Richard S. Finkel, MD, a pioneering pediatric neurologist and director of the Center for Experimental Neurotherapeutics (CENT) at St. Jude Children’s Research Hospital, has been distinguished on Time Magazine’s TIME100 Health list for 2025. This prestigious list honors 100 individuals worldwide who are redefining and positively impacting global health, underscoring Dr. Finkel’s groundbreaking contributions to pediatric neurology and translational neuroscience. His leadership at CENT, since joining St. Jude in 2020, marks a pivotal expansion in the hospital’s historic mission, broadening from catastrophic pediatric cancers to encompass debilitating neurological disorders that affect children worldwide.
Dr. Finkel’s tenure at St. Jude symbolizes a crucial evolution in pediatric medicine. CENT represents the clinical wing of the Pediatric Translational Neuroscience Initiative (PTNI), an innovative research platform focused on turning laboratory discoveries into tangible clinical interventions for catastrophic neurological diseases. By integrating cutting-edge neuroscience with clinical application, CENT aims to address urgent unmet needs in pediatric neuromuscular disorders, including spinal muscular atrophy (SMA), Duchenne muscular dystrophy, inherited neuropathies, and neurometabolic disorders. This expansion aligns with St. Jude’s broader vision to extend its decades-long legacy of curing childhood cancer to crippling neurologic diseases.
Among Dr. Finkel’s most notable clinical achievements is his leadership in conducting the first in utero treatment of spinal muscular atrophy using risdiplam, an orally administered drug. SMA is a genetic neuromuscular disorder characterized by progressive muscle wasting and weakness due to the degeneration of motor neurons. Traditionally diagnosed postnatally, SMA results in severe disability or death if untreated. Dr. Finkel’s prenatal intervention represents a revolutionary paradigm shift in treatment, leveraging the prenatal environment’s unique immunological and developmental properties to arrest disease progression even before birth.
This landmark in utero treatment, performed in 2022, demonstrated remarkable efficacy. The infant treated prenatally with risdiplam showed no detectable manifestations of SMA over two years after birth, a stark contrast to the expected clinical trajectory of untreated SMA patients. The underlying mechanism involves risdiplam’s ability to increase the production of survival motor neuron (SMN) protein by modifying the splicing of the SMN2 gene, thereby compensating for the loss of function mutation in SMN1. Administering the therapy during fetal development maximizes the preservation of motor neuron populations before irreversible degeneration occurs, highlighting the critical window that prenatal therapy opens for neurodegenerative diseases.
Published in a letter to the New England Journal of Medicine in early 2025, these findings provide robust proof of concept for prenatal intervention as a viable therapeutic strategy. This study not only underscores the biological plausibility but also opens new investigative avenues for other genetic neuromuscular disorders traditionally treated postnatally or symptomatically. The clinical outcomes have profound implications for developmental neurobiology, pharmacokinetics in utero, and fetal immune tolerance mechanisms, which collectively influence the safety and efficacy of early pharmacological intervention.
Dr. James R. Downing, president and CEO of St. Jude Children’s Research Hospital, emphasized that Dr. Finkel’s designation as a TIME100 Health honoree illuminates the significance of pioneering pediatric neuromuscular diseases that have historically been underserved. His work encapsulates the hospital’s expanding commitment to eradicate not only life-threatening cancers but also the devastating neurological disorders that compromise childhood development and survival worldwide. This recognition amplifies ongoing efforts to develop therapies that provide durable, disease-modifying benefits, substantially improving quality of life for affected children.
The research implications of Dr. Finkel’s work extend beyond SMA into a broad spectrum of neurological diseases caused by genetic mutations, neurodegeneration, and metabolic imbalances. His extensive clinical practice focuses on optimizing therapeutics involving gene modulation, neurometabolic stabilization, and neuroprotective strategies. By combining clinical acumen with translational neuroscience, Dr. Finkel accelerates the bench-to-bedside pathway, enabling novel interventions to move rapidly through preclinical models to clinical trials and eventually standard of care.
