European Hematology Association - Genetic Modification of Autologous Hematopoietic Stem Cells: A Potential Treatment for Mucopolysaccharidosis Ty

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- Mucopolysaccharidosis type I Hurler (MPS-IH) is caused by a deficiency in alpha-L-iduronidase (IDUA) and is typically managed by allogeneic hematopoietic stem cell (HSC) transplantation, which has shown high rates of success when patients are treated at a young age. However, skeletal abnormalities remain and progressive neurocognitive deterioration over time severely affect a patient's quality of life. In this first-in-human phase I/II clinical study, we aimed to investigate the efficacy of treatment with autologous HSC genetically modified to overexpress human IDUA in 8 patients with MPS-IH.

All patients showed rapid hematologic recovery and any IDUA antibodies were cleared within 3 months after transplantation. Adverse events were generally mild and comparable to other autologous HSC transplantation procedures. Importantly, all patients exhibited the presence of transplanted cells in the bone marrow and peripheral blood as early as day 30 and showed high levels of IDUA activity in blood and cerebrospinal fluid, which was maintained at 12-months follow-up. These findings were paired with stable cognitive and language performances, continued motor development, and improved clinical and laboratory parameters. In summary, these data highlight the therapeutic potential of genetically modified autologous HSC transplantation in patients with MPS-IH, potentially improving clinical outcomes with a favorable safety profile.

The results of this study will be presented by Professor Maria Ester Bernardo in the Presidential Symposium.

Presenter: Professor Maria Ester Bernardo

Affiliation: Ospedale San Raffaele, Milan, Italy

Abstract: #S102 HEMATOPOIETIC STEM CELL-GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE I, HURLER: EVIDENCE OF EXTENSIVE METABOLIC CORRECTION AND EARLY CLINICAL RESPONSE

About the EHA Annual Congress: Every June, EHA organizes its Annual Congress in a major European city. This year, due to the persisting COVID19 pandemic, EHA organizes a virtual Congress for the second time. The Congress is aimed at health professionals working in or interested in the field of hematology. The scientific program topics range from stem cell physiology and development to leukemia; lymphoma; diagnosis and treatment; red blood cells; white blood cells and platelet disorders; hemophilia and myeloma; thrombosis and bleeding disorders; as well as transfusion and stem cell transplantation.

Website: www.ehaweb.org file://eha.local/gebruikers$/i.vanderbeek/AppData/Roaming/Microsoft/Outlook/Temp/www.ehaweb.org

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CONTACT: European Hematology Association, Ineke van der Beek, Tel: +31 (0)6 2011 1055, Email: communication@ehaweb.org