Can Drug Treatments Break Through the Blood-Brain Barrier?

Can Drug Treatments Break Through the Blood-Brain Barrier?

Neurological drug trials have historically been the graveyard of drug development. The vast majority of drugs can’t cross the blood-brain barrier, meaning diseases like Alzheimer’s and Parkinson’s have been very difficult to treat.

Fortunately, new developments offer hope. This graphic from Scottish Mortgage Investment Trust shows how one of their portfolio companies, Denali Therapeutics, is engineering drugs that can potentially reach the brain.

What is the Blood-Brain Barrier?

The body has a special shield, made of blood vessels and tissues, known as the blood-brain barrier. You can think of it as a strong wall with tiny security guards that only allow certain things to go from your blood into your brain. It’s very picky about what it lets in, so that it can keep out germs and toxins. 

However, the blood-brain barrier makes it very challenging to treat brain diseases. Drugs have historically been unable to cross the barrier.

Engineering Brain Delivery

To tackle this problem, Denali Therapeutics is developing drugs that are better positioned to cross the blood-brain barrier. Here’s how it works:

• Transferrin is a naturally occurring protein that carries molecules like iron into the brain by binding to transferrin receptors. The receptors are like a VIP pass that allow the molecules to pass by the barrier’s security guards.
• Denali’s small molecule drugs bind to these transferrin receptors using transport vehicles, such as antibodies or enzymes.
• By binding to these receptors, Denali hopes its drugs can sneak through the blood-brain barrier and into the brain.

The company has a number of drug candidates that are in various stages of clinical trials. Through studying ‘degenogenes’—genes that cause neurodegeneration when they mutate—Denali can develop drugs that target specific disease pathways.

An Opportunity for Investors

Denali envisions a future where degenerative diseases are defeated, and people can live out their last years of life with dignity.

Alongside this meaningful purpose, Scottish Mortgage believes Denali has numerous qualities that position it for growth.

• Scale of opportunity: One in three seniors dies with Alzheimer’s or another form of dementia.
• Expertise: Denali’s leadership has deep scientific expertise, particularly in neurodegenerative diseases.
• Broad portfolio: The team has 10 drugs in development at the same time, which reduces its risk from any one drug failing.
• Initial success: Denali’s most advanced trial for Hunter’s disease has shown promising results.
• Strategic partnerships: Working with companies like Sanofi has led to $1.3 billion in upfront payments that covers program costs while allowing Denali to maintain significant control.

If Denali is able to tackle one of the largest medical challenges of our time, it will have a wide-reaching impact for patients and investors.

Want to invest in transformative companies like Denali Therapeutics?

Discover Scottish Mortgage Investment Trust, a portfolio of some of the world’s most exciting growth companies.

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