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Abstract #1967

A Single-Center, Phase 1, Open Label, Dosage-Escalation Study of Creatine Monohydrate in Subject with Amyotrophic Lateral Sclerosis

Eva-Maria Ratai1,2, Nazem Atassi, 2,3, Stuart Wallace, 2,4, Jeffery Bombardier1, David Greenblatt5, Merit Cudkowicz, 2,3, Allitia Dibernardo, 2,3

1Department of Radiology, A. A. Martinos Center for Biomedical Imaging, Massachusetts General Hospital, Charlestown, MA, United States; 2Harvard Medical School, Boston, MA, United States; 3Neurology, Massachusetts General Hospital, Charlestown, MA, United States; 4Psychiatry, Massachusetts General Hospital, Charlestown, MA, United States; 5Pharmacology & Experimental Therapeutics, Tufts University School of Medicine, Boston, MA, United States


The purpose of this study was to evaluate the serum pharmacokinetics of orally administered creatine in subjects with ALS and to assess whether oral intake produces increased concentrations of creatine in the brain utilizing in vivo MR Spectroscopy. Six ALS patients were enrolled in this open-label pilot study. Patients escalated weekly through 3 different dose levels. Creatine serum levels increased with daily use of 5, 10, 15 gm BID. MR Spectroscopy results are suggestive that creatine crosses the blood brain barrier when given at a high dose of 15 gm BID. Furthermore, glutamine and glutamate levels decreased post treatment.