Medicine stands at the precipice of an exciting new era.
We’re closer than ever to achieving functional cures for once-intracable diseases, including HIV. Stem-cell treatments are repairing blinding eye damage and stabilizing failing hearts. Emerging cancer treatments promise to extend patients’ lives and decrease the likelihood that their disease will return. And cutting-edge treatments are sparing children from devastating genetic diseases.
That’s the question circling my brain as I look ahead to 2026. While I’d love to focus solely on how far we’ve come, it’s impossible to ignore the ground we’ve lost in recent months.
Exciting developments to watch
As anticipated, 2025 was a breakthrough year for gene therapy, and I expect 2026 to bring more exciting developments in the field.
KJ Muldoon, a baby born with a rare genetic disease, became the first person to receive a customized CRISPR treatment. The two CRISPR-based therapies approved to date are one-size-fits-all, and they require cells to be removed, edited in a lab, and then reintroduced into the body. KJ’s treatment, by contrast, was made to tweak a specific mutation in his cells, and the editing took place inside his body.
One of KJ’s doctors told me that they’re now working with the Food and Drug Administration to make these bespoke treatments easier for patients to access, so hopefully, more people will benefit from such therapies in the coming months. (Notably, though, baby KJ’s treatment used mRNA — a molecule that also formed the basis of the first COVID-19 vaccines. The federal government is retreating from mRNA vaccines, but other uses of the technology may be spared.)
In the meantime, scientists are trialing a gene therapy for Huntington’s disease that may slow its progression — a feat never realized with any existing treatment. A CRISPR treatment for high cholesterol is making its way through trials, as is a gene therapy for congenital deafness and a new cancer therapy that involves base editing immune cells. And in preclinical research, scientists are developing new gene-editing systems that could someday enable “mutation agnostic” treatments that work for many people, as a complement to therapies that correct very specific mutations.
This year, we also saw results from a U.K.-based clinical trial of “mitochondrial donation,” a technique that’s been in the works for years and is finally being tested in people. The approach, done in the context of in vitro fertilization, aims to prevent mothers who carry harmful mutations in their mitochondrial DNA from passing those mutations to their kids. In the early trial, the approach appeared to be successful, and I’ll be interested to see how the research proceeds.
We’ve also seen GLP-1s — Ozempic and other drugs in the same class — become more commonplace, and we’ve been learning about their potential benefits beyond weight loss and blood-sugar control. There are early signals that these drugs may help treat migraine, alcohol use disorder and heart failure, for instance. I expect these findings will spur interesting research into the underlying relationship between these conditions and metabolism.
That said, I don’t think the drugs will be a silver bullet for all diseases — they just failed in a hotly anticipated Alzheimer’s disease trial, for example. Nonetheless, research on GLP-1s may uncover previously unappreciated drivers of disease that could be tackled by other means in the future.
I’ll also be keen to follow emerging research on senolytics — drugs that clear senescent, or biologically aged, cells from the body.
Xenotransplantation — the transplantation of animal organs into humans — continues to progress by leaps and bounds as experiments and trials with humans unfold around the world.
And as research increasingly reveals the role of viruses in dementia, I expect the next few years of studies could fundamentally rewrite our understanding of neurodegenerative disease and how to treat it.
Dissolution of public health
From a technological and research standpoint, there’s a lot to be excited about. But the horizon looks darker when you cast your eyes to the realm of public health and the systems that fund and regulate research and new drugs, at least in the United States.
President Trump’s second administration ushered controversial new appointments across the country’s leading health agencies — as well as deep budget cuts. Vaccine and medical-establishment skeptic Robert F. Kennedy Jr., now at the helm of the Department of Health and Human Services, spearheaded dramatic changes across its divisions, such as the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC).
The NIH has signaled that it’s deemphasizing the practice of studying both sexes. Given that females are understudied at baseline, experts worry that such a move will widen existing knowledge gaps. The agency’s leadership has also argued that collecting demographic data — on study participants’ race, ethnicity or gender identity — should be avoided except in circumstances deemed “scientifically justified,” a phrase with no clear definition.
In the next year, I expect these moves to derail research aimed at understanding health disparities and improving care for marginalized and understudied populations. Disrupting this research today means prolonging these disparities in the future.
