Rens Dimmendaal & Banjong Raksaphakdee / Better Images of AI / Medicines (flipped) / Licenced by CC-BY 4.0 The potential of using artificial intelligence in drug discovery and development has sparked both excitement and skepticism among scientists, investors and the general public. "Artificial intelligence is taking over drug development," claim some companies and researchers. Over the past few years, interest in using AI to design drugs and optimize clinical trials has driven a surge in research and investment. AI-driven platforms like AlphaFold, which won the 2024 Nobel Prize for its ability to predict the structure of proteins and design new ones, showcase AI's potential to accelerate drug development. AI in drug discovery is "nonsense," warn some industry veterans. They urge that "AI's potential to accelerate drug discovery needs a reality check," as AI-generated drugs have yet to demonstrate an ability to address the 90% failure rate of new drugs in clinical trials.

Clinical trials are an indispensable part of the drug development process, bridging the gap between basic research and clinical application. During the development of new drugs, clinical trials are used not only to evaluate the safety and efficacy of the drug but also to explore its dosage, treatment regimens, and potential side effects. This review discusses the various stages of clinical trials, including Phase I (safety assessment), Phase II (preliminary efficacy evaluation), Phase III (large-scale validation), and Phase IV (post-marketing surveillance), highlighting the characteristics of each phase and their interrelationships. Additionally, the paper addresses the major challenges encountered in clinical trials, such as ethical issues, subject recruitment difficulties, diversity and representativeness concerns, and proposes strategies for overcoming these challenges. With the advancement of technology, innovative technologies such as artificial intelligence, big data, and digitalization are gradually transforming clinical trial design and implementation, improving trial efficiency and data quality. The article also looks forward to the future of clinical trials, particularly the impact of emerging therapies such as gene therapy and immunotherapy on trial design, as well as the importance of regulatory reforms and global collaboration. In conclusion, the core role of clinical trials in drug development will continue to drive the progress of innovative drug development and clinical treatment.

The drug development process is a critical challenge in the pharmaceutical industry due to its time-consuming nature and the need to discover new drug potentials to address various ailments. The initial step in drug development, drug target identification, often consumes considerable time. While valid, traditional methods such as in vivo and in vitro approaches are limited in their ability to analyze vast amounts of data efficiently, leading to wasteful outcomes. To expedite and streamline drug development, an increasing reliance on computer-aided drug design (CADD) approaches has merged. These sophisticated in silico methods offer a promising avenue for efficiently identifying viable drug candidates, thus providing pharmaceutical firms with significant opportunities to uncover new prospective drug targets. The main goal of this work is to review in silico methods used in the drug development process with a focus on identifying therapeutic targets linked to specific diseases at the genetic or protein level. This article thoroughly discusses A-to-Z in silico techniques, which are essential for identifying the targets of bioactive compounds and their potential therapeutic effects. This review intends to improve drug discovery processes by illuminating the state of these cutting-edge approaches, thereby maximizing the effectiveness and duration of clinical trials for novel drug target investigation.

Large Language Models (LLMs) have recently demonstrated remarkable performance in general tasks across various fields. However, their effectiveness within specific domains such as drug development remains challenges. To solve these challenges, we introduce \textbf{Y-Mol}, forming a well-established LLM paradigm for the flow of drug development. Y-Mol is a multiscale biomedical knowledge-guided LLM designed to accomplish tasks across lead compound discovery, pre-clinic, and clinic prediction. By integrating millions of multiscale biomedical knowledge and using LLaMA2 as the base LLM, Y-Mol augments the reasoning capability in the biomedical domain by learning from a corpus of publications, knowledge graphs, and expert-designed synthetic data. The capability is further enriched with three types of drug-oriented instructions: description-based prompts from processed publications, semantic-based prompts for extracting associations from knowledge graphs, and template-based prompts for understanding expert knowledge from biomedical tools. Besides, Y-Mol offers a set of LLM paradigms that can autonomously execute the downstream tasks across the entire process of drug development, including virtual screening, drug design, pharmacological properties prediction, and drug-related interaction prediction. Our extensive evaluations of various biomedical sources demonstrate that Y-Mol significantly outperforms general-purpose LLMs in discovering lead compounds, predicting molecular properties, and identifying drug interaction events.

