May 21, 2016

Reshaping human health and changing lives through gene editing

Gene editing is going to fundamentally change our lives and how we traditionally think about health throughout the first half of the 21st century.

Gene editing is going to change the way people are treated by curing the roots of diseases instead of merely treating the symptoms. It’s going to change the way we think about what we put into our bodies, as gene editing will put healthier food on our plates without polluting the planet. This food will not only be safe to eat, it will also meet the environmental challenges related to sustainable growth and climate change.

As a result, people will no longer focus on whether or not we should engage in gene editing from an ethical standpoint. The question isn’t “When will gene editing become a significant reality for the majority of the world?” The truth is, this is neither science fiction nor a prediction — gene editing is happening now, as evidenced by the first cancer patient having been treated by TALEN®-based gene-edited T-cells. Additionally, this fall, fields across the United States will harvest TALEN®-based gene-edited soybeans and potatoes. There are even gene-edited pigs and hornless cows that are currently walking around the barnyard.

The landscape in gene editing is anything but clear, but the recent emergence of new gene-editing technologies, with new players in the space, has led to an inevitable ethical debate.

For example, three early clinical-stage startup companies, all based on CRISPR technology, have struck major alliances with big pharma and biotech companies: Editas Medicine (Juno Therapeutics), CRISPR Therapeutics (Vertex and Celgene) and Intellia Therapeutics (Novartis).

While these alliances are important, the long-term successes of these companies depend upon their ability to deliver on their promises. Turning CRISPR innovations into approved and effective drugs is a core focus, and it will still take years of more hard work before an effective, approved drug will result from these efforts — if at all.

Additionally, Sangamo BioSciences and Precision BioSciences are two other well-established companies that operate in the gene-editing space. Precision Bio, which has so far used its ARCUS gene-editing technology to advance the research efforts of its biotechnology partners, is now aiming to use its technology to develop its own products.

Sangamo is a clinical-stage biopharmaceutical company that is researching ways to commercialize Zinc finger nucleases, which modify a cell’s DNA at a location, thereby correcting or disrupting a specific gene. Its lead therapy, SB-728, is a potential functional cure for HIV/AIDS, and recent published data further support the company’s ongoing progress, which has been described as a major step toward immunological functional control of HIV.

All of this brings us to the subsequent ethical debate, which centers on the potential threat of gene editing, specifically gene-edited humans.

The fear of gene editing — and the concerns around what people could do through gene editing — isn’t based on any kind of rational fact.

It’s important to remember that animal transgenesis took place more than 35 years ago through a process that was immediately transposable to humans, and this has not led to any wave of transgenic humans. The same goes for the ability to knock out genes in human embryonic stem cells, cloning humans after Dolly the Sheep technology or using human iPS cells to create new clones. The fear of gene editing — and the concerns around what people could do through gene editing — isn’t based on any kind of rational fact.

People often ask: “What is gene editing? Should I be concerned about this. What happens if ill-intentioned people get their hands on this technology?”

The answer is complicated. Technologies such as cell phones and social media have fundamentally changed global society. For the vast majority, these changes have been for the good, even if bad people misuse them.

Gene editing is similar to this; it is a fundamental change in the way we look at the basic building blocks of life. It provides us with the ability to rethink how we treat diseases, how we grow our food and how we think about ourselves as humans.

The ultimate act of civilization was initially thought of as growing plants and breeding animals, hence genetic selection and cloning. Cloning, or selecting the best breeds, was initially done to improve survival. Since then, humans continued to perfect this technology.

With a population that’s close to reaching more than 9 billion humans on the planet, much of our survival may depend on the strength of gene editing. Furthermore, who cares today if a person is the result of in vitro fertilization? Do you remember the debate on this in the 1970s? This is no longer a debate.

2015 was a pivotal year, and gene editing is now transforming our lives in very real ways. The first leukemic patient — who could not be saved by any other therapy — was injected with a gene-edited CAR T-cell product candidate at the Great Ormond Street Hospital in the United Kingdom. She was the first patient helped by gene editing.

According to experts at the European Medicine Agency, this is the most complex product they have ever seen. It is the result of very sophisticated reprogramming of T-cells — adding some genes while suppressing others — to convert T-cells into a powerful cancer-killing machine.

This product can be produced by the thousands, stored long-term, provided to hospitals around the world and given to any patient who is in medical need. Today, this may be complex to produce, but it is simple to administer to patients. Tomorrow, it has the potential to become a standard in medicine.

2015 was an equally positive year for commercial agriculture, as gene-edited harvests across the United States were abundant, making it conceivable for gene-edited potatoes and soybeans to make it to consumer plates within two years. For the previous 50 years, the focus of plant breeding was increasing yield that resulted in greater productivity but included increased use of herbicides and pesticides. Until recently, the health of consumers was not a focus, resulting in a negative impact of mass agriculture and the rise of organic agriculture.

Today, organic agriculture represents less that 10 percent of current U.S. production. Nevertheless, with a growing population and an ever-diminishing cultivation space (not speaking about global warming, sustainability or equitable growth), a strong demand for healthier products and respect of nature is a paradox that can be solved either by economic shrinkage or technology. This upcoming harvest is the first step to finding an answer to this margin squeeze, and sets the stage for a new route to human expansion and sustainable development.

Again, it is not a question of if or when gene editing will happen; rather, it’s whether or not we would like to be the first to make it happen. As President Obama stated in his most recent State of the Union address: “Let’s make America the country that cures cancer once and for all.” This came one day after the launch of the Cancer MoonShot 2020 effort, led by big pharma and biotech companies. But we don’t have to wait until 2020 to administer a treatment that eliminates cancer cells. We are well on our way with gene editing.

Featured Image: Bryce Durbin

from TechCrunch

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