The success of stem cell therapies has been some time in coming, explains Michael Jacobson, PhD, founding partner at Cambridge Biostrategy Associates. “Stem cells—specifically Mesenchymal Stem Cells (MSCs)—not CAR-T, were where the commercial focus was ten years ago,” he says.
Three or so stem cell companies are still hanging on, he adds. They have shown some clinical successes, but also some setbacks, indicating, he says, that clear signs of commercial and clinical success remain slow to come. Among the stem cell companies with late-stage development products is Athersys.
“The first-generation [of CAR-T] was accelerated in development because they were showing evidence of good therapeutic effect in patient populations who had no other option,” explains Gil Van Bokkelen, PhD, Athersys’ CEO. “But the reality is they’re very expensive, cumbersome, and can only deal with one patient at a time—they’re the antithesis of scalable.”
In contrast, he explains, MultiStem uses cells with robust growth properties to generate millions of clinical doses from a single donor bank. “The general pattern in the industry is we’re seeing the emphasis shift from expensive gene-modified personalized therapies to more scalable off-the-shelf therapies that can be produced in larger-scale bioreactors.”
To supply large groups of patients with stroke, for example, the company is developing large-scale manufacturing processes. As he explains, “We do all our own process development at our European subsidiary in Belgium and our U.S. subsidiary in Ohio, and all our tech development work,” he explains.
A key goal is to reduce Athersys’ dependence on contract manufacturers (CMOs), who, he says, are struggling to keep pace with surging global demand. With capital investment in gene and cell therapy doubling in a year and meaningful growth in late-stage clinical trials, he argues that many companies are building their own manufacturing facilities to complement or replace CMOs.
Athersys is already planning to establish a manufacturing facility to complement their trusted CMO relationships. With the company’s Phase III trial of a stroke treatment in Japan a “pivotal study, which represents a big opportunity,” he says, “we’ve been working hard to establish an end-to-end process, which is very scalable and well controlled, so we can produce a large quantity of material—because we’re not far from the finish line.”