Pharmaceutical companies have spent billions of dollars on Alzheimer’s research. The target has been the toxic protein, amyloid-beta.
According to a recent article in NPR News, this theory was dramatically challenged by the failure of two of Biogen’s major drug trials of the amyloid drug, aducanumab.
Aducanumab showed promise by eliminating toxic protein in the brain. It also appeared to slow the symptoms of dementia.
Phil Gutis Felt the Impact
Phil was one of the Alzheimer’s patients in the aducanumab trial. His reaction to the trial’s cancellation was: “It was like being punched in the stomach.”
Phil, age 57, was a reporter for the New York Times. He had high hopes that aducanumab would preserve what was left of his memory.
Phil’s disappointment and frustrations showed recently when he poured over photos of Abe, his dog who died last year. Phil could not remember anything about Abe or the 12 years he had him as a companion.
Phil is hoping to participate in another clinical trial as he is willing to “try anything” at this point.
Although the knee-jerk reaction after all these years of frustration would be for the scientists to give up, that is not the way scientists “operate”.
Enter Dr. Daniel Alkon
Dr. Alkon, currently President of the biotech company Neurotrope, spent a great deal of his career at the National Institutes of Health, focusing on how the “wiring” in the brain helps cells to communicate.
Dr. Alkon points out that in Alzheimer’s the brain loses that wiring. This causes a gradual loss of cognitive function. This theory led Dr. Alkon and his team to study genetically altered mice with symptoms that mimic Alzheimer’s.
The scientists observed a loss of wiring that they believed could be regenerated. Actually, it is the brain that creates the regeneration by forming new connections. This occurs in the brain cells and is controlled by the protein PKC-epsilon.
Enter Bryostatin-1
The scientists searched worldwide for a drug that could safely tweak PKC-epsilon. Years ago bryostatin had been studied at the National Cancer Institute. Dr. Alkon recalled that it could activate PKC-epsilon and might be effective against Alzheimer’s.,
The team experimented on animals with the substance that looks like seaweed and originates from minute marine animals.
The next step was to try the drug on humans. One such individual was named Frank. Dr. Alkon describes Frank’s condition when they first examined him as hallucinating and just “staring at the ceiling.”
After only a few weeks of treatment, Frank returned to some of his former activities such as playing pool and swimming. He was able to eat unaided and also to communicate.
Three studies of bryostatin followed. Two were preliminary and showed some improvement for patients with advanced Alzheimer’s. The third involved 150 patients with modest results.
Armed with these results, the Neurotrope team is planning another trial.
While Dr. Alkon and team are focusing on bryostatin, other scientists are concentrating on the modulation of the brain’s immune system or searching for drugs that defend against toxins in healthy brain cells.
Drugs are being developed to protect and improve the function of brain cells. If successful, these drugs can be used to treat Parkinson’s and other degenerative brain disorders.
