One Final Gift and One Urgent Challenge: Advancing Research on Rare Cancers

Jerry Pelletier’s Legacy: How a Lifetime of Science and One Final Gift Are Advancing Precision Cancer Care for Rare and Hard-to-Treat Cancers

Ahead of World Cancer Day, we are reminded that progress against cancer is driven not only by discovery, but by extraordinary acts of generosity.

Jerry Pelletier—a respected researcher, mentor, and colleague—passed away from cancer in 2023. In the face of his own diagnosis, Jerry made a decision that continues to shape cancer research today: he donated his tumour to science.

Since then, Jerry’s tumour has been studied at the Rosalind & Morris Goodman Cancer Institute (GCI) and across McGill University by former colleagues and collaborators. This work has generated critical insights into the biology of the rare cancer he faced. Insights that have helped advance new approaches in precision oncology and deepened understanding of cancers that remain poorly characterized and difficult to treat.

Jerry’s contribution did not end with that gift. In the years since his passing, his science, his values, and the urgency of his experience have continued to shape how researchers at McGill approach some of cancer’s most challenging problems, particularly rare cancers and other aggressive, hard-to-treat diseases, where options are limited and time matters.

From fundamental science to collective momentum

Throughout his career, Jerry Pelletier made foundational contributions to our understanding of RNA biology and cancer. His work spanned multiple domains, from the discovery of internal ribosome entry sites (IRESs) and advances in mRNA translation, to uncovering the genetic basis of Denys–Drash syndrome and identifying deregulated translational control as a vulnerability in cancer.

These contributions were highlighted during a 2024 symposium at McGill University commemorating Jerry’s legacy, where colleagues traced how his research consistently moved from defining core biological mechanisms to identifying opportunities for therapeutic intervention. This included early efforts to target components of the translational machinery and the development of compounds aimed at inhibiting eIF4A, a key regulator of protein synthesis in cancer cells.

Speakers also noted Jerry’s role beyond individual projects. In addition to his academic work, he co-founded a biotechnology company, helped introduce emerging technologies such as CRISPR-based genome editing to McGill, and actively engaged in shaping larger research efforts.

That same perspective informed Jerry’s engagement with larger research programs. According to colleagues, he actively supported and advocated for the vision behind DNA to RNA (D2R), a major McGill-led initiative aimed at accelerating the translation of RNA-based discoveries into clinical impact.

“Jerry believed deeply in the concept of what became D2R” says Philippe Gros, Chief Scientific Officer, D2R. “He was consistently making the case that strong, curiosity-driven RNA science needed to be paired with the infrastructure to translate discoveries into impact. That perspective helped shape how D2R was conceived.”

Today, D2R stands as a $164-million research initiative, reflecting a model of discovery and translation that Jerry helped inspire through his work, his advocacy, and his engagement with the broader scientific community.

Addressing rare cancers through a DNA to RNA (D2R) funded project

Following Jerry’s passing, a team led by the GCI’s Morag Park and D2R Scientific Director Mark Lathrop, along with many collaborators, received a $3-million D2R translational grant for what is formally known as the Jerry Pelletier Initiative: Diagnostic and Therapeutic Innovations for Rare Cancers, named in his memory.

Jerry succumbed to a rare urothelial cancer, part of a broader category of rare cancers defined by the U.S. National Cancer Institute as affecting fewer than 15 people per 100,000. Individually uncommon but collectively significant, rare cancers account for a substantial number of cancer deaths, often because their underlying biology is poorly understood and effective treatment strategies are lacking.

Rare genitourinary (GU) cancers, in particular, have long faced this challenge. Compared with more common GU tumours—such as clear-cell or papillary renal cell carcinoma—rare subtypes suffer from limited molecular characterization and few targeted therapeutic options. As a result, patients are frequently treated with standard therapies that offer modest and short-lived benefit.

Part of the funded translational project seeks to address this unmet need directly. By focusing in part on understudied GU cancers, including rare urothelial tumours, the project aims to uncover actionable genomic vulnerabilities and advance more precise, personalized therapeutic strategies.

One early opportunity enabled by the initiative is the completion of the molecular characterization and drug sensitivity profiling of Jerry’s own tumour—work that was not possible within the timeframe of his illness.

“Jerry’s experience underscored the challenges of rare cancers, but it also showed what can happen when a community comes together around a shared goal,” says Professor Morag Park. “This project grew out of that moment—from the recognition that we needed to move faster and work more collectively to make a difference.”

IMPACT: turning urgency into infrastructure

Jerry’s experience also highlighted something else: what can happen when researchers move quickly, and what is lost when they cannot.

As his disease progressed, colleagues across the GCI and McGill University rallied. Experts in genomics, precision medicine, immunology, and preclinical model development worked together, combining their expertise in an effort to better understand his tumour and identify potential vulnerabilities.

“By the time the disease had metastasized, the broader research community came together,” says Professor Sidong Huang. “We did everything we could and showed that the science was possible—but we simply did not have enough time.”

That experience helped catalyze the creation of the Integrated Molecular Platform for Accelerating Cancer and Immune Therapeutics (IMPACT), a soon to be established facility at the GCI led by Professors Morag Park and Heather Melichar.

IMPACT is designed to rapidly integrate patient-derived preclinical models, multi-omic data, functional genomics, and therapeutic response profiling to identify cancer vulnerabilities and inform treatment strategies. The goal is speed with rigor, providing clinically relevant insights within timelines that matter to patients.

At its heart, IMPACT represents sustainability: ensuring that future patients with rare and aggressive cancers can benefit from a coordinated, rapid-response research pipeline, something Jerry himself did not have access to.

Continuing the science: MG-002 and hard-to-treat cancers

Jerry’s legacy also continues through the science he helped initiate. Throughout his career, he believed that understanding cancer at a molecular level should ultimately lead to therapeutic innovation.

One such effort is MG-002, a second-generation inhibitor targeting dysregulated translation initiation in cancer. Developed through Jerry’s work at McGill, MG-002 reflects his long-standing interest in compound screening and drug development. True to character, he named the compound after the institution rather than himself.

Today, MG-002 is being further advanced by Professors Josie Ursini-Siegel, Sidong Huang and Ivan Topisirovic, through a CIHR-funded research project. This work focuses on metastatic breast cancer, a hard-to-treat cancer, exploring whether MG-002 can selectively disrupt cancer cell survival and reduce tumour spread in preclinical models.

While this research is not specific to rare cancers, it addresses a closely related challenge shared by many rare and aggressive diseases: resistance to standard therapies and an urgent need for new treatment strategies.

Colleagues note that even as his health declined, Jerry remained committed to ensuring this work moved forward—seeing the grant process through so the science could continue.

A full circle in rare cancer research

Early in his career, Jerry made a landmark contribution to cancer genetics. In 1991, his research demonstrated that mutations in the WT1 gene could disrupt normal kidney and genitourinary development while also driving childhood cancer—helping establish the modern understanding of inherited cancer risk and the developmental origins of cancer.

In many ways, his legacy has come full circle. A scientist who helped illuminate the biology of a rare pediatric cancer would later inspire a research ecosystem focused on improving outcomes for patients with rare and hard-to-treat cancers more broadly.

For World Cancer Day, we honour Jerry Pelletier not only for his scientific achievements, but for the generosity, urgency, and collaborative spirit that continue to shape cancer research today—bringing us closer to better outcomes for patients who are too often left behind or who too often have far too little time.