CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin

Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. The results were published in Haematologica.

Researchers Discover Common Mechanism Causing Autoimmune Disease and Blood Cancers

In a study by Yale Cancer Center, researchers report on the discovery of a common mechanism that promotes both autoimmune diseases and blood cancers, including the blood diseases Acute Lymphoblastic Leukemia (ALL), Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL).

Yale Study Shows Combined Liver–Cytokine Humanization Rescues Circulating Red Blood Cells for Testing of Novel Therapeutics

In a new study by the Yale Department of Immunobiology and Yale Cancer Center, researchers report combined liver and growth factor humanization enhances human red blood cell production and survival in circulation the immunodeficient murine host.

Researchers Discover Mechanism to Overcome Drug-Resistance in B-Cell Acute Lymphoblastic Leukemia

In a new study led by Yale Cancer Center, researchers have discovered a novel metabolic gatekeeper mechanism for leukemia. This mechanism depends on a molecule called PON2, which could lead to a new treatment for the disease. The findings were published online today in the Proceedings of the National Academy of Sciences.

UT Southwestern Leads National Efforts Around Childhood Blood Disorders

DALLAS – Oct.15, 2020 – When a child has a rare blood disorder, clinicians can struggle to find the best diagnostic and treatment methods. New research led by UT Southwestern shows the effectiveness of a treatment for aplastic anemia and reveals the range of diagnosis and treatment options used by hospitals around the country for a related disease – myelodysplastic syndrome (MDS).

Six patients with rare blood disease are doing well after gene therapy clinical trial

UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.

Likelihood of Prenatal Screening for Blood Disorders Varies Between Type of Healthcare Provider

A Rutgers Cancer Institute of New Jersey investigator and other collaborators examined prenatal screening practices for blood disorders between different types of care providers and found different variations in prenatal guidance.