Clinical trial testing innovative HIV/AIDS stem cell therapy gets under way

The first time you try something new, particularly in the world of science, it is often a time of both optimism and caution. That’s particularly true when it’s the first time a project you have funded is starting a clinical trial, moving out of the lab and the world of theory and into people and the real world. So while we are delighted to be able to announce that Calimmune has begun a clinical trial using its innovative approach to HIV, we are also very mindful that this is just the first step on what could be a long road.

In this trial Calimmune is using an innovative gene-based stem cell therapy to help protect individuals with HIV from the effects of the AIDS virus. It employs a two-pronged approach using a gene medicine called Cal-1. This medicine is a combination of the individual’s own T cells (which help direct the body’s immune defense system) and a blood forming stem cell that has been genetically modified to block CCR5, a protein known to play a key role in helping the AIDS virus infect cells. By blocking CCR5 it is hoped this approach can stop the virus from entering and damaging cells. The therapy also uses a second mechanism to reduce the likelihood that the virus will develop resistance to this procedure.

Our President, Alan Trounson, PhD, says the trial is an important one for several reasons:

“CIRM funding of this Phase l/ll trial is an important milestone for us,” says Alan Trounson, PhD, President of the stem cell agency. “One of our goals is to support research that moves the most promising science out of the lab and into clinical trials in people. To be able to do that with a disease as devastating as HIV/AIDS highlights the importance of our funding and the potential impact it could have on the health of people around the world.”

Calimmune CEO Louis Breton echoed those feelings in our news release:

“This study is an early but important step in an emerging area of scientific exploration, representing the culmination of more than a decade of research and development. We are optimistic that what we learn from this study may bring us closer to the day when a one-time or infrequent treatment could lessen, delay or provide an alternative to a lifetime of antiretroviral therapy.”

Altogether 12 patients will be enrolled in this phase of the trial, which will be carried out at clinical sites in San Francisco and Los Angeles, the company announced. The goal, as with all early stage trials, is to test the safety and measure any preliminary indicators of the effectiveness of the therapy. If it is successful the impact on our ability to treat HIV/AIDS could be enormous. But that’s a long way away.

This is not the only research we are funding that is combining stem cells and gene modification to try and ‘disable’ the CCR5 protein. A team from USC and the Beckman Research Institute at the City of Hope are taking a slightly different approach to achieve the same goal. You can read about their work here. There’s also more information on our website about all the HIV/AIDS projects we’ve funded.

Kevin McCormack

Learn more about the California Institute for Regenerative Medicine