Ataxia Advisory Committee for Therapeutics

The Ataxia Advisory Committee for Therapeutics (Ataxia ACT) aims to optimize the planning and execution of preclinical and clinical drug development programs for (all forms of) ataxias. Ataxia ACT provides confidential, unbiased, multi-stakeholder and multidisciplinary expert advice to optimize therapy development, by either a) steering studies along a realistic and well-informed plan to clinical trial/later phase clinical activity or b) encouraging a change of direction or approach, to avoid failure further down the line. It is based on the blueprint of the successful Treat-NMD TACT.

Ataxia ACT can address: 

  • Trial design
  • Drug formulation
  • Bioavailability and toxicology
  • Regulatory considerations
  • Recommendations including go-no-go milestones

The Ataxia ACT meets twice a year: once in the autumn and once in the spring. The applicants are asked to pay a contribution towards the meeting costs.

The Ataxia ACT process is independent of any funding stream, but applicants can use its reports to support funding applications. An Ataxia ACT review is NOT an endorsement by Ataxia ACT or Ataxia Global Initiative that a drug has any particular status in terms of regulatory or funding potential.

Terms of Reference
Pre-Application Form

Upcoming Ataxia Advisory Committee Meetings

11 and 12 November 2024, London, UK

Pre-application deadline: 1 July 2024
Full application deadline: 1 August 2024

Two places are currently available


Spring 2025, USA

Four places are currently available.


Autumn 2025, tbc

Four places are currently available


Interested in coming to Ataxia ACT?

Get in touch with us at ataxiaglobaloffice [at] med.uni-tuebingen.de to talk about how we can help improve your therapeutic development program.

Ataxia ACT Core Committee

Guided by two chairs, the Ataxia ACT Core Committee makes decisions on the strategic direction of the ACT and reviews and provides feedback on all potential applications. The Core Committee covers a wide range of expertise in a drug development process. Core members attend every ACT meeting and review each application unless a conflict of interest has been identified.

 

Adelaide Fernandes Borralho

Adelaide Fernandes is an Associate Professor. She received her degree in Pharmaceutical Sciences in 2002 and her PhD in Pharmacy (Biochemistry) in 2006, from the University of Lisbon.

Adelaide's research focuses on inflammation-associated disorders. Main projects address inflammatory cross-talk between Central Nervous System resident cells, immune cells and peripheral inflammation in neurodegenerative disorders, as Multiple Sclerosis, Alzheimer’s Disease and ARSACS.

Maria Judit Molnar

Judit Molnar is a Professor of Neurology, Psychiatry, Clinical Genetics, and Clinical Pharmacology, Doctor of the Hungarian Academy of Sciences, director of Semmelweis University’s Institute of Genomic Medicine and Rare Disorders.

She is the member of the Committee of Orphan Medicinal Products of the EMA, among others president of the Hungarian Medical College of Clinical Genetics, past president of the Hungarian Human Genetic Society, elected Co- Chair of the Neurogenetic Scientific Panel of the EAN, secretary of the Hungarian Society of Personalized Medicine.

Susan Perlman

Core Committee Chair

Susan Perlman has had clinical experience with the diagnosis and management of Friedreich’s ataxia and Spinocerebellar ataxia patients and families for 35 years and has participated in multiple clinical trials.

She is an active member of the Collaborative Clinical Research Network for Friedreich’s ataxia and the Clinical Research Consortium for Spinocerebellar Ataxia (CRC-SCA) and Director of our Ataxia Center of Excellence.

Thorsten Schmidt

Thorsten Schmidt has been working for almost 25 years in the field of inherited, neurodegenerative disorders and in particular on ataxias.

He is leading since 2006 the SCA3 research group and since 2015 the Research laboratory at the Institute of Medical Genetics and Applied Genomics at the University Hospital Tübingen.

Barbara Tate

Barbara Tate, PhD, has been the Chief Scientific Officer of the Friedreich Ataxia Research Alliance since spring of 2020. Previously, she was the Chief of Strategy and a venture partner at the Dementia Discovery Fund (DDF), SV Life Sciences. She has worked in both large and small pharmaceutical companies, where she has held executive positions.

She was a founder and the first CEO of Tiaki Therapeutics, a DDF portfolio company. She was also a founder of Ataxion (later Cadent) and a Founder and the Vice President and Head of Biology at Rodin Therapeutics, both Atlas Venture companies.

Sophie Tezenas du Montcel

Since 2022: PI - ARAMIS Team, Paris Brain Institute, INRIA, INSERM

Since 2017: Associate professor - Pitié-Salpêtrière Hospital (Medical information department) and Sorbonne Université, Paris

2004-2017: Associate professor - Pitié-Salpêtrière Hospital (Biostatistics department) and UPMC/Sorbonne Université, Paris

2003: PhD Genetic Epidemiology - University Paris XI, Paris, France

1998-2004: Assistant Professor - Pitié-Salpêtrière Hospital and UPMC, Paris

1998: Medical degree (MD) - Université Aix - Marseille II, France

Bart van de Warrenburg

Core Committee Chair

Bart van de Warrenburg is professor of Neurology and research group leader at the Radboudumc, Nijmegen, Ther Netherlands. He was trained in neurology at the same center and was a movement disorders fellow at Queen Square, London, UK.

He is founder and head of the Radboudumc expert center for rare and genetic movement disorders (appointed by the Minister of Health and also formal partner in the European Reference Network for Rare Neurological Disorders). He is a recognized clinical expert in rare movement disorders, with a focus on ataxias.

Willeke van Roon-Mom

One of the disease areas Willeke van Roon-Mom has been working on for more than 20 years are Spinocerebellar Ataxias caused by CAG repeat expansions in the coding regions of genes. This research has focused on studying disease mechanisms and developing splice modulating RNA targeting therapies. She further expanded her expertise in the use of induced pluripotent stem cell (iPSC) based models and animal models for neurodegenerative diseases.

Willeke is a co-director of the Dutch Center for RNA Therapies (DCRT), which focus is on developing RNA targeting treatments for patients with unique mutations (RNAtherapy.nl).

Ataxia ACT Extended Committee

In addition to the Core Committee who attend all Ataxia ACT reviews, we have a pool of global experts who are referred to as our extended committee. For each Ataxia ACT application, a bespoke panel is put together with invited reviewers selected from the extended committee who are best placed to answer specific questions and provide tailored multidisciplinary advice.