This month Chinese scientists are using the CRISPR method to target metastatic non-small cell lung cancer in patients. The specific technology employed will reprogram T-cells from the patients to remove the PD-1 gene. The PD-1 gene serves as a check on the cell’s ability to generate an immune response and thus prevents healthy cells from being attacked during an immune response.
CRISPR is more specific than alternative immunotherapy methods (which are typically antibody based) because you are going into the cell’s genome and editing the genes. This makes CRISPR a potentially more effective method at targeting cancer cells, but it also has larger repercussions if done incorrectly. For example, it has been known to cause gene edits in the wrong place in the genome, which can have very harmful effects. Also, since T cells are involved, as in the above article, then there may be potential for an autoimmune response since T cells are involved in a variety of immune responses in a somewhat non-specific way. As mentioned in the article, one potential way to correct this is to sample T-Cells from the tumor, so they are confer specificity to the tumor.
In my opinion, CRISPR is very exciting, but also a bit scary due to the infancy of the technology and the problems mentioned above. CRISPR is no doubt a powerful biological tool, and as a result, a bit controversial. Furthermore, because we are modifying the genome, serious consideration should be given regarding any long term effects from this technology that may not be foreseen, especially for applications outside immunotherapy.
Aside from the drawbacks, however, these methods are very promising because they offer great hope when traditional therapies fail. With specific regard to immunotherapy, as we gain more knowledge about the immune system, and as our technologies continue to develop, I think (and hope) that these methods will be a viable solution to cancer therapy.
As of now, I am unaware of any CRISPR methods for PH, however, there are immunotherapy clinical trials underway for PH (for example, targeting CD20+ B cells).
I think it is way too early to see PH trials involving the CRISPR method. But depending on the initial results in cancer trials (as in the above article), we may start to see more application of this technique to PH. I did, however, find one interesting article about the application of CRISPR to the treatment of cystic fibrosis which could be very interesting.