Unmet Need

  • Schizophrenia is a severe mental disorder with limited effective treatments due to medication resistance.
  • Deferiprone (iron chelator) shows antipsychotic-like activity in mouse models, but systemic exposure is undesired.
  • Current option is oral deferiprone (Ferriprox®) for iron overload; it can cause serious side effects including agranulocytosis.
  • Unmet need: a brain-directed therapy that minimizes peripheral exposure and side effects.

Our Innovation

  • Intranasal, nose-to-brain delivery of deferiprone alone or combined with antioxidants (N-acetylcysteine [NAC], vitamin E) using a nanocarrier with permeation-enhancing agents.
  • DESI-MS imaging to map spatial brain distribution and the unique metabolomics signature after intranasal vs intraperitoneal administration.
  • Rationale for combination: In preclinical neurodegenerative and psychiatric disorders associated with brain iron overload (e.g., Alzheimer’s and Parkinson’s disease), iron chelation with deferiprone alone may exacerbate oxidative stress by reducing glutathione, the brain’s principal endogenous antioxidant. Accordingly, combining deferiprone with antioxidants such as NAC and vitamin E has been proposed to better restore brain function and protect against iron toxicity; in schizophrenia models, the combination would be more effective than deferiprone alone.

Advantages

  • Direct brain delivery with nanocarrier aims to minimize peripheral exposure and related side effects.
  • Potentially enhanced efficacy via iron-chelation + antioxidant synergy (NAC, vitamin E with excellent safety profiles).
  • Non-invasive intranasal route tailored for CNS targeting using permeation enhancers.
  • Imaging-guided development (DESI-MSI) enables data-driven optimization of dose and formulation.

Commercial Opportunity

  • We are seeking collaboration with pharmaceutical companies focused on CNS (psychiatric/neurological disorders), companies with nanotechnology/drug-delivery expertise, and academic institutions for preclinical/clinical testing.

Contact in Yissum: Ariela Markel