San Diego, CA, August 13, 2008 --Bioengineering Professor Shyni Varghese is one of five UC San Diego researchers and physicians awarded New Faculty grants from the California Institute for Regenerative Medicine (CIRM). The new awards, announced today, add $11.5 million to the more than $20 million in funding that researchers at UC San Diego have received from CIRM.
|Bioengineering Professor Shyni Varghese’s $2.3M grant from the California Institute for Regenerative Medicine will enable her to explore embryonic stem cell-based transplantation therapy for treating muscle wasting.|
“These grants demonstrate CIRM’s strong commitment to the dynamic and innovative careers of these young faculty members,” said Larry Goldstein, Ph.D., director of UC San Diego’s stem cell program. “This investment in the future of the next generation of ‘big thinkers’ is especially critical in an atmosphere of tight funding, allowing institutions like UC San Diego to mount broad programs that will improve health outcomes for Californians and patients around the world.”
Varghese will focus on the most common form of muscle wasting, Duchenne muscular dystrophy (DMD), which is characterized by progressive skeletal muscle degeneration in young children. She plans to optimize the micro-environmental factors that contribute to differentiation in the embryonic stem cells, then engineer a population of clinically viable ESC-derived muscle progenitor cells.
“We will investigate differentiation of our embryonic stem cells in a three-dimensional environment, in order to derive progenitor cells that can be used to test therapies for treating Duchenne muscular dystrophy using animal models,” said Varghese. Successful completion of this study could offer broad applicability of stem cell-based therapies for treating various types of muscle-wasting diseases.
The five UC San Diego researchers represent diversity in background and research focus, which ranges from maternal and infant health to cancer and skin disease. They are among the 12 scientists and 11 physician scientists in California that will receive New Faculty awards, which fund promising M.D. and Ph.D. scientists in the critical early stages of their careers as independent investigators. A total of $59 million in funding for 23 researchers was approved at today’s meeting of 29-member Independent Citizens Oversight Committee (ICOC).
The grantees were chosen from 55 applications received by CIRM from 32 institutions. Information on the research project of the other four UC San Diego-affiliated researchers is below.
Catriona Jamieson, M.D., Ph.D., assistant professor of medicine and Director for Stem Cell Research at Moores UCSD Cancer Center ($3,065,572)
The aim of Jamieson’s research is to develop highly active stem cell therapy that may halt progression to acute leukemia in a form of blood cancer called myeloproliferative disorders (MPDs.) A patient with MPD makes too many blood cells, caused by a mutation expressed in the stem cell, the early stage cell that goes on to differentiate to become either red or white blood cells.
“Because human embryonic stem cells have robust self-renewal capacity and can provide a potentially limitless source of tissue-specific stem and progenitor cells, they represent an ideal model system for generating and characterizing human MPD stem cells,” said Jamieson. “This research harbors tremendous potential for developing life-saving therapy for cancer patients by providing a new platform to rapidly test new therapies to target cancer stem cells.”
Human clinical trials for an inhibitor that can stop the over-proliferation of blood cells – developed as a result of collaborative discoveries by Jamieson, along with other stem cell researchers from UC San Diego, Dana-Farber Cancer Institute, the Mayo Clinic and a San Diego pharmaceutical company, TargeGen – are currently being conducted at major research institutions across the country, including UC San Diego.
Mana Parast, M.D., Ph.D., assistant professor in UC San Diego School of Medicine’s Department of Pathology ($3,078,580)
Parast’s research targets the placenta for stem cell therapy, looking at generating trophoblast stem (TS) cells, the primary cell type that carries out major functions of the placenta such as establishing blood supply from mother to fetus. The trophoblast “shell” that becomes the placenta comprises a second type of stem cell in the human blastocyst, along with the embryonic stem cells that become the embryo and give rise to adult organs. Abnormal development and function of the placenta can lead to premature or preterm birth – the leading cause of neonatal death in the United States. Parast aims to prevent such problems before they start, in utero, by treating the placenta. Her work will identify specific trophoblast markers that could be developed into diagnostic tools and eventually lead to TS-based therapeutics for in-utero interventions, designed to decrease such complications.
Benjamin Yu, M.D., Ph.D., assistant professor in the Division of Dermatology in the UC San Diego School of Medicine ($3,056,649)
Yu’s research focuses on adult stem cells that are already present in our bodies but for unknown reasons lie dormant. A major obstacle to applying adult stem cells to regenerative therapies is that these cells are often too limited in number to study and are difficult to grow. The aim of his research is to identify the mechanisms that control the proliferation and fate of adult stem cells.
As a model to study adult stem cells, Yu studies the hair follicle, an organ that regenerates itself many times during a lifetime. He has helped develop an approach to manipulate the proliferation of adult stem cells in the hair follicle using cell-permeable proteins.
"By developing non-genetic approaches to manipulate adult stem cell proliferation and differentiation in the hair follicle, we hope to apply these molecular tools to adult stem cells in other organs," said Yu. "This research might be used someday to stimulate regeneration in our own bodies without risking permanent genetic changes."
In addition, Bing Ren, Ph.D., associate member of the Ludwig Institute for Cancer Research and associate professor in the UC San Diego Department of Cellular and Molecular Medicine, received a New Faculty grant, awarded to the Ludwig Institute, for $1,726,564.
Ren will study transcription factors that are essential for embryonic stem cells to maintain their identity or differentiate along specific lineages. Results from the proposed study will improve the understanding of the mechanisms that control pluripotency – the cells’ ability to differentiate into many different cell types – and lay a foundation for development of better tools for manipulating and reprogramming human embryonic stem cells for regenerative medicine.
This round of grants brings the total awarded by CIRM to UC San Diego to $32,853,328. In addition, the San Diego Consortium for Regenerative Medicine (SDCRM) – comprised of UC San Diego, the Salk Institute for Biological Studies, The Scripps Research Institute and The Burnham Institute – received a $43 million facilities grant from CIRM earlier this year to help build a joint stem cell research facility.