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Amyotrophic Lateral Sclerosis (ALS) JUNE 2017

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Amyotrophic Lateral Sclerosis (ALS) JUNE 2017 Research Brief Amyotrophic Lateral Sclerosis (ALS) JUNE 2017 Amyotrophic lateral sclerosis (ALS) is a neurologists practicing in the state of Texas progressive disease of the nervous system in were asked if they diagnosed or provided care which degeneration of nerves causes muscle for ALS patients… Neurologists were asked to weakness, paralysis, and ultimately, death. submit one‐page case reports for ALS patients Quantifying the prevalence of ALS in the under the doctor’s care who were alive at some population is difficult because ALS is not a point between January 1, 2009 and December nationally notifiable disease. The federal 31, 2011” (ATSDR, 2017b, p.1). The following table Agency for Toxic Substances and Disease shows results of both surveillance reports: Registry (ATSDR) implemented a National ALS DEMOGRAPHIC CHARACTERISTICS OF ALS PATIENTS Registry in 2009 and launched a secure web JANUARY 2009‐DECEMBER 2011 portal in October 2010 through which UNITED STATES TEXAS individuals with a diagnosis of ALS can register # %#% and complete brief risk‐factor surveys online. TOTAL REPORTED ALS 12,187 1,423 “The National ALS Registry is a congressionally GENDER mandated registry for persons in the U.S. with Male 7,409 60.8 796 55.9 ALS. It is the only population‐based registry in Female 4,740 38.9 627 44.1 Unknown 38 0.3 the U.S. that collects information to help RACE scientists learn more about who gets ALS and White 9,638 45.2 1,135 79.8 its causes” (ATSDR, 2017a). Numbers of Black 798 0.9 93 6.5 individuals diagnosed with ALS can also be Asian NR* 21 1.5 gleaned from existing administrative databases Other 535 1.6 1 0.0 including Medicare, Medicaid, the Veterans Unknown 1,216 52.3 173 12.2 Health Administration, and the Veterans ETHNICITY** Hispanic 25 1.3 213 15.0 Benefits Administration (Centers for Disease Control Non‐Hispanic 889 46.2 1,053 74.0 and Prevention [CDC], 2016). Unknown 1,012 52.5 157 11.0 The Centers for Disease Control and Prevention AGE (CDC) (2014) published the first surveillance Under 40 506 4.2 82 5.7 report on the prevalence of ALS in the United 40‐49 1,356 11.1 201 14.1 50‐59 2,590 21.3 351 24.7 States in July 2014 using data obtained from all 60‐69 3,795 31.1 426 29.9 five sources listed above. Texas state data was 70‐79 2,895 23.8 276 19.4 collected through a surveillance project funded 80+ 952 7.8 76 5.3 by ATSDR “to gather reliable and timely data to Unknown 93 0.8 11 0.8 describe the incidence and demographic *Not reported in CDC surveillance characteristics of ALS and to assist ATSDR in **Ethnicity reported for web portal only, evaluating the completeness of the Registry” not available in the national databases (CDC, 2014; ATSDR, 2017b) (ATSDR, 2017b, p.1). As part of that project “all Page 1 of 6 Amyotrophic Lateral Sclerosis Research Brief June 2017 WHAT IS ALS? deterioration of upper and lower motor “Amyotrophic lateral sclerosis, is a progressive neurons (nerve cells)” neurodegenerative disease that affects nerve Primary Lateral Sclerosis (PLS) is the rarest cells in the brain and the spinal cord. A‐myo‐ form of ALS where “the upper motor trophic comes from the Greek language. "A" neurons (nerve cells) deteriorate. If the means no. "Myo" refers to muscle, and lower motor neurons are not affected "Trophic" means nourishment – "No muscle within two years, the disease usually nourishment." When a muscle has no remains a pure upper motor neuron nourishment, it "atrophies" or wastes away. disease” "Lateral" identifies the areas in a person's Progressive Bulbar Palsy (PBP) affects spinal cord where portions of the nerve cells approximately 25% of PALS and “starts with that signal and control the muscles are located. difficulties in speaking, chewing and As this area degenerates it leads to scarring or swallowing due to lower motor neuron hardening ("sclerosis") in the region” (ALS (nerve cell) deterioration” Association, 2017c). ALS is also commonly referred Progressive Muscular Atrophy (PMA) is to as Lou Gehrig’s disease (after the famous characterized by deterioration of the lower baseball player who died from it), Charcot’s motor neurons. “If the upper motor disease (after the physician who first identified neurons are unaffected within two years, it in 1865) and motor neuron disease (which the disease usually remains a pure lower describes the disease mechanism) (ALS Therapy motor neuron disease” Development Institute [ALSTDI], n.d.b). Familial ALS “affects more than one ALS affects only the nerves involved in member of the same family and that’s due voluntary muscle movement (motor neurons). to a known gene mutation. This type of ALS Other nervous system functions such as accounts for a very small