Stephen L. DeFelice, M.D., Founder and Chairman | 3/9/2025
A recent clinical study with the GM1 ganglioside offers broad promise for the treatment of a variety of neurological diseases and disabilities (See Study).
In the past the Italian pharmaceutical company, Fidia, possessed know-how on a method to extract from bovine brains a mixture containing gangliosides marketed under the brand name, Cronassial.
Gangliosides are natural substances found in the brain and neuron membranes that serve critical functions including neuronal growth. It was approved in a number of countries but subsequently removed for safety concerns of possible Guillain Barre and the purity of the extract.
At the request of Fidia, my organization assembled a Peer group of medical authorities to review the available data, and they concluded that was sufficient evidence to support clinical trials. I agreed to plan and manage the U.S. clinical studies on the bovine extract at daily intramuscular doses of 40mg . A double-blind study was conducted on ALS patients by Walter Bradley , the Chairman of Neurology, at the University of Vermont. No activity was detected.
Another double-blind study on diabetic neuropathy was conducted at the Peter Bent Brigham Hospital by neurologist, Mark Hallet. No activity was detected.
Dr. Hallet, who subsequently became Chief of the Medical Branch of Neurology at the NIH and now retired, informed me of a recently published Chinese clinical study which reported that GM1, a purified ganglioside extract, was impressively efficacious in reducing ataxic dysfunction in patients with Spinocerebellar Ataxia Type 3, a neurodegenerative disorder.
Our negative U.S. clinical findings on the mixture may have been due to a)too low a dose or b) the pure GM1 alone is effective. Also, unlike the U.S. studies where the dose of the mixture was administered intramuscularly, the purified GM1 extract was administered intravenously assuring optimum availability. It’s also important to note is that only the high dose of GM1 was effective- a 400mg loading dose followed by 200mg daily c) The 40mg GM1 dose was ineffective which indicates that a higher dose of Cronassial may also be effective. The positive clinical results were seen after only 12 weeks of GM1 administration. This presents the opportunity to conduct creative multiple indication, small and short- term efficacy studies at low-cost, including biomarkers, at an acceptable risk-benefit profile, instead of costly, long- term studies.
Also, I understand that, though costly, GM1 can be synthesized which presents a commercial venture opportunity. I am not aware if GM1 is marketed in any country. For the record, I have no economic stake in this pursuit.