Approximately 60 Principal Investigators, and several Post Docs, both basic science/laboratory and clinical researchers, work within the Research Institute and the Medical Center.
California Pacific Medical Center physicians from many different disciplines engage in active research. Take a look at our Clinical Trials section for some of these projects.
Dr. Robert Miller, and New Advances in ALS Research at CPMCRI
For the 30,000 people in the U.S. with amyotrophic lateral sclerosis (ALS), progressive neurodegeneration and death of motor neurons causes loss of control of voluntary muscles and resulting symptoms including muscle weakness, spasticity, slurred speech and respiratory difficulties. While a cure is elusive, researchers at CPMCRI are exploring new approaches to treatment, and pioneering advances that bring improved quality of life for people with ALS.
Drs. Robert Miller, Jonathan Katz and colleagues at CPMC’s Forbes Norris MDA/ALS Research and Treatment Center have begun new studies of approaches to reduce the neuroinflammation suspected to underly the gradual progression of ALS.
“We are seeking to understand the mechanisms that selectively trigger motor neurons to degenerate in ALS, and to find effective approaches that halt the processes leading to cell death,” said Dr. Miller, Director of the Forbes Norris MDA/ALS Research Center.
Currently, the only FDA-approved drug treatment for ALS (riluzole/Rilutek®) reduces damage to motor neurons by decreasing the release of glutamate. The drug has established safety data and prolongs survival in patients by several months.
Ongoing clinical trials led by Dr. Miller and Dr. Katz will assess the efficacy and safety of a new drug (NP-001) that has shown promise in halting disease progression in a subset of patients. A separate trial of the drug tirasemtiv is nearing completion. This drug is a fast skeletal muscle troponin activator, which is helpful in a disease such as ALS characterized by weakened and wasted muscles.
Other trials led by the ALS team will determine optimal use of non-invasive ventilation (NIV) machines to ease breathing in patients, and a multicenter, randomized study of the NeuRX® Diaphragm Pacing System—an approach to support patients with diaphragm weakness.
Later this year, the ALS group will begin collaborating with Cedars-Sinai Regenerative Medicine Institute on a study funded by the California Institute for Regenerative Medicine (CIRM). The new project will test the effects of combined stem cell and gene therapy, along with the protein GDNF (glial cell-derived neurotrophic factor) to promote the survival of damaged motor neurons in ALS patients.
Other collaborative initiatives include a project with researchers at the University of California, San Diego to study mutations in RNA-binding proteins that cause neuronal dysfunction and death, and several studies of cognition and behavioral issues in ALS patients.
Upcoming research being presented at the 2014 Annual Meeting of the American Academy of Neurology (AAN)
Dr. Miller will deliver a platform presentation on ALS quality measures at the AAN annual meeting in April. Systematic assessment of guidelines and consensus papers from 2006 to 2013 resulted in the development of 11 quality measures for ALS. The measures are focused on evaluating the use of effective therapeutic options in patients, and increasing awareness of advanced planning and patient safety.
“The AAN quality measures, when implemented by providers, have the potential to significantly improve care for people with ALS,” said Dr. Miller.
More About CPMC’s Forbes Norris MDA/ALS Research and Treatment Center
In recognition of the ALS group’s successes and leadership in the area of motor neuron disease research, the Forbes Norris Clinic was one of only five centers selected to present research on quality and outcomes measures at the AAN’s meeting last year.
The research on “Quality improvement in neurology: Quality measures in ALS” was published in a December 2013 issue of Neurology. Furthermore, the non-profit organization ALS Worldwide published a book about best practices in the management of the illness, and highlighted the Forbes Norris Clinic.
Dr. Robert Miller, Director of the Forbes Norris MDA/ALS Research Center
Dr. Jonathan Katz, Director of CPMC’s Neuromuscular Research Program and Director of the ALS Clinic
For more information about the program and team members, visit:
- Peggy Cawthon, PhD, MPH
- Stewart Cooper, MD
- Steven Cummings, MD
- Shanaz Dairkee, PhD
- Robert Debs, MD
- Pierre Desprez, PhD
- Dan Evans, PhD
- Gantt Galloway, PharmD
- Mohammed Kashani-Sabet, MD
- Sean D. McAllister, PhD
- John Mendelson, MD
- John Muschler, PhD
- Karin L. Petersen, MD
- Michael C. Rowbotham, MD
- Liliana Soroceanu, MD, PhD
- Katie Stone, PhD
- Gregory James Tranah, PhD
- Cassandra Vieten, PhD
- Esther Wei, ScD
- Li Xi Yang, MD, PhD
- Garret L. Yount, PhD