Cancer, a multifactorial and heterogeneous disease, poses a significant global health challenge. Despite current treatments such as surgery, radiotherapy, and chemotherapy, tumour recurrence and treatment side effects are common. These pitfalls necessitate a dire need for alternative therapeutic strategies with minimal side effects. This necessity has broadened the horizons of drug discovery into the marine domain, an exciting frontier for novel therapeutic agents. The marine ecosystem serves as a hub of diverse chemical groups with potential anti-cancer properties. Few marine-derived drugs are approved for cancer, and preliminary studies show that marine lead compounds can inhibit cancer cell growth and induce apoptosis. In this context, this review encapsulates an overview of ‘the current state of marine biodiscovery’. It explores the ‘potential of marine natural products in combating cancer’ with a particular focus on glioblastoma multiforme as a case study. Additionally, it discusses the ‘key strategies for advancing marine-derived anti-cancer compounds from the research stage to clinical use’. By tapping into the vast, unlocking the hidden treasures of the ocean, marine natural compounds could offer a hopeful perspective in the fight against cancer.

Sulforaphane (SFN), a bioactive compound derived from glucoraphanin in cruciferous vegetables such as broccoli, has been extensively studied for its therapeutic potential across diverse disease categories. SFN exerts its effects through well-characterised pathways, including the Keap1/Nrf2 axis, which regulates phase II detoxification enzymes, and epigenetic mechanisms such as histone deacetylase inhibition. This review evaluates clinical trials registered on ClinicalTrials.gov, focusing on those using SFN or broccoli-derived extracts.

As a result, we identified 84 trials, of which 39 have been published. Results suggest SFN’s potential in regulating redox and inflammatory pathways, improving metabolic and cardiovascular outcomes, and exerting anti-cancer and neuroprotective effects. For healthy subjects, SFN enhanced detoxification and reduced inflammation. In cancer patients, SFN showed promise in early-stage prostate and breast cancer, particularly in GSTM1-positive individuals, but had limited effects in advanced cases. For brain disorders, SFN demonstrated symptomatic improvements in autism spectrum disorder and cognitive benefits in schizophrenia but lacked robust biomarker integration. SFN had minimal impact on respiratory diseases but showed supportive roles in allergic rhinitis therapy. Metabolic disease studies revealed glycaemic control improvements in type 2 diabetes but no benefits for hypertension. Approximately 50% of completed trials remain unpublished, raising concerns about publication bias. While published results highlight SFN’s therapeutic potential, limited sample sizes and inconsistent outcomes underscore the need for more extensive, stratified trials. This review emphasises the importance of integrating mechanistic insights and precision medicine approaches to maximise SFN’s clinical utility.

Prospective consent in neonatal research poses significant challenges, particularly during urgent, time-sensitive clinical windows of study enrollment. This is especially true at referral centers for large geographic regions. A partial waiver of consent offers a potential translational science approach to enhance access to research participation in critically ill neonates. We compared enrollment rates in a study evaluating pulse oximetry accuracy across neonates with varying skin pigmentation before and after implementing a partial waiver of consent. Overall enrollment increased significantly without creating a racial disparity in enrollment, thereby improving generalizability and efficiency in neonatal clinical research.

Antenatal corticosteroids are given to pregnant people at risk of preterm birth to reduce newborn morbidity, including respiratory distress syndrome. However, there has been concern surrounding potential adverse effects on subsequent generations. Animal studies have demonstrated endocrine and metabolic changes in those exposed to corticosteroids in utero (F1) and in the second generation (F2). We aimed to assess the effects of parental antenatal corticosteroid exposure on health of the second generation (F2) of Auckland Steroid Trial (AST) participants. In the AST, women (F0) expected to birth between 24 and 36 weeks’ gestation were randomised to betamethasone or placebo. When their children (F1) were 50 years old, they and their children (F2) were followed up with a self-report questionnaire and data linkage. The primary outcome for this analysis was body mass index (BMI) z-score in the F2 generation. Secondary outcomes included respiratory, cardiovascular, neurodevelopmental, mental and general health, and social outcomes. Of the 213 F2 participants, 144 had BMI data available. There was no difference in BMI z-score between participants whose parent was exposed to betamethasone versus placebo (mean (SD) 0.63 (1.45), N = 77 vs 0.41 (1.28), N = 67, adjusted mean difference (95% confidence interval) = 0.16 (-0.37, 0.69)). There was no evidence of a difference in rates of overweight, diabetes, respiratory disease, cardiometabolic risk factors, neurodevelopmental difficulties, mental health difficulties and social outcomes between parental betamethasone versus placebo exposure groups, but confidence intervals were wide. These findings are reassuring regarding the intergenerational safety of antenatal corticosteroids.

