UCSF Plays Significant Role in Stem Cell Research

By Jennifer O'Brien, UCSF Today | October 27, 2006

It has been a robust year for stem cell research at UCSF. Previously made up of numerous pioneering labs working relatively independently, the research program here has coalesced into one of the premier stem cell institutes in the United States.

Scientists in some 60 labs representing the fields of molecular biology, developmental and cell biology, neurobiology, immunology, and cancer research are bringing their expertise to bear on studies of cell differentiation, or specialization, and tissue regeneration -- all studies aimed at illuminating and treating disease.

The researchers are carrying out their studies in adult and embryonic stem cells -- as well as similar early-stage cells -- of fruit flies, zebrafish, worms, mice and humans. They are working to characterize the nature of the cells and to identify the genes that are expressed as the cells move increasingly toward becoming particular cell types, such as those of the heart, brain or pancreas. They are also exploring the use of stem cells to treat animals with experimental diseases.

They are making headway. The scientists, based in labs throughout the University, are gaining insights into the earliest stages of development and the genetic missteps that lead to such conditions as birth defects, infertility, the development of some cancers and the onset of a host of diseases, including breast cancer and Parkinson's disease.

They also are making advances that could someday lead to the use of stem cells and other early-stage cells as the basis for therapies to treat or replace damaged tissues in diseases such as heart disease, diabetes, Parkinson's disease, childhood epilepsy, Huntington's disease, amyotrophic lateral sclerosis (ALS), musculoskeletal diseases and spinal cord injury.

Laying the Foundation
"It is an extraordinarily exciting time in the development of the stem cell field and UCSF's role in it," says Arnold Kriegstein, director of the UCSF Institute for Regeneration Medicine. "UCSF, with the breadth and depth of its research enterprise, is in a position to play a significant role in laying the foundation for the field and, eventually, to help move discoveries toward clinical trials in patients."

The range of research underway at UCSF is broad:

Arturo Alvarez-Buylla, professor of neurological surgery, discovered a population of neural stem cells in the brain, and is carrying out a range of studies on these cells. One line of his research suggests that adult neural stem cells may be the cause of malignant gliomas, the most common form of brain tumor. If this hypothesis proves true, scientists may be able to target and destroy such rogue stem cells.

Yerem Yeghiazarians, assistant professor of medicine, interventional cardiology, and director of the Translational Cardiac Stem Cell Program, is exploring the potential of treating cardiomyopathy, a condition that results in weakening of the heart muscle and its pumping ability, with stem cells.

Mike German, professor of medicine, and Matthias Hebrok, associate professor of medicine, are studying the genes that prompt stem cells to become insulin-producing pancreatic beta cells. Their goal is to create pancreatic islet cells that could be transplanted into diabetic patients to treat their disease.

Susan Fisher, co-director of the UCSF Human Embryonic Stem Cell Research Center, is working to grow human embryonic stem cell lines in "feeder-free" conditions -- i.e., without exposure to mouse or human cells that traditionally have been used to nourish the cells in culture. The goal is to create conditions for the cells that would qualify them under the Food and Drug Administration's biological safety standards for transplantation, with an eye toward cell-based therapies.

Renee Reijo Pera, co-director of the UCSF Human Embryonic Stem Cell Research Center, is studying human embryonic stem cells with the goal of identifying genetic mutations that could play a role in some cases of birth defects, infertility and cancer. She also is carrying out studies of somatic cell nuclear transfer, or therapeutic cloning, with the ultimate goal of providing a strategy for creating patient-specific cell lines to study and treat diseases.

Read more at Jennifer O'Brien, UCSF Today