By JONATHAN D. ROCKOFF
Eli Lilly & Co. is taking its Alzheimer's disease research in a new direction, a move that deepens the drug maker's efforts to find tests and treatments for the memory-robbing disorder despite a costly setback last year.
Like its rivals, Lilly had focused on the sticky plaques that build up in the brains of Alzheimer's patients, but today it is also setting its sights on some twisted fibers that are considered another hallmark of the disease, company officials said. Lilly is in the early stages of probing several potential treatments targeting these neurofibrillary tangles of a protein called tau, and just bought the rights to two tests for measuring the tangles in the brain, the officials said.
"The most meaningful impact in Alzheimer's might involve targeting multiple pathways and using combinations of drugs," said Dan Skovronsky, Lilly's vice president of tailored therapeutics and CEO of its Avid Radiopharmaceuticals molecular imaging subsidiary.
Alzheimer's is a disease affecting 36 million people world-wide and five million in the U.S. The few approved medicines, which can only ease the symptoms, have about $3 billion in yearly sales. Analysts say the market could be $10 billion or higher if a drug was found to slow or halt the disease, but that has proven difficult.
Lilly has been studying an agent that targets the sticky amyloid plaques that can be seen in Alzheimer's patients. After reporting last year that its Alzheimer's agent called solanezumab failed to work in two late-stage trials, the company decided, after seeing positive signs in patients with an earlier and milder form of the disease, to conduct a new trial testing the experimental drug in those patients.
In the wake of such setbacks attacking amyloid plaques, researchers are taking a fresh look at the tau protein and neurofibrillary tangles as targets for drug development, said Lon Schneider, who directs the California Alzheimer's Disease Center at the University of Southern California. He said he has consulted for Lilly and other drug makers.
Tau is a protein that plays a vital role in the life of neurons. Its buildup into tangles kills neurons and is "tightly correlated" with Alzheimer's, Dr. Schneider said. "However it doesn't necessarily follow that" agents attacking the tangles will cure the disease, he said.
Companies, including large firms Biogen Idec Inc., Roche Holding AG and AstraZeneca PLC, are working on such agents, and will have to see what kind of impact they have, Dr. Schneider said.
"The whole field has been amyloid-centric, amyloid-driven," Dr. Skovronsky said. "But we need more than that. That's why we're investing in tau."
Lilly, which said it hasn't discussed its anti-tau program outside of scientific circles previously, began looking at tau in 2006. In 2009, the company hired Michael Hutton, a tau specialist from the Mayo Clinic, to bolster its efforts. Within the next two years, the company expects to begin testing in patients at least one molecule targeting the tangles, Dr. Skovronsky said.
The Indianapolis drug maker said it bought the rights to the tau-measuring tests from Siemens AG for an undisclosed sum.
Lilly hopes to validate and use these tests to identify the stage of a living Alzheimer's patient's disease—something that can only be determined now by giving patients questionnaires or looking at the brains of dead patients, Dr. Skovronsky said. Lilly also hopes the tests could be used to gauge the effectiveness of anti-tau and other Alzheimer's agents.
GE Healthcare's medical diagnostics unit is also developing a diagnostic imaging test against tau tangles.
Write to Jonathan D. Rockoff at jonathan.rockoff@wsj.com
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