The SUMMIT trial (NCT01953926), led by Ghassan K. Abou-Alfa, MD, a medical oncologist at Memorial Sloan Kettering Cancer Center, has brought new hope for the treatment of fibrolamellar carcinoma. The trial evaluated the use of neratinib (Zejula) both as a single agent and in combination with pembrolizumab (Keytruda), an anti-PD-1 therapy.
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Response Rates: The single-agent therapy showed a response rate of about 5% and a disease control rate of 13.3%. However, the combination therapy marked a significant milestone by achieving partial responses, a first for fibrolamellar carcinoma treatment.
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Patient Tolerance: Patients tolerated the therapy very well. Although grade 3 and 4 adverse events occurred in about two-thirds of the patients, mainly related to hyperglycemia, hepatic failure, anaphylaxis, the combination therapy did not introduce additional adverse events.
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Conclusion: The combination of neratinib with checkpoint inhibitors is deemed critical. While the added value of mTOR inhibition in combination remains uncertain, the trial represents a step in the right direction for treating this rare disease.
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Challenges: The rarity of fibrolamellar carcinoma limits sponsor interest, highlighting the importance of collaboration in advancing treatment options for young patients suffering from this terrible disease.
