Gene Therapy, NFL's CTE Risks, and Personalized Cancer Vaccines

Gene therapy is any effort to alter the roughly 20,000 protein-encoding genes in the human body to improve its function and produce the proteins it needs. Single-gene mutations can be harmless, but they may also result in serious or life-threatening conditions, including cystic fibrosis, Tay-Sachs disease, and muscular dystrophies.

> Deaf kids and adults are hearing for the first time with a novel gene therapy. (More)
> Prenatal gene therapy is eliciting interest with a planned clinical trial. (More)

Gene therapies aim to correct errors in variant genes by replacing them with healthy copies that were created in the lab. These genes can be delivered into the body using a virus emptied of its disease-causing instructions and instead carrying functional genes. Inside the body, the virus does what it typically does—invades the patient's cells—but instead of making someone sick, it delivers healthy genes so the body has the instructions it needs to produce the proteins it needs, shut off protein production, or perform other necessary actions.

> See how viruses act as delivery vehicles for the new DNA. (More)

> Gene therapy can also be conducted outside the body. (More)

> There's been some recent progress in animals regarding a rare genetic disorder that can cause people to become deaf and blind. (More)

Gene-editing technologies like CRISPR have enabled breakthrough treatment options in recent years. In the US, there are currently approved gene therapies for conditions including sickle cell disease, Duchenne muscular dystrophy, leukemia and lymphoma, inherited retinal and hearing loss disorders, and more.

> Explore this list of more than 12 FDA-approved gene therapies.

> The FDA has recently proposed a way to speed reviews for gene therapy approvals without lengthy clinical trials. (More)

> Retinal cells have recently been regenerated in the lab with gene therapy. (More)

Discover more: 

> Elizabeth Taylor had two rows of eyelashes due to a gene mutation. (More)

> Base editing might be CRISPR 2.0. (More)

> On an emergency basis, a baby with a toxic ammonia buildup disorder was successfully treated with a bespoke gene therapy. (More)
>... explore everything we've learned about Gene Therapy.

In partnership with Earth Echo

Blue Zone Costa Ricans' Chocolate Breakfast

Most of us file breakfast sweets under guilty pleasure. So, it's a little disarming that Costa Ricans in one famous blue zone drink cacao every morning—and researchers keep studying why they stay so lean and energetic into their 70s, 80s, and 90s.

The short version: this morning ritual is linked to steadier blood sugar and healthy insulin sensitivity, normal blood pressure, and hearts and arteries that stay strong with strikingly little heart disease. Men in the region are seven times more likely to become centenarians than the rest of Costa Rica.

Turns out "healthy" and "delicious" don't have to be at odds. Discover Costa Rica's blue zone breakfast secret.

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 News in Context 

1440 brings you the knowledge and context behind recent intriguing stories:

FDA approved a drug to treat the root cause of narcolepsy this month, and researchers are exploring if this type of medication can be repurposed for other disorders such as attention deficit hyperactivity disorder. (More, free w/email)

> Narcolepsy can cause hallucinations.

> Sleep deprivation leads to the buildup of dangerous molecules in the gut.

At least 1 in 4 NFL players end up with CTE, according to brain examinations of 878 players who died between 2016 and 2021; at least 215 of their brains showed hallmarks of the neurodegenerative condition. (More, free/email)

> National Hockey League commissioner denies CTE risk for the sport. 
> Learn more about CTE, which is linked to dementia and behavioral issues. 

People on GLP-1s are sometimes injecting "zombie filler"—fat from corpses. (More)

> After stopping GLP-1s, people gain two-thirds of the weight back. 

> Medicare began covering GLP-1s in some instances last month.

Traditional scalpel-based cataract surgery is often being replaced by laser surgery, even though the former is cheaper and outcomes are just as good. (More)

> Globally, 1 in 2 people who need cataract surgery don't have access to it.

> Cataract surgery with lasers or scalpels often employs sound waves to break up the cloudy lens.

