Crossing the BBB for treating Parkinson’s
The biotech will receive up to $1m depending on the completion of various milestones in the development of its delivery system to bypass the blood-brain barrier (BBB).
Successful development of the “Trojan horse” delivery technology would allow for therapeutics to be delivered to the brain for the treatment of Parkinson’s disease.
Gene Johnson, chief scientific advisor to MJFF, said: "Trojan horse technology has intrigued Parkinson's researchers for years because of its theoretical potential to overcome the drug delivery hurdle, one of the single most significant challenges facing the entire field.
"It is fitting that the MJFF, which is searching urgently for treatments that will significantly modify Parkinson's disease, should provide funding to vet the potential of this technology to yield new therapies that could transform patients' lives."
The technology is being developed to deliver glial cell derived neurotrophic factor (GDNF), which has been shown to promote the survival of a range of neurons but cannot currently be adequately delivered to the affected regions.
ArmaGen is hoping to overcome this problem through the development of its “Trojan horse” technology, which works by fusing GDNF to a genetically engineered monoclonal antibody (mAb) that can cross the BBB using endogenous receptor mediated transport (RMT) systems.
RMTs transfer circulating peptides across the BBB, providing a system that researchers have used to deliver therapeutics to the brain.
The GDNF-fusion protein is described by ArmaGen as being a bi-functional molecule as it binds both with the transfer protein and to a specific neurotrophin receptor on brain cells to protect them from damage.
The MJFF’s funding is to be used to test the efficacy and toxicity of the GDNF-fusion protein on primates that have been induced with an experimental form of Parkinson’s.
It is hoped that this will form the basis for performing the work that will eventually lead to the submission of an investigational new drug (IND) application with the US Food and Drug Administration (FDA).
ArmaGen believes its technology could have applications in the treatment of a range of other diseases where crossing the BBB poses a problem, including Alzheimer’s, cancer and schizophrenia.
