Professor Timor Baasov and Dr. Daniel Pilch

Professor Timor Baasov and Dr. Daniel Pilch
New Antibiotics for Treating Cystic Fibrosis and Other Genetic Diseases

  
Many human genetic diseases are the result of mutations causing premature termination of protein synthesis  and hundreds of these ‘nonsense’ mutations are known. They have been shown to underlie cystic fbrosis (CF), Duchenne muscular dystrophy (DMD), Tay-Sachs, Hurler syndrome (HS) and other fatal diseases for which there is currently no effective treatment. Professor Timor Baasov at Technion’s Schulich  Faculty  of Chemistry  showed  that  some  aminoglycoside antibiotics allow reading past the false stop signal to generate full-length functional proteins. However, to date, the high toxicity of these drugs in humans limits their therapeutic use. Combining his experience with the complementary expertise of Dr. Daniel Pilch from Rutgers University and The University of Medicine and Dentistry at Piscataway, and other laboratories, Prof. Baasov developed novel aminoglycosides programmed to fix nonsense mutations in genetic diseases that are less toxic to mammalian cells.

“In breakthrough research, we have shown for the first time that by chemically modifying an existing antibacterial drug, we can target it selectively to human cells, while, at the same time, lowering its toxicity. This novel drug line can potentially be used to repair ‘faulty’ genes in human beings,” explained Professor Baasov. “This is a completely new direction, and we are currently investigating the potential of these new compounds for treating certain types of cancer in which nonsense stop mutations play a key role. ‘Chemistry’ can be used to do many good things, such as redesigning toxic compounds to produce valuable drugs. We are making sense out of ‘nonsense’,” Prof. Baasov added. Together they have applied for a license for these new drugs, and are now looking into using them to treat many other genetic diseases, which have been incurable until now.