Understanding glioblastoma and the unmet need in oncology
Rare cancers account for approximately 20–24% of all cancer diagnoses and represent a major unmet need in oncology due to their biological complexity, limited clinical evidence, and restricted therapeutic options.
cases per 100,000 individuals per year (EU definition)
of all cancer diagnoses
he most common malignant primary brain tumor in adults
Glioblastoma Multiforme (GBM) is characterized by rapid proliferation, diffuse infiltration, and high molecular heterogeneity.
A key driver of its aggressiveness is the presence of cancer stem cells, which sustain tumor growth, promote therapeutic resistance, and drive disease recurrence.
Current standard of care – surgery, radiotherapy, and temozolomide -provides only limited benefit, and prognosis remains poor.
StemGen’s approach targets cancer stem cells through differentiation therapy – turning aggressive cancer stem cells into “normal” mature brain cells.
hrBMP4 has completed phase I in recurrent GBM and the final results, recently published (Bos E. et All Molecular Cancer 2023), showed that intratumor and peri-tumor administration of hrBMP4 by Convection Ehanced Delivery (CED) was safe and well tolerated. The pattern of tumor response and overall survival revealed promising evidence of efficacy, with two patients experiencing complete and durable tumor response and another patient who showed partial response. Overall median survival was clearly extended. Planning of confirmatory studies in Recurrent and Newly Diagnosed GBM is underway. The results are available in Referza Bos et al. 2023. ClinicalTrials.gov; Identifier: NCT02869243.