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US regulator weighs up CRISPR cancer therapy trial

20 June 2016

By Rachel Reeves

Appeared in BioNews 856

A US federal safety board is set to review an application for the first in-human use of CRISPR/Cas9 genome-editing technology to treat cancer.

The National Institutes of Health's (NIH) recombinant DNA advisory committee – established to review all human experiments that alter DNA – will consider a proposal to use CRISPR technology to treat cancer patients, in meetings on 21–22 June.

Proposed by scientists at the University of Pennsylvania, the experiment would first involve the removal of immune cells called T-cells from cancer patients. CRISPR would then be used to edit two genes in the T-cells so they express receptors allowing them to target melanoma, myeloma and sarcoma tumour cells, before being injected back into patients.

While the trial would represent a first-in-human use of CRISPR in the US or Europe, other immune-centred genetic engineering techniques are already being evaluated in cancer and other diseases. Last year a genome-editing technique called TALENs, transcription activator-like effector nucleases, was used to reverse advanced leukaemia in a one-year-old British girl (see BioNews 827). Scientists consider CRISPR, which was developed less than four years ago, to be more efficient and accurate than previous methods.

'Researchers in the field of gene transfer are excited by the potential of utilising CRISPR/Cas9 to repair or delete mutations that are involved in numerous human diseases in less time and at a lower cost than earlier gene-editing systems,' said Carrie Wolinetz, associate director of science policy at NIH, in a blog post.

Although CRISPR has been touted as carrying much potential to improve gene therapy-based treatments, safety concerns remain. The MIT Technology Review, which broke the news of the NIH evaluation, says that modified T-cells are potentially dangerous as they 'bypass the usual checks and balances that keep the immune system from attacking a person's own tissues'.

The committee meeting will be live-streamed on the NIH website.

Under the Poliscope (National Institutes of Health policy blog) | 16 June 2016
STAT | 16 June 2016
MIT Technology Review | 16 June 2016


12 September 2016 - by Dr Özge Özkaya 
Scientists from China have managed to shrink the size of tumours in mice using CRISPR/Cas9 genome-editing technology...
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...
27 June 2016 - by Rachel Reeves 
The first in-human use of the genome-editing technology CRISPR has been approved by a US federal safety board...

13 June 2016 - by Dr Jane Currie 
Scientists have used CRISPR to create part-pig, part-human embryos in an attempt to grow human organs for transplant...
06 June 2016 - by Ayala Ochert and Amina Yonis 
The team that developed the CRISPR/Cas9 DNA-editing technique have now developed a new system that could allow RNA editing...
25 April 2016 - by Dr Özge Özkaya 
Scientists have fine tuned the genome-editing tool CRISPR so that it can now edit a single 'letter' of DNA...
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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