The ability to edit the human genome has been eagerly anticipated for many years.
While the possibilities are exciting for many, there are serious ethical concerns that are already beginning to be bypassed.
In discussions of genetics and gene editing, cancer, cystic fibrosis, and other well-known diseases often come to the forefront, and as clinical trials are already underway for cancer patients for genetic therapies, it is clear that this will remain the case.
However, it is rare diseases that will benefit the most from gene editing.
Currently, new therapies cost over $1bn to develop. In addition, only approximately one in 10 drugs in Phase I testing will go through FDA approval.
As a result, from a financial perspective, therapies for diseases affecting a smaller population will likely result in a poorer return on investment.
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By GlobalDataThe incentive to develop drugs for rare diseases is not sufficient to meet current needs. The potential for gene editing for individuals with these rare diseases would mitigate this current lack of therapies, opening avenues for new genetic therapies.
Changing the game
In 2015, breakthroughs shifted these ambitions from the realm of science fiction into a tangible possibility.
As ethical concerns mounted, the scientific community called the International Summit on Human Gene Editing.
The statement issued at the close of the meeting concluded that creating hereditary modifications by editing germline cells would be “irresponsible” unless relevant safety issues have been resolved and there is “broad societal consensus” about the use of such applications.
A little over a year later, the US National Academy of Sciences and National Academy of Medicine released a report that concludes clinical trials on “heritable germline genome editing should be permitted,” under oversight by the committee for specific criteria.
Professor Francoise Baylis, who was a member of the organising committee for the 2015 summit, comments that in a mere 14 months, germline editing has shifted from “irresponsible” to “permitted,” and the requirement for societal consensus, as vague a requirement as it is, has been omitted.
Gene editing has already begun in clinical trials.
Two children with leukaemia treated with edited immune cells in 2015 are both healthy almost two years later.
Due to the recent advances the process of editing genes takes mere weeks, where previously this took many years.
As a result, a human trial is already underway in China, with a US trial greenlit and expected to begin next year.
Human trials have come about startlingly quickly
According to the New Scientist, there are now 20 trials awaiting approval, predominantly in China. This competition has been likened to the space race, a Sputnik 2.0, with China and the US racing to be the first to use the breakthroughs in patients.
In the realm of cancers and diseases such as HIV, it is easy to be excited for the possibilities of gene editing.
If proven to be safe, gene editing will likely become a widespread treatment and cure for many diseases.
The issue arises when we move away from easily defined and pathogen-led diseases into murkier waters.
Many deaf people do not view deafness as a disability requiring treatment; it is simply a part of their culture and community.
People with Down syndrome similarly do not require treatment to live a full life. There is no robust line between treatment and enhancement in many cases, and with gene editing there becomes a risk of medicalising differences.
With germline editing now considered permissible, the demarcations of acceptable changes must be discussed and set, not only by the scientific community but by the wider population as well.
By enabling widespread genome editing, humans could be in control of our evolution, with possibilities to cure numerous diseases and minimise suffering.
However, only the most optimistic can believe these treatments will be available to all. If genetic treatments move towards enhancements, social inequality could become encoded into our DNA.
Gene editing presents vital issues that deserve to be debated outside the scientific community.
Perhaps just because we can, does not mean we should.
