Study testing treatment to regenerate nerve tissue in neck
By Nancy DiFiore
A paralyzing injury to the neck during recreational activities such as horseback riding or playing football usually has permanent, lifelong effects that change a person’s life dramatically. With no options for the repair of spinal cord injuries, many are left with little hope for recovery.
Now researchers at Rush are exploring a new therapy that uses stem cells to treat spinal cord injuries within the first 14 to 30 days of injury. Rush is one of only two centers in the country currently studying this new approach.
“There are currently no therapies that successfully reverse the damage seen in the more than 12,000 individuals who suffer a spinal cord injury each year in the United States alone,” says Richard G. Fessler, MD, PhD, professor of neurological surgery at Rush. An estimated 1.3 million Americans are living with a spinal cord injury.
“These injuries can be devastating, causing both emotional and physical distress, but there is now hope. This is a new era where we are now able to test whether a dose of stem cells delivered directly to the injured site can have an impact on motor or sensory function,” Fessler continues. “If we could generate even modest improvements in motor or sensory function, it would result in significant improvements in quality of life.”
Potential progenitor of paralysis repair
Fessler is the principal investigator at Rush of a study involving a set of what are called progenitor cells, which are likely to develop into a certain types of cell. Specifically, the researchers at Rush are studying nerve cells known as oligodendrocyte progenitor cells, which potentially can make poorly functioning nerves function better. A San Francisco Bay-area biotechnology company, Asterias Biotherapeutics, develops the cells and is sponsoring the study.
The clinical trial is designed to assess safety and efficacy of escalating doses of the special cells, known as AST-OPC1, to treat individuals with a cervical spinal cord injury that resulted in tetraplegia, the partial or total paralysis of arms, legs and torso. As of mid-August, one individual has been enrolled in the study at Rush.
The trial involves testing three escalating doses of AST-OPC1 in patients with subacute, neurologically complete injury to the cervical spinal cord, that is, the spinal cord in the neck (and specifically, the spinal nerves known as C5 to C7). These individuals essentially have lost all sensation and movement below their injury site and have severe paralysis of the upper and lower limbs.
For this therapy to have a chance to work, the spinal cord has to be in continuity and not severed. Patients must be able to start the treatment within 25 days of their injury.
The researchers will administer AST-OPC1 between 14 to 30 days post-injury. Following the treatment, patients will receive frequent neurological exams and imaging so that the researchers can assess the results. Patients will be followed for 15 years thereafter.
“If this treatment proves to be safe and effective, in the future, it also might be used for peripheral nerve injury or other conditions that affect the spinal cord, such as multiple sclerosis or ALS,” Fessler says.
The study seeks male and female patients ages 18 to 65 who recently experienced a cervical spinal cord injury at the neck that resulted in partial or total paralysis of arms, legs and torso. Participants must be able to provide consent and commit to a long-term follow-up study. To enroll in the trial or for more information, contact Michelle Smreczak at (312) 942-8614 or firstname.lastname@example.org.