UAE researchers have achieved a remarkable feat in the fight against one of the most aggressive forms of cancer: glioblastoma, a type of brain tumor notorious for its rapid growth and resistance to conventional treatments. In a development that underscores the nation's burgeoning prowess in artificial intelligence applied to healthcare, scientists have leveraged generative AI to identify and refine a promising drug candidate known as ISM0387. This compound, targeting the PRMT5 enzyme through a mechanism called synthetic lethality in tumors lacking the MTAP gene, was brought from concept to preclinical nomination in less than 12 months—a timeline that compresses what traditionally takes over a decade and billions of dollars into a fraction of the time.

The innovation stems from a collaboration between the Emirates Drug Establishment (EDE), the UAE's regulatory body for pharmaceuticals, and Insilico Medicine, a global biotech firm with a significant presence in Abu Dhabi. Using Insilico's Chemistry42 platform, which integrates more than 40 generative AI models, the team analyzed vast datasets of biological and chemical information to generate over 90 potential molecules. These were virtually screened, optimized, and tested in preclinical models, demonstrating dose-dependent tumor suppression and the critical ability to cross the blood-brain barrier—a major hurdle in brain cancer therapy.

This breakthrough not only highlights the speed of AI-driven discovery but also positions the UAE as a pioneer in AI-powered biotechnology, fostering an ecosystem where academia, industry, and government converge to accelerate medical advancements.

The Challenge of Glioblastoma and Why AI Matters

Glioblastoma multiforme (GBM), the most common and lethal primary brain tumor in adults, affects approximately 3 in 100,000 people annually worldwide, with median survival times hovering around 15 months even with aggressive multimodal therapy including surgery, radiation, and chemotherapy. In the UAE, cancer incidence is rising, with brain and central nervous system tumors accounting for about 2% of all new cases, according to recent health ministry data. The blood-brain barrier, a protective layer that prevents many drugs from reaching the tumor site, exacerbates the problem, leading to high recurrence rates—over 90% within two years.

Traditional drug discovery relies on trial-and-error screening of millions of compounds, a process plagued by high failure rates (over 90%) and exorbitant costs. Enter artificial intelligence (AI), particularly generative models trained on massive datasets of molecular structures, protein interactions, and clinical outcomes. These systems predict drug-target interactions with unprecedented accuracy, enabling rapid iteration from hypothesis to candidate.

In the UAE context, this aligns with national strategies like the UAE Centennial 2071, which emphasizes knowledge-based economy and advanced tech integration in healthcare. Universities play a pivotal role here, training AI specialists who fuel such innovations.

ISM0387: From AI Design to Preclinical Promise

The journey of ISM0387 exemplifies AI's transformative power. Starting with identification of the PRMT5 target—crucial in MTAP-deleted cancers common in GBM—the Chemistry42 platform generated novel molecular structures. Reinforcement learning refined these for optimal binding affinity, pharmacokinetics, and safety profiles. Within six months, 90 molecules were prioritized, leading to ISM0387's selection after in vitro validation showing superior activity over existing chemotherapies by more than 60% in select cell lines.

Preclinical studies in animal models confirmed its efficacy: at 30 mg/kg doses over 20 days, it significantly reduced tumor growth without notable toxicity. Its ability to penetrate the blood-brain barrier positions it as a game-changer for CNS malignancies.

Dr. Alex Aliper, co-founder of Insilico Medicine, noted, “This achievement marks a turning point in global drug development,” emphasizing AI's role in overcoming longstanding bottlenecks.

UAE Universities Fueling the AI Revolution in Oncology

While the ISM0387 project is industry-led, UAE's higher education institutions are the backbone of this ecosystem. Mohamed bin Zayed University of Artificial Intelligence (MBZUAI) in Abu Dhabi, the world's first graduate research university dedicated solely to AI, has been instrumental. MBZUAI researchers have pioneered AI models for cancer prognosis, such as Human-in-the-Loop for Prognosis (HuLP), which integrates physician expertise with deep learning to predict head and neck cancer survival more accurately than standard methods.

At Khalifa University, faculty in biomedical engineering and AI labs are developing tools for precision oncology, including AI-enhanced imaging for tumor segmentation and response prediction. Their work on multi-omics integration complements drug discovery efforts like ISM0387.

