In Vivo CAR-T Cell Therapy for Cancer Treatment

Recent advancements in cancer treatment using genetically modified immune cells, specifically T cells, have shown tremendous promise in battling diseases such as multiple myeloma, a type of blood cancer. Traditionally, T cells are harvested from patients, modified in a lab to create chimeric antigen receptors (CAR), and then reintroduced into the body. This process is often time-consuming and expensive, leading researchers to explore the potential of creating these modified cells directly in the body for faster and more effective treatment.

Exciting preliminary results from recent clinical trials have highlighted the effectiveness of the in vivo approach. In one study, four patients with multiple myeloma successfully developed in vivo CAR-T cells within their bodies after receiving a specially engineered virus, which introduced the necessary genetic changes. This technique allowed their immune cells to target and eliminate cancerous cells from their bone marrow, showcasing the strategy's potential to induce quick and powerful responses against the disease. 

Although the early data is encouraging, there are still challenges to tackle. Patients in these trials experienced some side effects, including a drop in blood pressure and oxygen levels, as well as mild confusion. This indicates that while the in vivo method might reduce some complications seen in conventional CAR-T therapies, new side effects may still arise. Current CAR-T treatments can also lead to cytokine release syndrome (CRS), a severe immune reaction, though it was observed at a lower severity in the in vivo trials. “The question is no longer can you really do this,” says MIMG faculty member Yvonne Chen. “The question now is can you reach the level of efficacy that’s expected and will the safety profile meet the target.”

In another trial, researchers reported similar positive outcomes, with all patients showing no detectable cancer cells in their bone marrow after treatment. The side effects in this trial were milder, suggesting that understanding and mitigating adverse reactions could be crucial as this new approach develops further. Experts believe this in vivo CAR-T therapy could transform how we treat multiple myeloma and potentially other hard-to-treat cancers in the future.

While these trials mark a significant step forward, experts stress the need for more extensive studies to ensure the safety and effectiveness of in vivo CAR-T cell therapy. Long-term patient monitoring will be crucial to comprehensively evaluate the results and refine the approach, but the current findings do offer hope for those battling cancer and highlight the ongoing innovation in cancer immunotherapy.

Read the full article in Science.