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BOSTON—Interim results from a small, preliminary study support a new type of treatment for progressive multiple sclerosis (MS). Results from the first six people enrolled in the phase I study, which was designed to enroll 10 people, were presented at the 69th Annual Meeting of the American Academy of Neurology. “While these results are very preliminary, and much more research is needed, we are excited there were no serious side effects,” said study author Michael P. Pender, MD, PhD, a Professor and Director of the Multiple Sclerosis Research Group at the University of Queensland in Brisbane, Australia. 
The study investigated the relationship between MS and the Epstein-Barr virus (EBV). Previous research has suggested a role for EBV in MS pathogenesis. The study involved six people with progressive MS who had moderate to severe disability (ie, Expanded Disability Status Scale scores between 5.0 and 8.0).
In some people with MS, EBV-infected autoreactive B cells might accumulate in the CNS because of defective cytotoxic CD8+ T-cell immunity. Elimination of EBV-infected B cells may reduce the destruction of myelin in MS.
For the study, researchers removed the participants' own T cells and stimulated them to boost their ability to recognize and destroy cells infected with EBV. They then injected participants with infusions of escalating doses of T cells every two weeks for six weeks. They followed the patients for 26 weeks to look for evidence of side effects and possible improvement of symptoms.
Three participants showed symptomatic and objective clinical improvement, starting two to eight weeks after the first infusion.
“One person with secondary progressive MS showed striking improvement,” Professor Pender said. This participant had normalization of lower extremity tone and plantar responses for the first time in 16 years. “This participant had a significant increase in ambulation from 100 yards with a walker at the start of the study, and over the previous five years, to three quarters of a mile, and was now also able to walk shorter distances with only one-sided assistance. Lower leg spasms that had persisted for years resolved.”
Professor Pender said another participant with primary progressive MS had reduced fatigue, increased productivity, and improved balance. Another responder had improved color vision, visual acuity, and manual dexterity; reduced fatigue; fewer lower extremity spasms; and less urinary urgency. All three responding participants had improvements in fatigue and ability to perform daily activities.
“The best responses were seen in the two people who received T cells with the highest amount of reactivity to the EBV,” Professor Pender said. None of the six participants had serious side effects.
“Much more research needs to be done with larger numbers of participants to confirm and further evaluate these findings,” Professor Pender said. “But the results add to the mounting evidence for a role of the Epstein-Barr virus infection in MS and set the stage for further clinical trials.”
The study was a collaboration between the QIMR Berghofer Medical Research Institute, Royal Brisbane and Women's Hospital, and the University of Queensland. The study was supported by MS Queensland, MS Research Australia, QIMR Berghofer Medical Research Institute, and Perpetual Trustee Company Limited.
—Glenn S. Williams
Suggested Reading
Pender MP, Csurhes PA, Smith C, et al. Epstein-Barr virus-specific adoptive immunotherapy for progressive multiple sclerosis. Mult Scler. 2014;20(11):1541-1544.
BOSTON—Interim results from a small, preliminary study support a new type of treatment for progressive multiple sclerosis (MS). Results from the first six people enrolled in the phase I study, which was designed to enroll 10 people, were presented at the 69th Annual Meeting of the American Academy of Neurology. “While these results are very preliminary, and much more research is needed, we are excited there were no serious side effects,” said study author Michael P. Pender, MD, PhD, a Professor and Director of the Multiple Sclerosis Research Group at the University of Queensland in Brisbane, Australia.
The study investigated the relationship between MS and the Epstein-Barr virus (EBV). Previous research has suggested a role for EBV in MS pathogenesis. The study involved six people with progressive MS who had moderate to severe disability (ie, Expanded Disability Status Scale scores between 5.0 and 8.0).
In some people with MS, EBV-infected autoreactive B cells might accumulate in the CNS because of defective cytotoxic CD8+ T-cell immunity. Elimination of EBV-infected B cells may reduce the destruction of myelin in MS.
For the study, researchers removed the participants' own T cells and stimulated them to boost their ability to recognize and destroy cells infected with EBV. They then injected participants with infusions of escalating doses of T cells every two weeks for six weeks. They followed the patients for 26 weeks to look for evidence of side effects and possible improvement of symptoms.
Three participants showed symptomatic and objective clinical improvement, starting two to eight weeks after the first infusion.
