One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part of the body. Not only does delivery to the correct tissue ensure the effectiveness of the treatment, but delivery to the wrong tissue can be detrimental for the patient. A drug administered to the wrong part of the body can often cause harmful side effects. It’s clear this is an area that necessitates further research.
That said, nanoscale therapeutics do still hold great potential as a treatment option for many rare conditions such as cystic fibrosis and Muscular Dystrophy. It’s clear this type of therapy could be life-changing, and researchers have already spent years of work establishing it. Now we just need to figure out how to get it to patients safely.
Dr. Tilton’s Solution
Doctor John Tilton is the Director of Immunobiology in the CWRU School of Medicine. He has developed a way to deliver nanoscale therapies to specific areas of the body. He calls the product a NanoPOD.
NanoPODs utilize viruses, which are often co-opted by scientists for use as a vehicle to administer treatments. They’re effective because they already have expertise in penetrating cells. However, Dr. Tilton’s research focuses on a specific type of cell. He wants to solve the issue of therapy delivery for diseases that occur as a result of genetic mutations.
Ten percent of all genetic conditions are caused by a mutation which prematurely stops protein synthesis. This mutation is a result of the introduction of a “termination codon.” Cystic fibrosis is just one of the many rare conditions caused by this mutation. Dr. Tilton believes that delivery of treatment through a nanoPOD could effectively reduce the effects of this mutation.
This research has thankfully just received financial support from the Marian Falk Medical Research Trust in the amount of 300,000 dollars. This money will support Dr. Tilton’s research for one year.
His first project with the nanoPODS will be an investigation in mice. He will work to package therapies into the pods and then deliver them to precise tissues in mice who have a genetic condition.
Overall, nanoscale therapeutics are still in very early stages. However, research has shown tremendous promise for RNA therapeutics as well as protein therapeutics as a treatment for various autoimmune diseases and cancers. Researchers can only predict the impact these therapies will have in the future for many rare conditions.
NanoPODs could potentially be used to package a wide variety of therapies which have yet to be used in patients due to a lack of an effective delivery method. If Dr. Tilton’s research in mice models shows safety and efficacy, we could see a much faster progression of the delivery of these innovative new therapies to the patients who need them most.
You can read more about Dr.Tilton’s investigation of nanoPODs here.