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 and key partners in the industry and academic research communities, including Biohaven Pharmaceuticals, Ionis Pharmaceuticals, Roche Pharmaceuticals, Servier Group, Triplet Therapeutics, uniQure, 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), which can manifest in impaired gait, coordination of movement and speech. Such a 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 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
CPTA creates a neutral space where stakeholders, including regulators, from across different fields and backgrounds can come together in the precompetitive space to share their expertise, insights, and data to collaborate on development of solutions to common medical product development issues. Data contributed to CPTA is hosted on C-Path’s Rare Disease Cures Accelerator – Data and Analytics Platform, an FDA-funded initiative that provides a centralized and standardized infrastructure for data across rare diseases. Sharing data on this platform multiplies the impact of ataxia data and facilitates advanced data analysis approaches that would be impossible from individual studies in isolation. 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.
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 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.