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Biotech firm Oncopeptides has confirmed that its Peptide Drug Conjugate (PDC) platform successfully crosses the blood-brain barrier in glioblastoma patients — a milestone that has stumped cancer researchers for decades. Early data from three patients in the INSULA trial showed meaningful drug concentrations inside brain tumors and good tolerability. It's a promising first step toward effective systemic treatment for one of the deadliest brain cancers.
For decades, the blood-brain barrier (BBB) has been the ultimate gatekeeper — blocking more than 90% of conventional cancer drugs from ever reaching brain tumors. Now, Oncopeptides says its Peptide Drug Conjugate (PDC) platform has cleared that hurdle in actual patients.
Early data from the INSULA trial (OP-701), a Window-of-Opportunity study conducted at Oslo University Hospital, showed that the company's drug melflufen successfully penetrated the BBB in all three evaluated patients with recurrent glioblastoma. Intra-tumoral drug concentrations matched what was seen in preclinical models, and the treatment was well tolerated — with the first patient completing five monthly doses without major drug-related toxicity.
The study's design — administering the drug before scheduled tumor-removal surgery — allowed researchers to directly measure drug uptake in resected tissue, providing unusually direct proof of concept. Recruitment continues toward a target of ~10 patients.
By the Numbers:
90% of conventional systemic cancer drugs fail to cross the blood-brain barrier
Why it matters: Glioblastoma is the most aggressive primary brain cancer with virtually no effective systemic treatment options. Confirming BBB penetration with a cytotoxic payload in humans is a critical clinical proof-of-concept that could open the door to a new class of brain cancer therapies.