Bloomberg News

Alzheimer’s Therapy May Come From Effort to Revive Failed Drugs

April 11, 2014

NIH Director Francis Collins

National Institutes of Health Director Francis Collins speaks with editors and reporters at a Bloomberg Government breakfast Q&A session in Washington, D.C. on April 11, 2014. Photo: David Banks/Bloomberg

An effort to find new uses for drugs that have failed in clinical trials may offer hope for Alzheimer’s patients, a top U.S. health research scientist said.

Research began a year ago on a therapy targeting another disease “is looking pretty interesting” in Alzheimer’s, National Institutes of Health Director Francis Collins said today at a breakfast sponsored by Bloomberg Government in Washington.

The NIH awarded $12.7 million to nine academic groups in June to help fund research in eight disease areas as part of a pilot initiative. Eight drugmakers including New York-based Pfizer Inc. (PFE:US) and Paris-based Sanofi (SAN) offered 58 failed treatment candidates relegated to companies’ freezers, Collins said.

“A year from now, I think we’ll know if we’ve hit a home run in there somewhere,” he said at the breakfast.

Since 1998 there have been more than 100 attempts to develop a treatment for Alzheimer’s and all have failed. More than 5 million Americans have Alzheimer’s disease, the most common form of dementia, a patient population that is expected to triple by 2050, according to the Alzheimer’s Association. As many as a half-million people in the U.S. may die from the disease each year, making it the country’s No. 3 killer, researchers reported last month in the journal Neurology.

“These compounds have already gone through most of the pipeline,” Collins said of the NIH-funded research. “You can go almost immediately to a clinical trial. And if it works, wow, you’ve saved 10 years.”

‘Very Preliminary’

Still, Collins said in a follow-up e-mail, the Alzheimer’s finding is “very preliminary.”

NIH also is teaming with pharmaceutical companies to study new approaches to treat Alzheimer’s, diabetes, lupus and arthritis. Collins said he worked with the companies’ research and development units for three years to make the “unprecedented” partnership happen. The research will be made public for any scientist to use.

About 60 percent of mid-stage clinical trials on drugs fail and about 40 percent of final-stage trials don’t pan out.

“The chance when you start a drug development program of actually getting FDA approval 10 or 12 years later is well under 1 percent,” Collins said, referring to the U.S. Food and Drug Administration. “A lot of the really well-characterized drug targets were identified a while back. Now we’re into the space that’s more difficult and, I think, the data that companies were depending on to make their bets about drug targets were clearly not that good.”

NIH has struggled the last decade to secure the government money necessary to fund research. After the agency’s budget rose to $27 billion in 2003, NIH’s funding has remained almost unchanged. If inflation is considered in the equation, it’s worse, Collins said, meaning the agency has lost 25 percent of its purchasing power.

“We are in a real squeeze,” he said.

To contact the reporter on this story: Anna Edney in Washington at aedney@bloomberg.net

To contact the editors responsible for this story: Reg Gale at rgale5@bloomberg.net Andrew Pollack, Bruce Rule


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