Pig kidney transplants, new schizophrenia drugs: Here are five of the biggest medical advances of 2024

From pig kidney transplants to reversing genetic hearing loss, 2024 was a year filled with medical advances.

This breakthrough includes the discovery of the causes of autoimmune diseases, the development of “game-changing” drugs, and potential hope for people experiencing end-stage organ failure.

Here are five of the biggest highlights of this year's medical achievements.

Gene therapy restores hearing in children

Children with inherited hearing loss regain hearing thanks to a type of gene therapy, according to results of a clinical trial published in a medical journal. LanceIn January.

Mass Eye and Ear InvestigatorA specialist hospital in Boston tested six children with a type of hereditary hearing loss called DFNB9, which is caused by a genetic mutation that prevents the transmission of sound signals from the ear to the brain.

The gene therapy involved an inactivated virus with a functioning gene that was introduced into the inner ears of six children.

After 26 weeks, five of the six children had regained their hearing and were even able to have “normal conversations.”

“For children with this hereditary hearing loss…the only treatment for them right now is [a] It's a cochlear implant,” Dr. Chen Yi Chen, associate scientist and study co-investigator at Mass Eye & Ear's Eaton Peabody Institute, told ABC News. [a] Cochlear implants can help them a lot, but they have their own limitations. ”

“But with this gene therapy, these children regained their hearing and were able to speak. So, in a sense, their lives were completely changed,” he continued. “This research really opens up a whole field and will allow us to develop treatments for other diseases in the future.” [types] It is a genetic hearing loss and there is currently no cure. ”

Groundbreaking animal organ transplant

Surgeons at Massachusetts General Hospital (MGH) performed the world's first gene-edited pig kidney transplant into a living human in March 2024.

inside 4 hour procedureThe surgical team connected the blood vessels and ureters of the pig's kidneys (the tubes that carry urine from the kidneys to the bladder) to the blood vessels and ureters of Richard Suleiman, 62, a man living with end-stage renal disease.

“We know that transplantation is the best treatment option for patients with kidney failure, but unfortunately we are facing a huge organ shortage,” said Leonard, Medical Director of Kidney Transplantation at MGH. Dr. Riera told ABC News. “That means there are more than 100,000 people waiting for a kidney transplant in the United States, and more than 17 people die every day on the waiting list.”

“So the idea here is, how can we overcome this organ shortage barrier? And having another species of kidney that can be donated in a timely manner to patients who develop kidney failure is an important part of this field. “It could be a game-changer for the whole thing,” he added.

Suleiman died in May this year, but there is no evidence that his death was the result of a transplant. MGH.

Through Suleiman's treatment, Riera is learning more about how best to treat animal organs when used for transplants, in hopes of making the treatment more widely available to patients waiting for a new organ. He said he learned.

The cause of lupus revealed

Brigham's team and Women's Hospital and Northwestern Medicine announced they have discovered the cause of the autoimmune disease lupus and a potential way to reverse it.

Lupus is thought to be caused by the body's immune system mistakenly attacking its own healthy cells and tissues, which can cause inflammation and damage to organs and systems. Centers for Disease Control and Prevention.

In a study published in a magazine nature In July, researchers compared blood samples from 19 patients with lupus to 19 patients with lupus and found that there was an imbalance in the types of T cells that lupus patients produced.

T cells are a specific type of white blood cell that play an important role in the body's immune response to disease.

“We identified a fundamental imbalance in the immune response that occurs in lupus patients and defined specific mediators that can correct this imbalance and attenuate the pathological autoimmune response,” said co-author and rheumatologist. said Dr. Deepak Rao. said Co-Director of the Brigham and Women's Hospital and Cell Profiling Center. press release at that time.

First new class of schizophrenia treatment in more than 30 years

In September, the FDA First new class of drug to treat patients with schizophrenia More than 30 years later.

Manufactured by Bristol-Myers Squibb, the pill, called Cobenfy, combines two drugs, xanomeline and trospium chloride, and is taken twice a day.

Clinical trials have shown that this combination helps manage symptoms of schizophrenia, such as hallucinations, delusions, and disorganized thinking.

Dr. Rene Kahn, chair of the department of psychiatry at the Icahn School of Medicine at Mount Sinai, said the first schizophrenia drug took years to develop, but the drug is effective in preventing psychosis and increases dopamine production. He said it works by blocking receptors.

“Blocking dopamine receptors directly or indirectly can be very unpleasant. For patients, it can sometimes lead to unpleasant side effects, such as low energy or feeling depressed. , which can cause side effects of Parkinson's disease,” Khan told ABC News. .

“This is a game-changer in the sense that it is the first drug that does not directly, particularly directly, affect the dopamine system and does not reliably block dopamine receptors,” he said of Cobenfi. This is very important. We show that we don't need to block or influence the dopamine system directly, but can do it through another mechanism.”

Khan said the next step is to monitor the drug, which has been prescribed to thousands of schizophrenia patients, to ensure it is effective and has minimal side effects.

First over-the-counter combined influenza and coronavirus test for non-emergency use

In October, the FDA authorized the first over-the-counter combination coronavirus and influenza test for non-emergency use.

Helgen Quick Check COVID-19/Influenza AThe &B antigen test can be purchased at pharmacies without a prescription.

Although there are other over-the-counter combination tests currently available, this is the first to be sold to consumers through traditional approval channels outside of a public health emergency, the FDA said.