Alexion to Begin Soliris Trial for Pediatric Patients with NMOSD

Alexion Pharmaceuticals is conducting a Phase 2/3 study to evaluate the safety and efficacy of eculizumab (Soliris) specifically in pediatric participants with relapsing neuromyelitis optica spectrum disorder (NMOSD).

This study is an interventional, open-label, single-arm trial for pediatric individuals aged 2 to 17 years with anti-aquaporin-4 antibody-positive NMOSD. It is open to participants in Japan, Korea, Spain, and the United States. All locations are currently recruiting except Korea at the time of this writing.

Dosing of eculizumab will be based on the participant’s body weight, and as such, as weight may change, so may the participant’s weight cohort and dosing. Beginning with a weight-based weekly dose of eculizumab during an induction phase, participants will receive intravenous infusion every two weeks during the Primary Treatment Period for a total of 52/53 weeks. After completing the Primary Treatment Period, participants may continue to receive eculizumab in the Extension Treatment Period for 104 weeks.

Notable inclusion criteria in addition to the anti-AQP4 antibody positivity and a confirmed NMOSD diagnosis, participants must be age 2 to 17 years (must be less than 18 years) and with a bodyweight greater than or equal to 10 kilograms (or 22.0462 pounds). Participants should have experienced at least two relapses in the past two years, with at least 1 of the relapses occurring in the past year before screening. Those who participate in the study and also receive supportive immunosuppressive therapies to prevent relapse, whether in combination or as a single therapy, must be on a stable dosing regimen for a long enough amount of time.

Exclusion criteria include those with unresolved or chronic infections that have not been appropriately treated with antibiotics, or prior to screening have used rituximab or other biologicals within six months, mitoxantrone within three months, IVIG or plasma exchange (PLEX) within three weeks, or immunomodulatory therapies for multiple sclerosis (MS) within three months.

Those interested in this study may find the full inclusion/exclusion criteria here and here and should contact Alexion Pharmaceuticals by phone, +1 855-752-2356, or by email at

Ignition Survey for MOGAD Results

In Spring 2020, The MOG Project partnered with the Siegel Rare Neuroimmune Association and The Sumaira Foundation for NMO to understand treatment practices among those with MOG antibody disease (MOGAD). The intention of the survey was to learn from the MOGAD community experience and share results with physicians and each other.

267 individuals with MOGAD and their caregivers completed a survey asking a range of questions regarding acute and preventive treatments and their associated side effects. 89% of respondents had been prescribed an acute treatment during their most recent attack/relapse, the most commonly prescribed being IV Steroids (86%) followed by Oral Steroids (50%). Nearly 70% of those diagnosed with MOGAD are currently on a preventive treatment. Rituximab (45%), Mycophenolate Mofetil (25%) and IVIG (25%) were cited as commonly prescribed preventive treatments. Side effects were common, with 66% of respondents reporting side effects from their acute treatments. 49% of those on Rituximab, 32% on Mycophenolate Mofetil, and 53% on IVIG reported side effects from their treatments.

It is important to note that this respondent pool may skew towards patients with more severe cases of MOGAD, as these people may have been more likely to respond to the survey. You can view the detailed survey results here. Special thanks to the members of the MOGAD community and their caregivers for participating in this survey. It is our hope that these survey results help those affected by MOGAD know they are not alone; ours is a shared experience as a community.

Update on UT Southwestern Q Cell Stem Cell Trial in Transverse Myelitis

During these unprecedented times of COVID-19 we have fielded many questions about the impact of the pandemic on our clinics, health care systems and patients. I hope everyone has been able to stay safe during these days of isolation and social distancing. While I am confident that a solution to the COVID-19 pandemic will be found, the impact of these events will be felt for years.

Beyond the personal health impacts, economic impacts and changes to our medical system, the pandemic has dramatically impacted basic science and clinical research efforts across the world. The UT Southwestern Q Cell Stem Cell trial is included. Since the mandates to enact social distancing were put into effect, basic science labs and clinical trials were paused. Thus, we have been unable to proceed with our first surgery as planned. Based on the number of questions received, we wanted to provide this update to our community.

