Nature Medicine Reveals 11 Clinical Trials to Watch in 2024
On the 7th (local time), the international academic journal Nature Medicine selected and revealed 11 clinical trials that the medical community is paying attention to in 2024. Ben Johnson, senior editor at Nature Medicine, stated that the chosen clinical trials are mainly aimed at addressing important global health concerns.
Stem Cell Treatment for Parkinson’s Disease and DNA Editing for Genetic Diseases
Phase 1 results of the stem cell treatment for Parkinson’s disease (STEM-PD) are expected to be released next year. The trial, led by researchers at Skåne University Hospital in Sweden, involves transplanting dopamine neurons extracted from human embryonic stem cells into the brains of patients with moderate Parkinson’s disease. This groundbreaking trial holds the potential to provide therapeutic benefits for numerous patients if proven effective.
Meanwhile, Verb Therapeutics in the US is conducting a phase 1 clinical trial on a DNA editing treatment for the inherited disease ‘heterozygous familial hypercholesterolemia’. This DNA editing treatment aims to continuously reduce LDL, a risk factor for cardiovascular disease, by inactivating the PCSK9 gene in the liver.
AIDS and Malaria Vaccines
Vir Biotech of the United States is conducting phase 1 clinical trials for a vaccine (VIR-1388) to prevent HIV infection. The vaccine is designed to stimulate a T-cell response among immune cells, and results from this trial are expected to have a significant public health impact.
University of Oxford in the UK and the Serum Institute of India are conducting a phase 3 clinical trial on a malaria vaccine (R21) targeting infants in four African countries. Researchers are optimistic that the R21 vaccine, utilizing nanoparticles with concentrated antigens, will be more effective than existing malaria vaccines.
Advanced AI in Medical Diagnostics
Nottingham University Hospitals NHS Trust in the UK is testing a deep learning algorithm (qXR) that can analyze chest X-ray and CT images to diagnose lung cancer. Preliminary results have shown a significant reduction in diagnosis time, offering hope for earlier detection and improved survival rates for lung cancer patients.
Additionally, researchers at the Erasmus Medical Center in the Netherlands are conducting a clinical trial on CT screening for AI (4-IN THE LUNG RUN) targeting 26,000 people to reduce the death rate from lung cancer. Meanwhile, a machine learning algorithm (RISKINDEX) is being tested at Maastricht University Medical Center to classify high-risk patients in the emergency room, aiding in determining appropriate treatment methods.
Other notable clinical trials include treatments for breast cancer, melanoma, and depression. AstraZeneca in the UK is conducting a phase 3 clinical trial for an antibody-drug combination for breast cancer patients, while the Netherlands Cancer Institute is testing a combined immunotherapy treatment for patients with stage 3 melanoma.
In summary, the clinical trials revealed by Nature Medicine represent cutting-edge advancements in medical research and have the potential to significantly impact global healthcare in the coming year.
On the 7th (local time), the international academic journal ‘Nature Medicine’ selected 11 clinical trials that the medical community should pay attention to next year./Nature Medicine
The first results are expected to be released as early as next year to determine whether the stem cell treatment for the incurable disease Parkinson’s disease and the vaccine to prevent acquired immune deficiency syndrome (AIDS) are actually effective and safe for people. Also expected to be released are the results of clinical trials targeting humans for early diagnosis of lung cancer, artificial intelligence (AI) and depression treatment apps.
On the 7th (local time), the international academic journal ‘Nature Medicine’ selected and revealed 11 clinical trials that the medical community is paying attention to in 2024. Ben Johnson, senior editor at Nature Medicine, said, “The trials clinical trials chosen this time are mainly efforts to solve diseases that are considered important around the world.”
◇ Will stem cells to treat Parkinson’s disease and DNA editing to cure genetic diseases succeed?
Symptoms of Parkinson’s disease./CarrotsMitHummus
The results of phase 1 clinical trials for stem cell treatment for Parkinson’s disease (STEM-PD) are expected to be released first.
Researchers at Skåne University Hospital in Sweden are conducting a clinical trial to transplant dopamine neurons extracted from human embryonic stem cells into the brains of patients with moderate Parkinson’s disease between the ages of 50 and 75. This is the first clinical trial to treat Parkinson’s disease with human embryonic stem cells. The first group of patients received the treatment in February, and phase 1 results are due to be released at the end of next year.
Merlin Palmer, head of the developmental and regenerative neurobiology research team at Lund University in Sweden (professor at the New York Stem Cell Institute, USA), said, “This is a clinical trial targeting patients with moderate disease (whose disease has already moving forward) , so if efficacy is confirmed, it could be of therapeutic benefit to many patients.” “It will,” he expected.
Verb Therapeutics in the US is conducting a phase 1 clinical trial on a DNA editing treatment for the inherited disease ‘heterozygous familial hypercholesterolemia’. This clinical trial is the first study in the world to edit DNA bases in vivo.
Heterozygous familial hypercholesterolemia is a common genetic disease affecting 1 in 300 people. It is caused by a mutation in the gene (PCSK9) which breaks down low density lipoprotein (LDL), also known as ‘bad cholesterol.’ Conventionally, patients are treated with drugs called statins to prevent them from developing cardiovascular disease, but most patients do not respond well to these drugs.
