Spinal cord injury hasn't been a big-ticket malady from the perspective of
Wall Street's drug companies, whose neuroscience bets have more typically
been placed on treatments for Alzheimer's and Parkinson's and other more
common (i.e., profitable) nerve disorders.
But because technology and research results have begun to catch up with
expectations, SCI cure research is getting ready to leap from the lab to the
clinic.
Two years ago, physician-turned-entrepreneur Ron Cohen started Acorda
Therapeutics, a "virtual" company with a New York City address. It's the
first bio-tech company to specifically focus on the commercialization of SCI
research.
Cohen first caught the bio-tech bug in 1986. He left an internal medicine
practice to join a San Diego start-up company in the artificial skin
business. But running a business excited him less than creating one, so he
looked for something new.
Cohen says his first love has always been neuro-medicine. He didn't pursue
it after med school "because there was so little -- nothing -- that could be
done for patients."
Twenty-some years later, he was looking for a bio-tech project. "SCI," he
found, "was conspicuous by its absence. That really got me interested," says
Cohen. "As I became more aware, I saw that the technology was there, the
animal model was good. The field was not well-funded by the NIH, but the
scientists were a cohesive group."
Cohen raised some private money -- the total is about $1 million now -- and
has a line on venture capital well beyond that. He envisions Acorda as the
product development arm for paralysis research projects around the world.
His crack science team includes well-known SCI researchers Wise Young of New
York University; Andrew Blight of the University of North Carolina; Richard
Bunge, science director for The Miami Project; Dennis Choi of Washington
University in St. Louis; Carl Cotman of the University of California at
Irvine; and Jerry Silver of Case Western Reserve University in Cleveland.
The key to Acorda is collaboration, says Cohen. (That's the origin of the
name -- there's the "cord," of course, but also "accord.") He cites the
discovery of the gene for Huntington's disease: "At the time they began,
people were laying odds it would take 75 years to find. But Nancy Wexler's
team of eight labs found it in 10 years.
"A single scientist is tilling just a small piece of the garden," he says.
"If you want to cover the whole farm, you have to pool together the
technologies and expertise, and in SCI, that's never been done."
Acorda's first major project, slated for mid-1996, is a large-scale clinical
trial for the drug 4-AP, which in smaller trials has been shown to
temporarily boost nerve transmission in fibers that lack myelin insulation.
Cohen says 18 SCI centers have agreed to admit people to the trials, which
will be coordinated by Dr. John Ditunno of Thomas Jefferson Hospital in
Philadelphia. About three out of 10 people with spinal cord injury are
expected to benefit from the drug.
The 4-AP work segues to another tier in Acorda's strategy. Cohen has
licensed with Dr. Moses Rodriguez of the Mayo Clinic for the rights to
develop an antibody molecule that appears to stimulate myelin production.
Similar antibodies have resulted in "spectacular recoveries" in a handful of
patients.
Another part of the plan is to bring to market a growth-promoting compound
called L1. This molecule seems to counteract substances in the damaged cord
that block growth. The L1, says Cohen, could be combined with growth
factors, genetically engineered cell lines or other antibodies that promote
axon growth.
There won't be a single treatment for paralysis, Cohen says. "We want to use
an integrated approach, using perhaps the L1 molecule, growth factors and
biomaterials for appropriate delivery. I foresee the use of cells that are
genetically altered to express the L1 molecule and a growth factor, along
with a biomaterial substance -- all implanted at the lesion site with a
time-release to promote functional regeneration."
When will all this happen? "Our science team has set as an internal goal --
I want to be clear that we're not making a prediction -- that with the
science available today, we can in four or five years have some construct,
some therapy to test in humans.
"This is very hard work," Cohen says, "but given the technology and the
scientists we have, it is not ridiculous, it is not pie in the sky. It's
within the realm of possibility."