Researchers at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital recently created and launched ModelMatcher, a new virtual global networking/matchmaking platform designed to facilitate and enhance the pace of preclinical discovery and therapeutic development for numerous new and existing genetic disorders.
Finding the cause, diagnosis and treatment for a genetic disorder is a highly complex and time-consuming endeavor involving several stakeholders – patients and their families, physicians, scientists, patient support groups and non-profit organizations, pharmaceutical/biotechnology companies and federal agencies – who, despite mutual interests and shared goals, tend to function primarily in separate silos.
ModelMatcher is the brainchild of Duncan NRI investigators and Baylor College of Medicine faculty Dr. Shinya Yamamoto and Dr. Zhandong Liu. Dr. Yamamoto is a fruit fly biologist, and Dr. Liu is a computational biologist. Their teams, including Duncan NRI and Baylor graduate student, Michael Harnish, and staff programmers Lucian Liu and Seon Young Kim, created ModelMatcher as a common online space where diverse stakeholders could come together to connect, interact, generate new ideas, innovate and share discoveries with the common goal of improving the lives of patients and families.
“As a parent of a child diagnosed with a rare disease at Texas Children’s, I know from first-hand experience that basic and preclinical research in animal studies are the starting point for developing viable therapies,” Brian Broadbent, a parent active in the Undiagnosed Diseases Network Participant Engagement and Empowerment Resource (UDN PEER), a patient-representation and advocacy arm of the UDN, said. “Basic scientists, who are at the forefront of cutting-edge discoveries, may not always have access to or close interactions with physicians, patients and families, who bring a wealth of clinical knowledge and critical insights about the day-to-day life of these patients. These insights have the potential to transform research and speed up the pace of discovery.”
Similarly, patients and families often lack access to scientific or clinical collaborations and discussions.
“Our goal in creating this online networking tool was to remove these systemic barriers by creating an open, transparent and international collaborative ecosystem with established guidelines where seamless scientific and clinical collaborations can take place,” Yamamoto said. “We are hopeful that this will speed up the entire drug discovery pipeline, from the initial gene discovery to the development and delivery of safe and effective therapies.”
A desire for commitment and collaboration is at the core of ModelMatcher. Scientists with diverse expertise are asked to register information about themselves and provide a list of genes that fall into their area of expertise. Clinicians seeking information about specific genetic variants that are identified as the potential cause of rare diseases can then easily identify these scientists and connect with them directly and establish collaborations to facilitate diagnosis and/or therapeutic research.
Additionally, this platform will immediately increase the opportunity for seamless partnering among patients, patient families, support groups, clinicians and scientists. The goal is to advance research and patient care in a way that is not currently possible. Interested scientists (including graduate students, postdoctoral fellows and staff), clinicians, patients/families and other stakeholders of rare and undiagnosed disease research can register here to get connected.
In the long term, the ModelMatcher creators envision that this platform will also expedite drug discovery by creating and facilitating collaborative networks among pharmaceutical and biotech companies, venture capitalists and federal agencies with a common goal of accelerating the discovery process for specific disorders.
“ModelMatcher is a highly anticipated one-of-a-kind networking platform that has rightfully earned every bit of the excitement surrounding it and is a giant leap forward in opening up possibilities for newer treatments and therapeutic options,” Troy Evans, a rare disease patient and UDN PEER Co-Chair, said. “The ability to address the growing need for connecting rare disease patients and families with key stakeholders is a monumental step that will improve our lives in tangible ways,”
The ModelMatcher team is grateful to the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Baylor College of Medicine, the Undiagnosed Diseases Network and the Rare Diseases Models and Mechanisms Network for their support.