In the ongoing fight to cure Alzheimer’s disease, the US Food and Drug Administration (FDA) approved the drug Leqembi as a possible treatment on January 6. Leqembi is the second drug in a new category of drugs approved to treat the disease, according to FDA press release. FDA approval Fast track to approvalwhich approves drugs for serious conditions where there is an unmet medical need.
“Alzheimer’s disease immeasurably damages the lives of those who suffer from it,” said Billy Dunn, director of the Office of Neurology at the FDA’s Center for Drug Evaluation and Research, in the press release. “This treatment option is the latest therapy to target and affect the underlying Alzheimer’s disease process, rather than just treating the symptoms of the disease.”
While this may sound like promising news, you may be wondering: What is this drug and how does it slow cognitive decline?
Main trial against Lekembi
The drug targets the “fundamental pathophysiology” of Alzheimer’s, or the changes seen at the cellular level. Leqembi works to reduce the amount of beta-amyloid present in the brain, which is a major identifier of Alzheimer’s.
Plaques can form in the brain when pieces of beta-amyloid, a protein, stick together. Excessive accumulation blocks cell-to-cell signaling at synapses in the brainas well as other neurological functions leading to Alzheimer’s disease.
In a study of 856 participants with Alzheimer’s disease, researchers provided treatment to patients who suffered from mild cognitive impairment or mild stage of Alzheimer’s dementia. Of note, participants had to have the presence of amyloid beta pathology.
The results of the study showed a statistically significant reduction in the amount of amyloid beta plaque in patients who received the treatment compared to the placebo group. The placebo group had no reduction.
Read more: Alzheimer’s drug approval sparks firestorm
Limitations of Leqembi
Although the approval of Leqembi is exciting news, the drug, like many other newly approved drugs, comes with side effects and certain drawbacks.
According to the press release, the prescription information for Leqembi includes a warning about amyloid-related imaging abnormalities (ARIA). While ARIA usually has no symptoms, it can cause serious and life-threatening events related to Leqembi. These events include swelling of the brain or small spots of bleeding in or on the surface of the brain.
The most common side effects of Leqembi are infusion-related reactions, including flu-like symptoms, nausea, vomiting, and changes in blood pressure. Headache is also common with the drug.
Leqembi is also only intended to treat patients with mild cognitive impairment or those in the mild dementia stage of Alzheimer’s disease. As this is the only population studied in Leqembi’s clinical trials, it cannot be prescribed to patients outside this population. Currently, there are no data on the effectiveness of the treatment for patients in the early or later stages of the disease.