The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes.
Dr. Stephanie Lee, of the Fred Hutchinson cancer center and former ASH president commented at a press briefing that the studies presented will focus on new drug targets, genetic disorders, and malignant diseases.
Multiple Myeloma
Promising response rates and additional disease control are shown in the first study following the enrollment of 288 patients. The drug talquetamab generated a significant response for patients who had not responded to previous treatment. A total of 74% of patients who received 0.4 mg/kg of talquetamab each week and 73% of patients who received 0.8 mg/kg twice each month saw a significant improvement in their cancer after treatment.
Talquetamab binds to multiple myeloma cells and T-cells, thereby activating an immune reaction that destroys myeloma cells.
Immune Thrombocytopenia
In the second study, fifty percent of patients with immune thrombocytopenia (ITP) who were administered efgartigimod (EFG) experienced an improved platelet count, thus lowering the risk of severe bleeding.
Platelets are blood cells that help to control bleeding. ITP is a rare and chronic autoimmune disease that increases the reduction of platelets.
The normal platelet count is between 150,000 and 400,000 per microliter of blood. ITP patients generally have a count of under 50,000 or as low as 20,000. The patients in this study had a median platelet count of approximately 30,000 per microliter.
EFG medication reduces autoantibodies proven to cause an increase in clearing platelets in ITP patients. 131 patients from six countries were enrolled in the study.
The primary endpoint was met at the twenty-four-week mark with 21.8% of participants receiving EFG versus 5% who had received a placebo.
Beta Thalassemia
The third study confirmed that beta thalassemia patients were transfusion free after only one gene therapy treatment and experienced a significant improvement in their quality-of-life.
The study was the largest of its kind thus far. Over 80 percent of participants who previously had depended on continuous blood transfusions to remain alive were transfusion free after receiving only one transfusion of their own stem cells that had been modified to correct the disease-causing genetic mutation.
Patients in this study have an acute type of beta thalassemia which is a disease whereby the body does not make a sufficient amount of hemoglobin. It is a molecule found in red blood cells that carries oxygen throughout the body’s cells.
Patients in this study were given their own blood-forming stem cells that were modified genetically with the addition of healthy copies of the beta-globin gene.
63 patients in four studies received a procedure involving the harvesting of their blood-forming stem cells. The cells were modified genetically and re-infused into the patients who had received an initial dose of chemotherapy to kill the remaining unhealthy stem cells.
49 people were transfusion independent for a minimum of one year. A total of 68 patients in two studies followed by 89 patients in two other studies were eventually transfusion-independent.
The therapy, known as beti-cel, was FDA approved for the treatment of beta-thalassemia.
Sickle Cell Disease
The fourth study involving sickle cell disease (SCD) evaluated the cost-effectiveness of a gene therapy for the illness. The research found that, if effective, a gene therapy would be cost-effective for this disease.
