ALS findings give reason to hope
By Leo Greene
Never has the research produced such promise. Do I dare to dream?
At a recent Muscular Dystrophy Association ALS conference in Anaheim, I found the stuff of dreams.

Medical sleuths talked of dramatic breakthroughs, promising treatments and the real and imminent possibility of growing new nerves to withered muscles.

"Hopefully within the next two to three years, there will be other more definitive treatments that will either cure the disease or significantly slow down the disease," said Dr. Tahseen Mozaffar, director of the MDA/ALS Research and Clinical Center at UC Irvine.

The neurologist wasn't the only one to speak in terms of "two to three years."

Do I dare to dream?

As early as next year, UC Irvine Stem Cell Research Center scientists could be attempting to grow new nerves in infants with spinal muscular atrophy, commonly known as SMA, a genetically inherited disease like ALS.

SMA is twice as common as ALS and is the leading genetic killer of children under the age of 2.

Stem Cell Research Center co-director Hans Keirstead spearheaded the project, in which embryonic stem cells were implanted in animals and coaxed into becoming working motor nerves.

"To date, there has been no method of isolating high-purity cultures, 95percent and up, where we can generate unlimited quantities of this tissue type," he said.

Those nerve tissues could be used as replacement therapies for ALS, SMA, MS and spinal-cord injuries.

The center is trying to raise $480,000 for an FDA-required safety study. Once that's done, human trials would follow with FDA approval.

"We're working as fast as we can," Keirstead said.

And by the way, there exists no alternative to human embryonic stem cells for growing new nerves.

If I could hang on for another couple of years, perhaps I could qualify for a nerve growth trial.

Do I dare to dream?

Mozaffar had good things to say about his university's stem-cell research and about some high-tech investigative work going on in Arizona.

The Phoenix-based TGen research company has been applying cutting-edge technology to decipher the genetic underpinnings of diseases.

TGen announced late last year that it had uncovered genetic abnormalities common to those with the sporadic form of ALS.

Ninetypercent to 95percent of those with the disease, including me, suffer from sporadic ALS. A genetically inherited form of the disease afflicts the remaining 5percent to 10percent.

Most ALS research has involved the inherited form simply because the offending gene is known and can be inserted into laboratory animals. Whether fixing the inherited form will apply to sporadic variety isn't yet known.

"The TGen study for the first time addressed the issues which are completely relevant to sporadic ALS," Mozaffar noted.

The next step for TGen involves finding drugs or other therapies to fix what the genes are doing.

A similar effort is under way in Cambridge, Mass. At the ALS Therapy Development Institute, medical researchers have been scanning genes, proteins, viruses and other possible culprits in an effort to find out what's askew in ALS. As the damage is defined, treatments will be tried initially in laboratory mice to see whether they extend life.

"And if it does indeed increase survival in the mouse, then what we'll do is build a drug that has that same property, or perhaps take gene therapy into the clinic," said ALSTDI President Sean Scott.

The institute's $36million ALS project represents a collaboration with MDA and Augie's Quest, a California foundation started by fitness entrepreneur Augie Nieto, who was diagnosed with ALS in March 2005.

The Massachusetts lab is already testing a potentially promising drug in humans. It's an old malaria medicine that appears to slow progression of the genetic form of ALS. The drug is being reformulated to reduce toxicity and will be tried on those with the more common form of the disease, Scott said.

If it works, the drug could be available in a couple of years.

Other encouraging research is being conducted at places such as Johns Hopkins University, Columbia University, UC San Diego, Emory University and Laval University in Canada, just to name a few.

The big corporate pharmaceutical companies have largely ignored relatively rare diseases such as ALS, opting instead to develop medicines for more common ailments with potentials for higher returns.

That sad state of affairs may soon change, however. Because of the 1983 Orphan Drug Act, which provides tax breaks and other forms of financial encouragement, ALS actually turns out to be a sizable market, with up to $18billion in available revenue, Scott said.

"I think people are going to start realizing that they're missing the boat by not participating in ALS research."

So, with all this promising research, do I dare to dream?

After all, "research is difficult, and we can't predict its outcome," Keirstead said.

I do dream. I dream with the knowledge that an effective treatment might not come in time for me and many others.

My dreams of the future are bright, but brief.

I reserve my energies for the efforts at hand.

Copyright © 2007 Los Angeles Newspaper Group



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