Acute myeloid leukaemia (AML) is the most common acute leukaemia in adults, with around 20,000 annual diagnoses in developing countries. Its pathogenesis involves genetic mutations, such as NPM1, CEBPA, and FLT3-ITD, alongside chromosomal translocations that lead to the proliferation of poorly differentiated myeloid cells. While AML is a primary concern, it is essential to consider other leukaemia types and associated risk factors. Prognosis in AML varies among patients with similar cytogenetic profiles, categorized as favorable, intermediate, or adverse. Standard treatments include chemotherapy regimens, such as anthracycline and cytarabine, and allogeneic stem cell transplantation. Recent research highlights the significance of mutations in signal transduction pathways and transcription factors in myeloid differentiation. Continued exploration of these genetic factors will enhance our understanding of AML and improve treatment outcomes, emphasizing the need for a comprehensive approach to patient management. Finally, AML genetic research and clinical consequences may improve treatment regimens and patient outcomes.

We demonstrated a facile microwave-assisted synthesis of 16 novel phthalimide (Pht)-hydroxyethylamine (HEA)-based compounds isolated without tedious column chromatography. Among all the tested compounds, 3b, 3f, and 3h displayed 50% inhibitory concentration in the micromolar range (0.17 – 1.60 µM) against the Pf3D7 strain. Of particular note, compound 3f showed the highest potency with an IC50 value of 0.17 ± 0.001 µM. Further, stage-specific assay revealed that hit 3f was predominantly active at the ring stage. None of the compounds exhibited marked cytotoxicity up to 500 µM concentration on HepG2 liver cells. Further, preliminary computational studies suggested that aminopeptidase N could be the potential target for hit 3f, which needs to be validated through enzymatic assays.

Alzheimer's disease causes cognitive decline, and drug discovery focuses on its pathophysiology. Current research is concentrating not only on amyloid β markers and tangles but also on synaptic defects, mitochondrial dysfunction, and inflammation as key therapeutic targets. Treatments for the amyloid pathway include monoclonal antibodies (e.g., aducanumab and lecanemab) to remove formed Aβ. Ongoing preliminary studies of small molecules and beta-secretase inhibitors are indeed targeting Aβ production to interrupt this toxic cascade. Small molecule strategies involve using small molecules and antisense oligonucleotides to reduce tau hyperphosphorylation and its levels. Future treatments for neurodegenerative diseases aim to modulate microglial activation and cytokine signaling using potential NLRP3 inhibitors, for example, to balance the immune response. Biological mesenchymal stem cells (MSCs) and neural progenitor cells (NPCs) are being discussed for neurodegenerative treatment and stem cell therapy. Nanotechnology has improved drug delivery across the blood-brain barrier, resulting in better targeting and reduced toxicity. Gene editing via CRISPR-Cas9 gene therapy targeting the genetic basis of Alzheimer's disease is on the horizon. Biomarkers are assisting in identifying and monitoring treatment response in Alzheimer's disease, while novel uses of FDA-approved drugs offer new treatment options, improve management strategies, and potentially enhance patient outcomes.

In drug discovery, efficiency and precision are crucial elements that play pivotal roles in saving lives, time, and financial resources while pursuing groundbreaking advancements. To aid this, commercial software platforms are explicitly crafted. These platforms leverage the power of classical with quantum mechanics, machine learning and artificial intelligence to predict molecular behaviours and interactions, moving beyond traditional trial-and-error methods. These approaches fundamentally revolutionize identifying, designing, and optimizing potential drug candidates. This review compares commercial tools such as Discovery Studio, Molecular Operating Environment (MOE) and Schrödinger. Our focus is primarily on Schrödinger due to our hands-on experience on it. In addition to the comparison, we highlight Schrödinger's modules, advantages, achievements, and capacity to streamline the discovery of PROTACs, small molecule inhibitors, and antibodies.