The mission of the MMRF has always been to provide a cure for each and every patient. We know that multiple myeloma is different in every patient. Our goal is to generate and collect as much patient data as possible and make it available to researchers worldwide, to speed new discoveries and propel new clinical options for myeloma patients into the clinic as quickly as possible.
The MMRF is laser-focused on the promise of precision medicine, meaning treating each patient with therapies that work best for them. This year marks the 10th anniversary of the first sequencing of the multiple myeloma genome, a groundbreaking MMRF initiative made possible by patients who generously donated their samples to our Tissue Bank. This effort demonstrated the first actionable mutations detected in myeloma and led directly to our landmark CoMMpass Study, which is the largest genomic dataset in all of cancer. The actionable mutations discovered through CoMMpass paved the way for our MMRC MyDRUG® trial, where for the first time, myeloma patients are treated with targeted therapies already approved for these actionable mutations in other cancers.
Our Chief Scientific Officer, Dr. Daniel Auclair, recently gave an exciting presentation on our MyDRUG trial at the European School of Haematology’s 3rd How to Diagnose and Treat Multiple Myeloma virtual meeting. Dr. Auclair described MyDRUG and showed some of the first data from the trial, which demonstrated that a drug that targets a specific genomic mutation in a myeloma patient was effective in treating their disease.
MyDRUG is a platform trial, where eligible relapsed refractory myeloma patients receive different therapies based on their genomic profile. Eligible patients undergo genomic sequencing to determine if they have any actionable gene mutations that can be treated with therapies that target that specific mutation, then enter the arm in the trial where they will be treated with that targeted therapy, along with an all-oral backbone therapy of Ninlaro®, Pomalyst®, and dexamethasone. Find out more about the MyDRUG trial here or by contacting our Patient Navigation Center.
The RAS/RAF mutation arm of the trial is now fully enrolled, and interesting results are beginning to emerge. Dr. Auclair described the case of one patient who relapsed 22 months after their first line of treatment (a stem cell transplant (ASCT) plus maintenance therapy) and 4 months after their second line of therapy. Genomic testing revealed an actionable NRAS mutation; the patient was enrolled in MyDRUG and received Cotellic® (cobimetinib), a therapy that is FDA approved for melanoma patients with NRAS mutations, and is only available to myeloma patients through the MyDRUG trial. Cotellic targets the pathway activated by the NRAS mutation. The patient experienced an immediate decrease in their M-spike when treated with the targeted therapy alone; the response became even deeper when the backbone therapy was added, and response is still ongoing at 12 months. While this is a single case, if future data validate these findings, this treatment could become an option for the 53% of myeloma patients who harbor RAS/RAF mutations.
These results demonstrate the potential benefit of a precision medicine approach for patients whose disease may not respond well to more traditional myeloma therapies. As the MyDRUG trial continues, arms that show evidence of activity will continue enrolling, while arms that fail to provide patient benefit may be discontinued. More arms will be added as more targeted therapies become available.
The MMRF continues to collect even more patient data through our CureCloud® patient registry and make it available to researchers, bringing the promise of using precision medicine approaches as a tool to provide personalized treatment and a cure for each and every patient closer to reality. We encourage every myeloma patient to participate in the CureCloud. Learn more about this study here.