Could a cancer drug help treat early-stage Alzheimer’s disease?

Credit: Michelle Bixby/Penn State.

A new study suggests that a drug initially developed to treat cancer might also help in the fight against Alzheimer’s disease, a common form of dementia.

Researchers from Penn State, Stanford University, and an international team of collaborators have found that blocking a specific enzyme called IDO1 could restore memory and brain function in models that mimic Alzheimer’s disease.

Alzheimer’s disease, which affects millions of people worldwide, leads to the progressive loss of memory, language, and the ability to think clearly.

Currently, there are treatments available to manage symptoms and slow the disease’s progression, but there are no approved treatments that can stop the disease from starting in the first place.

This new research, published in the journal Science, offers hope for developing such a treatment.

The enzyme in question, IDO1 (indoleamine-2,3-dioxygenase 1), plays a role in breaking down a substance called tryptophan, which is also found in turkey and known for making people sleepy. When IDO1 breaks down tryptophan, it creates a compound called kynurenine.

The study found that when too much kynurenine is produced, it reduces the brain cells’ ability to use glucose—a critical source of energy—leading to problems in brain function.

The research team used various models, including cells with amyloid and tau proteins (which are associated with Alzheimer’s) and mouse models of the disease, to test their theory.

They discovered that blocking IDO1 helped restore normal glucose metabolism in brain cells called astrocytes.

These star-shaped cells support neurons, the brain cells responsible for thinking, memory, and communication.

When IDO1 was blocked, the neurons received better metabolic support, which improved their ability to function.

What makes this study exciting is that IDO1 inhibitors, which are currently being developed to treat cancers like melanoma, leukemia, and breast cancer, could potentially be repurposed to treat early-stage Alzheimer’s disease.

This could be a major step forward in finding ways to protect the brain from the damage caused by aging and neurodegenerative diseases.

The research also suggests that IDO1 could be involved in other neurodegenerative diseases, such as Parkinson’s disease and various forms of dementia. By targeting the brain’s metabolism, scientists may not only slow down these diseases but potentially reverse their progression.

This discovery opens up new possibilities for treating Alzheimer’s and other neurodegenerative diseases, offering hope to millions of people and their families.

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