Funds to map brain may speed treatments
Spotlight On Biotech
Updated 8:31 pm, Tuesday, February 19, 2013
Roche and Eli Lilly, two drugmakers racing to develop treatments for some of the least-understood brain disorders, may gain the most from a U.S. government boost in funding to fully map the human brain.
The National Institutes of Health is in the planning stages of a massive effort called the Brain Activity Map to understand how neurons actually process information, according to Story Landis, director of the agency's Neurological Disorders and Stroke division. It may take at least five years to develop the tools needed to map out all this information, she said.
'The last great frontier'
"A lot of people see neurological research as the last great frontier in biomedical science," Landis said Monday. "There are lots of people looking at individual circuits. This would take the science to a whole new level."
Better funding should improve the odds for finding effective Alzheimer's, Parkinson's and autism treatments, which so far have been plagued by failures because much of how the human brain works remains a mystery. There have been 101 unsuccessful attempts to develop a treatment for Alzheimer's disease since 1998, including recent setbacks by Pfizer, Johnson & Johnson and Lilly, according to the Pharmaceutical Research and Manufacturers of America.
"This attention to neuroscience research is exciting," said Luca Santarelli, the head of neuroscience at Swiss company Roche, Europe's second-largest drugmaker by sales. "Advancements in the understanding of the molecular and circuit basis of brain disorders will dramatically advance the development of treatments for patients."
Alzheimer's, the most common form of dementia, is the sixth-leading cause of death in the U.S., according to the Alzheimer's Association. The number of people with the disease is expected to double within 20 years as the world's population ages, to as many as 65.7 million in 2030 and 115 million by 2050, the World Health Organization said last year. There is no cure, only therapies to address symptoms.
Drugs in pipeline
Roche has 16 drugs in development for neurological disorders, including four for Alzheimer's disease and two for autism. Lilly is working on 10 neurosciences drugs, while Pfizer has 11 in testing, including two for Alzheimer's.
The NIH initiative would be akin to what happened with the Human Genome Project, where DNA sequencing was already occurring at a small scale before the project to map the entire human body was started, said George Church, a genetics professor at Harvard Medical School and director of the Personal Genome Project.
While researchers can already track the activity of perhaps 100 or so brain neurons at a time, the new opportunity is to connect those on a large scale and then measure activity at the detail level and the whole brain level, Church said.
"This is about basic technology as well as knowledge," Church said. "We stand at the doorway of a fundamental intersection of nanochemical sensors that can read or write neurons and synthetic biology. It's overripe for this merger."
It won't be easy, said Maria Carrillo, vice president of medical and scientific relations at the Alzheimer's Association. The initiative should go hand-in-hand with others, such as the National Alzheimer's Plan to develop effective treatments by 2025, she said.
"Even though the human genome is a changing entity, the human brain is incredibly more complicated because we are all so different," Carrillo said.
Still, it has been the limited knowledge of how the brain actually works that has been holding back the development of new drugs, said Richard Mohs, who leads early-stage neuroscience research at Lilly. For conditions like depression and schizophrenia, researchers don't fully understand what causes the symptoms, and a detailed map of the brain's circuitry could help, he said.
"The brain is so complex, and there are so many possible biologic targets," Mohs said. "Once you do have a target, trying to screen drugs for activity against that target in living human beings is very hard."
Drugmakers are testing more than 600 drugs for psychological disorders, according to PhRMA.
Like the Human Genome Project, the value of brain mapping "is in the revelation of connections that one would otherwise never have thought existed," he said. "These networks will be disrupted in major diseases. If we don't know they're there, we can't develop drugs that target brain disease networks. Brain activity mapping will undoubtedly provide many new targets for a new generation of drugs for brain diseases."