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Living with ALS (2024) / Chapter Skim
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Pages 139-150

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From page 139...
... • The Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT) is a data platform, created in partnership with NEALS, that houses the largest collection of ALS clinical trials datasets.
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From page 140...
... Network for many All clinical trials Natural history study diseases, including ALS. (Phase 1, 2 and 3)
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From page 141...
... by NIH and FNIH via clinical trials.
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From page 142...
... NEALS can provide a large network of trial-ready sites and clinicians, community engagement and education and standardized clinical outcomes training, but it does not have the scalable trial infrastructure, harmonized data banks, and funding model that an expanded, supported, NIH network could offer. Also, the development of the ALS clinical trials workforce is hampered under the currently fragmented research networks.
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From page 143...
... As FDA notes, historical controls can be effective if the natural history of the disease is well defined which is not the case yet for ALS (FDA, 2023) Should the natural history of ALS become better understood, using natural history studies to serve as an external control could become increasingly important as investigators bring more gene-targeted ALS drugs to human clinical trials.
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From page 144...
... The study is collecting detailed clinical and demographic information, speech and respiratory functional data, multi-omic datasets, and longitudinal biofluid collection of cerebrospinal fluid, blood, and urine. Although a variety of mechanisms exist for conducting clinical trials and natural history studies, a centralized, dedicated ALS clinical trials network that builds on and brings together existing ALS clinical trial consortia could provide a coherent approach to clinical trials and natural history studies that permits faster answers to multiple questions at once.
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From page 145...
... Such a clinical trials network dedicated to ALS would create an exciting opportunity for scores of multidisciplinary ALS centers in diverse geographic areas to bring clinical trial and biomarker research closer to ALS individuals' homes across the nation, which otherwise would not be possible. Recommendation 5-1: Create an ALS clinical trials network.
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From page 146...
... Prognostic biomarkers identify the likelihood of a clinical event, •  disease recurrence, or disease progression in patients with a disease or medical condition of interest. Prognostic biomarkers are associated with differential disease outcomes, but predictive biomarkers discriminate those who will respond or not respond to therapy.
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From page 147...
... Currently, FDA accepts slowing of disease-related decline, stabilization, and improvement of function in daily activities, as measured using either the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) 5 or a scale of combined function and survival outcomes (Berry et al., 2013; Cedarbaum et al., 1999; Quintana et al., 2023)
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From page 148...
... In addition, adverse effects such as nausea, diarrhea, or dry skin may sound minor compared to the risks associated with a fatal disease, but adverse effects that may be tolerable for a drug that works are intolerable for a drug that does not. When there is inadequate evidence of benefit, expanded access programs offer a mechanism for ineligible for clinical trials to receive treatment while definitive data are being collected.
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From page 149...
... b.  Create and sustain a comprehensive, robust, and indefinitely ongoing natural history study across diverse patient popula tions and different stages of disease that could serve as external and concurrent controls for some clinical trials.
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From page 150...
... ESTABLISHING A COMPREHENSIVE ALS REGISTRY In the committee's view, a robust registry of people with ALS is a necessary tool to measure progress toward making ALS a more livable disease. It would collect data on care, outcomes, and risk factors, providing a valuable population-level perspective on living with ALS.
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Key Terms

  • amyotrophic lateral
  • als centers
  • clinical trials
  • als clinical
  • national als

  • als registry
  • comprehensive als
  • als tdi
  • natural history
  • people living

  • lateral sclerosis
  • als research
  • clinical care
  • als clinics
  • individuals living

  • als clinic
  • history studies
  • lateral scler
  • person living
  • clinical trial

  • trials network
  • multidisciplinary als
  • genetic carriers
  • integrated als
  • patient registries

  • developing als
  • history study
  • clinical practice
  • cated als
  • risk factors


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