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Incredible medical breakthroughs of 2022, from treating blood cancer to slowing Alzheimer's disease

Dec 23, 2022, 16:02 IST
Insider
Alyssa was the first patient with an aggressive blood cancer to receive white blood cells from a donor that scientists genetically modified with a new technology. She is pictured on the day she received the cells in May 2022.Great Ormond Street Hospital for Children
  • Scientists made breakthroughs in 2022 that may pave the way for new drugs and vaccines in 2023.
  • From a RSV vaccine to an infusion for Alzheimer's, scientists laid the groundwork for future success.
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In 2022, scientists made breakthroughs that pave the way for new drugs and vaccines for serious medical conditions with few treatments.

From a vaccine for respiratory syncytial virus (RSV) to an infusion that slowed down Alzheimer's for some people with the disease, here are three momentous advances from 2022.

An RSV vaccine showed promise for the first time in 50-years

Two vaccines are poised to be approved for RSV by the end of 2023, according to their makers, after almost 50-years without any meaningful progress.

RSV typically causes mild, flu-like symptoms that get better within weeks without treatment, according to the Centers for Disease Control and Prevention.

However, it can be serious, especially for infants and older people, with the potential to cause pneumonia and inflammation of the small airways, called bronchiolitis, which can lead to respiratory failure and death. More than a hundred babies and 10,000 adults over the age of 65 in the US die of RSV every year, the CDC states. As Insider reported, RSV hit the US earlier than usual this year, with toddlers and school-age kids getting sick too.

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There is no approved vaccine or treatment for RSV. In fact, in the late 1960s, a RSV vaccine made symptoms worse in kids and babies.

The two new vaccines, which are for use in pregnant people and elderly adults, respectively, target a part of the virus called the fusion protein that attaches to cells before it infects them. The vaccines that made the illness worse targeted it afterwards.

GSK said its vaccine for people older than 60 was about 94% effective at preventing severe disease in a trial of approximately 25,000 people.

Pfizer said its vaccine for pregnant people, who pass on antibodies to their child, was about 69% effective at preventing severe RSV in newborns during the first six months of life in a trial of approximately 7,400 people.

Dr. Wilbur Chen, an infectious disease expert at the University of Maryland School of Medicine who researches vaccines and is an independent advisor to the CDC, previously told Insider that if the vaccines are approved for use in the US "we're going to see a significant decrease over time for RSV hospitalizations and deaths."

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A woman gets her COVID vaccine. A new Pfizer vaccine for pregnant people protected their babies from RSV, in a clinical trial.Jack Guez/AFP via Getty Images

Scientists treated an aggressive blood cancer with gene-editing

In a world-first, scientists treated an aggressive blood cancer with gene-editing technology that was invented just six years ago.

The 13-year-old patient named Alyssa was diagnosed with T-cell acute lymphoblastic leukemia in May 2021, after what her family thought was a period of colds, viruses, and general tiredness.

T-ALL causes the body to make too many white blood cells, such as T-cells, which don't work as well as normal T cells against infections. Doctors initially treated Alyssa with chemotherapy and a bone marrow transplant, but her disease came back.

During a clinical trial in May, researchers at Great Ormond Street Hospital, UK, treated Alyssa with T-cells from a healthy donor that were genetically altered, before giving her another bone marrow transplant.

Our DNA, or genetic code, is composed of single letters called bases. Researchers altered the donor T-cells by chemically converting single letters, called base-editing.

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The scientists adjusted the code so Alyssa's immune system, and other cancer treatments, could no longer attack the donor T-cells, and the modified T-cells could destroy cancerous T-cells, but not themselves.

According to GOSH, Alyssa's disease was in remission 28 days after the experimental treatment.

Dr. Robert Chiesa, a consultant in bone marrow transplant and CAR T-cell therapy at GOSH, said in a statement: "This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months."

Alyssa relaxing at home with her dog, Holly, after the treatment.Family's own

An Alzheimer's drug slowed down the disease

An experimental drug slowed down the progression of Alzheimer's disease in a study that ended decades of research failures, and which experts hope suggests that new drugs to treat Alzheimer's — the most common form of dementia — are possible.

Current treatments for Alzheimer's can help to control symptoms, but can't reverse or cure the disease.

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In the study, cognitive decline slowed by around a quarter over the 18 month treatment period for 898 people aged between 50 and 90 who received the drug, compared to 897 people who researchers gave a dummy drug.

The drug called lecanemab is a monoclonal antibody — a lab-made version of proteins the body makes to attack viruses or bacteria — that has been engineered to tell the immune system to clear amyloid protein from the brain.

As Insider's Yeji Jesse Lee reported, the controversial amyloid hypothesis that the accumulation of beta-amyloid, or proteins also called Aβ clumps, in the brain causes Alzheimer's underpins the drug. These clumps build up in the brain of people with Alzheimer's and eventually kill brain cells, according to the hypothesis.

John Hardy, a world-leading neurogeneticist at UCL, UK, who uncovered the mutation that lead to the amyloid hypothesis around 30 years ago, told BBC News that the findings were "historic" and he was optimistic "we're seeing the beginning of Alzheimer's therapies."

The infusion, which is given fortnightly, may slow disease progression but it isn't a miracle cure and taking it comes with risks.

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Overall, almost 7% of people given the drug had to stop because of side effects, and we don't know its effects beyond 18 months.

The FDA is assessing data to decide whether lecanemab to approve for wider use next year.

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