We have received FDA clearance to proceed with this phase one trial in paralyzed TM patients. The trial currently plans to enroll nine patients for the surgical implantation of Q Cells. This is a safety study with many regulatory requirements, including a requirement focused on training the surgical team to perform the procedure with novel technology. As we worked during the year to get complete licensing and credentialing requirements for the team, the COVID-19 pandemic began, halting plans for first enrollment and surgery.

Nevertheless, we are preparing to reopen for research at UT Southwestern. As such, we continue to work to identify the nine candidates for this clinical trial. Those interested can find information on the SRNA website. We appreciate your support during these difficult times and remain excited about what lies ahead.

Benjamin M. Greenberg, MD, MHS, FANA, FAAN
Director, Perot Foundation Neurosciences Translational Research Center
O’Donnell Brain Institute
University of Texas Southwestern
Board of Directors, Siegel Rare Neuroimmune Association (Formerly the Transverse Myelitis Association)

Dr. Greenberg has received funding from the SRNA and serves as an unpaid member of the board of the SRNA.

Case Report: Transverse Myelitis following COVID-19

A case report was recently published of a 60-year-old man who received a diagnosis of transverse myelitis after infection with SARS-CoV2, which is the coronavirus that has been spreading throughout the world. He was admitted to the hospital with respiratory symptoms and was given a throat swab test that showed he was positive for the virus. He recovered from COVID-19 pneumonia and was sent home after five days. Three days later, he was unable to urinate and had weakness in his legs. The man was unable to walk unassisted and had other signs of spinal cord dysfunction. An MRI of his spine showed a lesion at T9 consistent with myelitis. He had no lesions in the brain. He had a spinal tap which showed that his cerebrospinal fluid had an increased cell count and protein levels, also consistent with myelitis. The coronavirus (SARS-CoV2) was not found in his cerebrospinal fluid, and all other testing for other bacteria and viruses was negative.

He was treated with an intravenous antiviral medication and an antibiotic, but this treatment was stopped after 8 days when no infectious agents were found in his cerebrospinal fluid. He improved slightly after three days. On the 7th day, he was given a lower dose of steroids than is typically given in transverse myelitis because of the known prior infection with the virus. He then improved rapidly. He was discharged after 13 days with some sensation impairments, but he had normal bladder function and was able to walk independently. The authors of the report believe this was not the case of a direct infection of SARS-CoV-2 into the spinal cord, but an overreaction of the immune system to the virus (i.e., post-infectious myelitis).

We will continue to monitor cases of neuroimmune issues after COVID-19 and will share with our community as we hear about them. For additional information, please visit our COVID-19 page.

Munz M, Wessendorf S, Koretsis G, et al. Acute transverse myelitis after COVID-19 pneumonia. J Neurol. 2020 May 26;1-2.

Survey about Disability Identity Development

Over the past five years, Anjali Forber-Pratt, PhD, has been working to develop and validate a measure of disability identity development. While the study has made a lot of progress, it is in need of another sample of approximately 1,000 individuals with disabilities to complete the reduced set of items. This is a one-time email as we would like to invite you to participate in the study by taking the following survey, which contains a significantly reduced set of questions:

The survey will take no more than 20 minutes to complete and contains basic questions about yourself, your disability, your interactions with others, your involvement with the disability community and perceptions about your identity. If you choose to participate, you may enter your contact information (or you may opt out by not entering it) to enter a drawing for a $100 Amazon gift card. Three gift cards will be drawn. Your participation in this study is voluntary. In order to be eligible to participate in this study, you must be:

  • 18 years of age or older
  • Identify as having a disability (visible or hidden)
  • Able to reliably complete self-report measures in English
  • Living or residing in the United States

Thank you for your consideration! Feel free to share with others who may be eligible to participate. Email with any questions.

Anjali J. Forber-Pratt, Ph.D. is an Assistant Professor at the Department of Human & Organizational Development at Vanderbilt University. Her research agenda adopts a social-ecological framework and looks at issues surrounding identity, equity and empowerment through methodology for individuals who are different in some way, with a large focus on disability. She completed her degrees at the University of Illinois. Dr. Forber-Pratt is a Paralympic medalist and currently serves as a member of the Board of Directors of SRNA.