Over his distinguished career, Dr. Finkel has authored more than 150 peer-reviewed articles and book chapters, reflecting his deep scientific insight and commitment to collaborative neurology research. He has played an instrumental role in designing innovative clinical trials that incorporate biomarkers, electrophysiological metrics, and advanced imaging to measure therapeutic efficacy objectively. His approach exemplifies precision medicine tailored to the unique genetic and phenotypic profiles of pediatric patients suffering from debilitating neuromuscular disorders.
The success of in utero therapy for SMA challenges existing paradigms of treatment timing and delivery, suggesting that early intervention—potentially initiated during gestation—could prevent irreversible neurological damage more effectively than postnatal treatments. This has profound implications for future drug development targeting other monogenic neurological conditions, advocating for the integration of prenatal diagnostic tools and therapeutic planning into neonatal care. This clinical innovation could dramatically shift global health policies around fetal medicine and pediatric neurology.
Dr. J. Paul Taylor, executive vice president and scientific director at St. Jude and director of PTNI, pointed out the critical unmet clinical need in catastrophic neurological diseases, areas where research has lagged behind oncology. Unlike cancer or sickle cell disease, many neurological disorders have lacked effective disease-modifying therapies. The translational neuroscience platform led by Dr. Finkel is transforming this landscape by combining molecular biology, genetics, and pharmacology to exploit new therapeutic targets and innovative delivery systems, including oral small molecules such as risdiplam.
St. Jude Children’s Research Hospital’s historic mission has evolved from groundbreaking pediatric oncology to embracing complex neurological diseases, leveraging its multidisciplinary expertise and infrastructure. The hospital remains a world leader in pediatric biomedical research, integrating genomic sequencing, cellular biology, and clinical trials to foster therapeutic development. By sharing discoveries openly with global collaborators, St. Jude ensures advances benefit children worldwide, supporting a collaborative, data-driven approach to medicine.
In conclusion, Dr. Richard S. Finkel’s recognition as a TIME100 Health honoree is a testament to his visionary leadership and translational impact in pediatric neurology. His achievements in prenatal treatment for SMA represent a transformative milestone that reshapes how we understand, diagnose, and treat genetic neuromuscular disorders. With ongoing clinical and scientific efforts, Dr. Finkel and the St. Jude team continue to push the boundaries of pediatric neurotherapeutics, offering hope and healing to children and families facing devastating neurological diseases.
Subject of Research: Pediatric Neuromuscular Disorders, Prenatal Therapy for Spinal Muscular Atrophy
Article Title: Richard S. Finkel Named to TIME100 Health 2025 for Pioneering Prenatal Treatment of Spinal Muscular Atrophy
News Publication Date: 2025
Web References:
https://time.com/collections/time100-health-2025/7279665/richard-finkel-kelly-hennings/?filters=pioneers
https://www.stjude.org/directory/f/richard-finkel.html
https://www.stjude.org/research/initiatives/pediatric-translational-neuroscience-initiative.html
https://www.stjude.org/care-treatment/treatment/neurological-disorders/spinal-muscular-atrophy.html
https://www.stjude.org/media-resources/news-releases/2025-medicine-science-news/promising-results-from-first-prenatal-therapy-for-spinal-muscular-atrophy.html
Image Credits: St. Jude Children’s Research Hospital
Keywords: Spinal muscular atrophy, Neurology, Neurological disorders, Pediatrics, Neuropathology
Tags: addressing neurological diseases in childrenCenter for Experimental NeurotherapeuticsDuchenne muscular dystrophy treatmentinnovative clinical interventionspediatric medicine evolutionpediatric neurology advancementspediatric neuromuscular disordersRichard Finkel pediatric neurologistspinal muscular atrophy researchSt. Jude Children’s Research HospitalTIME100 Health list 2025translational neuroscience initiatives