New York University Abu Dhabi (NYUAD) contributes with smart MRI molecules that activate in tumor microenvironments, lighting up glioblastomas on scans while releasing therapeutic payloads—dual detection and treatment in one. Led by researchers like Mostafa El-Sayed, this innovation directly addresses brain tumor challenges.

These universities collaborate with EDE and companies like Insilico through initiatives like the UAE Research Program for Innovative Science and Technology (UIST), training PhD students in AI-health intersections. MBZUAI alone graduates hundreds of AI experts annually, many entering oncology research.

Step-by-Step: How AI Accelerates Drug Discovery

• Target Identification: AI scans genomic databases to pinpoint vulnerabilities like MTAP deletion in GBM cells.
• Molecule Generation: Generative models create billions of virtual compounds, prioritizing those with desired properties.
• Virtual Screening: Physics-based simulations predict binding and ADMET (absorption, distribution, metabolism, excretion, toxicity) profiles.
• Optimization: Reinforcement learning iterates designs for better efficacy and safety.
• Preclinical Validation: Synthesized candidates tested in cell lines and animal models, confirming ISM0387's promise.

This pipeline slashed the hit-to-lead phase from years to months, a model UAE universities are replicating in labs.

Photo by Markus Winkler on Unsplash

Stakeholder Perspectives: From Labs to Clinics

Dr. Fatima Al Kaabi, EDE Director General, highlighted, “This demonstrates UAE's maturity in translating research into applications.” Academics echo this: Prof. Timothy Baldwin at MBZUAI stresses AI's role in “democratizing drug discovery for underserved cancers like GBM.”

Patients and oncologists in UAE hospitals like Cleveland Clinic Abu Dhabi and Tawam see hope: early data suggests ISM0387 could extend survival beyond current standards. Multi-perspective views include ethicists at UAE University advocating for equitable access, ensuring AI benefits all demographics.

Challenges remain—clinical trials needed, regulatory hurdles—but UAE's fast-track approvals position it ahead.

Statistics and Real-World Context in UAE

Brain tumors represent 1.5-2% of UAE cancers, with GBM comprising 50% of malignant cases. Incidence rises 2-3% yearly, linked to aging population. Traditional therapies fail 90% long-term; AI promises 20-30% survival gains per models.

UAE invests AED 100B+ in R&D, with 20% health-focused. MBZUAI's 500+ AI grads/year bolster this; Khalifa's cancer center integrates AI diagnostics, reducing scan times 40%.

Implications for UAE Higher Education and Research Careers

This breakthrough elevates UAE universities globally. MBZUAI's focus on generative AI for biology attracts top talent; Khalifa's interdisciplinary programs yield patents yearly. NYUAD bridges US-UAE research, fostering exchanges.

Opportunities abound: PhD positions in computational oncology, postdocs in AI-drug design. UAE's visa reforms ease talent inflow; salaries competitive (AED 20K+/month entry AI research). Programs like NAFIS mandate Emiratization, prioritizing nationals.

Future: Clinical trials 2027, potential Phase I by 2028. UAE aims 10 AI drugs by 2031.

Challenges, Solutions, and Ethical Considerations

• Challenge: Data Bias Solution: UAEU diverse datasets ensure inclusive AI.
• Regulatory Speed EDE's adaptive frameworks fast-track AI drugs.
• Ethics MBZUAI ethics board mandates transparency in AI decisions.

Balanced view: AI complements, not replaces, human insight.

Global Impact and UAE's Leadership

UAE joins elite like US, China in AI pharma. Partnerships with Insilico expand to MENA. For brain tumors, ISM0387 could set new standards; universities train next gen.

Khaleej Times on the breakthrough details the excitement.

Outlook: By 2030, AI halves UAE cancer mortality via precision therapies.

Photo by KOMMERS on Unsplash

Actionable Insights for Researchers and Students

• Join MBZUAI's MSc/PhD in AI for Health.
• Collaborate via Khalifa's innovation hubs.
• Explore NYUAD interdisciplinary projects.

UAE's vision: World-class research powering human health.