AFM Diagnoses of 2018 – An Urgent & Important Request

Centers for Disease Control and Prevention (CDC) is conducting a case series activity by attempting to interview all confirmed 2018 Acute Flaccid Myelitis (AFM) patients and their families with a standardized questionnaire. The detailed questionnaire includes questions about exposures, illness just prior to onset, and healthcare-seeking behavior prior to limb weakness. CDC’s Institutional Review Board (IRB) package allows three months for data collection, and the last day for interviews is September 11, 2019. CDC is working through state and local health departments to conduct the interviews, and as of the third week of August, they have completed questionnaires for approximately 1/3 of the 233 cases in 2018. CDC and state and local health departments are making a limited number of phone calls and sending a text message to families.

If you or your child are one of those impacted by AFM in 2018, we encourage you to respond quickly to the call you have received or will receive from CDC or your local health department. If you’ve made an appointment for an interview to be conducted, please be available at the time you’ve scheduled. If you are a provider or a friend of a family impacted in 2018, please check with the family to see if they have reached back out in response to the call or text they may have received. The interviews are critically important in understanding the etiology and impacts of AFM. The findings will assist in assessing risk factors, establishing prevention measures, and developing treatments. Our hope is that every one of the 233 families will respond and have their stories heard by CDC. Your time, your story, and your family matters. Your contribution in sharing your story with CDC will assist all of us as we move forward and focus on what must be done in the future for all of those diagnosed with AFM. As an organization dedicated to awareness and research of this devastating disorder, we sincerely appreciate your participation and spreading the word about this important activity. If you have any questions, please feel free to reach out to us at

SRNA-Supported Research at the 2019 American Academy of Neurology Annual Meeting

SRNA maintains our commitment to advancing the scientific understanding of and therapy development for AFM, ADEM, MOG Antibody Disease, NMOSD, ON, and TM. We are proud to announce that once again SRNA-supported research was presented at the American Academy of Neurology Annual Meeting, which was held from May 4th-10th in Philadelphia, PA.

Dr. Olwen Murphy, a current James T. Lubin Fellow, and a team at The Johns Hopkins Hospital presented research on sarcoidosis-related myelitis. The research was a retrospective study of patients diagnosed with sarcoidosis-related myelitis at the Johns Hopkins Transverse Myelitis Center. The goal of the study was to identify characteristic clinical, imaging and CSF features of sarcoidosis-related myelitis. They found that distinct imaging patterns occur in sarcoidosis-related myelitis and recognition of these features may aid in coming to a correct diagnosis. Most of the patients in the study had a long symptom evolution (81%), meaning it took more than three weeks from symptom onset to when symptoms were at their worst. Most had sensory symptoms (87%) and motor symptoms (53%). Enhancement patterns suggest that the blood-spinal-cord barrier may play a role in the development of sarcoidosis-related myelitis lesions. Dr. Murphy and colleagues also presented a case report on a 68-year-old man who presented with a 2-year history of severe muscle spasms in the lower back and pelvic region. The patient was ultimately referred to their clinic for evaluation of “treatment-resistant” stiff person syndrome after baclofen and benzodiazepines had no clinical effect. He was correctly diagnosed with an anterior disco-osteo-arterial conflict and underwent surgery to correct the problem. Six months post-surgery he reported marked improvement in his symptoms and increased exercise tolerance.

Dr. Jonathan Galli, another James T. Lubin Fellow, worked with Dr. Clardy on another research study about stiff person syndrome. The goal was to describe epidemiological characteristics, antibody status, and treatment outcomes of stiff person syndrome patients within University of Utah Health. Stiff person syndrome (SPS) is an autoimmune disease that classically causes severe muscle rigidity and spasms. They identified 31 patients with stiff person syndrome. Patients were predominantly female (78%). Some of their cohort had co-existing autoimmune diseases (63%) and malignancy (13%). Diazepam and baclofen was effective in a majority of patients. IVIg was the most commonly utilized immunotherapy (used in 69% of patients) with benefit demonstrated in 41% of patients who received this treatment. Dr. Galli and colleagues also presented a case series with the goal to describe atypical epilepsy presentations in patients with common variable immunodeficiency (CVID) within the University of Utah Healthcare system. Patients with CVID are at increased risk of infection, malignancy, and autoimmune disease. They presented a case series of 5 patients with CVID and co-existing epilepsy. All patients had atypical seizure symptoms including behavioral arrest, alterations in consciousness, and/or amnestic episodes. Most of the patients had improvement with antiepileptic therapy.

