- Despite promising response rates among blood cancer patients treated with CAR-T, T-cell-based immunotherapy is still not considered a "breakthrough cure," according to a GlobalData analyst Cal Xuan.
- When Xuan spoke to Pharma Times, she emphasized that treatments are very expensive, ranging from $300,000 to $500,000—a price tag that suggests the treatment should be curative in order to justify the price.
- CAR-T also comes with some big safety concerns, including an increased risk of tumor lysis syndrome (TLS) and cytokine release syndrome (CRS). Both can be fatal.
Chimeric antigen receptor therapy, also known as CAR-T, has generated a great deal of hype and a great deal of hope. That's driven by exciting, but early, outcomes in the few clinical trials conducted to date.
Researchers at the University of Pennsylvania documented a 90% clinical response rate in children with relapsed acute lymphoblastic leukemia (ALL) who were treated with CAR-T. There are also early-stage trials that show promise.
Nonetheless, not every treatment effort is a home run. And with price tags hovering at the half-million dollar mark, other treatments such as conventional mainline therapy and stem-cell transplanats may be better alternatives for some patients.
There are also major concerns associated with the most common side effects of CAR-T—TLS and CRS. TLS is potentially life threatening and occurs when chemotherapy kills cancer cells so quickly that the kidneys can't break down the byproducts of this process that reside in the blood.
Cytokine release syndrome (CRS), an extreme inflammatory response to CAR-T, leads to flu-like symptoms and can be fatal. Both are real threats to patients undergoing T-cell therapy and must be factored into the overall value equation.