Gene therapy cures leukaemia in eight days

NewScientist ^ | 3-26-2013 | Andy Coghlan

[Sir Napsalot]

WITHIN just eight days of starting a novel gene therapy, David Aponte's "incurable" leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing.

The cured trio, who were all previously diagnosed with usually fatal relapses of acute lymphoblastic leukaemia, have now been in remission for between 5 months and 2 years. Michel Sadelain of the Memorial Sloan-Kettering Cancer Center in New York, co-leader of the group that designed the trial, says that a second trial of 50 patients is being readied, and the team is looking into using the technique to treat other cancers.

The key to the new therapy is identifying a molecule unique to the surface of cancer cells, then genetically engineering a patient's immune cells to attack it.

In acute lymphoblastic leukaemia, immune cells called B-cells become malignant. The team were able to target a surface molecule known as CD19 that is only present on B-cells. Doctors extracted other immune cells called T-cells from the patients. These were treated with a harmless virus, which installed a new gene redirecting them to attack all cells bearing CD19. When the engineered T-cells were reinfused into the patients, they rapidly killed all B-cells, cancerous or otherwise.

(snip)

The treatment is not the first to re-engineer T-cells to attack a form of leukaemia. Last year, an international company called Adaptimmune used the approach to treat 13 people with multiple myeloma – it left 10 in remission.

[Sir Napsalot]

Gene therapy is not new. But we may be seeing more and more promising results of making a great stride against cancer(s).

[massgopguy]

Well at least I’m not getting blamed for causing a disease today. Gene cause Cancer. Gene causes Dipsomania (look it up).

[Sergio]

bkmk