![Helen Waller-Evans](https://profiles-cardiff.imgix.net/attachments/1245?w=200&h=200&auto=format&crop=faces&fit=crop&q=90")
Dr Helen Waller-Evans
- Welsh speaking
Teams and roles for Helen Waller-Evans
Lecturer
School of Biosciences
Overview
I am a neuroscience lecturer at the Medicines Discovery Institute. In order to develop better neuroscience drugs, we need to fully understand the roles of potential drug targets and the pathways they are involved in. Work in my laboratory focusses on characterising the function of potential neuroscience drug targets and developing assays to assess the effects of novel drugs on this function. I have a particular interest in developing therapies that act on lysosomal proteins for use in psychiatric and neurological diseases.
Publication
2025
- Best, H. L. et al. 2025. Niemann-Pick C-like endo-lysosomal dysfunction in DHDDS patient cells, a congenital disorder of glycosylation, can be treated with miglustat. International Journal of Molecular Sciences 26 (4) 1471. (10.3390/ijms26041471)
- Kirkham, E. et al., 2025. Lysosomal pH measurements via dual-imaging microscopy. In: Hughes, S. M. and Basak, I. eds. Lysosomes: Methods and Protocols. Vol. 2976, Methods in Molecular Biology New York, New York, USA: , pp.11-24. (10.1007/978-1-0716-4844-5_2)
- Reeks, J. et al., 2025. High throughput cryo-EM provides structural understanding for modulators of the lysosomal ion channel TRPML1. Structure 33 (8), pp.1374-1385. (10.1016/j.str.2025.05.014)
- Riseley, R. et al., 2025. Unusual reactivity of 2-amino-1H-indole-3-carboxylate esters. ARKIVOC 2025 (5) 202512417. (10.24820/ark.5550190.p012.417)
2024
- Cubitt, J. et al. 2024. Beware of N-Benzoyloxybenzamides. Molecules 29 (21) 5143. (10.3390/molecules29215143)
2023
- Best, H. et al. 2023. The Batten disease associated protein CLN3 is required for the efflux of lysosomal K. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.47-47. (10.1016/j.ymgme.2022.107030)
- Best, H. L. et al. 2023. Disease phenotypes present in CLN7 patient fibroblasts are improved following treatment to reduce glycosphingolipid synthesis.. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.17. (10.1016/j.ymgme.2022.107029)
- Best, H. L. et al. 2023. The role of glycoconjugates as receptors for insecticidal proteins. FEMS Microbiology Reviews 47 (4)(10.1093/femsre/fuad026)
- Cook, S. R. et al. 2023. Investigating lysosomal and cellular phenotypes of the lysosomal disorder cystinosis. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.28-28. (10.1016/j.ymgme.2022.107061)
- Davies, M. et al. 2023. Acid ceramidase inhibition as a mechanism to treat lysosomal disorders. Molecular Genetics and Metabolism 138 (2) 107069. (10.1016/j.ymgme.2022.107069)
- Fenn, G. D. et al. 2023. Development of a novel anthraquinone-derived fluorescent lysosomal probe. Molecular Genetics and Metabolism 138 (2) 107097. (10.1016/j.ymgme.2022.107097)
- Fenn, G. D. , Lloyd-Evans, E. and Waller-Evans, H. 2023. Investigation of cannabidiol as a potential therapeutic in Niemann-Pick disease. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.42-42. (10.1016/j.ymgme.2022.107098)
- Honeybun, L. , Waller-Evans, H. and Lloyd-Evans, E. 2023. Unbiased phenotypic drug screen for CLN3 disease. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.167-167. (10.1016/j.ymgme.2022.107150)
- Lloyd-Evans, E. et al. 2023. Glycosphingolipid reduction with miglustat as a therapeutic strategy for CLN3 and other neuronal ceroid lipofuscinoses.. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.84. (10.1016/j.ymgme.2022.107212)
- Somogyi, A. et al., 2023. The synthetic TRPML1 agonist ML-SA1 rescues Alzheimer-related alterations of the endosomal-autophagic-lysosomal system. Journal of Cell Science 136 (6) jcs259875. (10.1242/jcs.259875)
- Waller-Evans, H. et al. 2023. Filipin composition and imaging modality dramatically affect cellular cholesterol visualisation with implications for assessing cholesterol levels and localisation across the lysosomal disorders. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.133. (10.1016/j.ymgme.2022.107352)
