Lab Progress Towards Drug for Auto-Immune Disease

Researchers said Monday they had pinpointed two compounds -- one naturally derived from fasting and intensive exercise -- that show promise for combating arthritis, multiple sclerosis, gout and other auto-immune diseases.

The compounds throw a wrench into a molecular mechanism that causes inflammation, a driver of these diseases, the teams reported in Nature Medicine.

Called beta-hydroxybutyrate (BHB) and MCC950, the compounds may open up an intriguing avenue in drug research, they said.

The hope is that the mechanism they target, NLRP3, proves to be common to all auto-immune diseases, regardless of which part of the body is affected.

"Drugs like aspirin or steroids can work in several diseases, but can have side effects or be ineffective," said Luke O'Neill, a biochemistry professor at Trinity College Dublin, whose team worked with Australian scientists on MCC950.

"What we have found is a potentially transformative medicine, which targets what appears to be the common disease-causing process in a myriad of inflammatory diseases."

BHB was found separately by researchers at the Yale School of Medicine.

It is a metabolite -- a product of metabolism that results from fasting, high-energy exercise or the low-carb "ketogenic" diet.

So far, the compounds have lowered inflammation in mice with multiple sclerosis, a condition called Muckle-Wells syndrome and familial cold auto-inflammatory disease -- fever and joint pain that occur after exposure to cold temperatures.

Years of trials will be needed to see if they work on humans and are safe.