EARLY CAREER INVESTIGATOR SPOTLIGHT
What are your research goals?
Dr. Agianda: My research goal is to accelerate the path to effective therapies for children with rare neurogenetic diseases by identifying and validating blood-based biomarkers that can serve as objective, minimally invasive outcome measures in clinical trials. I focus on hereditary spastic paraplegia (HSP), a heterogeneous group of disorders affecting the corticospinal tract, where the absence of validated biomarkers has slowed trial design and made it difficult to detect meaningful treatment effects over short timeframes. Using plasma proteomics platforms, my broader aim is to identify a biomarker robust enough to support regulatory-grade trial endpoints, so that as targeted and gene therapies reach clinical testing, sponsors and families have reliable ways to measure whether a therapy is working, shortening the distance between discovery and meaningful change in children's lives.
How are you involved with the RDCRN?
Dr. Agianda: I serve as the inaugural SP-CERN Career Enhancement Program Fellow, a role created to support early career investigators in building independent research programs within the RDCRN. In this position, I am leading a biomarker development project spanning the 13 SP-CERN sites across the United States, coordinating sample collection, harmonizing proteomic and clinical data, and analyzing plasma biomarkers across the spectrum of hereditary spastic paraplegia subtypes. This multi-site structure gives me access to a patient population far larger and more genetically diverse than any single institution could provide. The fellowship has also given me direct experience with the operational and regulatory complexity of rare disease research networks, from data-sharing agreements to cross-site quality control.
How does participating in the network support your long-term development?
Dr. Agianda: Participating in SP-CERN connects me to a network of physician-scientists across the country who are actively developing and testing therapies for spastic paraplegia, giving me mentorship and collaborative relationships I could not build within a single lab or institution. Being embedded in this community lets me see how biomarker science translates into real trial design decisions, and exposes me to the full arc of rare disease drug development, from natural history studies through regulatory engagement. These relationships are central to my long-term goal of becoming a physician-scientist in pediatric neurology who runs an independent biomarker-driven research program; the network provides both the scientific collaborators and the patient-facing perspective needed to keep my research grounded in what matters most to families.
