‘Not time to give up’ on tau protein therapeutics, expert says
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SEATTLE — Although successful tau therapeutics have not yet been identified, new insights into the protein’s biology may soon lead to treatments, according to a presenter at the 2022 American Academy of Neurology annual meeting.
“The general principle in tauopathies [is] that both the amount of inside tau and the anatomical location of the tau protein in autopsy is strongly correlated with the severity and also the clinical features of disease,” Adam J. Boxer, MD, PhD, Endowed Professor in Memory and Aging at the Weill Institute for Neurosciences at the University of California, San Francisco, said during the presentation. “If we could decrease accumulation of the tau protein, maybe we could decrease the severity or types of symptoms that people have.”
According to Boxer, tau protein plays a central role in three kinds of neurodegeneration: primary, where a microtubule-associated protein tau (MAPT) mutation may lead to progressive supranuclear palsy (PSP), Pick’s disease and corticobasal degeneration; secondary, where beta-amyloid peptide in cerebrospinal fluid is a diagnostic indicator of Alzheimer’s disease; and contributory, where epilepsy or Parkinson’s disease are likely diagnoses. For all three, tau gain of function eventually leads to neuronal dysfunction and death, making reduction in this gain of function a priority and prompting AD, PSP and MAPT mutation carriers to be the focus of current clinical trials.
Time is of the essence, Boxer stated, and the quicker the tau biomarker is discovered, the better. Inexpensive, easy biomarker tests are required for this purpose, he added, and a simple plasma P-tau test, which costs approximately $50 to $100 may be the answer, as opposed to a tau PET scan, which may cost $5,000 to $10,000, or an MRI to assess brain atrophy, which can cost as much as $2,000.
Boxer noted there have been no successful tau therapies and no evidence of clinical efficacy to date, although research and clinical trials are underway.
“These are still early days for therapeutics, and not time to give up yet,” Boxer told attendees. “But we need to double down, accelerate our clinical development and use what we’re learning about the biology of tau.”