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Raynor Cerebellum Project

New Directions to Transform Understanding and Therapy of Cerebellar Disorders

The Raynor Cerebellum Project (RCP), anchored at UT Southwestern Medical Center, is one of the major efforts of the Peter O'Donnell Jr. Brain Institute. RCP was established with a generous commitment from the Once Upon a Time Foundation and will dedicate more than $25 million to develop a “virtual institute” supporting independent and collaborative projects in selected thematic areas pursued by investigators across the country and internationally. At least $6 million of the funding will support research outside of UT Southwestern.

The goal of the Raynor Cerebellum Project is to empower novel interdisciplinary research aimed at impacting the lives of patients with cerebellar disease within five to seven years.

The Challenge

Currently, there are no meaningfully effective therapies for patients suffering from cerebellar disease-related motor or cognitive symptoms. The treatment of patients with cerebellar disease is largely empiric and of limited impact. In contrast, the lives of individuals with parkinsonism can be improved dramatically by mechanistically informed pharmacologic or circuit-based therapies. 

There are many reasons for this discrepancy. Perhaps most impactful is the centrality of cerebellar function for motor and cognitive function. Indeed, the challenge of treating cerebellar ataxia has been likened to treating blindness caused by occipital cortical disease; in both situations, the relevant circuits are deafferentated, blinded to the outside world. Another barrier to effective therapy for cerebellar disease is a relative lack of understanding of cerebellar functional organization, both locally and for its extensive CNS connections.

Raynor Cerebellum Project Big Ideas Summit

To meet this challenge, the first investment of the RCP was the Raynor Cerebellum Project's Big Ideas Summit held in September 2022. The summit brought together an interdisciplinary group of ~30 outstanding researchers to brainstorm and identify the most critical questions in the field with a strong but not exclusive focus on advancing areas with the greatest potential to improve the lives of individuals suffering from cerebellar diseases. The goal was to identify and develop 4-6 major funding initiatives that will be used to guide future publication of requests for applications (RFA) by RCP. In keeping with our commitment to fund the best science, applications for funding in response to future RFAs will be open to all investigators and will not be limited to Summit participants.

Summit participants are in an especially advantageous position to receive funding as they will help shape the research initiatives and demonstrate their commitment to RCP’s collaborative vision. 

The challenge to participants of the RCP Summit is to share what they believe to be the most critical questions to be tackled, how best to do so, and to be open to entertaining innovative but perhaps unfamiliar ideas from others.

Examples of big questions might include:

  1. There is not a non-human primate (NHP) model of cerebellar disease similar to the MPTP model of parkinsonism. Would it be valuable to develop an NHP model of cerebellar disease? If so, which aspect of cerebellar dysfunction would be best to model and what physiological or behavioral readouts should be followed?

  2. Could circuit-based neuromodulation (e.g., DBS, TMS) provide a therapeutic opportunity for the treatment of cerebellar disorders? What would we need to know and how should we go about exploring these possibilities?

  3. Related to #2, many ataxias are caused by ion channel mutations with many of these channels concentrated in cerebellar circuits. Are there strategies to therapeutically target these channels?

  4. What can we learn from patients with cerebellar disease using non-invasive monitoring, motion tracking, and related technologies? Can we devise cerebellar-specific rehabilitation strategies based on a systems-level understanding of ataxia and how specific interventions promote recovery? Relatedly, what are the best outcome measures to guide such therapy and how might this therapy benefit from TMS or pharmacological interventions to facilitate plasticity?

  5. The cerebellum receives input from and projects to all cortical areas and many subcortical targets. Clinical and laboratory data increasingly implicate the cerebellum in non-motor cognitive behaviors, especially when cerebellar damage occurs in the developing brain during childhood. How can we better define the character and natural history of these symptoms and define their underlying pathophysiology? How can this knowledge be employed to devise novel therapeutic strategies?

How the Summit Works

The Summit and accompanying virtual sessions were facilitated by Knowinnovation (KI), an international organization that provides services to a variety of organizations whose missions include driving advances in innovation and achieving excellence in research. KI's clients range from national funding bodies to regional colleges and have included National Institutes of Health, the National Science Foundation, and the Engineering and Physical Sciences Research Council UK.

  • In Person

    The Summit will be an intensive, interactive, and free-thinking workshop that aims to stimulate thinking in promising new research approaches and theories that can deepen our understanding of cerebellar disorders. The Summit will bring together PI-level researchers in systems and cognitive neuroscience, molecular/genetic neuroscience, disease modeling, neuroimaging, neurorehabilitation, and human translational neuroscience who are interested in forming new collaborations and developing innovative approaches to address challenges that advance scientific questions related to cerebellar research.

    Participants will be expected to engage constructively in dialogue with each other, the facilitators, and “Subject Guides." Collaboration will be encouraged, especially by bringing diverse minds together to embrace this challenge. The approach of the Summit is not to discuss ideas that are already well developed but not yet published. Rather, the goal is to bring individuals from different disciplines together to interact and engage in free-thinking on first principles, to learn from one another, and create an integrated vision for future research projects. It is expected that the sharing of these ideas will be encouraged within the Summit but their confidentiality will be respected outside the Summit. Interdisciplinary teams will work together to ideate and develop a roadmap for how to tackle selected challenges in this field. Through the course of the three days, teams will form, pitch, and refine ideas (based on input from subject guides and other participants) for interdisciplinary pilot projects. 

  • Virtual

    In addition to the in-person Summit, there will be three virtual sessions. The first will be a networking event scheduled before the Summit. The other sessions are scheduled after the Summit and will give participants an opportunity to further refine and develop their project ideas, receive feedback, and present their final project proposals to the entire group.

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