A world-first gene therapy which turns white blood cells into a disease-busting “living drug” has reversed previously untreatable blood cancers, according to recently released research funded by Blood Cancer UK.
Great Ormond Street Hospital (GOSH) and King’s College Hospital in London treated nine children and two adults with T-cell leukaemia using the technique, which scientists said triggered a “deep remission” in the majority. Seven are still disease-free three years later.
Developed by GOSH and University College London (UCL), the technique involves editing the genetic code of donor T-cells to target cancer cells without being rejected by the body. This base-editing is an advanced version of CRISPR technology.
“A few years ago this would have been science fiction,” said UCL’s Prof Waseem Qasim. “Now we can take white blood cells from a healthy donor and change a single letter of DNA code in those cells and give them back to patients to try to tackle this hard-to-treat leukaemia.”
In 2022, 13-year-old Alyssa Tapley from Leicestershire was the first in the world to receive the BE-CAR7 treatment. She had not responded to standard therapies, such as chemotherapy or a first bone marrow transplant, and her family had been discussing palliative care options before the research opportunity was proposed.
Dr Rob Chiesa, study investigator and bone marrow transplant consultant at GOSH said: “Although most children with T-cell leukaemia will respond well to standard treatments, around 20% may not. It’s these patients who desperately need better options and this research provides hope for a better prognosis for everyone diagnosed with this rare but aggressive form of blood cancer.”
“I’ve now been able to do some things I thought earlier in my life would be impossible,” said the now 16-year-old, who is cancer-free and has been discharged to long-term follow up. “My ultimate goal is to become a research scientist and be part of the next big discovery that can help people like me.”
Main image: National Cancer Institute
