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Guiding Clinical Care for People at Risk for ALS and FTD

An international workshop co-led by University of Miami Miller School of Medicine neurologist Michael Benatar, M.D., Ph.D., laid the foundation for developing guidance for the clinical care of people at elevated genetic risk for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Participants at the ALS Hope Foundation-sponsored workshop came together to discuss care guidelines for patients at elevated risk for ALS and FTD.

Dr. Benatar, the Walter Bradley Chair in ALS Research and executive director of the Miller School’s ALS Center, and Terry Heiman-Patterson, M.D., professor of neurology at Temple University, led the Sept. 21-22 workshop in Philadelphia.

Sponsored by the ALS Hope Foundation, the workshop brought together clinicians, scientists, genetic counselors, and members of the at-risk ALS and FTD communities. The Miller School was also represented by Joanne Wuu, Sc.M., research associate professor of neurology and associate director of research at the ALS Center, and Danielle Colato, R.N., a clinical research nurse who works with Dr. Benatar and Wuu in the ALS Center.

Working with Limited Evidence

“The clinical care needs of people at elevated genetic risk for ALS and FTD have not historically been addressed,” said Dr. Benatar. “While evidence to guide management is somewhat limited, we need to work with what we have to provide the best framework and guidance that can assist physicians who are faced with an increasing number of pre-symptomatic carriers seeking care.”

At the workshop, clinicians, community members, lawyers and policy experts discussed the use of F.D.A.-approved drugs for patients who already have ALS, the role of lifestyle and environmental exposures, and the ethical, social, and legal issues relevant to the care of pre-symptomatic carriers.

“I am grateful for everyone coming together at this workshop to advance the clinical science of treating and preventing ALS and FTD,” said Neil Thakur, Ph.D., chief mission officer of the ALS Association, which provided co-sponsorship for the workshop.

Pre-symptomatic Testing Reveals Clinical Need

“With increasing knowledge of the genetic causes of ALS and the growing use of clinical genetic testing in the ALS patient population, we are seeing an increasing number of individuals and their family members seeking pre-symptomatic testing,” said Dr. Heiman-Patterson. “In turn, that raises a number of questions for clinicians about medications, lifestyle modifications, nutritional recommendations and psychological support.”

Disability and life insurance coverage, for example, could be affected if a patient’s medical record references genetic risk for disease.

“We also need to consider the cost of medications, along with the potential benefits,” added Dr. Benatar, noting that a low-cost drug with few side effects has little downside compared to an expensive medication that requires a monthly injection for decades and is associated with significant risk of serious side effects.

Drs. Benatar and Heiman-Patterson will be partnering with workshop attendees to draft a general guidance for physicians based on presentations and discussions at the workshop. Dr. Benatar also expressed that hope that workshops of this sort will provide an impetus for further research that can refine the recommendations for clinicians providing care for those at elevated risk for ALS and FTD.

Tags: ALS, ALS Center, dementia, Dr. Michael Benatar, USNWR Neuro