Clinical validation of NANO-PL: a hydrogel-based formulation of a small molecule for a highly targeted therapy against Glioblastoma Multiforme (GBM)

Glioblastoma (GBM) is considered the highest unmet need in oncology. The average life expectancy of GBM patients is 12-18 months, and the 5-year survival rates remain below 7%. Despite the efforts, the available therapy today counts only with a few (ineffective) approved therapies.Using a machine learning algorithm, we have discovered a correlation between a small molecule and an unexplored target in GBM. We have developed a hydrogel-based formulation of the molecule with great safety and efficacy in vivo. Our formulation showed 40% long-term survival in a GBM rat model, with complete tumour eradication after a single dose treatment. NANO-PL is a one-time treatment, applied locally during surgery, which is effective even in GBM cells that are resistant to the standard of care or those involved in recurrence. NANO-PL also showed a synergistic efficacy with temozolomide, standard-of-care. The EIC will enable the clinical validation of NANO-PL, completely unlocking the GBM treatment.