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Mount Sinai Clinical Investigators Complete Study Evidencing Unprecedented Therapy for Blood Cancer Patients With No Treatment Options

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Mount Sinai Clinical Investigators Complete Study Evidencing Unprecedented Therapy for Blood Cancer Patients With No Treatment Options

Mount Sinai researchers have found a new type of therapy to be effective for patients with a particular type of bone marrow cancer that is resistant to several standard therapies, according to results of a clinical trial published in The New England Journal of Medicine in August.

The Study:  STORM Part 2

This trial tested selinexor with dexamethasone, a combination that significantly knocked down the cancer in more than a quarter of patients, including two patients who went into complete remission. Proteins and messenger RNAs play an important part of cancer cell growth, and selinexor has an unprecedented mechanism that blocks the export of protein and messenger RNAs from the nucleus of the cancer cell to the cytoplasm, causing the cancer cell to die. This therapy caused at least a minimal response in almost 40 percent of patients who had multiple myeloma, a cancer of a type of white blood cell called a plasma cell.

The clinical trial, called the STORM Part 2 Study, studied the response of 122 patients taking selinexor and dexamethasone, both oral drugs, in trials across the United States and Europe. Mount Sinai enrolled a quarter of the patients in the international study.

Results

Patients generally saw a response to the drugs within one or two months. While there was no organ toxicity, side effects included low blood count (without bleeding), nausea, vomiting, a lack of appetite, and fatigue.

This study is a major milestone in myeloma research for the team at The Tisch Cancer Institute at Mount Sinai, which recently launched a new Center of Excellence for Multiple Myeloma. The new Center brings together internationally recognized physicians to provide comprehensive, compassionate care for patients with multiple myeloma and related diseases and to advance innovative research and personalized treatments that lead to cures. Dr. Jagannath will serve as Director of the Center, which treats the largest number of myeloma patients in the country.

Selinexor

It is a selective inhibitor of nuclear export used as an anti-cancer drug. It works by quasi-irreversibly binding to exportin 1 and thus blocking the transport of several proteins involved in cancer-cell growth from the cell nucleus to the cytoplasm, which ultimately arrests the cell cycle and leads to apoptosis. It is the first drug with this mechanism of action.

Selinexor was granted accelerated approval by the U.S. Food and Drug Administration in July 2019, for use as a drug of last resort in people with multiple myeloma. In clinical trials, it was associated with a high incidence of severe side effects, including low platelet counts and low blood sodium levels. The drug was developed by Karyopharm Therapeutics of Newton, MA.

Clinical Investigator Comments

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 noted. “This study proved that a novel, first-in-class drug with a new mechanism of action can kill a patient’s cancer cells.”

Lead Research/Investigator

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

Ajai Chari, MD, Director of Clinical Research in the Multiple Myeloma Program at the Tisch Cancer Institute at Mount Sinai

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