dysarthria, is difficult to treat. Almost all patients with
bulbar-onset ALS and nearly 70% of patients with
spinal-onset ALS develop dysarthria. Alternative augmentative communication devices and speech patholo-gists can be helpful. The Speakit application is a free
speech-generating device available for the iPad, and a
DynaVox device is a more costly augmentative and alternative communication tool. These devices help patients to communicate their needs and to stay connected
As ALS advances, patients might require palliative
medicine or hospice care. Before a patient reaches advanced stages of the disease, he or she should fill out a
directive and Physician Orders for Life-Sustaining Treatment form, said Dr. Weiss. Patients must meet one of
two criteria to receive Medicare reimbursement for hospice care. The first is a forced vital capacity of less than
30% with dyspnea at rest. The second is rapid progression of the disease with life-threatening complications
(eg, infections) over 12 months or severe nutritional
impairment over 12 months. Treatments for terminal
dyspnea include morphine sulfate, oxygen, lorazepam,
and chlorpromazine. These medications may depress
respiratory drive, however, said Dr. Weiss.
Current Research and Recently Completed Trials
Smith et al found that dextromethorphan–quinidine
may improve bulbar function. In addition, recent
studies have found that diaphragmatic pacing, an
FDA-approved technique, shortened the survival of
patients with ALS by one year. An interanalysis found
that patients using the diaphragmatic pacer died pre-
maturely, compared with patients who had sham
surgery in which electrodes were not implanted. Sever-
al phase II studies have suggested that tirasemtiv is sub-
stantially beneficial in decelerating slow vital capacity.
Finally, a 24-week randomized controlled trial found
that edaravone improved function in patients with defi-
nite or probable ALS. Edaravone was FDA-approved in
May 2017. NR
Miller RG, Mitchell JD, Moore DH. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane Database Syst Rev.
Miller RG, Jackson CE, Kasarskis EJ, et al. Practice parameter update:
the care of the patient with amyotrophic lateral sclerosis: drug, nutritional,
and respiratory therapies (an evidence-based review): report of the Quality
Standards Subcommittee of the American Academy of Neurology. Neurology
Paganoni S, Deng J, Jaffa M, et al. Body mass index, not dyslipidemia, is
an independent predictor of survival in amyotrophic lateral sclerosis. Muscle
Nerve. 2011;44( 1): 20-24.
Shefner JM, Wolff AA, Meng L, et al. A randomized, placebo-controlled,
double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in
patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2016; 17( 5-6):426-435.
Smith R, Pioro E, Myers K, et al. Enhanced bulbar function in amyotrophic
lateral sclerosis: The nuedexta treatment trial. Neurotherapeutics. 2017 Jan
9 [Epub ahead of print].
Traynor BJ, Alexander M, Corr B, et al. Effect of multidisciplinary amyotrophic
lateral sclerosis (ALS) clinic on ALS survival: a population based study, 1996-
2000. J Neurol Neurosurg Psychiatry. 2003;74( 9):1258-1261.
Weiss MD, Macklin EA, Simmons Z, et al. A randomized trial of mexiletine
in ALS: Safety and effects on muscle cramps and progression. Neurology.