Q&A with Dr. Steinberg
Stroke Progress and Innovation Awards 2016
Gary K. Steinberg, MD, PhD
Gary K. Steinberg, Douglas Kondziolka, Lawrence R. Wechsler, L. Dade Lunsford, Maria L. Coburn, Julia B. Billigen, Anthony S. Kim, Jeremiah N. Johnson, Damien Bates, Bill King, Casey Case, Michael McGrogan, Ernest W. Yankee, Neil E. Schwartz
SPOTLIGHT: Q&A with Dr. Steinberg
What is the key take-away message from your article?
This was the first clinical trial for stroke patients in North America using intracerebral transplantation of stem cells. These patients were suffering from chronic motor disability 6 months to 3 years following their stroke. Although it was a small Phase 1/2a study of 18 patients, it demonstrated not only that the procedure was safe and feasible, but a statistically significant improvement in neurologic recovery. It also changes our prior notion of what happens following a stroke. Since chronic stroke patients generally reach maximal recovery by 6 months, we previously thought the underlying neural circuits were “dead’ or irreversibly injured after that time. We now know that it is possible to resurrect these circuits in certain stroke patients, even years after the stroke.
What prompted you and your co-authors to perform this study?
Stroke is the leading cause of adult disability, with the highest annual incidence of any neurological disorder, including Alzheimer’s disease, traumatic brain injury, epilepsy and Parkinson’s disease. Each year 795,000 people in the U.S. will experience stroke, and approximately 7 million people in the U.S. are currently living with disability from stroke. Stroke-related costs are more than $34 billion dollars a year, and these numbers are predicted to rise as the age of the general population increases. Due to improved acute medical management, the stroke mortality rate is declining. Thus, the high annual incidence of stroke and declining mortality rate have changed stroke from an acute killer to a chronic disabling disease. Yet very few treatment options exist to target this chronic phase of stroke to improve the neurologic functional status and recovery of patients. Such treatments for recovery remain focused on physical medicine—occupational therapy, physical therapy and speech/cognitive therapy. However, these are time-consuming and costly, and have a very modest benefit on functional recovery. These facts define a common, devastating, chronic disease with no existing medical therapy and establish stroke recovery as a major area of unmet need. Stem cell therapy for chronic stroke has shown exciting potential in preclinical studies, with stem cell transplantation in the weeks to months after stroke significantly improving functional recovery in rodents. While various routes of stem cell administration have been used, intraparenchymal (directly into the brain) delivery has been associated with the greatest efficacy. This prompted us to design this study.
What is innovative about this work? And what are its applications?
This was the first clinical trial for stroke patients in North America using intracerebral transplantation of stem cells. These patients were suffering chronic motor disability, 6 months to 3 years following their stroke. If a current randomized controlled, double blinded, multicenter Phase 2b study of 156 chronic stroke patients confirms the efficacy of the same approach, a definitive larger randomized, blinded, controlled Phase 3 study will be conducted. If this is also positive, it would potentially lead to FDA approval and commercialization of the stem cell therapy. In this case, it could substantially improve the neurologic condition and lives of millions of stroke patients. In the future, it might also be applied to patients suffering from other neurologic injury such as traumatic brain injury and spinal cord injury, and perhaps even to patients with disabling degenerative neurologic diseases like Parkinson’s disease, ALS (Lou Gehrig’s disease) or even Alzheimer’s disease.
Tell us about the biggest challenge you came across while conducting this study.
The greatest challenge was obtaining the detailed clinical and radiologic follow-up for the patients in the study.
Is there anything more you would like to add about your work?
While stem cell therapy holds great promise, there is also a lot of hype surrounding this treatment. We don’t want to oversell the results of this small Phase 1/2a study. However, we are excited about the clinical benefits observed and look forward to confirming them in the current larger multicenter randomized controlled, double blinded study.
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