- Who is conducting this study?
The University of Texas Southwestern, Q Therapeutics, Inc., and The Transverse Myelitis Association are collaboratively sponsoring the first human safety study to treat central nervous system (CNS) disease. Q Therapeutics is a privately-held biotechnology company founded on the work of Dr. Mahendra Rao during his time at the University of Utah and the National Institutes of Health. The University of Texas Southwestern is a medical education and biomedical research institution in the United States. The Transverse Myelitis Association is a patient advocacy organization for individuals with rare neuro-immune disorders (acute disseminated encephalomyelitis, acute flaccid myelitis, MOG antibody-associated disease, neuromyelitis optica spectrum disorder, optic neuritis, and transverse myelitis).
- What are Q-Cells®?
Q Therapeutics developed a glial-restricted precursor cell, called a Q-Cell, that develops into oligodendrocytes. Oligodendrocytes produce myelin, the insulation around nerves, and other factors that are necessary for healthy Central Nervous System (CNS) function. In mice born with nerve cells without myelin, Q-Cells have been shown to produce myelin and other factors that restored the mice to normal function.
- What is the difference between these cells and stem cells?
Stem cells are undifferentiated cells that can turn into other types of cells. Q-cells are not stem cells because they can only turn into oligodendrocytes and astrocytes, the support and repair cells of the CNS.
- How is this study being funded and what are these funds going to be used for?
The cost of the Phase I study is approximately $2.5 million. The CONQUER program at The University of Texas Southwestern secured a $1.5 million gift towards funding of the Phase I trial. The TMA through The Pauline H. Siegel Eclipse Fund and Q Therapeutics, Inc. have committed to raise the remaining funds to cover participant travel expenses, the production and testing of the cells for the trial, and the cost of a contract research organization (CRO) that will manage the clinical trial.
- Who can participate, and why were these inclusion criteria picked?
This study is enrolling patients diagnosed with transverse myelitis who are between one and ten years from their event and remain unable to walk. These criteria were picked because Phase I studies are focused on safety. Given the surgery that is required to implant these cells it was decided to start with a patient population with the least potential for harm and simultaneously the greatest need for intervention. There are other inclusion/exclusion criteria that are listed at clinicaltrials.gov
- How can someone sign up to participate?
Please visit this page to begin the screening process: https://ais.swmed.edu/redcap/surveys/?s=AKMY84JALP
- How many people will participate?
The Phase I trial will include nine non-ambulatory adult transverse myelitis patients.
- How are the cells being used?
The Q-cells will be surgically implanted into the spinal cord at the level of a lesion. Participants will be followed for both safety assessments and multiple measurements to determine if the cells are inducing any level of repair.
- What are the outcomes the study will look at?
As a Phase I study, the majority of outcomes in this study are focused on assuring the safety of this treatment modality. We will look for any complications from surgery, evidence of immune rejection of the cells or abnormal growth of cells. In order to assess for clinical benefit, we will do serial physical exams, MRI imaging and electrophysiology studies to determine if the cells are producing new myelin.
- Are there any risks to participating?
There are always risks to participating in research and those are greatest in Phase I studies, based on our limited experience. In this study there are risks to the surgical procedure, the cell implantation, and the medications used to prevent rejection. All of these elements have been used in other studies and found to be reasonably safe, but they have not been attempted in patients with transverse myelitis. Detailed discussions of risks will occur during the informed consent process.
- What would cause the study to end early?
Studies can be stopped early for many reasons, including concerns about safety, evidence of harm, or regulatory concerns.
- What will it take to get the study to a Phase II or efficacy trial?
If successful, the data from the Phase I study will be used to justify raising the funds necessary for a Phase II trial. Sometimes, a second Phase I trial is needed to explore various dosing options or patient selection criteria. Moving forward from the first study is based on the data collected and funding.
- Is it possible this might cure TM?
After an attack of monophasic transverse myelitis, damage is done to the spinal cord, which causes neurological symptoms such as weakness, paralysis, sensory changes, bladder, bowel, and/or sexual dysfunction etc. Therefore, a cure would be a therapy that repairs the damage to the spinal cord, which improves neurological symptoms. This trial is a safety trial, meaning the safety of this therapy is being assessed. This is the first step needed before it moves into an efficacy trial. It is too early to assess whether this cell therapy will repair the damage to the spinal cord, but it is hoped that it will, as this has also been shown in animal models.
- Since this study is only in transverse myelitis, how may it help people with neuromyelitis optica spectrum disorder, MOG antibody-associated disease, acute disseminated encephalomyelitis, or multiple sclerosis?
If it is shown that this therapy repairs damage to the spinal cord in individuals with transverse myelitis, this therapy may be used to repair damage to the spinal cord from neuromyelitis optica spectrum disorder, MOG antibody-associated disease, acute disseminated encephalomyelitis, multiple sclerosis, or even traumatic spinal cord injuries.
- Where can I find the press release?
You can find the press release about the study here.
- Where can I find the study on Clinicaltrials.gov?
You can find the study on Clinicaltrials.gov here.