An interesting article on the front page of today’s Buffalo News ,” Finding hope in a spider's bite : Venom holds a key to possible therapy for cruel illness, “ describes efforts by a local grandfather to help move an innovative, promising treatmment for Duchenne muscular dystrophy through the pipeline , by funding the pre-clinical research needed to reach clinical; trials. Hiis grandchild is suffering with the disese and is running out of time.

The full article is at:
http://www.buffalonews.com/city/article244438.ece

BTW an article published by UB in Jan 2010, stated that this treatment, “GsMTx4 appears to have additional applications. Robert Plunkett, associate professor of neurosurgery, has shown that the peptide stimulates neuronal growth and may be useful in the treatment of Parkinson’s disease….. “

Excerpts From the current article on Duchenne’s--
“…By age 10, affected children, almost always boys, usually need braces to walk.
By their teen years, most depend on a wheelchair.
And as they grow into manhood, they lose their ability to breathe. Few survive beyond their 30s.

There is no cure, and the standard treatment of steroids can slow -- but not stop -- the progressive muscle weakness, forcing families into a race against time.
All of which is why Jeffrey Harvey, a Clarence stockbroker, couldn't sit still when his grandson, also named Jeffrey, was diagnosed with the disease more than a year ago.
Harvey began to look for potential new treatments, surfing the Internet for anything with promise. Out of curiosity, he searched in his hometown, which led him to a laboratory at the University at Buffalo and to a tarantula spider named Rosie.

The story of his search highlights a novel potential treatment for muscular dystrophy -- one of several therapies on the horizon. It also shows how challenging it can be to bring a drug from the laboratory to a patient's bedside….”
"There is just no drug for these kids other than the steroids to strengthen the muscles, and the steroids have serious side effects," said Harvey, whose grandson is now 3.
“UB biophysicists had found a protein in tarantula venom that showed promise against muscular dystrophy, but the idea was greeted skeptically. No one responded when they shopped it around to pharmaceutical companies earlier in the decade.

Then Harvey called.,,”

Harvey and the researchers formed a small biotech company that hopes to do what the big drug companies wouldn't do,,.
"I want my grandson to be able to have this drug," Harvey said…

“In September, the Food and Drug Administration gave GsMTx4 orphan drug status, a designation for experimental treatments of rare conditions that helps speed development.
Now, comes the difficult part -- seeking an estimated $3 million to fund testing before the drug goes into trials in children.

"The thing is, unlike a lot of others, we already have the drug. If we had the money to get it through preclinical testing, we might have it on the market in three years," Harvey said.
That's not much money or time in the world of drug development, where companies typically spend hundreds of millions of dollars and more than a decade on one product..”

Maybe this is part of our answer. Bypass the drug companies...Scientists, working with patients and families . .. Understand the urgency of getting treatments to patients sooner, not later... Welcome patients at the table, instead of banning them from scientific meetings.

Are the Doldrums based on business or scientific oppotunities?

We don’t know if the Tarantula cure will pan out, but at least its being given a chance based on the science, not sitting on a lab shelf, because of a business decision.