Rainwater Foundation funds neuro-research April 19, 2013
A new line of prion-inoculated mice could speed up medical research to develop drug combinations to treat neurodegenerative diseases, including Alzheimer’s and other prion-related forms of dementia, the Nobel laureate who discovered prions said in Fort Worth on April 12.
Prions are infectious proteins that cause brain disorders, including Creutzfeldt-Jakob disease, frontotemporal dementias, Parkinson’s disease and Huntington’s disease and play a major role in long-term cell damage from traumatic brain injuries, Dr. Stanley Prusiner said.
Prusiner is director of the Institute for Neurodegenerative Diseases and professor of neurology at the University of California at San Francisco. He won the Nobel Prize in physiology or medicine in 1997 for his work with prions. He was keynote speaker for Research Appreciation Day at the University of North Texas Health Science Center in Fort Worth.
“Prions explain the many different forms of neurodegenerative diseases and the fact that they are sporadic, infectious and inherited,” Prusiner said. The biggest problem in finding drugs to treat or cure them has been the fact that they contain no DNA or RNA and they quickly become drug resistant.
“There’s not a single drug that stops or even slows the progression of neurodegenerative diseases,” Prusiner said. “There are zero drugs in the pipeline for Alzheimer’s – 600 in the pipeline for cancer and zero for Alzheimer’s. There’s nothing,” he said in an interview after his presentation.
The few drugs currently available, such as levodopa used for Parkinson’s and Aricept for Alzheimer’s, seem to work for a few months and then just stop, he noted.
But scientists are now “beginning to understand the whole process,” and the new breakthrough in research animals should make it quicker and easier to test various compounds and combinations of compounds early in the disease process, with the hope of developing drugs that penetrate the blood-brain barrier and remain effective over a longer time.
Already medical researchers have demonstrated the ability to double the life span of prion-infected mice – from 100 to 200 days, the equivalent of about nine years in humans – Prusiner said.
The line of prion-inoculated mice should speed up the process to find new compounds that are effective against prion diseases so they can be added to the therapeutic mix before the mice develop resistance to the compounds that are keeping them alive.
To produce drug cocktails that remain effective over time, “we need many thousands of compounds in developing one useful drug,” Prusiner said. “When we did cell work and animal work in the past, the animal models were poor. Now we have [better] animal models … and the potency of new compounds is 10 to 100 times better. We can now work with animals and cultured cells that we know are either susceptible or resistant,” he said. “This is brand new. … It opens a whole new line of investigation. The key is to have biological systems to weed out whether we are going forwards or backwards or standing still. … I’m sure we will find some [compounds] that will work.’’
He is optimistic that such drugs will be available in the next five to seven years despite federal budget cuts that threaten research funded by the National Institutes of Health and other federal agencies.
“I am 100 percent hopeful,” he said. “One of the great things about science is you can go to bed and wake up in the morning and you or somebody else on the planet can have discovered something that changes everything – like that!” he said, snapping his fingers.
Prusiner is part of the Tau Consortium, a group of 30 research scientists from around the world that was founded and funded by the Fort Worth-based Rainwater Charitable Foundation. The foundation was established by Fort Worth billionaire investor Richard Rainwater in 1991. Rainwater was diagnosed in 2009 with progressive supranuclear palsy, a rare neurodegenerative disease.
To date, the foundation has given more than $265 million to support higher education, at-risk children and medical research, in particular research focused on finding cures for neurological diseases, according to the foundation’s website.
Rainwater is the investment banker formerly associated with Bass Enterprises who resurrected the Disney empire and founded or co-founded numerous successful companies, including Ensco International, Columbia Hospital Corp., Mid Ocean Limited and Crescent Real Estate Equities.
Progressive supranuclear palsy resembles Parkinson’s in the beginning and then progresses more like Alzheimer’s. It is grouped with Parkinson’s, Alzheimer’s and other degenerative brain diseases in a category known as “tauopathies” because they are characterized by tau – tangled clumps of protein that grow and multiply in the brain.
The Tau Consortium is an international group of scientists organized and funded by the Rainwater Foundation to investigate the causes and potential treatment for tauopathies.
Prusiner thanked the Rainwater Foundation and decried the fact that in recent years hundreds of thousands of young researchers have been trained to find the cause and the cure as well as ways to prevent neurodegenerative diseases, and now funding for research has been drastically cut by the federal government.
“It sucks – it just sucks. It’s really hard right now. … To expect private funding to take up the slack is really not okay,” he said. “There are now 5.3 million people with Alzheimer’s disease in this country; there will be 13.4 million by 2050. ... We have 500,000 new cases a year, and there are 100,000 dying each year.”
Healthpoint Biotherapeutics and the Rainwater Charitable Foundation were underwriters for the 2013 Research Appreciation Day on April 12.