Research Studies

Study Title

CRiALS PRESS-ALS (Pre-Symptomatic Studies in ALS)

Enrollment Status

Current

Principal Investigators

Michael Benatar, MD, PhD and Joanne Wuu, ScM

Study Type

Observational

Goals

Study the early regional manifestations of ALS

Enrolling

ALS patients, Controls

Eligibility

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Diagnosis of ALS and has at least one region (arm, leg, bulbar) that is unaffected based on clinical examination and EMG
Healthy controls

Study Involvement

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5 study visit (3 months apart) at UMiami over a period of 1 year
Study visits may entail:

  • Blood, urine, and (optional) CSF collection
  • MRI
  • Neurological exam
  • Neuropsychological assessment
  • Breathing test
  • Fine motor skills testing
  • Surveys/questionnaires

Primary Funding Source

ALS Recover Fund, Kimmelman Estate

Related Publications

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Benatar M, Wuu J, McHutchison C, Postuma RB, Boeve BF, Petersen R, Ross CA, Rosen H, Arias JJ, Fradette S, McDermott MP, Shefner J, Stanislaw C, Abrahams S, Cosentino S, Andersen PM, Finkel RS, Granit V, Grignon AL, Rohrer JD, McMillan CT, Grossman M, Al-Chalabi A, Turner MR; attendees of the First International Pre-Symptomatic ALS Workshop. Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases. Brain. 2021 Oct 22. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/34677606

Si Y, Kazamel M, Benatar M, Wuu J, Kwon Y, Kwan T, Jiang N, Kentrup D, Faul C, Alesce L, King PH. FGF23, a novel muscle biomarker detected in the early stages of ALS. Sci Rep. 2021 Jun 8;11(1):12062.

https://pubmed.ncbi.nlm.nih.gov/34103575

Gray E, Thompson AG, Wuu J, Pelt J, Talbot K, Benatar M, Turner MR. CSF chitinases before and after symptom onset in amyotrophic lateral sclerosis. Ann Clin Transl Neurol. 2020 Aug;7(8):1296-1306. Epub 2020 Jul 14.

https://pubmed.ncbi.nlm.nih.gov/32666680

Benatar M, Wuu J, Lombardi V, Jeromin A, Bowser R, Andersen PM, Malaspina A. Neurofilaments in pre-symptomatic ALS and the impact of genotype. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Nov;20(7-8):538-548. Epub 2019 Aug 21.

https://pubmed.ncbi.nlm.nih.gov/31432691

Gertsman I, Wuu J, McAlonis-Downes M, Ghassemian M, Ling K, Rigo F, Bennett F, Benatar M, Miller TM, Da Cruz S. An endogenous peptide marker differentiates SOD1 stability and facilitates pharmacodynamic monitoring in SOD1 amyotrophic lateral sclerosis. JCI Insight. 2019 May 16;4(10):e122768.

https://pubmed.ncbi.nlm.nih.gov/31092730

Benatar M, Wuu J, Andersen PM, Lombardi V, Malaspina A. Neurofilament light: A candidate biomarker of presymptomatic amyotrophic lateral sclerosis and phenoconversion. Ann Neurol. 2018 Jul;84(1):130-139. Epub 2018 Aug 16.

https://pubmed.ncbi.nlm.nih.gov/30014505

Gendron TF; C9ORF72 Neurofilament Study Group, Daughrity LM, Heckman MG, Diehl NN, Wuu J, Miller TM, Pastor P, Trojanowski JQ, Grossman M, Berry JD, Hu WT, Ratti A, Benatar M, Silani V, Glass JD, Floeter MK, Jeromin A, Boylan KB, Petrucelli L. Phosphorylated neurofilament heavy chain: A biomarker of survival for C9ORF72-associated amyotrophic lateral sclerosis. Ann Neurol. 2017 Jul;82(1):139-146.

https://pubmed.ncbi.nlm.nih.gov/28628244

Gendron TF, Chew J, Stankowski JN, Hayes LR, Zhang YJ, 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 LF, Elman LB, Toledo JB, McBride JD, 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 are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis. Sci Transl Med. 2017 Mar 29;9(383):eaai7866.

https://pubmed.ncbi.nlm.nih.gov/28356511

Shepheard SR, Wuu J, Cardoso M, Wiklendt L, Dinning PG, Chataway T, Schultz D, Benatar M, Rogers ML. Urinary p75(ECD): A prognostic, disease progression, and pharmacodynamic biomarker in ALS. Neurology. 2017 Mar 21;88(12):1137-1143. Epub 2017 Feb 22.

https://pubmed.ncbi.nlm.nih.gov/28228570

Benatar M, Wuu J, Ravits J. Opportunity and innovation in studying pre-symptomatic amyotrophic lateral sclerosis. Muscle Nerve. 2013 May;47(5):629-31. Epub 2013 Mar 15.

https://pubmed.ncbi.nlm.nih.gov/23494825

Related Presentations & Lectures

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Gray E (presenter), AG Thompson, Wuu J, Pelt J, Talbot K, Benatar M, Turner MR. Chitotriosidase level and activity rises in the pre- and early symptomatic phases of ALS. Platform presentation at the 30th International Symposium on ALS/MND, Perth, Australia, Dec 4-6, 2019.

McMillan CT (presenter), Wuu J, Cook P, Olm C, Duda J, Gee J, Clayman CE, Elman L, McCluskey L, Irwin DJ, Grossman M, Benatar M. Baseline and longitudinal volumetric changes associated with presymptomatic C9ORF72 repeat expansion carriers. Platform presentation at the 29th International Symposium on ALS/MND, Glassgow, UK, Dec 7-9, 2018.

Rogers ML (presenter), Shepheard S, Wuu J, Andersen P, Schultz D, Benatar M. Urinary p75 neurotrophin receptor extracellular domain: A biomarker relevant to ALS therapy development. Platform presentation at the International Symposium on ALS/MND, Boston, MA, Dec 8-10, 2017.

Benatar M (presenter), 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.

Benatar M (invited speaker). The challenge of early therapeutic intervention in ALS. Invited presentation at the 25th International ALS/MND Meeting. Brussels, Belgium, December 2014.

Benatar M (invited speaker). Identifying Biomarkers in Familial ALS. ALS Association Drug Discovery Meeting. Washington, DC, March 2012.