Spider venom key in UB medical breakthrough

Researchers at the University at Buffalo have discovered a protein in the venom of a Chilean Rose Tarantula that can be synthesized and used as treatment for those battling Muscular Dystrophy.

BUFFALO, NY - If you were playing the word association game, the immediate response to hearing Chilean Rose Tarantula would be 'nope.'  According to a statisticbrain.com survey, 30% of the U.S. population are afraid of spiders - seems low. 

But the eight-legged mongers, specifically venomous arachnids, serve a large role in the researching of new medical treatments. 

“The venom produced by herself, and her species, are what we were working with,” said Distinguished Professor of Physiology and Biophysics Dr. Fred Sachs. 

Sachs has been working with spider venom in his research since the mid-1980's. His research in cell mechanics led him to developing a treatment for cardiac arrhythmias.

“We discovered that cells have sensors, string sensors," said Dr. Sachs, "We discovered it by accident in skeletal muscle of a chicken.”

The question raised by Dr. Sachs and his team was 'why do cells have this mechanical sensor?' To find out the group needed to develop a drug that would keep this cell sensor on all the time, or off all the time then observe how a mouse would react.

This is where the spider venom  comes into play.

“Some animal must have invented a venom that paralyzed its prey and screw up its muscular coordination,” Sachs said.

The Chilean Rose Tarantula was that animal, thus the name Rosie. This is actually the second Rosie, the first died of natural cause after forty years….yes they live that long.

The researchers at UB determined a protein in the spider venom could trigger the cells muscle sensor and in turn be synthesized into a drug treatment.

But drug companies at the time, at this point the mid-1990's,  weren’t interested in such a therapy for cardiac arrhythmias, Dr. Sachs then focused his attention on Muscular Dystrophy.

Approximately 1 in every 3,500 boys are born with the genetic disease and the life expectancy ranges from late teens to late twenties. Currently, there is no cure for Muscular Dystrophy, only treatment, and those treatments only benefit about ten percent of those living with the disease. Being a degenerative disease, quality of life for those living with MD deteriorates with each passing year.

These grim stats and the disease hitting close to home were a reason why a local investor partnered with Dr. Sachs to further develop the synthesized spider venom treatment.

“My Grandson, who is now nine, was diagnosed with MD at fourteen months," said Jeffery Harvey, "They noticed he wasn’t rolling over or crawling so they brought him in for a DNA test.”

Harvey and Dr. Sachs formed Tonus Therapeutics in 2009. The team, including other researchers from UB, oversee the development of the drug, called AT-300. The drug combats the symptom of Muscular Dystrophy at the core genetic level, whereas current Steroid therapies only battle the disease from a single genetic variant and there are nearly fifty genetic variations of MD. AT-300 doesn’t have the side effects current Steroids do.

“He gets tired easily, he falls down because he doesn’t have the core muscle strength you and I do,” said Harvey.

Based on their scientific data in pre-clinical trials, there was a forty percent increase in mobility and other effects the disease has on the body. In all, the team hopes their synthesized spider venom will buy those suffering from MD, just a little more time until a full cure can be developed.

“Our hope is it will prolong their life, give them more years of walking and enjoy life a little bit longs," Harvey said. 

Tonus Therapeutics has licensed AT-300 to  Massachusetts-based pharmaceutical company Akashi Therapeutics. Akashi plans to submit the drug to the FDA for additional trials. In a statement to WGRZ, Akashi CEO Marc Blaustein explained what's next in the drug testing protocol:

We will need to do a series of what are called IND-enabling studies prior to a first human clinical trial.  IND stands for Investigational New Drug Application.  FDA must approve a company’s IND Application before clinical studies can proceed.  These IND-enabling studies consist of safety/toxicity studies and product manufacturing and related studies.  They are designed to ensure safety and quality of the product before entering any human clinical trial.  We may also do additional studies in mice or other model systems to help inform the design of future clinical trials.

Blaustein said safety and toxicity studies could take 12-24 months, and then clinical trials in humans could begin. 

 


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