Sangamo HIV project could rewrite story of state's stem cell agency
Read more: Sangamo HIV project could rewrite story of state's stem cell agency | San Francisco Business Times
San Francisco Business Times - by Ron Leuty Date: Thursday, March 10, 2011, 2:21pm PST
A consortium that includes Sangamo BioSciences Inc. is pursuing a next-generation stem cell treatment that could provide a longer-term — if not lifetime — barrier to the AIDS virus.
But the fate of California's $3 billion stem cell research funding agency also may rest on the success or failure of the Sangamo collaboration and 13 other "disease teams" that are trying to get into clinical trials within four years.
Backed by a grant from the California Institute for Regenerative Medicine, researchers at Richmond-based Sangamo (NASDAQ: SGMO), the University of Southern California and the City of Hope medical center near Los Angeles believe they can draw stem cells out of bone marrow and knock out a gene that produces the door knob that HIV uses to access a cell. Researchers then reassemble the stem cell and place it back in the body so it can produce blood cells sans the key gene and, therefore, are resistant to HIV.
It is experimental — this specific gene therapy has not yet been tested in humans — but it has been successful in mouse models, said Paula Cannon, an associate professor of molecular microbiology and immunology at USC, who last week presented data from her lab’s work at the 18th Conference on Retroviruses and Opportunistic Infections in Boston.
The collaboration could be trotted out as a success story for CIRM, the stem cell research funding agency set up when California voters in 2004 approved of the state sale of $3 billion in bonds to finance the agency. CIRM earmarked $14.5 million in fall 2009 for the Sangamo-USC-City of Hope project.
The project received the highest science score among applicants for so-called disease team grants. Those 14 grants, totaling $230 million, were aimed at groups of researchers — academic collaborators or public-private partnerships — with stem cell-based treatments for a wide range of diseases.
The caveat? The projects must be in or on the cusp of clinical trials within four years of receiving the money.
“The clock’s ticking,” Cannon said. “There’s pressure, but there’s also pressure from ourselves to do it right and do it safely.”
CIRM, based in San Francisco, will need poster children like this to convince Californians that it has used its $3 billion wisely and is worthy of more cash. That’s a story that has been difficult to tell as CIRM’s internal follies — including the aborted selection of a new chairman at the end of last year — has seemed to grab more headlines.
CIRM has funded a research building boom — new facilities have been built at the University of California, San Francisco, and Stanford University, and are under construction at UC Berkeley. New research buildings are one thing, however, treating or curing a friend or loved one’s disease is another.
To that end, CIRM in January hired Dr. Ellen Feigal, who was executive medical director of global development at Amgen Inc. (NASDAQ: AMGN), as vice president for research and development.
“We’ve kind of been managing (the disease team projects) intensively because we want them to make their timelines,” said CIRM President Alan Trounson. “Now with Ellen Feigal, we can keep them on track and get in the clinic in the most efficient way possible.”
There’s one disease-team project that is taking cells from heart biopsies, growing them outside the body and transplanting them back; another is working on carrying cytotoxic drugs to inoperable brain tumors.
“All of them are on schedule at the moment, including some pretty interesting work in cancer,” Trounson said.
CIRM wasn’t the first money into Cannon’s project, which is about five years old. The agency initially received $100,000 over two years from the California HIV/AIDS Research Program, overseen by the University of California Office of the President, followed by money from the National Institutes of Health. Cannon’s lab started receiving the CIRM money in spring 2010.
But moving the project forward, Cannon said, would have been difficult because the generally conservative NIH likely would have deemed it too risky.
“It’s what we need,” Cannon said of the CIRM money. “It will fund us for four years so we can go to the government, the FDA, with all the preclinical information.
“Normally, Big Pharma companies are the only ones that have the money to do that regulatory work that’s needed, but this consortium is taking on the job ourselves.”
Sangamo has another gene-therapy approach in a Phase I/II trial — with investigators at the University of California, San Francisco, and UCLA — that blocks the CCR5 gene in white blood cells known as T cells.
Sangamo CEO Edward Lanphier has called it a “functional cure” for HIV.
Both SB-728, Sangamo’s T cell treatment, and the experiments in Cannon’s lab use the company’s “zinc finger” gene-editing technology. But Cannon’s work is further down the chain, changing the hematopoietic stem cells whose progeny include all blood cells, including T cells — so it could provide a more basic defense to HIV.
“Most other gene therapy strategies for HIV are all about putting something into the cell that will stop the virus,” Cannon said. “What I liked about this is, it’s just taking off the door knob (used by HIV) and you don’t have to keep taking off the door knob. Once it’s gone, it’s gone.”
Read more: Sangamo HIV project could rewrite story of state's stem cell agency | San Francisco Business Times
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