Dumping buckets of ice water over friends’ heads has become a trend on social media as people, including president Rao and Richmond mayor Dwight Jones, partook in the ALS ice bucket challenge to raise awareness.
James Bennett, professor of neurology, psychiatry, physiology and biophysics poured the ice water over Rao before taking the challenge as well. For Bennett, the challenge had special significance, however.
Bennett has been involved in ALS or amyotrophic lateral sclerosis research for about 15 years and has developed a drug that has gone through clinical testing as well as two novel treatments for the disease that currently has no cure.
His team works with inducible pluripotent stem cells (IPSC) which come from the blood cells of ALS patients. He hopes to bring his therapies into the “ALS arena” within the next year.
“(The ice bucket challenge) is a very transient 24-second cold water experience and some would say it’s kind of a stunt,” Bennett said. “But it’s a stunt that’s caught on and has led to many people through social media becoming a little bit more aware of ALS and it’s led to almost a hundred billion dollars being contributed to one of the national ALS organizations.”
Bennett explained that he developed a drug called R(+) pramipexole (R(+)PPX). Knopp Biosciences, the company that licensed R(+)PPX from the University of Virginia in 2006, refers to the drug as Dexpramipexole.
He said that Knopp carried out an approximately 120-person Phase 2 study of Dexpramipexole in ALS patients and found a significantly beneficial effect. They then sub-licensed the drug to a larger international company, Biogen-Idec, that carried out a much larger Phase 3 study with approximately 920 participants, but could not repeat the same findings.
“There’s been substantial disagreement between Knopp and Biogen about the Biogen Phase 3 study. Therefore, Dexpramipexole remains ‘debated’ as to its effectiveness in ALS,” he said.
Bennett has also develeoped two novel therapies. One is a naturally occurring mitochondrial protein, and the other is a small molecule that can mimic a neurotrophin.
“ALS, on an emotional level, I would say it’s the worst disease in all of internal medicine,” Bennett said. “Meaning that when you give someone an ALS diagnosis you are basically giving them a death sentence because there’s nothing that we know about yet that will stop the progression of the disease.”
Bennett said ALS is similar to polio except it happens spontaneously. People with the disease lose the ability to move their arms, legs and face, the ability to swallow and ultimately the ability to breathe. Unless they choose to go on a ventilator, the loss of breathing control is what kills many ALS patients.
The disease is most prevalent in middle-aged individuals, but Bennett said the disease does not seem to affect one gender or race over another. He said ALS is a rare disease that is often misdiagnosed, but affects between 30 to 50 thousand people in the United States.
“It’s a very aggressive, really nasty, awful disease with no treatment and what I talk about is even the worst kinds of cancer that people can get, you at least have some kind of treatment,” Bennett said. “But in ALS there’s really nothing.”
Human genetics graduate student David Brohawn who participates in research under Bennett described ALS as a “particularly scary disease” because 90 percent of patients do not have a family history of it, meaning it can affect anyone.
He said the huge amount of donations to the ALS foundation could allow for more grant money which VCU could potentially apply for.
Laura O’Brien, graduate physiology and bio statistics student is also researching ALS under Bennett.
She is responsible for taking human cells and transforming them into motor neurons, which are the cells lost through ALS. Although O’Brien did not take the ice bucket challenge, she said she thinks it’s a fantastic way to raise awareness and funds.
”I’m curious to see what the ALS Association does with all the extra money,” she said.