Medicinal cannabis has been trialled for Tourette syndrome in adults, but it has not been studied in adolescents. This open-label, single-arm trial study evaluated the feasibility, acceptability and signal of efficacy of medicinal cannabis in adolescents (12–18 years), using a Δ9-tetrahydrocannabinol:cannabidiol ratio of 10:15, with dose varying from 5 to 20 mg/day based on body weight and response. The study demonstrated feasibility of recruitment, acceptability of study procedures, potential benefits and a favourable safety profile, with no serious adverse events. Commonly reported adverse events were tiredness and drowsiness, followed by dry mouth. Statistically significant improvement was observed in parent and clinician reports on tics (paired t-test P = 0.003), and behavioural and emotional issues (paired t-test P = 0.048) and quality of life as reported by the parent and young person (paired t-test P = 0.027 and 0.032, respectively). A larger-scale, randomised controlled trial is needed to validate these findings.

Current evidence underscores a need to transform how we do clinical research, shifting from academic-driven priorities to co-led community partnership focused programs, accessible and relevant career pathway programs that expand opportunities for career development, and design of trainings and practices to develop cultural competence among research teams. Failures of equitable research translation contribute to health disparities. Drivers of this failed translation include lack of diversity in both researchers and participants, lack of alignment between research institutions and the communities they serve, and lack of attention to structural sources of inequity and drivers of mistrust for science and research. The Duke University Research Equity and Diversity Initiative (READI) is a program designed to better align clinical research programs with community health priorities through community engagement. Organized around three specific aims, READI-supported programs targeting increased workforce diversity, workforce training in community engagement and cultural competence, inclusive research engagement principles, and development of trustworthy partnerships.

Significant improvements have been achieved to enhance the patient-centricity of clinical research, including the development and utilization of novel clinical trial endpoints. These include endpoints that harness outcomes that are important to patients and reflect the patients’ lived experiences. This may take the form of utilizing variables such as days alive and out of hospital (DAOH) and quality-of-life adjusted outcomes. The use of composite outcomes can be used to enrich patient-centricity by weighting or ranking events. These approaches have several nuances that should be considered including selecting appropriate events, defining outcomes, how to elicit or construct weights, and whose opinions to consider. After weights have been determined, a variety of approaches exist to combine weights with outcomes and make comparisons between groups. The approaches, including the win ratio, weighted win ratio, desirability of outcome ranking (DOOR), multicriteria decision analysis (MCDA), and variations of time-to-first composite event analyses, have unique advantages and challenges depending on the clinical scenario. While improving patient-centric outcomes is of high importance to multiple stakeholders, more comparative work is needed to characterize the implications of alternative approaches.

The risk of losing access to crucial means-tested programs — like Medicaid, Supplemental Security Income (SSI), the Supplemental Nutrition Assistance Program (SNAP), and Temporary Assistance for Needy Families (TANF) — poses a barrier to the enrollment of low-income Americans in clinical trials. This burden likely disproportionately affects members of racial and ethnic minority groups, people with disabilities, elderly individuals, and rural populations, and may frustrate efforts to reflect the US population in clinical trial enrollment. To help achieve representative clinical trials for myriad conditions, Congress should pass legislation excluding payments to clinical trial participants from gross income and expand the clinical trial compensation exclusions for means-tested programs established in the Ensuring Access to Clinical Trials Act of 2015.

Conducting scientific medical research with human subjects presents risks that raise both ethical and human rights concerns. We argue in this article that applying a human rights framework to the problems that arise in the context of scientific medical research can contribute to a better understanding of the impact on individuals, the related obligations of the State, and the avenues to make the State accountable when things go wrong. We start our analysis with a case brought to the European Court of Human Rights, which we use as an illustration throughout the article. We then discuss the relevance of human rights to the field of scientific medical research with a focus on the right to life and the right to health. The article draws on international human rights jurisprudence that deals with concrete disputes arising from the clinical reality. We use case law to highlight the role of human rights law in tackling the real-life problems that may occur during scientific medical research. Our analysis contends that human rights law can provide valuable guidance for healthcare professionals and equip them to handle concrete situations in the clinical reality when the safety of research subjects is at stake.