Reshaping rare cancer drug discovery

This paid-for advertorial by IRBM appeared in DDW Volume 24 – Issue 2, Spring 2023

The landscape for treating rare cancers is changing with advances in molecular genetics, novel drug modalities and patient-derived models. Dr Alessandro Carugo, IRBM, explains how building unique drug discovery strategies and expert alliances is reshaping the rare cancer drug ecosystem.

Why are rare cancers now getting the attention they deserve?
In the last decade we have seen a revolution in how tumours are diagnosed and classified, with molecular and genetic profiles revealing similarities between rare and more common forms of cancer. Such insights are helping drug hunters to design novel drug development strategies. We can bucket tumour subtypes together to identify new tractable targets and design stratified, biomarker associated trials that can include patients with rare cancer phenotypes. Patient advocacy groups are helping too by building awareness and improving access to novel trials and treatments. Such advances are encouraging new pharmaceutical investment into the rare cancer space. All this makes the prospect of new and effective treatments for rare and neglected cancers a realistic possibility and will improve outcomes for thousands of patients who until now, had few treatment options.

How can we reshape drug discovery in rare cancers to keep up the pace?
As drug hunters, we are finally changing our mindset and considering that no cancer is ‘rare’. Now, organisations that are at the forefront of cancer drug discovery and development may need to rethink their traditional approach and adapt integrated discovery platforms to target any cancer type. Whether it’s a rare cancer, an uncommon tumour subtype with an associated biomarker,or an unprecedented genetic alteration, it’s our capabilities and know-how that allow us to build bespoke cancer drug discovery programs. The chances of success are further enhanced by bringing together a wide network of industry and academic leaders who can add value to our drug discovery strategies by providing unique patient material, or their expert opinion on a specific rare cancer.

Which innovations are driving progress in the rare cancer space?
Limited patient material has always been an issue, but now, with sophisticated, patient- derived cell and organ models, we can guide the progression of novel therapeutic leads. High- content screening capabilities and spatial imaging technologies are also accelerating progress by providing intricate and comprehensive insights at unparalleled resolution. However, the most significant advancement is the large armamentarium of drug modalities we can now deploy; from small molecules to monoclonal antibodies, and from PROTACs to peptide-based theranostics, these innovations are enabling us to develop ambitious drug discovery strategies to address almost any therapeutic need.

What’s next for rare cancer drug discovery?
With rare cancers, integrated drug discovery programmes that incorporate different drug modalities will remain essential but can only be part of the equation. It is our capability to build strategic alliances that include all interested parties that will truly reshape the traditional way of developing therapeutic agents. This collaborative ecosystem is already transforming the drug development pipeline and will bring rare cancer treatments into the mainstream, ensuring better outcomes for all cancer patients.


Dr Alessandro CarugoDr Alessandro Carugo is Director of Oncology at IRBM. He is an experienced drug hunter and has led teams in both industrial and academic settings to identify and progress multiple therapeutic agents for rare and neglected tumours. Carugo holds a PhD in Molecular Oncology from the European School of Molecular Medicine (SEMM), and a MS (Hons) in Medical Biotechnology from the University of Milan-Bicocca, Italy.

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