Table of Contents
- 1 CRISPR: A Nobel, He Jiankui’s bombshell, and an ugly patent fight
- 2 Infectious disease: From Zika to Ebola to Covid-19
- 3 Opioid crisis: Fentanyl drives overdose deaths and lawsuits stack up
- 4 Drug pricing: Anger all around over hikes but little action
- 5 AI in medicine: Tremendous strides but its real impact still unknown
- 6 Gene and cell therapies: Balancing potential for cures with high costs and safety issues
The past five years have been packed with medical and scientific advances, a series of public health crises that have gripped the world, and uproar over rising prescription drug costs.
They’ve also been a heck of a time to launch a publication about health and medicine.
As STAT celebrates its five-year anniversary, our reporters took a look back at six areas we’ve covered closely — CRISPR, infectious disease, the opioid crisis, drug pricing, AI in medicine, and cell and gene therapy — to recap the biggest headlines and controversies and cast an eye to what may lie ahead.
CRISPR: A Nobel, He Jiankui’s bombshell, and an ugly patent fight
Even before STAT published its first stories, we knew CRISPR would be big: Breakthrough scientific papers in 2012 and early 2013 showed that this technique for changing the DNA of plants and animals was so easy to use that labs across the world would seize on it to understand basic biological processes as well as develop cures for genetic diseases. That’s why my first story for STAT profiled one of CRISPR’s inventors, biologist Feng Zhang of the Broad Institute. Check out his “Twinkle, twinkle, little star” analogy.
Sure enough, just five years later, CRISPR became Nobel big: Earlier this month, biochemist Jennifer Doudna of the University of California, Berkeley, and microbiologist Emmanuelle Charpentier of the Max Planck Institute for Infection Biology won the Nobel Prize in chemistry for their discovery of the CRISPR-Cas9 genetic scissors. The award was the first science Nobel won by two women.
What I never suspected was how fast a CRISPR nightmare might come true, how agonizingly long drug development takes, and what an ugly fight over patents CRISPR would spawn.
CRISPR’s inventors knew from the get-go that it would be theoretically possible to use the technique to alter the genes of human embryos, creating “designer babies.” That seemed like something a rogue researcher might try in, oh, 10 years. Yet there I was in Hong Kong in November 2018, at the second international conference on human genome editing, when China’s He Jiankui dropped his bombshell: He’d CRISPR’d human embryos, resulting in the birth of twin, genetically altered girls. That ignited a firestorm of condemnation and hand-wringing that the global scientific community hadn’t tried hard enough to stop him.
Also in the hand-wringing category: The fight over CRISPR patents between the Broad Institute and the University of California has been an eye-opener with its legal costs (well into eight figures; think of the science that would buy), ugly accusations, and sheer persistence.
Two happier CRISPR surprises: significant improvements on the original technique and the growing list of human diseases it might treat or cure, if success in lab mice is any indication.
With several companies as well as academic scientists already using CRISPR in clinical trials, one message from 2015 has stuck: CRISPR might actually live up to its hype, becoming the powerhouse genetic cure scientists dreamed of.
— Sharon Begley
Infectious disease: From Zika to Ebola to Covid-19
One of the first stories I wrote for STAT, five days after its official launch, was an almost eerie prediction of what was to come in 2020. Coronavirus researchers reported that a new SARS-like virus, isolated from bats, seemed capable of infecting people. Anyone else have goosebumps?
Before we got to Covid-19, though, there was the 2016 Zika outbreak. A virus spread by mosquitoes was shown to trigger microcephaly in some babies born to women infected in pregnancy. Zika didn’t prove to be a worldwide threat, but it devastated affected families.
Ebola again reared its ugly head — this time in what was effectively a war zone — in 2018. The North Kivu-Ituri outbreak on the eastern border of the Democratic Republic of the Congo was protracted and deadly. But it was also the first ever in which a new vaccine was widely used and therapies were successfully tested.
On New Year’s Eve 2019, I read a ProMed post asking for information about unexplained cases of pneumonia in China. I had covered the 2003 SARS outbreak, and this sounded eerily familiar. Here we go, I thought.
And here we are. The virus responsible for those pneumonia cases, now called SARS-CoV-2, prompted China to take the unprecedented move of placing tens of millions of people into lockdown, for weeks. That fact alone should have conveyed the urgency of the situation, yet the response outside of China was regrettably slow, and, in the U.S., laced with politics. In the most polarized of times, taking precautions against Covid-19, the disease SARS-2 causes, has broken down along red state, blue state lines. Even after President Trump contracted Covid-19 and spent days in hospital, getting experimental treatments, he persists in insisting the virus will soon disappear and is nothing to fear.
