Prothena and the Warren Center for Neuroscience Drug Discovery have entered into a collaborative research agreement to develop new small molecule therapeutics for the treatment of Alzheimer’s disease in individuals with Down syndrome.
Down syndrome is a condition in which a person’s having a third copy of a specific chromosome causes developmental differences in learning, language and memory, and health conditions—including heart defects at birth. Scientists are studying the cause of Down syndrome; currently it is believed that the third chromosome is created from a random error in cell division within the involved egg or sperm or, more rarely, after fertilization.
Beyond health issues at birth, research has revealed that people with Down syndrome have a greatly increased risk of developing a dementia that is either Alzheimer’s disease or something very close to it. According to the CDC, about 30 percent of people with Down syndrome who are in their 50s have Alzheimer’s dementia, and about 50 percent of people with Down syndrome in their 60s have Alzheimer’s dementia.
“This collaboration plays to the center’s strength in small molecule central nervous system drug discovery and Prothena’s deep expertise in protein dysregulation for neurodegenerative diseases,” said Craig Lindsley, William K. Warren, Jr. Chair in Medicine, University Professor of pharmacology, biochemistry and chemistry and director of the WCNDD. “I have long been a fan of the scientific leadership at Prothena and a great admirer of their CNS antibody portfolio. Being able to work together on a small molecule effort is great for patients.”
If successful, the collaboration will deliver development candidates suitable for the initiation of IND-enabling studies. Other WCNDD scientists involved in the collaboration are Bruce Melancon, Paul Spearing, Olivier Boutaud and Carrie Jones.
“We are enthusiastic about this collaboration with Vanderbilt and its Warren Center for Neuroscience Drug Discovery,” Prothena Chief Scientific Officer Wagner M. Zago said. “People with Down syndrome have a greatly increased risk of developing dementia due to Alzheimer’s disease. The vision of leveraging unique areas of expertise from Prothena and Vanderbilt to focus and create potentially transformative new medicines for this underserved community is unprecedented.”
Prothena is a late-stage clinical company with expertise in protein dysregulation and a diverse pipeline of novel investigational therapies for neurodegenerative and rare peripheral amyloid diseases.
“We’ve been in discussions with Prothena around several areas of potential collaboration in recent years, and we’re excited that we’re moving forward with this collaboration,” said Thomas Utley, senior licensing officer at the Center for Technology Transfer and Commercialization. “The relationship among all areas of Prothena have been a great experience, and we are looking forward to seeing where things lead now.”