Patients with serious illnesses like cancer — of the pancreas, breast, and rectum — may need to take in with extra care journalistic reports on medical advances that might affect their treatment.
Two N’s will matter a lot to them — nuance and the scientific short-hand in which medical scientists communicate how many subjects participated in their research (the value described as N=).
The New York Times, in not one but three recent news articles, reported with a borderline Gee Whiz! approach that would be tempered by close reading of the stories in question that were headlined, thusly:
- Reprogrammed Cells Attack and Tame Deadly Cancer in One Woman (emphasis mine)
- A Cancer Trial’s Unexpected Result: Remission in Every Patient
- Breast Cancer Drug Trial Results in ‘Unheard-Of’ Survival
It doesn’t take a skeptic long to give more than a cursory glance at these reports to say, Well, yes, that’s interesting and perhaps of journalistic value. Other news organizations reported on some of these matters. Coverage of these studies, two funded by drug makers, arose from publication in respected medical journals and reports of research at an annual specialists meeting.
BUT: Step back from their novelty and issues arise. As is rapidly made clear about the first of these three stories, involving pancreatic cancer:
“Researchers have managed to tame pancreatic cancer in a woman whose cancer was far advanced and after other forms of treatment had failed. The experiment that helped her is complex and highly personalized and is not immediately applicable to most cancer patients. Another pancreatic cancer patient, who received the same treatment, did not respond, and died of her disease.”
So, what, the study could be reported as N=1 or N=2? Medical journals are filled with “case studies,” reports on just one unusual patient. These rarely get reported on by news organizations. That’s because it can be a significant challenge to generalize medical care from just one or two cases, or even several more.
Still, because notice of the pancreatic cancer case appeared in the New England Journal of Medicine, hundreds of words followed in the New York Times about what the publication’s editor describes as a “proof of concept” effort that is but one step among many to follow. How many? Not reported. How long will this take? Hard to guess and not delved into. Cost? Zero mention.
OK, but what about that cancer trial with remission in 100% of the cases? It sounds great. Hope it is. The smaller subhead, though, should start the head scratching. That’s because it explains this:
“The study was small, and experts say it needs to be replicated. But for 18 people with rectal cancer, the outcome led to ‘happy tears.’”
Sounds great? It might take reading several news articles, including two from the Washington Post, as well as the New York Times report, though, to get the full nuance of this admirable work, described at one point by the purported “paper of record” as “astonishing.” Here is a full paragraph of “yes, but …” notes from the Washington Post:
“The study does have caveats: The sample size of patients, while diverse in age, race, and ethnicity, was small. And even the earliest patients in the trial still have several more years of observation to ensure that the tumors haven’t reemerged or metastasized elsewhere in the body. The results also only pertain to those who carry a specific abnormality to their rectal cancer known as mismatch repair-deficiency, which impedes the body’s function to normalize or ‘repair’ abnormalities when cells divide and instead results in mutations. The deficiency occurs in roughly 5% to 10% of all rectal cancer patients and tends to resist chemotherapy.”
The New York Times also reported this:
“On average, one in five patients have some sort of adverse reaction to drugs like the one the patients took, dostarlimab, known as checkpoint inhibitors. The medication was given every three weeks for six months and cost about $11,000 per dose. It unmasks cancer cells, allowing the immune system to identify and destroy them.”
Hmmm. Let’s look now at the breast cancer study, which does have a sounder N=557. The author of the New York Times report includes this energetic description of this research, writing that patients received “treatment with a drug that targeted cancer cells with laser-like precision [and] was stunningly successful, slowing tumor growth and extending life to an extent rarely seen with advanced cancers.”
Before patients besiege their oncologists, the nuance of this care is worth noting:
“The trial focused on a particular mutant protein, HER2, which is a common villain in breast and other cancers. Drugs that block HER2 have been stunningly effective in treating breast cancers that are almost entirely populated with the protein, turning HER2-positive breast cancers from those with some of the worst prognoses into ones where patients fare very well. But HER2-positive cases constitute only about 15% to 20% of breast cancer patients, said Dr. Halle Moore, director of breast medical oncology at the Cleveland Clinic. Patients with only a few HER2 cells — a condition known as HER2-low — were not helped by those drugs. Only a small proportion of their cancer cells had HER2, while other mutations primarily drove the cancer’s growth. And that posed a problem because the cancer cells evaded chemotherapy treatments.”
Just for nuance’s sake, patients should consider this added information about trastuzumab deruxtecan, sold as Enhertu and the drug involved in this research:
“In patients who took trastuzumab deruxtecan, tumors stopped growing for about 10 months, as compared with 5 months for those with standard chemotherapy. The patients with the experimental drug survived for 23.9 months, as compared with 16.8 months for those who received standard chemotherapy. ‘It is unheard-of for chemotherapy trials in metastatic breast cancer to improve survival in patients by six months,’ said [Dr. Shanu Modi of Memorial Sloan Kettering Cancer Center], who enrolled some patients in the study. Usually, she says, success in a clinical trial is an extra few weeks of life, or no survival benefit at all but an improved quality of life.”
By the way, it’s worth noting at least two more things about Enhertu, as the New York Times reported:
“Like all chemotherapy, trastuzumab deruxtecan has side effects, including nausea, vomiting, blood disorders, and, notably, lung injuries that led to the deaths of three patients in the trials.”
And there’s this, quoting Dr. Susan Domchek, a breast cancer specialist at the University of Pennsylvania’s Abramson Cancer Center:
“She says that even before the Food and Drug Administration approves trastuzumab deruxtecan for HER2-low patients, she will see if the data from the new study will be enough to convince insurers to approve the drug, which has a wholesale price of about $14,000 every three weeks.”
In my practice, I see not only the harms that patients suffer while seeking medical services, but also their struggles to access and afford safe, efficient, and excellent health care. This has become an ordeal due to the skyrocketing complexity, uncertainty, and cost of treatments and prescription medications, too many of which turn out to be dangerous and bankrupting drugs.
Make no mistake about it: Doctors, hospitals, and medical scientists have made great progress in treating cancer and much credit is due to their hard work, persistence, and creativity. At the same time, it is important for all of us to remember that medicine is conservative — often for good reason. Yes, our medical-scientific knowledge explodes almost by the day. Still, respected clinicians take pains to protect patients, never experimenting with or on them with recklessness or negligence. Good doctors make every effort to safeguard those in their care. It may not be ideal and can be improved. But studies have shown it typically takes 17 years for innovations developed in labs or in rigorous clinical trials (as reported in medical journals) to make their way into mainstream care.
Still, as grizzled editors would remind, a huge component of the news is newness, so good journalists will jump on developments, even though their full import may not be apparent — for a while. That puts a lot of pressure on the injured and sick and their loved ones to, as a former president described it, never get too high or too low. We all need to not only stay current with medical-scientific news but also to increase our savvy, our capacity to process it ourselves and keep it in context, especially with our own medical care and needs. This is painfully so with cancer care, with its own specialists recognizing the “financial toxicity” of the disease’s costly, burdensome treatment.
We have much work to do to ensure we improve the safety, quality, effectiveness, affordability, and access of our medical services. We should neither look askance at advances that may change our lives nor swallow still developing, incomplete, or punched up information that can disappoint or even harm us.