Umbrella trials and platform trials are shaping the future of cancer research. These trials, which allow researchers to study several treatments at once, hold the promise of making effective, highly targeted, new drugs available—and available more quickly—to high-risk multiple myeloma patients.

With its launch of MyDRUG (Myeloma – Developing Regimens Using Genomics), the first ever multiple myeloma platform trial, the MMRF is leading the charge in this new research approach. The MyDRUG trial was developed based on key findings from the MMRF CoMMpass Study and the Multiple Myeloma Research Consortium (MMRC) Molecular Profiling Initiative, which revealed that myeloma is not one uniform disease but rather a variety of different disease subtypes defined by different genetic mutations. In other words, an individual’s genomic blueprint determines how myeloma behaves.

Using this information, the MyDRUG trial is testing different treatments that are known to specifically target genetic mutations in patients with high-risk myeloma. In the MyDRUG trial, high-risk patients are those who received treatment at diagnosis but whose response to this treatment did not last long. Historically, these patients have unfavorable long-term results. Therefore, the idea behind MyDRUG is to offer treatments to these patients that are different from what is currently available…and thus different from what has not worked. The strategy behind MyDRUG is to individualize treatments to these patients based on their specific genetic blueprint rather than simply applying the conventional, one-size-fits-all, approach to treatment. Ultimately, MyDRUG is a way to combine information about the biology of myeloma with new treatments.

Patients enrolled in the MyDRUG trial undergo genome sequencing, a test that analyzes a person’s genetic structure and can reveal the presence of cancer-related mutations. Based on the results, patients are matched to a specific treatment that targets their unique myeloma subtype and assigned to the branch of the MyDRUG trial that is studying that treatment. In addition to receiving the specific, subtype-matched treatment, all patients are given the standard-of-care regimen of Ninlaro + Pomalyst + dexamethasone. High-risk patients with no mutations detected receive a four-drug combination (Darzalex + Ninlaro + Pomalyst + dexamethasone)—a treatment that is otherwise unavailable.

Currently, MyDRUG is testing the following treatments, many of which are already approved for use in other cancers, that target specific genetic mutations or cytogenetic abnormalities:

  1. Cobimetinib (RAF/RAS mutations)
  2. Enasidenib (isocitrate dehydrogenase [IDH] mutations)
  3. Abemaciclib (cyclin-dependent kinase [CDK] mutations)
  4. Erdafitinib (fibroblast growth factor receptor [FGFR] mutations)
  5. Venetoclax (chromosomal translocation t[11;14])
  6. Any other mutations not listed

Another unique feature of the platform approach is that the trial can enroll patients indefinitely (by contrast, traditional clinical trials have set enrollment deadlines). Treatments that are shown not to work are removed from the study. Also, when additional information about genetic mutations and/or new drugs that target those mutations becomes available, the treatments can be added to the trial.

The MyDRUG trial is led by MMRF CSO Dr. Daniel Auclair and Dr. Shaji Kumar at the Mayo Clinic in Rochester, Minnesota and is enrolling patients at 17 cancer centers and academic research institutions throughout the United States. You can listen to Dr. Kumar review the MyDRUG trial and other innovative clinical trials at
To learn more about joining the MyDRUG trial, contact a Nurse Patient Navigator at the MMRF Patient Support Center at 888-841-6673.

Additional Resources
MMRF. The MMRF Launches MyDRUG – First-ever Platform Clinical Trial in Multiple Myeloma [press release]. November 29, 2018.
MMRF. What is the MyDRUG study?
NIH. US National Library of Medicine. Myeloma-developing regimens using genomics (MyDRUG). Updated December 12, 2018.