Serena S
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Promising new gene therapy treatment for leukaemia is a step closer

Serena S
Posted by
21 Jun 2010

A promising new cancer treatment for patients with leukaemia has been brought a step closer after a study by UK and Dutch scientists has overcome one of the possible side effects linked with this therapy.

The research, which is published online in the journal Nature Medicine on Monday 19 April, directly addressed safety concerns associated with this new treatment, called TCR gene therapy, to ensure that it brings the most benefit to patients with leukaemia.

TCR gene therapy, which engineers the patients’ own immune cells with the ability to target and kill cancer cells in their body, has been successfully tested in patients with skin cancer in the USA and will shortly be trialled in adults and children with leukaemia in the UK.

The treatment uses genetic engineering to modify white blood cells, an important part of the immune system that fight infection, to home in on and destroy leukaemia cells in the blood.

Scientists, supported by blood cancer charity Leukaemia & Lymphoma Research, explored new strategies for delivering this treatment to patients so that it avoids also damaging healthy cells, causing serious side effects.

Laboratory results had shown that the treatment should be more effective for those patients with leukaemia who do not respond to standard chemotherapy, but there were concerns that there could be side effects that were more severe.

One side effect is a condition called graft versus host disease (GVHD) in which the newly delivered white blood cells attack and destroy the patient’s healthy cells.

Dr Gavin Bendle, a UK scientist working in the laboratory of Prof Ton Schumacher at The Netherlands Cancer Institute thanks to a visiting fellowship grant from Leukaemia & Lymphoma Research, led the research.

He said: “TCR gene therapy is likely to be an important part of treatment for those patients with leukaemia who cannot be cured with standard therapies. By investigating the way in which these genetically engineered white blood cells target leukaemia cells in the blood, we have devised new strategies for delivering this treatment that will minimise the side effects experienced by patients.”

Two national clinical trials, both supported by Leukaemia & Lymphoma Research, are due to open in the UK later this year, offering TCR gene therapy to children and adults with leukaemia, who cannot be cured using treatments that are currently available.

Dr David Grant, Scientific Director at Leukaemia & Lymphoma Research, said, “TCR gene therapy is likely to offer new hope to patients not only with leukaemia, but with all blood cancers, including lymphoma and myeloma, who do not respond to standard treatments. This research is vital to ensure that all these patients get the best possible standard of treatment and chance of survival.”