UK medics laud world-first resistant leukaemia treatment
London: Doctors in Britain have hailed a pioneering treatment for an aggressive form of leukaemia, after a teenager became the first patient to be given a new therapy and went into remission. The 13year-old girl, identified only as Alyssa, was diagnosed with T-cell acute lymphoblastic leukaemia in 2021. But her blood cancer did not respond to conventional treatment, including chemotherapy and a bone marrow transplant.
She was enrolled on a clinical trial of a new treatment at London's Great Ormond Street Hospital for Children (GOSH) using genetically engineered immune cells from a healthy volunteer.
In 28 days her cancer was in remission, allowing her to receive a second bone marrow transplant to restore her immune system. Six months on, she is "doing well" back home in Leicester and receiving follow-up care. "Without the experiment, Alyssa's only option was palliative care," the hospital said on Sunday.
Robert Chiesa, a GOSH consultant, said her turnaround had been "quite remarkable", although the results still nee ded to be monitored and confirmed in the next few months.
Acute lymphoblastic leukaemia (ALL) is the most common kind of cancer in children and affects cells in the immune system, known as B cells and T cells, which fight and protect against viruses.
GOSH said Alyssa was the first patient known to have be en given base-edited T cells, which involves chemically converting single nucleotide bases letters of DNA codewhich carry instructions for a specific protein. Researchers at GOSH and University Colle ge London helped develop the use of genome-edited T cells to treat B-cell leukaemia in 2015.
But to treat some other types of leukaemia, the team had to overcome the challenge that T cells designed to recognise and attack cancerous cells had ended up killing each other during the manufacturing process. Multiple additional DNA changes were needed to the base-edited cells to allow them to target cancerous cells without damaging each other.
"This is a great demonstration of how, with expert teams and infrastructure, we can link technologies in the lab with real results in the hospital for patients," said GOSH consultant immunologist and professor Waseem Qasim. "It's our most sophisticated cell engineering so far and paves the way for other new treatments".
Alyssa said she was spurred to take part in the trial notjust for herself but for other children. The researchers we re presenting their findings at the American Society of Hematology's annual meeting.