Rik van der Kant, researcher at Alzheimer Center Amsterdam, has been researching new medicines for Alzheimer's disease for years. He makes use of groundbreaking technology that allows him to test hundreds of potential medicines simultaneously. With a fellowship grant from Alzheimer Nederland, he moved to San Diego, USA, with his wife in 2013 to work in a leading Alzheimer's disease lab.
Role of cholesterol
“Here I learned to make living brain cells from skin pieces of patients with Alzheimer's,” he says. “This allowed me to investigate the role of cholesterol in the disease. I discovered that an accumulation of cholesterol in the brain cells of Alzheimer's patients directly leads to an accumulation of the toxic proteins Tau and Amyloid. That was big news, also internationally. The drug Efavirenz subsequently turned out to be suitable for reversing this accumulation.”
Testing effectiveness
Van der Kant calls it a 'eureka moment'. “You don't believe it at first, but all subsequent tests showed the same result. I came back to Amsterdam to take the clinical step, together with my colleague Jort Vijverberg. We adjusted the dose of Efavirenz and prepared everything for the clinical trial to test the effectiveness of the drug. Very special and unique to be able to do it all yourself, within the walls of Amsterdam UMC.”
“It's cool to see how a young researcher makes such a wonderful discovery,” says Vijverberg. “Compared to other cholesterol-lowering medications, Efavirenz can reach the brain much more easily and reduce cholesterol even in low concentrations. We are very curious about what this means for patients with Alzheimer's.”
Promising step
The efforts of Van der Kant and Vijverberg offer hope for new therapeutic approaches in the fight against Alzheimer's disease. “It remains a matter of first seeing and then believing, but I see it as a promising step in the right direction,” Vijverberg concludes.
Sign up for the trial
In total, forty patients with early-stage Alzheimer's (between 50 and 75 years old) are needed to participate in the clinical trial, which will last three months. More information about the trial and registration can be found here.
This article was originally posted on the website of Alzheimer Center Amsterdam. Read it here.