CRISPR the Next Frontier in Gene Therapy Cancer Research

  • | Sep 06 2016 |

In our last blog, we described the progress and promise of CAR T, an immunotherapy that has been particularly successful with leukemia and lymphoma. As a companion to CAR T, and all forms of cell and gene therapies, CRISPR is the talk of the scientific town these days and is the next tcell_ssfrontier in genetic medicine.

CRISPR. Sounds like something that belongs in your kitchen, doesn’t it? This science is neither therapy nor treatment. It’s a fantastically precise tool that permits genetic editing. Once we completed mapping the human genome, we have the knowledge, and the technology, to effect change in the base DNA that makes up our unique identity. A small change in that code can make a huge difference in health and longevity.

What is CRISPR technology?

Clustered Regularly Interspaced Short Palindromic Repetition is the actual term, should you have an opportunity to prove your medical mettle in conversation. A simpler translation: molecular medical scissors used to repair or replace a portion of the genetic code. #genomics

The technology was discovered just three years ago when researchers, using a DNA cut and paste method using bacteria to protect against viruses, realized they could also edit human genes. In CRISPR, either the cancerous cell, once cut, will repair itself, or the body’s natural killer T cells will take aim and destroy diseased cells. Unbelievably simple once the barriers caused by mutated or damaged genes are removed.DNA_scissors

Sounds like Sci-Fi? Well, it is a huge leap into the future. So new that scientists from around the world gathered earlier this year for the first international summit on human gene editing, in Washington DC, and agreed to avoid using the science for human genetic engineering. No test tube animals, no Frankenstein. On the other hand, CRISPR is being used to simulate cancers in laboratory animals, for the purpose of study, and much will be learned in this way.

CAR T, Immunotherapy and CRISPR

CRISPR has the potential to battle not only disease, but also genetic disorders like allergies or neurological dysfunction. The opportunities are limitless. Our own Scientific Advisory Council member, Dr. Carl June, at the University of Pennsylvania, is heading a research team funded by Facebook financier Sean Parker. Dr. June aims to enhance his pioneering immunotherapy by making even more powerful killer T cells using the CRISPR scissors. Cells will be edited to bypass cancer’s natural protective obstacles to treatment, known as checkpoint inhibitors. [See our blog on CAR T.]

“This is the first example of CRISPR in clinical trials in humans, period,” Parker reported at the Forbes Philanthropy Summit in August. This sort of advancement may not have been possible without private funding.


gene_editing_ssThe race is on to refine the technique. In China, scientists are studying the potential for CRISPR for lung cancer and have secured approvals for first level trials. In the US, the FDA is expected to approve Phase I trials later this year, with patients suffering melanoma, sarcoma and myeloid cancers. The trials will examine toxicity and side effects as well as patient response.

Treating Cancer Patients

Are there challenges? Of course. Medical interventions, even perfected in the lab, must be refined in practice. Humans have been blessed with powerful immune systems that have been too often neutralized when confronted with cancers, but both CAR T and CRISPR seek to change all that.

The future is happening right now, and ACGT is still the only non-profit in the nation dedicated exclusively to innovative cell and gene therapies for cancer. Stay tuned!

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