Top 10 Multiple Myeloma Stories of 2019
Myeloma Research News brought you daily coverage of important findings, treatment developments, clinical trials, and other events related to multiple myeloma throughout 2019.
As a reminder of what mattered most to you in 2019, here are the top 10 most-read articles of last year with a brief description of what made them interesting and relevant to the myeloma community.
We look forward to reporting more news during 2020 to patients, family members, and caregivers dealing with multiple myeloma.
No. 10 – “FDA Delays Decision on Selinexor’s Approval for Heavily Treated Multiple Myeloma“
In the spring, the U.S. Food and Drug Administration was expected to make a decision on whether to approve selinexor in combination with the corticosteroid dexamethasone for myeloma patients who had failed at least three prior therapies. But after a recommendation from its Oncologic Drugs Advisory Committee, the agency decided to wait for the findings of the BOSTON Phase 3 clinical trial (NCT03110562), which would also reassure researchers after concerns regarding safety and trial design.
The selinexor combination was approved — with conditions — just a few months later, in June, for patients who received and failed at least four prior therapies. A full approval is dependent on the final BOSTON results.
Selinexor is sold as Xpovio by Karyopharm Therapeutics.
No. 9 – “Study Tracing Myeloma Progression from Precursor Conditions Seeks Participants“
In January, recruitment began for the PROMISE study, which hopes to identify the molecular changes that occur in people with precursor conditions who eventually develop multiple myeloma.
The study intends to recruit 50,000 participants, to include U.S. African-American adults, and people with a parent, sibling, or child with multiple myeloma or one of its precursor conditions, regardless of race. For more information on enrollment, visit the study’s website.
The ultimate goal is to provide useful information to support the development of therapies designed to stop the disease from progressing.
No. 8 – “GSK’s Belantamab Mafodotin Shows Promise for Relapsed or Refractory Multiple Myeloma“
Findings from a Phase 2 clinical trial called DREAMM-2 (NCT03525678) demonstrated that GlaxoSmithKline’s investigational antibody-drug conjugate, belantamab mafodotin, significantly reduced tumor burden in 60% of participants with hard-to-treat myeloma. It also helped them live without signs of disease progression for a median of one year.
The trial enrolled 196 patients who had failed at least three prior lines of therapy, had not responded to a proteasome inhibitor and an immunomodulatory agent, and had relapsed or were refractory to an anti-CD38 antibody. Additional findings from DREAMM-2 will be presented at an upcoming scientific meeting.
No. 7 – “Multiple Myeloma Patients Best Treated at Recognized Care Centers with High Volume, US Study Reports“
A study published in the Journal of the National Comprehensive Cancer Network showed that people with multiple myeloma who are treated by myeloma specialists at nationally recognized centers — which see larger numbers of patients — have greater chances of survival than those treated in their community by local oncologists.
The findings were based on medical records from 1,029 patients diagnosed between 2006 and 2012. While provider expertise and hospital volume overlap, the study demonstrated that both have a significant impact on patient survival. Only the highest-volume community oncologists — those seeing more patients than 80% of other oncologists in that community — matched the survival rates achieved by specialists at high-volume facilities, according to an October story in Myeloma Research News.
June came with news that a candidate chimeric antigen receptor (CAR) T-cell therapy for multiple myeloma, called bb2121, reduced tumor burden in 85% of heavily treated myeloma patients included in the CRB-401 Phase 1 clinical trial (NCT02658929). The therapy, being developed by Celgene and Bluebird Bio, completely eliminated all traces of cancer in nearly half of the study’s participants.
The trial enrolled people who had received at least three prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent, or were resistant to both types of medications.
The investigational treatment, which had a favorable safety profile, also eliminated the very small traces of cancer cells that remain after treatment and may cause relapse — a status called minimal residual disease negativity.
Early in the year, we reported on a Phase 3 clinical trial, called Myeloma XI (NCT01554852), showing that long-term maintenance treatment with Revlimid (lenalidomide) significantly improved the time patients live without the disease getting worse after first-line therapy.
Revlimid was tested in 1,137 patients who achieved at least a minimal response to first-line treatment, and were compared with 834 patients under observation only. The goal was to delay disease progression or death and extend overall survival. In addition to delaying disease worsening or death by a total of 19 months in the overall population, the maintenance therapy extended the lives of patients who received a stem cell transplant as part of their first-line therapy.
No. 4 – “Donor-derived CAR T-cell Therapy ALLO-715 Enters Clinical Testing for Multiple Myeloma“
Allogene Therapeutics announced in July it was launching the first clinical trial testing its “off-the-shelf” CAR T-cell therapy ALLO-715 in people with multiple myeloma who have failed at least three prior therapies.
Dubbed UNIVERSAL (NCT04093596), the single-arm, open-label, Phase 1 trial is recruiting up to 90 participants across five sites in the U.S. The goal is to study ALLO-715’s safety and preliminary signs of efficacy, including proportion of responders, duration of response, time to disease progression or death, and overall survival. For enrollment information, visit here.
In a presentation at last year’s ASH meeting, researchers from the University of Pennsylvania revealed that the gene editing technology CRISPR-Cas9 could be safely used to genetically modify a patient’s T-cells and create an effective CAR T-cell therapy.
In a pilot Phase 1 study (NCT03399448), the team evaluated a new engineered T-cell therapy, called NYCE, to treat up to 18 patients with advanced myeloma, sarcoma, or melanoma. The treatment was well-tolerated, so far, by the first three patients. A single administration resulted in the expansion and survival of NYCE T-cells in the blood, which effectively bound to their tumor target, NY-ESO-1, the researchers said.
The scientists are optimistic about these early results, and believe this approach may be used to treat several diseases.
No. 2 – “Keytruda Plus Standard Therapy Fails to Improve Outcomes in Multiple Myeloma“
Disappointing news was reported in September, after two Phase 3 trials — KEYNOTE-183 (NCT02576977) and KEYNOTE-185 (NCT02579863) — failed to demonstrate any benefits of Keytruda (pembrolizumab) as an add-on to standard therapy for both untreated and pretreated multiple myeloma patients.
The trials compared standard treatment with Revlimid or Pomalyst (pomalidomide), in combination with dexamethasone, with a combination of standard treatment plus Keytruda. The Keytruda combination increased the number of adverse events and led to worse survival outcomes than standard treatment. That prompted the FDA to close several clinical trials of Keytruda in multiple myeloma. The future of immune checkpoint inhibitors targeting the PD-1 and PD-L1 in multiple myeloma is now uncertain.
No. 1 – “Scientists Find ‘On/off Switch’ of Malignant MM Cancer Cells, May Lead to New Therapies, Study Says“
Our most-read article of 2019 reported on a study describing a new set of genes that work as an “on/off switch” for malignant multiple myeloma cancer cells. Specifically, these genes can “wake up” otherwise dormant, “asleep” malignant myeloma cells in the bone marrow, which will reactivate and travel to other regions in the body, giving rise to aggressive cancer metastases.
The findings may shed light into new therapeutic targets for the disease, either aiming to maintain malignant cells in a dormant state indefinitely or to deliberately wake them up and make them susceptible to being targeted with conventional chemotherapy.
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At Myeloma Research News, we hope these stories and our continued reporting throughout 2020 contribute to informing and improving the lives of everyone affected by MS.
We wish all our readers a happy 2020.
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