Clustered Regularly-Interspaced Short Palindromic Repeats, or CRISPR, is on the path to human trails to treat cancer. This new biotechnology uses an enzyme called Cas9 to edit out sequences in human DNA that transfer hereditary illnesses and it can even be used to modify human traits such as eye color.
The development of this new technology can be traced back to 1987 when Japanese scientist Yoshizumi Ishino performed a research project aimed at discovering how bacteria fight off viral infections. Since this discovery, CRISPR has advanced into the most modern and innovative biotechnology tool, and it recently passed key safety reviews that could result in the first clinical trials to engineer genetic immune cells to fight off cancer.
CRISPR Function and Clinical Application
CRISPR works by targeting illnesses in the body that are identified by the Cas9 enzyme. Essentially, the Cas9 enzyme identifies invading sources of DNA from viruses, and these sources are copied and indexed in the genome as things called spacers in CRISPR. If the bacteria’s RNA identifies an incoming sequence of invading DNA, they guide CRISPR to that particular sequence, and then Cas9 enzymes cut and completely disable that gene to prevent it from killing the bacteria. These spacers in essence give the bacteria’s RNA molecules the ability to take down and fight invading viruses.
In humans, CRIPSR and Cas9 have the ability to cut, splice, and replace any gene sequence, giving scientists the capability to eradicate certain illnesses. The emergence of this new biotechnology is now being proposed for a new clinical trial that could be used to engineer immune cells to kill cancer. In a recent development of CRISPR, the technology won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health. The proposed clinical trial is backed by the Parker Institute for Cancer Immunotherapy, and a total of $250 million is being allocated towards this new possible method of treating cancer.
CRISPR Cas 9 Mechanism
Image Source: cancer.gov ucsf.edu
CRIPSR and Cancer
The proposed CRIPSR clinical trial is set to take a total of 2 years and will treat a total of 18 patients with myeloma, sarcoma, and melanoma that have stopped responding to existing cancer treatments. During the trial, scientists want to remove T-cells from patients and use a non-malignant virus to boost cells with a receptor for NY-ESO-1, a common protein found in cancer tumors. After this process, the modified T-cells are then placed back into the patient to fight off NY-ESO-1 tumors.
While CRISPR has been approved the RAC, the trial must now gain approval from internal ethics boards and the Food and Drug Administration (FDA). Although this technology could potentially eradicate many illnesses, such as HIV, ethical considerations must be taken into account, since the technology can be used for many other applications beyond treating illnesses such as changing the eye color of a baby or increasing bone density of humans.
