Dr. Florian Eichler is a co-founder of ALD Connect. He is a Professor of Neurology at Massachusetts General Hospital (MGH) and Harvard Medical School. His career has been dedicated to advancing the care and treatment for devastating neurogenetic conditions. Following neurogenetics training at Johns Hopkins with the late Dr. Hugo Moser and residency in pediatric neurology at MGH, he became the Director of the Leukodystrophy Service that cares for patients with an increasing variety of neurogenetic conditions. Dr. Eichler runs a laboratory at MGH that explores the relationship of mutant genes to specific biochemical defects and their contribution to neurodegeneration. In 2015, he became Director of the Center for Rare Neurological Diseases at MGH. The Center aims to eradicate rare disorders of the nervous system by leveraging the power of biological insights towards design and implementation of clinical trials. Dr. Eichler is the Principal Investigator of several NIH-funded studies on neurogenetic disorders as well as a gene therapy trial of adrenoleukodystrophy.
Main Area of Research: My laboratory studies the relationship of the ALD gene defect to lipid accumulation and immune responses and their contribution to neurodegeneration. A large focus lies in advancing new gene therapy trials for different phenotypes of ALD. This encompasses ex vivo lentiviral and in vivo AAV- based approaches. In collaboration with Boston Children’s and other centers we were able to achieve a first FDA approved gene therapy for childhood cerebral ALD. For women with ALD we are working on natural history studies as well as new therapeutics aiming to reactivate the healthy X chromosome.