The tremendous heterogeneity of multiple myeloma enables it to defy treatment, and its reputation for relapse means the disease is generally considered incurable. But as genetic sequencing and other molecular profiling techniques become more affordable, this formerly intractable cancer may be a prime target for strategy based on precision medicine. 

“I do think that is a direction we’re heading,” said Brian Van Ness, PhD, professor of genetics, cell biology, and development at the University of Minnesota, Saint Paul. “Particularly in myeloma, it is such an incredibly heterogeneous background that we’re faced with … To say one patient has the same disease as another, even though we’re calling them all myeloma, really does an injustice to the fact there’s lots of differences between 1 patient and another.”

Dr Van Ness and his coauthors laid out the case for personalizing myeloma treatment in a recent review article in Leukemia.1“As molecular profiling methods become increasingly affordable and reliable, it is easy to envision a future for disease management that is completely data driven,” the authors wrote. 

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But Dr Van Ness acknowledged the challenge of conducting large clinical trials comparing treatments when the goal is to provide each patient a completely individualized therapy, tailored to their genetic profile. “The question ultimately is, is the care going to show better outcomes when you profile individuals for their best individual therapy?”

In an attempt to answer that question, the Multiple Myeloma Research Foundation (MMRF) has launched a platform clinical trial called MyDRUG. The trial will consist of 6 treatment arms, each testing the efficacy of treatments tailored to each patient’s particular mutational profile.

“MyDRUG represents the most comprehensive effort so far in trying to address that,” said Shaji Kumar, MD, of the Mayo Clinic in Rochester, Minnesota. Dr Kumar is leading the trial.