Video launched about the use of sheep to treat neurological diseases

BROOKINGS, S.D. – The South Dakota Agricultural Heritage Museum (SDAHM) and the Shepherd’s Gift: GM1 for HD launched GM1 Sheep Can Treat Neurological Diseases, an animated video outlining how sheep can play a role in treating neurological diseases. This video débuted in the exhibit The Unspun Tale: Sheep in South Dakota, at the South Dakota Agricultural Heritage Museum, but is now available to view and share on YouTube.

The video discusses how sheep with the recessive gene disorder Gangliosidosis have the potential to treat neurological diseases by providing the GM1 Ganglioside molecules (GM1) needed for proper nerve cell function. Patients with Parkinson’s, Huntington’s and Alzheimer’s and other neurologic diseases have a deficiency of GM1 molecules in their nerve cells. The film answers basic questions about the science behind the research into treatment options and explains how people can get involved in bringing this treatment to patients afflicted with these diseases.

“We wanted to include information about the GM1 Gangliosidosis research to treat neurological diseases in the exhibit because it uses sheep and research that is being done in South Dakota,” stated South Dakota Agricultural Heritage Museum Director Gwen McCausland. “I personally believe it has the potential to both make a huge impact in treating neurological diseases and for the sheep industry. We hope our exhibit introduces new audiences to this research.”

The South Dakota Agricultural Heritage Museum wrote the narration for the video and worked with SoDak Animation to design the short film. The Shepherd’s Gift: GM1 for HD and SDAHM co-sponsored this project.

Why sheep?

In the early 1990s, the Italian pharmaceutical company Fidia Pharma Spa, pioneered the development of a drug platform harvesting GM1 from cattle. The company was in phase II of clinical trials to treat Parkinson’s disease when Europe had an outbreak of Mad Cow disease, stopping the use of cattle in the process of further development of this drug.

At that time veterinarian Dr. Larry Holler, currently of White, S.D., was working on his Ph.D. studying GM1 Gangliosidosis in a new sheep model. When the Mad Cow disease outbreak happened, he realized the potential these sheep had to treat neurologic disease and pursued research using sheep as a safe and viable source of GM1 molecules.

In 2012, researchers at the University of Alberta proved that an infusion of GM1 into the brain restored normal motor behavior in mice with Huntington’s disease and demonstrated the potential to reverse the damage created by this neurological disease.

Dr. Holler’s research shows that sheep with Gangliosidosis have up to 40 times the amount of GM1 molecules compared to normal sheep, providing a safe and natural source of GM1 for treatment of neurological diseases. For over 20 years, Dr. Holler and his wife, Sue, have dedicated their life’s work to making this research into a viable treatment option.

What is GM1?

GM1 Ganglioside (GM1) is a natural glyco (sugar) lipid (fat) molecule found in the membrane of cells in all mammals. GM1 molecules are concentrated on cells found in the nervous system. They serve as the communication port for the cell. Other cells, enzymes and viruses attach to a GM1 molecule to communicate complex processes to the cell. Once it is used, the GM1 goes back into the cell to be recycled into new GM1 molecules.

What is Gangliosidosis?

Gangliosidosis is a genetic disorder where the body lacks the enzyme needed to break down the used GM1 molecules in the cell. The used GM1 molecules fill up the cell, clogging it until the cell stops working, ultimately causing death. Sheep with Gangliosidosis are harvested a few months after birth and the GM1 molecules can be extracted for medicine.

Can you raise a whole herd with Gangliosidosis?

No. Gangliosidosis is a recessive gene. If two parents are carriers, their offspring have a 25% chance of getting this disease, 50% chance of being a carrier, or 25% chance of being normal. Research is being conducted to increase these percentages.

Why isn’t this available for patients right now?

GM1 molecules are naturally made by mammals and cannot be recreated in a lab. This causes issues for pharmaceutical companies trying to mass-produce a drug for thousands of patients. It is estimated that it would take one or two sheep with Gangliosidosis to treat one patient for one year. Multiply this by the number of patients with Parkinson’s, Huntington’s, and other neurological diseases, and that would require millions of sheep. The focus of Dr. Holler’s research is creating a viable, safe, plentiful source of GM1 molecules to treat patients. His research has developed a new, verified, ovine (sheep) source for GM1 ganglioside production that will exceed the levels previously found using cattle by 30 to 40 times, and provide needed raw materials for pharmaceutical applications.

Is there a large enough supply of sheep?

There are many producers open to introducing this recessive gene into their flocks. It all comes down to the economics of supply and demand. When wool was a great source of income, there were millions more sheep than there are today. If this treatment makes it to market, the demand for sheep with Gangliosidosis will increase and may offer an additional value-added product for sheep producers.

Path to approval

To get this treatment into the hands of patients, it needs to be approved by the U.S. Food and Drug Administration (FDA). Dr. Steven Hersch, a clinician and researcher at Harvard/Massachusetts General Hospital, has been working with Dr. Holler on this research. In January 2015, the FDA approved the sheep as an acceptable source of GM1 to progress towards an Investigative New Drug (IND) application, which would lead to human clinical trials.

The clinical trial phase requires additional funding. Funding needed to pay for the research is scarce. The Holler family has been utilizing private funding to support the research as part of a grassroots approach.