Children with Aggressive Blood Cancer Offered Treatment Cure Hope
In a groundbreaking development at Great Ormond Street Hospital, children with aggressive blood cancer are being offered a new treatment that experts believe could be the key to curing the disease. The treatment, which targets T-cell acute lymphoblastic leukaemia (T-ALL), involves CAR T-cell therapy that specifically targets a protein found only on cancerous white blood cells.
CAR T-cell therapy works by genetically modifying the body’s immune cells to identify and destroy cancer cells. While this therapy has shown success in other cancers, it has not been effective in treating T-ALL. However, researchers now have a clearer target for attacking this deadly cancer through the identification of the protein CCR9 on cancerous T cells.
Dr Sara Ghorashian, a consultant haematologist at GOSH, along with her team from GOSH and the University College London Great Ormond Street Institute of Child Health, are conducting a new study recruiting 12 children with T-ALL that has relapsed or is resistant to conventional treatments like chemotherapy and bone marrow transplants. The study, financed by a £2.4 million grant from the Great Ormond Street Hospital Charity, will commence in April next year at both GOSH and University College London Hospital.
A significant aspect of this research is the simultaneous testing of the treatment on both children and adults, marking a departure from the traditional approach of delaying paediatric clinical trials compared to adult trials. This innovative strategy aims to expedite access to targeted therapies for both children and adults, preventing delays in treatment availability for children with aggressive cancers such as T-ALL.
Dr Ghorashian emphasised the need for early-phase clinical studies in paediatrics to ensure tailored treatments for children with cancer, as opposed to borrowing dosing and delivery methods from adult treatments. The therapy’s success in clearing leukaemia in lab and animal studies offers hope for its effectiveness in treating T-ALL, with the added benefit of reduced side effects compared to traditional treatments like chemotherapy and bone marrow transplants.
Maris Hanson, who tragically lost her son Frank to T-ALL, is now actively raising funds and awareness for GOSH Charity. Frank’s story underscores the urgent need for improved treatments for children with aggressive blood cancers, driving efforts to establish a new Children’s Cancer Centre at GOSH. Dr Aoife Regan, GOSH Charity’s director of impact and charitable funding, expressed pride in funding this critical study, which may pave the way for a more efficient clinical trial design benefiting seriously ill children in the future.
The hope is that this innovative approach to treating aggressive blood cancer in children will not only provide new treatment options but also set a precedent for inclusive and timely clinical trials for both paediatric and adult patients. The ultimate goal is to ensure that no child is left behind in accessing life-saving treatments, ushering in a new era of cancer care that prioritises the needs of children battling these devastating diseases.