The UCSF CIRM Scholars Research Program is designed to equip the next generation of basic and clinical scientists with the expertise and motivation to make major strides in the fields of regenerative medicine, stem cell research, and gene therapy. To achieve this goal and fill the gap of needed experts in these fields in California requires robust training at multiple levels of education. This past year, the program selected four graduate students, seven post-doctoral fellows, and two clinical fellows from a large and highly competitive pool of applicants. Applicants were selected based on their prior work, proposed research, and potential for impact on the stem cell, gene therapy, and regenerative medicine fields. The selected applicants represent a diverse pool of backgrounds and specialties from stem cells to tissue engineering to gene editing all working toward a shared goal of improving human health. They each have established well thought out research and training programs under the guidance of strong and committed mentors. In addition to their individualized research plans, the trainees have been engaged in a series of community and patient oriented activities. For example, trainees have given presentations to local high school students, participated in the Bay Area Science Fair, and put on first UCSF "CIRM Days" aimed at educating and engaging high school students and undergraduates in the possibilities of stem cells and gene editing in improving human health. The trainees have also had the opportunity to directly engage with patients and their doctors who have been treated with or used stem cell/ gene editing based therapies to tackle specific diseases such as sickle cell , alpha thalassemia, severe combined immunodeficiency, and cancer. Research wise, the trainees have made much progress in their projects, which is shared with the other trainees and their mentors in a monthly research-in-progress meeting. The trainees are making inroads into areas such as engineering liver tissue, converting unhealthy fat to healthy fat, developing new cell therapies to target leukemia, engineering islets for treatment of diabetes, developing tumor cellular vaccines, improving genetic editing approaches, and much more. The trainees have also taken individualized courses specific to their research and attended conferences to both present their work as well as keep abreast of the latest conceptual, technical, and translational advances in the field. For example, many of the trainees attended and presented at the annual International Society of Stem Cell Research meeting this past summer. While we are only in the first year of the program, we are highly enthusiastic of the progress made by our trainees. They are well on the path to be future leaders in the stem cell/ gene therapy fields with great potential to positively impact patient care.
Reporting Period:
Year 2
The UCSF CIRM Scholars Research Program aims to support the next generation of basic and clinical scientists in their journey toward advancing in the fields of regenerative medicine, stem cell research, and gene therapy. To achieve this goal and generate these experts for the advancement of science in California, we must support trainees at multiple levels of education. This past year, the program selected one clinical fellow and two postdoctoral fellows from a highly competitive pool of applicants. Our trainees were ultimately selected based on their prior work, proposed research, and potential for impact on the fields of stem cell, gene therapy, and regenerative medicine. The cohort for this year combines these new candidates with our trainees continuing from the first year of the grant.
Overall, our cohort this year represents a diverse pool of backgrounds and specialties from stem cells to tissue engineering to gene editing all working toward a shared goal of improving human health. They each have established, rigorous research and training programs under the guidance of committed mentors. In addition to their individualized research plans, the trainees have been engaged in a series of community and patient oriented activities. For example, our trainees have independently mentored and tutored to students from backgrounds underrepresented in the sciences at multiple education levels (from high school to masters students). A majority of our trainees participated in UCSF’s “CIRM Days,” offering tours and talks aimed illuminating the possibilities of stem cell and gene editing therapies to high school students and undergraduate students. The trainees have also had the opportunity to directly engage with physicians and patients about their experience with stem cell/ gene editing based therapies for diseases such as sickle cell , alpha thalassemia, severe combined immunodeficiency, and cancer.
In their second year, the trainees have persistently made impressive strides in their research projects that model human diseases and devise innovative therapeutic strategies and tools to combat these conditions. Collectively, they are addressing a wide spectrum of diseases including leukemia, retinal disorders, diabetes, neural degeneration, liver insufficiencies, and obesity. Research progress was shared among the entire cohort and their mentors in a monthly Research-In-Progress (RIPS) meeting. The trainees have also taken individualized courses specific to their research and attended conferences to both present their work as well as keep abreast of the latest conceptual, technical, and translational advances in the field. Three of the trainees who have recently left our training program will start or have already started faculty positions at other institutions, including UT Austin and UC San Diego. Two of our trainees have developed inventions from their research and one has published a paper in Cell Stem Cell. We excited to see the progress of this year and future cohorts as the grant progresses.
Grant Application Details
Application Title:
Scholars Research Training Program in Regenerative Medicine, Gene Therapy, and Stem Cell Research
Statement of Benefit to California:
We envision that the citizens of the state of California will benefit in many ways from our university’s proposed Scholars Training Program for graduate students, postdoctoral fellows, and clinical scientists. Collectively, the basic research, translational strategies, and clinical therapies that emerge from the work of our university’s California Institute for Regenerative Medicine (CIRM) funded trainees will be an important stimulus to the state economy, particularly the biotechnology sector and associated medical enterprises. Additionally, specific groups of individuals will directly benefit from work that is focused on cell-based therapies for repairing tissues and organs whose damage leads to common medical conditions, for example, diabetes, cardiovascular disease, Parkinson’s disease, paralysis and/or immune dysfunction. On the way to achieving the CIRM’s ultimate goals in terms of novel regenerative therapies for patients, we envision that numerous other benefits will emerge. For example, human embryonic stem cell (hESC) systems are powerful tools for unraveling the molecular basis of human development, which remains largely a black box. A fundamental lack of understanding regarding the mechanisms that give rise to the hundreds of cell types that form tissues and organs makes it extremely difficult to discern why these processes sometimes go awry, leading to birth defects and/or setting the stage for many diseases. Additionally, novel therapies for other medical conditions are also likely to emerge. In this regard, some forms of cancer are now thought to be associated with the proliferation of stem cells that carry mutations in genes that promote their self-renewal, rather than differentiation and integration into the compartment that they normally occupy. Other important applications include drug development. For example, hESCs and their differentiated progeny could be used to screen promising compounds for efficacy, safety and/or toxicity. Where will the workforce come from that will enable this revolution in how the medical establishment approaches patient care? Stem cell and gene therapy is a rapidly growing field that must be rapidly populated with scientists and clinicians who are specially trained in all aspects of regenerative medicine, a new specialty. This necessity makes the funding of CIRM-sponsored training programs especially critical for institutions such as ours that have the ability to make important research discoveries and translate them into clinical therapies. Our university has a long and distinguished history of training leaders in science and/or medicine who easily traverse the boundaries between academia and industry. Our past successes strongly suggest that our CIRM-funded training programs will be equally successful. Accordingly, we expect that our trainees will become leaders in the field.
