A recent study presented at the American Heart Association’s Epidemiology and Prevention │ Lifestyle and Cardiometabolic Scientific Sessions 2024 has raised concerns about the potential risks associated with time-restricted eating (TRE), a popular dietary trend.
According to the study, individuals who adhered to an 8-hour daily eating window faced a staggering 91% higher risk of dying from cardiovascular disease. This alarming finding sheds light on the potential dangers of TRE, particularly for those with existing heart conditions or cancer.
Metachromatic leukodystrophy (MLD) is a rare genetic disease that affects the brain and nervous system. It is caused by a deficiency of an enzyme called arylsulfatase A (ARSA), leading to the accumulation of fatty substances in cells. This buildup results in damage to the central and peripheral nervous system, leading to motor and cognitive decline and, ultimately, premature death. With approximately one in every 40,000 individuals in the United States affected by MLD, the approval of Lenmeldy represents a significant milestone in the treatment of this devastating condition.
Lenmeldy marks the first FDA-approved gene therapy for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile MLD. Unlike traditional treatments that focus on supportive care and symptom management, Lenmeldy offers a promising new approach to addressing the underlying cause of MLD.
The groundbreaking therapy involves a one-time, individualized infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), genetically modified to include functional copies of the ARSA gene. These modified stem cells are then transplanted back into the patient, where they engraft within the bone marrow and begin producing the ARSA enzyme. By breaking down the harmful buildup of fatty substances, Lenmeldy has the potential to halt the progression of MLD and improve patients’ quality of life.
The approval of Lenmeldy represents a significant advancement in the field of gene therapy and offers hope to children and families affected by MLD. With further research and development, gene therapies like Lenmeldy have the potential to revolutionize the treatment of rare genetic diseases, offering new pathways to improved health and well-being.