BarcelonaAlthough it is impossible to accurately predict what the scientific community might deliver as a shock to population health, the journal Natural medicine Dozens of renowned researchers were asked to identify which clinical trials they lead could have the greatest impact on medicine in 2024. ProposalsThey all range promising, from gene editing to treat chronic diseases or cancer to developing artificial intelligence tools to diagnose diseases and improve healthcare resources.
Amit Khera, a cardiologist at Boston’s Brigham and Women’s Hospital and a Harvard Medical School professor, is also a vice president of Verve Therapeutics, a biotechnology company that wants to develop a drug to permanently control familial hypercholesterolemia. This inherited genetic condition is very common – affecting 1 in 300 newborns – and results from a defect in chromosome 19. In these cases, the body is unable to get rid of low-density lipoprotein (LDL), known as bad cholesterol, and when Its concentration in the blood is very high, as it accumulates in the arteries and clogs them, which increases the risk of cardiovascular disease.
“Despite the statins [els fàrmacs contra el colesterol] “It reduces the risk. Most patients do not have ideal levels of bad cholesterol,” says Khera. The researcher is working on a clinical trial to prove that gene editing of the mutation that causes this condition, in the PCSK9 gene, can better control it in the long term. 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 allows any of the letters in individual DNA to be changed without breaking the DNA. In this case, the PCSK9 gene is located in the liver. The drug is called Verve-101, and preliminary results were presented last November at the scientific sessions of the American Heart Association, and indicate a reduction in bad cholesterol levels in the blood by about 50%.
David Baldwin is a pulmonologist at Nottingham University Hospital and suggested nature It is the use of artificial intelligence as a tool to diagnose lung cancer early. Nearly three out of four cases are identified in late stages, and the researcher suggests that a chest X-ray, followed immediately by a computed tomography (CT) scan, could advance detection.
The experiment is being conducted on 150,000 patients from six British hospitals and aims to show that applying artificial intelligence reduces the time required to conduct a CT scan from 63 to 32 days. The team plans to finish the recruitment process in July 2024. The idea is to show that halving the waiting time between tests 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,” Baldwin says.
The list of articles also includes a selection by Carlin M. Van der Aalst, associate professor at the Department of Public Health and Erasmus Medical Center Rotterdam. In this case, we want to see whether lung cancer screening with CT every two years is as effective in preventing deaths as annual screening for those who have no abnormalities at the first screening.
The lymphocyte vaccine VIR-1388 has the potential to prevent human immunodeficiency virus (HIV) infection, says the head of clinical research at Vir Biotechnology in San Francisco (USA), infectious disease scientist Cary Hwang. The Phase 1 study includes adults ages 18 to 55, and the vaccine is based on vectors of cytomegalovirus, a herpes virus that induces “robust, unique and sustained” defense responses that can prevent HIV infection.
University of Liverpool child psychiatrist Atif Rahman highlights 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 perform cognitive therapy for pregnant women with major depression during the second or third trimester of pregnancy. “This number reinforces therapeutic messages and helps solve problems through empathy and mutual support,” and they just have to follow the app’s instructions.
The study Rahman proposes is funded by the UK Department of Health, through the Research and Innovation for Global Health Transformation programme, but was inspired by Pakistan’s Rural Health Thinking programme, endorsed by the World Health Organization (WHO). The psychiatrist adds: “We hope that the results of this study will stimulate more innovation and research in this important field that could change the rules of the game in addressing the treatment gap for a common mental disorder around the world.”
“Most clinical trials for Parkinson’s disease are conducted with patients at an advanced stage of the disease. As a result, those at an earlier stage of the disease, who could benefit most from treatment, are often excluded,” says the head of the research team in Neurobiology Development and Regeneration. At Lund University, Sweden, Malin Parmar.
She is also a professor at the New York Stem Cell Foundation and, in 2024, proposes to conduct a STEM-PD trial on the radar, in which neurons derived from human embryonic stem cells (called dopamine) would be transplanted into patients’ brains. They are aged 50 to 75 years and suffer from moderate Parkinson’s disease. The first patients received the doses last February, and researchers hope to have preliminary results by the end of 2024. This is the first time this treatment has been tested in Parkinson’s disease, which has so far been useful in treating leukemia and lymphomas.
In emergency services, good triage is essential; This means identifying patients’ risks in order to prioritize their care and refer them to appropriate services. To do this, Stephen Meeks, Head of the General Clinical Chemistry and Hematology Unit at the Central Diagnostic Laboratory in Maastricht (Netherlands), is betting on using an already existing clinical risk score, Riskindex, which allows predicting 31-day mortality. Patients who requested treatment in the emergency department. It is hypothesized that if the AI model is validated, 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 system has outperformed internal medicine specialists, but how useful these models are when implemented in clinical practice remains unknown,” Meeks admits. Recruitment for the study is halfway through, and researchers hope to see results as early as 2024.
Christian Blank is Professor of Medical Oncology at Leiden University and researcher at the Netherlands Cancer Institute. His suggestion is to use immunotherapy to fight skin cancer. In fact, his team is pioneering the use of checkpoint inhibitors, which make a patient’s immune system fight cancer. Now they have to confirm, in a phase III trial called “Our Club,” how this treatment works in melanoma.
The study included 420 patients from Australia, Europe and the United States with stage III primary or cutaneous melanoma. In these cases, adjuvant anti-PD-1 therapy (which opposes proteins generated by the immune system itself and allows some tumors to evade treatment) improves survival, but a large proportion of patients still suffer relapses. With this change, they hope to see an overall survival benefit, “which could become a practice change in the treatment of stage III melanoma.”
According to the University of Oxford, children aged 5 to 17 months who have been vaccinated three times with the malaria vaccine, R21/Matrix-M, can get 80% protection if they also receive a booster dose one year later. If this cap is confirmed, his vaccine will 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 the University of Oxford, who confirms that only two vaccines have shown beneficial effectiveness, the Oxford vaccine and Moskirksi, from GlaxoSmithKline. He points out that “the effectiveness of this decreases from 55% to 30% four years after vaccination.”
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