A 13-year-old has been freed from leukemia with the help of a revolutionary new treatment, doctors say.
Alyssa, whose surname was not used by the family, was diagnosed with T-cell acute lymphoblastic leukemia in 2021.
Conventional treatment, including chemotherapy and bone marrow transplantation, failed to prevent the disease from recurring.
In May, Alyssa received universal CAR (chimeric antigen receptor) T cells, prefabricated from healthy donors, as part of a clinical trial.
Twenty-eight days later, she was in remission and was able to undergo a second bone marrow transplant.
She is said to be “recovering well” at home while her condition is monitored at Great Ormond Street Hospital where she is being treated.
Alyssa’s mother, Kiona, said the family was “on a weird cloud nine”, adding: “Hopefully this proves that the research works and they can give it to more children – all of which need to be doing.”
Alyssa, from Leicester, said: “Once I do that, people will know what they need to do anyway, so doing it will help people – of course I’ll do it.”
If not treated, the next step is palliative care
Edit prefabricated cells using new techniques.
The edited CAR T cells can then be given to patients so they can quickly find and destroy T cells in the body, including cancerous cells.
The person can then undergo a bone marrow transplant to restore their depleted immune system.
Without treatment, the only next step for Alyssa is palliative care, scientists say.
Dr Robert Chiesa, GOSH consultant for bone marrow transplantation and CAR T cell therapy, said the results were “remarkable” but said Alyssa’s condition must continue to be monitored over the next few months.
He said: “Since Alyssa developed leukemia last May, she has never been in complete remission – neither chemotherapy nor the first bone marrow transplant.
“She was leukemia-free only after she received CD7 CAR-T cell therapy and a second bone marrow transplant at GOSH.”
“Sick children end up with better futures”
Professor Waseem Qasim, Consultant Immunologist at GOSH, said: “This is a great example of how we can connect cutting-edge technology in the laboratory with real outcomes for patients in the hospital, through our expert team and infrastructure.
“This is our most complex cell engineering to date, paving the way for other new treatments and ultimately a better future for sick children.”
The study will be presented this weekend at the American Society of Hematology annual meeting in New Orleans.
Great Ormond Street Hospital hopes to enroll up to 10 patients with T-cell leukemia who have exhausted all conventional options for the clinical trial. They will be referred by a specialist.