AUSTIN (KXAN) –  An Austin-based pharmaceutical company is making promising strides toward developing a new treatment for Alzheimer’s disease. 

Cassava Sciences has been researching neurodegenerative conditions for over a decade. In a recent trial, one of their medications – simufilam – showed slowed cognitive decline by 38% in patients with mild-to-moderate Alzheimer’s disease who had the medication versus patients taking a placebo. 

“In my opinion, that’s a remarkable finding,” CEO and president of Cassava Sciences, Remi Barbier, said. 

“Alzheimer’s makes cancer look like child’s play. What we’re talking about is a brain with over three billion cells, all of which are interconnected, starting to act up and decay. How do you treat 3 billion anything? It’s very, very difficult,” Barbier added. 

More often in clinical trials, one group will receive the experimental medication while another group will receive a placebo – something like a sugar pill or saline injection – to examine the drug’s effectiveness. 

In this trial, both groups took simufilam for a year. After this period, the patients were randomized to receive either the placebo or simufilam for an additional six months. Of the 157 patients with mild-to-moderate Alzheimer’s disease from 16 U.S. clinical sites, 38% showed slowed cognitive decline on the ADAS-Cog – a common test used to assess dementia severity– than those who took the placebo for six months. 

“It is a trial design that responds to the question: ‘What happens when patients are taken off a drug?’” Barbier said. 

Developing Alzheimer’s drugs

Developing a drug for neurodegenerative disorders, including Alzheimer’s Disease, is challenging, Barbier said. 

Alzheimer’s disease is a complex brain disorder that causes cells in certain parts of the brain to die and leads to progressive memory and thinking problems. It is the most common form of dementia– affecting more than six million Americans over 65 each year – and costs the U.S. healthcare system hundreds of billions of dollars annually.  

Much of the treatment development thus far has focused on targeting certain plaque proteins – called Amyloid plaques – and tau tangles in the brain associated with Alzheimer’s disease.

Cassava Sciences’ drug does not try to eliminate any plaques or tangles in the brain but focuses on addressing a misfolded protein called Filiamin A. 

Barbier said that several independent academic researchers have studied the role this misfolded protein plays in the disease, however, Cassava Sciences has a patent on the treatment.

“Without patents, we would never be able to invest the type of money and resources and people that are required in order to come up with a drug innovation,” he said.

Barbier said it takes usually around ten to 15 years for a drug to get FDA approval. Cassava Sciences has two ongoing clinical trials studying simufilam’s effectiveness and hopes to have the results of the projects in the next couple of years. 

He said he feels heartened by recent accelerated FDA approvals of some Alzheimer’s Drugs. One of those drugs was Biogen’s Lequembi, an intravenous monocular antibody injection that slows the rate of cognitive decline by 27%. 

“There is hope for patients with Alzheimer’s disease, and there’s hope beyond these monoclonal antibodies,” Barbier said. 

“The important point is that the FDA has shown a certain flexibility and willingness to listen to data, look at data and be open-minded about what it takes to treat patients with Alzheimer’s.”