Under CEO Paul Stoffels, Belgian biotech Galapagos is testing a device that manufactures CAR-T cancer therapies in hospitals, bringing down the wait time and price. When it works, CAR-T cancer therapy can appear miraculous. With a single infusion of their own immune T-cells—genetically modified to find and kill cancer in the blood—about half of patients with leukemia and lymphoma, and about a third with myeloma, have a complete remission, achieving a functional “cure.” For the most prevalent type of pediatric cancer, acute lymphoblastic leukemia, CAR-T therapy has demonstrated complete remission rates as high as 90%. The first two patients treated with CAR-T therapy, in 2010—adult men who had end-stage chronic lymphocytic leukemia—were still in remission a decade later. The U.S. Food and Drug Administration has approved a total of six CAR-T therapies since 2017, all for the treatment of blood cancers.

A 2022 survey by Mayo Clinic researchers found that the median time on the waiting list for CAR-T therapy was six months, and that only a quarter of patients eventually received it.

Unlike regular drugs, so-called autologous CAR-T infusions are “living medicines,” custom-made for each patient. Typically, patients will get blood drawn in a hospital or special center. The T cells are separated out, and shipped refrigerated or frozen to a central biomanufacturing facility where they’re genetically  “reprogrammed” to express a tumor-seeking molecule, called a chimeric antigen receptor, or CAR, on their surface. The modified cells are “expanded” in an incubator for a few days or weeks to boost their numbers enough to create a therapeutic dose. After several quality testing steps, the modified CAR-T cells are frozen and sent back to the hospital to deliver to the patient. The process typically takes at least two weeks and up to eight weeks, “vein to vein.”

Current CAR-T treatments have list prices in the high $300,000 to high $400,000 range. “The reason it’s so costly,” explains Travis Young, VP of Biologics at the nonprofit California Institute for Biomedical Research (Calibr), “is because every point in that manufacturing process has to be very highly controlled. It requires a technician that has a lot of training, it requires clean rooms, it requires the infrastructure for shipping and cryopreservation, and then the biggest time is the pre-release testing to ensure the sterility and potency of the product.” There are numerous opportunities for glitches. “The supply chain is in its infancy,” Young says. “And it’s not just the infrastructure, it’s the number of people that have the training to do this.”

Companies are hacking away at these challenges in different ways, aiming to reduce the complexity, time, and ultimately cost of delivering CAR-T treatments to more patients. One of the more unlikely contenders is Belgium-based Galapagos NV, which last June announced a bold plan to manufacture these expensive therapies more quickly and affordably, by developing them not in a centralized facility, but at the point of care, using a small, highly automated device the size of a home microwave.

Source: https://www.fastcompany.com/