Unraveling the promise of genetics for treating progressive illness 

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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 …

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What is needed to develop disease-modifying therapies for Parkinson’s and Alzheimer’s 

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Developing disease-modifying therapies for neurodegenerative diseases remains a pressing need.

The incidence of neurodegenerative disease is ramping up in the U.S. and elsewhere as much of the global population ages. One out of three seniors dies with Alzheimer’s disease or another form of dementia, according to the Alzheimer’s Association.

Parkinson’s disease is also becoming more widespread. Between 2015 and 2040, the number of people with Parkinson’s could nearly triple, rising from 6.3 to 17.5 million.

But developing drugs that can slow or stop the progression of such diseases poses a significant challenge for drug developers. Eli Lilly’s donanemab, for instance, showed promise earlier this year in treating Alzheimer’s in a Phase 2 study summarized in NEJM.

Another Alheimer’s candidate, aducanumab from Biogen, has also shown promise, although late l…

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The quest for a disease-modifying drug for Parkinson’s disease continues

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The need for therapies that change the course of Parkinson’s disease is considerable.

“There are zero disease-modifying treatments for Parkinson’s,” said Dr. Allan Levey, professor and chairman of Emory University’s department of neurology. 

Parkinson’s disease affects some 10 million people worldwide, according to the Parkinson’s Foundation. 

Current drugs for Parkinson’s make it possible to “reverse symptoms pretty well for many people for many years,” Levey said. “But the reality is their symptoms are sort of masked. The disease is still getting worse on a daily basis.” 

Most Parkinson’s treatments focus on managing dopamine levels in the brain. The basis of that strategy traces back to formative research in the 1960s involving levodopa. Today, levodopa-based therapies continue to b…

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