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Felt like palliative care was the only option left for a 13-year-old leukemia Patient Alyssa Tapley, until she became the first person in the world to receive a treatment that edits healthy immunity cells To fight cancer.
Leicester teen was diagnosed T-cell leukemia In May 2021, after a long period of what The family thought it was a cold.Viruses and general fatigue.
He did not respond Chemotherapy Or a bone marrow transplant, and he thought Cancer was “incurable” Unless a research opportunity was offered.
When Alyssa received therapy in 2022, she remained cautiously optimistic even after her leukemia was undiagnosed.
But three years later, at age 16, treatment proved life-changing. She has been discharged for long-term follow-up and has dreams of becoming a research scientist herself.
Alyssa said, “I decided to participate in the research because I thought even though it didn’t work for me, it might help others. Years later, we know it works and I’m doing really well. I did all the things you’re supposed to do as a teenager.”
“I’ve gone sailing, spent time away from home for my Duke of Edinburgh award, but even going to school is something I dreamed of when I was sick. I’m not taking anything for granted.
“Next on my list is to learn to drive, but my ultimate goal is to become a research scientist and be part of the next big discovery that can help people like me.”
Eight children and two adults with an aggressive form of leukemia that “seemed incurable” have since been treated at Great Ormond Street Hospital (GOSH) and King’s College Hospital (KCH).
About two-thirds of patients with T-cell acute lymphoblastic leukemia (T-ALL) involved in a clinical trial of the treatment — known as BE-CAR7 — are now cancer-free.
BE-CAR7, which was developed by scientists at Gosh and University College London (UCL), is a groundbreaking treatment that works by editing healthy immune cells to fight cancer.
Typically CAR T-cell therapy involves a doctor collecting T cells from a patient, which are then modified in a lab with proteins called chimeric antigen receptors (CARs) on the surface to recognize and kill cancer.
These immune cells are then infused back into the patient’s bloodstream through a drip.
But applying this concept to leukemia, which develops from abnormal T cells, is more complicated.
For BE-CAR7 the researchers used healthy T cells obtained from a donor and modified them using a method called base editing – which can be used to change single letters of the DNA code inside living cells.
This allows T cells to work after chemotherapy and deactivates them to prevent attacks against normal cells.
When base-edited CAR T-cells are given to a patient, they rapidly seek out and destroy all T-cells in the body, including leukemia T-cells.
If the leukemia is in remission within four weeks, the patient’s immune system is rebuilt with a bone marrow transplant over a period of several months.
This theory was followed up by a clinical trial to explore whether BE-CAR7 could cure leukemia before a planned bone marrow transplant, in the hope that it might prevent the cancer from returning.
For the study, nine children and four adults with T-ALL were treated with BE-CAR7 and more than half (64 percent) are now disease-free.
82 percent of participants who achieved deep remission were able to undergo stem cell transplants without any disease.
According to the researchers, although there were side effects, such as low blood count and rashes, these could be controlled.
Wasim Kassim, Professor of Cell and Gene Therapy at UCL and Honorary Consultant Immunologist at GOSH, said: “We have previously shown promising results using precision genome editing for children with aggressive blood cancers and this large number of patients confirms the impact of this type of treatment.
“We have shown that universal or ‘off-the-shelf’ base-edited CAR T-cells can seek out and destroy very resistant cases of CD7+ leukemia.”
Dr. Rob Chiesa, study investigator and bone marrow transplant consultant at Gosh, said: “Although most children with T-cell leukemia will respond well to standard treatment, about 20 percent may not.
“These are patients who desperately need better options and this research offers hope for a better prognosis for everyone suffering from this rare but aggressive form of blood cancer.”
The results of the clinical trial have been published in the New England Journal of Medicine and presented at the 67th American Society of Hematology annual meeting in Florida.