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2019-10-14 16:34
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1- Gene therapies to cure diseases got the FDA green light

For the past 30 years, gene therapy — which involves modifying a person’s DNA to treat or prevent disease — has been one of the most promising areas of medicine. Think about it: Instead of having to take a drug every day to manage an illness for the rest of your life, you could have your genes altered to permanently fix the problem.

But no gene therapy treatments have ever been brought to the US market — until this year.

In a first, the Food and Drug Administration approved several gene treatments for market. There was Luxturna, for adults and children with an inherited form of vision loss that can result in blindness; Yescarta, to treat adult patients with certain types of large B-cell lymphoma; and Kymriah, for children and young adult patients with a form of acute lymphoblastic leukemia.

“2017 is absolutely a watershed for the whole concept of gene therapy,” said Stephan Grupp, who directs the cancer immunotherapy program at the Children’s Hospital of Philadelphia. (Grupp’s program was behind two of the new therapies.)

There are other promising gene therapies on the horizon. In a British trial of 13 patients with hemophilia, a genetic disease in which people lack the protein needed to stop bleeding, a gene therapy treatment helped 11 of the patients reverse their disease. In the same issue of the New England Journal of Medicine, researchers published an article on another successful gene therapy for hemophilia.

“You could read articles 20 and 30 years ago about the promise of gene therapy, but there was no real delivery on that promise until we could work out the practical problems of delivering a gene therapy in a living human being instead of a test tube,” Grupp said. “The field is really gathering amazing momentum.”

2- CRISPR became an even more precise tool to fix nature’s “mistakes”

Scientists made several crucial advances this year in rewriting the genetic code using CRISPR/Cas9 — a tool made of protein and RNA that can be customized to efficiently edit DNA.

For years, hype has been building around CRISPR/Cas9’s tremendous potential — to permanently cure genetic diseases like cystic fibrosis and help the body fight infections, as well as create better biofuels and more resilient crops. The technology works by borrowing a system bacteria use to fight viruses and programming it to snip targeted sections of the genome and swap in a replacement.

But a big hurdle was the concern that getting an edit wrong could create a problem that doesn’t just harm an individual organism but ripples through generations of progeny. In October, researchers at Harvard University and MIT presented two approaches to get around this.

The first technique gets at the problem of permanence. Rather than modifying DNA, researchers engineered a version of CRISPR that targets RNA, a sister molecule to DNA. While DNA is the definitive record and instruction set for making a bacterium, plant, or animal, RNA serves as a go-between, a transcript of individual genetic recipes that is then translated into the language of proteins. Changing RNA alone ensures that modifications are transient.

The other approach refines the precision and accuracy of the CRISPR/Cas9 technology, allowing the complex to target and modify a single base pair out of the 3 billion base pairs that form the human genome. The majority of human genetic diseases are caused by a mutation in a single base pair, so this technique could one day be used to precisely and permanently fix the genome without introducing any new mistakes.

In August, scientists also reported they’d used CRISPR to fix a mutation that causes a heart disorder in human embryos. The conclusions of that study have since been called into question, but the research undoubtedly nudged the field forward.

And finally, this month scientists were able to reduce the severity of genetic deafness in mice. And a private company, CRISPR Therapeutics, announced it will begin clinical trials on a CRISPR-based therapy for the blood disorders beta thalassemia and sickle cell anemia.

3- The WHO stopped a frightening plague outbreak

Of all the public health stories we’ve covered in the past few years, the plague epidemic that was brewing in Madagascar frightened us most. Starting last summer, plague infected some 2,300 people in the capital and other cities, and killed 207. Making things worse, the type of bacteria that spread was the highly contagious pneumonic plague, a rare and more dangerous form of the disease that attacks the lungs and passes from person to person through droplets from coughing. Untreated pneumonic plague is always deadly; patients typically die within 24 hours of disease onset — unless they’re lucky enough to get antibiotics.

But by the end of November, Madagascar’s situation had radically improved. And that was largely thanks to health officials who responded to the outbreak by acting on lessons learned from the tragic 2013–’16 Ebola epidemic in West Africa.

As soon as it became clear that Madagascar’s plague season was unusually forceful this year, the World Health Organization delivered 1.2 million doses of free antibiotics to treat those infected and prevent the disease in people who might have been exposed to the bacteria. The United Nations agency also supported the Ministry of Public Health of Madagascar in a response that included strategies like training 4,400 “contact tracers,” who followed up with people who might have been exposed to someone infected with plague, and boosting the epidemiological surveillance in all of Madagascar’s affected districts so that they could more quickly identify cases and stop plague from spreading.

