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Financial problems persisted in the pharmaceutical sector in 2023, prompting industry observers to wonder when and how the field would recover.

• Discovery of a new antibiotic in the human nose 

The renowned “Nature” journal surveyed experts who anticipated a qualitative leap in the sector in 2024, with numerous clinical trials paving the way for the production of new drugs.

Among the prominent clinical trials:

1. Treatment for Familial Hypercholesterolemia Approximately 1 in every 300 individuals suffers from familial hypercholesterolemia, a common inherited genetic condition. This disease results from mutations in the “PCSK9” gene, encoding a protein that breaks down low-density lipoprotein (LDL) receptors, a type of cholesterol.

• Extra hours of sleep on weekends “protect your heart” 

Despite the cholesterol-lowering effects of “statin” drugs, most patients cannot achieve optimal cholesterol levels. In 2024, a clinical trial will test the drug “VERVE-101,” which inhibits the “PCSK9” gene in the liver to reduce harmful cholesterol levels.

The components of “VERVE-101” are encapsulated in nano-sized lipid particles and delivered to the body through intravenous injection. A study on this drug was presented at the American Heart Association conference in 2023.

1. Artificial Intelligence for Early Lung Cancer Diagnosis Early diagnosis of lung cancer saves lives. A trial involving 150,000 patients across 6 UK hospitals will assess the application of artificial intelligence to chest X-rays, reducing the time needed for computed tomography (CT) scans and diagnosis.

• “Noma” mouth eater… The neglected serious disease! 

1. T-cell Vaccine for HIV In 2024, a trial will evaluate the safety and immune response of the “VIR-1388” vaccine for preventing human immunodeficiency virus (HIV) infection. Adults aged 18-55 with general good health and without HIV will receive one of three doses of VIR-1388 or a placebo.

“VIR-1388” is a cytomegalovirus (CMV) vector vaccine that stimulates strong, unique, and sustainable T-cell responses to prevent HIV infection. Safety was demonstrated in a proof-of-concept trial.

1. Stem Cells for Parkinson’s Disease The “STEM-PD” trial, overseen by the New York Stem Cell Foundation, implants dopamine neuronal cells derived from human embryonic stem cells into the brains of patients aged 50-75 with moderate Parkinson’s disease. This marks the first test of human embryonic stem cell therapy for Parkinson’s.

• World Health Organization” warns of a new global threat 

Initial doses were administered in February 2023, with preliminary results expected by the end of 2024.

1. Machine Learning for Patient Triage The “RiskIndex” system, using artificial intelligence to predict patient deaths in the emergency department, outperformed internal medicine specialists in a recent retrospective study. A clinical trial on this system is expected to reveal its clinical value in 2024.
2. Immunotherapy for Skin Cancer The international phase III trial “NADINA” will compare the effectiveness of the new adjuvant “ipilimumab” with the current adjuvant “nivolumab” in stage III melanoma patients. The trial aims to improve survival in resectable skin cancer.

• Spread of Measles in Gaza… Is it turning into an epidemic? 

1. Long-term Efficacy of Malaria Vaccine R21 Oxford University initiated clinical trials of the R21/Matrix-M malaria vaccine in African children. Results, expected in 2024, will address the challenge of achieving a sustained high antibody response for malaria vaccines.