Dr. Cynthia Wang, a former James T. Lubin Fellow, and colleagues presented a case report of a 6-year-old boy with new-onset seizures and altered mental status associated with multifocal right hemispheric lesions resulting from primary CNS vasculitis. Primary CNS vasculitis is a rare vascular inflammatory brain disease. The child was treated with cyclophosphamide with gradual improvement in cerebral edema, and he also underwent cranioplasty four weeks after hemicraniectomy. He improved significantly over one month in inpatient rehabilitation and he regained the ability to ambulate independently. The researchers note that this case demonstrates an unusual presentation of this condition in a child. They also note that hemicraniectomy should be considered in patients with medically refractory increased intracranial pressure.

Dr. Benjamin Greenberg and colleagues also presented about the upcoming study to investigate the safety of the transplantation of human glial restricted progenitor cells into subjects with transverse myelitis. Funding and support for this trial has been provided via the UT Southwestern CONQUER Program, the Transverse Myelitis Association and Q Therapeutics. The primary objective of the study is to evaluate the safety of oligoprogenitor Q-Cells® transplantated into the posterior columns of the spinal cord in patients with Transverse Myelitis. A secondary objective of the study is to obtain preliminary data regarding the clinical activity of Q-Cells® in patients with TM. For more information about this study, visit the study page here.

For more information on other research and clinical publications supported through the generosity of SRNA community, please visit

Now Enrolling for Phase I Remyelination Trial using Q-Cells in Transverse Myelitis

A Phase I remyelination trial using Q-Cells in Transverse Myelitis conducted by the University of Texas Southwestern Medical Center (UTSW), Q Therapeutics, and The Siegel Rare Neuroimmune Association has just opened enrollment! The trial is designed to study the safety and efficacy of implanting cells that produce myelin into the spinal cord. It is the first study of its kind in transverse myelitis. You can read more about the study in UTSW’s press release.

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. 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. If you’d like to learn more about the study, please visit the FAQ page.

If you are a person diagnosed with transverse myelitis, are between one and ten years from your event, and you remain unable to walk, you may be eligible to participate. To sign up to participate in this trial, please fill out UTSW’s survey here. Please note that the study will enroll nine non-ambulatory adult transverse myelitis patients, so filling out the survey does not guarantee enrollment in the study. You can find more information on the study, including inclusion and exclusion criteria for participation, on on the study page here.

If you have any questions about enrollment in the study, please email us at

CAPTURE: An update to the long-term study of pediatric transverse myelitis and acute flaccid myelitis

By Rebecca Whitney, Pediatric Programs Manager at SRNA

In 2014, the Transverse Myelitis Association (SRNA) and seven clinics across North America, under the direction of Dr. Benjamin Greenberg at UT Southwestern Medical Center, began a research journey to follow pediatric transverse myelitis diagnoses. I honestly didn’t know what to expect from this new venture, having previously only been on the participation end of a research study, but knew in my heart of hearts that this study, CAPTURE, was incredibly valuable and would make an impact in the history of our children and families. I was ready to be a part of it, although it was difficult, often heartbreaking work. But, that’s also why I knew I needed to be a part of it. It is a matter of my heart and means something so incredibly personal to me as a parent of a young child diagnosed with transverse myelitis.

CAPTURE is the first of its kind – a prospective, observational study in pediatric transverse myelitis (TM) and acute flaccid myelitis (AFM). Designed to not only utilize the imaging and clinical diagnosis details from physicians, CAPTURE also collects information from the children and families themselves about their outcomes, relative to the treatments they received. Initially, study criteria required enrollment within three months of diagnosis. After listening to feedback from SRNA and parent community, Dr. Greenberg and his team changed the study’s enrollment to include patients who are within six months of diagnosis. Even for the most well-intentioned families vowing to help by participating in a research study, those first three months post-diagnosis are oftentimes so bewildering and surreal. We found we experienced a better enrollment rate and continued participation if we allowed families time to adjust to this new realm of medicine they suddenly found themselves in.

