Milestones are useful things. They measure how far we have come on a journey, and give us a sense that we are on the right path. One of the projects we are helping fund just passed a big milestone, and it’s given the researchers the go-ahead to move on to the next, perhaps even more important stage.
The project is Calimmune’s stem cell gene modification study, which takes blood stem cells from people who are HIV-positive, genetically modifies them so they carry a gene that blocks the AIDS virus from infecting cells, and then re-introduces the modified cells to the patient. The hope is that those stem cells will then create a new blood system that is resistant to HIV.
The milestone it passed is that the Data Safety Monitoring Board (DSMB) looked at the results from the first group of four patients treated with this approach, found that there were no serious adverse events or dangerous side effects from it, and gave Calimmune the go-ahead to start treating the next group of patients.
In a news release we put out jointly with Calimmune, their CEO Louis Breton said this is a big step forward for them:
“We are very excited and encouraged by this development. This recommendation from the DSMB is an important step in bringing this one-time therapy to the patients, and takes us closer to our ultimate goal of eradicating AIDS.”
It’s a pretty big deal for us too, as our President and CEO C. Randal Mills noted in the same release:
“The mission of CIRM is to efficiently accelerate the development of stem cell treatments for patients suffering from unmet medical conditions. While still early in clinical development this announcement demonstrates real progress towards this mission. The accomplishments of Calimmune’s team is a great example of how CIRM partnerships are working to impact patient’s lives today.”
Now, just treating four people might not seem particularly impressive, after all HIV/AIDS has killed more than 25 million people worldwide and has infected another 25 million more – around 1.1 million here in the U.S. But every treatment has to begin with a simple premise, that whatever you do is not going to hurt the patient. Getting the green light from the Data Safety Monitoring Board, an independent panel of experts who review data and advise the researchers doing clinical trials, shows this approach appears to be safe.
The next step is to repeat this same process in 3 or 4 more patients but to give those patients a preconditioning regimen, treating them with a medication before returning their modified stem cells to them, to try and make the therapy more effective. This could show that the therapeutic approach, called Cal-1, is not only safe but also is working to protect patients against HIV.
If the safety data from that second group also looks good, then Calimmune can move on to the next group of patients. Each step, no matter how small, moves us ever closer to our end goal of developing a cure for HIV/AIDS.
That’s still a very distant goal right now, but with each milestone we pass it shows that we are heading in the right direction.
Want to know more about Calimmune’s path towards clinical trial? Check out Calimmune CEO Louis Breton’s recent video describing their progress towards a cure for HIV.