Research leads to potential treatment for rare disease

UofSC School of Medicine cannabinoid research results in FDA approval of new treatment for autoimmune hepatitis

Research from University of South Carolina School of Medicine investigators Mitzi Nagarkatti and Prakash Nagarkatti has led to Food and Drug Administration approval of cannabidiol (CBD), a nonpsychotropic compound found in cannabis, for orphan drug designation as a treatment for autoimmune hepatitis.

Revive Therapeutics Ltd. licensed the Nagarkattis’ patented research to develop a novel cannabinoid-based therapy. In late June, CBD received the special designation for the treatment of the rare disease, which can cause liver fibrosis or cirrhosis, and even liver failure.

"We were very pleased to have received orphan drug designation as it validates the novel research by the Nagarkattis' and the potential for CBD to treat autoimmune hepatitis," said Fabio Chianelli, president of Revive Therapeutics Ltd. "We are excited about advancing the research of the Nagarkattis with the goal to commercialize a safe therapeutic option for this rare disease."

The Nagarkattis are world leaders in cannabinoid research, and one of only a few labs in the nation licensed to work with cannabinoid compounds.

The team began their work at Virginia Commonwealth University nearly 20 years ago. Their early work found cannabinoid compounds could be used to treat certain types of cancer. Upon arriving at the School of Medicine in 2005, the team shifted their focus to studying how cannabinoids and other dietary supplements can be used to treat inflammation.

“Inflammation is the underlying cause of many diseases, including cardiovascular, neurodegenerative and autoimmune diseases, as well as obesity, aging and cancer. Our research looks at inflammation from various angles, including how naturally occurring dietary supplements can be used to prevent and/or reduce inflammation,” says Mitzi Nagarkatti, Ph.D., Carolina Distinguished Professor and chair of pathology, microbiology and immunology.

In 2008, the Nagarkattis successfully patented their findings that CBD binds to certain receptors on immune cells, mediating a wide range of effects, including anti-inflammatory properties. This research indicated CBD could be used as a treatment for various autoimmune diseases, including autoimmune hepatitis.

“By binding to the vanilloid receptors, CBD is able to suppress the processes that create inflammation, which can result in a decreased risk of liver failure and an increased life expectancy in patients with autoimmune hepatitis,” says Prakash Nagarkatti, Ph.D., Carolina Distinguished Professor and vice president for research.

The Nagarkattis anticipate clinical trials with autoimmune hepatitis patients may start in 2019. 

“We are proud to be leading the charge to get regulated cannabinoid therapies to the patients who need them. Products like CBD can be produced from hemp, a crop recently reintroduced to South Carolina. So, we believe this work can have significant health and economic benefits to our state,” says Prakash Nagarkatti.  

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