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Carrier-mediated delivery of therapeutic proteins into the brain

Principal Investigator:
  • Roman Melnyk, The Hospital For Sick Children
Team Members:
  • Sheena Josselyn, The Hospital For Sick Children
  • James Ellis, The Hospital For Sick Children
  • Eyal Grunebaum, The Hospital For Sick Children
  • Berge Minassian, The Hospital For Sick Children
  • SickKids Foundation

Project Overview

It is estimated that diseases of the brain currently affect over 1.5 billion people worldwide and account for approximately a third of global disease burden. For many neurological diseases, such as Parkinson’s disease, Huntington’s disease, autism and epilepsy, the available treatments are focused on symptom relief, rather than a cure. From a therapy standpoint, treating brain disorders is complicated by the fact that the brain is shielded from the rest of the circulation by a biological barrier called the blood-brain-barrier. This barrier protects the brain from unwanted chemicals, but at the same time, prevents drugs from reaching their site of action in the brain. For this grant, Dr. Melnyk and his team are proposing the development of a safe and non-invasive approach to ferry drugs across the blood-brain-barrier, by attaching them to an established carrier molecule that naturally and safely enters the brain. This carrier has been injected into millions of children in its role as a carrier of bacterial components for numerous childhood vaccines. Through their re-engineering of this system, they will deliver curative doses of proteins to specific destinations in the brain to restore a missing functionality. This approach has broad applicability for many neurological diseases and in particular, has great potential to restore function in diseases where the genetic defect responsible for disease is known. They have assembled a multidisciplinary team of researchers at the Hospital for Sick Children to bring this innovative research endeavour all the way from the test-tube to the patient.