New Initiative at UCSF Targets Multiple Myeloma

A major new initiative at UCSF will focus on the discovery and development of promising new treatments for multiple myeloma, a cancer of the blood that kills nearly 11,000 Americans every year.

The Multiple Myeloma Translational Initiative (MMTI) kicked off in April with a $2 million gift from Stephen and Nancy Grand, longtime supporters of UCSF. The gift will fund the first two years of the initiative, supporting the research of UCSF scientists and working to secure the University’s place as a national leader in the fight against cancer.

“The potential here is to have the best cancer program not just at UCSF, but anywhere in the United States,” said Marc Shuman, MD, UCSF professor of medicine and urology, clinical director of the California Institute for Quantitative Biosciences (QB3), and co-leader of the Prostate Cancer Program at the UCSF Helen Diller Family Comprehensive Cancer Center. “An initiative like this could not be done at most medical institutions in this country.”

Multiple myeloma begins in the plasma cells, a type of white blood cell that forms antibodies, an important part of the immune system. In 2008, nearly 20,000 new cases of multiple myeloma were diagnosed in the United States and the disease claimed roughly 10,700 lives, according to the American Cancer Society.

UCSF’s multiple myeloma program is already the largest on the West Coast, but the new funding will allow for the expansion of existing labs and clinical research initiatives. In addition, MMTI translational and clinical scientists will strengthen their own efforts by closely collaborating with other cancer researchers, both on and off campus, said Jeffrey Wolf, MD, director of the multiple myeloma program and the new MMTI.

“The Grand gift will accelerate the translation of multiple myeloma research from bench to mouse to bedside,” he said at the April 27 MMTI launch ceremony, where UCSF scientists involved with the initiative provided a glimpse of their ongoing efforts to better understand and combat this debilitating disease.

Two of those scientists, Kevan Shokat, PhD, and Davide Ruggero, PhD, hope to use a portion of the Grand gift to fund a clinical trial of a new drug they discovered that blocked cancer’s main source of growth when tested in mice. The compound, dubbed TORKinib, inhibits the signaling molecules that prompt cells to manufacture proteins for growth – signals that cancer exploits for its own growth. Shokat, UCSF professor of cellular and molecular pharmacology, and Ruggero, UCSF assistant professor of urology, plan to test TORKinib in a new mouse model of multiple myeloma, with the eventual goal of a human clinical trial, they said.

The MMTI will also benefit from the expertise of a team of UCSF scientists investigating a new cancer-fighting strategy that involves manipulating a basic, life-or-death response within cells known as the unfolded protein response. Led by Peter Walter, PhD, UCSF professor of biochemistry and biophysics, the six-person team also includes Shokat and Shuman, among others. It is funded by a $4 million Collaborative Innovation Award from the Howard Hughes Medical Institute (HHMI). Walter, who also holds the title of HHMI investigator, and his fellow researchers believe that cancer may exploit the unfolded protein response to keep up its own out-of-control growth. So far, the strongest evidence of that theory has been found in multiple myeloma, and the new MMTI will give Walter’s team the opportunity to probe more deeply and possibly develop new forms of treatment for the disease.

That sort of research ties in directly with the larger mission of UCSF’s cancer program, said Eric Small, MD, professor of medicine and urology, leader of the Prostate Cancer Program, and director of investigational therapeutics at the Cancer Center.

“The MMTI couldn’t fit better with the direction the center is going in,” he said at the launch event. “The key goal is translation. When it comes down to it, the real benefit of this initiative comes to our patients.”

The Grands made their gift to UCSF through the Multiple Myeloma Research Foundation (MMRF), a Connecticut-based organization that funds research aimed at developing new treatments for multiple myeloma. The MMRF’s sister organization, the Multiple Myeloma Research Consortium, works to facilitate clinical trials of new drug therapies at UCSF and 14 other member institutions.