Hematology

Gene-editing therapy in sickle cell disease and advances in multiple myeloma dominated hematology news in 2020.

The UCSF hematologist discusses the growing portfolio for the sickle cell therapy after ASH 2020.

The historic phase 3 HOPE-B trial shows all but 1 treated patient discontinued prophylaxis after a single dose of etranacogene deaparvovec.

The second process of ARU-1801 in a new patient resulted in up to 4 times greater engraftment of transduced hematopoietic stem cells and progenitors at 6 months.

What started out as a journey to better understand regulatory T cells has now led to an intriguing approach with an investigational cell therapy designed to prevent the risk of graft-versus-host disease and to improve relapse-free survival rates in patients undergoing hematopoietic stem cell transplantation.

An off-the-shelf CAR T-cell therapy that targets B-cell maturation antigen, ALLO-715, elicited responses in heavily pretreated patients with relapsed/refractory multiple myeloma in early findings from a first-in-human study presented at the 2020 ASH Meeting.

Treatment with the BCMA-targeted CAR T-cell therapy idecabtagene vicleucel was found to yield clinically meaningful improvements in the quality-of-life of triple-class exposed patients with relapsed/refractory multiple myeloma.

December 6, 2020 - Odronextamab, is a novel CD20xCD3 bispecific antibody, continues to show intriguing antitumor activity and an acceptable safety profile in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, including those who have previously received chimeric antigen receptor T-cell therapy.

The furthest-progressed gene-edited therapy for a genetic disease reported historic efficacy data in ongoing phase 1/2 trials.

The complexities associated with the disease and lack of validated risk predictors makes such a model challenging to implement.

In adolescent patients with sickle cell disease, hydroxycarbamide therapy was associated with improvements in neurocognition variables such as working memory, verbal memory, and nonverbal IQ, according to study findings.

The Institute for Clinical and Economic Review (ICER) published a draft evidence report on crizanlizumab (Adakveo), voxelotor (Oxbryta), and L-glutamine (Endari) for sickle cell disease, as well as a draft scoping document on valoctocogene roxaparvovec, an investigational gene therapy, and emicizumab (Helimbra) for hemophilia.

Adult patients with beta thalassemia will now have an FDA-approved treatment available with luspatercept-aamt (Reblozyl). The therapy treats the rare inherited blood disorder, which requires patients to have regular red blood cell transfusions.

The only thing that should matter in these patients is their genetic code, but often it’s your zip code that really restrains your ability to access the healthcare you deserve, explained Ahmar Zaidi, MD, pediatric hematologist-oncologist, Comprehensive Sickle Cell Center, Children's Hospital of Michigan, when discussing the possibility of a gene therapy for sickle cell.

Neil Minkoff, MD, chief medical officer and vice president, EmpiraMed, discusses his hopes if the first gene therapy for sickle cell disease is approved and if he has any concerns with payer reactions.

Voxelotor, a first-in-class oral therapy, is both safe and effective in sickle cell disease, according to a phase 1/2 randomized study assessing the drug. These findings were consistent across all doses, ranging from 500 to 1000 mg.

Gene therapy provides an opportunity for every patient to become not a patient, to become an individual, and you can’t put a cost on that, explained Sophie Schmitz, BA, MA, managing partner, Partners4Access.

Crizanlizumab is being developed to prevent painful and unpredictable vaso-occlusive crises in patients with sickle cell disease.

The therapy attempts to restore fetal hemoglobin production.

The first patient has been treated in a phase 2b dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. Once the dosing of AMT-061 is confirmed, the safety and efficacy of the therapy will be evaluated in the global phase 3 HOPE-B clinical trial.

The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.

The FDA gave a gene therapy for bleeding in hemophilia B breakthrough status.

Researchers discovered a clinically translatable gene therapy protocol, which is a gene delivery system that introduced the production of therapeutic clotting proteins in platelets, so that the inhibition of inactivating antibodies is avoided.

A gene therapy treatment for men with hemophilia B proved effective, and the researchers involved in the study estimate it saved $2.5 million for the trial's patients.

Scientists have successfully engineered platelet cells in dogs to express higher levels of factor VIII, paving the way for further tests in humans.