- Waller-Evans, H. et al. 2023. Measuring ion flux via lysosomal channels: Primary and secondary assays for drug discovery across the lysosomal disorders. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.133-134. (10.1016/j.ymgme.2022.107353)
2022
- Best, H. L. et al. 2022. The crystal structure of Bacillus thuringiensis Tpp80Aa1 and its interaction with galactose-containing glycolipids. Toxins 14 (12) 863. (10.3390/toxins14120863)
2020
- Badell Grau, R. A. et al. 2020. Investigating the prevalence of reactive online searching in the COVID-19 pandemic. Journal of Medical Internet Research 22 (10) e19791. (10.2196/19791)
- Cook, S. R. et al. 2020. Detrimental effect of zwitterionic buffers on lysosomal homeostasis in cell lines and iPSC-derived neurons. AMRC Open Research 2 21. (10.12688/amrcopenres.12903.1)
- Cook, S. R. et al. 2020. Visualisation of cholesterol and ganglioside GM1 in zebrafish models of Niemann-Pick type C disease and Smith-Lemli-Opitz syndrome using light sheet microscopy. Histochemistry and Cell Biology 154 , pp.565-578. (10.1007/s00418-020-01925-2)
- Fenn, G. et al. 2020. Crystallization and structure of ebselen bound to cysteine 141 of human inositol monophosphatase (IMPase). Acta Crystallographica Section F: Structural Biology Communications F76 (10), pp.469-476. (10.1107/S2053230X20011310)
- Lloyd-Evans, E. and Waller-Evans, H. 2020. Biosynthesis and signalling functions of central and peripheral nervous system neurosteroids in health and disease. Essays in Biochemistry 64 (3), pp.591-606. (10.1042/EBC20200043)
2018
- Morimoto, M. et al., 2018. Bi-allelic CCDC47 variants cause a disorder characterized by woolly hair, liver dysfunction, dysmorphic features, and global developmental delay. American Journal of Human Genetics 103 (5), pp.794-807. (10.1016/j.ajhg.2018.09.014)
2015
- Waller-Evans, H. and Lloyd-Evans, E. 2015. Regulation of TRPML1 function. Biochemical Society Transactions 43 (3), pp.442-446. (10.1042/BST20140311)
2014
- Kaisaki, P. J. et al., 2014. Genetic control of differential acetylation in diabetic rats. PLoS ONE 9 (4) e94555. (10.1371/journal.pone.0094555)
2013
- Waller-Evans, H. et al. 2013. Nutrigenomics of high fat diet induced obesity in mice suggests relationships between susceptibility to fatty liver disease and the proteasome. PLoS ONE 8 (12) e82825. (10.1371/journal.pone.0082825)
2012
- Prömel, S. et al., 2012. Characterization and functional study of a cluster of four highly conserved orphan adhesion-GPCR in mouse. Developmental Dynamics 241 (10), pp.1591-1602. (10.1002/dvdy.23841)
2010
- Lloyd-Evans, E. et al. 2010. Endolysosomal calcium regulation and disease.. Biochemical Society Transactions 38 (6), pp.1458-1464. (10.1042/BST0381458)
- Waller-Evans, H. et al. 2010. The orphan adhesion-GPCR GPR126 is required for embryonic development in the mouse. PLoS ONE 5 (11) e14047. (10.1371/journal.pone.0014047)
2009
- Langenhan, T. et al., 2009. Latrophilin signaling links anterior-posterior tissue polarity and oriented cell divisions in the C. elegans embryo. Developmental Cell 17 (4), pp.494-504. (10.1016/j.devcel.2009.08.008)
Articles
- Badell Grau, R. A. et al. 2020. Investigating the prevalence of reactive online searching in the COVID-19 pandemic. Journal of Medical Internet Research 22 (10) e19791. (10.2196/19791)
- Best, H. L. et al. 2025. Niemann-Pick C-like endo-lysosomal dysfunction in DHDDS patient cells, a congenital disorder of glycosylation, can be treated with miglustat. International Journal of Molecular Sciences 26 (4) 1471. (10.3390/ijms26041471)
- Best, H. L. et al. 2023. The role of glycoconjugates as receptors for insecticidal proteins. FEMS Microbiology Reviews 47 (4)(10.1093/femsre/fuad026)
- Best, H. L. et al. 2022. The crystal structure of Bacillus thuringiensis Tpp80Aa1 and its interaction with galactose-containing glycolipids. Toxins 14 (12) 863. (10.3390/toxins14120863)
- Cook, S. R. et al. 2020. Detrimental effect of zwitterionic buffers on lysosomal homeostasis in cell lines and iPSC-derived neurons. AMRC Open Research 2 21. (10.12688/amrcopenres.12903.1)
- Cook, S. R. et al. 2020. Visualisation of cholesterol and ganglioside GM1 in zebrafish models of Niemann-Pick type C disease and Smith-Lemli-Opitz syndrome using light sheet microscopy. Histochemistry and Cell Biology 154 , pp.565-578. (10.1007/s00418-020-01925-2)
