A recent clinical study on GM1 ganglioside has shown significant promise for treating a variety of neurological diseases and disabilities. (See the study here).

Previously, the Italian pharmaceutical company Fidia developed a method to extract gangliosides from bovine brains, marketed under the brand name Cronassial. Gangliosides are vital substances found in the brain and neuron membranes, playing a critical role in neuronal growth and development.

The product was approved in several countries but was later removed from the market due to safety concerns, specifically regarding Guillain-Barré syndrome and the purity of the extract.

At the request of Fidia, my organization assembled a peer group of medical experts to review the available data. They concluded that there was sufficient evidence to support the initiation of clinical trials. As a result, I took the responsibility to plan and manage U.S. clinical studies on the bovine extract, administered in daily intramuscular doses of 40mg.

A double-blind study conducted on ALS patients under the leadership of Walter Bradley, Chairman of Neurology at the University of Vermont, showed no significant activity. Similarly, another study on diabetic neuropathy, conducted by renowned neurologist Mark Hallet at Peter Bent Brigham Hospital, also yielded no noticeable effects.

Dr. Hallet, who later became Chief of the Medical Branch of Neurology at the NIH, shared with me the findings of a Chinese clinical study that demonstrated the impressive efficacy of GM1, a purified ganglioside extract, in reducing ataxic dysfunction in patients with Spinocerebellar Ataxia Type 3, a neurodegenerative disorder.

Our negative findings in the U.S. may have been due to several factors:

• The dose used in the study may have been too low.
• Pure GM1 could be more effective than the mixture used in our trials.
• In contrast to the U.S. trials, the purified GM1 was administered intravenously, ensuring optimal availability in the body.

The Chinese study indicated that the high dose of GM1 was effective—a 400mg loading dose followed by 200mg daily. Notably, the 40mg dose in our study was ineffective, suggesting that a higher dose of Cronassial may also yield positive results.

The positive clinical results from the Chinese study were evident after just 12 weeks of GM1 administration. This opens up opportunities to conduct cost-effective, short-term efficacy studies, including the use of biomarkers, with an acceptable risk-benefit profile. This approach could replace the need for lengthy, expensive studies.

Furthermore, GM1 can be synthesized, providing a potential commercial opportunity. While I am unaware of GM1 being marketed in any country, it’s important to note that I have no financial stake in this pursuit.