The WHO had been widely criticized for responding to the recent Ebola epidemic too slowly. But the agency seems to have taken the criticism to heart and changed course. This bodes well for the inevitable public health emergencies of the future.

4- Scientists got closer to finding another planet that can support life

Way back in February, NASA and the European Southern Observatory announced that they had discovered seven(!) Earth-size planets orbiting a small star 40 light-years away.

Three of the planets are directly in the star’s habitable zone, meaning water could mostly likely exist on the surface of them.

It’s uncertain whether these planets could harbor life; their close proximity to the star — a red dwarf — may have left them incapable of forming atmosphere. Regardless, the discovery was another success in a continuing hopeful trend.

In recent years, scientists have overseen an explosion in the number of exoplanets — planets beyond our solar system — discovered throughout the galaxy. And we’re going to keep finding more, especially as astronomers turn to artificial intelligence to automate the search.

It seems wherever we look, we find exoplanets, many in the sweet spot known as the “habitable zone,” in which temperatures may be suitable to have liquid water. There’s even an exoplanet orbiting with a slight potential to be hospitable around the next nearest star. And in November, astronomers announced they discovered Ross 128 b, an exoplanet just 11 light-years away (we’re practically neighbors, in cosmic terms) that may even be a better candidate for finding life. Its star, also a red dwarf doesn’t seem to flare as much atmosphere-stripping radiation as some other stars in its class. Ross 128 b is also likely to be rocky, and is the right distance from its star to have a temperature suitable for liquid water.

Many scientists believe it’s a question of when — and not if — we will find an exoplanet that looks extremely likely to harbor life.

The James Webb Space Telescope, set to launch in 2019, will be able to measure the chemical composition of exoplanet atmospheres. If the atmospheres contain telltale gases like ozone, oxygen, or methane, life could exist there.

The more Earth-like exoplanets astronomers find in the galaxy, the more they update their estimates of how many Earth-like planets could be out there. And the more exoplanets there are, the more likely it is that life exists on at least one of them.

And if life on Earth is looking dreary, just look up and think: Something new, unknown, possibly frightening, and exciting must be up there. That’s hope.

5- Bariatric surgery was shown to be an effective treatment for obesity

Weight loss surgery — in particular the gastric sleeve and bypass operations — is far and away medicine’s best treatment for severe obesity. But it’s still far from a cultural norm. Only 1 percent of Americans who are eligible get surgery, and surveys show a third still think it’s dangerous or ineffective.

Doctors have been hesitant to prescribe surgery for severely obese patients, in part because of stigma around the surgeries, and because there’s been a shortage of long-term data about the effects of the current bariatric surgical methods.

But over the past year, a number of long-term studies — in both adults and teens — have come out, and it’s looking like the only truly effective treatment for obesity is getting its due.

In one of the best long-term studies on the effects of bariatric surgeries, published in JAMA Surgery in December, researchers followed more than 1,000 adults for up to seven years and found that bypass patients lost 28 percent of their original bodyweight and kept it off.

The results of bariatric surgery have proven to be similarly striking in teens. In a 2017 Lancet study, following 58 American teens for eight years who underwent gastric bypass, the teens lost an average of 30 percent of their bodyweight. A study out of Sweden this year tracked 81 teenagers with obesity who had a gastric bypass for five years. They lost about 28 percent of their original bodyweight. That’s far better than the 5 percent of trimming that people achieve, on average, with eight years of intensive diet and exercise programs, according to one of the best long-term studies of weight loss.

In all these papers, surgery also helped reverse or prevent people’s obesity-related health conditions, like diabetes, high blood pressure, or high cholesterol. That’s why doctors are increasingly recommending the surgery for young people, in whom obesity-related health problems are more easily prevented and reversed.

To be sure, surgery is no magic bullet; it comes with uncertainties and potential side effects. But it’s far better than the standard weight loss prescriptions on offer, and given the science, 2017 may be the year the public starts to see bariatric surgery for what it is: an effective and underused treatment for severe obesity.

https://www.vox.com/2017-in-review/2017/12/22/16769574/best-science-stories-news-2017

 

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