Cyclosporin, a direct mitochondrial neuroprotectant, substantially extends life in ALS mice. Mitogard®, the only cyclosporin formula designed for cerebrospinal fluid administration, was developed because systemic cyclosporin does not easily reach the brain and spinal cord. Dose escalation toxicology and pharmacokinetics safety studies of intrathecal Mitogard® will be performed under Good Laboratory Practices (GLP) by Northern Biomedical Research, enabling Investigational New Drug (IND) approval for ALS clinical trials.
ALS or Lou Gehrig's disease causes progressive death of motor neurons in the brain and spine leading to paralysis and death usually within three years. There are as many as 30,000 Americans with ALS with 5,000 dying each year.
Principal Investigator Jenny Karlsson, Ph.D., is Maas BiolAB’s Director of Preclinical Development. Dr. Karlsson received her doctorate at the Wallenberg Neuroscience Centre, Lund University, Sweden. She researched cyclosporin neuroprotection in ALS mice at the University of Hawaii and was an Assistant Professor at the University of New Mexico College of Pharmacy before joining Maas BiolAB. “I am very excited to take this promising drug from experimental research onto the path of FDA approval and clinical treatment of patients,” said Dr. Karlsson. “My personal goal is to deliver a safe and effective treatment for this rare and terrible disease.”
Marcus Keep, M.D., CEO of Maas BiolAB, stated, “This translational funding from the NIH is a substantial leap toward treating ALS patients. While the patients are few, the commitment from the NIH demonstrates their understanding of the importance of taking a bold chance on a promising therapy for real disease.”
Maas BiolAB, LLC is a private company in Albuquerque, NM, developing Mitogard®, the only cyclosporin formula designed for CSF delivery, to treat amyotrophic lateral sclerosis (ALS). Maas BiolAB holds world patents for cyclosporin neuroprotection against chronic neurological diseases and has FDA Orphan Drug Designation for cyclosporin to treat ALS. Maas BiolAB is located in the Technology Ventures Corporation Incubator (TVC SBIR Outreach Center director Barbara Stoller). NeuroPharma AB is a research partner developing NeuroSTAT®, an intravenous cyclosporin formula to treat traumatic brain injury. (http://www.maasbiolab.com)