Revolutionizing Liver Cancer Treatment with RN-005

ACHIEVE - Advancements in Cholesterol-targeted Hepatocellular Carcinoma: RN-005 as an Innovative and Effective approach

In this project, the University of Maastricht, Maastricht UMC, and Renatus Inc, aim to revolutionize hepatocellular carcinoma (HCC) treatment through the development of RN-005, a novel cyclodextrin compound. This newly established partnership aims to address the urgent need for more effective and less toxic therapies by exploring the therapeutic potential of RN-005, which selectively reduces cellular cholesterol accumulation.

HCC, the most common type of liver cancer, presents a significant challenge in oncology with a 5-year survival rate of ±20%. The current treatment modalities for HCC are limited by suboptimal efficacy and substantial side-effects, highlighting a critical need for more targeted, less toxic therapies. Moreover, HCC is associated with significant economic and healthcare burdens, with global liver cancer cases increasing.

Their project conceptualizes an innovative approach to combat HCC by leveraging RN-005’s ability to reduce cellular cholesterol accumulation. Unlike its predecessor, hydroxypropyl-beta-cyclodextrin (HPβCD), which has shown significant toxicity and inflammatory effects, RN-005 is engineered to selectively target intracellular cholesterol in cancer cells, reducing the risk of adverse effects. This project’s primary objective is to obtain preclinical evidence that RN-005-mediated depletion of cholesterol represents an effective intervention for curtailing HCC progression while concurrently augmenting the therapeutic effectiveness of existing treatments.

The project comprises three work packages: evaluating RN-005’s impact on tumor progression in vivo, its therapeutic efficacy in combination with conventional therapy in vivo, and its clinical value using patient-derived models. If successful, RN-005 could transform the HCC treatment landscape, offering a less toxic, more effective option and potentially extending its benefits to other cancers influenced by cholesterol metabolism. While immediate impacts on clinical practices may be incremental, the long-term potential of this research could lead to significant advancements in cancer treatment.

Summary
This consortium is exploring RN-005, a novel compound designed to target cellular cholesterol in liver cancer cells, aiming to reduce toxicity and improve treatment efficacy. Initial results are promising, showing that RN-005 can inhibit cancer cell growth without harming normal cells, hence more effective and safer therapies.
Technology Readiness Level (TRL)
1 - 4
Time period
36 months
Partners
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