Center for ALS Research

Established in 2005 to integrate and coordinate new and on-going research and clinical activities by investigators and clinicians in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases, the Center for ALS Research, directed by Robert Bowser, PhD, includes a multidisciplinary clinic for patient care and numerous basic science laboratories searching for causes and treatments of ALS. The Center for ALS Research also administers the Stephen Tuttle ALS Tissue Donation Program, providing a resource for human tissue samples for ALS and control subjects for use in research activities both within the University of Pittsburgh and throughout the country.

_________________________________________________________________________________

Research
Current research emphasizes the basic mechanisms and cell biology of disease. Various University faculty utilize molecular biology and genetics, cell biology, and various animal models to investigate motor neuron diseases. Studies include:

Examining the biologic function of ALS specific protein biomarkers and role in disease pathogenesis.

Determining how environmental factors such as selenium participate in ALS and contribute to motor

neuron cell death.

Examination of the role that cell cycle proteins play in motor neuron cell death and gliosis during ALS.

Identification and examination of metabolic pathways that contrbute to the pathobiology of ALS.

Determining new genetic factors that contribute to sporadic ALS.

Clinical
The medical director of the Center for ALS Research is David Lacomis, MD. The clinical research component of the center includes the identification and validation of protein biomarkers for the diagnosis of ALS, as well as the search for protein biomarkers of ALS disease progression. The MDA-ALS clinic provides accurate clinical diagnosis and continued treatment of ALS patients through a multidisciplinary approach and patients are followed longitudinally and recruited to participate in various clinical studies. Currently, there are three clinical research studies and two experimental clinical trials ongoing for ALS, with additional clinical trials planned for 2008.

A Two-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Research subjects for this study are being recruited at the University of Pittsburgh Medical Center under the direction of the Principal Investigator, Dr. David Lacomis. Subjects will be randomly assigned to receive either placebo or the study drug (2 tablets of KNS-760704). This study involves two phases which total nine months of participation. In the first phase, subjects will be randomly assigned to receive the study drug (at three different possible dosages) or placebo for three months. When phase one is completed, subjects will participate in a four-week "wash-out" period, during which all patients will receive placebo. Following the completion of the "wash-out," all patients will be randomly assigned to receive one of two dosages of the study drug for the remaining six months. To be eligible for this study, patients must be between the ages of 21 and 80 and have a diagnosis of probable or definite ALS and must have a vital capacity (breathing function) of greater than or equal to 70 percent of normal. There are other criteria that must be met in order for patients to participate in this research study and they cannot receive other experimental therapies. Approximately 80 subjects will be enrolled at all sites. The University of Pittsburgh plans to enroll approximately ten patients. Enrollment begins June 12. For more information, email Danielle Rowlands or phone 412-648-9053 or 412-692-2854.

UPCOMING CLINICAL TRIALS