Johns Hopkins’ Phoenix
In a society full of “what’s new must be better” attitudes, few “failed” technologies get a second chance. When a drug company, for example, discontinues work on a compound because it has failed its intended therapeutic target, the research rarely gets any attention. Once in a while, in science especially, revisiting these failed compounds turns out to be rewarding after all. Such was the experience of a team of Johns Hopkins medical scientists and an antibiotic formulated more than 100 years ago.
The original intent of clofazimine, synthesized in the 1890s, was to treat tuberculosis. After the compound failed, it was found later to be effective as a treatment for leprosy and achieved FDA approval. Now, efforts by the Johns Hopkins team has led them to discover the drug may be a potential therapy for multiple sclerosis, psoriasis, and type 1 diabetes.
“People have been working for years and spending tens of millions of dollars on developing a drug to inhibit a specific molecular target [known as the Kv1.3 postassium channel] involved in these diseases, and here, we have a safe, known drug that hits that target,” said Jun O. Liu in a prepared release.
The team searched through a listing of FDA-approved drugs and the John Hopkins Drug Library, which contained more than 3000 drugs in pharmacies or in Phase II trials. The drugs were tested one at a time against cells designed to mimic the immune system, after comparing the result of 200 hits, they found clofazimine had the highest inhibitor activity.
Of course the result of this work is not typical, but the discovery is inspiring at least, especially while the research and development sector is undergoing setbacks and increased market pressures.