One protein inhibitor could treat Chagas, leishmaniasis and sleeping sickness

An article by FierceBiotech
August 9, 2016

Wellcome Trust-backed scientists have found a compound that treats three neglected parasitic diseases in mice: leishmaniasis, Chagas disease and sleeping sickness.

The trio of diseases affects millions of people in Latin America, Asia and Africa, yet current treatments are limited and have a number of side effects. The Wellcome-funded team from the Novartis Research Foundation’s Genomics Institute (GNF) focused on the genetic and biological similarities between the “kinetoplastids,” single-celled parasites that cause the diseases.

Because of these similarities, the researchers sought a compound that would be effective against all three diseases. After testing more than 3 million chemicals, they landed on a compound dubbed GNF6702, which destroyed the parasites in mice and did not harm human cells in lab tests, according to a statement. The drug works by selectively inhibiting a protein complex inside kinetoplastids called the proteasome. It does not harm the mice as it does not inhibit the mammalian proteasome.

"We found that these parasites harbour a common weakness,” said GNF’s Frantisek Supek, a research investigator and senior author, in the statement. "We hope to exploit this weakness to discover and develop a single class of drugs for all three diseases."


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