23 June 2016
A key US ethics and biosafety committee has given its approval for the first human trials using CRISPR genome editing technology.
Although the green light from the US Food and Drugs Agency (FDA) must be received before the trial can start, this week’s approval by the advisory committee to the National Institutes of Health, brings the technology a step closer to clinical use – and sooner than widely anticipated.
Assuming they get the go ahead, scientists at the University of Pennsylvania (UPenn), will extract and modify the ‘T’ cells from the immune systems of 18 patients with myeloma, sarcoma or melanoma. The patients have all stopped responding to existing treatments.
The UPenn team have designed this small trial primarily to assess the safety of combining three CRISPR ‘edits’ to DNA in one treatment. The first edit has previously been shown to shrink tumour cells in people, but the effect quickly wears off. The two additional edits are intended to make the impact of the first edit longer lasting and more effective. Once all three edits have been made, the T-cells will be reintroduced into the patient.
The proposed two year trial is funded by the Parker Institute of Cancer Immunotherapy, founded earlier this year by Internet billionaire, Sean Parker.
Prior to this week’s announcement, the CRISPR- dedicated company Editas Medicine, had been vaunted to be the first off the starting block. The company hopes to start trials in 2017 for CRISPR-based treatment for inherited blindness.