Fred Sachs, Ph.D, distinguished professor of physiology and biophysics, has decided to take things into his own hands to revolutionize the world of medicine. Sachs and his colleagues have teamed up to start their own pharmaceutical company, Rose Pharmaceutical, to advance the clinical testing of a certain protein that they believe has the potential to treat a slew of diseases.
Sachs discovered a protein in the venom of a tarantula known as GsMTx4, a place he admits would seem unlikely to find such a therapeutic substance.
The mechanism by which the protein works is completely unheard of in the realm of drug therapies. Where most medical drugs act on various chemical receptors found on the different cells of the body, GsMTx4 acts on a type of sensor on cell membranes sensitive to mechanical stress. The researchers at Sachs's lab were the first to discover, accidently about 20 years ago, that such structures even existed on cells.
According to Sachs, these sensors are still at such an early stage of being researched that their actual function is still the subject of much debate.
'Exactly what they do for a living remains to be determined,' Sachs said. 'The current model we have is that you could look at them as pain receptors at a cellular level. What they're looking for is weak spots on the reinforcement to the membrane.'
Sachs's lab discovered – through an act of serendipity – that GsMTx4 acted on these stress-sensitive structures to produce a number of extremely positive effects in the bodies of test mice. These included an increase in muscle strength, pain inhibition and a correction of cardiac arrhythmia. Sachs was quick to try to market the protein.
With the experimental research to prove it, Sachs attempted to advance the protein as a treatment for muscular dystrophy, sickle-cell anemia, cardiac arrhythmia, and peripheral neuropathic pain. Sachs gave seminars to all the large pharmaceutical companies.
Unfortunately, they were uninterested or not capable of accepting the discovery he had made.
Sachs said the companies' hesitation to advance the drug was due almost entirely to its complete novelty and the unconventional way in which it functioned.
'Big pharmaceutical companies didn't pick up on this,' Sachs said. 'I tried. I gave them a lot of seminars and stuff, but the idea of working with biomechanics and drugs was so [unorthodox] that no one wanted anything to do with it.'
According to Sachs, he gave up completely and decided to do the science and let someone else pick up on his research in the future. The next step found him.
A local stockbroker with a 2-year-old grandson diagnosed with muscular dystrophy found Sachs' work on the Internet, and the two discussed his work over lunch. This encounter and their subsequent correspondence eventually led to the formation of Rose Pharmaceuticals this past July. The company is affectionately named after the lab's pet tarantula, 'Rose.'
Although they are hopeful about the progress they will make, the company is still in its infancy and facing one major problem.
'Like all start-ups we don't have the money,' Sachs said. 'We have the idea, but we don't have the money, so we'll keep working on it.'
The next step is to get FDA approval.
'There's no obvious reason this wouldn't work at this point,' Sachs said. 'So we're going after four of these diseases because we have data that it can be active on these four diseases, and anything you learn from any one of them can be applied to the other. So there is really only one set of toxicity tests that need to be done with this.'
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Photo: Courtesy of Laron Fomby
