The Miracle of Alyssa: Girl, 13, defeats terminal leukemia after revolutionary gene therapy
LEICESTER, ENGLAND: A teenage girl has been cleared of her terminal cancer after using a revolutionary new type of gene therapy that scientists have described as the most sophisticated cell engineering to date. Alyssa, 13, from Leicester, had already undergone chemotherapy and an initial bone marrow transplant - in hopes to alleviate her leukemia, but without success.
Scientists said that without the cancer treatment the only next step she would be left with is palliative care. But after receiving an infusion of T-cells altered with the help of the first-ever use of base-edited cell therapy at Great Ormond Street Hospital for Children (GOSH), Alyssa is recovering. She has been in remission for six months. These pre-manufactured cells, which a healthy volunteer donated, were edited using the new technology which allowed them to hunt down and kill cancerous T-cells without attacking each other.
READ MORE
Breast cancer in men: Shame and ignorance obscures a lethal American health issue
Alyssa spoke about the experimental new treatment: "Once I do it, people will know what they need to do, one way or another, so doing this will help people - of course, I'm going to do it."
She became the first patient enrolled in a new clinical trial, funded by the Medical Research Council, during which she was given universal CAR (Chimeric Antigen Receptor) T-cells that had been pre-manufactured from a healthy volunteer donor in May this year. "The doctors have said the first six months are the most important,” said her mother, Kiona to GOSH. "We don’t want to get too cavalier, but we kept thinking: 'If they can just get rid of it, just once, she’ll be OK.' And maybe we’ll be right." She was also able to have a second bone marrow transplant and is said to be "doing well at home" as she continues her recovery with follow-up monitoring at GOSH.
"This is our most sophisticated cell engineering so far, and it paves the way for other new treatments and ultimately better futures for sick children," said consultant immunologist Professor Waseem Qasim, one of the project’s leaders. "We have a unique and special environment here at GOSH that allows us to rapidly scale up new technologies and we're looking forward to continuing our research and bringing it to the patients who need it most."
“We are in a strange cloud nine, to be honest," said Kiona while admitting that she felt "amazing" at home. "Hopefully, this can prove the research works and they can offer it to more children - all of this needs to have been for something," she added.
While Dr Robert Chiesa, consultant in bone marrow transplant and CAR T-cell therapy at GOSH, described the outcome as "quite remarkable", he cautioned that it must be monitored and confirmed over the next few months.
T-cells are a type of white blood cell which are part of the immune system and develop from stem cells in the bone marrow. Moreover, T-cell leukemia affects this type of white blood cell. Alyssa, who was diagnosed with T-cell acute lymphoblastic leukemia (T-ALL) in 2021, was given all the conventional treatments, but the prospects of her survival remained grim at that time. According to the DailyMail, T-ALL is the most common cancer in kids and young people, affecting 500 each year, and the survival rate for those whose T-ALL has relapsed is just 10 percent.