"Danny Go!," an upbeat children's program often used in schools to promote movement and dance, dropped a second season on Netflix Aug. 24. (More)

> "Danny Go's" star and his wife are mourning the recent death of their teenage son and supporting other parents experiencing grief.

A robot that uses ultrasound and infrared light is now authorized to perform patients' blood draws. (More)

> Why don't bears form blood clots when they hibernate?

 Medical Developments Spotlight 

We love spending time learning about the latest medical breakthroughs and spotlighting research that piques our interest, may influence future research directions, or inform healthcare conversations. Here's what we found this week.

The fetal 'DNA fragmentome' may one day provide a first-trimester snapshot of likely pregnancy complications and improve triage care.

Science Translational Medicine | Kate E. Stanley, Lore Lannoo, et al. In a new study, scientists examined cell-free DNA fragments floating in a mother's blood, a concept formally called the DNA fragmentome, seeking signs of potential pregnancy complications due to infection, pregnancy with twins, and immune-mediated diseases. Researchers retrospectively examined first-trimester blood samples from almost 2,000 pregnant women at high risk of such complications and correctly found several high-risk complications more than half the time, suggesting this approach should be studied further. (More, paywall may apply) 

> The first newborn disease screening was developed 65 years ago.

Childhood cancer predisposition may be detectable with a genomic newborn screening test.

MedPage Today | Mike Bassett. Researchers are calling for universal newborn cancer screening using routinely gathered blood samples after they accurately detected cancer-associated mutations in about 7% of nearly 2,000 children who developed cancer by age 8. The researchers scanned the blood for a panel of 11 genes linked with cancer or likely cancer. (More, free w/email)

> The earliest known cancer was discovered in a toe of a 1.7-million-year-old fossil.

Personalized mRNA vaccines, combined with immunotherapy, slowed melanoma's spread and recurrence, according to a late-stage randomized trial.

Reuters | Michele Gershberg. Merck and Moderna announced that a personalized skin cancer vaccine, when combined with the immunotherapy drug Keytruda, showed a "clinically meaningful" benefit compared with a placebo treatment. That's based on a randomized trial involving 1,137 participants. The companies have been working on this approach since 2016. The personalized vaccine, called Intismeran, teaches a patient's immune system to recognize targets specific to the patient's tumor. Still unknown: whether this approach extends overall survival as well as specific details of the trial. (More, w/video)

> mRNA vaccines alter immune responses—not DNA.

In partnership with Earth Echo

This "Hot Chocolate" May Support Stem Cells

Is chocolate the secret to a long life? Earth Echo put "healthy chocolate" claims to the test. Turns out, sun-dried ceremonial-grade cacao holds onto far more of its natural heart-healthy polyphenols than the highly processed cocoa powder in most supermarket chocolate or cocoa.

See why chocolate lovers are switching to ceremonial-grade cacao. In one 30-day study, high-flavanol cacao roughly doubled circulating stem cells, curbed sweet cravings, and gave smooth energy—no jitters, no crash—so your morning cup feels like a win. See why chocolate lovers are switching.

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 Etcetera 

> GLP-1 use may hinder the effectiveness of oral birth control.

> A rare measles complication can kill seemingly recovered patients years later.

> See how Tommy John surgery, repairing a torn tendon, can save a pitcher's arm.

> Some recent research suggests bedbugs may be able to transmit MRSA.

> A physician is using AI to try to match the roughly 3K FDA-approved drugs with some 9K diseases without therapeutic options to see if they can be repurposed.

> There are two malaria vaccines available, but they don't work well enough to be recommended for most people.

> Cow's milk transfusions were tried out before modern blood transfusions.
 

> There are viruses that infect bacteria, and they're super common.

> Humans could survive without a bunch of their organs, but not the liver.
 

> Turophobia is the fear of cheese.

> Goldfish are supertasters.

> There were 174 reported cases of leprosy in the US last year.