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Genome editing successfully treats leukaemia

25 January 2017

By Dr Loredana Guglielmi

Appeared in BioNews 886

Doctors at London's Great Ormond Street Hospital have used genome editing to successfully treat two children with leukaemia.

The initial results were widely reported in the media in November 2015 (see BioNews 827), and the two case studies have now been published in the journal Science Translational Medicine. Both children showed complete remission of the disease within 28 days and were free of cancerous cells one year after receiving the treatment.

Layla Richards was the first child to receive the experimental cell-based treatment in June 2015. She was three months old when she was diagnosed with B-cell acute lymphoblastic leukaemia (B-ALL) and had previously undergone chemotherapy and a bone-marrow transplant. But these failed, and the leukaemia returned. She received the experimental treatment when she was one year old. The London team then repeated the procedure on a second baby girl, aged 16 months and with a similar medical history to Layla, in December 2015.

The treatment was based on an existing system – CAR T-cell therapy – which uses a patients own T cells and genetically modifies them to target cancerous cells. But it is hard to collect enough healthy T cells from leukaemia patients.

Professor Waseem Qasim at University College London, who led the study, created a universal CAR T-cell therapy using T cells taken from a healthy person, overcoming the need to fine-tune the infants' own immune cells. They used the genome editing tool TALENs – transcription activator-like effector nucleases – to silence the gene that would cause the T cells to be rejected.  

'The patient could be treated immediately, as opposed to taking cells from a patient and manufacturing them,' said Dr Julianne Smith, vice-president of CAR-T development for Cellectis, the New York biotech company that manufactures the cell treatment.

'In the off-the-shelf approach, blood is collected from a donor and then turned into hundreds of doses that can then be stored frozen,' said Dr Smith. 'We estimate the cost to manufacture a dose would be about $4000,' she added. The cost of the personalised therapy using a patient's own cells is around $50,000.

The results have been challenged by some researchers, who have pointed out the children received standard chemotherapy on top of the cell-based therapy. 'There is a hint of efficacy but no proof,' noted Professor Stephan Grupp, director of cancer immunotherapy at the Children's Hospital of Philadelphia, Pennsylvania. 'It would be great if it works, but that just hasn't been shown yet.'

Rights to this genome-editing therapy have been sold to Cellectis, and the pharmaceutical companies Servier and Pfizer are now attempting to develop it into a treatment for other B-ALL patients.

New Scientist | 25 January 2017
Science Translational Medicine | 25 January 2017
MIT Technology Review | 25 January 2017
Eurekalert (press release) | 25 January 2017


04 September 2017 - by Meetal Solanki 
The world's first cancer treatment which uses a patient's own genetically modified immune cells has been approved...
14 August 2017 - by Ebtehal Moussa 
Over a 100 new genes that may be essential for cancer immunotherapy to work have been identified using a new CRISPR-based screen...
17 July 2017 - by Dr Loredana Guglielmi 
A drug for leukaemia that genetically alters patients' own cells to fight cancer, has cleared a critical hurdle in gaining commercial approval...
12 June 2017 - by Ebtehal Moussa 
Two trials of a new gene therapy have successfully treated blood cancers in patients who were unresponsive to standard treatment...

25 July 2016 - by Rachel Siden 
Chinese scientists plan to start the first-ever clinical trial of CRISPR genome-editing technology in humans – on patients with lung cancer – this August...
22 February 2016 - by Julianna Photopoulos 
Trials using genetically engineered immune cells have shown 'extraordinary results' in treating blood cancers in terminally ill patients, say researchers...
09 November 2015 - by Lone Hørlyck 
An experimental cell-based treatment using gene editing, previously only tested on mice, has successfully reversed advanced leukaemia in a one-year-old girl...

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