CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials.
On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR/Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells.
This first trial is small and designed to test whether CRISPR is safe for use in people, rather than whether it cures cancer or not. It will be funded by a US$250 million immunotherapy foundation formed in April by former Facebook president Sean Parker. The trial itself does not yet have a budget. The University of Pennsylvania will manufacture the edited cells, and will recruit and treat patients along with centers in California and Texas.
The researchers will remove T cells from 18 patients with melanoma, sarcoma, or myeloma, and perform three CRISPR edits on them. One edit will insert a gene for a protein engineered to detect cancer cells and instruct the T cells to target them, and a second edit removes a natural T cell protein that could interfere with this process. The third is defensive: removing the gene for a protein that identifies the T cells as immune cells and preventing the cancer cells from disabling them. The researchers will then infuse the edited cells back into the patient.
http://www.nature.com/news/federal-...linical-trial-1.20137?WT.mc_id=TWT_NatureNews
On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR/Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells.
This first trial is small and designed to test whether CRISPR is safe for use in people, rather than whether it cures cancer or not. It will be funded by a US$250 million immunotherapy foundation formed in April by former Facebook president Sean Parker. The trial itself does not yet have a budget. The University of Pennsylvania will manufacture the edited cells, and will recruit and treat patients along with centers in California and Texas.
The researchers will remove T cells from 18 patients with melanoma, sarcoma, or myeloma, and perform three CRISPR edits on them. One edit will insert a gene for a protein engineered to detect cancer cells and instruct the T cells to target them, and a second edit removes a natural T cell protein that could interfere with this process. The third is defensive: removing the gene for a protein that identifies the T cells as immune cells and preventing the cancer cells from disabling them. The researchers will then infuse the edited cells back into the patient.
http://www.nature.com/news/federal-...linical-trial-1.20137?WT.mc_id=TWT_NatureNews