Gene-editing therapy in sickle cell disease and advances in multiple myeloma dominated hematology news in 2020.
This year’s popular pieces in blood cancers and blood diseases appeared early in the year and offered glimpses into research that would bear greater fruit at the end of the year during the recent annual meeting of the American Society of Hematology. Here are the most read hematology articles on AJMC.com:
5. MRD: We’re Aware of It, Say Oncologists, but We Don’t Use it for Decisions.
An August survey from investigators at the University of Chicago Medical Center found that the concept of measurable, or minimal, residual disease (MRD) in multiple myeloma (MM) has reached clinicians but they had not yet incorporated it into decision-making. A total of 84 respondents completed the survey over a 2-month period, with 55% responding before FDA approved an NGS assay for MRD in MM. Whether the survey results still hold, the use of MRD has made its way into clinical research as an end point.
4. Once-Weekly KRd Shows Promise in Newly Diagnosed MM.
Patients with newly diagnosed multiple myeloma (MM) often receive a twice-weekly triplet combination of carfilzomib (Krypolis), lenalidomide, and dexamethasone (KRd), but data published in October suggest that once-a-week dosing can be equally successful. The study reported an overall response rate of 97%, including those with a very good partial response (VGPR) or better among those who had a stem cell transplant and an ORR of 93.3% among those who did not have a stem cell transplant.
3. CAR T-Cell Therapies Show Promise for Multiple Myeloma, but Hurdles Remain.
In July, a review article in Frontiers in Immunology outlined the progress and challenges in bringing chimeric antigen receptor (CAR) T-cell therapy to the treatment of MM, which has already seen advances in the development of immunomodulatory drugs. But MM is still incurable, and 2020 saw significant leaps forward with solutions that involve targeting the B-cell maturation antigen (BCMA), which the authors note is highly expressed on the surface of malignant plasma cells, with restricted expression in normal tissues/cells except for a low-level expression in mature B-cells. Other possible targets include CD38, CD138, CD19, and immunoglobulin kappa light chain (Ig-Kappa). The greatest success has been in targeting BCMA, as other targets have yielded high response rates but not durable responses.
2.Janssen's Wildgust Breaks Down Bispecific Antibodies in Development for Non-Small Cell Lung Cancer and Multiple Myeloma.
This interview with Janssen’s Mark Wildgust, PhD, vice president of Global Medical Affairs/Oncology, appeared in the special recap issue of Evidence-Based Oncology™ from the American Society of Clinical Oncology (ASCO). During the meeting, Janssen presented results from the CHRYSALIS study on amivantimab, a bispecific antibody under development to treat non–small cell lung cancer (NSCLC). The pharma giant, along with its parent company, Johnson & Johnson (J&J), had previously received a breakthrough therapy designation for teclistamab, another bispecific antibody indicated for potential treatment of multiple myeloma. Updated phase 1 results for teclistamab were recently presented during ASH 2020.
1. Revolutionary Treatment for Sickle Cell Disease Shows Promise in Clinical Trial.
Our January 2020 article on results from just 3 adult patients that had been presented at ASH 2019 offered a glimpse of what was to come: in this case, stem cells genetically engineered to produce healthy hemoglobin produced remarkable results in patients with sickle cell disease (SCD), a debilitating, incurable disease that results from painful sickle cell buildup, causing lack of oxygen. Dana-Farber Cancer Institute researchers targeted BCL11A, which preclinical research showed could regulate the switch of fetal hemoglobin to adult hemoglobin. The concept of targeting this protein has taken flight and expanded; a different research group took center stage at ASH 2020, focusing on the use of CRISPR technology to silence BCL11A with an infusion of modified stem cells.