CReATe Consortium

The Clinical Research in ALS and related disorders for Therapeutic Develoment (CReATe) Consortium is a collaborative effort to foster the discovery and validation of biomarkers relevant to therapy development for people living with ALS and related disorders including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), and progressive muscular atrophy (PMA). CReATe is a Rare Diseases Clinical Research Consortium (RDCRC) that forms part of the National Institutes of Health (NIH) Rare Diseases Clinical Research Network.

In partnership with The ALS Association, CReATe is able to advance its goals through the release of an annual request for applications to fund biomarker pilot projects. CReATe also fosters biomarker development by making its substantial repository of biological samples available to the broader scientific community.

The ALS Association Commitment

The Association supports three areas through CReATe:

• Pilot Biomarker Study: Dr. Benjamin Murdock at the University of Michigan in Ann Arbor, Mich. The project aims to identify immune cell populations to serve as biomarkers and therapeutic targets in ALS patients. The Association committed $50,000 over 1 year.
• Sample Collection: Dr. Michael Benatar at the University of Miami in Miami. Supports a CReATe Consortium Biorepository to store biological samples collected from all study participants. Biomarker studies require that samples are collected, stored and processed in a harmonized manner, along with collection of clinical data. These samples are used to support biomarker studies within CReATe Consortium and within the broad scientific community. The Association committed $400,000 over 3 years. The CReATe Biorepository was announced in September 2015.
• Sequencing Study: Drs. Paul Taylor and Jinghui Zhang at St. Jude Children’s Hospital in Memphis, Tenn. and Michael Benatar at University of Miami in Miami. The project aims to characterize clinical variability and progression amongst a large cohort of people living with ALS. The investigators plan on performing whole genome sequence analysis of at least 750 CReATe participants. All data will be made public to the ALS community. The Association committed $835,937 over 60 months.

Goals

The overall goal of CReATe is advance the discovery and validation of biomarkers ALS relevant to therapy development for people living with ALS and related disorders. In addition, researchers aim to understand the relationship between clinical phenotype (observable traits) and underlying genotype (DNA sequence).

How CReATe advances the search for a treatment for ALS

• Supports a pilot biomarker project (see above) in partnership with The ALS Association.
• Maintains a CReATe repository (see above) for biological samples from individuals where their observable traits (i.e. phenotypes) were well characterized by Consortium members for future biomarker development. Funding by The Association will allow more extensive samples to be collected, stored and shared with the ALS community.
• Supports CReATe Connect, an international online system to help facilitate communications between doctors/scientists and people living with ALS and their caregivers that works in parallel to the National ALS Registry. There is also an option to be contacted about future clinical research opportunities and to receive updates on the research progress and new educational opportunities sponsored by CReATe.

Key Players

Michael Benatar, M.D., Ph.D., Director of CReATe, Professor of Neurology and Epidemiology and Public Health at the University of Miami in Miami.

The Consortium has sites at the following centers: University of Miami, Florida, University of Kansas Medical Center, University of California at San Diego, California Pacific Medical Center, University of Tübingen, Germany, University of Pennsylvania, The Ohio State University, University of Texas Southwestern, University of Texas Health Sciences Center San Antonio, University of Toronto, University of Iowa, University of Virginia, Wake Forest University and Cleveland Clinic, Ohio.

Progress

As of March 2016, 67 subjects have enrolled and 48 have had their whole genomes sequence completed. CReATe aims to enroll 700+ participants overall.

Website

https://www.rarediseasesnetwork.org/cms/create