Tiny llama proteins could treat Alzheimer’s disease

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A new scientific discovery may change how doctors treat complex brain diseases like schizophrenia and Alzheimer’s.

The secret comes from an unexpected source—camels, llamas, and alpacas. Scientists have found that these animals produce tiny proteins called nanobodies that could become a powerful new type of medicine for the brain.

A study published on November 5 in the journal Trends in Pharmacological Sciences explains how nanobodies can travel into the brain more easily and act more precisely than traditional drugs. In animal studies, these small proteins were able to reach brain cells more effectively and with fewer side effects, raising hopes for new treatments in humans.

Researchers from the Centre National de la Recherche Scientifique (CNRS) in France led the work. “Camelid nanobodies open a new era of biologic therapies for brain disorders,” said Professor Philippe Rondard, one of the study’s authors. “They can form a new class of drugs that sit between conventional antibodies and small molecules.”

The story of nanobodies began in the early 1990s, when Belgian scientists were studying the immune systems of camelids. They discovered that, unlike humans, camels and llamas produce a simpler form of antibodies made of only heavy chains.

From these, scientists isolated the active part—tiny fragments now known as nanobodies. Each nanobody is about one-tenth the size of a normal antibody. These small but powerful molecules exist only in camelids and certain fish, making them a rare natural defense system.

Antibody-based drugs have been used for years to fight cancer and autoimmune diseases. However, when it comes to brain disorders, most antibody treatments struggle to work effectively because they cannot easily cross the blood-brain barrier—a protective shield that stops many drugs from reaching the brain.

Even the few antibody treatments that work for Alzheimer’s disease often cause harmful side effects such as brain swelling.

Nanobodies could overcome this problem. Their small size allows them to slip through the blood-brain barrier and directly reach the brain’s target cells. In experiments with mice, nanobody treatments helped restore normal behavior in models of schizophrenia and other neurological diseases.

Because they can reach their targets more efficiently, nanobodies might provide the same or better results with fewer side effects than current drugs.

According to co-author Dr. Pierre-André Lafon from CNRS, nanobodies are also easier to make and purify than traditional antibodies. “They are highly soluble small proteins that can enter the brain passively,” he explained.

“Unlike many small-molecule drugs, they do not stick to the wrong targets or cause unwanted reactions.” Scientists can also engineer nanobodies to focus on specific brain molecules, giving them great potential for precision medicine.

Before these new treatments can be used in people, scientists need to perform more safety tests. Researchers must study how long nanobodies stay active in the brain, whether they remain stable over time, and how to store them properly without losing their effect.

Toxicology and long-term safety studies will also be needed to ensure that nanobody-based therapies are safe for chronic use.

The CNRS team has already started testing these questions in the lab. They are working on improving nanobody stability, checking that the molecules fold correctly, and preventing clumping. According to Dr. Lafon, some nanobodies already show promise for long-term treatment without major side effects in animal studies.

In reviewing the study, it’s clear that nanobodies could represent a big leap forward in brain medicine. Traditional antibody drugs often fail to reach the brain or cause dangerous inflammation, but nanobodies appear to avoid both problems.

Their small size, flexibility, and precision could make them ideal for tackling disorders that have long been difficult to treat safely.

However, much work remains before they reach clinical use. Scientists must prove that nanobodies are safe in humans and understand how to deliver them in the right doses. If these challenges can be solved, nanobody-based drugs could transform the treatment of brain disorders—offering patients more effective therapies with fewer side effects.

If you care about Alzheimer’s disease, please read studies about the protective power of dietary antioxidants against Alzheimer’s, and eating habits linked to higher Alzheimer’s risk.

For more health information, please see recent studies that oral cannabis extract may help reduce Alzheimer’s symptoms, and Vitamin E may help prevent Parkinson’s disease.

The study is published in Trends in Pharmacological Sciences.

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