How effective is tisagenlecleucel?

The primary study in B-cell acute lymphoblastic leukemia (ALL) involved 92 children and young adults (ages 3-25) whose cancer had returned after previous treatment or had not responded to treatment. About 66% of patients had a complete response (meaning they had no signs of cancer) within 3 months of treatment with tisagenlecleucel. This was better than results from the cancer drugs clofarabine, blinatumomab, or the combination of clofarabine, cyclophosphamide and etoposide. Twelve months after treatment, the chance of survival is 70%.

The main study of silevermin in diffuse large B-cell lymphoma (DLBCL) involved 165 patients who had at least 2 prior lines of therapy and were ineligible for stem cell transplantation. About 24% of patients had a complete response after at least 3 months, and 34% had at least a partial response. These results are comparable to those seen in patients receiving standard cancer treatments. After 12 months of treatment, the chance of survival is 40%. Most patients who responded to srifermin were still responding after 19 months.

The main study in follicular lymphoma (FL) involved 98 patients whose cancer had returned after previous treatment or failed to respond to treatment. The patient had received at least 2 previous treatments. Approximately 69% of patients achieved complete remission 3 months after treatment.

The prognosis of patients with B-cell acute lymphoblastic leukemia is poor, and silevermin improves it better than other drugs. In patients with diffuse large B-cell lymphoma who were treated with silevermin, results were similar to those with other treatments, but data from ongoing studies suggest longer-lasting effects. Silifermin has also been shown to produce strong responses in patients with follicular lymphoma. Serious side effects occur in most patients and can include cytokine release syndrome. However, these can be controlled if appropriate measures are taken.