Recently, members of the Denali Therapeutics team provided insights into their work and partnership with Takeda to develop a therapeutic to treat FTD-GRN (Frontotemporal Dementia caused by GRN gene mutation) and shared what inspires them to engage in neurodegenerative disease research.

 

Could you share your company’s approach to developing an FTD-focused therapeutic?

Denali is committed to developing therapies for neurodegenerative diseases like FTD. Historically, many attempts to tackle such diseases have not been successful. We believe we can overcome these odds by adhering to three core scientific principles:

1. Denali investigational treatments are designed to target disease processes or pathways that are at the root cause of disease based on evidence from human genetics.
2. The human brain contains approximately 400 miles of blood vessels. These blood vessels are lined by closely linked endothelial cells to form the Blood-Brain Barrier (BBB), which protects the brain from toxins, but also has been an obstacle to delivering treatments to the brain. Denali has engineered investigational treatments that aim to cross the BBB and optimize delivery of treatments to the brain.
3. We use biomarkers (substances that can be measured in the blood or other body fluids and that reflect a desired biological effect) to monitor FTD-GRN progression and the effects of treatment. This approach helps us understand safety and the impact of the investigational treatment on the disease. It also helps in selecting the right patients and the right dose for clinical development.

In collaboration with Takeda, we are applying these principles to develop TAK-594/DNL593, a potential treatment for frontotemporal dementia caused by GRN gene mutations (FTD-GRN).

TAK-594/DNL593 is an investigational therapy and is not approved by any Health Authority.

What inspires you and your colleagues to work in the neurodegenerative disease and FTD space?

Denali was founded to defeat neurodegeneration. Neurodegenerative diseases are one of the biggest public health challenges. Changes to a person’s behavior and personality, as well as impairment of their verbal abilities as symptoms of FTD progression, are devastating for individuals and their families. Denali is working to develop a potential treatment for those living with FTD-GRN.

As we seek to raise FTD awareness, what do you hope people will come to understand about the disease?

The scientific community has made tremendous progress in understanding the genetic and biological root causes of FTD. This research has resulted in several potential new therapies that are being investigated in the laboratory and in clinical trials.

There is good reason to hope that effective treatments for some patients with FTD are on the horizon. Many of the initial treatments being developed are aimed at genetic forms of FTD, and the lessons learned from these efforts will help support the way for treatments for non-genetic forms and other neurodegenerative diseases as well.

This year, AFTD is focused on making sure every FTD story counts. Why do you think it is important for people to share their stories, and have outlets to do so?

When courageous patients, caregivers, and family members share their journey and hope for the future, this raises awareness and brings urgency to the fight against FTD. These individuals inspire our daily work in developing future treatments that will make a difference for families living with FTD. Please contact patients@dnli.com for additional information.

 

AFTD would like to extend our gratitude to Denali Therapeutics and Takeda for their support of the 2023 Education Conference. Recorded sessions from this year’s conference can be viewed on AFTD’s YouTube channel.

Sharing your story is one of the most powerful ways to raise awareness, educate and support others, and self-advocate. If you’d be interested in sharing your FTD experience with our community, please reach out to info@theaftd.org.

Every story brings awareness to make things better for the next family. 

Every story moves us closer to a future free of FTD.