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I’m going to keep this short because I really want you to check out Jason Mast’s new story. I’ve got a bit of a teaser below, but the whole remarkable tale is worth your time. Stay well and thanks for reading.
A biotech company by and for Amish and Mennonite people
First, they saved the cow. Petunia was suffering from a bovine form of maple syrup urine disease, a life-threatening condition unusually prevalent in Pennsylvania’s Mennonite community.
At the local veterinary hospital, Petunia was given a novel gene therapy. Today, she is a healthy 5-year-old heifer.
The doctor behind this procedure wanted to save the calf, of course. But his mission was bigger — Kevin Strauss is building a biotech company by and for the people he treats inside a wooden, mortise-and-tenon frame medical center. The goal is to develop innovative gene therapies to treat the unique set of rare diseases that afflict Amish and Mennonite people.
Jason Mast has an inside look at the new company, including its effort to resurrect treatments abandoned by pharma or left to gather frost in an academic freezer, often because there are too few patients for a company to generate profit.
Our roundup of the big European cancer conference
My colleague Adam Feuerstein and I are leaving Madrid after a packed four days at the European Society for Medical Oncology’s annual meeting. Selecting which readouts to cover is a form of triage given the massive amount of research being detailed (if you think we missed something game-changing, let us know), but one highlight in terms of outcomes didn’t come from some new drug. Rather, it was the combination of Amgen’s Imdelltra and AstraZeneca’s Imfinzi showing that it greatly extended survival in difficult-to-treat small cell lung cancer — and showing the potential of paired approaches.
Other highlights:
Yes, the big readout was Merck and Moderna’s cancer vaccine, and yes, the wind got a bit taken out of the presentation’s sails with the embargo break. But it’s still a milestone in cancer research. Here’s our deep dive into what the results mean for melanoma care, and what they could mean for moving cancer vaccines into other tumor types. And don’t miss Adam chatting to Moderna CEO Stéphane Bancel about the impact for the company, and where it goes from here. (We also got some early data from a vaccine designed to prevent tumors in an inherited condition that leaves people at much higher risk of cancer.)
Scientists figuring out how to target KRAS led to a major advance in pancreatic cancer, as you’ve probably heard. Now more KRAS-targeting drugs are coming, often going after specific mutations. Roche unveiled strong data from divarasib in non-small cell lung cancer, and Incyte showed promising results from INCB161734 in pancreatic cancer.
Three years ago at ESMO (coincidentally the last time the conference was in Madrid, so a real hardship assignment for me), I wrote about how antibody drug conjugates were all the rage. That continues to be the case, with important readouts from Merck, AstraZeneca, GSK, and beyond. One thing I’m thinking about going forward: These drugs in theory should be safer than the standard chemotherapies they’re designed to replace, but some of them carry quite serious side effects themselves. With so much money pouring into ADC design, is there a way to reduce rates of those adverse events? — Andrew Joseph
Too many emergency departments can’t handle child patients
A few years ago, parents rushed their baby to a small rural hospital. But the facility didn’t have pediatric specialists, pediatric nurses, or a children’s intensive care unit. As the little boy’s condition worsened, the hospital’s staff scrambled to identify a facility that could take him. But every children’s hospital in northern California was overwhelmed. No one had room for one more.
The baby died of RSV. And the problem has only gotten worse since then, write two pediatric care experts in a new First Opinion.
“In this new normal, community hospitals across the country are transferring children to dedicated pediatric centers not just because a child is critically ill, but increasingly because smaller hospitals have quietly lost the ability to manage even common conditions,” they write. “The nation’s relatively few children’s hospitals can’t accommodate every sick child, much as we would like to. We now routinely turn sick children away and hope one of our colleagues somewhere else will have a bed.”
What’s the solution? Read on.
The Washington news you need this week
Every week over in D.C. Diagnosis, STAT’s John Wilkerson highlights the most important news he’s watching. As midterms creep ever closer, here’s what’s on his mind:
- Tuesday is the second day of the National Organization for Rare Disorders’ Breakthrough Summit, which will feature the heads of the FDA and NIH.
- On Wednesday and Thursday, Secretary of Health and Human Services Robert F. Kennedy Jr. will stump for Iowa GOP gubernatorial candidate Zach Lahn.
- Also Wednesday, outgoing White House senior adviser Calley Means holds a fireside chat at Tufts University’s Food is Medicine Institute.
Election day is only a week away! Keep up with the latest health care politics news on our midterms page.
A possible treatment for brain-eating amoebas, found in the sea
Brain-eating amoebas are the stuff of zombie novels and spooky season. They are also, unfortunately, real-world parasites found in warm freshwater. Infections caused by N. Fowleri are hard to treat and nearly always fatal. But researchers have isolated a compound that could help stop the disease.
The substance was taken from a marine bacterium, and has been able to kill multiple strains of N. Fowleri faster than the current treatment. There is much work to be done before this discovery can translate to medicine though. Researchers say their next focus will be studying whether the compound can cross the blood-brain barrier.
What we’re reading
- Is rabies making a comeback? New York Times
- New clues point to why IBS is twice as common in women as in men, The Washington Post
- ‘It’s a mess right now’: RFK’s sweeping changes place new vaccines in limbo, The Guardian
- Cancer vaccines are finally here. How do we get them to patients? STAT