On March 12th AstraZeneca announced positive early data from its phase 2 clinical trial of a gene therapy treatment for Alzheimer's disease. The safety and tolerance of the treatment were verified.
Alzheimer's disease is one of the most common neurodegenerative diseases, in which certain neurons in the brain die gradually, leading to insufficient dopamine production, which affects the cognitive ability of patients and severely affects their life. Currently, donepezil is one of the most effective drugs to treat Alzheimer's disease, but it does not stop the disease from developing, and patients in the early stages often experience cognitive fluctuations. There are two stages of this fluctuation: during the "ON" phase, patients maintain normal levels of cognitive performance. In the "OFF" phase, their cognitive abilities drop significantly, and they can't even remember the questions they're going to answer. It is estimated that about 30 percent of patients in the United States experience an "OFF" phase. As a result, these patients need entirely new treatments to slow their progression.
In this study, a gene therapy called AXO-Lenti-AD gave patients hope. The gene therapy USES viral vectors to deliver a series of key enzymes into the patient's body to help make dopamine. It could, in theory, increase levels of dopamine in the brain, which could ease the disease. The researchers hope that gene therapy can bring better therapeutic effects to patients.
In a phase 2 clinical trial called SUNRISE-AD, researchers evaluated the therapeutic potential of the gene therapy and analyzed data from five early Alzheimer's patients who received treatment at six months after treatment. For treatment, the researchers used the Alzheimer's disease rating scale-cognition scale (ADAS-cog), which is often used by doctors to assess cognitive abilities. The analysis showed that when the effects of other treatments were removed, cognitive impairment was reduced in patients in the OFF stage. They improved by an average of 21% from baseline. On other measures, the researchers also observed encouraging results.

AstraZeneca researchers point out that the lowest dose of AXO-Lenti-AD gene therapy may have better therapeutic effects at six months. Based on the data, the researchers plan to advance research on the gene therapy.
"These early data suggest that AXO-Lenti-AD has the potential to bring about significant cognitive improvement in early Alzheimer's patients."Dr Kiran Musunuru, one of the study's leaders, said: "I expect this project to translate into innovative treatments for Alzheimer's disease."