April 3, 2023

Gene therapy expands to a skin condition

Aaron-Owens_by-Constanza-Hevia-H.-15

Constanza Hevia for STAT

Aaron Owens (above) has lived his life covered in bandages. His skin is so fragile the slightest friction tears his tissue. An 18-year-old high school student, he has the genetic condition epidermolysis bullosa, or EB. A few years ago he enrolled in clinical trial testing B-VEC, developed by Krystal Biotech, against some wounds and a sham dose against others. It worked.

Owens said that before the treatment, 70% of his back was completely raw. "Now it's 100% healed. I can sleep on my back," he told STAT's Andrew Joseph. "I don't have to worry about so much pain and blood in the shower. It's more than life-changing. It's easier to do everything." The therapy is not only the first treatment for EB, but also the first topical gene therapy and the first for a skin condition. The FDA is set to make a decision on approval by May 19. Read more.

medicine

White House Covid adviser urges doctors to combat medical misinformation

The coordinator of the Biden administration's Covid-19 response urged doctors to take a leadership role with patients to battle medical misinformation and disinformation. "The reason it has taken root is because there was an information vacuum," Ashish Jha said at a conference for physicians on Friday. "It is critical that we fill that vacuum because if we don't, others will."

Jha connected misinformation and disinformation with the 250 to 500 daily Covid deaths he's tracked while working in the White House. "If you are up to date on your vaccines and you get treated with Paxlovid, if you get an infection, you just don't die of this virus," he said. "Almost every one of those deaths is preventable. And yet people are still dying. And that is the power of misinformation. That is the power of disinformation that we all have to work on countering." I have more here.

pandemic
Leaders of long Covid clinical trials call for treatment centers

Where are the answers on long Covid? Patients, doctors, and scientists have been clamoring for some clarity on what defines the condition, who gets it, how to prevent it, how to treat it, and whether it ever goes away. The NIH has mounted its own multisite research effort, called the RECOVER trial, but its pace has been compared to a "slow moving glacier," STAT's Rachel Cohrs reported in March 2022. Today 15 principal investigators from a University of Arizona Health Sciences consortium, all of whom lead RECOVER enrollment sites, are issuing a call to action to fill current gaps in helping people with long Covid by creating treatment centers. That could mean:

Converting RECOVER recruitment centers into treatment centers
Obtaining $37.5 million in fiscal 2024 spending to build a network of centers

For patients, that would translate into coordinating clinical care and rehabilitation, reducing healthcare disparities, and addressing complex medical and psychosocial needs while focusing on people who now receive fragmented or no care.

Closer Look

ChatGPT in medicine misses the mark in early tests. Here's why

Photo illusration by Leon Neal/Getty Images

ChatGPT is a pretty good student. The AI tool got more than 80% of questions on a board exam right, something even the best students need years to achieve. But moving into the real world of medicine was much different, a Stanford study found. Almost 60% of ChatGPT's answers either disagreed with human specialists or offered information that wasn't clearly relevant. That's not surprising given specialists' reliance on medical records and ChatGPT's internet-sourced knowledge.

That highlights a bigger problem: The early testing of the model examined only its textbook knowledge, not its ability to help doctors make faster, better decisions in real-life situations, particularly messier ones when information is incomplete. "We still need to figure out what the evidence bar is to decide where they are useful and where they are not," said Philip Payne of Washington University in St. Louis. STAT's Casey Ross explains.

insurance
Medicare Advantage insurers get more time

Health insurance companies got a big win Friday. After the companies' intense lobbying, the Biden administration said it would phase in changes to Medicare Advantage's risk adjustment program over three years, rather than deploying them all at once. What's at stake are the higher payments insurers get from the government by coding more illnesses. Medicare officials say insurers sometimes exploit the system by saying that their members are sicker than they are.

To fix that problem, Medicare is removing or consolidating codes for certain conditions that lead to inflated payments. Insurers said the administration's initial proposal amounted to slashing Medicare benefits, but CMS Administrator Chiquita Brooks-LaSure disagreed. "Any reductions in benefits would be decisions made by companies, not because of [Medicare] policies." STAT's John Wilkerson has more.

PHARMA
FDA cites multiple failures at eye-drop manufacturer linked to serious infections

The reports in February were horrifying: Drug-resistant infections were being traced to over-the-counter eye drops that caused serious illness from the bacteria Pseudomonas aeruginosa, eventually including systemic infections, permanent vision loss, and three deaths. Now, STAT's Ed Silverman writes in an exclusive story, an FDA inspection report says the manufacturer of the eye drops failed to follow numerous procedures to make sure its products did not become contaminated.

During a recent visit to Global Pharma Healthcare in Tamil Nadu, India, FDA inspectors found the company failed to take basic steps during sterilization, relied on deficient manufacturing processes and methods to ensure and verify sterility, and did not have adequate systems to create aseptic conditions, among other issues, the inspection report noted. The case follows the deaths of dozens of children linked to cough syrup made in India, renewing long-standing concerns about the Indian pharmaceutical industry. Read more.

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