Precision Medicine Tool for Relapsed, Refractory Multiple Myeloma


A new precision medicine platform utilizing both DNA and RNA sequencing helps identify targeted therapy options for patients with relapsed or refractory multiple myeloma.

While recent therapeutic advancements have improved survival rates in multiple myeloma, relapse remains prevalent and often leads to mortality. A significant challenge in the treatment of patients with relapsed disease is the timely identification of personalized treatment options. Currently, approaches used in precision oncology aim to match specific DNA mutations to drugs, but including genome-wide RNA profiles may have clinical utility.

Alessandro Laganá, PhD, Icahn School of Medicine Mount Sinai Medical Center (New York, NY), and colleagues designed a precision medicine novel computational platform for relapsed or refractory multiple myeloma on the basis of DNA and RNA sequencing. Researchers modeled their approach after conventional DNA-based approaches to integrate somatic mutations and copy number alterations with RNA-based drug repurposing and pathway analysis. A total of 64 patients with relapsed or refractory disease were included in a pilot precision medicine clinical trial utilizing this approach.

Results of the trial were published in JCO Precision Oncology (online August 8, 2018; doi:10.1200/PO.18.00019).

Dr Laganá and colleagues were able to generate treatment recommendations in 63 of the patients. Treatment was implemented in 26 of these patients, of which 21 were assessable. Researchers reported that 11 patients received a drug that was based on RNA findings, eight patients received a drug that was based on DNA, and two patients received a drug that was based on both RNA and DNA.


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Additionally, researchers noted that 16 of the assessable patients demonstrated a clinical response—including a 66% overall response rate—and five patients had ongoing responses at the conclusion of the trial. Median duration of response was 131 days.

“Our results…represent proof of principle of how RNA sequencing can contribute beyond DNA mutation analysis to the development of a reliable drug recommendation tool,” authors of the study concluded. “Although more work still needs to be done, the precision medicine paradigm holds great promise for the treatment of cancer and represents a unique opportunity for accelerated development and application of novel therapeutic approaches.”—Zachary Bessette