When pets treat diseases: researchers create potential therapy for muscular dystrophy from the venom of Rosie, their pet tarantula



Discovery could prolong life of sufferers by decades

Chemical in spider venom key to new therapy

Firm to create treatments already set up



She may look like any other giant spider, but scientists say Rosie, a Chilean rose tarantula, may actually hold the key to treating muscular dystrophy.

The spider started out as a pet for Professor Frederick Sachs of the University at Buffalo.



However, he discovered a key chemical in the venom of the spider can fool the body into thinking it is under attack and cause it to beat the disease.

Even Professor Sachs admits the therapy is unusual.

'No one in their right mind would give spider spit to a kid with dystrophy,' he said.

'It's only through the basic science that you can end up here. … If you keep your eyes open, you see things you would never have looked for.'



Researchers are developing a treatment for muscular dystrophy using a chemical found in the venom of a Chilean rose tarantula

Frederick Sachs, a professor of physiology and biophysics, first purchased a spider of Rosie’s kind—the original Rosie—as a laboratory pet some 20 years ago.

The spider was a mascot for his research team, which was investigating the effect of venoms on important cellular structures called mechanosensitive ion channels.

These channels, which Sachs co-discovered in 1983, are tiny tunnels that connect the inside of a cell with the outside world. Normally, the tunnels are closed. But when a cell is stretched or contorted, the tunnels open and let calcium and other substances into the cell.

The effect is exactly what happens in muscular dystrophy.

Due to a defective gene, patients are missing a reinforcing protein that helps muscle cells keep their shape. This causes the cells to buckle, prompting the ion channels to open and calcium to flood in.



The end result of this chain reaction is that the body 'starts digesting your muscle from inside out,' said Professor Sachs.

Professor Sachs thought that venoms, which are very complex in their chemical makeup, might contain molecular compounds that could block the ion channels.



To test this theory, Sachs hired Tom Suchyna, a recent PhD graduate, to screen the venoms of centipedes, scorpions and spiders.



'There were no drugs for the mechanosensitive channels, so what you do is you go out to look for one,' Sachs said.



The most promising hit came from the exact species of spider that the researchers had adopted as a pet. In the venom of the Chilean rose tarantula, the team discovered a protein that kept the ion channels shut, and has even formed a company to try and develop it.



JB, a Duchenne muscular dystrophy sufferer, whose grandfather Jeff Harvey started a company with researchers to develop a treatment

The fi rm, Tonus Therapeutics, was formed after the grandfather of a sufferer contacted Professor Sachs.

Jeff Harvey, a stockbroker from Williamsville, told him JB’s story and said he was looking to invest in promising treatments for muscular dystrophy.

He had discovered Sachs through a Google search.

'I didn’t know what to make of it at first,' Sachs said. 'We had lunch together, and I explained the history of my attempts to get big pharma interested. In my discussions with Jeff, it seemed like maybe what we should do is make a biotech company and then try to raise funds to take care of what big pharma wouldn’t do.'



'I have an image in my head of a boy standing up out of a wheelchair and walking away,' Sachs said.



'It’s a little fanciful, probably, but that’s the image I have. As I get older, I would feel really good to have helped some kids.'

Today, the UB researchers’ company, Tonus Therapeutics, is testing the protein therapy in dystrophic mice. Initial experiments found that the animals gained strength and suffered no toxic reactions while on the drug for more than 40 days.

The protein used in studies is synthesized in the lab, and is a mirror image of the original tarantula protein, a change that improves the drug’s effectiveness.

The researchers say the therapy is not a cure. But if it works in humans, they believe it could extend the lives of children like JB for years—maybe even decades.