Monday, February 28, 2011

Sangamo Press Release

We will be blogging the Conference on Retroviruses and Opportunistic Infections, which is happening right now in Boston. But first, we want to post a Sangamo press release. Anyway, more later.

February 28, 2011

Sangamo BioSciences Announces Presentation of Positive Clinical Data From Novel ZFN Therapeutic Approach for the Treatment of HIV/AIDs at Conference for Retroviral and Opportunistic Infections

First Steps in Development of a 'Functional Cure' for HIV/AIDS: Demonstration of Successful Engraftment and Expansion of ZFN- CCR5-Modified T-Cell Product, SB-728-T, with Durable Improvement in Total CD4+ T-cell Counts and CD4:CD8 Ratios

RICHMOND, Calif., Feb. 28, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the presentation of positive preliminary clinical data from its Phase 1 trial (SB-728-902). The trial is being conducted in immunologic non-responders, HIV-infected subjects who are currently on highly active antiretroviral therapy (HAART) and have undetectable levels of virus but suboptimal CD4+ T-cell counts. The study is designed to evaluate safety and clinical outcomes of Sangamo's zinc finger nuclease (ZFN)-generated CCR5-modified, autologous T-cell product (SB-728-T) for the treatment of HIV/AIDS. CCR5 is the major co-receptor used by HIV to infect cells of the immune system.

"These compelling data provide a mechanistic 'proof of concept' for this novel approach to HIV therapy which shows the most promise of any yet tested," stated Carl June M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine, and an investigator in a second SB-728-T Phase 1 trial that is led by Pablo Tebas, M.D. at the University of Pennsylvania.

"From a single infusion of ZFN-modified cells, substantial and sustained increases in total CD4+ T-cell counts were observed in most subjects. This improvement is greater than we have seen in any previous adoptive T-cell approach. The data are consistent with CCR5-ZFN-modified T-cells being resistant to HIV infection and having a selective advantage in the presence of the virus — just as we saw in the preclinical studies. This is very encouraging as we continue to evaluate the drug in HIV-infected subjects with active infections."

Summary of Clinical Data

The data demonstrate that a single infusion of SB-728-T was well tolerated; the CCR5-modified cells successfully engrafted in all subjects and resulted in a durable improvement in total CD4+ T-cell counts in five of six of the subjects analyzed. In addition, five of the six subjects also exhibited sustained improvements in their CD4:CD8 T-cell ratio, which is an indicator of immunologic health. The ZFN-CCR5-modified cells also exhibited normal T-cell growth kinetics and trafficking and were observed to undergo selective expansion in the gut mucosa, a major reservoir of virus in the body, suggesting, as predicted, that the cells were resistant to HIV infection. These data represent the necessary first steps in the development of a "functional cure" for HIV/AIDS by providing a protected CD4+ T-cell population in these subjects that is resistant to HIV infection.

Wednesday, February 9, 2011

Is AIDSWatch Keeping Up? - AIDS Policy Project

We approached the National Association of People with AIDS several weeks ago, asking them to include AIDS cure advocacy in the agenda for their national AIDS lobby day, AIDSWatch.  A number of major U.S. AIDS organizations wrote letters in support of this idea. NAPWA said yes. But a few days ago, they reversed this decision (you can read their explanation below). 

You will not see another group speaking truth to power in this way.  We believe very strongly in solidarity and have a long track record of practicing it. But however strong or weak they are, NAPWA, in addition to a tiny group of other national advocates, does influence whether or not there is money for AIDS research, for treatment (there are currently 6,000 sick Americans with AIDS on waiting lists for their drugs), housing, and other critical services that keep people with AIDS alive.
  Decisions about AIDS research, including the search for a cure, have implications for people with AIDS around the world.

We hope that NAPWA will change its mind.

You can read the comments about this blog post by going to

Is AIDSWatch Keeping Up?

By Jose De Marco and Stephen J LeBlanc
(Members of the Board of Directors, The AIDS Policy Project)

Just three years ago, everyone thought a cure was a distant dream.

But the case of the Berlin Patient, cured of AIDS in 2007 and HIV-free today, has inspired new research in laboratories from San Francisco to Melbourne, Australia. An activist campaign is calling for $240 million for AIDS cure research at the NIH, funded with money from a department that is being shut down. People affected by HIV are becoming aware of the possibilities. A recent poll by the National Minority AIDS Council showed that fixing ADAP and an AIDS cure were the top two policy priorities of its constituents.

But you won't find anyone talking about a cure at AIDSWatch, the annual lobby event organized by the National Association of People with AIDS. Starting February 16th, it promises to advocate for "the most crucial issues facing the 1.3 million people living with HIV/AIDS in the United States." A cure is not on their list.

Four hundred people with AIDS and their allies will fan out across Capitol Hill to talk about the need for AIDS funding with their legislators. But despite letters of support from organizations of people with AIDS ranging from ACT UP Philadelphia to the National Minority AIDS Council, NAPWA has declined to include AIDS cure research on the agenda for discussion or for lobbying. Their agenda is set, they say. According to one organizer, there is no time for a speaker to discuss AIDS research, let alone the cure. He told us there will be no specific dollar requests in the lobby visits. Otherwise, volunteer lobbyists would suffer from "information overload."

Funding for the National Institutes of Health has been stagnant since 2003. The NIH's main AIDS research division, NIAID, must reject funding for 77% of the AIDS research grant applications it receives because there is no money to pay for more.

Worse, the NIH spends only 3% of its AIDS research budget trying to find a cure for AIDS. Three percent. There is no commitment yet to increase this funding, even as the entire field has been reinvigorated and exciting research is being proposed in labs all over the world.

Only 5 million of the 33 million people with HIV worldwide currently have access to AIDS meds. Only 1/3 of the very sick receive them. In 2009, nearly 2 million people died this way.

We need a cure.

Congressional briefings, paid lobbyists, and advocacy reports have not, so far been enough to increase federal funding for the NIH, let alone AIDS cure research. With Congress trying to cut spending back to 2008 levels and some members of Congress trying to de-fund the US Agency for International Development (and therefore US-funded global AIDS treatment), what else can we do? What could work?

Hundreds of thousands of people care about AIDS in this country. We need to leverage their voices to loudly, publicly, and specifically call for more AIDS funding--for the ADAP, for housing, for the NIH, and for a cure. This movement requires the grassroots power of people with AIDS. Expertise is essential--but expertise without political power cannot get the goods.

NAPWA must reverse its mistake and include AIDS cure research, and indeed overall NIH funding on its agenda for policy briefing and lobbying on February 16th-18th. Research advocates want to share what they know with the hundreds of people with AIDS making the trip to Washington from states across the US. We want them to talk to their representatives in Congress about AIDS cure research, and the need for more NIH funding. The fact that there are promising research avenues for a cure for AIDS that remain starved for funding will come as news to nearly all of them.

AIDSWatch is the only national AIDS lobby day we have. It is a critical chance for people with AIDS and their allies to push for funding and educate Congress about the issues. Including progress toward a cure.

The NIH, despite its meager financial commitment, is still the leading funder for cure research worldwide. AIDS research is a global issue. What happens at AIDSWatch affects the world, but the world doesn't have a say in what is on the agenda at AIDSWatch.

Political power, including focused grassroots power, is critically needed to move funding for an AIDS cure forward. Hundreds of people coming to Washington in two weeks can help--if they get the chance.

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