Researchers at Weill Cornell Medical College may have identified a possible new treatment for Alzheimer’s diseases after concluding a preliminary, small-scale study with Baxter International. The focus of the study was Baxter’s Gammagard, an intravenous immunoglobulin treatment for patients with immunodeficiency and its effects on slowing or even stopping the progression of Alzheimer’s.
“The IVIG study was a Cornell investigator-initiated study. We conceived and designed the study and then approached Baxter and other sources for support,” said Prof. Normal Relkin, neurology and neuroscience, lead researcher at Weill Cornell Medical College.
This study was Phase II of a three-phase study. In Phase I, researchers discovered the existence of a new class of naturally occurring antibodies in human blood against beta amyloid oligomers, the toxic proteins in Alzheimer’s disease.
“This could be part of an innate defense mechanism against Alzheimer’s and other age-related neurodegenerative disorders,” stated Prof. Marc Weksler, medicine, senior investigator in the Phase I study of medicine at Weill Cornell Medical College, in a Cornell press release.
“Phase II was a six month randomized double-blind, placebo-controlled study in 24 patients with probable Alzheimer’s. [It] cost close to $2 million dollars all told, and the majority of that did come from Baxter with supplements from the National Institutes of Health, Citigroup Foundation, and private donors,” Relkin said.
“Phase III will be an 18 month double-blind, placebo-controlled study in 360 patients. Two different doses of Gammagard will be compared to placebo. Phase III is scheduled to begin in the beginning of 2008 and should be completed by 2010-2011. [It] will cost in excess of $30 million, $8 million of which will come from a grant from the NIA, the [remaining] balance from Baxter.”
Alzheimer’s disease is a neurodegenerative disease commonly found in people over age 65. In the U.S., more than 5 million Americans are estimated to suffer from it and was the seventh leading cause of death in 2004, with 65,829 deaths and rising, according to the National Center for Health Statistics.
Financially, the disease costs over $100 billion per year, making it the third most costly disease in the U.S., after heart disease and cancer. The Diagnostic Center for Alzheimer’s Disease projects that 14.3 million Americans will have the disease by 2050 — a 350 percent increase from 2000.
In the most recent clinical study phase, 16 out of 24 patients reacted positively to Gammagard, displaying better cognitive response than the eight patients who were given a placebo. Responding to the encouraging findings, the Alzheimer’s Disease Cooperative Study group decided on Aug. 28 to officially pursue a multi-center Phase III study.
“The effect of this treatment is not ‘curative’ in the sense that it cannot restore brain cells that are already destroyed by the disease. It has a symptomatic benefit that is evident within a few months of initiating treatment, and a long-term effect in stabilizing the progression of symptoms. It does this over and above what the currently available medications can do,” said Relkin.
“Other forms of immunotherapy are being studied to combat Alzheimer’s disease, but this is the only line of research being carried out using human antibodies. In addition to providing a new form of treatment, our studies of Gammagard IVIG are providing new insights into innate immunological defenses against neurodegenerative disorders, [so] in that sense, I think this can be considered revolutionary.”