Alzheimer's Meeting to Focus on Progress

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BOSTON -- Although no cure for Alzheimer's disease is yet apparent, researchers gathering here for a week-long meeting will see ample evidence that one -- or more -- may be just over the horizon.

Novel treatment approaches, new diagnostic and treatment-monitoring technologies, and recent findings on potentially modifiable risk factors for the disease are among the topics to be covered at the Alzheimer's Association International Conference, which formally begins Sunday, although pre-meeting activities are underway today.

Already, conference organizers have released results from three studies by Boston-based researchers, and announced the availability of whole-genome sequencing results from an 800-person cohort in a "big data" project.

Findings from the Boston studies included discovery of new inhibitors of the gamma-secretase enzyme -- the target of several drugs with disappointing results in phase III trials -- that showed promise in animal models of succeeding where the others failed.

Another group found that a combination of two different types of brain scans plus cognitive tests could identify patients in the earliest stages of Alzheimer's disease.

And a third research team, examining genomic data from more than 1,600 individuals, uncovered variants in five genes that were associated with the speed of cognitive decline in Alzheimer's patients.

The meeting will also feature the first detailed report on a closely watched, failed phase III trial of intravenous immunoglobulin (IVIG). Topline results announced by the product's manufacturer in May had indicated that patients receiving IVIG infusions showed no slowing in cognitive or functional decline relative to placebo.

But a subgroup analysis suggested the possibility of benefit in certain patients, about which AAIC attendees will be eager to learn more.

Other areas to be addressed at the meeting include:

  • New studies of drugs targeting beta-amyloid protein plaques, including the phase III trial of solanezumab in individuals with "preclinical" Alzheimer's disease just getting underway
  • Novel imaging technologies and other biomarkers for detecting and measuring Alzheimer's disease pathology
  • Reviews of national goals on preventing and treating Alzheimer's disease
  • Reaction to the Centers for Medicare and Medicaid Services' proposal to disallow Medicare coverage of beta-amyloid brain scans except in approved clinical trials

Sam Gandy, MD, PhD, a prominent Alzheimer's disease specialist at Mount Sinai School of Medicine in New York City, told MedPage Today that he expects AAIC attendees would also be talking about the FDA's recent draft guidance document detailing the agency's data demands for new Alzheimer therapies.

In it, the FDA said it would not approve new drugs solely on the basis of biomarker data, such as results from brain amyloid scans or levels of amyloid proteins in blood or cerebrospinal fluid. The drug's maker must demonstrate positive effects on clinical measures of cognitive and functional ability.

But officials did indicate that, in patients with early and mild symptoms, relatively subtle improvements in clinical outcomes could be the basis for approval.

That announcement has been "the biggest news for the field," Gandy told MedPage Today. "This means that new drugs are now realistically likely to be approved in the next 5 years."

The "big data" project unveiled Friday involved some 200 terabytes of genomic information obtained from some 800 individuals participating in the Alzheimer's Disease Neuroimaging Initiative.

In collaboration with the Brin Wojcicki Foundation, the Alzheimer's Association announced that it would be made available to researchers worldwide through the Global Alzheimer's Association Interactive Network (GAAIN), funded with $5 million from the association.

The group said it represents "the first massive whole-genome sequencing project in Alzheimer's disease." GAAIN will be led by Maria Carrillo, PhD, vice president of medical and scientific relations at the Alzheimer's Association, along with academic researchers from the U.S. and Italy.