- Cubitt, J. et al. 2024. Beware of N-Benzoyloxybenzamides. Molecules 29 (21) 5143. (10.3390/molecules29215143)
- Davies, M. et al. 2023. Acid ceramidase inhibition as a mechanism to treat lysosomal disorders. Molecular Genetics and Metabolism 138 (2) 107069. (10.1016/j.ymgme.2022.107069)
- Fenn, G. et al. 2020. Crystallization and structure of ebselen bound to cysteine 141 of human inositol monophosphatase (IMPase). Acta Crystallographica Section F: Structural Biology Communications F76 (10), pp.469-476. (10.1107/S2053230X20011310)
- Fenn, G. D. et al. 2023. Development of a novel anthraquinone-derived fluorescent lysosomal probe. Molecular Genetics and Metabolism 138 (2) 107097. (10.1016/j.ymgme.2022.107097)
- Kaisaki, P. J. et al., 2014. Genetic control of differential acetylation in diabetic rats. PLoS ONE 9 (4) e94555. (10.1371/journal.pone.0094555)
- Langenhan, T. et al., 2009. Latrophilin signaling links anterior-posterior tissue polarity and oriented cell divisions in the C. elegans embryo. Developmental Cell 17 (4), pp.494-504. (10.1016/j.devcel.2009.08.008)
- Lloyd-Evans, E. and Waller-Evans, H. 2020. Biosynthesis and signalling functions of central and peripheral nervous system neurosteroids in health and disease. Essays in Biochemistry 64 (3), pp.591-606. (10.1042/EBC20200043)
- Lloyd-Evans, E. et al. 2010. Endolysosomal calcium regulation and disease.. Biochemical Society Transactions 38 (6), pp.1458-1464. (10.1042/BST0381458)
- Morimoto, M. et al., 2018. Bi-allelic CCDC47 variants cause a disorder characterized by woolly hair, liver dysfunction, dysmorphic features, and global developmental delay. American Journal of Human Genetics 103 (5), pp.794-807. (10.1016/j.ajhg.2018.09.014)
- Prömel, S. et al., 2012. Characterization and functional study of a cluster of four highly conserved orphan adhesion-GPCR in mouse. Developmental Dynamics 241 (10), pp.1591-1602. (10.1002/dvdy.23841)
- Reeks, J. et al., 2025. High throughput cryo-EM provides structural understanding for modulators of the lysosomal ion channel TRPML1. Structure 33 (8), pp.1374-1385. (10.1016/j.str.2025.05.014)
- Riseley, R. et al., 2025. Unusual reactivity of 2-amino-1H-indole-3-carboxylate esters. ARKIVOC 2025 (5) 202512417. (10.24820/ark.5550190.p012.417)
- Somogyi, A. et al., 2023. The synthetic TRPML1 agonist ML-SA1 rescues Alzheimer-related alterations of the endosomal-autophagic-lysosomal system. Journal of Cell Science 136 (6) jcs259875. (10.1242/jcs.259875)
- Waller-Evans, H. et al. 2013. Nutrigenomics of high fat diet induced obesity in mice suggests relationships between susceptibility to fatty liver disease and the proteasome. PLoS ONE 8 (12) e82825. (10.1371/journal.pone.0082825)
- Waller-Evans, H. and Lloyd-Evans, E. 2015. Regulation of TRPML1 function. Biochemical Society Transactions 43 (3), pp.442-446. (10.1042/BST20140311)
- Waller-Evans, H. et al. 2010. The orphan adhesion-GPCR GPR126 is required for embryonic development in the mouse. PLoS ONE 5 (11) e14047. (10.1371/journal.pone.0014047)
Book sections
- Kirkham, E. et al., 2025. Lysosomal pH measurements via dual-imaging microscopy. In: Hughes, S. M. and Basak, I. eds. Lysosomes: Methods and Protocols. Vol. 2976, Methods in Molecular Biology New York, New York, USA: , pp.11-24. (10.1007/978-1-0716-4844-5_2)
Conferences
- Best, H. et al. 2023. The Batten disease associated protein CLN3 is required for the efflux of lysosomal K. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.47-47. (10.1016/j.ymgme.2022.107030)
- Best, H. L. et al. 2023. Disease phenotypes present in CLN7 patient fibroblasts are improved following treatment to reduce glycosphingolipid synthesis.. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.17. (10.1016/j.ymgme.2022.107029)
- Cook, S. R. et al. 2023. Investigating lysosomal and cellular phenotypes of the lysosomal disorder cystinosis. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.28-28. (10.1016/j.ymgme.2022.107061)
- Fenn, G. D. , Lloyd-Evans, E. and Waller-Evans, H. 2023. Investigation of cannabidiol as a potential therapeutic in Niemann-Pick disease. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.42-42. (10.1016/j.ymgme.2022.107098)
