Individualized Systems Medicine Functional Profiling for Recurrent Glioblastoma

Individualized Systems Medicine Strategy for Targeting Cancer Stem Cells in Patients With Recurrent Glioblastoma (ISM-GBM)

A study to determine the feasibility and safety of individualized cancer stem cell targeted therapy based on high-throughput functional profiling of FDA/EMA-approved drugs in patients with GBM that has recurred or progressed following standards-of-care (RT, TMZ).

This protocol describes a prospective single-center phase 1 study to evaluate the feasibility and safety of a high-throughput drug sensitivity and resistance testing (HTS) platform of individualized cancer stem cells (CSC) to predict targeted therapies in patients with recurrence of GBM after standards-of-care. Secondary outcome include efficacy of drug treatment. The underlying hypotheses is that treatment of patients based on functional profiling og autologous CSCs using HTS a) is feasible within an acceptable time window for clinical translation, b) safely delay disease progression and c) increase survival. There are increasingly published literature that strongly support the importance of a targeting CSC to improve therapy and prevent tumor recurrence in GBM, as an additional strategy to improve the overall prognosis of patients.

Inclusion Criteria:

• Recurrence of histologically verified glioblastoma
• Adequate biopsy to generate enough live cells to allow functional screening
• Must be ambulatory with an Eastern Cooperative Oncology Group (ECOG) performance status 0-1
• Must be 18 to 70 years of age
• Adequate bone marrow, liver and heart function
• Must be competent to give consent
• Signed informed consent and expected cooperation of the patients for the treatment and follow up must be obtained and documented according to International Conference on Harmonization Good Clinical Practice guidelines (ICH GCP), and national/local regulations.

Exclusion Criteria:

• Patients taking part in other clinical trials which could make inclusion or follow-up difficult
• Any reason why, in the opinion of the investigator, the patient should not participate