Researchers in NRW Discover a Substance That Could Revolutionize Cancer Treatment! A Combination of Existing Drugs and a Novel Autophagy Inducer

The endpoint and "now" as told by primary information

The university's press release (August 5) and the announcement by the West German Tumor Center (WTZ) (August 7) clearly state the flow from mechanism identification→synergistic effect of combination→patent application (WO2025056601). The intention to aim for clinical trials is also specifically mentioned. Local media (August 14) succinctly reported the research team's stance of **"already obtained a patent, want to proceed to clinical trials."**uni-due.deWestdeutsches Tumorzentrum Essen (WTZ)ruhr24.de

Why BAP1 is important: A "common factor" across diseases

BAP1 mutations are frequently observed in uveal melanoma, renal cell carcinoma, cholangiocarcinoma, and pleural mesothelioma, and are known to be strongly associated with metastasis risk and poor prognosis. Its impact on immune environment and treatment responsiveness is also suggested, increasing its value as a biomarker. The strength of the current proposal lies in its placement in the context of "cross-disease" precision medicine.PMC

SNS reactions: Expanding through the author's own dissemination

On X (formerly Twitter), Samuel Peña-Llopis, one of the lead researchers, posted that **"BAP1 deficiency suppresses autophagy via SRC→BECN1. Therefore, the combination of SRC inhibitors and autophagy inducers opens the way."** The research introduction account of Taylor & Francis also spread the word, attracting attention primarily among the medical and research communities. Posts from the lab account and co-authors followed, sharing the message that "clinical trials are premised on patient selection (BAP1 testing)."

Key points of reactions (summary):
・"The mechanism is clear and the reproduction system is multi-layered. Showing organoids is reassuring" (Researcher)
・"Repurposing existing drugs could shorten development time" (Clinician)
・"Safety and optimal dosage of autophagy inducers need careful examination" (Pharmacology/Translational Researcher)
(The above is a summary of the gist of each post and not a verbatim quote)

However, "the real challenge starts now": Points to note and the next steps

• Not yet clinically tested: Efficacy and safety in humans are unknown. Particularly, autophagy induction can work positively or negatively depending on the cellular context, so optimization of dosage and timing is essential.PubMed

• Biomarker linkage: Accurate determination of BAP1 deficiency/low expression is a prerequisite for treatment selection. Standardization of pathology/genome testing is key.PMC

• Verification of expanded applicability: While there is potential for application beyond UM and ccRCC (such as cholangiocarcinoma, mesothelioma), differences in tumor microenvironment for each tumor type should be noted.PMC

• Intellectual property and development: **Patent for combination strategy (WO2025056601)** has been obtained. The next step is to build a collaborative research/clinical trial system.uni-due.deWestdeutsches Tumorzentrum Essen (WTZ)

From Local to Global: NRW's "Bridge-Building" Power

The driving force behind this achievement is the strong ecosystem of translational (bridge-building) research centered around the university's medical school and the West German Tumor Center (WTZ). Networks like NCT West and DKTK, along with support from EU MSCA and DFG, are narrowing the gap between research and clinical practice. Regional reporting conveys this "driving force" to the general reader, while the global expert community delves into specifics through papers and social media—a multi-layered information cycle is pushing the research to the next step.Westdeutsches Tumorzentrum Essen (WTZ)journalonko.deruhr24.de