Case Suggests Soliris as Potential Treatment for Progressive MS
SPMS patient treated with Soliris for related disorder has no relapses for 5 months
Note: This story was updated July 20, 2022, to correct the headline to reflect that Soliris might be a potential treatment for progressive MS based on data from a single case report.
The patient was treated for a suspected diagnosis of neuromyelitis optica spectrum disorder (NMOSD), an autoimmune disease that, like multiple sclerosis (MS), also causes demyelination (myelin loss) in the brain and spinal cord. However, her symptoms continued to worsen, ultimately leading to a diagnosis of SPMS.
While the medication did not stop her symptoms from getting gradually worse, her “ultimate diagnosis of secondary progressive MS offers the opportunity to make preliminary observations of the role of eculizumab therapy in progressive MS,” the investigators wrote.
The report, “Eculizumab therapy in a patient with secondary progressive multiple sclerosis,” was published in Neuroimmunology Reports. Of note, one of the researchers receives grants and consulting fees from Alexion, which developed Soliris.
Soliris is an antibody that works by blocking C5, a protein of the complement system that is involved in the inflammatory reactions driving nerve damage in NMOSD. The complement system is a set of blood proteins that are part of the immune system’s first line of defense.
The medication is approved to treat NMOSD patients who test positive for antibodies against the aquaporin-4 (AQP4) water channel protein. It also is indicated for other diseases caused by an excessive complement system activity.
The complement system has also been implicated in MS development and progression, suggesting that “drugs targeting the terminal complement proteins may therefore become potential effective therapies for MS,” the researchers wrote.
In the report, they documented the case of a 41-year-old female patient who received Soliris for a suspected NMOSD diagnosis, who turned out to have SPMS instead.
The patient was initially diagnosed with MS around age 19, after an MRI scan of the brain revealed white matter lesions consistent with the disease. Her first neurological symptoms were unsteady walking, numbness in her toes, and optic neuritis (inflammation in the optic nerves, which carries messages from the eyes to the brain).
Over the years, the patient tried a number of MS treatments, but her symptoms continued to worsen gradually. Eventually, she lost bladder control and became bound to a wheelchair. She experienced muscle weakness and spasms, and had two more episodes of optic neuritis.
The patient also experienced progressive symptoms in her upper limbs and, by the time she first went to neuroimmunology clinic, she had no sensation in her feet. Based on the pattern of damage to the spinal cord and the optic nerves, which are the areas most commonly affected in neuromyelitis optica, the doctors considered a diagnosis of NMOSD.
Testing for anti-AQP4 antibodies in the blood came back negative, as did testing for genetic diseases and others. “Given the patient’s progressive disease course despite optimal therapy [for MS], eculizumab was proposed as an off-label treatment,” the scientists wrote.
For five months, the woman tolerated Soliris well and did not have any more relapses. However, her muscle weakness and spasms continued to gradually worsen.
A new MRI scan, taken four months into treatment with Soliris, revealed signs consistent with MS in the brain and spinal cord. Based on these findings and a lack of clinical improvement despite treatment with Soliris, the doctors made a diagnosis of secondary progressive MS.
“While our patient had no clinical improvement on eculizumab, this case raises interesting questions about the role of complement overactivation in MS,” the researchers wrote. “Future large retrospective studies and randomized trials are needed to further address the safety and efficacy of eculizumab in MS.”
The patient then began treatment with Ocrevus (ocrelizumab), but her symptoms continued to build up over time.
“She may simply have a more disabling disease course that lies higher on the spectrum of progressive MS,” the researchers wrote.