Over the past decade, our ability to alter the human genome has rapidly improved, largely due to advancements in genome editing technologies like CRISPR. Scientists are vigorously pursuing the ultimate goal of making any change to any DNA sequence in any cell of the body. The versatility of these tools has opened the door to treating a range of debilitating diseases, from prevalent neurological conditions to rare forms of cancer. Yet, alongside promising therapeutic applications, we should be worried about the potential unethical and inequitable uses of these technologies.
Universal concern over the potential use and misuse of genome editing reached a peak in 2018 following the surprise announcement of the first edited babies. He Jiankui, a professor overseeing a university lab in China, publicly disclosed his efforts to engineer HIV immunity into human embryos. The international scientific community decried He’s experiment, and the Chinese government eventually filed criminal charges against this rising young scientist. He Jiankui is currently serving a three-year prison term after a Chinese court found him guilty of “illegal medical practice”.
Criticized as unsafe and unnecessary, altering human embryos for reproductive purposes arguably crossed a line and added urgency to the question: How should human genome editing be regulated? A national approach is clearly flawed. A menagerie of different country-level regulations would likely lead to medical tourism—traveling outside of one’s home country for access to a restricted medical procedure. But what would governance on a global scale look like?
In 2020, the United States’ National Academies and United Kingdom’s Royal Society released a report describing what boxes should be checked before attempting to initiate a pregnancy with an edited human embryo. For instance, doctors should sequence the embryo’s DNA to look for any unintended changes.
Despite providing a list of experimental do’s and don’ts, this report did not settle how to—and who should—oversee the ethical use of this technology on a global level. To answer this question, an advisory committee to the World Health Organization began a two-year inquiry looking specifically at this issue.
In July, the committee, composed of leading scientific experts from around the world, released two reports: a framework for governance and recommendations on human genome editing. These documents provided several case studies, covering the many different ways genome editing can be used to alter human cells, ranging from curing diseases through to enhancing physical ability.
The report addressed the clear harms of altering embryos and suggested ways to monitor unethical research on a global scale. For instance, the committee recommended creating a whistleblowing mechanism for scientists to report illegal activity.
The committee also confronted an equally, if not more, concerning consequence of human genome editing that has received much less attention than embryo editing: Following in the footsteps of other therapeutics such as cancer drugs and vaccines, genome editing therapies appear to be on course to further amplify existing global health inequities.
The committee stressed a need for global health justice, recognizing that as genome editing technology progresses, many countries are poised to be left behind. If not addressed, patients with rare genetic diseases may be left finding their cures siloed in high-income countries where hospitals have the sophisticated equipment, trained staff, and payment structures necessary to offer these therapies.
To date, gene therapies for diseases like Leber congenital amaurosis (a hereditary eye disease) and spinal muscular atrophy (a degenerative nerve disease) are approved in several countries. Gene therapies, which randomly insert a healthy version of DNA into the genome, work differently than genome editing, which makes precise genetic alterations. Approved gene therapies currently on the market are priced anywhere from $300,000 to $2.1 million, staggering numbers for individuals even in high-income countries. Pharmaceutical company Bluebird Bio recently pulled its gene therapy for beta-thalassaemia, a rare blood disease, from European countries over disagreements about price setting, signaling the difficulty of bringing these expensive, lifesaving therapies to patients.
So, what does global health justice look like for a seven-figure drug? More importantly, who’s role is it to ensure that patients around the world benefit from future genome editing therapies? To begin answering these questions, we must start with understanding where the technology is now, where it’s headed, and who will most benefit.
In the U.S. alone, billions of dollars are going toward developing genome editing technology to treat a long list of debilitating diseases. This approach is called “somatic cell editing,” a less controversial counterpart to “germ cell editing,” which involves altering egg, sperm, and embryos. Changes to somatic cells will not be passed down to future generations but can still be used to cure a disease for the lifetime of a patient.
The landscape of diseases that could potentially be cured using genome editing is vast. Scientists are developing new ways to target cells throughout the body, from neurons in the brain to retinal cells in the eye. Early research and clinical testing, conducted in China, the U.S., and elsewhere, have shown the potential of this technology for correcting muscular dystrophy, Huntington’s disease, and even certain forms of blindness. Despite promising early results, genome editing therapies are still in clinical trials and no treatments are approved by regulators to enter any country’s health care markets. While still hypothetical, these clinical efforts are clear indications of what the future therapeutic landscape may look like.
