Several UA researchers have discovered that the drug ampicillin may be a breakthrough in helping patients with movement disorders such as dystonia.
The research was performed in the lab of biology professors Guy and Kim Caldwell in collaboration with scientists at Harvard and the University of Alabama at Birmingham.
“There has not been a new drug lead for dystonia for decades, and most clinical advances have involved dangerous, invasive brain surgery,” Guy Caldwell said in an e-mail. “It is our hope that this work serves as an inspiration to dystonia patients that true hope is on the horizon from the standpoint of drug development to combat the disease.”
Dystonia is a movement disorder that causes muscles to contract and spasm, affecting millions of people throughout the world. Ampicillin is an antibiotic in the penicillin group that activates a protein called torsinA, which protects cells from the stresses known to impact movement disorders.
Apart from the Caldwells and their collaborators at Harvard and UAB, the research team includes several current and former UA doctoral students. The team has been working on this project for five years to satisfy the need for a rapid drug lead, Guy Caldwell said. The team’s findings were published in the scientific journal “Disease Models and Mechanisms” on March 10.
“What we were hoping to do was circumvent a lot of the cost in bringing pharmaceutical help to dystonia patients,” said Kim Caldwell in a UA news release. She said that by examining molecules that are already FDA-approved, the team can avoid the 12-year, $800 million process of bringing a non-approved drug from the research stage to a patient.
“Kim and I are most excited by the potential that exists when such a lead is found,” Guy Caldwell said. “One enters science with the ideal of contributing to the cumulative knowledge of mankind, but it is even more exciting when that knowledge may extend toward helping cure suffering for millions.”
In addition to dystonia, Guy Caldwell said his team plans to evaluate the drugs in models of other diseases like Lou Gehrig’s disease, Alzheimer’s and cystic fibrosis. Kim Caldwell has received a $750,000 stimulus grant from the National Science Foundation to employ doctoral students and undergraduate researchers while continuing dystonia research.
“We love seeing our students realize the true significance of making such discoveries,” Guy Caldwell said. He also gave advice to undergraduates interested in research opportunities.
“If you elect to pursue research, give it you all to find out if it is truly for you or not,” he said. “Success comes from getting integrated into the fabric of a project, sharing intellectual discussions with grad students and your mentors, and fully embracing the culture of a research lab just as one would if they joined the Crimson Tide or Million Dollar Band. Co-authoring a paper that was your own work, not someone else’s, is another very important goal and an outcome of serious commitment.”
