Illustration of blood cells

New treatment reverses incurable blood cancer in some patients

A revolutionary new cancer therapy is providing a lifeline to patients with previously incurable leukemia. Scientists at Great Ormond Street Hospital (GOSH) and University College London (UCL) have developed a world-first treatment using base-edited T-cells. Early trial results show that this experimental therapy, known as BE-CAR7, successfully cleared cancer in patients with relapsed T-cell acute lymphoblastic leukemia (T-ALL). This breakthrough offers hope where all other treatments, including chemotherapy and stem cell transplants, had failed.

This advancement represents a major leap forward in the field of immunotherapy. It demonstrates the potential of next-generation gene editing to treat the most aggressive and resistant forms of cancer.

Precision Engineering with Base Editing

The core innovation of BE-CAR7 lies in its use of base editing, a highly precise form of genome engineering. Unlike traditional CRISPR methods that cut the DNA double helix, base editing chemically changes individual DNA letters. This technique allows scientists to modify healthy donor T-cells so they can hunt down and destroy cancerous T-cells without attacking each other or the patient.

This level of precision reduces the risk of unintended genetic damage, making the therapy safer and more effective. It allows for the creation of “universal” off-the-shelf T-cells that can be used for multiple patients. This scalability addresses a major bottleneck in current CAR-T therapies, which often require manufacturing bespoke cells for each individual patient.

Achieving Remission in “Untreatable” Cases

The clinical results from the Phase 1 trial are incredibly promising. The therapy was administered to children and adults who had exhausted all other treatment options. In one highlighted case, a 13-year-old girl named Alyssa became the first person in the world to be treated with this technology. After just 28 days, she was in remission and was able to proceed to a bone marrow transplant.

Data presented at the American Society of Hematology annual meeting showed that remission was achieved in a significant majority of the trial participants. These patients, who previously had a dire prognosis, were given a second chance at life. This success validates the therapeutic potential of base-edited cells in a clinical setting. You can read more about Alyssa’s story and the trial from Great Ormond Street Hospital.

A Universal “Off-the-Shelf” Solution

A major positive aspect of BE-CAR7 is its potential to democratize access to advanced cancer care. Current CAR-T therapies are expensive and time-consuming because they must be custom-made from the patient’s own cells. BE-CAR7 uses donor cells that are engineered to be “universal,” meaning they can be manufactured in batches and stored for immediate use.

This “off-the-shelf” capability could drastically reduce waiting times for critically ill patients. It also has the potential to lower manufacturing costs, making life-saving immunotherapy accessible to more people. This shift toward universal cell therapies is a key goal for the future of oncology. The National Institute for Health and Care Research (NIHR) supports the development of these innovative treatments within the NHS.

Expanding the Frontier of Medicine

The success of this trial marks the beginning of a new era in medicine. It proves that base editing can be safely applied to humans to treat complex diseases. Researchers are already looking to expand this technology to treat other types of blood cancers and potentially solid tumors.

This progress offers a hopeful vision for the future of cancer treatment. It suggests that even the most resistant cancers may eventually be curable through the precision of genetic engineering. The Medical Research Council (MRC) funds much of the foundational science that makes these breakthroughs possible. Furthermore, Blood Cancer UK provides resources and support for patients navigating new treatment options.

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