NCI-2024-08329

A Phase 1 Study of ALN-BCAT as Monotherapy and in Combination With Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma

This is a Phase 1 research study evaluating the safety and dosing of a new drug, ALN-BCAT, for patients with advanced liver cancer (HCC), both when given alone and in combination with the approved immunotherapy, Pembrolizumab. ALN-BCAT is an RNAi therapeutic that acts as a targeted inhibitor. It works by silencing the gene for beta-catenin in cancer cells. This is crucial because an overactive beta-catenin pathway is known to drive cancer growth and make the tumor resistant to the body's immune system. Pembrolizumab is an immune checkpoint inhibitor (like Keytruda). It works by releasing the "brakes" (PD-1 checkpoint) on the patient's own immune T-cells, allowing them to detect and attack the cancer. The combination aims to achieve synergy: ALN-BCAT is intended to dismantle the tumor's defense (beta-catenin-driven resistance), making the cancer vulnerable, while Pembrolizumab then unleashes the immune system to mount a strong, effective attack.

For more information about the trial, click the link below:

https://clinicaltrials.gov/ct2/show/NCT06600321

Clinical Trial Site: Sinai

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