FF-10502-01 is well tolerated with manageable side effects, in patients with solid tumors, including cholangiocarcinoma, according to a new study published in the journal Cancer. The treatment also had limited hematologic toxicity. 

The study also found that in patients with cholangiocarcinoma who were heavily pretreated with gemcitabine, FF-10502-01 led to durable partial responses and disease stabilizations. 

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“FF-10502-01 . . . may represent an effective therapy,” wrote the study authors. 

To explore the safety, tolerability, and antitumor activity of FF-10502-01 in patients with solid tumors, including cholangiocarcinoma, a team of researchers, led by Gerald S. Falchook, MD, conducted a phase 1/2a clinical trial in patients with inoperable metastatic tumors not responding to standard therapies. 

The phase 1 portion of the trial enrolled 40 participants to determine the dose of the treatment to be used in the phase 2 portion. Then 66 patients, of which 36 had cholangiocarcinoma were enrolled in the phase 2 portion. 

The results showed that in patients treated with 90 mg/m2 of FF-10502-01 as determined in the phase 1 portion, common adverse events included grade 1–2 rash, pruritus, fever, and fatigue. There were also grade 3 and 4 hematologic toxicities, such as thrombocytopenia and neutropenia, but these were low in incidence. 

A total of 5 patients who had tumors refractory to gemcitabine treatment showed confirmed partial responses. 

In patients with cholangiocarcinoma, the median progression-free survival rate was 24.7 weeks, while the overall survival rate was 39.1 weeks. In these patients, prolonged progression-free survival was associated with mutations in the BAP1 and PBRM1 genes.

FF-10502-01 is a cytidine nucleoside analog, which is structurally similar to gemcitabine but with different biologic effects. It has shown promising activity in models of gemcitabine-resistant tumors both alone and in combination with cisplatin.


Janku F, Javle MM, Sen S, et al. A phase 1/2a safety, pharmacokinetics, and efficacy study of the novel nucleoside analog FF-10502-01 for the treatment of advanced solid tumors. Cancer. Published online March 7, 2023. doi:10.1002/cncr.34709