The Clinical Research in ALS and related disorders for Therapy Development (CReATe) Consortium is a Rare Diseases Clinical Research Consortium (RDCRC) that forms part of the National Institutes of Health Rare Diseases Clinical Research Network (RDCRN). CReATe comprises a multi-disciplinary group of clinicians, scientists, educators, patient advocacy groups and other strategic partners, and aims to advance therapeutic development for patients with amyotrophic lateral sclerosis (ALS) and related disorders including progressive muscular atrophy (PMA), primary lateral sclerosis (PLS), frontotemporal dementia (FTD), multisystem proteinopathy (MSP), and hereditary spastic paraplegia (HSP). We recognize that the obstacles to therapeutic development are multifactorial and include: (a) etiological and biological heterogeneity; (b) phenotypic heterogeneity; (c) limitations of existing clinical outcome measures for use in early-to-mid phase clinical trials; (d) a paucity of biomarkers that have been validated as ?fit for purpose?); (e) the relatively late stage at which symptoms appear, diagnosis is made, and treatment is initiated; and (f) variable (often low) rates of patient participation in clinical research studies. CReATe investigators are engaged in a range of clinical trial readiness activities that aim to overcome these obstacles, and to thereby advance therapeutic development for this group of rare diseases. In addition, CReATe aims to promote and support collaborative research in the field of ALS and related disorders; develop and disseminate resources such as the CReATe Biorepository and our electronic health record-based ALS Toolkit that are of value to the broader scientific community; lower barriers to patient participation in research; cultivate and enhance the careers of young clinical investigators and translational scientists dedicated to the study of this group of rare diseases; and engage both lay- and scientific-community stakeholders in a partnership that fosters efforts to develop treatments for patients afflicted with these rare diseases.
ALS, PLS, HSP, PMA and FTD are characterized by degeneration of motor and frontotemporal neuronal systems. Effective therapies for these disorders are sorely needed. Disease heterogeneity, a paucity of biomarkers, delayed diagnostic and treatment, and relatively low rates of patient participation in research, have hampered therapeutic development efforts. Through a series of clinical trial readiness activities, CReATe aims to overcome these obstacles and thereby to advance therapeutic development for this group of rare diseases.
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