New drug discovery program underway for fibrolamellar carcinoma

At the International Liver Cancer Association Annual Conference, Blueprint Medicines Corporation announced a new drug discovery program intended for patients with fibrolamellar carcinoma, according to a press release issued by the company.

“We disclosed some of our data on targeting [protein kinase cAMP-activated catalytic subunit alpha (PRKACA)] in cancer, including potent and selective small molecule inhibitors and evidence of pathway modulation in tumors in vivo,” Christoph Lengauer, PhD, chief scientific officer of Blueprint Medicines, told Healio.com/Hepatology. “Ongoing work is focused on non-clinical development and bringing PRKACA inhibitors to the clinic.”

FLC is a rare, disparate subtype of liver cancer affecting 1% of patients, usually impacting young adults.

“We became interested in [fibrolamellar carcinoma (FLC)] as a result of a computational biology effort investigating gene translocation and kinase fusion events in cancer. The PRKACA fusion is observed at a very high frequency — estimated at greater than 90% in FLC,” Klaus Hoeflich, PhD, senior director of biology of Blueprint Medicines, told Healio.com/Hepatology. “It is the only recurrent genetic event in fibrolamellar tumors, and PRKACA is considered to be the driver of the disease.”

Currently, surgery is the only option since there are no approved therapies.

“This is a tumor type of very high medical need and we are passionate about helping patients with FLC,” Lengauer told Healio.com/Hepatology. – by Janel Miller

Disclosure: Hoeflich and Lengauer are employed by Blueprint Medicines.