Date: Jul 25, 2014 Source: (
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David Radin, PhD, Principal Investigator
BioStrategies, LC
"Lectin-assisted transnasal delivery of corrective enzyme for GM1 gangliosidosis"
The grant is a two-year $80,000 milestone-based grant.
BioStrategies LC is a start-up biotechnology company located at Arkansas State University. The company develops and manufactures human and animal protein therapeutics in plant - based production systems. Dr. David Radin and Dr. Carole Cramer, the co-founders of BioStrategies, previously conducted breakthrough research which was funded by several NIH SBIR (Small Business Innovation Research) grants and which led to FDA approval of a treatment for Gaucher disease, a lysosomal storage disease. If they are successful in this project with GM1 gangliosidosis, this approach will apply to other similar diseases.
A summary of the proposal follows:
A major limitation to enzyme replacement therapies (ERTs), which are used to treat many lysosomal storage diseases, is that the blood brain barrier protects the central nervous system, preventing needed enzyme from reaching the brain. Much research has been done to find a way to get treatments to the brain, but there has been only limited success so far.
Dr. Radin's group has worked with an experimental approach to crossing the blood brain barrier, attempting to deliver enzyme into the brain through the nose. His team has experience working in transnasal delivery. They are currently working on a vaccine candidate using transnasal delivery and have previously worked on delivering an enzyme using this method.
This group has developed a new method to do this using a plant protein called RTB that has been shown to facilitate delivery through the lining of the nose. They then developed a fusion protein linking RTB to B-galactosidase, the enzyme deficient in GM1.
With support from NTSAD's Research Initiative, Dr. Radin's group aim to treat mice with GM1 with this new therapy, looking for evidence of B-galactosidase reaching the brain. They will also look to see if levels of stored GM1 decrease and if the mice have improvements in health.
Because the transnasal approach is new, they plan to compare mice treated by nasal delivery and mice treated by intravenous administration.
They believe that their new drug targeting/delivery technology has the potential to provide significant future advances in ERT drug delivery challenges and also in treating symptoms affecting brain function.
Like other Research Initiative grants, this grant will be paid over time, with payments contingent upon successfully completing research plan milestones every 6 months.
