Medicine, here are the news for 2024

Medicine, here are the news for 2024

2024 is now upon us, ready to bring a breath of innovation to the world of medicine. As usual, Nature Medicine has decided to draw up a list of innovations to keep an eye on next year, asking 11 experts to talk about the most promising trial they are working on, which could produce results during 2024. And among genetic engineering, impossible vaccines, artificial intelligence at the service of health, the news certainly shouldn’t be missing.

Base editing for familial hypercholesterolemia

The first expert expert consulted by Nature is Amit Khera, vice president of the Genomic Medicine division of Verve Therapeutics, and his choice falls on testing the first gene therapy for heterozygous familial hypercholesterolemia, an inherited disorder that causes high levels of cholesterol in the blood . The disease affects approximately one in three hundred people, and is caused by mutations in a gene, PCSK9, which codes for the production of a protein responsible for modulating the presence of LDL cholesterol (the bad one) in the blood. The disease is almost always asymptomatic, and if it is not treated adequately those who suffer from it risk suffering from heart attacks and serious cardiovascular problems at a relatively young age. Even when diagnosed early, drugs do not always help achieve optimal cholesterol control, and for this reason Verve Therapeutics is working on an in vivo gene therapy that aims to cure the genetic mutation that causes the disease using a innovative genetic engineering technique called base editing.

The phase 1 trial is recruiting patients to test the effectiveness of the treatment. According to preliminary data presented during the annual meeting of the American Heart Association, the therapy appears promising, although for now serious doubts remain about its safety profile. The very high cost of this type of treatment also raises doubts about the cost/effectiveness profile, especially in the case of relatively common and treatable pathologies such as familial hypercholesterolemia. In short, only trials will be able to tell us whether this new gene therapy will ever see the light, and the first results should arrive during 2024.

Ai and early diagnosis of lung cancer

Early diagnosis in oncology is always synonymous with hope. In the case of lung cancer, unfortunately it remains a remote eventuality, because to date almost three quarters of diagnoses arrive when the neoplasm is in the advanced stages, and therefore has less chance of being cured. Even in these cases, however, speeding up the time that passes between imaging tests and diagnosis improves the possibilities of intervention by surgeons and oncologists, and this is what David Baldwin, pulmonologist at Nottingham University Hospitals, hopes to achieve with the the help of artificial intelligence. In a randomized controlled clinical trial, 150,000 patients at six large UK hospitals were assessed using an AI developed to spot signs of lung cancer in images obtained from a chest x-ray. The hypothesis of Baldwin and his collaborators is that this tool could reduce the time needed to go from the first tests to the diagnosis by up to 50%. If the results of the trial are as hoped, Baldwin believes that the use of AI in reading chest x-rays could quickly become the standard of care in this field.

A vaccine against HIV

It’s more or less the holy grail in the field of vaccinia. So elusive that many believe it impossible. Carey Hwang, vice president and director of clinical research at Vir Biotechnology in San Francisco, hopes to change that soon. The company is in fact working on a viral vector vaccine against HIV, developed to act particularly effectively on T lymphocytes, a type of immune system cell that could prove particularly important in preventing the infection. A phase 1 trial will evaluate the safety and immune response induced by the vaccine. The first results should arrive within a couple of years, and if they prove promising they will push us to continue with the more advanced phases of experimentation.

Therapy for perinatal depression

Mental health protection is not guaranteed in the same way all over the world. And it is particularly lacking especially in low- or middle-income countries, where many communities do not have access to the services of psychologists, psychiatrists and psychotherapists. For this reason, Atif Rahman, professor of child neuropsychiatry at the University of Liverpool, thought of developing an app capable of guiding a lay person in the administration of cognitive therapy interventions against perinatal depression. The app is designed to be used by other women in the pregnant woman’s community, so that they can intervene in case of symptoms of major depression during the second and third trimesters of pregnancy. The tool will now be tested in a trial, which will see its effectiveness compared with that of a psychological assistance program provided by community health workers in some rural areas of Pakistan.

