Melanoma Research: A Model for Cancer Breakthroughs with Far-Reaching Impact

May is Melanoma Awareness Month, a global campaign to raise awareness about the deadliest form of skin cancer. Researchers at the Rosalind and Morris Goodman Cancer Institute (GCI) are turning to the challenges posed by this cancer as an opportunity, using melanoma to pioneer new understandings of cancer genetics, treatment, and immunology.

Breakthrough Therapies: A Paradigm Shift in Melanoma

Until recently, late-stage melanoma was considered an untreatable disease and patients typically survived less than a year after diagnosis. However, in the 2010s melanoma emerged as the proving ground for some of the most advanced therapies and scientific discoveries: targeted therapy and immunotherapy. Today, many late-stage melanoma cases can now be completely eliminated by immunotherapy – a class of cancer drugs which harness a patient’s own immune system to attack cancer.

The dramatic improvement in melanoma outcomes over just a few years has shown how breakthroughs in cancer research can transform a once-hopeless diagnosis into a treatable disease. Even more importantly, the success of precision medicine and immune-based therapies in melanoma is now shaping treatment approaches for many other types of cancer.

Extending the Reach of Immunotherapy

While immunotherapy can effectively treat late-stage melanoma and other cancer types, some patients don’t respond to these therapies for reasons that scientists don’t entirely understand. A recently published study led by GCI investigator Ian Watson has identified a mutational mechanism that may predispose melanoma patients to respond favorably to a type of immunotherapy. The underlying mutation is present in several other cancer types, which could stand to benefit from immune-based therapies.

Melanoma’s tendency to metastasize, or spread, to the brain makes it especially deadly - and especially valuable for studying immune interactions in this immune-privileged organ. In a study from the GCI, researchers Logan Walsh, Daniela Quail, and Peter Siegel, investigated the immune cells within tumors that had originated in the brain or metastasized from other tumor types including melanoma. Their research identified distinct immune environments depending on the tumor’s origin, helping to refine the use of immunotherapies in metastatic cancers.

Targeted Therapies and the Promise of Precision Medicine

Another reason melanoma stands as a success story in oncology is its role in advancing targeted therapy. Roughly 50% of melanomas share a common mutation, allowing researchers to develop highly specific drugs that attack only the cancerous cells, sparing healthy ones. Another study from Ian Watson’s team has taken these therapies further by demonstrating that these same drugs can also be used treat melanomas driven by other, previously untargetable mutations – mutations which drive other cancer types beyond melanoma. This kind of drug repurposing and pathway discovery is paving the way for precision medicine in cancers beyond melanoma.

A Blueprint for the Future of Cancer Care

While research is still needed to improve melanoma outcomes, successes in treating the disease have shaped modern cancer therapy and pushed the boundaries of what is possible. Researchers at the GCI are continuing to advance our understanding of melanoma therapies, progressing our quest for the #knowledgetocure.

 

The studies highlighted in this article can be found below.

Berry, D., Moldoveanu, D., Rajkumar, S., Lajoie, M., Lin, T., Tchelougou, D., ... & Watson, I. R. (2025). The NF1 tumor suppressor regulates PD-L1 and immune evasion in melanoma. Cell Reports, 44(3).

Karimi, E., Yu, M. W., Maritan, S. M., Perus, L. J., Rezanejad, M., Sorin, M., ... & Walsh, L. A. (2023). Single-cell spatial immune landscapes of primary and metastatic brain tumours. Nature, 614(7948), 555-563.

Rajkumar, S., Berry, D., Heney, K. A., Strong, C., Ramsay, L., Lajoie, M., ... & Watson, I. R. (2022). Melanomas with concurrent BRAF non-p. V600 and NF1 loss-of-function mutations are targetable by BRAF/MEK inhibitor combination therapy. Cell Reports, 39(1).