As reported in the European Medical Journal, Novartis has paused eight clinical studies evaluating its experimental CAR-T cell therapy, known as rap-cell, after three trial participants died following severe immune-related complications. The decision has prompted a broad safety review as the company works with independent experts and health authorities to better understand the events.
The affected patients were enrolled in studies investigating rap-cell for autoimmune and neurological conditions. According to Novartis, all three experienced serious immune reactions, a recognized risk associated with CAR-T therapies, which can sometimes trigger excessive immune system activation and lead to damage in multiple organs.
Comprehensive Investigation Underway
Following the fatalities, Novartis initiated a comprehensive assessment of the therapy’s safety profile. The company is collaborating with independent monitoring committees to review the circumstances surrounding the cases and determine whether earlier warning signs of severe toxicity can be identified in future patients.
CAR-T treatments are created by collecting and genetically modifying a patient’s own immune cells so they can target specific disease-causing cells. The technology has transformed treatment options for several blood cancers, but researchers have increasingly explored its use in autoimmune diseases and other non-cancer indications.
Novartis stated that it continues to share data with regulators and is closely monitoring participants who have already received the experimental treatment.
Autoimmune and Neurological Programs Impacted
The trial suspension affects multiple studies involving inflammatory and autoimmune disorders, including:
- Systemic lupus erythematosus (lupus)
- Rheumatoid arthritis
- Vasculitis
The pause also extends to investigations in neurological diseases characterized by nerve or muscle dysfunction, such as:
- Multiple sclerosis (MS)
- Myasthenia gravis
However, Novartis emphasized that the suspension does not apply to separate rap-cell studies being conducted in patients with lymphoma and leukemia. Those oncology trials are continuing without interruption.
Investor Response Remains Positive
Despite the safety setback, Novartis shares rose approximately 4% following the announcement. Market sentiment was supported by positive Phase 3 results for remibrutinib, the company’s investigational oral therapy for multiple sclerosis.
In the study, remibrutinib demonstrated superior relapse reduction compared with teriflunomide, an established MS treatment. The findings strengthened expectations that the drug could become a major commercial product if approved.
Broader Industry Caution
The Novartis announcement comes amid heightened scrutiny of CAR-T therapies being developed for autoimmune diseases. Reports indicate that Bristol Myers Squibb has also temporarily paused certain autoimmune CAR-T studies as a precaution while safety considerations in the emerging treatment area continue to be evaluated.
The developments highlight both the promise and the challenges of expanding CAR-T technology beyond cancer care. While the approach has delivered significant benefits in oncology, researchers and manufacturers continue to navigate the complex safety risks associated with manipulating the immune system for a broader range of diseases.
