2023-12-07 21:21:06
BarcelonaDespite the impossibility of predicting exactly what the scientific community can offer as a shocker for the health of the population, the magazine Nature Medicine has asked a dozen renowned researchers to name which of the clinical trials they lead may have the greatest impact on medicine in 2024. The proposalsall of them promising, range from genetic editing for the treatment of chronic diseases or cancer to the development of artificial intelligence (AI) tools to diagnose pathologies and optimize healthcare resources.
Boston Brigham and Women’s Hospital cardiologist and Harvard Medical School professor Amit Khera is also the vice president of Verve Therapeutics, a biotech that wants to develop a drug to permanently control familial hypercholesterolemia. This inherited genetic condition is very common – affecting about one in 300 newborns – and is caused by a defect in chromosome 19. In these cases, the body is unable to eliminate low-density lipoprotein (LDL), known as as bad cholesterol, and when its concentration in the blood is very high, it accumulates in the arteries and clogs them, which increases the risk of cardiovascular disease.
“Although statins [els fàrmacs contra el colesterol] reduce the risk, most patients do not have optimal levels of bad cholesterol,” says Khera. The researcher is working on a clinical trial to demonstrate that genetic editing of the mutation that triggers this condition, in the PCSK9 gene, could control better long-term hypercholesterolemia.
The trial is called Heart-1 and seeks to apply, for the first time in humans, base editing, a type of genome editing that would allow changing any of the letters in individual DNA without breaking the In this case, the PCSK9 gene in the liver. The drug is called Verve-101 and the provisional results were presented last November at the scientific sessions of the American Heart Association, and they show a reduction in the levels of bad cholesterol in the blood by around 50%.
David Baldwin is a pulmonologist at Nottingham University Hospital and his proposal to Nature is to use AI as a tool to diagnose lung cancer early. Almost three out of four cases are identified in late stages, and the researcher suggests that taking a chest X-ray, followed immediately by a computed tomography (CT) scan, could advance detection.
The trial is being conducted on 150,000 patients from six British hospitals and aims to show that applying AI reduces the time to do a CT scan from 63 to 32 days. The team plans to finish recruiting in July 2024. The idea is to show that cutting the waiting time between tests in half allows cancer to be detected earlier. “If our trial finds a significant improvement, it will likely lead to an immediate change in the standard of care,” says Baldwin.
The list of essays also includes the choice of Carlijn M. van der Aalst, assistant professor in the Department of Public Health and the Erasmus Medical Center in Rotterdam. In this case we want to see if screening every two years for lung cancer with CT is as effective in preventing deaths as annual tests for those who do not have abnormalities in their first scan.
The head of clinical research at Vir Biotechnology in San Francisco (USA), infectologist Carey Hwang, argues that his lymphocyte vaccine VIR-1388 has the potential to prevent human immunodeficiency virus (HIV) infection. The phase 1 study includes adults aged 18 to 55, and the vaccine is based on cytomegalovirus vectors, a herpes virus that induces “strong, unique and sustained” defense responses that could prevent acquisition of HIV
University of Liverpool child psychiatrist Atif Rahman puts the spotlight on mental health care for pregnant women. Specifically, in a mobile application that facilitates interaction between women who do not have any kind of psychological training so that they do cognitive therapy for pregnant women with major depression during the second or third trimester. “This figure reinforces therapeutic messages and helps solve problems through empathy and mutual support,” and they just have to follow the app’s guidelines.
The study that Rahman proposes is funded by British health, through the Research and Innovation for Global Health Transformation programme, but is inspired by the Thinking Healthy program in rural Pakistan, endorsed by the World Health Organisation. Health (WHO). “We hope that the results of this study will stimulate further innovation and research in this important area that can be a game changer in addressing the treatment gap for a common mental disorder around the world,” adds the psychiatrist.
“Most clinical trials for Parkinson’s disease are done with patients with advanced disease. As a result, those in a much earlier stage of the disease, who could benefit most from treatment, are often excluded “, poses the head of the research team in neurobiology of development and regeneration at the Swedish University of Lund, Malin Parmar.
The professor at the New York Stem Cell Foundation proposes, for this 2024, to have the STEM-PD trial on the radar, from which neurons derived from human embryonic stem cells (called dopaminergic) will be transplanted in the brains of patients aged 50 to 75 years with moderate Parkinson’s. The first patients received the doses last February, and the researcher hopes to have preliminary results by the end of 2024. This is the first time this therapy has been tested in Parkinson’s disease, which has so far been useful for leukemias and lymphomas.
In the emergency services it is essential to make a good triage; that is to say, identify what the patients’ risk is in order to prioritize their care and refer them to the appropriate services. To do this, the bet of the head of the general clinical chemistry and hematology unit of the Central Diagnostic Laboratory in Maastricht (Netherlands), Steven Meex, is to use the already existing clinical risk score Riskindex, which allows predicting 31-day mortality of patients who sought treatment in an emergency department. If the AI model is validated, he posits, it could identify patients at risk of deterioration more accurately than doctors themselves.
The tool was developed and evaluated in four Dutch hospitals, based on 266,327 patients. “The Riskindex outperformed internal medicine specialists, but the extent to which these models have beneficial value when implemented in clinical practice is still unknown,” admits Meex. Recruitment for the study is halfway through, and researchers hope to see results as early as 2024.
Christian Blank is a professor of medical oncology at Leiden University and a researcher at the Netherlands Cancer Institute. His proposal is the use of immunotherapy to fight melanoma. In fact, his team is pioneering the use of checkpoint inhibitors, which make the patient’s own immune system fight cancer. Now they have to confirm, in a phase 3 trial called Nadina, how it works in melanoma.
The study includes 420 patients from Australia, Europe and the US with stage III primary or cutaneous melanoma. In these cases, adjuvant anti-PD-1 therapy (which goes against the proteins that the immune system itself generates and allows some tumors to evade treatments) improves survival, but a considerable proportion of patients still suffer relapses. With this change, they hope to see an overall survival benefit, “which could become a practice change for the treatment of stage III melanoma.”
According to the University of Oxford, children aged 5 to 17 months vaccinated three times with their malaria vaccine, R21/Matrix-M, can get 80% protection if they also receive a booster dose after one year. If this extreme is confirmed, his vaccine would have the highest levels of coverage ever achieved in this disease. This is defended by Adrian Hill, director of The Jenner Institute and professor of vaccinology at Oxford, who emphasizes that only two vaccines have shown useful effectiveness, Oxford’s and Mosquirixi, from GlaxoSmithKline. “But the effectiveness of this drops from 55% to 30% four years after vaccination,” he points out.
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