A remarkable breakthrough in medical science is shining a light of hope for patients battling blood cancers. A pioneering gene therapy, dubbed a “living drug,” has successfully transformed white blood cells, leading to the reversal of previously untreatable blood cancers, as revealed by exciting new research supported by Blood Cancer UK.
At Great Ormond Street Hospital (GOSH) and King’s College Hospital in London, a group of nine children and two adults with T-cell leukaemia underwent this innovative treatment. The results were truly uplifting, as the majority experienced a “deep remission,” with seven individuals remaining free of the disease even three years later.
Developed through a collaboration between GOSH and University College London (UCL), this groundbreaking approach involves editing the genetic code of donor T-cells. By making precise modifications, these cells can effectively target cancer without being rejected by the body. This advanced technique builds upon the promising CRISPR technology, showcasing the incredible potential of modern medicine.
Reflecting on this achievement, UCL’s Prof Waseem Qasim shared, “A few years ago this would have been science fiction. 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.” This transformation from concept to reality is a testament to the dedication and innovation within the scientific community.
Among those who benefited from this treatment is 13-year-old Alyssa Tapley from Leicestershire, who made history as the first person in the world to receive the BE-CAR7 therapy in 2022. After struggling to respond to conventional therapies like chemotherapy and a prior bone marrow transplant, her family was considering palliative care when they discovered this groundbreaking research opportunity.
Dr. Rob Chiesa, a key investigator and bone marrow transplant consultant at GOSH, emphasized the importance of this research, stating, “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.”
Now 16 years old and thriving, Alyssa expressed her joy, saying, “I’ve now been able to do some things I thought earlier in my life would be impossible.” With a bright future ahead, she aspires to become a research scientist, eager to contribute to the next significant discovery that can help others like herself.
This inspiring journey highlights the profound impact of scientific advancement and the unwavering spirit of those who strive for a healthier tomorrow.
