Drug Combination Leads to Effective Response, Remission in Patients with Multiple Myeloma

Researchers at Mount Sinai Hospital may have found an effective new therapeutic option for patients with multiple myeloma, according to a study published in The New England Journal of Medicine.

A drug combination of selinexor with dexamethasone significantly reduced cancer in more than one-quarter of patients tested, including 2 patients who experienced complete remission. Selinexor works by blocking the export of protein and messenger RNAs from the nucleus of the cancer cell to the cytoplasm, which plays an important role in cancer cell growth—this causes the cancer cell to die.

The clinical trial, called the STORM Part 2 Study, examined the responses of 122 patients taking selinexor and dexamethasone, both oral drugs, in trials across the United States and Europe. Additionally, 25% of the patients were enrolled in the international arm of the study.

"This study proved that a novel, first-in-class drug with a new mechanism of action can kill a patient's cancer cells," said the study's senior author, Sundar Jagannath, MBBS, Director of the Multiple Myeloma Program and Professor of Medicine (Hematology and Medical Oncology) at The Tisch Cancer Institute at Mount Sinai. "This proved that the drug worked in patients who had exhausted every other treatment and who would have been placed on hospice care otherwise."

Patients reported a response to the drugs within 1 or 2 months. While there was no organ toxicity, adverse events included low blood count without bleeding, nausea, vomiting, lack of appetite or fatigue. According to the report, more than 40% of patients with multiple myeloma had a minimal response to this drug combination.

Selinexor was approved by the FDA for patients resistant to multiple therapies in early July and is being investigated in multiple myeloma in combination with other drugs approved for the disease, as well as in other malignancies, such as lymphoma and ovarian cancer.

Reference

• Unprecedented Therapy Found Effective for Blood Cancer Patients with No Treatment Options [press release]. New York, NY. Mount Sinai website. Published August 22, 2019. https://www.mountsinai.org/about/newsroom/2019/unprecedented-therapy-found-effective-for-blood-cancer-patients-with-no-treatment-options. Accessed August 26, 2019.