UC San Diego seeks patients to help test new treatment for Alzheimer’s disease

Scientists are experimenting with gene therapy to try to find an effective treatment for Alzheimer's disease.
(UC San Diego)

The Phase 1 clinical trial involves a type of gene therapy that will be used for the first time in humans.


UC San Diego is seeking patients to help test a new therapy that’s meant to slow Alzheimer’s disease and possibly improve memory, a goal that has proved to be one of the toughest challenges in medical science.

The treatment involves giving patients a genetically altered protein that is being tested for the first time in humans.

The protein is known as brain-derived neurotrophic factor, or BDNF, which plays the dual role of preserving brain cells and stimulating cell growth. Scientists say people with Alzheimer’s have diminished amounts of the protein.

A team led by Dr. Mark Tuszynski is using gene therapy to place healthy BDNF inside a harmless virus that will carry the protein to specific areas of the brain where it might have a therapeutic effect. The therapy is designed to focus only on degenerating cells to avoid triggering possible side effects, including seizures.

“We are hoping that the BDNF protein will slow down or stop cell death, literally build new connections between cells in the brain and, as a result, slow down memory loss or actually improve memory,” Tuszynski said.

He said he has successfully tested the therapy in rats, mice and monkeys. Such treatments frequently do not work in humans, and some animal experiments can have a negative effect, which is why human clinical trials begin with small numbers of people.

Tuszynski is running a three-year Phase 1 trial that will test the safety and efficacy of the new therapy in 12 patients, with an additional 12 people serving as a control group. The study will focus on people who have Alzheimer’s or mild cognitive impairment.

“If in the first half of the study BDNF is shown to be safe in Alzheimer’s patients, we will then go on to see if it is also safe in patients with even earlier stages of memory loss, patients with mild cognitive impairment,” Tuszynski said.

To learn more about the trial, call Michelle Mendoza at (858) 249-3015 or email ◆