A revolutionary leukaemia treatment could soon be approved for use in the USA after clinical trials saw complete remission in 90% of patients with a type of aggressive blood cancer.
The gene therapy ‘CTL019’ has produced unprecedented results, and was regarded as such a success that it was extended to include a larger pool of patients than originally planned.
It is now awaiting the thumbs up from Food and Drug Administration (FDA) officials in America, who could green light the treatment for September, making it the first gene therapy permitted for use in the country.
‘CTL019’ therapy was developed by the University of Pennsylvania and sees a custom-made, personalised drug administered to individuals with resistant acute b-lymphoblastic leukaemia, a disease that accounts for 25% of all cancers in children.
Current standard treatment programs for b-lymphoblastic leukaemia are based around chemotherapy followed by bone marrow transplantation.
If the standard treatment fails, as in 15% of cases, a bone marrow donor is unavailable or the patient relapses, there are no other therapeutic options and the chances of survival are slim.
The proposed new therapy sees a single shot of immune cells taken from the patient, which are then grown and genetically modified in a lab for expression of a molecular receptor called CAR, and then re-enter the body via blood transfusion.
This editing means that the cells are reprogrammed to kill the patient’s specific leukemic cells.
In the trials 30 patients who had the disease, 90% of them saw the leukaemia vanish completely, although the team are keen to stress that long-term effects of these ‘CTL019’ cells living in the patient’s body are not yet known.
Maria Teresa Esposito, at the University of East London, wrote in a blog post: “As scientists, we all hope and work hard to find a definitive cure for all cancers. We don’t know yet if the patients that undertook the CTL019 trials can be considered cured or whether they will eventually relapse.
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“But this drug is an opportunity to prolong the survival of patients that currently have no other option. In the meantime, we will celebrate every success in the battle to prolong the survival of cancer patients.”
It would be the fourth approval of its kind worldwide, following European approval for Glybera and Strimvelis – therapies to tackle genetic diseases – and Gendicine in China to treat cancer.
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