number of people cognition and the senses (hearing, seeing, with ALS in the United States (between 5 smelling, tasting, and touch) are not affected. and 10 percent)” (Johns Hopkins Medicine, n.d.) “Because people with ALS usually retain their ability to perform higher mental processes such RISK FACTORS as reasoning, remembering, understanding, and A large variety of potential risk factors for problem solving, they are aware of their developing ALS have been studied with no clear progressive loss of function and may become causes coming to light. There is still little known anxious and depressed” (National Institute of about why the disease manifests in any Neurological Disorders and Stroke [NINDS], n.d.). particular individual. Even in the small number In general, ALS is categorized as either sporadic of genetically linked forms, mutations have (occurs at random with no identifiable risk been identified on over a dozen different genes factors) or familial (inherited genetic disease). but not one is found in all PALS. Aside from 90‐95% of all ALS cases are considered to be being white, male, and between 40‐70 years sporadic (NINDS, n.d.). All forms of ALS are old (average age of 55 at diagnosis), the only progressive – neurodegeneration continues real identified risk at this time is military inexorably. There is evidence to suggest that service. Military veterans are approximately the rate of decline varies from person to person twice as likely to develop ALS (ALS Association, but is constant for each individual (Turner and 2017d; Turner & Swash, 2015). The evidence was so Swash, 2015). strong that, “In July 2008, in response to the evidence, Secretary of Veterans Affairs Dr. MEDICAL CLASSIFICATION James B. Peake announced that ALS is to be There are five distinct classifications for ALS considered a presumptively compensable used by physicians: illness for all veterans with 90 days or more of Classical ALS affects approximately two continuously active service in the military” thirds of PALS and is “characterized by a (ALSTDI, n.d.a). Page 2 of 6 June 2017 Amyotrophic Lateral Sclerosis Research Brief DIAGNOSIS Most patients who received an initial diagnosis There is no definitive test to diagnose ALS. A of Probable or Possible ALS will ultimately physician reviews the patient’s medical history progress to Definite ALS. The average length of and symptoms and conducts a series of tests to time that PALS survive after diagnosis is 30 rule out other neurological diseases which may months with most people living between two be treatable. The process can be lengthy (up to to five years. Most individuals die of respiratory 12‐14 months) because a primary sign of ALS is failure and/or other complications for the the progression of the signs and symptoms disease (ALS Association, 2017d; ALSTDI, n.d.a). “ALS through a series of neurological tests over time has the shortest median survival among (ALSTDI, n.d.b). The current requirement calls for neurodegenerative disorders” (Turner and Swash, “repetition of clinical and electrophysiological 2015, p.671). examinations at least six months apart to SYMPTOMS ascertain evidence of progression” (ALS Onset of symptoms for ALS vary per individual Association, 2016b). and “it has been estimated that one‐third of Some of the tests used to rule out other large motor neurons must be lost before there neurological diseases include: is visible atrophy” (Turner and Swash, 2015, p.668). Electromyography (EMG) which measures Typical signs and symptoms that a person may the ability of muscles to respond to experience include: electrical stimuli (contract) Trouble grasping or lifting objects Nerve Conduction Study (NCS) which tests Change in vocal pitch when speaking the ability of the nerves to provide Tripping sufficient stimuli to cause the muscles to Dropping things contract Abnormal fatigue of the arms and/or legs Magnetic Resonance Imaging (MRI) of the Slurred speech brain and spinal cord, while normal in PALS, Difficulty swallowing may rule out other conditions such as Weakness in hands, legs, feet, or ankles tumors, cysts, herniated disks, etc. which Muscle cramps and twitches may mimic ALS symptoms Uncontrollable periods of laughing or crying Routine Laboratory tests including blood (ALSTDI, n.d.b; ALS Association, 2016c) and urine samples and thyroid function TREATMENT tests There is no cure for ALS, the disease is always (NINDS, n.d.) fatal. Treatment revolves around extending There are three types of ALS diagnoses, the quality of life for the individual: distinctions of which are primarily used for Medications can help relieve some inclusion in clinical trials: symptoms Definite ALS is diagnosed when the loss of Ease muscle cramps both upper and lower motor neurons is Reduce fatigue present in two or more regions of the body Reduce excess
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