Brain Targeting Program

The Wyss Institute's Brain Targeting Program is developing shuttles that dramatically improve delivery of drugs to the brain.

We know more than ever about brain diseases – their causes, their impact on patients and families, and potential approaches to their treatment. But drugs developed for Alzheimer’s disease, Parkinson’s disease, brain cancers, and rare genetic disorders of the brain often fail in the clinic because these drugs cannot cross the blood brain barrier (BBB). In 2019, the Wyss Institute established the Brain Targeting Program to confront this challenge and improve outcomes for the millions of patients suffering with brain diseases.

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Diseases we are targeting

• Neurodegenerative Diseases: Alzheimer’s disease, Parkinson’s disease, ALS
• Brain Cancers: Glioblastoma multiforme and metastasis
• Rare Brain Diseases: Enzyme deficiencies such as Pompe’s & Lafora’s disease
• Psychiatric Diseases: Bipolar, Depression, Schizophrenia, Anxiety

Targeting of drugs to the brain continues to present one of the greatest challenges in medicine today. For Alzheimer’s disease, almost 300 experimental drugs have been tested in more than 1,000 clinical trials over the past 10 years. Sadly, 99.6% of these trials failed, in part because the drugs could not reach their optimal concentration in the brain. Even in breast cancer, one-third of patients treated with Herceptin die due to brain metastases that are inaccessible to the drug. New brain targeting approaches are urgently needed to improve the lives of millions of brain disease patients and their family members.

Creating a premier center for engineering brain-targeted drugs

At the Wyss Institute, we join the freedom of academia with the focus of industry to solve seemingly impossible problems. The Wyss Brain Targeting program focuses on two major areas, which together enable dramatic improvement in treatments for multiple diseases of the brain.

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• First, we are discovering and developing new brain transport shuttles that are safe and effective. Our first-generation Wyss brain shuttles increase uptake of test drugs into the brain by 10- to 100-fold. These shuttles will improve transport of new drugs and will rescue drugs that failed to cross the BBB in past clinical trials.
• Second, we are developing novel brain-targeted therapies that could enter clinical trials within the next 5 to 7 years. These new therapies have the potential to greatly improve treatment for diverse brain diseases.

We are ready to conduct proof-of-concept pilot projects in diverse brain diseases and to launch ambitious long-term projects to translate these discoveries into exponentially improved brain disease treatments.

Gathering competitors in novel collaborations to discover new brain targeting technologies

The Wyss Brain Targeting Program is organized as an R&D hub to promote collaboration among diverse stakeholders, including competing companies and academic labs. Our first pre-competitive project supported discovery of brain-targeting shuttles that can benefit all participants’ drug programs while advancing the entire field. During the past 3 years, this project attracted discovery support from 8 companies and 2 government agencies. With the help of these industry-academic interactions, we have discovered brain targeting shuttles that can help revolutionize treatment of brain diseases. We are now beginning to combine these shuttles with known and novel drugs to invent brain-targeted therapeutics for treatment of specific brain diseases.

A Brain Targeting Scientist’s Story

Reimagining treatment for brain diseases to improve the lives of patients and their families

“To sit next to her, to hold her hand, but know that the person who was playing with me and spent so much time with me doesn’t know who I am and is not there is the most difficult part. It’s hard to see your grandmother doesn’t recognize you anymore.”

Ana Raquel Santa Maria has been inspired to Reimagine the World with better understanding, treatment, and prevention of brain diseases after seeing her grandparents suffer as they age, especially her grandma who has dementia. Read and listen to Ana tell her story.