2014 also brought to light a “new variant” of TM that was popping up across the country – Acute Flaccid Myelitis or AFM. Due to the outbreaks of AFM, we found our correspondence with families and enrollment were increasingly with those experiencing AFM rather than TM. Regardless, the study remained open for both diagnoses, and we have continued to learn so much, often debated amongst clinicians and governmental entities, about the acute treatments that might provide the best outcomes for our children. CAPTURE has been and continues to be a critical part of the conversation and debate regarding acute treatments as it is an already established mechanism for collecting essential data and follows children’s progress for up to a year after their diagnosis. To date, we have enrolled 140 children into CAPTURE.

CAPTURE, through the work of SRNA and Dr. Greenberg’s team in Dallas, has been able to move forward and is still continuing to enroll new participants with TM and AFM within six months of diagnosis through our online cohort. This is incredible considering that when we began, the study was scheduled to end recruitment in 2018. The more information we can obtain, the clearer the way we move forward becomes – how we develop treatments and therapies, engage potential stakeholders, including government officials and entities, and ultimately, provide the best outcomes for children, or better yet, stop TM and AFM from even being a threat to our children. Even more incredible is the fact that recent IRB changes to CAPTURE will allow us to continue to hear from those children and families previously enrolled, should they so choose, through 2026!

Let’s think about this for a moment… By following current and future enrollees in CAPTURE through 2026, we will have 10 years – a decade – of real, patient-reported data. For some, this means when they report their recovery and outcomes, we will follow them into adulthood. We all know how quickly children grow and quite frankly, a diagnosis of a rare neuroimmune disorder can be starkly different for a child than an adult, including their recovery. What an excellent opportunity to impact change and the future of medicine related to these disorders!  As it stands right now, we can’t refer back to medical literature or history books and find what is to be done for a child amid an AFM outbreak. The potential for learning from our heart-wrenching experiences is astounding. As I sit here and watch my own child recover from major surgery as a result of his TM diagnosis, now almost 12 years out, how he’s doing today looks so different than what we thought or imagined 5 or 10 years ago, or even six months post-diagnosis. Since we are forced to face this diagnosis and the ramifications of what it means for his well-being, his growth, his mobility, his quality of life, then we are going to learn and grow from it, and hopefully, we can help other children and families as we do.

CAPTURE must and will continue for our children. We will continue to enroll those newly diagnosed within six months and into the long-term study. Those who were previously enrolled in CAPTURE will be contacted by Tricia Plumb of UTSW to see if they wish to extend their participation. I hope that each one of the 140 participants to date will consider the impact they may have on our community and future children by continuing to share their stories via the brief surveys. The aim is for each child/family to complete a survey and have a short, secure video chat with the study leader(s) every 4 months. If you or your child has been recently diagnosed or you’ve participated in CAPTURE and wish to extend your participation, please contact Tricia Plumb or me.

Oftentimes over the last few years, we’ve been contacted by families further out from six months, even years post-diagnosis, and have heard, “what about my child? What about her experience and her recovery?” If you or your child were diagnosed with TM or AFM before six months ago, your story, your recovery, your outcomes matter, too! A new study called CORE TM is available and currently enrolling participants, and it is similar to CAPTURE. For more information about CORE TM or to enroll, please do so via the SRNA Registry. You may also contact Tricia Plumb or me for more information.

For families of children diagnosed with Acute Disseminated Encephalomyelitis (ADEM) or whose diagnosis may have changed from TM to ADEM and are no longer eligible for CAPTURE, there is the possibility for study participation for you, too. APERTURE is currently enrolling and is also an observational study being conducted online. For more information on APERTURE, please contact me, Tricia Plumb, or Dr. Cynthia Wang.

Further to the opportunities noted above, the SRNA Registry is always available, and everyone with a rare neuroimmune disorder is invited to participate in the registry.

