A new treatment freed a teenage girl from incurable cancer.
In May 2022, 13-year-old Alyssa from Leicester became the first registered patient in the world to receive a new type of CRISPR called base editing.
“Radically modified T-cells at Great Ormond Street Children’s Hospital (GOSH) in partnership with the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) cured Alyssa’s “incurable” T-cell leukemia, leaving her with zero detectable cancer cells,” researchers said in a press release.
“When I do this, people know what they need to do one way or another, so it helps people—of course I do,” Alyssa said in a press release.
The result should be monitored and confirmed in the next few months.
Alyssa was diagnosed with T-cell acute lymphoblastic leukemia (T-ALL) in 2021 and has already received chemotherapy, a bone marrow transplant, and other anti-cancer treatments. But all was in vain, and his illness returned. No other treatment options were available at the time.
He became part of an attempt to receive genetically modified CAR T cells from a healthy donor. The cells were altered with a new core editing technology, allowing them to attack and kill one another.
“This is quite significant, although it is still a preliminary result that needs to be followed up and confirmed over the next few months,” said Dr. Robert Chiesa, GOSH’s consultant on bone marrow transplantation and CAR T-cell therapy.
“We’re honestly on cloud nine—it’s good to be home,” said Alyssa’s mother, Kiona.
Researchers had to choose an “unconventional” CAR-T cell therapy
Leukemia is characterized by the rapid growth of abnormal blood cells in the bone marrow. Chemotherapy is usually given to kill all the cells in the bone marrow, and then the bone marrow is replaced with a transplant. Although this approach is popular and successful in most cases, doctors try another method called CAR-T therapy when the above fails.
T-cell leukemia is difficult to treat with traditional CAR-T cell therapy, however, because the T cells designed to recognize and attack cancer cells also kill each other during the manufacturing process before they can be administered as therapy, according to the release.
Therefore, the trial teams used base editing, a technique invented just six years ago, to create a new type of CAR T cell therapy to attack cancer T cells.
How does basic editing work?
Single letters are chemically altered to alter T cells. This technique has been used to modify healthy donor T cells.
Donor T cells are edited and modified so that they are not attacked by the patient’s immune system. Finally, patients receive engineered CAR T cells to find and destroy T cells in the body. If successful, the patient receives a bone marrow transplant to restore the weakened immune system.
“This is an excellent example of how, with experienced teams and infrastructure, we can combine cutting-edge technologies in the laboratory with real results in the patient’s hospital.” “This is our most advanced cell design to date and will pave the way for other new treatments and ultimately a better future for sick children,” said UCL GOS ICH cell researcher Waseem Qasim, Professor of Gene Therapy and Consultant Immunologist at GOSH.
Alyssa’s mother hopes her daughter’s treatment is the result of research. “The doctors said the first six months are the most important, and we don’t want to be too harsh, but we thought if they could get through all of this once, he’d be fine.” “And maybe we’re right,” he said.