PHILADELPHIA – Piera Pasinelli, Ph.D., co-director of the Frances and Joseph Weinberg Unit for ALS Research at the Farber Institute for Neurosciences at Thomas Jefferson University was recently awarded a Margaret Q. Landenberger Research Foundation grant to identify why drug therapies that showed promise in Amyotrophic Lateral Sclerosis (ALS) mouse models have proven unsuccessful in human clinical trials. Dr. Pasinelli and her research team will closely examine critical aspects of drug delivery in the central nervous system at the metabolic level with the hope of advancing drug development for ALS. The grant totals $300,000 and will be issued in $100,000 disbursements over three years.
"In spite of recent progress toward understanding the origination of ALS, finding an effective treatment has remained elusive. Our hypothesis is that one reason there is no viable drug therapy for ALS may be that in designing both mouse and human clinical trials, scientists have overlooked critical aspects of drug bioavailability and metabolism." said Dr. Pasinelli. "Proteins called multi-drug resistance transporters are increased during the disease, rendering pharmacological treatments for ALS mostly ineffective. This grant will support research towards finding ways to inactivate these transporter proteins to counteract their negative effect on ALS therapy. We are extremely grateful to the Margaret Q. Landenberger Research Foundation for seeing the potential our work has and awarding us this grant money."
The Margaret Q. Landenberger Research Foundation, which was formed in 1992, is based in Philadelphia, Pennsylvania. Brown Brothers Harriman Trust Company, N.A., serves as the foundation's corporate trustee.
"The Landenberger Foundation provides initial funding for novel medical research projects in the hope that this will help the scientists further expand their research through additional grants from other sources," said William R. Levy, Executive Vice President, who oversees the operations of the foundation for the Brown Brothers Harriman Trust Company. "Although it is a small foundation, it has an excellent scientific advisory board that helps direct the grant-making decisions. The Foundation's mission is to be at the cutting edge of medical research."
ALS, commonly known as Lou Gehrig's disease or motor neuron disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed. According to the ALS Association, every year about 5,600 people are diagnosed with the disease, which is currently untreatable.
About The Weinberg Unit for ALS Research
The Weinberg Unit for ALS Research at Jefferson's Farber Institute for Neurosciences, co-directed by Drs Piera Pasinelli and Davide Trotti, is a center solely dedicated to the study of disease mechanisms in ALS and to discovering therapies for this devastating disease. The Unit is supported by the Farber Family Foundation and is named after the late parents of Vickie Farber whose father succumbed to ALS. As the first center solely dedicated to basic ALS science in the Philadelphia area, the mission of the Weinberg Unit is to understand how ALS develops and progresses and to translate the results of basic research programs into the development of effective therapies.