MRI scans over time of a patient with partial response.
glioblastoma, tumour, distal bile duct adenocarcinoma
SOLID TUMOUR, LUNG METASTASIS, LUNG ADENOCARCINOMA, MESOTHELIOMA, TUMOUR, REGRESSION, GLIOBLASTOMA, COLON ADENOCARCINOMA, PANCREATIC ADENOCARCINOMA
The patient showed a sustained tumour regression (partial response according to RANO criteria) over time for 3 years on monotherapy with 2-OHOA (500 mg BID, cohort 2). MRI transverse (upper scans) and sagittal (lower scans) brain images show the glioblastoma (red circle) reduction, which was determined to be ca. −93% of the initial volume.Twenty-four patients with other advanced solid tumours were included in the intended-to-treat population. Although no patients achieved a PR, five of those 24 patients (20.8%; 1 mesothelioma, 1 lung metastasis of distal bile duct adenocarcinoma, 1 pancreatic adenocarcinoma, 1 metastatic lung adenocarcinoma, and 1 colon adenocarcinoma) had at least 1 assessment of SD, 3 of which lasted for 3 months or more (Fig. Therapeutic activity had been shown at low doses of 1 g/day while MTD was established at 12 g/day which is a wide therapeutic window that point out the potential of the drug and its safety profile.A Kaplan–Meier plot of PFS in the ITT is shown in Supplemental Fig. 