A Laser Focus on Alzheimer’s & Parkinson’s

By Tara Kirkpatrick

Critical Path Institute continues to help drive needed therapies for neurodegenerative diseases, especially Alzheimer’s disease and Parkinson’s disease.

C-Path’s Critical Path for Alzheimer’s Disease consortium, which started in 2008, has contributed to some definite wins in accelerating drug development for this condition, over the past 20 years. 

These include the first-ever computerized tool to transform clinical trial design in Alzheimer’s disease that is endorsed by the Food and Drug Administration and the European Medicines Agency. Also, the regulatory endorsement of viable drug development biomarkers in Alzheimer’s disease and Parkinson’s disease has contributed to a new era of biomarker-informed clinical trials.

“Thanks to our talented team and the fact that we had all the support from the FDA and EMA, we have worked with key opinion leaders in the field, and we were able to look at data in new ways,” said Diane Stephenson, C-Path’s VP of neurology. “So, we brought forward these modeling tools and biomarkers at a time when, again, people were uncertain as to how to link the right drug to the right patient at the right time. It’s really game-changing.”

The latest optimism centers around a protein linked to Alzheimer’s deterioration, called “tau.”. To advance the momentum of using tau imaging in clinical trials, the CPAD consortium gathered more than 50 global experts together to develop a groundbreaking standard for analyzing tau in clinical trials for earlier stages of Alzheimer’s–what C-Path calls a harmonization approach.

The Critical Path for Parkinson’s consortium, started in 2015, has achieved numerous regulatory successes for establishing biomarkers and accepted clinical trial tools.

It’s now delving deeper into sex differences in Parkinson’s pathophysiology and disease progression that can impact drug development. Long considered “an old man disease,” new Parkinson’s cases are increasingly showing up in women. 

“The frequency of younger people of both sexes with Parkinson’s is growing, to even the childbearing years,” Stephenson said. “We don’t know why. It’s probably both genetics and environment, but it really raises the urgent need for new approaches to detection and treatments.”

“And when you go and talk to women with Parkinson’s, they actually experience the disease very different,” she said. “They get diagnosed way later. They have different responses to the currently approved medications.”

This innovative initiative – called Global Evidence in Medicine for Parkison’s Disease, or GEM-PD, was recently launched in Vienna and already, many leading experts want to be involved and offer data, Stephenson said. “What’s so incredible about this whole field…everything we do is going to make a dent.”

“We are excited about this whole project because we’re sitting on a lot of data,” Stephenson said. “We have data from 15,000 patients, and no one’s probably ever, ever looked at sex differences.”

Stephenson has a personal stake in both diseases, having a mother who died from Alzheimer’s and a brother from Parkinson’s. “To watch the two people you love…suffer from the same diseases you’re working on, it was the most hopeless feeling.”

But continuing her C-Path work in neurodegeneration gives her hope for future patients. “I feel really encouraged…the science is advancing at a pace that I thought probably wouldn’t happen in my career.”

READ THE FULL SPECIAL REPORT HERE.