According to the findings of a clinical trial, scientists at Mt. Sinai have found a new therapeutic effective for patients with a particular type of bone marrow cancer. The cancer is known to be resistant to several standard therapeutics. Findings were published in the The New England Journal of Medicine and involves testing the drug selinexor with dexamethasone--a combination that treated the cancer in a quarter of patients where two of these patients went into complete remission.
In cancer cell biology, proteins and messenger RNAs play a critical role in cancer growth. Selinexor was found to inhibit the export of protein and messenger RNAs from the nucleus of the cancer cell to the cytoplasm resulting in cancer cell death.
"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 enrolled in the clinical trial, known as the STORM Part 2 Study, who took selinexor and dexamethasone, saw a response within one or two months. Even though no toxicity was present, patients did experience low blood count, nausea, vomiting, and lack of appetite or fatigue.
"This study is meaningful for patients with multiple myeloma who haven't had success on multiple other therapies," said the study's first author Ajai Chari, MD, Director of Clinical Research in the Multiple Myeloma Program at The Tisch Cancer Institute at Mount Sinai. "An increasing number of patients have resistance to the standard drugs used in the treatment of multiple myeloma, and the overall survival in these patients is short, sometimes less than three months."
Selinexor has been approved by the FDA for patients resistant to multiple therapies and the drug is being studied for multiple myeloma as well as in other malignancies such lymphoma and ovarian cancer.
In the Tisch Cancer Institute at Mount Sinai, the study brings a major milestone in myeloma research.
"The Center of Excellence for Multiple Myeloma is part of The Tisch Cancer Institute and uses the most advanced diagnostic and treatment approaches within state-of-the-art facilities of a National Cancer Institute-designated cancer center," said Ramon Parsons, MD, PhD, Director of The Tisch Cancer Institute, Chair of Oncological Sciences, and Ward-Coleman Professor in Cancer Research of the Icahn School of Medicine at Mount Sinai. "Coordinated care teams include experts from the Bone Marrow Transplant Program, pathology, radiology, immunology, genomics, infectious diseases, orthopedic surgery, cardiology, and nephrology, and patients will have broad access to comprehensive supportive services, including from social workers, financial counselors, and clergy."