Mounting the attack on Lou Gehrig's disease
Research scientist Claire LeMasters works at Knopp Biosciences. The South Side company is involved in ALS research and drug development
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Neil Alexander received the news that he had amyotrophic lateral sclerosis more than seven decades after baseball legend Lou Gehrig, who lent his name to the disease, received the same diagnosis.
Yet in that time, little progress has been made in the treatment of ALS. For the average patient, the degenerative motor neuron disease still leads to paralysis and death two to five years after diagnosis. The one drug for ALS that has received approval from the U.S. Food and Drug Administration -- riluzole -- only moderately slows the progression.
That's why, when Mr. Alexander, 46, of O'Hara, received his diagnosis last June, his doctor's advice was direct: "Do what you love."
Mr. Alexander has spent the past several months creating LiveLikeLou.org -- a foundation to raise money for ALS patient support, research and awareness -- and in September, he did something that Gehrig, who died two years after receiving his 1939 diagnosis, could not do.
- Neil Alexander continues to work, coach as he raises ALS awareness
- Executive finds new strength in disease's legacy
- Living with Lou Gehrig's Disease
- ALS charity makes first major donation
He joined a study for a promising drug for the treatment of ALS called dexpramipexole. The drug won't cure patients of ALS, but there is some suggestion it could slow the progression of the disease. The Phase III study involving 943 ALS patients in the United States, Canada, Europe and Australia is being conducted by Weston, Mass.-based company Biogen Idec and will conclude in the fall. The results -- whether the drug works and if so, how significantly it slows the progression of the disease and extends a person's life -- will be available by the end of 2012.
"It would be a significant development in an area that has had nothing positive," said Mr. Alexander, who doesn't know whether the pill he has been taking twice a day since September is the drug or the placebo.
If Mr. Alexander, who with his wife of 20 years, Suzanne, has a 10-year-old daughter and an 8-year-old son, is awaiting the results of the study eagerly, so is South Side-based Knopp Biosciences.
When the company, named after the late Pittsburgh real estate developer and ALS patient Walter Knopp, was founded in 2004, its main focus was working with University of Pittsburgh researchers to develop biomarkers for ALS, which could be used to create a diagnostic test for the disease.
Knopp grew into a drug development and research company after a researcher at the University of Virginia told Knopp he had identified dexpramipexole as a potential treatment for ALS. The drug seems to increase the efficiency of energy production by mitochondria in the nerve cells that become dysfunctional when ALS strikes, thereby slowing the disease's progression.
The company licensed the drug in 2006 and studied it in two phases -- first with about 80 healthy human volunteers to analyze how it behaves in the blood system, and then with about 100 ALS patients to examine how the drug affects the progression of the disease, said Tom Petzinger, executive vice president for business development and public affairs at Knopp.
The outcome of the Phase II study revealed a trend showing the drug could have an effect on the progression of the disease and a survival benefit. Biogen Idec found the Phase II data "very compelling," said Jim Baker, the company's senior manager for public affairs. Those results convinced Biogen Idec to license the drug and run the Phase III trial.
There are other researchers exploring treatments for ALS, but dexpramipexole is potentially the closest to receiving FDA approval, Mr. Baker said.
As Knopp waits to see Biogen Idec's Phase III results, CEO Mike Bozik said his company is "guardedly optimistic and quietly confident."
The study may be winding down, but Knopp is growing.
In the past six months, Knopp has doubled its office and lab space on the South Side as well as its employee head count. Its 40-person staff includes about 20 biologists and chemists.
Researchers are turning their attention to learning how dexpramipexole works, testing the efficacy of new compounds developed by Knopp chemists and to identifying what's called the molecular target of the drug, the site where the drug acts.
One goal is to develop the next generation of drugs that target mitochondria, which could be used as treatment on ALS as well as similar diseases such as Parkinson's.
Mr. Bozik said his vision for the company is to create a sustainable biotech company that is an anchor company for similar companies in Western Pennsylvania.
"Our goal is to be a sustainable, impactful biotech company that gets the ball rolling here in Pittsburgh," he said.
The work Knopp has been doing requires absolute rigor, but researchers realize they are working on a disease where every day counts for the people who have it, Mr. Petzinger said.
One of those people, Mr. Alexander, said he can tell the disease is progressing as he nears the first anniversary of his diagnosis. The muscles in his hand are atrophying, and his left hand is getting difficult to use. He clears his throat frequently, and he can feel the disease advancing in his legs.
He created LiveLikeLou.org, partly in an attempt to improve the outlook for the people who develop ALS in the future.
If scientists can develop drugs to slow the progression of the disease, the hope is that ALS, much like AIDS, can become a chronic disease rather than a death sentence, Mr. Petzinger said.
"The best thing we can do is get a meaningful treatment approved, because there are millions of people with ALS who haven't been born yet and haven't developed the disease yet," he said.
First Published June 18, 2012 12:00 am