UPenn May Beat Editas to First Human CRISPR Treatment
CRISPR will need to fix up and look sharp next week for an appearance before a federal safety board. The National Institutes of Health report that the gene-editing technique is about to be considered for its first human use, as part of a cancer immune therapy.
Proposed by researchers from the University of Pennsylvania, the therapy will target myeloma, melanoma and sarcomas by reprogramming the patient’s own circulating immune cells using precision gene editing.
Carrie Wolinetz, associated director for science policy at the NIH, revealed the news in a blog post yesterday, writing that the NIH’s Recombinant DNA Advisory Committee (RCA) “will review a protocol involving the first-in-human use of gene editing via CRISPR/Cas9 technology.”
Biotech company Editas Medicine, which raised $94.4 million during its initial public offering in February this year, had been the hot tip for first-in-man trials of CRISPR. But UPenn might be about the pip them to the post. Editas previously announced its intention to begin a trial in 2017 using CRISPR to treat a rare eye disease. Thus far neither UPenn nor Editas have released a statement about the news.
The RCA have a tricky cost-benefit analysis to do around the proposed study, looking into the potential benefits weighed against the risks of gene therapy. Tinkering with the immune system can be dangerous because there is a risk that any alterations could trigger severe autoimmune responses.
“While the application of new gene editing technologies in this field has great potential to improve human health, it is not without concerns,” writes Wolinetz.
Last year British toddler Layla Richards had her aggressive leukaemia successfully treated with TALEN-based genome editing, marking the first time that TALEN had been used to treat a patient.