A team of doctors in Germany and scientists in Italy were able to help a boy from Syria with a genetic disorder that left him with untreatable wounds covering 80 percent of his body.
The 7-year-old fled with his family from Syria to Germany in 2013, and by the time he started to receive treatment at Ruhr University Bochum, he was running out of time. He has a disease called junctional epidermolysis bullosa, caused by a mutation of the LAMB3 gene, which produces the protein that makes the top layer of skin connect to deeper layers underneath. The condition made his skin fragile and quick to blister, and his epidermis was still intact only on his head and a patch of his left leg. With all options exhausted, doctors reached out to scientists in Italy, asking if they could grow replacement skin for their young patient.
The scientists had regenerated healthy skin in a lab before, but never for as tall an order as this. They took epidermal cells from an area of his skin that did not have blisters, and genetically modified it in the lab, using a virus to correct the LAMB3 defect. The scientists then grew colonies of cells with corrected genes into sheets of genetically modified skin, and over two months, they grafted the skin to the patient. The grafts grew together and self-renewed, to the delight of the boy's medical team.
Once the epidermis has regenerated, the stem cells take over as in a healthy person, Michele De Luca at the University of Modena told The Guardian. Two years after the surgeries, the boy is doing well, does not take any medication, attends school and plays sports, and when he has a cut, his skin heals. Catherine Garcia
Children whose vision improved following gene therapy, plus their parents, doctors, and scientists, will speak in front of a Food and Drug Administration panel on Thursday, as the committee decides whether it will recommend approving the therapy.
The FDA has until Jan. 18 to decide if it wants to approve Luxturna, which would be the first gene therapy available in the United States for an inherited disease and the first where a corrective gene is directly given to a patient, The Associated Press reports. Luxturna has been tested on people with Leber congenital amaurosis, who are unable to make a protein needed for the retina due to flaws in the RPE65 gene. They typically are only able to see blurred shapes and bright lights, until they lose their sight all together.
A study, funded by Luxturna's manufacturer, Spark Therapeutics, found that while it does not give patients 20/20 vision, it did improve the vision of nearly everyone who participated in the trials. The company hopes patients would only need one treatment, which involves injecting a modified virus with the corrective gene into the retina. It's not known yet how long the benefits last, but it usually only takes about a month for sight to start to improve. Thanks to the therapy, children have been moved from Braille classrooms to sighted classrooms, and adults who have never held jobs before due to limited sight can now work, Dr. Katherine High, president of Sparks Therapeutics, told AP. Catherine Garcia
Put away the masks, creams, cleansers, and serums — a University of California, San Diego, professor says he has developed an acne vaccine that can take care of the disease.
"This is the first vaccine for human beauty," Prof. Eric Huang of the Department of Dermatology told NBC 7. "I think this vaccine has a huge market in the whole world." Huang said acne is caused by an overgrowth of the p. acnes bacteria inside a lesion, and when the bacteria releases a toxin called Christie-Atkins-Munch-Peterson (CAMP) factor, it causes inflammation. The human body can't neutralize this factor on its own, but the vaccine can. "It does not kill the bacteria," he said. "The vaccine neutralizes the bacteria, which everybody has."
After five years of work, there are two types of vaccines — therapeutic and preventative — which will be given to children in elementary school. The vaccine has been tested on mice and worked well, Huang said, and now, he needs to team up with a pharmaceutical company for large-scale clinical trials; if that happens soon, after FDA approval, the vaccine could be available within three to five years. Catherine Garcia
Tesla and SpaceX CEO Elon Musk is setting his sights on something new: "neural lace" technology, which involves implanting tiny electrodes into the brain that could one day help humans function at a higher level.
His new company, Neuralink, will pursue developing these cranial computers, which at first would most likely be used to treat people with brain disorders like epilepsy and major depression. While Musk would not comment to The Wall Street Journal about Neuralink, several people with information about the company said he is actively setting it up and could have a significant leadership role. Musk has said it's important for humanity to not be left behind as advances are made in artificial intelligence. Catherine Garcia
A new study shows that eating a diet rich in staples of a Mediterranean diet — fish, olive oil, nuts, vegetables, and whole grains — may help reduce the risk of developing a type of breast cancer that cannot be treated with hormone therapy.
The study, conducted for the World Cancer Research Fund and published Monday in the International Journal of Cancer, found that the Mediterranean diet might greatly reduce the chances of women getting post-menopausal ER-negative cancer. Piet van den Brandt of Maastricht University in the Netherlands, the study's lead researcher, said this "usually has a worse prognosis than other types of breast cancer," and the research "can help to shine a light on how dietary patterns can affect our cancer risk."
The study followed 62,573 women between 55 and 69 who tracked their diets over two decades, starting in 1986. Over the course of the study, 3,354 women were found to have breast cancer, with 1,033 cases not analyzed because of family histories of breast cancer or incomplete diet data. The researchers concluded, after analyzing individual components of the subjects' diets, that nut intake was most strongly inversely associated with ER-negative breast cancer, The Guardian reports, followed by fruit and fish. They suggested that if everyone ate the highest defined Mediterranean diet — low in red meat, sugar, and white rice and bread — close to 33 percent of ER-negative breast cancer cases and 2.3 percent of all breast cancer cases could be avoided. Catherine Garcia
NASA successfully launched a new generation of weather satellite from Cape Canaveral, Florida, Saturday night, hailing a new era in weather forecasting. "This is a quantum leap," said Sandra Cauffman, deputy director of Earth sciences at NASA. "It will truly revolutionize weather forecasting."
— NASA (@NASA) November 19, 2016
The new GOES-R satellite differs from its predecessors in that it can take a high-resolution image of an entire hemisphere of the Earth every five minutes while simultaneously focusing on specific weather events. Previous satellites took 30 minutes to take the same photo and could not multi-task. Bonnie Kristian
The next time you have a kidney stone, don't go to the doctor or stay home in excruciating pain — head to your local amusement park.
After hearing from patients who said their kidney stones passed without pain after going for a ride on Big Thunder Mountain at Disney World, researchers at Michigan State University decided to conduct a test. Using a 3D printer, they created silicone models and put in kidney stones of various sizes, then took them for a ride. The scientists say even the largest stones were dislodged after two or three rides, and sitting in the back was more effective than being in the front.
Dr. Clayton Lau, a urologist at City of Hope in Duarte, California, told ABC Los Angeles the bumpiness of a roller coaster likely does not create enough turbulence to pass a stone, and the rush of adrenaline probably causes movement in the ureter, helping propel the stones. The researchers say their models show the stones did pass all the way after at least one ride, and suggest that people who are prone to getting kidney stones go for regular rides to keep them at bay. Lau doesn't see thrill ride therapy becoming the next big thing, saying, "I think that's the last thing you want to do when you're in pain is jump on a roller coaster," but ask any patient and even while hurting, they might choose Disneyland over the doctor's office. Catherine Garcia
Scientists use wild ant-eating frogs as tiny scouts who are able to search for insects in places people can't go. Then they capture the frogs and carefully make them vomit up the results of their latest explorations. The frogs are released unharmed.
In this case, the frog puke contained a single (and dead) member of the newly identified ant species, Lenomyrmex hoelldobleri. "Sometimes people think that our world is very well explored. Nothing could be farther from the truth," said Christian Rabeling of the University of Rochester, New York, who led a study on the new ant. He added, "The difficulty is finding the ants!" Bonnie Kristian