An anonymous reader quotes a report from NPR: The Food and Drug Administration approved the first gene therapy to restore hearing for people who were born deaf. The decision, while only immediately affecting people born with a very rare form of genetic deafness, is being hailed as a milestone in the quest to treat hearing loss. "It's the first time in history there's a new drug for hearing loss," says Zheng-Yi Chen, an associate scientist at Mass Eye and Ear in Boston who was not involved in the development of the therapy approved by the FDA Thursday. But his research team reported very promising results with a similar approach Wednesday. "I think it's an historical event, a landmark, a great development for the whole field," he says of the approval. [...] The FDA's decision was based on the results from the treatment of 20 patients born with a defective version of a gene known as OTOF, which is necessary to transmit sound from the ears to the brain. Doctors infused billions of adeno-associated viruses into the patients' ears by making a small incision behind the ear to open a small hole in the skull. The viruses carried a healthy version of the OTOF gene that had been split in half to fit inside the virus. The gene provides instructions to make the otoferlin protein, which is necessary for hair cells in the inner ear to transmit sound to the brain. Most of the patients began to hear for the first time within weeks, with the quality of their hearing improving over the following months, according to [Regeneron Pharmaceuticals, which developed the gene therapy and plans to offer it for free in the U.S. It should be available within weeks.]. The amount of hearing patients gained varied, but 80% achieved at least some significant hearing restoration and 42% ended up with normal hearing, which included the ability to hear whispers, Regeneron says. The hearing ability has lasted at least two years so far. The treatment can only help patients with the very rare form of deafness that Smith was born with, which only affects about 50 children each year in the U.S. But similar gene therapies are showing promise for other forms of genetic deafness. And researchers hope someday gene therapy may help with common types of hearing loss, like from aging and loud noise.

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A 63-year-old man in Norway appears to be cured of HIV after receiving a stem cell transplant from his brother, who turned out to have a rare mutation that makes immune cells resistant to HIV. "Four years after the transplant, and two years after the man stopped antiretroviral therapy, he still appears to be free of the infection," reports Gizmodo. From the report: According to the report, the man was first diagnosed with myelodysplastic syndrome, a type of cancer that weakens blood cell production from bone marrow, in 2018. Though he seemed to initially respond to treatment, the cancer returned after two years, and doctors decided to perform a stem cell transplant. Because the man also had HIV (diagnosed in 2006), the doctors were hoping to treat both conditions at once, though they knew their chances were low. Most of these cases have involved the use of stem cells taken from people with two copies of a particular mutation in their CCR5 gene, which regulates the CC5R receptor on white blood cells. This mutation, named CCR5-delta 32, makes immune cells naturally resistant to infection from strains of HIV-1 (the most common type of the virus). However, only about 1% of the population carries two copies of the mutation. After initial screening failed to find someone who both possessed the mutation and had compatible bone marrow, the doctors decided to move ahead with the man's brother, who was already known to have compatible bone marrow. But to everyone's surprise, testing on the day of the transplant showed that the brother also had the mutation. Though the man did experience some complications from the procedure, his body successfully started to produce new blood cells with the mutation. The doctors decided to take him off antiretroviral medication two years after the transplant. And in the two years since then, regular follow-up tests have failed to show any signs of the virus in his system. [...] According to AFP, there have only been roughly 10 cases worldwide involving an HIV cure through stem cell transplantation. This is the first to involve a family donor.

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