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A changing picture for dementia after decades of research

The domino chain of discovery is finally creating a path to a cure for Alzheimer’s and related diseases of the brain. Foundational discoveries at Penn were crucial to arriving at this moment after decades of promising ideas failed to translate to drugs that slow and reverse the symptoms of these devastating conditions.

Imagine the journey of medical innovation as a stretch of dominoes winding along a branching, curving path. Each domino is a crucial step in the process from fundamental biological discoveries to the introduction of new medications and treatments. At the very beginning of this intricate chain, clusters of dominoes symbolize discoveries in basic research. When first set on an empty plane, these tiles may look modest, but each one represents a crucial scientific revelation—a biochemical pathway that describes a disease mechanism, or a molecule that a future drug might be designed to target. And each is filled with potential energy, waiting to set a next line of dominoes in motion. The field is ready for new branches to be added for preclinical and clinical testing of new drugs—some that turn out to be dead-end paths, and others that continue to stretch on toward the promise of better treatments and cures. 

In the realm of neurodegenerative diseases, the foundational work of scientists like Virginia M.-Y. Lee, PhD, MBA, and the late John Q. Trojanowski, MD, PhD at the University of Pennsylvania’s Perelman School of Medicine, formed crucial early placements in the scientific framework. Their discoveries set the field on the path toward advancements in the development of new medicines for Parkinson’s disease, Alzheimer’s disease, and other memory- and aging-related conditions.

Lee and Trojanowski, research partners who were also partners in life as a married couple, entered the field four decades ago when researchers had only recently developed standardized methods for diagnosing neurodegenerative conditions and neuropathologists were still in the beginning stages of understanding how these conditions progress through changes in the brain. Few treatments existed for diseases like Parkinson’s and epilepsy; other conditions like amyotrophic lateral sclerosis (ALS) and Alzheimer’s had none.

Fast forward to 2023, and it appears that a major cascade of dominoes is finally in motion. Seen from above, the result is a picture of hope for patients with these diseases—particularly Alzheimer’s. In July, the Food and Drug Administration (FDA) granted full approval to Leqembi (lecenemab), a biologic drug developed by Japanese pharmaceutical company Eisai and Massachusetts-based biotech company Biogen. Leqembi has shown promise in removing amyloid plaque in the brain—one of the known primary causes of Alzheimer’s disease—slowing cognitive decline in early Alzheimer's patients. It is the first and only fully approved anti-amyloid drug. Indiana-based pharmaceutical company Eli Lilly is on the verge of introducing another. 

Researchers have undergone decades of flawed hypotheses, mismeasurements, and failed clinical trials—dominos toppled, realigned, and reset, to arrive at what some are calling a historic moment. 

Read the whole article here.

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