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From the Day Neuromuscular Research Laboratory (Drs. Cudkowicz and Brown), Massachusetts General Hospital (MGH), Charlestown; the Department of Neurology (Drs. Cudkowicz and Brown, and P.M. Sexton and T. Ellis) and General Clinical Research Center (D.L. Hayden), MGH, Harvard Medical School, Boston, MA; the Department of Pharmaceutical Sciences (P.R. Gwilt), College of Pharmacy, University of Nebraska Medical Center, Omaha, NE; and Transcend Therapeutics, Inc. (Dr Wahlen), Cambridge, MA.
Address correspondence and reprint requests to Dr. Cudkowicz, Department of Neurology, Massachusetts General Hospital, 32 Blossom Street, Rm. ACC 836, Boston, MA 02114; e-mail: Cudkowicz{at}helix.mgh.harvard.edu
In 13 subjects with ALS, we studied the safety and pharmacokinetic properties of Procysteine, a cysteine prodrug that increases levels of intracellular glutathione. We found that oral administration of Procysteine was safe. Procysteine enters CSF after both IV and oral dosing and accumulates to significant levels in CSF. We also observed that CSF levels of glutathione fall dramatically with aging.
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