Leonard van den Berg

Leonard van den Berg did a fellowship at the Neurological Institute at Columbia University in New York and obtained his PhD degree in 1995 in Utrecht. He has been professor of Experimental Neurology since 2005 and leads a research group focused on translational research into ALS and other diseases of motor neurons. His research has been focused on the search for effective treatment for patients with motor neuron diseases and motor neuropathies by delineating the biological and molecular pathways that initiate and/or drive motor neuron degeneration. These efforts are based on clinical, lifestyle, environmental,genetic and imaging data obtained from large population-based case–control studies. Part of the research activity has been focused on characterizing the phenotype, diagnosis, pathology and treatment options for multifocal motor neuropathy, which is an important treatable ALS mimic. He is principal investigator of the largest, prospective population-based case-control study in ALS (PAN) to provide class I level of evidence of both environmental/lifestyle and genetic factors that determine risk and outcome of ALS and related motor neuron disorders. His research group has strong bioinformatic expertise in genome-wide association studies, copy number variation, gene expression pathways and other systems biology approaches. He is founder and director of The Netherlands ALS Center, which aims to improve the diagnosis, treatment/care and scientific research for ALS in The Netherlands. He promotes international collaborations on ALS research as chairman of the European Network to find the Cure for ALS (ENCALS)) and as coordinator of awarded EU grants (FP7 Euro-MOTOR (systems biology), JPND SOPHIA (biomarkers)).He promotes international collaborations on ALS research as chairman of the European Network for the Cure of ALS (ENCALS)), as coordinator of awarded EU grants (FP7 Euro-MOTOR(systems biology), JPND SOPHIA (biomarkers)) and as initiator of the worldwide ALS genetics research Project MinE.

Prof. Leonard van den Berg has regular interaction with pharmaceutical and other industrial partners. He has not received personal fees or other personal benefits.

UMCU has received major funding by Julius Clinical for participating in clinical studies sponsored by Amylyx and Ferrer.

UMCU has received funding by Apellis and Orphazyme for providing outcome measure training during clinical trials.

UMCU received minor funding (€200-10,000 per industrial partner) for consultation and invited lectures by Neuropath, Orphazyme, Avexis, BrainEver, Amylyx, Argenx, Calico Life Sciences, Ferrer, Biogen, QurAlis, Brainstorm Cell Therapeutics, Arrowhead, Egeen, Corcept, Sanofi, Kadimastem, Medscape, Takeda, Ionis, RRD, Uniqure and Novartis.

UMC Utrecht participation in following clinical trials (all payments are received by the UMC Utrecht)

UMC Utrecht executes the investigator initiated study: Safety and Efficacy of Tauroursodeoxycholic (TUDCA) as add-on Treatment in Patients Affected by Amyotrophic Lateral Sclerosis (ALS). Van den Berg is local principal investigator. The UMC Utrecht sponsors this trial (2019-)

UMC Utrecht executes the industry initiated study: An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis. Van den Berg is local principal investigator. (2020-)

UMC Utrecht executes the industry initiated study: A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene. Van den Berg is local principal investigator. (2020-)

UMC Utrecht executes the industry initiated study: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD). Van den Berg is local principal investigator. (2021-)

UMC Utrecht executes the industry initiated study: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Amyotrophic Lateral Sclerosis. Van den Berg is local principal investigator. (2021-)

UMC Utrecht executes the industry initiated study: A Multi-arm, Adaptive, Group-sequential trial NETwork to evaluate lithium efficacy in patients with Amyotrophic Lateral Sclerosis (ALS). This trial is sponsored by the UMC Utrecht. Van den Berg is local principal investigator. (2021-) TRICALS and the UMC Utrecht sponsor this trial.

UMC Utrecht executes the industry initiated study: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Subjects With Amyotrophic Lateral Sclerosis (ALS). Van den Berg is local principal investigator. (2021-)

UMC Utrecht executes the industry initiated study: A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study, Followed by an Open-Label Extension, to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis. Van den Berg is local principal investigator. (2021-)

UMC Utrecht executes the investigator initiated study study: Targeting Metabolic Flexibility in ALS (MetFlex); Safety and Tolerability of Trimetazidine for the Treatment of ALS. Van den Berg is local principal investigator. The UMC Utrecht sponsors this trial (2021-)

Research line

Neuromuscular Disease, Motor Neuron Disease

Most recent key publications

1: Walhout R, Westeneng HJ, Verstraete E, Hendrikse J, Veldink JH, van den Heuvel MP*, van den Berg LH*. Cortical thickness in ALS: towards a marker for upper motor neuron involvement. J Neurol Neurosurg Psychiatry 2015;86:288-94.
2: Diekstra FP, Van Deerlin VM, van Swieten JC, Al-Chalabi A, Ludolph AC, Weishaupt JH, Hardiman O, Landers JE, Brown RH Jr, van Es MA, Pasterkamp RJ, Koppers M, Andersen PM, Estrada K, Rivadeneira F, Hofman A, Uitterlinden AG, van Damme P, Melki J, Meininger V, Shatunov A, Shaw CE, Leigh PN, Shaw PJ, Morrison KE, Fogh I, Chiò A, Traynor BJ, Czell D, Weber M, Heutink P, de Bakker PI, Silani V, Robberecht W, Veldink JH*, van den Berg LH*. C9orf72 and UNC13A are shared risk loci for amyotrophic lateral sclerosis and frontotemporal dementia: A genome-wide meta-analysis. Ann Neurol 2014;76:120-33.
3: Van den Berg LH. Therapy of amyotrophic lateral sclerosis remains a challenge. Lancet Neurol 2014;13:1062-3
4: Vlam L, van der Pol WL, Cats EA, Straver DC, Piepers S, Franssen H, van den Berg LH. Multifocal motor neuropathy: diagnosis, pathogenesis and treatment strategies. Nature Rev Neurol 2011;8:48-58.
5: Hardiman O, van den Berg LH, Kiernan MC. Clinical diagnosis and management of ALS. Nat Rev Neurol. 2011 Oct 11;7(11):639-49