A therapy used in preparing patients for bone marrow transplantation which has been developed in Southampton over the past 20 years has been awarded Orphan Drug Status (ODD) by the United States Food and Drug Administration (FDA).
Bone marrow conditioning is performed prior to hematopoietic stem cell transplant (HSCT), a procedure where the patient’s bone marrow is cleared of cells and replaced by stem cells (cells that can develop into different types of cells), to encourage production of new bone marrow that produces healthy blood cells.
Traditional conditioning regimens are associated with morbidity and mortality from chemotherapy, limiting their use, particularly in paediatric transplantation and for rare diseases.
TLX66 (90Y-besilesomab) was developed by Southampton researchers at both the University of Southampton and Southampton General Hospital over the past two decades.
TLX66 has the potential to add to the depth of conditioning, thereby more effectively destroying disease-causing cells. In addition, TLX66’s potential to reduce the toxicity of existing conditioning regimens could increase the number of patients that are eligible for transplant and reduce the long-term side-effects associated with conventional conditioning regimens.
HSCT is being used increasingly in malignant haematological conditions, such as multiple myeloma and acute myeloid leukaemia, and in non-haematological malignancies and rare diseases including AL-amyloidosis.
The first in man Phase I trial started in Southampton in 2002 with other Phase I and Phase II trials taking place since, in collaboration with other centres to test the therapy autologous transplants for myeloma, AL-amyloidosis and in Paediatric allogeneic transplantation.
Dr Kim Orchard, Senior Lecturer and Consultant Haematologist in the Faculty of Medicine, has led the research since the beginning.
Dr Orchard commented: “We are really pleased to receive this status from the FDA – this work has only been possible through the collaboration and enthusiasm of teams within the departments of nuclear medicine, radiopharmacy, medical physics and the BMT program in Southampton.
“This work has led to improved responses post-transplant in adults and children undergoing HSCT in the early phase trials and we are now working with Telix to develop further clinical trials.
“The technology has the potential to improve transplant outcomes and could replace conventional conditioning with total body irradiation (TBI) thus avoiding the early and late organ damage associated with TBI.”
The therapy previously achieved Orphan drug status in the EU in 2018.