“One person with secondary progressive MS showed striking improvement,” Professor Pender said. This participant had normalization of lower extremity tone and plantar responses for the first time in 16 years. “This participant had a significant increase in ambulation from 100 yards with a walker at the start of the study, and over the previous five years, to three quarters of a mile, and was now also able to walk shorter distances with only one-sided assistance. Lower leg spasms that had persisted for years resolved.”
Professor Pender said another participant with primary progressive MS had reduced fatigue, increased productivity, and improved balance. Another responder had improved color vision, visual acuity, and manual dexterity; reduced fatigue; fewer lower extremity spasms; and less urinary urgency. All three responding participants had improvements in fatigue and ability to perform daily activities.
“The best responses were seen in the two people who received T cells with the highest amount of reactivity to the EBV,” Professor Pender said. None of the six participants had serious side effects.
“Much more research needs to be done with larger numbers of participants to confirm and further evaluate these findings,” Professor Pender said. “But the results add to the mounting evidence for a role of the Epstein-Barr virus infection in MS and set the stage for further clinical trials.”
The study was a collaboration between the QIMR Berghofer Medical Research Institute, Royal Brisbane and Women's Hospital, and the University of Queensland. The study was supported by MS Queensland, MS Research Australia, QIMR Berghofer Medical Research Institute, and Perpetual Trustee Company Limited.
—Glenn S. Williams
Suggested Reading
Pender MP, Csurhes PA, Smith C, et al. Epstein-Barr virus-specific adoptive immunotherapy for progressive multiple sclerosis. Mult Scler. 2014;20(11):1541-1544.
BOSTON—Interim results from a small, preliminary study support a new type of treatment for progressive multiple sclerosis (MS). Results from the first six people enrolled in the phase I study, which was designed to enroll 10 people, were presented at the 69th Annual Meeting of the American Academy of Neurology. “While these results are very preliminary, and much more research is needed, we are excited there were no serious side effects,” said study author Michael P. Pender, MD, PhD, a Professor and Director of the Multiple Sclerosis Research Group at the University of Queensland in Brisbane, Australia.
The study investigated the relationship between MS and the Epstein-Barr virus (EBV). Previous research has suggested a role for EBV in MS pathogenesis. The study involved six people with progressive MS who had moderate to severe disability (ie, Expanded Disability Status Scale scores between 5.0 and 8.0).
In some people with MS, EBV-infected autoreactive B cells might accumulate in the CNS because of defective cytotoxic CD8+ T-cell immunity. Elimination of EBV-infected B cells may reduce the destruction of myelin in MS.
For the study, researchers removed the participants' own T cells and stimulated them to boost their ability to recognize and destroy cells infected with EBV. They then injected participants with infusions of escalating doses of T cells every two weeks for six weeks. They followed the patients for 26 weeks to look for evidence of side effects and possible improvement of symptoms.
Three participants showed symptomatic and objective clinical improvement, starting two to eight weeks after the first infusion.
“One person with secondary progressive MS showed striking improvement,” Professor Pender said. This participant had normalization of lower extremity tone and plantar responses for the first time in 16 years. “This participant had a significant increase in ambulation from 100 yards with a walker at the start of the study, and over the previous five years, to three quarters of a mile, and was now also able to walk shorter distances with only one-sided assistance. Lower leg spasms that had persisted for years resolved.”
Professor Pender said another participant with primary progressive MS had reduced fatigue, increased productivity, and improved balance. Another responder had improved color vision, visual acuity, and manual dexterity; reduced fatigue; fewer lower extremity spasms; and less urinary urgency. All three responding participants had improvements in fatigue and ability to perform daily activities.
“The best responses were seen in the two people who received T cells with the highest amount of reactivity to the EBV,” Professor Pender said. None of the six participants had serious side effects.
“Much more research needs to be done with larger numbers of participants to confirm and further evaluate these findings,” Professor Pender said. “But the results add to the mounting evidence for a role of the Epstein-Barr virus infection in MS and set the stage for further clinical trials.”
The study was a collaboration between the QIMR Berghofer Medical Research Institute, Royal Brisbane and Women's Hospital, and the University of Queensland. The study was supported by MS Queensland, MS Research Australia, QIMR Berghofer Medical Research Institute, and Perpetual Trustee Company Limited.
—Glenn S. Williams
Suggested Reading
Pender MP, Csurhes PA, Smith C, et al. Epstein-Barr virus-specific adoptive immunotherapy for progressive multiple sclerosis. Mult Scler. 2014;20(11):1541-1544.