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US medicines regulator approves use of CAR-T cell therapy for childhood leukaemia

Bloodwise
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Updated 30 Aug 2017

The Food and Drug Administration (FDA) has approved the use of a new drug, which engineers patients’ own immune cells to fight cancer, for the treatment of children with acute lymphoblastic leukaemia who do not respond to standard treatment.

The treatment, known as CAR-T cell therapy, has shown considerable promise in early clinical trials treating children who did not respond to standard chemotherapy. The first child to be treated with the therapy on a US clinical trial is still in remission six years later.

How does the treatment work?

Immune cell samples are taken from the patient and genetically modified in a laboratory to recognise, seek out and kill cancer cells, providing a personalised treatment for each patient. Because the body will always retain a number of these white blood cells that recognise leukaemia cells as harmful, patients could theoretically have a life-long ‘immunity’ to the cancer, much like in diseases such as chickenpox.

What still needs to be done? 

CAR-T cell therapy is a completely new approach that carries some risks and will only be approved for use in children who do not respond to chemotherapy or who relapse after treatment. CAR-T therapies, which are also being developed for other cancers, do not work for everybody and some clinical trials have reported severe and fatal side effects. As it is still in the early stage of development, the long-term effects on patients is still not known. The treatment is manufactured individually for each patient and is extremely expensive. 

Why is this week’s news important? 

Gene therapy drugs are viewed as one of the most promising developments in cancer treatment in recent years and many other gene therapy drugs are currently being developed to treat for different types of cancer.  While a number of different clinical trials have been completed, this is the first time ever that a gene therapy drug has been approved for widespread use.  

Dr Alasdair Rankin, Director of Research at Bloodwise, said: 

“We welcome the approval of the first CAR-T cell therapy in the USA and it is particularly exciting that it will benefit children with acute lymphoblastic leukaemia who do not respond to current treatments. We look forward to this therapy being submitted for approval in the UK soon. 

“The development of CAR-T cell therapies is a very promising area of blood cancer research, which has the potential to save many lives. There are still a number of questions that still need to be answered, however, particularly around long term effectiveness and side-effects and the cost of treatment. More follow-up time and further studies will be needed to refine the treatment and better understand its effect on patients.”

Bloodwise currently has around £2.5 million invested in different projects and clinical trials looking at using gene therapy to treat a range of blood cancers, including myeloma and a number of different types of leukaemia and lymphoma.

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