Unraveling the promise of genetics for treating progressive illness

May 11, 2021

For almost every major common disease, researchers have less understanding of the severe forms than milder cases. And as a result, people with severe forms of diseases often have few treatment options available.  

Thus, the significant unmet medical need for many diseases ranging from multiple sclerosis to Alzheimer’s is to halt disease progression and treat severe forms of the disease. “Most patients eventually do progress. We don’t understand what is causing that progression,” said Dr. Jeffrey Gulcher, chief scientific officer of Genuity Science (Boston). 

What causes disease progression? 

This basic concept concerning disease severity is evident in a range of clinical areas. Consider, for instance, non-alcoholic fatty liver disease, where abnormal amounts of fat are stored in the liver. Roughly one in four people in the U.S. have the condition, which can progress to non-alcoholic steatohepatitis (NASH) in a limited number of individuals. “Most of us can walk around fine with plenty of fat in our liver, and it doesn’t really affect us,” Gulcher said. “But a small percentage of people who do have fatty liver disease ultimately develop fibrosis or cirrhosis.” 

A similar theme is evident in diseases ranging from asthma to multiple sclerosis (MS). “In MS, the unmet medical need is for the progressive forms of the disease, where after each attack, the patient doesn’t fully recover,” Gulcher said. 

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