Killing cancer is the ultimate goal for many researchers, and they’re studying various ways of doing it. Some scientists are working on a vaccine that would kill tumor cells in a patient and stop cancer from spreading. Others are looking to edit genes to enhance the immune response of the organism and eliminate cancerous growth. A federal panel approved the latter type of proposed treatment, marking the first use of CRISPR in humans.
If CRISPR sounds familiar, that’s because we talked about this gene-editing technology before. As Stat reports, it was widely expected that the first human use of CRISPR would occur in 2017, in a clinical trial supposed to treat a rare form of blindness called Leber congenital amaurosis.
However, a federal biosafety and ethics panel on Tuesday approved a CRISPR-based clinical trial that would see doctors from various centers treat certain types of cancer by editing immune cells. Proposed by physicians from the University of Pennsylvania, the experiment still needs approval from the medical centers where it would be conducted, and from the FDA.
The trial would focus on patients with multiple myeloma, melanoma, and sarcoma, and would be funded by the Parker Institute for Cancer Immunotherapy, which was launched earlier this year by Sean Parker.
The scientists plan to remove T cells (immune cells) that are in the first line of defense against foreign cells that make it inside the body, and then edit them so that they can target and kill cancerous cells.
The genetic altering process of the T cells would ensure that the cells produce a “chimeric antigen receptor,” or CAR, which would then bind to molecules-antigens protruding from tumor cells. This way, T cells would go after a specific type of cell, and only take out cancer.
But for CAR to work, additional gene-editing is needed. CRISPR techniques will be used to slice out two genes in T cells. One gene is for PD-1, a molecule found on cancer cells that disable T-cells when meeting them. The other is for endogenous TCR, T cell’s natural receptors, as studies showed that removing the TCR results in better functioning.
With PD-1 and TCR genes removed, T cells would be more efficient at fighting cancer, and they would have an increased persistence inside the human body.
This new type of treatment, as exciting as it may be, also comes with some ethical side effects and conflicts of interest. For starters, we, as a species, are opening doors to something we haven’t done before, playing with human genes. Secondly, the financial interests behind the success of such treatments are enormous, and the committee already spotted a conflict of interest in this matter that it wants to keep in check.
More details about the first human trials are available at the source link.