Who we are
Officially launched in February 2021, the Critical Path to Therapeutics for the Ataxias (CPTA) is a public-private partnership with the mission to optimize clinical trials for inherited ataxias. CPTA is a collaborative effort between C-Path, the Ataxia Global Initiative, the National Ataxia Foundation, Ataxia UK, the Ataxia Charlevoix-Saguenay Foundation and key partners in the industry and academic research communities, including Biogen, Biohaven Pharmaceuticals, Ionis, PTC Therapeutics, Roche, Servier Pharmaceuticals, Triplet Therapeutics, and VICO Therapeutics. Through facilitating a collaborative and consensus-driven community of patient advocates, researchers, and regulators, CPTA aims to work collaboratively to develop tools and complete analyses to accelerate the development of new therapies for inherited ataxias.
Inherited ataxias are a diverse collection of genetic disorders that are characterized by ataxia (impaired coordination of voluntary movements, such as gait and speech). The clinical presentation is typically associated with atrophy of the part of the brain called the cerebellum. Each individual ataxia has a unique genetic cause and spectrum of clinical presentation, affecting many different body systems. For example, spinocerebellar ataxias (SCAs) often include dysphagia, stiffness, hyperreflexia, spasticity, and some cognitive impairment, while autosomal recessive ataxias often include peripheral nerve neuropathy and seizures, but also many additional symptoms specific to each disease.
Although the pipeline of new medical products for the ataxias is expanding, there remain large gaps in our understanding of these diverse disorders. In particular, as novel therapies approach the clinic, there is an urgent need for actionable tools to understand the natural history, progression, and variance in these diseases, as well as how to measure therapeutic effects in clinical trials.
Our mission is to bring together experts from across different fields of ataxia research, advocacy, and medical product development to create regulatory tools and strategies that will catalyze research in the ataxias. In doing so, we aim to increase the efficiency and effectiveness of medical product development for these disorders and deliver innovative treatments to patients in need.
How we do it
Critical Path Institute (C-Path)’s Critical Path to Therapeutics for the Ataxias (CPTA) consortium creates a neutral, pre-competitive environment where stakeholders, including industry drug development professionals, subject matter experts and regulators, can collaborate and share their expertise, insights, and data to generate novel quantitative solutions to accelerate drug development. Data contributed to CPTA is hosted on C-Path’s Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP), an FDA-funded initiative that provides a centralized and standardized infrastructure for data across rare and orphan diseases. Sharing data on this platform enhances the impact of ataxia data and facilitates advanced analytical approaches. These data will inform ataxia research, leading to a better understanding of these disorders, how disease progression and drug effects are measured, and will help optimize the development of novel medical products for inherited ataxias.
CPTA is focused on collaboratively driving advancement in the following areas: 1) Analysis of existing and new endpoints to optimize measurement of disease progression and drug effects in the various ataxias. 2) Driving towards regulatory acceptance of new endpoints and/or biomarkers for use in ataxia clinical trials. 3) Aggregation and sharing of existing clinical data across the ataxias to inform community understanding of natural history of the different ataxias.