Position statement on possible therapies for neurological conditions

The American Academy of Neurology (AAN) has issued a position statement on therapies for neurological conditions that have limited evidence or no approved use by the Food and Drug Administration (FDA). The statement is published in Neurology.

The statement includes guiding principles for discussions with patients and policymakers about these therapies. Although the statement includes illustrative examples, it does not provide clinical recommendations regarding the use of specific therapies.

"As the experts in brain health, neurologists and neuroscience professionals are continually working toward achieving scientific breakthroughs through rigorous research," said American Academy of Neurology President Natalia S. Rost, MD, MPH, FAAN, FAHA. "Still, people often ask about emerging therapies not yet supported by science. In our efforts to provide brain health resources for all, the American Academy of Neurology has issued this position statement to help facilitate conversations between patients and their doctors about emerging therapies."

The position statement says the AAN recommends that a patient and their neurologist carefully review all available evidence and discuss the potential risks and benefits of the therapy, including when patients are considering using their "Right to Try." The Right to Try Act allows people with life-threatening diseases who have tried all other approved treatments, and who cannot participate in a clinical trial, to try an experimental treatment.

"There is a growing and unmet need for effective treatments for a variety of neurological disorders," said author Larry B. Goldstein, MD, FAAN, FAHA, of the University of Kentucky, and who serves on the AAN Board of Directors. "Whenever considering a potential treatment that has not been thoroughly researched, a person and their neurologist need to have a discussion about the available data supporting effectiveness and what is known about any risks to their health."

The statement says the AAN supports the "off-label" use of FDA-approved therapies when high-quality evidence indicates that the benefit of the therapy outweighs the risks, with shared decision making between the patient and physician.

When discussing such therapies more broadly, including with policymakers, the statement says that for those that have FDA approval but a high risk or incomplete data on side effects, or newly recognized and potentially serious side effects, the AAN supports waiting to take a definitive position until after further FDA review.

If a suspended therapy is reintroduced, or as the data otherwise warrants, the AAN supports FDA required post-approval monitoring. The AAN supports neurologists and patients incorporating data about side effects in their shared decision making about the relative risks and benefits of a possible therapy.

The statement notes the proliferation of unproven treatments for neurological diseases that people may want to use to self-treat their conditions, yet those treatments may have limited or no supporting data and can have the potential to cause harm. These may be of interest especially to people with rare neurologic conditions with no approved therapies.

The statement says that rigorous investigation of any treatment is necessary to determine its efficacy and ensure safety and recommends patients discuss any potential treatment with their neurologist.

More information:
Larry B. Goldstein et al, Principles for Novel Neurologic Therapeutics, Neurology (2025). DOI: 10.1212/WNL.0000000000213850 www.neurology.org/doi/10.1212/WNL.0000000000213850

Provided by American Academy of Neurology