98% Of Drugs Never Make It To The Brain, But This Method Could Change That

Treating the brain isn’t like the rest of the body. Your blood-brain barrier shields it; filtering the blood to ensure nothing untoward makes it through. This protection is normally a good thing, but it becomes a problem if you want to deliver therapeutic drugs through it. This method could be a solution. 

Smuggling therapeutics

Many diseases like Parkinson’s and Alzheimer’s disease are extremely difficult to treat. Only very specific molecules can make it through the brain’s secure barrier, and most drugs don’t make the cut. This poses a challenge when you want to treat disease inside the brain, and so efforts have been focused on finding a way to overcome this. New research has now demonstrated a way of treating Parkinson’s disease with a surgical treatment that opens up a small route to bypass the barrier; essentially a smuggling hatch into your brain. 

Drugs can be administered through the nose and diffused through a mucosal graft (A, red) which is 1,000 times more porous than the normal blood-brain barrier. Credit: Garyfallia Pagonis and Benjamin S. Bleier, M.D.

“Although we are currently looking at neurodegenerative disease, there is potential for the technology to be expanded to psychiatric diseases, chronic pain, seizure disorders and many other conditions affecting the brain and nervous system down the road”

How did they do it?

One way of helping Parkinson’s patients is an injection of a molecule called GDNF (glial derived neurotrophic factor) directly into their brain. GDNF has proved effective in trials, but the delivery process has some complications and can be very unpleasant for the patient. In an effort to improve on this, scientists operated on mice and removed a small patch of the brain barrier through the nose. They then repaired this with something called a mucosal graft, which is far more permeable. In patients with a brain tumour this operation is already safe and well established; surgeons cut a window in the barrier, remove the tumour and then recreate the wall with the nasal lining. The new lining still effectively protects against infection. Because the procedure is already approved, this work could quickly move to human trials for Parkinson’s or other degenerative conditions. 

“Brain diseases are notoriously difficult to treat due to the natural protections the body builds against intrusion. Dr. Bleier’s group has identified a potential avenue to pass that barrier, and we look forward to the next stage of research. It is a platform that opens doors for new discovery and could enable drug development for an underserved population.”

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