Drug development for brain disease is complex. No blueprint exists to easily identify the most promising targets or critical challenges in research. The NDC is uniquely positioned to speed breakthroughs by providing scientists with the funding, tools and collaborative consortium partnerships.
The NDC accelerates the pace of innovation by bringing drug discovery to the earliest stage of exploration for novel therapeutics. We are driving progress in the following critical areas:
The native immune system in the brain, which is separate from the peripheral immune system, is critical for brain health. Many genetic risk factors for neurodegenerative diseases map to these cells, implicating them in disease initiation and progression. We identify drug targets in microglia and astrocytes that regulate neuroinflammation.
Preserving and restoring neurons is a key goal of neurodegeneration drug discovery. In many cases, neurons die due to active cellular processes that can be targeted with therapeutics. We identify these processes and develop therapeutics to stop them.
Sleep and Brain Detoxification
Sleep is essential for healthy brain aging, since the brain removes toxins like misfolded proteins during sleep. Neurodegenerative diseases, like Alzheimer’s disease and Parkinson’s disease, cause sleep disruptions early in disease onset. Restoring rejuvenating sleep is an important goal for therapeutic discovery.
Neurofibrillary Tau Toxicity
The protein tau forms neurofibrillary tangles that cause brain cell death in Alzheimer’s disease and other neurodegenerative conditions. Research is uncovering the mechanisms by which aggregated tau proteins become toxic. By harnessing that knowledge, we develop therapeutics to block this key pathogenic trigger.
Autophagy and Lysosomal Biology
Neurodegeneration is ultimately a disease of misfolded proteins. One key clearance mechanism for these toxic molecules is through autophagy, in which unwanted cellular debris is degraded by fusing with lysosomes inside cells. Unfortunately, aging and inflammation impair these processes. Our goal is to identify drug targets that promote degradation of toxic proteins and lipids.
Drug Delivery to the Brain
Less than 2% of small molecule drugs adequately penetrate the blood brain barrier, which also prevents effective delivery of biologics, cell therapies, antisense nucleotides, and other innovative therapeutics. We want to unlock the blood brain barrier to unleash a new generation of central nervous system therapeutics.
"The NDC is a unique and potentially transformative idea: We will bring together academic research and drug discovery expertise at the earliest stages of discovery, and greatly accelerate the pace of progress."
~ Giulio Draetta, M.D., Ph.D.