AI, omics, and systems biology can now help scientists design targeted drugs for cardiovascular disease pathways once thought “untreatable.”

A new article published in Frontiers in Science says these tools could transform heart drug development and save lives—but that global, equitable health policy leadership is urgently needed.

Despite major advances in cardiovascular care, heart disease remains the world’s leading cause of death. This is partly because cardiovascular medicine still largely relies on broad-brush treatments that don’t account for the enormous diversity within cardiovascular diseases (CVDs). Although these one-size-fits-all treatments—like statins—help many patients, they do not work for everyone.

The new review calls for a fundamental shift in how heart drugs are discovered, developed, and tested. Using AI, omics, and big data to identify and design drugs for disease-related genes and proteins, this “innovation paradigm” could drive the development of truly personalized treatments for CVD.

“CVDs are likely to claim 26 million deaths per year by 2030, up from 19 million in 2020,” said lead author Prof Masanori Aikawa, from Brigham and Women's Hospital and Harvard Medical School. “This approach could usher in new drugs that succeed where conventional ones have failed—but global health leadership is needed to drive that transformation and ensure it saves lives worldwide.”

One promising avenue is RNA-based therapeutics. Unlike conventional drugs, which reach only a small percentage of all potential protein targets, RNA therapies can be designed to influence almost any gene. They may also be quicker to develop, and early trials already show their potential for lowering cholesterol more effectively than standard treatments.

“This is an exciting time for cardiovascular medicine,” said Prof Aikawa. “RNA therapies are already opening the door to tackle disease pathways long considered ‘undruggable,’ and with strong global leadership, we can bring new precision medicines to patients faster and save lives from CVD.”

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Next-generation heart medicine

CVDs vary between patients in terms of their symptoms, underlying mechanisms, and how well they respond to treatment. Even patients with identical diagnoses present differently, which reflects the broad spectrum of our genes, environments, and lifestyles.

To better serve this variance, the authors say the new approach should harness such technologies as:

• omics approaches, which provide detailed information about molecules within cells, such as proteins (proteomics) and genes (genomics)

• systems biology, which looks at how networks of genes and proteins interact to shape disease

• AI, which can analyze disease pathways to identify new drug targets, and design new drugs targeted to specific proteins and genes.

The authors predict that, with sufficient investment and support, this innovation paradigm will give rise to a whole new generation of therapies—especially RNA-targeted and digitally designed drugs—that can intervene in disease pathways previously considered ‘undruggable.’ Such advances could dramatically reduce the time, cost, and failure rate involved in the development of cardiovascular drugs.

“There’s no single version of CVD, so to treat patients more effectively, we need bespoke medicines that reflect the broad spectrum of CVDs. This means harnessing technological advances to map the complex networks of genes, proteins, and pathways that differ between individuals—and identify how best to target them,” said senior-author Prof Joseph Loscalzo, from Brigham and Women's Hospital and Harvard Medical School. “This is how we make the ‘untreatable’ treatable: by spotting new drug targets within individual patients, and designing new molecules specifically for them.”

Bold investment; open science

But turning these tools into real treatments will take more than lab breakthroughs. The authors call for stronger collaboration between scientists, industry, and healthcare, alongside global policy leadership to ensure precision medicine reaches people everywhere.

“To save lives from CVD, we urgently need a new standard approach,” said co-author Dr Sarvesh Chelvanambi, from Brigham and Women's Hospital and Harvard Medical School. “That means bold investment, open science, and new partnerships across academia, industry, and healthcare. Right now, this paradigm isn’t in place—even in high-income countries. Global leadership is vital to mobilize the funding, resources, and policies that will make it a reality worldwide.”

The authors say that with the right leadership, precision medicine could finally change the course of the world’s leading killer.

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The article is part of the Frontiers in Science multimedia article hub ‘Transforming cardiovascular medicine with precision approaches.' The hub features an explainer, two editorials, two viewpoints, and a version of the article for kids, from other eminent experts: Dr Valentin Fuster, Prof Filip K. Swirski, and Dr Girish Nadkarni (Mount Sinai Health System, USA), Dr Siyeon Rhee and Prof Joseph C. Wu (Stanford University, USA), Prof Hugo ten Cate (Maastricht University, the Netherlands), and Prof Rainer Schulz (Justus-Liebig University Giessen, Germany).

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Frontiers in Science is Frontiers’ multidisciplinary, open-access journal focused on transformational science to accelerate solutions for healthy lives on a healthy planet.

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