About The Center
Amyotrophic lateral sclerosis (ALS), also known in the U.S. as Lou Gehrig’s disease, is a degenerative disease of the nervous system that affects motor nerve cells (neurons) and results in relentlessly progressive muscle weakness. ALS is inherited (familial) in 5-10% of patients, and the majority have non-inherited (sporadic) ALS.
Although the cause of sporadic ALS is unknown and there is no cure yet, research on the disease is advancing at a rapid pace. There is one drug that does modify the disease course somewhat; other drugs are undergoing clinical trials; and, symptomatic treatments are available.
News
On October 6, 2011, Robert Ferrante, PhD, MS, Co-Director received the Leonard Gerson Distinguished Scholar Award established by Sandy Gerson Snyder in memory of her father Leonard Gerson who passed away from ALS at age 70. The award supports educational programs for faculty and staff at the University of Pittsburgh School of Medicine as well as the community at large, with the goal of broadening horizons and pushing the boundaries of knowledge as scientists, physicians, and patients work together in the fight against ALS.
The Center for ALS Research has been designated as a site for the second phase III trial of dexpramipexole. The start date is not yet known.
Scientists identify another protein, ubiquilin, that accumulates abnormally in motor neurons in sporadic and familial ALS. [link to pdf Nature article] Investigators hope that this discovery will lead to therapies that act on this protein pathway, but there are no immediate implications to patient care.
The Center gratefully acknowledges donations from the 2nd Annual Jon Obusek Memorial Bike Run and funding support from the Walter S. and Lucienne Driskill Foundation for research studies to collect blood, CSF and post-mortem tissue samples from ALS patients to discover biomarkers for the progression of ALS.
Two groups of investigators identified a new gene in a non-coding region of chromosome 9 that causes an inherited form of ALS with frontotemporal dementia. These papers are published in the September 10th issue of Neuron. (Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS and A Hexanucleotide Repeat Expansion in C9ORF72 is the Cause of Chromosome 9p21-Linked ALS-FTD)
research
Phase III Study of Dexpramipexole in ALS (EMPOWER)
Clinical trial of Ceftriaxone in ALS
Phase III Study of Dexpramipexole in ALS (ENDEAVOR)- pending
Other Clinical Research
A Multicenter Study for the Validation of ALS Biomarkers
Optimizing Noninvasive Ventilation for People with ALS
Epidemiology of ALS in Western PA
