By Adin Blumofe
In 2020, the Nobel Prize in Chemistry was awarded to Jennifer Doudna and Emmanuelle Charpentier for their 1987 invention of the gene-editing tool CRISPR, which allows scientists to change specific nucleotides in DNA. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) revolutionarily provided the ability to edit mutated genetic code directly. Thirty-five years from initial conception, the possibilities this technology provides are finally beginning to be fully utilized.
Typically, the immune system, which comprises over a dozen types of cells, works in a complex, multilayered defense to destroy potential foreign or domestic threats to the human body. When a cell stops regulating its growth properly, reproduction becomes cancerous, sucking up resources and spreading rapidly. Usually, cancer cells are quickly destroyed by the immune system. However, sometimes the cancerous cells fly under the radar of the immune system or are simply capable of over-powering the body’s defenses. If left untreated, metastasizing cancer will have derelict effects on the human body. Until this point, medical interventions have been primarily focused around chemotherapy and radiation, approaches that have serious weaknesses, such as the possibility that these non-targeted treatments will induce future cancers.
Using CRISPR technology, Antoni Ribas’s team at the University of California, Los Angeles, is creating a new weapon for the doctors’ toolbox by developing specific cancer-targeting T-cells. They edited sixteen patients’ T-Cells using CRISPR, reworking them to address each patient’s exact type of cancer. The idea is that designer T-Cells will better detect and destroy tumors, which have thus far been successfully hiding from the body’s defense system. The treatment counterintuitively requires infusing a few gene-edited T-cells, while killing off much of the unadulterated antibodies, so that, after many cycles of reproduction, most of the body’s remaining T-cells will be of the altered form. By saturating the blood with the ‘new’ T-cells, the immune cells can diffuse into hard surface tumors, which are harder to penetrate. Once inside the tumor, the T-cells can tear apart the cancer from the inside out.
According to Scientific American, “One month after treatment, five of the participants experienced stable disease, meaning that their tumours had not grown. Only two people experienced side effects that were likely due to the activity of the edited T-cells.” The results from the trial serve as a promising proof of concept. Now that it has been established that the method is functional, CRISPR can be expanded to different types of cancers across the human body. Not only is this process more effective in tackling the cancer, but there is also the benefit of not having to worry about inducing secondary cancers from the treatment, as CRISPR does not involve radiation.
With all the exciting news, it is important to temper expectations. In the coming years, one should not expect to see cancer cures in CVS pharmacies. Producing these CRISPR therapies “requires a tremendously complicated manufacturing process,” as it practically necessitates making a new medicine for each patient. The necessary high-degree of specificity means the treatment will always be exorbitantly expensive compared to the generalized, current alternatives. Additionally, insurance companies are far warier of funding cutting-edge techniques due to potential doubts of efficacy and the sticker shock they carry. Despite all the short-term caveats, one thing is for sure—CRISPR, in time, will fundamentally alter the human condition.