More than 42 million people worldwide have been infected and more than 1 million have died. The U.S., with its dismal pandemic response, has more cases than any other country on the planet, more than 8.5 million, and the highest number of deaths, surpassing 225,000. Vaccines should soon start to become available, but pre-Covid lifestyles will remain out of reach for some time.
— Helen Branswell
Opioid crisis: Fentanyl drives overdose deaths and lawsuits stack up
In STAT’s first year, I was dispatched to Huntington, W.Va., where in one afternoon, more than two dozen people overdosed. The spate earned national attention for the danger posed by the potent synthetic opioid fentanyl and its chemical cousins, which were expanding across the country at that point into new territory. It also came as families were growing more vocal about losing loved ones to overdoses, a movement that has reshaped how addiction is viewed and inspired policymakers to dedicate more attention to the problem.
Since then, fentanyl has become a leading source of overdose deaths in more places. But even as the toll from fentanyl grew, other bright spots emerged: The Affordable Care Act, in particular its expansion of Medicaid, provided treatment coverage for more Americans. Congress and the Trump administration approved billions of dollars to fight addiction. And for the first time in decades, U.S. overdose deaths saw a year-to-year decline from 2017 to 2018, driven by drops in deaths from prescription opioids. More than 67,000 people still died that year — a sign of just how deeply entrenched the problem was — but the wish was that the tide was starting to turn.
Tragically, that doesn’t seem to have been the case. Overdose deaths ticked up again last year and are expected to rise again this year, fueled by the isolation and economic collapse from the pandemic. It’s not just fentanyl that’s causing the concerns, either. Deaths from stimulants like methamphetamine have surged in recent years, and unlike for opioid use disorders, there are no approved treatments for meth addiction.
Another key story over the past five years has been the stacks of lawsuits filed against opioid manufacturers, distributors, and other players in the supply chain for their roles in allegedly seeding the epidemic. The lawsuits have helped push Purdue Pharma, the maker of OxyContin, into bankruptcy proceedings, and just last week, Purdue agreed to plead guilty to three felony criminal charges in a settlement with the federal government. A separate three-year legal fight waged by STAT helped crack open new details about how members of the Sackler family steered Purdue’s strategy as scrutiny of its medications grew. In Oklahoma, a judge has ordered Johnson & Johnson to pay hundreds of millions of dollars to the state. (The company is appealing.)
More broadly, a so-called multidistrict litigation involving thousands of lawsuits against a range of defendants could lead to a national settlement akin to the tobacco litigation. The question then would become: What happens to the money, and can it make a dent in the country’s addiction crisis?
— Andrew Joseph
Drug pricing: Anger all around over hikes but little action
There are rare moments when working at STAT’s outpost in Washington, D.C., leaves us on the outside looking in. Health and medicine are huge political issues, of course. But sometimes, the chaos of the Trump era (Mueller, Charlottesville, impeachment — take your pick) dominates the day’s news in ways that have little to do with health care or life sciences.
The national uproar surrounding prescription drug prices has provided the perfect antidote: It’s our window into the very heart of what goes on at the White House and on Capitol Hill.
In recent years, we’ve watched drug prices morph from a D.C. afterthought to a marquee political issue. Once President Trump used his first post-election press conference to allege that drug companies were “getting away with murder,” we were off to the races. Democrats soon made runaway drug costs a central theme in the 2018 midterm elections. Seizing on the momentum, House Speaker Nancy Pelosi made an aggressive drug pricing bill a centerpiece of her 2019 agenda, though Congress remains gridlocked without Senate Republicans’ support.
Despite mounting pressure, the pharmaceutical industry remains as powerful as ever. They spend millions on lobbying and contribute to thousands of politicians at the federal, state, and local levels. That’s been true for decades — but recently, we’ve seen big money line up behind efforts to regulate drug prices, some from grassroots groups but mostly from a Texas billionaire couple billing itself as a pharma “counterweight.”
Congress, nonetheless, has done little, leaving a flailing Trump administration to attempt major reforms on its own. Despite four years of anti-pharma rhetoric, however, the White House’s own efforts have been beset by chaos and infighting. With his reelection odds fading, Trump is growing more brazen. Weeks before Election Day, he unveiled a slate of executive orders that almost certainly won’t take effect before Nov. 3 — including a transparently political effort to send $200 prescription drug coupons to millions of seniors.
