Breakthrough Cancer Therapy Halts Tumor Growth Without Harming Healthy Cells, Enters Human Trials

Scientists from the Francis Crick Institute and Vividion Therapeutics have unveiled a groundbreaking cancer therapy that effectively stops tumor growth without causing damage to healthy cells. This innovative approach, detailed in the journal Science on October 9, 2025, represents a significant advancement in precision oncology.

sciencedaily.com reported, The research team identified a novel compound designed to block a critical signal that drives cancer cell proliferation. Unlike previous treatments, this compound specifically targets the interaction between the cancer-driving RAS gene and the PI3K enzyme, a pathway often implicated in tumor development.

Crucially, the new therapy demonstrated remarkable efficacy in preclinical studies. In mice models with lung and breast tumors, the compound successfully halted tumor growth. Importantly, these animal studies showed no evidence of the harmful side effects, such as elevated blood sugar levels, commonly associated with earlier cancer drugs.

economictimes.com noted, This breakthrough addresses a long-standing challenge in cancer treatment: precisely targeting cancerous cells while preserving healthy tissue. Julian Downward, Principal Group Leader at the Francis Crick Institute, noted that previous attempts to disrupt RAS-driven signaling were often hampered by severe side effects.

The promising results have paved the way for the therapy to advance into human clinical trials. This critical next step will evaluate the compound's safety, tolerability, and preliminary efficacy in patients, marking a pivotal moment in its development.

bioengineer.org reported, Matt Patricelli, Chief Scientific Officer of Vividion Therapeutics, expressed optimism about the therapy's potential. He highlighted that by selectively blocking a key cancer growth signal while allowing healthy cell processes to continue, this discovery could make a real difference for patients.

The collaboration between the Francis Crick Institute, a leading biomedical research center, and Vividion Therapeutics, a biopharmaceutical company known for its innovative discovery technologies, underscores the power of combining academic research with industry expertise to tackle complex medical challenges.

• Targeting the RAS-PI3K Pathway: The core of this new therapy lies in its ability to precisely disrupt the interaction between the RAS oncogene and the PI3K enzyme. The RAS gene is mutated in approximately one in five cancers, acting as a cellular "on" switch that, when mutated, continuously signals cells to grow and divide uncontrollably. PI3K is a key downstream effector in this growth pathway. Previous attempts to inhibit PI3K broadly led to severe side effects like hyperglycemia because PI3K also plays a vital role in insulin signaling and blood sugar regulation.
• Mechanism of Selective Action: Researchers at Vividion Therapeutics identified small molecules that irreversibly bind to the surface of PI3K near the RAS binding site. This specific binding prevents RAS from interacting with PI3K, effectively shutting down the cancer growth signal. Crucially, the compound allows PI3K to maintain its interactions with other molecules, including those involved in the insulin pathway, thereby preserving normal cellular functions and avoiding metabolic toxicities.
• Preclinical Success and Broad Potential: In mouse models, the compound not only halted the growth of RAS-mutated lung tumors but also showed efficacy against HER2-driven breast cancers, which also engage with PI3K. This suggests the therapy could be effective across a broader spectrum of cancers beyond just those with RAS mutations. Furthermore, combining the new compound with other drugs targeting the same pathway resulted in stronger and longer-lasting tumor suppression.
• Vividion Therapeutics and Bayer's Role: Vividion Therapeutics, a wholly owned and independently operated subsidiary of Bayer AG since its acquisition in 2021, leverages a proprietary chemoproteomic screening technology to identify novel binding pockets on "undruggable" proteins. This platform was instrumental in discovering the selective compounds. Bayer's investment in Vividion highlights a strategic focus on expanding its oncology portfolio through innovative targeted therapies.
• Advancement to Human Trials: The investigational drug has now entered Phase 1 clinical trials. These initial trials are designed to assess the safety, tolerability, and pharmacokinetics of the compound in humans, as well as to gather preliminary data on its efficacy in patients with tumors harboring RAS or HER2 mutations. This is a critical step in translating promising laboratory findings into viable treatments for patients.
• Context of Targeted Therapies: This new therapy aligns with the growing field of targeted protein degradation (TPD) and precision oncology. TPD strategies, such as PROTACs (Proteolysis Targeting Chimeras), aim to selectively degrade disease-causing proteins rather than just inhibiting their activity, offering new ways to overcome drug resistance and target previously inaccessible proteins. This approach represents a shift towards more precise treatments that minimize harm to healthy cells.