Skip to Content

How This Biotech Wants to Become a Stem Cell Therapy Success Story

Athersys Stem Cell TherapyAthersys Stem Cell Therapy
Athersys won a recent FDA fast track for its multi-purpose stem cell therapy.Westend61—Getty Images

The world of stem cell-based medicine has had its fair share of disappointments and regulatory recriminations over wildly inflated claims. But Athersys CEO Gil Van Bokkelen says the stem cell field isn’t getting a fair shake.

“It’s a misconception to say stem cells haven’t really delivered,” he tells Fortune in an interview. “This is a very hot field right now, and things have really changed.”

Van Bokkelen points to his own company’s recent achievements, including a number of cell therapies in mid- or late-stage clinical trials. Just two weeks ago, Athersys’ experimental MultiStem treatment received the Food and Drug Administration’s (FDA) coveted fast track designation in a devastating lung-wasting disease called acute respiratory distress syndrome (ARDS). These designations speed the regulatory process and is typically granted for products that treat disorders with a dearth of available treatments.

And devastating ARDS surely is. It’s a consequence of trauma to the lungs in patients who are typically already in the hospital for some kind of severe injury or illness, and it results in fluid leaking in to the lungs, making the very act of breathing even more difficult (or impossible) in patients who are already extremely sick, according to the American Lung Association.

Athersys’ studies of MultiStem found that patients who received the treatment “within several days after being diagnosed with ARDS and being placed on a ventilator experienced lower mortality, increased ventilator-free days, and increased ICU-free days” compared to a placebo group. Those preliminary results led to the FDA fast track.

What makes the MultiStem platform intriguing is, well, what puts the “Multi” in the name. Van Bokkelen says that Athersys is trying to avoid the main problem many stem cell upstarts face: Scaleability and multi-faceted uses. Rather than requiring an individual, perfect donor match, Athersys is attempting to create a product that’s an “off-the-shelf” version of regenerative stem cell treatment that can help repair tissue.

“The drug isn’t just doing one thing, it’s doing a bunch of different things,” says Van Bokkelen. For instance, it’s being tested out in late-stage trials for ischemic stroke patients (yet another program with FDA fast track designation). “Market opportunity is way bigger in this space than pretty much any other one,” he adds.

We should find out soon enough whether Athersys can succeed where so many others have failed.

Subscribe to Fortune’s Brainstorm Health Daily newsletter for news on the most exciting health innovations delivered straight to your inbox.