- Honeybun, L. , Waller-Evans, H. and Lloyd-Evans, E. 2023. Unbiased phenotypic drug screen for CLN3 disease. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.167-167. (10.1016/j.ymgme.2022.107150)
- Lloyd-Evans, E. et al. 2023. Glycosphingolipid reduction with miglustat as a therapeutic strategy for CLN3 and other neuronal ceroid lipofuscinoses.. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.84. (10.1016/j.ymgme.2022.107212)
- Waller-Evans, H. et al. 2023. Filipin composition and imaging modality dramatically affect cellular cholesterol visualisation with implications for assessing cholesterol levels and localisation across the lysosomal disorders. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.133. (10.1016/j.ymgme.2022.107352)
- Waller-Evans, H. et al. 2023. Measuring ion flux via lysosomal channels: Primary and secondary assays for drug discovery across the lysosomal disorders. Presented at: 19th Annual WORLDSymposium™ 2023 Orlando, Florida 21-26 February 2023. Vol. 138.Vol. 2. Elsevier. , pp.133-134. (10.1016/j.ymgme.2022.107353)
Research
Research Interests
My research focuses on elucidating the functions of lysosomal proteins and their roles in neurological disorders, and developing novel drugs that target these proteins for use in both diseases of ageing and lysosomal storage disorders - rare genetic diseases caused by defects in lysosomal proteins.
My main research interest is the NPC1 protein, a lysosomal protein with a suggested role in cholesterol transport. Loss of NPC1 function auses Niemann-Pick type C disease, a lysosomal storage disorder, where affected childern have difficulty walking and swallowing, learning difficulties, epilepsy and dementia. Current treatments can slow disease progression but do not tackle the fundamental lack of NPC1 activity. I am developing functional assays for the lysosomal NPC1 protein, which will allow me to conduct drug screens for molecules that can activate NPC1 in Niemann-Pick type C patients.
NPC1 is also used as an intracellular receptor by several viruses, including Ebola, hepatitis C, and feline coronaviruses. I am investigating whether drugs that inhibit the NPC1 protein can prevent SARS-CoV-2 entering cells, which could enable their rapid re-purposing to treat Covid-19.
Current lab members
Gareth Fenn - PhD student
Sian Gardiner - senior technician
Marcus Hanley - PhD student
Bethany Tattersdill - research assistant
Gabby Ward - technician
Collaborators
Dr Emyr Lloyd-Evans (School of Biosciences, Cardiff University)
Dr Ceri Fielding (School of Medicine, Cardiff University)
Prof. Simon Pope (School of Chemistry, Cardiff University)
Prof. Colin Berry (School of Biosciences, Cardiff University)
Funding
Research in my laboratory is funded by the Academy of Medical Sciences, Niemann-Pick Research Foundation, Welsh Government, and Harrington Research Institute.
Biography
Career History
2019 - present: Neuroscience lecturer, Medicines Discovery Institute, Cardiff University, where I develop novel drugs targeting lysosomal proteins
2012 - 2019: Research Associate with Dr Emyr Lloyd-Evans, School of Biosciences, Cardiff University. Here I investigated the mechanisms leading to pathogenesis in several lysosomal storage disorders, and is where I developed my interest in lysosomes.
2010 - 2012: Research Associate with Prof. Alun Davies FRS, School of Biosciences, Cardiff University. Here I investigated the role of growth factors in neuronal development, and how this is affected in neurological disorders including Parkinson disease.
2008 - 2010: Research Associate with Prof. Dominique Gauguier, University of Oxford. Here I investigated the contribution of different genes to the severity of type 2 diabetes.
2005 - 2008: PhD with Dr Andreas Russ, University of Oxford, investigating the role of GPR126, an orphan GPCR, in mouse development.
2004 - 2005: Technician with Dr Andreas Russ, University of Oxford.
2003 - 2004: Postgraduate Diploma in Psychology (distinction), Oxford Brookes University.
2002 - 2003: Histologist with Prof. Nick Rawlins, University of Oxford.
1998 - 2002: BSc Hons (1st class) Applied Biology, University of Bath.
