Will a cure for Alzheimer's ever be found?
Unless an effective treatment is found, the costs to treat people with Alzheimer's disease will rise to more than $1 trillion by 2050, an increase from current costs estimated at $214 billion, according to data from the nonprofit Alzheimer's Association.
Affecting some 5 million people, Alzheimer's is the most common neurological condition, robbing the afflicted of their memories and so debilitating that many have to live in specialized care facilities. The disease is the only one in the top five causes of death that has no effective treatment or cure. While scientists have made progress in treating Alzheimer's symptoms, they're now focusing their efforts more on prevention and early detection, according to Dr. James Hendrix, director of the Global Science Initiative at the Alzheimer's Association.
"We have seen great progress around early detection," he said.
A cure -- which could be worth tens of billions dollars -- has proven to be elusive, however. Several drugs that scientists and drugmakers thought held promise failed during trials in recent years. Federal funding for Alzheimer's research is about $600 million, which the Alzheimer's Association considers inadequate. Last year, a blue-ribbon panel of scientists recommended to the U.S. Department of Health and Human Services that the figure be raised to at least $2 billion per year.
"Some people look at it as the golden lottery ticket," wrote John Carroll, editor-in-chief of the blog FiercePharma, in an email. "The odds against success are incredible, but if you come up with some winning data for an FDA that would like to approve something and millions of desperate patients with no real options, it would be worth a fortune, even if it's not very good."
Pharmaceutical Research and Manufacturers of America estimates 73 Alzheimer's drugs are in development, hoping to gain a foothold in what experts expect would be a multibillion-dollar market. Between 1998 and 2011, 101 drugs aimed at treating the disease failed. For every one medicine that got developed, 34 didn't even get that far.
Roche Holding recently pulled the plug on a Phase III trial of its Alzheimer's drug candidate gantenerumab because it was ineffective in patients with early stages of the memory-robbing disease. Gantenerumab is still being evaluated for as a treatment for the later stages of Alzheimer's.
In 2008, Myriad Genetics (MYGN) pulled the plug on the experimental drug Flurizan after Phase III clinical trials found the drug didn't improve cognitive functioning in an 18-month study of 1,684 patients. It spent about $200 million developing Flurizan.
Another drug called bapineuzumab, co-developed by Irish drugmaker Elan and Wyeth, failed to halt the mental decline of Alzheimer's patients. The drug wound up in the headlines again last year after former SAC trader Matthew Martoma was found guilty of insider trading for acting on an illegal trip about the study.
Eli Lilly (LLY) halted work on its Alzheimer's treatment, semagacestat, after the drug was found to be ineffective and made patients worse.
"Just imagine what people would pay or have to pay for something to ward off Alzheimer's," said pharmaceutical industry consultant Daniel Hoffman, who sees such a market being in the billions of dollars. "You would get ever person in the developed world taking it every night."
Of course, that's easier said than done, though Biogen Idec (BIIB) last month reported positive interim results for its prospective Alzheimer's treatment. Google-backed biotech firm Calico also is targeting diseases that affect the elderly.
Last month, the U.K.'s Telegraph newspaper published a story with the provocative headline "Has Stanford University found a cure for Alzheimer's disease?" Unfortunately, it overstated the importance of the California university's preliminary though promising research.
The Stanford study focused on a protein called EP2 found on a brain cell called microglia that manages inflammation and anti-inflammatory responses. It dovetails on previous work that has linked brain inflammation and Alzheimer's disease. Unfortunately, the study may not lead to a treatment for at least a decade if it happens at all.
The Telegraph article attracted considerable attention on social media, along with "anguished" emails from the loved ones of people affected by the disease, according to Bruce Goldman, a spokesman for Stanford Medical School.
Said Goldman: "They need cures as soon as they can get them, but science has to proceed in an orderly fashion."