Understanding the structure of the protein-ligand complex is crucial to drug development. Existing virtual structure measurement and screening methods are dominated by docking and its derived methods combined with deep learning. However, the sampling and scoring methodology have largely restricted the accuracy and efficiency. Here, we show that these two fundamental tasks can be accurately tackled with a single model, namely LigPose, based on multi-task geometric deep learning. By representing the ligand and the protein pair as a graph, LigPose directly optimizes the three-dimensional structure of the complex, with the learning of binding strength and atomic interactions as auxiliary tasks, enabling its one-step prediction ability without docking tools. Extensive experiments show LigPose achieved state-of-the-art performance on major tasks in drug research. Its considerable improvements indicate a promising paradigm of AI-based pipeline for drug development.

The field of drug discovery has experienced a remarkable transformation with the advent of artificial intelligence (AI) and machine learning (ML) technologies. However, as these AI and ML models are becoming more complex, there is a growing need for transparency and interpretability of the models. Explainable Artificial Intelligence (XAI) is a novel approach that addresses this issue and provides a more interpretable understanding of the predictions made by machine learning models. In recent years, there has been an increasing interest in the application of XAI techniques to drug discovery. This review article provides a comprehensive overview of the current state-of-the-art in XAI for drug discovery, including various XAI methods, their application in drug discovery, and the challenges and limitations of XAI techniques in drug discovery. The article also covers the application of XAI in drug discovery, including target identification, compound design, and toxicity prediction. Furthermore, the article suggests potential future research directions for the application of XAI in drug discovery. The aim of this review article is to provide a comprehensive understanding of the current state of XAI in drug discovery and its potential to transform the field.

Major drugmakers are using artificial intelligence to find patients for clinical trials quickly, or to reduce the number of people needed to test medicines, both accelerating drug development and potentially saving millions of dollars. Human studies are the most expensive and time-consuming part of drug development as it can take years to recruit patients and trial new medicines in a process that can cost over a billion dollars from the discovery of a drug to the finishing line. Pharmaceutical companies have been experimenting with AI for years, hoping machines can discover the next blockbuster drug. A few compounds picked by AI are now in development, but those bets will take years to play out.

Fox News contributor Dr. Marc Siegel weighs in on how artificial intelligence can change the patient-doctor relationship on'America's Newsroom.' Artificial Intelligence has an exciting future in health care, from streamlining insurance claims, to aiding radiologists, dermatologists, cardiologists and other specialties by enhancing data-based pattern recognition, from providing rapid information and improving efficiency in hospitals to a direct role in the doctor's office in informing both doctors and patients. Don't get me wrong, I have great respect for clinical judgment, creative solutions, and the need to preserve patient privacy. My personal empathy cannot be replaced by a computer voice, no matter how soothing it is. And so AI must work as a kind of co-pilot in the doctor's office.

Biotech buzzwords do not come much bigger than those of artificial intelligence (AI) and machine learning (ML). With the promise of expediting routes to market and reducing costs, such platforms may very well find themselves cemented as critical components in future drug development toolboxes. The application of ML approaches in the pharma industry has now matured to a stage where the first purely ML generated candidates have entered clinical trials. However, we are still a way off from using solely ML to uncover completely new disease mechanisms of action or targets, which many would consider as the holy grail of applications. Leading the charge towards this ambition are focused biotechs leveraging ML that are not only applying their platforms to bolster internal pipelines but striking deals with big pharma, which are likely to be the main clients, utilising ML to boost their own portfolios. The pharma industry continues to evolve and, based on current macro pressures, we are likely to see different approaches to increase efficiency and expedite drug discovery.

AI is being used in many areas of human biology, including genetics, drug development, and medical imaging. One potential advancement that could be achieved with the use of AI in human biology is the ability to personalize medical treatments based on an individual's genetic makeup. By analyzing an individual's DNA, AI algorithms could identify specific genetic variations that may make them more or less responsive to certain drugs. This could lead to more effective treatments with fewer side effects. Another area where AI is being tested is in the early detection of diseases.