Scientists from the Francis Crick Institute and Vividion Therapeutics have created a new class of compounds that can specifically block a key interaction between the cancer-associated protein RAS and the enzyme PI3K—a crucial driver of tumor growth. These compounds have now advanced into their first human clinical trial, marking a potential breakthrough in developing safer and more targeted cancer treatments.

The RAS gene is mutated in about 20% of all cancers. When this mutation occurs, it keeps the RAS protein in a permanently active state, constantly signaling cells to grow and divide uncontrollably. However, completely blocking RAS or its downstream pathways often harms healthy cells, since these same pathways are essential for normal functions like metabolism. PI3K, for example, also plays a vital role in regulating blood sugar through insulin signaling—so over-inhibition can lead to side effects like hyperglycemia.

Figure 1. Cancer-Fighting Drug.

To solve this problem, the researchers used a mix of chemical screening and biological testing to find compounds that could prevent RAS and PI3K from binding, without affecting PI3K’s other interactions. Scientists at Vividion Therapeutics discovered a set of small molecules that attach to PI3K near the RAS binding site, effectively blocking their interaction while preserving PI3K’s normal function in healthy cells. Figure 1 shows cancer-fighting drug.

When tested in mice with RAS-mutated lung tumors, one of these compounds successfully halted tumor growth without triggering hyperglycemia. Further experiments combining this drug with other treatments targeting the RAS pathway showed even stronger and longer-lasting tumor suppression than any of the drugs alone.

Encouraged by these results, the team extended their tests to other cancer types. In mice with HER2-mutated tumors—a mutation common in breast cancer—the new drug also suppressed tumor growth, even when RAS was not involved. This suggests the therapy could be effective against a broader range of cancers.

The compound has now entered its first human trial to evaluate safety, side effects, and potential effectiveness in patients with RAS or HER2 mutations.

Julian Downward, Principal Group Leader of the Oncogene Biology Laboratory at the Crick, said, “RAS mutations drive many cancers, but attempts to target this pathway have long been limited by toxicity [1]. Our collaboration has found a way around that by focusing on the specific RAS–PI3K interaction, keeping other essential PI3K functions intact. It’s exciting to see this work now move into clinical testing.”

Matt Patricelli, Ph.D., Chief Scientific Officer at Vividion Therapeutics, added, “This is a great example of how innovative discovery methods can uncover completely new ways to fight cancer. By selectively blocking RAS and PI3K from connecting while preserving healthy processes, we’ve designed a targeted approach that could benefit many patients. Seeing it progress to human trials is incredibly rewarding.”

References

1. https://scitechdaily.com/a-new-way-to-stop-cancer-growth-groundbreaking-drug-enters-human-trials/

Cite this article:

Keerthana S (2025), Groundbreaking Cancer-Fighting Drug Enters Human Trials, Offering a New Approach to Halting Tumor Growth, AnaTechMaz, pp.513