In the world of medicine, many promises fail due to failure in clinical trials. Very often, inventions that show great potential when studied in the laboratory are disappointed when tested in humans. But there are already several achievements that could bring great joy in the not-too-distant future.
Magazine Natural medicine selected 11 clinical trials – that is, interventions that are already being tested in humans – with the potential to change the medical landscape in the next year.
The compilation of such a list usually draws on the passion for pharmacological and technological innovation. What’s interesting about this work, however, is that it combines the great promise of cutting-edge treatments—base editors or antibody-drug conjugates—and artificial intelligence with simple and highly effective interventions.
Genetically modify a patient
The future is here. After years of waiting, the first therapy based on the gene cutter CRISPR was approved in 2023, the technology that has generated the most headlines over the past decade for its potential to transform medicine.
The approved therapy is based on extracting cells from the patient, modifying them and reintroducing them. However, Verve-101 wants to change them inside the patient’s own body.
Heart 1, a study of several patients with heterozygous familial hypercholesterolemia, has already shown promising results: with a single intravenous infusion, the level of LDL, or “bad” cholesterol, is reduced by half. these patients who, due to genetics, have excessively high blood sugar levels, which leads to serious cardiovascular problems.
In 2024, its results will be confirmed, and Verve-101 will be able to take the next step, also opening a new field of medicine, the one that has caused the most rivers of ink in recent years.
Brain cell transplantation
Another area that has been written about for many years and has not yet made much progress is embryonic stem cells, which have the potential to become any other cell in the human body.
“STEM-PD” will show its effectiveness in Parkinson’s disease, which has a double challenge: it is a pathology with few therapeutic options, and its origin is in the brain. an organ virtually impenetrable to modern treatments.
A few months ago, scientists at Skåne University Hospital in Sweden began transplanting dopaminergic stem cells (derived from embryonic stem cells) into the brains of patients aged 50 to 75 with mild Parkinson’s disease. By 2024, they plan to have preliminary results of this therapy: if it demonstrates benefits, there will be a “before” and “after” not only in neurology, but in all of medicine.
Changing views on cancer treatment
10 years ago, drugs burst onto the world to change the way we look at cancer. Their technical name is “immune checkpoint inhibitors,” but they are commonly known as immunotherapy.
It was a paradigm shift: The treatment did not attack the cancer cells, but rather activated the immune system to recognize and destroy them.. Despite this, the treatment system remains classic: step by step from surgery to the latest options.
Nadina aims to change this situation by introducing immunotherapy before surgery. Spanish doctors pioneered this new approach to treating lung cancer, and now the Netherlands Cancer Institute is about to apply it to melanoma.
The idea behind this new strategy is that activating the immune system benefits patients more at the beginning of treatment than at the end, and Nadina will be a validation of a new way to treat cancer.
If there are several drugs with different potential against cancer cells, why not use them simultaneously? This is the essence of antibody-drug conjugates: combine the specificity of monoclonal antibodies (they attack only tumor cells) and the destructive ability of chemotherapy..
These drugs have just reached patients, and now they want to go even further: to reach brain metastases, tumor cells to spread into areas inaccessible to current drugs.
In fact, patients with brain metastases are often left out of clinical trials. DESTINIY-Breast12 is trying to solve this problem by testing the effectiveness of trastuzumab deruxtecan (a drug already marketed as Enhertu) in breast cancer that has spread to other parts of the body.
Conjugates are biological drugs that are significantly larger in size than chemotherapy molecules. Theoretically, they are less able to cross the blood-brain barrier and eliminate brain metastases. Thus, if trastuzumab deruxtecan demonstrates its effectiveness, it will open up new areas of application for biological cancer therapy: it will achieve something that has not yet been achieved.
Artificial intelligence keeps its promises
Over the past year, artificial intelligence has become ubiquitous in conversations and, of course, could not help but be present in the most promising clinical trials of 2024.
Nature however, chose a “conservative” essay on the possibilities of AI: prioritizing patients to be treated, something in which machine learning has been working for many years.
Based on an index that ranks patients by risk of mortality in the next month, four Dutch hospitals are testing MARS-ED. AI that will help doctors triage patients entering the emergency department.
The algorithm took data from 266,327 patients and has already been shown to outperform GPs in stratification, but MARS-ED will aim to go one step further and help in environments where speed and accuracy are vital. Its results are expected in mid-2024.
Children’s mental health
Another hot topic, especially since the pandemic, is mental health concerns. Instead of new treatments for depression, such as psychotropic drugs, Nature prefers to focus on less dramatic interventions, but with proven effectiveness.
One of the most underserved mental health needs is that of children. The New Orleans Intervention Model provides assessment and intervention for orphaned and vulnerable children ages 0 to 5 years.
The Better Services Trial aims to evaluate this intervention in centers in London and Glasgow and will follow children for two and a half years. Its promoters believe that if its effectiveness is demonstrated, “could radically change the way these children are cared for, not just in the UK but around the world.“.
Application for depression in pregnant women
Continuing the theme of mental health, the magazine offers another new approach, focusing primarily on low- and middle-income countries. where access to cognitive therapy is not widespread.
This is an app that allows a woman to be trained to care for other people in the second or third trimester of pregnancy (and postpartum) in the same community who are suffering from major depression.
The study aims to demonstrate the potential of digital medicine in places where there are no healthcare providers, comparing this type of intervention with face-to-face contact with a professional.
Do malaria vaccines work long term?
Continuing to work with a dysfunctional environment, Nature focused on the malaria vaccine, one of the major medical advances of the decade, because it is a devastating disease that is widespread and for which prevention did not exist less than five years ago.
The problem is that The effectiveness of these vaccines decreases over time, from 55% in the first year to 30% in the fourth..
The study is enrolling 2,400 African children aged 5 to 36 months, with booster shots administered one year after the full three-dose regimen. They are using the R21 vaccine, which uses nanoparticles with a higher density of antigens on the surface than the other vaccine, RTS,S, and will be monitored for two years to see how effective it is.
New attack on HIV
After the recent failure of the most advanced HIV vaccines, hope is not lost. The Phase 1 trial (meaning it is the first time it is being tested in humans) aims to vaccinate people aged 18 to 55 years, without HIV and in good health, with VIR-1388, a new vaccine that produces a strong immunogenic response.
It builds on the previous vaccine VIR-1111, which is based on cytomegalovirus and has demonstrated good safety but poor immune response. VIR-1388 is less attenuated, so a strong response is expected..
People who have antibodies against cytomegalovirus have already received the vaccine to ensure its safety. Once this phase is over, it will be extended to the rest of the population over three years.
The bet on this vaccine is strong. The study, led by the US-South Africa HIV Vaccine Trial Network, is supported by the country’s National Institute of Allergy and Infectious Diseases and the Bill & Melinda Gates Foundation.
Back to Lung Cancer Screening
More people are talking about lung cancer screening every day, but it has not yet become a reality. It remains to be decided whether its utility outweighs the effort required to implement it and the trial chosen Nature will give you one of the keys.
“4-IN THE LENG RUN” is the name of the study, which is being conducted in six European countries and is targeting 26,000 people to determine whether a CT scan every two years is enough to prevent cancer deaths in people who are not diagnosed at the first test. identified deviations.
The idea behind the study is to reduce the cost of implementing a national screening program.as well as the possible harm of periodic imaging studies for part of the population.
When screening, it is important to balance the cost of the intervention against the benefit achieved. Performing annual imaging tests in the smoking population is very expensive and may not detect many cancers at an early stage, so costs and benefits must be adjusted for screening to work.
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