Researchers in the Abramson Cancer Center (ACC) helped bring new hope to patients with multiple myeloma who are out of all other options.

Earlier this month, the US Food and Drug Administration (FDA) granted accelerated approval to a drug called selinexor (Xpovio). It’s a cancer drug that’s unlike anything else oncologists have ever tried before. It’s not an immunotherapy, and it doesn’t target any specific mutation in a tumor. Instead, it’s a pill known as a selective inhibitor of nuclear export, and it targets the systems that cells have to keep some molecules in the nucleus—the command center of the cell—and others out. Cancer cells, in particular, need to export tumor-suppressing proteins from the nucleus so the tumor itself can grow and survive. Selinexor works by blocking the protein that shuttles the tumor suppressants out of the nucleus; a protein known as XPO1.

“The medication actually goes into the nucleus of the cell and affects where proteins are located, which is not something we’ve ever targeted before,” says Dan Vogl, MD, an associate professor of hematology-oncology in the Perelman School of Medicine and a member of the ACC. Vogl led the first part of the phase 2 trial on selinexor—published in the Journal of Clinical Oncology in 2018—and advocated to the FDA for its approval after completion of the second part of that trial.

The trial showed that 25% of multiple myeloma patients respond to treatment with a combination of selinexor and the steroid medication dexamethasone, in a population of patients who had already received and were no longer benefiting from all of the other highly effective myeloma medications available. That data led the FDA to conditionally approve the drug for multiple myeloma patients who have undergone four or more prior therapies for their disease. Still, the approach is not without risk. The drug also targets the nuclei of healthy cells, meaning patients can experience serious and potentially deadly side effects.