Experts point to efforts to cure sickle cell disease as foreshadowing how genome editing will affect patients and health care systems. Sickle cell disease is a genetic condition that results in distorted red blood cells, resulting in a reduced life expectancy of 42–47 years in the U.S. Multiple clinical trials to cure sickle cell using genome editing, led by industry and academic groups, are underway, but the final cost of any approved treatments will inevitably be enormous. These procedures involve removing the cells from the bone marrow of a patient, altering the DNA within those cells, and reinserting the edited cells back into the body. This process requires sophisticated equipment, highly trained personnel, and long stays in a hospital. Projections estimate that this therapy will be priced well over $1 million per patient, in line with the most expensive approved gene therapies, but this time for a much larger patient population—approximately 100,000 people in the U.S. live with sickle cell disease.
Large insurance companies and state Medicaid programs are scrambling to find ways to cover a future influx of genome editing treatments, challenging traditional ways of paying for drugs. In the U.S., individuals who are on small-employer insurance plans or don’t have insurance will find accessing these procedures extremely difficult, if not impossible. What, then, for people in low-income countries who could benefit from such treatments?
Assuming that a cure for sickle cell disease may be one of the first approved genome editing therapies, with the potential to benefit millions of people globally, how will a multimillion-dollar treatment help shift the needle toward global health equity?
The short answer is that it won’t. Indeed, if left to the market, global health inequities are likely to only be exacerbated. A different approach must be embraced to create effective treatments that are not only safe, but broadly accessible and also affordable. This was the mindset behind a collaboration between the Bill and Melinda Gates Foundation, pharmaceutical company Novartis, and the U.S.’s National Institutes of Health.
This collective effort aims to create a genome editing cure for sickle cell disease and HIV that will work for patients in low-resource settings like Africa or rural America. In short, they want to introduce genome editing molecules directly into the body without removing any cells, cutting down on cost and time. Technologically, this is a herculean goal. But if successful, it would offer an accessible cure for the 12–15 million people in Africa living with sickle cell disease, 90 percent of whom will not reach 18 years of age without interventions.
If the Gates project succeeds, will it be a one-off experiment or a model for future therapies? Translating this cross-sector collaboration into a standard approach for developing accessible cures would benefit from international oversight, a role that some say the WHO could fill—but many also wonder if it is up to the challenge.
The world is currently experiencing a disastrous example of global health injustice. While only 1 percent of people in low-income countries are vaccinated against COVID-19, U.S. citizens will likely begin lining up to receive booster shots in the coming months. Since the beginning of the pandemic, the WHO has worked to promote equitable access to COVID-19 vaccines through initiatives like COVAX, aiming to deliver doses for at least 20 percent of countries’ populations.
Yet their effort has been met with little success. Vaccine hoarding by high-income countries has presented itself as a significant obstacle to COVAX. Global vaccines access has also been stalled by an unwillingness of countries to agree to a temporary waiver of intellectual property rights associated with COVID-19 vaccines and related technologies.
The global pandemic has clearly illustrated a need for an organization that can show moral leadership, safeguarding the health and well-being of all citizens. The WHO has sought to do this over the past 18 months. Now the WHO’s advisory committee is calling on the organization to play a similar “moral” leadership role for genome editing, but vaccines, which have been around for more than a century, are a stark difference from genome editing, a technology with numerous unknowns.
Oversight of emerging technologies is outside of WHO’s original mission, but that function is beginning to change. WHO is currently assisting countries with evaluating how emerging technologies will affect the well-being of their citizens, and gene editing technologies are, arguably, one of the most significant new technologies for addressing health-related challenges. Even though WHO is still securing its footing on the rocky terrain of emerging technologies, it may be the best organization to help society direct its moral compass toward global health equity.
Part of this process will involve convening governments and policymakers, anticipating and articulating potential consequences of genome editing on health equity, and fostering innovative collaborations to ensure that genome editing therapies benefit all. Low-resourced countries were left on the periphery during the COVID-19 pandemic. Let’s make sure that geography doesn’t impede access to the benefits of future genome editing therapies.
Future Tense is a partnership of Slate, New America, and Arizona State University that examines emerging technologies, public policy, and society.