Kiadis Pharma has received approval to test in the UK an immune therapy to address the big challenges of bone marrow transplants, currently the only cure for blood cancers.
Kiadis Pharma, based in Amsterdam, is working on therapies to improve the outcome of hematopoietic stem cell transplantation (HSCT), a common treatment for multiple types of blood cancer. The company has just received approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) as well as hospitals in Manchester and Birmingham to start a Phase I/II trial with ATIR201 to treat thalassemia.
The trial is expected to recruit up to 10 patients with beta-thalassemia major and expand into Germany once Kiadis receives approval. First results are expected in late 2017.
Thalassemia is a genetic disease that results in insufficient hemoglobin production, leading to a shortage of oxygen in the body. ATIR201 is an adjunctive immune therapy to HSCT, the only treatment that offers a cure for this disease. Kiadis’ therapy provides the patient with functional immune cells to protect them from infections and graft-versus-host-disease (GVHD),