Duchenne Muscular Dystrophy is the most common genetic disease of boys. There is no known therapy or cure. In 1984 Sachs Lab, University at Buffalo's School of Medicine, Dr. Frederick Sachs, UB Distinguished Professor of Biophysics, Department of Physiology and Biophysics, and his colleagues, have discovered mechanosensitive ion channels, pores in the cell membrane that open under mechanical stress. When the genetic defects of dystrophy break down reinforcement of the cell membrane, these channels become activated and let calcium leak into the cell causing the muscles to atrophy. This is what causes muscular dystrophy.
Sachs Lab made the discovery of the channels when there were no drugs known to affect those channels. Taking a wild chance on Nature's chemical libraries, they examined venoms from scorpions, centipedes and spiders and in a tarantula venom, they discovered a drug that did work. The drug turned out to be a small, non-toxic, protein. Keep in mind, everything in a venom is not poisonous. The lab tested the protein (known as GsMTx4) on isolated dystrophic muscle since dystrophin makes a reinforcing network under the cell membrane, and its loss transfers stress to the membrane. This increased stress turns on the channels producing the calcium leak.
GsMTx4 caused the muscle less likely to be damaged from stress. In collaboration with Dr. Eric Hoffman of the Wellstone Institute for Muscular Dystrophy in Washington, Sachs Lab tested GsMTx4, and after a month of treatment there were no significant side effects.
Sachs Lab is close to having a therapy for muscular dystrophy.
To handle commercialization of GsMtx4 after preclinical testing, Sachs Lab formed Rose Pharmaceuticals, a research-based biotech drug company founded with the specific goal of treating muscular dystrophy. Rose Pharmaceuticals is located in Buffalo and started by Drs. Fred Sachs, Phil Gottlieb and Tom Suchyna and Mr. Jeff Harvey, and Rosie, the tarantula.
You can help the research. Donate (tax deductible) to the lab by sending whatever amount you feel is appropriate for "unrestricted research funds" to: Rosie's Therapy for Muscular Dystophy: Grammostola Spatulata Fund UB Foundation P.O. Box 900 Buffalo, NY 14226.
When this therapy passes FDA regulations and pre-clinical testing, not only will there be a therapy for muscular dystrophy, but this medication GsMTx4, with no known side effects, will also be able to treat other conditions that affect muscle walls such as various heart conditions and everyday chronic and acute pain. Right now, the only medications available for such conditions, including muscular dystrophy have severe side effects and are addictive.
Please join Sachs Lab, Rose Pharmaceuticals and the UB School of Medicine (all Buffalo based entities), in passing a medication that can literally help millions of people. Currently, everyone at Sachs Lab is funding this endeavor out of their own pockets.
Rose Pharmaceuticals has been featured in The UB Reporter, Technology Transfer Tactics, Innovations Report.com, Fox News National, Buffalo State News, Futurity.org Health & Medicine, and Channel 2 WGRZ. on National News.
The drug is in preclinical trials at the Wellstone Institute in Washington DC. There is no toxicity. There are no known side effects. There are no known addictive properties. There is no cure or therapy.
Until now.
You can help the research. Donate.
© Susan Marie 2010
Sachs Lab, Dr. Frederick Sachs
University at Buffalo School of Medicine
Rose Pharmaceuticals
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