MedDay, a Paris, France based biotechnology company specializing in treatment of neurological disorders, has released more information regarding the design of its (MS-SPI) clinical trial that is investigating the efficacy and safety of the investigational medicine MD1003 in treatment of primary and secondary progressive multiple sclerosis (MS).
Progressive MS is a serious and severely disabling condition with a high unmet medical need,” says Dr. Peder Walberg, CEO of Medical Need, a company that identifies and satisfies unmet medical needs of individual patients and / or situations in health care, in close collaboration with research institutions, industrial partners, health care professionals, patient organizations and other key stakeholders. Dr. Walberg observes that “MD-1003 has shown great promise for these severely affected patients which currently have a very poor prognosis and may represent a significant break-through in the treatment of progressive MS.”
MD1003 is a highly-concentrated pharmaceutical-grade biotin (vitamin H) which at a dosage of 300 mg/day corresponds to 10,000 times the official recommended daily intake (RDA) for biotin. At such doses, MD1003 is no longer considered a food supplement because of potential toxicity and its new therapeutic properties at this dosage recategorize it as an active pharmaceutical ingredient.
Biotin is a key co-factor for enzymes involved in energy production and synthesis of myelin. Biotin has potentially two targets related to progressive MS: (1) it activates the Krebs cycle in demyelinated axons to increase energy production; (2) it activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of long chain fatty acids required for myelin synthesis.
MD1003 — biotin at active pharmaceutical concentration administered at doses of 100 – 300 mg /day — is thought to have both pro-myelinotic effects and to enhance energy supply for nerve impulse transmission. A patent protecting the dose and use in multiple sclerosis (MS) has been allowed in both the U.S. and Europe. The medicine’s mode of action potentially influences two targets related to progressive MS: 1) it activates acetyl-CoA carboxylases (ACC1 and ACC2) the rate-limiting enzymes in the synthesis of long chain fatty acids required for myelin synthesis; and 2) it activates the Krebs cycle in demyelinated axons to increase energy production.
MD1003’s proof of concept has been demonstrated in a pilot open label study involving 23 subjects with primary and secondary progressive MS. Results were encouraging, with up to 90 percent of subjects exhibiting clinical improvement over time. Treatment efficacy was also assessed using electrophysiology studies and magnetic resonance spectroscopy. Results were published this year in the Journal of Multiple Sclerosis and Related Disorders.
The Open Access paper, entitled “High doses of biotin in chronic progressive multiple sclerosis: A pilot study“ (DOI: http://dx.doi.org/10.1016/j.msard.2015.01.005), is coauthored by MedDay co-founder and Chief Executive Officer Frederic Sedel, with Caroline Papeix, Agnes Bellanger, Valerie Touitou, Christine Lebrun-Frenay, Damien Galanaud, Olivier Gout, Olivier Lyon-Caen, and Ayman Tourbah.
The aim of the uncontrolled, non-blinded, proof of concept pilot study is to assess the clinical efficacy and safety of high doses of biotin in patients suffering from progressive MS. The coauthors note that biotin activates enzymes involved in energy production and myelin synthesis, and 91.3 percent of SPMS or PPMS patients improved clinically with high doses of biotin. Improvements were observed in chronic optic neuropathy, homonymous hemianopia or myelopathy, and in all cases improvement was delayed from two to eight months following the treatment’s onset.
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