How close are we to real treatments for neurodegenerative diseases? by Julie Andersen, Professor, Buck Institute

We are big fans of the Buck Institute, and for very good reason. Based in California, it stands as one of the world's premier research institutions dedicated entirely to investigating how we can achieve better health and healthier longevity, particularly when it comes to tackling complex neurological conditions like Parkinson's. A webinar hosted by the institute, featuring insights from leading neuroscientist Professor Julie Andersen, sheds light on how close science is getting to real, disease-modifying treatments. By shifting the focus away from merely managing symptoms and looking at these conditions through the lens of individual biological aging, researchers are uncovering groundbreaking avenues for hope. Moving Beyond Symptom Management: Historically, treatments for neurodegenerative conditions have focused almost entirely on easing symptoms rather than halting the underlying issue. In Parkinson's, the classic challenge is the loss of dopamine-producing cells in a region of the midbrain known as the substantia nigra (meaning "black substance"). Because these cells contain dopamine, which converts into the dark pigment melanin, their loss is a defining feature of the condition. Traditional medications essentially force the remaining cells to work harder and produce more dopamine, which manages motor symptoms for a time but does not stop the cells from dying off. When medications lose their efficacy, options like Deep Brain Stimulation can help stimulate the brain circuits to control voluntary movement, but the underlying progression carries on. The tide, however, is turning. Excitingly, a new immunotherapy called prasinezumab is moving into Phase 3 clinical trials. This therapy uses targeted antibodies to go after alpha-synuclein, the specific toxic protein that builds up and aggregates in the brains of people with Parkinson's. If successful, this could mark a massive shift toward a truly disease-modifying treatment that stops the condition in its tracks. The Micro-Robot Approach to Targeted Treatment: One of the biggest hurdles in modern medicine is delivering drugs exactly where they are needed without causing side effects elsewhere in the body. Professor Andersen highlighted a brilliant project in her lab led by research scientist Dr Chaska Walton, which aims to solve this using genetically engineered immune cells, specifically T-cells. Instead of flooding the body with a high dose of medicine in the hope that a small fraction crosses the blood-brain barrier, this approach turns T-cells into "little mini-robots" or mobile factories. These engineered cells travel through the bloodstream, cross into the brain, and actively seek out toxic protein clumps. Crucially, they remain switched off and do not produce any medicine until they hone in precisely on the pathology. Once there, they activate, release a combination of therapeutics—such as anti-inflammatories and growth factors—clear the damage, and then turn themselves off. Currently being tested pre-clinically, this targeted strategy could completely revolutionise how we treat neurological conditions. The Gut-Brain Connection and the Power of Diet: Perhaps one of the most fascinating shifts in modern research is the realization that Parkinson's is not simply a brain-centric issue, but a multi-system condition. Clinicians have long noted a striking clue: up to 80% of people diagnosed with Parkinson's experience gastrointestinal issues, such as constipation or irritable bowel syndrome, 10 to 20 years before any motor symptoms appear. Scientists are investigating two main theories for this connection: The Transport Theory: Alpha-synuclein might actually originate in the gut and physically travel up the vagus nerve directly into the midbrain. The Inflammation Theory: Early gut inflammation releases pro-inflammatory signals (cytokines) and immune cells into the bloodstream, which then enter the brain and trigger the neuroinflammation that drives protein clumping. To explore this, Professor Andersen’s lab collaborated with a gut inflammation specialist at the Buck Institute, Dr Dan Winer. Their collaborative research demonstrated that gut inflammation directly drives neurodegenerative pathology, and remarkably, it can be managed. In pre-clinical models, introducing a high-fiber diet successfully treated gut inflammation and prevented the manifestation of the condition's typical brain pathology. This directly highlights the value of specialized dietary frameworks like the MIND diet—a hybrid of the Mediterranean and DASH diets. It emphasizes complex carbohydrates (like whole grains) that break down slowly, nuts, berries, legumes, and high-quality fats like olive oil (which contains a highly beneficial component known as oleocanthal), whilst limiting processed foods and simple sugars. This way of eating fosters a diverse, healthy gut microbiome, which produces protective metabolites like short-chain fatty acids that help maintain the integrity of the blood-brain barrier. Navigating Supplements, Sleep, and Everyday Risks: While the exposome—the sum of our environmental exposures over a lifetime—accounts for roughly 90% of our aging risk, it also means a vast portion of our health destiny is tractable. We can actively take steps to alter our trajectory. Beyond diet, specific compounds like curcumin (found in turmeric) show genuine promise in clinical and pre-clinical studies for reducing neuroinflammation. However, Professor Andersen urges caution with over-the-counter supplements, noting that "more is not always better." High doses of curcumin can lead to liver toxicity, and overly high doses of other elements, like Vitamin D, can inadvertently disrupt metabolic health. Furthermore, everyday medications can have unexpected impacts. Many common over-the-counter sleep aids and allergy medicines are anticholinergic, meaning they actively block acetylcholine—a vital neurotransmitter for cognitive health. Long-term use of these can become an avoidable risk factor. Interestingly, the common mineral lithium is experiencing a research renaissance. A Harvard study recently revealed that lithium levels are statistically lower in brains affected by neurodegeneration. When researchers administered micro-doses of a form called lithium orotate—which crosses the blood-brain barrier far more easily than traditional medical lithium—it successfully preserved memory function without the toxic side effects of high-dose prescriptions. While getting backing for clinical trials on non-patentable, inexpensive minerals is notoriously difficult because big pharma cannot easily profit, academic institutions are now independently pushing these cheap, accessible treatments forward. Ultimately, the grand takeaway from the Buck Institute’s latest insights is one of profound optimism. While overall case numbers rise simply because the population is living longer, an individual’s personal risk of developing severe cognitive decline is actually on the decrease. By prioritizing quality sleep (which naturally flushes toxins from the brain), enjoying a high-fiber diet, exercising, and proactively managing cardiovascular health, we are actively rewriting our biological future. What is good for the heart truly is good for the brain.