Cell Therapy Mostly Used For Blood Cancer Shows Efficacy In Autoimmune Diseases As Well, But There Are Challenges

A revolutionary cell-based gene therapy is showing promise for treating autoimmune diseases.

Professor Georg Schett, a rheumatologist at University Hospital Erlangen in eastern Germany, used chimeric antigen receptor (CAR-T) cell therapy, typically used for blood cancers, for autoimmune conditions like lupus, when the immune system attacks healthy cells and tissues by mistake. This attack causes inflammation.

The Financial Times writes that the early success of CAT-T therapy has reignited discussions about its potential beyond blood cancers, though challenges remain.

Professor Georg Schett noted that this approach could revolutionize autoimmune disease treatment, providing a one-time intervention instead of the ongoing immune suppression traditionally required.

Despite only one of 15 patients experiencing a mild disease recurrence, the enthusiasm for CAR-T has grown.

The broader application of CAR-T faces hurdles, including high costs—up to $530,000 per treatment—and complex manufacturing processes.

Only 35,000 lymphoma and leukemia patients have been treated with CAR-T in the U.S. since its approval, while approximately 204,000 Americans suffer from systemic lupus erythematosus.

Challenges in scaling up the production are underscored by remarks from Peter Maag, CEO of Kyverna Therapeutics Inc KYTX, who compared CAR-T’s role to heart transplants in terms of necessity and impact.

Steven Nichtberger, chief executive of Cabaletta Bio, Inc. CABA, another biotech testing CAR-T for autoimmune disorders, in the FT report said the field would “rise and fall on the ability of a medicine to replicate Schett’s data.”

Patients typically undergo three chemotherapy sessions to prepare their bodies for CAR-T therapy, which often causes fevers or fatigue.

Cabaletta is exploring a version of the treatment that doesn’t require prior chemotherapy, potentially expanding its use to nearly all autoimmune patients, not just those severely ill, according to CEO Steven Nichtberger.

Pfizer-backed Allogene Therapeutics is using a manufacturing method called allogeneic CAR-T, which simplifies the process by using donor cells, but its effectiveness remains unproven.

Additionally, early data indicates that Amgen’s Blincyto, a cancer-targeting T-cell engager, showed promise in six rheumatoid arthritis patients and avoids the supply chain issues associated with CAR-T therapies.

Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.

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