Study Spotlight: Q-Cell Study for TM Patients
By Hannah Kelly
In a May episode of our “Ask the Expert: Research Edition” podcast, SRNA’s Dr. GG deFiebre and Dr. Benjamin Greenberg discussed an update to an ongoing clinical trial: the Q-Cell Study. The Q-Cell study is the first to investigate using stem cells to repair spinal cord damage in transverse myelitis and is taking place at UT Southwestern in Dallas, Texas. In this blog, we’re exploring the background and status of the study, the screening process, enrollment, and next steps.
The study is the first to investigate stem cells that will form cells that make myelin when placed into human tissue. After transplantation into the spinal cords of patients with transverse myelitis, these cells will hopefully produce myelin. Before reaching this stage, preclinical studies in animals demonstrated safety and efficacy of the stem cells. The FDA also approved the surgical device used to implant these stem cells into the spinal cord. Following a delay due to the COVID-19 pandemic, the first patient was enrolled in December of 2022.
“Where we are today has literally been 20 years in the making. And so, the steps to get to a clinical trial can be complicated, and for cell-based therapies, they’re particularly complicated.” shares Dr. Greenberg.
Exclusion criteria include patients with cervical spinal cord involvement and those able to walk or stand. By enrolling patients with more limited baseline function, the study hopes to minimize potential further damage to the spinal cord from the surgery. If the trial demonstrates that the procedure is safe and effective, the investigators will seek approval to enroll a broader range of patients. At the time the podcast episode was recorded, one surgery had been completed. The study investigators plan to enroll up to nine patients. After a review of their medical records and imaging, potential trial participants visit UT Southwestern for screening visits to ensure they meet inclusion criteria. In the podcast episode, Dr. Greenberg shares, “The discussion really focused around which patients should be first in a clinical trial of stem cells. And the priority, whenever we’re doing a first-in-human trial, is safety. And so, we design the trial to maximize safety and to maximize our ability to identify any danger signals during the course of the trial.”
The surgery involves implanting thawed stem cells into the spinal cord, followed by a short hospital stay. Afterwards, follow-up occurs in outpatient clinics over a period of months to ensure no toxicity occurs from the stem cells or from the immunosuppressive therapies patients must take to allow the stem cells to grow without triggering an unwanted immune response.
For now, the primary aim of this Phase 1/2a study is to determine the safety of the procedure and appropriate dosing requirements. Phase 2b of the study will be with a larger number of patients and will further investigate the safety and efficacy of the procedure. Since transverse myelitis is a rare and serious condition, the FDA may hopefully approve the therapy before conducting later-stage trials.
The podcast audio, video, and transcript are available in the Resource Library under the title “Update: Study to Investigate the Safety of the Transplantation of Human Glial Restricted Progenitor Cells into Patients with Transverse Myelitis.” For more details about the study, please visit the official webpage.