Drug makers have continued hiking prices throughout Trump’s term, and millions of Americans still can’t afford to fill prescriptions. In short: Despite yelling and screaming from almost every corner of D.C., we find ourselves today largely where we were five years ago. Drug industry allies are fearful, of course, of a hypothetical future in which Joe Biden is president and Democrats control all of Congress. But if we’ve learned anything, it’s that there’s little point predicting what happens next.
— Lev Facher
AI in medicine: Tremendous strides but its real impact still unknown
When STAT started investigating IBM’s Watson supercomputer, the rapid progress of artificial intelligence was fueling expectations for a revolution in health care. But our findings in 2017 gave reason for pause: Watson’s use in cancer care was not living up to the hype. In fact, doctors using it all over the world told us its treatment recommendations were biased and, in some cases, plainly wrong.
The story triggered an avalanche of reaction. Sources began reaching out with tips and internal documents showing that IBM’s executives had continued to aggressively promote the product — even as examples surfaced within the company of its unsafe and incorrect treatment advice. Watson went from being the leader of AI’s revolution to a cautionary tale.
Reporting that story reinforced the importance of the moment medicine is facing. Artificial intelligence is making tremendous strides, and I have continued to write about research demonstrating its potential to spot cancer early and flag declines in heart function in time to dramatically improve care. AI is also helping pharmaceutical companies discover new medicines, design better trials, and track patients’ response to treatments.
In addition, AI is becoming a major force of change in public health. During the Covid-19 pandemic, it has been deeply involved, and sometimes overhyped, at every phase of the crisis. It was used to help track its early spread, trace potential exposures, and create online symptom checkers to assess patients’ risk of developing Covid-19. Drug researchers used it to identify existing medicines that could be repurposed to improve patients’ chances of survival, and hospitals are now leaning on it in the midst of flu season, to help differentiate between viruses and predict which patients are likely to become severely ill.
But the story of AI’s real impact on health care is still unwritten. Its promise to improve health care is equaled by its potential to cause great harm — by violating patients’ privacy, increasing costs without producing real benefits, and perpetuating unequal treatment along racial and economic lines.
I just finished an investigation into how an earlier generation of analytics software has infused racial bias into decision-making about which patients should receive stepped-up care for chronic illnesses. Developers of the next generation of AI are vowing their products will not do the same. We at STAT will be watching every step.
— Casey Ross
Gene and cell therapies: Balancing potential for cures with high costs and safety issues
Last January, my colleague Matt Herper and I marked the start of a new decade by compiling a list of the 10 drugs that had the biggest impact on the companies that sell them, on medicine, and on society as a whole. Two of the 10 drugs on the all-star list were gene and cell therapies. We could have easily doubled that number.
Over the past five years, STAT has chronicled some of the remarkable scientific achievements made in the field of gene and cell therapy. Certain types of rare, inherited diseases can now be treated for the first time — cured, in some cases — by replacing malfunctioning genes with ones that are healthy and function normally.
The first U.S. approval of a gene therapy occurred in 2017. That one-time treatment, called Luxturna and developed by Spark Therapeutics, targeted a rare form of childhood blindness. Two years later, a small biotech called AveXis (later bought by Novartis) secured the second U.S. gene therapy approval. Zolgemsa is a lifesaving treatment for children born with spinal muscular atrophy, a progressive neuromuscular disease that, in its most severe form, causes infants to die or rely on permanent breathing support by the age of 2.
Patients with certain types of blood cancer are also living longer because their own immune cells are being reengineered in a laboratory to seek out and kill tumor cells. The first of these so-called chimeric antigen receptor T cell (CAR-T) therapies was approved in 2017. Both Kymriah, developed by Novartis, and Yescarta, developed by Kite Pharma (later acquired by Gilead Sciences), target blood cancers in children and adults caused by malignant B cells.
Still, some questions and concerns remain. Gene therapies are intended to be one-time treatments, yet how long patients benefit and what happens if these treatments stop working remains to be seen. The recent deaths of patients in a gene therapy trial are a reminder that, despite improvements in the technology, significant safety risks remain.
And then there’s the cost. Gene and cell therapies are extraordinarily expensive. At $2.1 million, Zolgensma is the highest-price medicine in the world. Who gets access to these potentially curative treatments, and how they are paid for, has become part of the ongoing, national debate over rising prescription drug costs.
There are now dozens of gene and cell therapies for inherited diseases and cancer in clinical development. Over the next several years, potentially curative gene therapies for hemophilia, Duchenne muscular dystrophy, and sickle cell disease could be approved and ready for patients. The cell therapy field in cancer is also evolving rapidly, with off-the-shelf treatments that are more convenient than today’s patient-specific offerings showing early but promising results.
— Adam Feuerstein