There are many approaches to treating cancer, ranging from invasive surgeries to extremely damaging radiation and chemotherapy. The teeny-tiniest clinical trial ever began at UCLA in yet another attempt to find another way to eradicate cancer. With only 16 participants, this trial combined two areas of research: gene editing and T-cell engineering. The reason for the miniscule sample size is the intensely customized nature of the treatment. Each patient’s tumor had completely unique mutations, so each patient needed equally unique T-cell engineering through gene editing.
One reason cancer is so hard to treat is because they have adapted to be resistant to the body’s own immune response. The patients that have cancers, especially ones in the later stages, have lost the battle against their cancer with their own immune system, so a new super-immune system must now be built. This army of new T-cells (white blood cells, which identify and kill bad cells, seen below) will need “training” for its difficult battle ahead. First, however, the researchers must determine how to train these cells so they will actually be successful. They used algorithms to find identifiable mutations in the tumor, something that the T-cell can seek out to differentiate the cancerous cells from the normal cells.
After testing to make sure that the T-cells can actually identify these mutations, T-cell receptors are designed specifically to their tumor. Then, each patient’s blood is taken so that the DNA code for the new receptors can be inserted using CRISPR, a genome editing technology at the cutting edge of genetic medical research. The DNA code is transcribed to mRNA, which is then used in the ribosome to build polypeptides, in this case, the receptor proteins for the T-cells. In order to ensure that these new T-cells (with the special receptors) are received, the patients had to take medication that suppressed the number of immune cells, so that the ones they are given can take hold.
One month into treatment, 5 of the patients’ tumors stopped growing, and only 2 of the participants had associated side effects. Although only 5 patients had the desired results, Dr. Ribas, one of the researchers, says that they “need to hit it stronger the next time” because they were limited to a small dosage of T-cells to start in order to establish safety. Additionally, the technology will only get better and better as the research progresses and the T-cells can have more and more mutation targets to look for in a tumor.