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New Gene Editing Tech Could Be Used in People Soon

A U.S. federal biosafety and ethics committee has unanimously approved the world's first human study on the powers of the genome-editing technology CRISPR/Cas9, according to media reports. The study aims to use CRISPR to create genetically-altered immune cells to attack cancer.

Kristopher Sturgis

A 3-D printed model of Cas9, a DNA-cutting enzyme that is used by CRISPR. (Image courtesy of NIH Image Gallery)

The new experiment was proposed by scientists from the University of Pennsylvania where the study will be conducted, pending approval from the Food and Drug Administration. Once FDA signs off on the study, Penn researchers will begin enrolling patients suffering from three different forms of cancer: multiple myeloma, melanoma, and sarcoma.

The study aims to be the first to use CRISPR (clustered regularly interspaced short palindromic repeats) -- a molecular editing system that offers the ability to edit the DNA of living systems -- in a clinical trial on human patients. The goal is to use the gene-editing technology to manufacture genetically modified T cells that can target and destroy tumor cells in the body. These genetically-modified T cells would be produced in a lab at the University of Pennsylvania before they can be infused back into human subjects as part of the study.

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Penn researchers could not be immediately reached for comment.

David Albert, former chief clinical scientist at GE Healthcare and current entrepreneur in the realm of mobile applications for electrocardiogram devices, believes the new CRISPR technology could be transformative when it comes to editing out harmful genetic defects.

"CRISPR presents the opportunity to repair specific genetic defects, which could be revolutionary," he says.

Given that it is still definitively unknown whether or not the system can safely alter human DNA, scientists have taken many steps to ensure that human trials are the logical next step. In early 2015, researchers from MIT reported that they had all but eliminated "off-target" editing errors, one of the major remaining technical issues that prevented the use of CRISPR as a means to modify human DNA.  A few months later, Chinese scientists announced they were the first researchers to use CRISPR to successfully modify human embryos.

The new trial looks to utilize a technique that involves T cells extracted from the patient, and alter them using the CRISPR technology, before being infused back into the patient with re-programmed instructions to identify and destroy specific antigens that protrude from tumor cells. The main goal of these early-stage clinical trials is to gauge the safety of the experimental therapy, while also learning how feasible it is to manufacture the CRISPR T cells needed for the therapy to be a success.

Many concerns that have arised in the past from the idea of altering human DNA.

Penn has a history in the field that should make its researchers well-aware of potential hazards: The university actually reported the first death of a patient participating in a clinical trial for gene therapy back in 1999.  Jesse Gelsinger had joined a clinical trial conducted at Penn that aimed to develop a treatment for infants born with a severe genetic disease. After his death, an FDA investigation concluded that the scientists involved in the trial broke several rules of conduct, and the university eventually reached an undisclosed financial settlement with the parents of Gelsinger.

The Gelsinger case was seen as a setback for scientists working in the field of gene therapy, and is undoubtedly an outcome that researchers believe they can avoid with their newest study.

The debate continues over whether or not the benefits of CRISPR are worth the potential dangers they pose. But this this group of researchers is on the cusp of exploring the real possibilities of using CRISPR to fight disease in humans.

Kristopher Sturgis is a contributor to Qmed.

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