Vaccines and the association with relapses in patients with neuromyelitis optica spectrum disorder

A retrospective study was conducted to determine whether vaccinations were associated with an increased risk of relapse in patients with Neuromyelitis Optica Spectrum Disorder (NMOSD). Patient records were reviewed from three NMOSD centers: the Johns Hopkins NMO Clinic in Baltimore, USA, NeuroCure research Center at Charité University Hospital in Berlin, Germany, and Neuroclinica in Medellín, Colombia. All patients with comprehensive health records related to their NMOSD and who had follow-up information for at least 90 days after their most recent vaccination were included in this analysis.

In order to determine whether there was an increased risk of relapse following a vaccination, the researchers compared relapses that occurred 30, 60, and 90 days after vaccination with relapses that occurred within randomly selected dates. For the purposes of this study, relapses were defined as “a new or worsening acute neurologic symptom lasting 24 hours, associated with a change in exam localizing to the [central nervous system] CNS and not explainable by fever, infection or metabolic condition.” The NMOSD patients were divided into two groups: those who were taking preventative immunotherapy medication such as rituximab, mycophenolate mofetil, azathioprine, methotrexate, or prednisone, and those who were not on a preventative immunotherapy, which included patients who were taking glatiramer acetate and interferon beta, as these medications have been found to worsen or not be effective in NMOSD.

Ninety patients who received a total of 211 vaccinations were included in this study. The median disease course was 6.6 years, and 340 relapses had occurred during this timeframe. Intramuscular influenza was the most common vaccine received (61% of the vaccines received).

The researchers found that vaccines were not significantly associated with relapses among patients on preventive immunotherapy such as rituximab, mycophenolate mofetil, azathioprine, methotrextate, or prednisone. However, the researchers found that vaccines were significantly associated with relapses in patients who were not on preventive immunotherapy. Also, among patients on preventive immunotherapy, routine vaccinations were associated with lower annualized relapse rates.

There were 7 patients who relapsed within 30 days of a vaccination, 6 patients who relapsed 31-60 days after a vaccination, and 3 patients who relapsed 61-90 days after a vaccination, for a total amount of 16 patients experiencing relapses within 90 days after a vaccination. Five of the inflammatory attacks were at the disease onset and eleven were relapses that occurred later in the course of the disease. The highest proportion of vaccination-associated relapses were after tetanus/diphtheria vaccines, as 15% of patients receiving this vaccination relapsed within 90 days.

118 of the 211 vaccinations in this study were administered to patients who were on immunosuppressive therapy. Most (13 of the 16) patients who experienced relapses were not on immunotherapy, and one patient was on glatiramer acetate, which is not an effective treatment for NMOSD. The remaining two patients were on immunosuppressive treatment for an average duration of 47 months.

As stated above, the researchers found that routine vaccination was associated with an 81% lower risk of relapse in patients who were using preventive immunotherapy than patients who were not vaccinated after their initial disease onset. A possible explanation for this finding is that relapses can be triggered by immune system activation, and vaccinations help prevent infections that cause immune system activation, which results in fewer relapses.

The researchers suggest that individuals with NMOSD take preventive immunotherapy treatment prior to receiving any future vaccinations.

The authors of the study note several limitations to this study. For example, they did not include patients who received live attenuated vaccines, such as Japanese encephalitis and yellow fever vaccines, which have been associated with relapses in NMOSD. Additionally, few patients had received the HPV vaccine, which has been associated with relapses in case studies of NMOSD patients. The authors also had limited information and could not include data on the adjuvants that were used for each of the vaccines. Also, there were few aquaporin-4 negative patients included in the study, so additional studies should confirm these findings with aquaporin-4 negative patients. Lastly, there are inherent biases in retrospective data analyses, which could have influenced the results of this study. The study also did not look at vaccine-preventable infections, such as the flu, and their potential association with relapses. The results of the study should be understood within the context of these limitations and biases.

To address the issues with this study, the researchers suggest that there should be a comprehensive, well-controlled prospective study that investigates relapses, vaccines, and infections.

Mealy MA, Cook LJ, Pache F et al. Vaccines and the association with relapses in patients with neuromyelitis optica spectrum disorder. Mult Scler Relat Disord. 2018 Jul;23:78-82. doi: 10.1016/j.msard.2018.05.003. Epub 2018 May 7.