C R E AT I N G A W O R L D W I T H O U T A L S Volume 9
New Clinical Trials New Results New Ideas in ALS Treatment By Richard Robinson
More than three dozen ALS clinical trials are currently recruiting patients. These trials are testing interventions ranging from an approved cardiac drug to test its neuroprotective ability to botulinum toxin injections to reduce accumulation of excess saliva. “This is an active time in ALS clinical research,” according to ALS Association Chief Scientist Lucie Bruijn, Ph.D. “There are multiple trials testing multiple types of interventions, all with a focus on improving patient outcomes.” There have been disappointments in recent large clinical trials. A trial of dexpramipexole, which acts on cell energy producers called mitochondria, found there was no evidence of effect from either low- or high-dose drug compared to placebo. Similarly, there was no effect on disease progression from dosing with ceftriaxone, which increases the level of a cell membrane transporter for glutamate. Impaired glutamate transport is believed to contribute to the ALS disease process. Despite these disappointments, researchers are more committed than ever to finding treatments to slow or halt the ALS disease process. Over a dozen biotech and large pharmaceutical companies are involved in therapy development programs. Some highlights of ongoing and upcoming trials include:
Fall 2013
alsa.org
The ALS Association’s Certified Center Cost Study By Kim Maginnis The multidisciplinary team approach to the treatment and management of patients with amyotrophic lateral sclerosis (ALS) has emerged as the preferred model of care with evidence for prolonged survival, improved quality of life and reduced hospital admissions. The Quality Standards Subcommittee of the American Academy of Neurology (AAN) developed evidence-based practice parameters for the care of people with ALS, which emphasized the need for multidisciplinary care and was first published in 1999 and later updated in 2009. Although multidisciplinary care is established as a desirable model for clinical practice for people with ALS, there are no published data available from the United States on the costs associated with this model for institutions adhering to the AAN Practice Parameters.
>Anti-SOD1 antisense. ISIS Pharmaceuticals has developed
“antisense” molecules that bind to and cause the degradation of the messenger created from the mutant SOD1 gene. The mutant gene is responsible for about 10 percent of familial ALS. A trial of the antisense molecules was recently shown to be safe in ALS patients, setting the stage for development of a more efficient antisense molecule and resumption of clinical trials, likely to begin in 2014. The same company is in the early
This review includes a three-month prospective, descriptive, multi-center study of the costs associated with care provided at a number of geographically diverse sites meeting the practice criteria for multidisciplinary ALS clinics and certified by The ALS Association. The study employs self-reported data collection tools evaluating costs associated
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Please turn to page 2