MDNews - Minnesota

September 2017

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" Recognizing and documenting the presence of 'atypical features' can sometimes allow us to recognize the limitations of the [disease] categorization process, and the systematic evaluation of what might appear to be unusual clinical observations — e.g., higher rates of schizophrenia in family members of those with ALS — can lead to new and exciting ways of subcategorizing patients based on pathobiology rather than clinical presentation." ORLA HARDIMAN, BSc, MD, FRCPI, FAAN, FTCD, MRIA, PROFESSOR OF NEUROLOGY AT TRINITY COLLEGE IN DUBLIN A Closer Look at an Incurable Condition THERE A RE NO effective pharmacologic remedies for ALS. Since the disease was discovered nearly 150 years ago, the FDA has approved only two medications to treat it, which underscores the critical importance of patient-focused drug development. The fi rst drug, riluzole, received approval more than 20 years ago, and it has little effect on survival. Typically, taking the drug consistently extends a patient's life by only two to three months, according to Calaneet Balas, MS, MBA, Executive Vice President, Strategy, at the Washington, D.C.-based The ALS Association. The second drug, edaravone, received FDA approval in May 2017. In the clinical trial on which approval was based, people who took edaravone experienced fewer declines in daily functioning than those who received a placebo during the same time period. Whether or not the drug, which is designed to delay disease progression and is administered intravenously, will improve survival rates is currently unknown. To complicate matters further, there is no identifi able biomarker for ALS. This makes it diffi cult for physicians to diagnose the condition in early stages or to test therapies' effectiveness. In the absence of biomarker data and curative pharmacologic agents, management strategies for ALS are primarily palliative. "We're really trying to make people comfortable and make sure that they have the technology that they need to communicate and eat, ..." Balas says. "That certainly helps promote the longevity of their life, as well as the quality of their life, but that's really how ALS is managed now." "Our solid, empirical evidence that a neurological disease — ALS — and a psychiatric disease — schizophrenia — have some common origins somewhat nullifi es the distinction between neurology and psychiatry, and I hope that this obsolescence might open up avenues in research that will help researchers and clinicians fi nd new ways to treat these diseases," he says. "As both ALS and schizophrenia are highly disabling conditions, we hope that the fresh ideas that our research injects into the fi eld of brain disorders will engender real progress and ultimately lead to cures for both diseases." ■ 1 3 M D N E W S . C O M /// M D N E W S M I N N E S O TA ■ 2 017

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