It’s through discovery that happens during clinical trials and research that allows for new advancements in medicine to be made. Providing the standard of care of is not always enough. New treatment therapies, technology and care delivery techniques must be developed, tested and perfected in order to keep advancing health care.
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Currently happening at Emory is the first-in-man clinical trial, testing a newly developed drug for a rare genetic condition called mucopolysaccharidosis type II (MPS II) or Hunter syndrome.
Hunter syndrome is a hereditary disease in which a critical enzyme is either missing, or there is not enough of it. Without enough of this enzyme, long chains of sugar molecules do not break down properly in the body and accumulate in the organs and tissues and become toxic.
To treat most cases of Hunter syndrome, an enzyme replacement medication is used, but this regimen does not cross into the brain to treat the most severe cases of Hunter syndrome. In this particular trial, Emory researchers are testing whether a man-made enzyme fused with an antibody and injected into the bloodstream will deliver the enzyme therapy into the brain. This is the first time this medication combination has been tested in a human.
The video below features Emory patient, Chris Dutcher. “By testing this new medication for the first time in a human, my hope is to help children with severe Hunter syndrome,” says Dutcher, whose younger brother also has the same disorder.
Chris has the mild version of Hunter syndrome, but his symptoms are still quite serious and involve the lining of the brain and spinal cord, which are not treated by existing medications. He is the first patient to receive the new treatment therapy.