Alan Finglas to Receive 2024 Patient Advocate Leader (PAL) Award

Congratulations to Alan Finglas, the recipient of the WORLDSymposium 2024 Patient Advocate Leader (PAL) Award. After reviewing numerous nominations, and considering many amazing individuals, the WORLDSymposium 2024 Awards Committee has selected Alan as the recipient of the 2024 PAL Award.

Alan and his wife Michelle founded MSD Action Foundation & in March 2015 along with family and friends, just 5 months after their first born, Dylan, was diagnosed with multiple sulfatase deficiency (MSD). At that time, there was no dedicated charity pursuing research on MSD anywhere in the world. Alan is known for his sheer determination, practical mindset, fundraising abilities, strategic planning, his ability to help secure grants, and being able to discover and foster collaborations. The charity has no employees and Alan’s position is 100% voluntary with zero expenses or travel bursaries.

Alan has played a vital role in changing the previous dynamic in MSD research from simply expanding knowledge, to pursuing therapeutic approaches. Alan guided the foundation to fund 14 translational research projects and he is actively collaborating closely with several other MSD research projects. Alan was key in securing over €500K in additional funding from the Health Research Board, Ireland, in co-funding for international research on MSD. Alan has helped to find MSD patients around the world, promote natural history studies on MSD, discover and validate new biomarkers on MSD, create new animal models, produce new MSD cell lines, and fund multiple drug screening approaches and new drug development approaches.

Alan brought experts together in 2017 for the 1st MSD conference which resulted in consensus clinical care guidelines for MSD being published. These guidelines are deemed to be a gold standard of care for MSD patients. Alan encouraged families in a number of countries, to create charities with similar goals to the MSD Action Foundation to promote and support research on MSD. Alan initiated an effort to apply for a dedicated ICD10-CM code for MSD, which was approved and updated in late 2018. Since 2018, Alan has been on the steering committee, the patient board and the lysosomal sub-network of MetabERN, which is the European Reference Network for inherited metabolic disorders. Alan received a Global Genes ‘Rare Champion of Hope’ award in conjunction with Genetic Disorders UK in London in 2017.

Alan was the key player in establishing a collaboration with AbbVie in relation to a small molecule drug repurposing effort. This collaboration has secured EU funding and involves REMEDI4ALL, Fraunhofer ITMP, University Medical Center Göttingen and EATRIS with assistance from AbbVie, with the goal to move to clinical trials on MSD patients in 2024.

Alan has collaborated with Steven Gray’s MSD AAV9 gene therapy program, The Jackson Laboratory, United MSD Foundation and clinical collaborators since 2018 (funded by United MSD Foundation). The Bespoke Gene Therapy Consortium via the Foundation for the National Institutes of Health (FNIH) awarded funding in May 2023 to further develop this gene therapy program and conduct an MSD clinical trial, and Alan will be a collaborator with the entire team for this clinical trial.

Alan is a husband and father to two boys. Alan is a Carpenter/Joiner for over 25 years, and has worked on set construction and with the shooting crew on a number of movies and TV series. He has brought his problem-solving skills and his extraordinary attention to detail to the lysosomal field. Alan acknowledges that the impact he has made on MSD has only been possible with the help of excellent collaborators and dedicated patient families around the world.

The 2024 Patient Advocate Leader Award will be presented at 7:30 AM PST on Tuesday, February 6, 2024, at the 20th annual WORLDSymposium in San Diego, California.

About the WORLDSymposium Patient Advocate Leader (PAL) Award

Each year, WORLDSymposium recognizes one individual for patient advocacy leadership in the field of lysosomal disease.

Award Nominations now closed.

WORLDSymposium’s Patient Advocate Leader (PAL) Award program began in 2016 in order to recognize individuals contributing directly to lives of patients and families dealing with a lysosomal disease through disease awareness and education, community mobilization, non-profit development and/or good governance activities, patient care, and support programs. The recipient is presented with an engraved award and is recognized at the WORLDSymposium scientific meeting held annually in February each year.

An announcement of the award recipient is included in the February special lysosomes issue of Molecular Genetics and Metabolism. (This award is given on a yearly basis, but can be omitted in any given year at the discretion of the Planning Committee.) Nominations are solicited annually from members of the lysosomal disease community, planning committee members and attendees of WORLDSymposium.

The 2024 Patient Advocate Leader Award will be presented at 7:30 AM PST on Tuesday, February 6, 2024, at the 20th annual WORLDSymposium in San Diego, California.

Past Award Recipients:

2016 Barbara Wedehase, National MPS Society
2017 Christine Lavery, UK Society for Mucopolysaccharide Diseases
2018 Jack Johnson, Fabry Support & Information Group
2019 Mark Dant, EveryLife Foundation for Rare Diseases, National MPS Society
2020 Cara O’Neill, MD, Cure Sanfilippo Foundation
2021 Terri L. Klein, NPGC, National MPS Society, USA
2022 Sue Kahn, National Tay-Sachs & Allied Diseases Association (NTSAD)
2023 Christine Waggoner, Cure GM1 Foundation