Gene Therapy Cures 'Untreatable' Blood Cancer in 11 Patients

Alyssa Tapley was 13 when doctors told her family to consider end-of-life care. Her aggressive blood cancer hadn't responded to chemotherapy or even a bone marrow transplant, and options had run out.

Today, at 16, she's cancer-free and planning to become a research scientist. She's one of eleven patients whose lives were saved by a revolutionary gene therapy that scientists once thought impossible.

Researchers at Great Ormond Street Hospital and University College London treated nine children and two adults with T-cell leukaemia using a technique called BE-CAR7. The therapy edits the genetic code of healthy donor white blood cells, programming them to seek and destroy cancer cells without being rejected by the patient's immune system.

The approach uses base-editing, an advanced version of CRISPR technology that changes a single letter of DNA code. Seven of the eleven patients remain in deep remission three years after treatment, according to research funded by Blood Cancer UK.

"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."

Around 20% of children with T-cell leukaemia don't respond to standard treatments. For these patients, the new therapy offers hope where none existed before.

The Ripple Effect

Alyssa became the first person in the world to receive BE-CAR7 treatment in 2022. Her recovery has opened doors for other patients facing the same desperate situation her family once confronted.

Dr Rob Chiesa, bone marrow transplant consultant at GOSH, said the research provides hope for better outcomes for everyone diagnosed with this rare but aggressive cancer. The success of these initial eleven patients is already informing new approaches to other hard-to-treat blood cancers.

"I've now been able to do some things I thought earlier in my life would be impossible," Alyssa said. Her dream of contributing to the next medical breakthrough that helps patients like her is now within reach.