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Precision Meds Shown Effective in Rare Cancer

Cancer on clipboard

(Nick Youngson,

8 Sept. 2022. Results from a clinical trial show patients with rare bile duct tumors respond to an experimental treatment blocking a specific cancer-causing protein. A team from Relay Therapeutics Inc. in Cambridge, Massachusetts and participating medical centers are scheduled to present their findings on 11 Sept. at a meeting of the European Society of Medical Oncology or ESMO in Paris.

The researchers are reporting on a test of Relay Therapeutics’ cancer treatment candidate code-named RLY-4008 in patients with cholangiocarcinoma or CCA, a rare solid-tumor cancer affecting the bile ducts. Bile is a fluid made in the liver that aids in digestion by breaking down fats into fatty acids. Bile ducts are tubes connecting the liver to the gall bladder and small intestine for transferring bile. Most current treatments for CCA are surgery to remove localized cancerous bile ducts, radiation, and chemotherapy. According to American Cancer Society, some 8,000 people in the U.S. are diagnosed with CCA each year, but the disease is also prevalent in Southeast Asia, caused by a common parasite infection there.

Relay develops therapies for rare and difficult-to-treat diseases with a process the company says addresses the dynamic nature of proteins. Relay says its Dynamo technology discovers new drugs with computational methods that analyze movements and changes of proteins in the body, including chemical and physical structure of protein molecules, rather than focusing on proteins as static entities. In July 2020, Science & Enterprise reported on Relay Therapeutics’ initial public offering that raised $400 million.

Test varying dosage levels

RLY-4008 is an oral precision-medicine drug that seeks to limit the fibroblast growth factor receptor 2, or FGFR2 protein. While the FGFR2 protein helps promote bone growth, it is also implicated in a number of solid tumor cancers. According to the ESMO paper authors, earlier non-selective attempts to inhibit FGFR2 proteins resulted in resistance and off-target toxicities, as well as response rates of 20 to 40 percent. RLY-4008, say the authors, is more highly targeted and addresses resistance and off-target effects.

The early- and mid-stage clinical trial assesses the safety and efficacy of RLY-4008, starting with a test of varying dosage levels, from 20 to 200 milligrams, in patients with unremovable or metastatic CCA. The study team is looking mainly for adverse effects from RLY-4008 and maximum tolerated dose over 24 months, but also any clinical benefits to trial participants to the treatments. The study has no control or comparison group.

Results reported at ESMO show the most common adverse effects from the first 38 participants are low-grade mouth inflammation or sores and swelling or blistering on the hands or feet, in 46 to 48 percent, and dry mouth by 31 percent. None of the adverse effects are rated as severe. About two-thirds (63%) of the 38 participants receiving any quantity of RLY-4008 report at least some tumor shrinkage in response to the drug. Among the 17 participants receiving the 70 milligram dose, 15 or 88 percent report reductions in their tumors. One patient reports near complete response to the drug, allowing for surgical removal of the tumor.

The authors say their findings support continuing the clinical trial to its next stages, expanding the treatment to other types of solid tumors associated with FGFR2 proteins, and later to CCA patients not responding to previous chemotherapy. The study expects to eventually enroll 440 patients at 41 sites worldwide.

Hat tip: STAT

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