An Alzheimer's drug to be tested mostly on an extended family predisposed to the disease will mark the first large-scale trial of an experimental treatment on people who don't yet show signs of memory loss.
The $100 million trial, by a group of government and academic researchers and the drug maker Genentech, will test the injectable drug crenezumab to see if it could prevent cognitive decline in 300 people who have a genetic mutation that makes it likely they would experience symptoms by age 45.
Most of these participants, some as young as 30, will be members of one extended family in Colombia, but a small group of unrelated individuals in the U.S. also will be studied, according to Eric Reiman, executive director of the Banner Alzheimer's Institute in Phoenix, which is part of the collaboration.
Crenezumab, from Genentech, a unit of Roche Holding AG,targets a sticky protein in the brain called amyloid that is thought to contribute to Alzheimer's when it clumps together. The trial also will seek to understand whether the amount of amyloid in one's brain could help predict later cognitive decline—a so-called biological marker. If it works, future trials could potentially use amyloid levels to determine whether a treatment is working rather than waiting to see if patients' memory worsens.
The trial was announced Tuesday at the unveiling of the National Plan to Address Alzheimer's Disease. That federal program aims to come up with effective treatment and prevention strategies by 2025 by bulking up on research funding, training health professionals and improving public-private partnerships.
Alzheimer's, a progressive brain disease, affects some five million Americans now and is projected to affect 8.7 million by 2030. The Alzheimer's Association, a research and advocacy group, estimates $200 billion will be spent this year on medical care for the disease.
No existing medications slow the course of the disease, and efforts to develop new drugs have been slow. Many experts believe medication would have the most benefit early in the course of the disease, ideally before an individual begins showing symptoms. However, studies that are designed to stave off memory loss are expensive and time-consuming because patients may not develop symptoms for years.
"We see this as a cornerstone of a larger effort to help launch a new era in Alzheimer's research," said Dr. Reiman.
William Thies, chief medical and scientific officer of the Alzheimer's Association, called the trial "ambitious" because crenezumab, which is in midstage clinical testing, is yet unproven in changing memory symptoms. The Alzheimer's Association isn't involved in the trial.
There have been few trials on how to prevent Alzheimer's and none using anti-amyloid compounds. One government-funded trial that examined whether the herb Ginkgo biloba could prevent Alzheimer's and related symptoms cost more than $20 million. Typically, trials of experimental Alzheimer's drugs are conducted on individuals who have started to show memory problems.
Several companies submitted proposals for their investigational compounds, and Genentech's was selected after vetting by an independent academic advisory group considering potential safety issues and efficacy, according to Dr. Reiman.
Other compounds, including Pfizer Inc.'s and Johnson & Johnson's bapineuzumab and Eli LillyLLY & Co.'s solanezumab, are in late-stage clinical trials.
The new trial is expected to begin in early 2013. Patients will be treated for as long as five years, according to Dr. Reiman.
Francis Collins, director of the National Institutes of Health, said in a speech to the Alzheimer's Research Summit Tuesday that the federal research agency would contribute $16 million over five years toward the prevention study. The Banner institute contributed an additional $15 million, and Roche is funding the rest.
The NIH also has made a $7.9 million grant for a study testing the effectiveness of an insulin nasal spray in treating patients in the early stages of cognitive impairment or Alzheimer's disease over the course of a year.
(Source: online.wsj.com)
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