MICA: Pre-Clinical Development of a Small Molecule anti-Wolbachia Candidate Macrofilaricide Drug

More than 157 million people worldwide are disabled by elephantiasis or river blindness, yet no safe drug exists to kill the adult parasitic worms that cause these diseases. The problem is that current treatments target the larval stage of the worms, not the adults. A drug called doxycycline can kill adult worms by attacking the *Wolbachia* bacteria living inside them, but the treatment takes weeks and is unsafe for pregnant women and children. This project has already identified a new compound, AWZ1066S, that kills *Wolbachia* more effectively than doxycycline in animal models and meets all safety requirements for further testing. The researchers now need funding to run the formal pre-clinical safety and manufacturing studies required before the drug can be tested in humans for the first time. If this succeeds, AWZ1066S could become the first safe, short-course oral treatment that kills adult filarial worms. That would transform control programmes for elephantiasis and river blindness, potentially eliminating the need for decades of annual mass drug distribution. For the 157 million people at risk, this would mean a genuine cure rather than just managing symptoms.