Stem cells against Parkinson’s

Malin Parmar is a professor at the Stem Cell Foundation in New York expert in regenerative neurobiology. His most recent research concerns the use of dopaminergic neurons obtained from embryonic stem cells in the treatment of Parkinson’s. Unlike many clinical trials of this type, it is aimed at patients in the early stages of the disease, who are therefore more likely to benefit from the treatment. The first patients were enrolled earlier this year, and have already received a neuron transplant. The first results, therefore, should arrive no later than the end of 2024.

Machine learning and triage

Triage is essential to guarantee the best possible care in emergency rooms, because it allows us to identify patients who need immediate care, and those who can wait a little longer before being examined. There are several algorithms for evaluating the clinical risk score of patients, but few of these have actually been evaluated with clinical trials. Recently, Maastricht University developed a clinical risk score called RISKINDEX, which exploits the potential of AI to predict the 31-day mortality risk of patients attending the emergency room. The algorithm was trained on data from 266,000 Dutch patients, and in simulations it performed better than several internal medicine specialists. It remains to be seen how effective it can be in clinical practice, and this is what the MARS-ED trial, currently underway, will test, the first results of which – according to Steven Meex, of Maastricht University – should arrive over the next year .

Immunotherapy for melanoma

Christian Blank is a professor of medical oncology at Leiden University, and is leading a trial that will evaluate the effectiveness of neoadjuvant therapy with immune checkpoint inhibitors in the treatment of melanoma. The trial is called NADINA, and will recruit 420 patients in Australia, Europe and the United States to compare the effectiveness of a neoadjuvant treatment with ipilimumab and nivolumab, followed by surgery, with that of the current standard of care, i.e. surgery and adjuvant therapy with nivolumab . The results will arrive next year, and according to Blank they could revolutionize clinical practice in the treatment of stage three melanomas.

Malaria vaccine

There are currently only two vaccines that have proven effective against malaria. One of these, RTS,S, has an effectiveness of 55% in preventing infection in the first months after vaccination, which however drops to 30% after just four years. For this reason, the scientific community anxiously awaits the long-term results of the clinical trial testing the other available vaccine, R21. The trial is headed by Adrian Hill, professor of vaccinia at Oxford, and hopes are high because this formulation contains particles with a greater density of antigens, which should make the immune response against the parasite that causes malaria more long-lasting. The preliminary results have already been made available in pre-print, and for the definitive ones all that remains is to wait until next year, hoping that the vaccine will reveal a more satisfactory immunizing capacity than its predecessor.

New drugs against brain metastases

Brain metastases are a problem affecting almost 50% of patients with Her2 positive advanced breast cancer. There is currently only one drug approved by the FDA for this therapeutic indication, but the DESTINY-Breast12 trial could change things. It will evaluate the effectiveness of an antibody-drug conjugate (a treatment that combines a powerful chemotherapy and a monoclonal antibody that precisely ferries it to tumor cells) that targets Her2. And as the director of the breast oncology division of the Dana-Farber Cancer Institute in Boston, Nancy Lin, explains, the results expected for 2024 will also allow us to better clarify the activity of antibody-drug conjugates at the brain level, opening the doors – yes hopes – to many new trials of this type for patients with brain metastases.

Mental health in pediatric age

Children who are entrusted to social services are an extremely fragile category. They have a higher risk than their peers growing up in the family of suffering from mental and physical disorders, of attempting suicide and of remaining unemployed once they reach adulthood. At least in the United Kingdom, where a trial is underway to verify the effectiveness of a new method of care, called New Orleans Intervention Mode, for the care of minors between zero and five years old. The randomized and controlled trial will evaluate the cost and effectiveness of the method by comparing it with that of the traditional approach of English social services. The duration of the study is 2 and a half years, and the results should arrive next year. As Dennis Ougrin, a child psychiatrist at King’s College London, explains, a positive outcome could radically change the care and psychological health of thousands of children in the United Kingdom and around the world.

Lung cancer screening

Lung cancer screening carried out with CT scans allows for a considerable reduction in lung cancer mortality. Several studies have now demonstrated this, but the diffusion of this prevention strategy is still relatively limited. Carlijn van der Aalst, professor of public health at the Erasmus Medical Center in Rotterdam, is working on a trial that will test the effectiveness of screening carried out every two years, versus that of annual screening, involving 26 thousand people from six European nations. If the results are as hoped for, she believes that by simplifying the screening procedure it will be possible to improve its diffusion, to the benefit of patients.

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