About the Study
Principal investigator: Michael Benatar, MD, PhD
The CRiALS Biomarker Study is aimed at increasing our knowledge of ALS and several other related neurological disorders, with the goal of identifying biomarkers related to therapy development. Research volunteers may participate in one or more procedures, including neurological examination, collection of biological samples, MRI, motor tasks, physiological and cognitive testing, and respiratory functions tests.
Eligibility Criteria
We are recruiting 4 groups of people for this study:
- Individuals diagnosed with ALS (sporadic or familial ALS)
- Individuals with neurological diseases that may be related to ALS
- Individuals at risk for developing ALS or related neurological diseases
- Individuals without neurological disease and with no apparent risk for developing neurological disease in the future
Funding Agencies
Collaborators
Physiology and Motor Function
- Ralf Reilmann, MD, PhD (University of Munster, Germany)
- Seward Rutkove, MD (Beth Israel Deaconess Medical Center)
Imaging
- Dr. Martin Turner, MD, PhD (University of Oxford)
Wet Biomarkers
- Oleg Butovsky, PhD (Brigham and Women’s Hospital, Boston)
- Mary-Louise Rogers, PhD (Flinders University, Australia)
- Claes Wahlestedt, MD, PhD (University of Miami)
- Zane Zeier, PhD (University of Miami)
- Leonard Petrucelli, PhD (Mayo Clinic Florida – Jacksonville)
Neuropsychology
- Heather Katzen, PhD
- Sharon Abrahams, PhD (University of Edinburgh)
Contact
Research coordinator:
- Ashley N. Manso, BS (Research Assistant, University of Miami)
Contact information:
- Email: alsresearch@miami.edu
- Phone: 1-888-413-9315
Links
Publications
- Shepheard SR, Wuu J, Wiklendt L, Dinning PG, Andersen PM, Chataway T, Schultz D, Benatar M, Rogers ML. Urinary neurotrophin receptor p75 extracellular domain: a longitudinal biomarker for ALS progression. Neurology. 2017 Feb 22. [Epub ahead of print]
- Gendron TF, Chew J, Stankowski JN, Hayes LR, Zhang Y-J, Prudencio M, Carlomagno Y, Daughrity LM, Jansen-West K, Perkerson EA, O’Raw A, Cook C, Pregent L, Belzil V, Van Blitterswijk M, Tabassian LJ, Lee CW, Yue M, Tong J, Song Y, Castanedes-Casey M, Rousseau L, Phillips V, Dickson DW, Rademakers R, Fryer JD, Rush BK, Pedraza O, Caputo AM, Desaro P, Palmucci C, Robertson A, Heckman MG, Diehl NN, Wiggs E, Tierney M, Braun L, Farren J, Lacomis D, Ladha S, Fournier CN, McCluskey L, Elman L, Toledo JB, McBride J, Tiloca C, Morelli C, Poletti B, Solca F, Prelle A, Wuu J, Jockel-Balsarotti J, Rigo F, Ambrose C, Datta A, Yang W, Raitcheva D, Antognetti G, McCampbell A, Van Swieten JC, Miller BL, Boxer AL, Brown RH, Bowser R, Miller TM, Trojanowski JQ, Grossman M, Berry JD, Hu WT, Ratti A, Traynor BJ, Disney MD, Benatar M, Silani V, Glass JD, Floeter MK, Rothstein JD, Boylan KB, Petrucelli. L. Poly(GP) proteins as a pharmacodynamic biomarker for C9ORF72-associated amyotrophic lateral sclerosis. Science Translational Medicine, in press
- Easnov R, Belle KC, van Blitterswijk M, Belzil VV, Rademakers R, Dickson DW, Petrucelli L, Boylan KB, Dykxhoorn DM, Wuu J, Benatar M, Wahlestedt C, Zeier Z. C9orf72 promoter hypermethylation is reduced while hydroxymethylation is acquired during reprogramming of ALS patient cells. Exp Neurol, 277: 171-7, 2016. [ Pubmed Link ]
- Zeier Z, Esanov R, Belle KC, Volmar CH, Johnstone AL, Halley P, DeRosa BA, Khoury N, van Blitterswijk M, Rademakers R, Albert J, Brothers SP, Wuu J, Dkxhoorn DM, Benatar M, Wahlestdt C. Bromodomain inhibitors regulate the C9ORF72 locus in ALS. Experimental Neurology, 271:241-250, 2015. [ Pubmed Link ]
- Turner MR, Benatar M. Ensuring continued progress in biomarkers for amyotrophic lateral sclerosis. Muscle Nerve. 2015 Jan;51(1):14-8. [ Pubmed Link ]
- Turner MR, Grosskreutz J, Kassubek J, et al. Towards a neuroimaging biomarker for amyotrophic lateral sclerosis. Lancet Neurology. 2011 May;10(5):400-3. [ Pubmed Link ]
- Metwalli NS, Benatar M, Nair G, et al. Utility of axial and radial diffusivity from diffusion tensor MRI as markers of neurodegeneration in amyotrophic lateral sclerosis. Brain Research. 2010 Aug;1348:156-64. [ Pubmed Link ]
- Carew JD, Nair G, Pineda-Alonso N, et al. Magnetic resonance spectroscopy of the cervical cord in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis. 2011 May;12(3):185-91. [ Pubmed Link ]
- Nair G, Carew JD, Usher S, et al. Diffusion tensor imaging reveals regional differences in the cervical spinal cord in amyotrophic lateral sclerosis. Neuroimage. 2010 Nov 1;53(2):576-83. [ Pubmed Link ]
Presentations and Lectures
- Zeier Z, Esanov R, Andrade N, Benatar M, Wahlestedt C. Epigenetic modeling and therapeutic targeting of the expanded C9ORF72 locus. Platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.
- Benatar M, Reilmann R, Schubert R, Reyes E, Seleski C, Cooley A, Wuu J. Quantitative Motor Testing: Biomarker of Pre-Symptomatic ALS? Platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.
- van Blitterswijk M, Finch NA, Wang X, Baker MC, Heckman MG, Gendron TF, Bieniek KF, Wuu J, DeJesus-Hernandez M, Brown PH, Chew J, Jansen-West KR, Daughrity LM, Nicholson AM, Murray ME, Belzil VV, Lee EB, Josephs KA, Parisi JE, Knopman DS, Petersen RC, Petrucelli L, Boeve BF, Graff-Radford NR, Asmann YW, Dickson DW, Benatar M, Bowser R, Boylan KB, Rademakers R. Expression of homeobox genes and transthyretin is changed in C9ORF72 expansion carriers. Platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.
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