ACT files to test embryonic stem cell-based therapy for macular degeneration

Advanced Cell Technology has filed an application with the FDA to begin an early phase trial of an embryonic stem cell-based therapy for macular degeneration. If the company name sounds familiar, that’s because it’s the same company that on November 22 received FDA approval to begin a trial for Stargardt’s macular degeneration. Both trials are testing the same cells. In a press release, the company said:

Company scientists view the use of the same hESC derived RPE cells for both trials as the most efficacious approach, as it permits the Company to leverage its experience with the FDA that it gained through the process of obtaining approval for the Stargardt’s clinical trial to expedite the approval of its clinical trial in Dry AMD.

As with the company’s Stargardt’s trial and Geron’s spinal cord injury trial, this new trial will be assessing safety in a very small number of patients — standard practice for any new therapy being tested. After proving safety in a phase I trial, a larger trial will assess whether or not the potential therapy is effective.

It’s exciting to see embryonic stem cell derived therapies reaching patients. In general, more trials fail than succeed and we don’t know in advance which ones are going to work. That’s why there are hundreds of cancer trials going on around the country, and why we need hundreds of stem cell trials too. Hopefully CIRM’s macular degeneration disease team led by Mark Humayun at USC won’t be far behind ACT with their own version of an embryonic stem cell-based therapy for macular degeneration. And hopefully, one of them will provide a cure for the roughly 30 million people worldwide who are losing or have lost vision due to the disease.

Here’s a video about the CIRM disease team project:

A.A.

4 thoughts on “ACT files to test embryonic stem cell-based therapy for macular degeneration

  1. I'm confused. Why would CIRM fund research that has already been researched? Dr Lanza of ACTC has already done the research on RPE cells and macular degeneration. ACTC has patents on RPE technology. You aren't going to catch up to them. ACTC is going to clinical right now. ACTC deserves a loan from CIRM. If CIRM doesn't give ACTC a loan for their clinical trials, I fail to see the point of CIRM.

  2. CIRM has just opened up our first round of funding for clinical trials, which ACT is welcome to apply for. If we do end up funding ACT, it's still a good policy to be funding more than one approach to macular degeneration. Think about it — if cancer clinical trials stopped when the first chemotherapy was in a phase I trial we'd be far behind where we are now. Instead, CIRM has the goal of funding a wide range of approaches to treating different diseases. As the science progresses those groups can learn from each other, make changes, and continue developing better, more effective therapies.

  3. Eigenman,
    What ACT is doing is very exciting, but literally no one knows if it will (A) be safe in humans and (B) actually work. It will take years to be sure.

    I hope it does work, but a clinical trial is an experiment and often they don't work out the way we think they should…even when we have strong preliminary preclinical data.

    By CIRM funding additional studies for the treatment of macular degeneration, they increase the overall odds that we as a field get a successful treatment for blindness.

    You might think it impossible, but the reality is that ACT's trial might fail and in the grand scheme of clinical trials, most do fail. Sometimes even the most promising.

    Even if ACT's trial is a success, work by others might prove clearly superior. Or it is possible that giving patients combinations of therapies such as both ACT's and one by another team might work best. For example, most cancers are treated by combinations of therapies.

    Paul

  4. It sounds to me like you have a bias against funding ACTC's research and are cleverly finding ways to make it seem like you are instead looking out for the greater good of research.
    colorwonder

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