Former CDC leaders have reported witnessing a profound disconnect between RFK Jr. and the agency’s scientific staff, a lack of strategy surrounding policy changes, and a dismissal of established research findings. Meanwhile, a new vaccine advisory committee handpicked by RFK Jr. has cast doubt on the well-established childhood vaccine schedule.
Some recent committee decisions have been more confusing than directly consequential, such as those regarding the measles vaccine and the COVID-19 vaccines. Still, set against the backdrop of RFK Jr. broadly undermining trust in vaccines, even these changes could lower vaccination rates in a country already poised to lose its measles elimination status. And other committee decisions, such as recommendations to delay hepatitis B vaccination for newborns, have the potential to cause direct and significant harm right away.
As the CDC is dismantled and its career scientists are ignored, devalued or fired, I anticipate further holes to be poked in the nation’s public-health safety net in 2026. Some decisions may primarily stoke confusion and mistrust around established medical practice. Others may bar access to care by revoking federal insurance coverage or withholding reimbursement to hospitals that provide certain types of care.
The exact impacts of forthcoming changes will likely be piecemeal, varying from state to state, similar to how we’ve seen abortion access splinter in the wake of Roe v. Wade’s overturn. But nationwide, it’s fair to expect upticks in vaccine-preventable disease.
For trustworthy health guidance, I would recommend sources such as the American Academy of Pediatrics, the American College of Obstetricians and Gynecologists and other professional medical associations; the independent health-policy resource KFF; and the University of Minnesota’s Center for Infectious Disease Research and Policy, including its Vaccine Integrity Project. Local health departments and regional coalitions, such as the West Coast Health Alliance, should also help fill the information gap left by federal agencies.
But given that the average person is already bombarded with conflicting health guidance — especially online — I’m concerned that the loss of centralized sources of science-backed information will ultimately put more people at risk of preventable disease.
Beyond America, and into the future
The Trump administration also shuttered the U.S. Agency for International Development (USAID) this year, pushing a handful of the agency’s prior functions under the Department of State.
USAID, previously the world’s largest foreign aid agency, had programs aimed at combating infectious diseases like HIV and tuberculosis, reducing malnutrition, cleaning water systems, and bolstering maternal health care around the world. Its loss left governments and organizations scrambling to make up the funding shortfall, but they likely won’t be able to fill the gap completely, stakeholders have warned. Even if they do, delays in funding still mean delays in care, which can be deadly.
Prior to USAID’s closure, experts worldwide were cautiously optimistic about bringing an end to the HIV epidemic by 2030. Now, models suggest that the loss of the agency could usher millions more HIV cases and deaths in low- and middle-income countries than anticipated over the next five years. Looking beyond HIV to all of USAID’s former programs, estimates suggest that the closures have already contributed to hundreds of thousands of deaths from infectious diseases and malnutrition worldwide.
The U.S. is not immune to the ripple effects of USAID’s dissolution.
“One of USAID’s most critical functions is to fight the spread of infectious diseases that have the capacity to spark a global pandemic,” Dr. Chris Beyrer, an epidemiologist and director of the Duke Global Health Institute, wrote for Live Science in March. “While much of this work is carried out far from the U.S., infectious diseases know no borders, and we have seen countless instances of viruses that arise in one part of the world but quickly find their way to other countries.”
This, to me, underscores a key point about public health: It’s a group project. Improving conditions for those most vulnerable to disease benefits everyone in the long run, not only by reducing suffering and saving lives but by cutting health care costs and bolstering economies. One could say the same about the efforts to curb climate change and environmental pollution — efforts that the current administration is also repudiating.
I look forward to following the development of groundbreaking medical treatments over the upcoming year. These emerging technologies promise to alleviate the suffering of individual patients — if they can access them. But even as we celebrate those accomplishments, I worry that their benefits simply won’t reach a huge portion of the populace.
Headlines about the next great gene therapy will run alongside news of rising infection rates and deadlier climate-driven disasters. Early data hint that senolytics could help stave off age-related diseases — but even as those drugs get developed, falling vaccination rates mean we could return to a time when a lot more people die in childhood than have in recent decades.
My hope for 2026 is that the scientists and stakeholders still committed to protecting public health will persevere and find ever-expanding support so that everyone can reap the benefits of medical science.
This article is for informational purposes only and is not meant to offer medical advice.
