Success Story - When Standard Treatments Fail: A Case for Virus-Specific T Cell Therapy

Key Takeaway:

• Virus-specific T cells (VSTs) used to successfully treat a newborn with a life-threatening CMV infection

• RareKids-CAN and the Riddell Centre for Cancer Immunotherapy supported the Health Canada regulatory submission for this project

• Alberta Children’s Hospital, Alberta Precision Laboratories, and the Riddell Centre for Cancer Immunotherapy worked together on this innovative therapy

• The baby is now free from detectable CMV after receiving the experimental therapy

• The success highlights the importance of local research, clinical infrastructure, and biomanufacturing capabilities in treating rare, severe conditions

In early 2025, a multidisciplinary team at the Alberta Children’s Hospital, Alberta Precision Laboratories, and the Riddell Centre for Cancer Immunotherapy came together to deliver a treatment via a single patient study for a baby girl with a rare and severe immune disorder caused by a genetic mutation. The child was battling a life-threatening cytomegalovirus (CMV) infection that had not responded to standard antiviral therapies and was preparing to undergo a stem cell transplant—the only potential cure for her condition.

However, the transplant required temporary immune suppression, which risked allowing the CMV infection - which had not responded to antiviral treatment - to worsen. To address this, the clinical team turned to an experimental therapy known as virus-specific T cells (VSTs). These immune cells were collected from one of the patient’s parents and engineered in the lab to selectively target CMV-infected cells. The manufacturing and quality control of the VSTs were carried out by Alberta Precision Labs in collaboration with the Riddell Centre’s Biomanufacturing Program. The Health Canada regulatory submission was supported by the Riddell Centre and RareKids-CAN.

This case demonstrates the successful use of virus-specific T cells to treat a life-threatening infection in a newborn—enabled by local manufacturing and clinical research infrastructure. The patient successfully received the therapy and, as of today, no longer has a detectable CMV infection

This initiative was made possible thanks to the generous funds provided by the Barb Ibbotson Chair in Pediatric Hematology, Alberta Children’s Hospital Foundation and the efforts of many groups coming together and generously contributing their time and efforts in-kind.

This case highlights how experimental cell therapies can offer hope to patients facing life-threatening conditions with no remaining standard options. By using virus-targeting immune cells from a parent, clinicians were able to control a severe infection and give this child a chance at a life-saving transplant.

While the Riddell Centre is primarily focused on advancing novel cell therapies for cancer, the infrastructure and expertise developed within the Centre are enabling rapid collaboration on other urgent medical challenges. This project exemplifies how the Centre’s capabilities in cell and gene therapy can be extended beyond oncology—bringing together science, compassion, and collaboration to deliver innovative treatments for patients with rare and complex conditions.