaged the broader San Diego community by participating in K12 outreach programs and mentoring high school and undergraduate summer interns. In their first year in the program, trainees made significant scientific progress in projects to advance the fields of stem cell biology and regenerative medicine. They made strides toward identifying strategies and therapies to differentiate neural progenitor cells to treat multiple sclerosis, regenerate heart tissue after heart failure, regenerate pancreatic beta cells for diabetes, and expand progenitor cells for lung and intestinal diseases. They also made significant progress in oncology, investigating the role of natural killer cells in allogeneic CAR-T cells for cancer cell therapy and identifying therapies that prevent tumor progression through suppression of cancer stem cells. Other trainees studied stem cells in the context of neurodevelopmental and neurodegenerative disorders. Through this program, trainees are not only developing advanced skills to pursue future careers in stem cell research and regenerative medicine, but they are also conducting fundamental and applied research that has potential to address critical unmet medical needs impacting millions.

Reporting Period:

Year 2

Reporting Period:

Year 3

Our trainees have continued to engage in the rigorous educational elements of the program. We welcomed several new postdoctoral trainees during our previous reporting period, who have been given the option to take the multi-institutional team-taught Core Course their predecessors have already taken, which provided current information in stem cell biology, medicine, and ethics. The trainees will also participate in and present at a multi-institutional retreat in December 2024 that encourages scholarly interaction between colleagues within the Sanford Consortium for Regenerative Medicine (a CIRM major facility) and UCSD. Trainees also engaged the broader San Diego community by participating in K12 outreach programs and mentoring high school and undergraduate summer interns. Our trainees have made significant scientific progress with their studies aimed at advancing the fields of stem cell biology and regenerative medicine. Specifically, they have made progress identifying and studying small molecules that target and function in stem cell populations to modulate diverse outcomes. In the context of tissue regeneration, trainees have identified or are studying molecules that target endogenous stem cell populations to promote regeneration and repair of cartilage and related tissues for the treatment of osteoarthritis; expand human retinal pigment epithelial cells for repair of retinal tissue in the context of age-related macular degeneration (AMD); or induce the regeneration or expansion of human β-cells for the treatment of diabetes. Trainees are also studying the role of the stem cell “niche” in the context of failed regenerative capacity in aging. Here, the role of mechano-sensing in stem cell activation is being explored, with the ultimate goal of identifying small molecules capable of modulating these processes for diverse regenerative medicine-related applications. Multiple efforts are ongoing aimed to studying and modulating the Hippo-YAP pathway, which plays critical roles in diverse stem cell-related physiologies. Here, two defined protein modifications are being studied in the context of cancer stem cells or cellular regeneration, and existing activators of the pathway are being studied in diverse contexts of tissue regeneration. Finally, the ability of an endogenous CNS-resident stem cell population to function in the context of adaptive immunity is being studied, and genetic screens involving blood stem cells in living mice are being conducted with the goal of identifying new ways facilitate macrophage-dependent tissue regeneration. Through this program, trainees are not only developing advanced skills to pursue future careers in stem cell research and regenerative medicine, but they are also conducting fundamental and applied research that has potential to address critical unmet medical needs impacting millions.

Grant Application Details

Application Title:

Training Scholars in Regenerative Medicine and Stem Cell Research

Public Abstract:

The goal of the program is to give trainees at The Scripps Research Institute (TSRI) a well-rounded understanding of regenerative medicine approaches, stem cell biology, and their clinical and ethical implications. The activities enabled by this training program will provide trainees a unique approach and set of tools to subsequently pursue regenerative medicine and stem cell biology in basic research, biotechnology or pharmaceutical enterprises. This training will lower future barriers for graduate students and postdoctoral fellows to engage in regenerative medicine research.

This program will be interdisciplinary in nature and will expand the existing postdoctoral and graduate programs at TSRI with training on understanding and controlling the cellular processes involved in human embryonic and adult stem cell self-renewal and differentiation, their application for expanding our mechanistic understanding of disease, the development of novel drugs to target those pathways, and ethical considerations. Trainees will also engage in a variety of outreach activities to help educate and inspire the broader San Diego community (K-12 students and undergraduates) on topics relating to regenerative medicine, with the goal of reducing disparities and disproportionate access to science.

Predoctoral trainees will be fully integrated with the Skaggs Graduate School, an interdisciplinary program that provides rigorous training in chemistry, chemical biology, synthetic biology, neurosciences, immunology, cell biology, and biophysics. The Doctoral Program in Chemical and Biological Sciences has ranked top-10 in chemistry and biology for the last 20 years (U.S. World and News).

CIRM trainees will have access to the full spectrum of research at TSRI. Uniquely, trainees will have the opportunity to collaborate with Calibr, TSRI’s drug discovery division, to take their scientific discoveries from the bench to the bedside. Though this process, they will gain exposure to all the challenges and opportunities of developing stem cell-based therapies and advancing them into the clinic, where they will gain an understanding of patient needs and contribute to the development of innovative new therapies for regenerative medicine in numerous diseases settings.

The CIRM EDUC4 training program will augment our existing training programs with access to courses and support for advancing regenerative medicine and include collaborative research projects between chemistry and biology graduate students and postdoctoral fellows. Such collaborations will provide a unique opportunity for trainees to assimilate the tools and approaches of both chemistry and biology in the context of important problems in stem cell biology. These joint projects may include chemists and biologists from neighboring institutions including UCSD, Salk, Sanford Burnham and the La Jolla Institute for Immunology, which, because of their close proximity, allow frequent and direct interactions.

Statement of Benefit to California:

The proposed research and training program will provide interdisciplinary training for a diverse group of graduate and postdoctoral fellows in regenerative medicine with the goal of advancing stem cell research and increasing the number of scientific leaders and researchers in stem cell research. The program will give trainees a well-rounded understanding of regenerative medicine approaches, stem cell biology, therapeutic development, and their clinical and ethical implications. Trainees will not only gain experience in chemistry and biology, but also have the opportunity to engage with translational research and precision and digital medicine. The activities enabled by this training program will provide trainees a unique approach and set of tools with which to subsequently pursue regenerative medicine and stem cell biology in basic research, biotechnology or pharmaceutical enterprises. Trainees will engage in a variety of outreach activities to educate the local community on topics relating to regenerative medicine, with the goal of reducing disparities and disproportionate access to science. We expect that these trainees will become the future scientific leaders and researchers in the field of regenerative medicine who will address critical unmet medical needs using stem cell and regenerative medicine-based therapies for the benefit of the public. The State of California will benefit not only from the scientific and therapeutic advances that trainees will make, but also the cultivation of talented young people in the field and development of a well-trained workforce.

Publications

RSC Chem Biol (2023): A covalent inhibitor of the YAP-TEAD transcriptional complex identified by high-throughput screening. (PubMed: 37920398)
bioRxiv (2023): Pharmacological inhibition of CLK2 activates YAP by promoting alternative splicing of AMOTL2. (PubMed: 37131806)

Training Scholars in Regenerative Medicine and Stem Cell Research

Grant Award Details

Progress Reports

Grant Application Details

Training Scholars in Regenerative Medicine and Stem Cell Research

Publications

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