VERVE-101, a DNA editing treatment, continuously reduces LDL, which causes cardiovascular disease, by inactivating the PCSK9 gene in the liver. This treatment consists of messenger ribonucleic acid (mRNA), which can edit adenine bases, and guide RNA, which takes the patient to the appropriate location on DNA.
◇ AIDS vaccine and malaria vaccine that are more effective than the current ones
Vir Biotech of the United States has developed a vaccine (VIR-1388) to prevent human immunodeficiency virus (HIV) infection and is conducting phase 1 clinical trials. The photo shows the ‘Walk to End AIDS’ event held in Manila, Philippines to mark World AIDS Day on the 2nd of this month./Yonhap News
Clinical trials for AIDS vaccines and malaria vaccines are also notable. Vir Biotech of the United States has developed a vaccine (VIR-1388) to prevent human immunodeficiency virus (HIV) infection and is conducting phase 1 clinical trials. Safety, reactivity and immunogenicity are being evaluated, that is, does an immune response occur when the virus actually invades the body.
VIR-1388 prevents AIDS by stimulating a T-cell response among immune cells that eat virus-infected cells. The vaccine and a placebo will be given to healthy adults between the ages of 18 and 55 with no history of AIDS to compare their effectiveness. The medical community expects that the results of clinical trials for this AIDS vaccine will have an enormous public health impact.
The University of Oxford in the UK and the Serum Institute of India are conducting a phase 3 clinical trial on the malaria vaccine (R21) targeting infants between 5 and 36 months of age in four African countries, including Burkina Faso, Kenya, Tanzania, and Mali. Academic circles have been developing malaria vaccines for over 100 years, but have repeatedly failed. The reason is that a high antibody response is needed for a vaccine to work against malaria parasites, but the effectiveness of those previously developed was too low.
Vaccines contain parts (antigens) of real pathogens and are based on the principle of activating the immune system in the body in advance without the presence of pathogens. Adrian Hill, professor of vaccination at the University of Oxford, expected that “R21 will be more effective than existing malaria vaccines because it uses nanoparticles with more concentrated antigens.” The human papilloma virus (HPV) vaccine, which is currently commercialized worldwide, is also effective for life because of these features.
◇ AI that detects lung cancer early, AI that classifies emergency patients into high-risk groups
Researchers at Nottingham University Hospitals NHS Trust in the UK are conducting clinical trials on a deep learning (qXR) algorithm that analyzes chest X-rays and computed tomography (CT) images. The photo is a chest X-ray./Mikael Häggström
Researchers at Nottingham University Hospitals NHS Trust in the UK are conducting clinical trials on a deep learning (qXR) algorithm that analyzes chest X-rays and computed tomography (CT) images.
Researchers are testing whether using this AI to differentiate chest X-ray or CT images of 150,000 lung cancer patients at six UK hospitals can reduce diagnosis time. The interim results showed that the time was almost halved from 63 days to 32 days. David Baldwin, Emeritus Professor of Respiratory Medicine at Nottingham University Hospital, said, “Three quarters of lung cancer patients are diagnosed late. If they are detected and diagnosed even slightly earlier, the survival rate will increase accordingly with that.” He predicted that this AI will reduce the time to detect and diagnose lung cancer by 50%.
Researchers at the Erasmus Medical Center in the Netherlands are conducting a randomized clinical trial on CT screening for AI (4-IN THE LUNG RUN) targeting 26,000 people. Interim results confirmed that using this AI can reduce the death rate from lung cancer as much as performing a CT scan every two years.
Researchers at Maastricht University Medical Center in the Netherlands are also conducting a randomized clinical trial on a machine learning algorithm (RISKINDEX) that correctly classifies who is high risk among patients who come to the emergency room. This AI predicts the patient’s 31-day mortality rate. It was developed in four hospitals in the Netherlands and learned based on data from 276,327 patients. Stephen Mix, head of the hematology department at the Central Diagnostic Research Institute at Maastricht University, said, “Classification of high-risk groups according to the severity of the patient’s crisis in the emergency room will help determine treatment methods.”
On the 7th (local time), the international academic journal ‘Nature Medicine’ selected 11 clinical trials that the medical community should pay attention to next year./Nature Medicine
Clinical trials related to the treatment of cancer, such as breast cancer and melanoma, also attract attention.
AstraZeneca in the UK is conducting a phase 3 clinical trial to test the efficacy and safety of the antibody-drug combination (ADC) ‘trastuzumab deruxtecan’ in breast cancer patients with the HER2 gene, regardless of the presence or absence of brain metastases. ADC is a form in which a drug is bound to an antibody that binds to a specific antigen.
The Netherlands Cancer Institute is conducting a phase 3 clinical trial on the combined treatment of the immunotherapy drugs ‘ipilimumab (Yervoy)’ and ‘nivolumab (Opdivo)’ for patients with stage 3 melanoma.
In addition, there is a randomized clinical trial of an app (THP-TA) that provides cognitive therapy to depressed women during mid to late pregnancy, and the New Orleans Intervention Model for the mental health of 0-5 year olds in care nutrition. ■ Randomized clinical trials are underway.
reference material
Nature Medicine (2023), DOI:
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