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July 16, 2019 News by Joana Carvalho, PhD

3-D Brain Models of PPMS and Parkinson’s Off to Space Station for Research in Microgravity

The National Stem Cell Foundation announced the start of a pioneering project to investigate the impact of microgravity on the neurodegeneration associated with primary progressive multiple sclerosis and Parkinson's disease. The project, a collaboration between the New York Stem Cell Foundation Research Institute, the Summit for Stem Cell, and investigators with Aspen Neuroscience, will send 3-D brain organoids derived from patients with these disorders, for a first time, to the International Space Station on SpaceX CRS-18. This flight, set to launch on July 21 from the Kennedy Space Center in Cape Canaveral, Florida, is a test run for a 30-day study of neurodegeneration in microgravity set to take place on the space station this fall. "The National Stem Cell Foundation is delighted to be funding innovative science at the frontier of new drug and cell therapy discovery. The leading-edge research findings that have developed through this collaboration between important research groups may fundamentally alter our understanding of how and why neurodegeneration occurs," Paula Grisanti, chief executive officer of the NSCF, said in a press release. These organoids, or 3-D cellular brain models, contain microglia — cells that normally support and protect neurons. Microglia are implicated in the brain inflammation and disease progression seen in people with Parkinson's, PPMS, and other neurodegenerative disorders. The project will allow scientists in the near absence of gravity to study how these cells interact with each other, migrate, send and receive chemical signals, change their genetic signature, and promote brain inflammation. As such, investigators may get a glimpse of all the biological mechanisms involved in PPMS and Parkinson's in ways not feasible on Earth. This work might lead to understandings and advancements with a direct impact on the development of medicines and cell therapies for neurodegenerative diseases. Space Tango is leading the transport and maintenance logistics, to ensure the cells arrive at the ISS in the best possible condition and remain viable during the 30 days they will remain in orbit. To that end, the company has developed a series of automated systems intended to surpass conventional lab techniques, and allow space station researchers to work with a higher number of samples than typical, and use high-throughput techniques to easily analyze them. According to the company, these automated systems are not intended exclusively for research on the ISS, but may also be used by research facilities worldwide to support and accelerate scientific innovation. "The vision the National Stem Cell Foundation brings to using new approaches to science and creating collaborations between leading experts in Parkinson’s disease and multiple sclerosis from across the country is truly unique," said Jana Stoudemire, commercial innovation officer at Space Tango. "In addition to supporting the development of tissue chip platforms for microgravity, Space Tango is excited to expand capabilities for human 3-D brain organoid models that will assist in studying some of the most challenging diseases we have yet to truly understand," Stoudemire added. "We are very pleased to support this important research on the ISS."

July 11, 2019 News by Ana Pena PhD

UK Researchers Pursuing Breath Test to Diagnose MS

Scientists at the University of Huddersfield, U.K., are trying to develop a breath test to detect volatile molecules exhaled only by people with multiple sclerosis (MS). Doing so might offer a non-invasive and simple way to diagnose the disease. The research team now plans to undertake…

July 3, 2019 News by Ana Pena PhD

NervGen to Advance NVG-291 as Potential Remyelination Therapy for MS

NervGen wants to advance the development of NVG-291, its lead investigational therapy for spinal cord damage, as a potential remyelination treatment for multiple sclerosis (MS), the company announced. Following preclinical data showing that NVG-291 promotes myelin repair and regeneration of damaged nerves in animal models of…

July 2, 2019 News by Jose Marques Lopes, PhD

Protein Linked to Microglia Cell Activation and Nerve Fiber Damage in Study That Also Notes Potential Treatment

A protein leads to nerve fiber and myelin damage, particularly in progressive forms of multiple sclerosis, by activating brain immune cells called microglia, according to a new study. Its researchers also noted this protein is the target of experimental MS treatment called temelimab (GNbAC1), which showed potential in Phase 2 clinical trials. The…

June 28, 2019 News by Patricia Inacio, PhD

Less-frequent Dosing with Arbaclofen ER Tablets Decreases Spasticity As Well As Standard Baclofen, MS Trial Data Show

Arbaclofen extended-release (ER) tablets taken twice a day can effectively reduce spasticity (muscle stiffness) in patients with multiple sclerosis (MS) with similar potency to that of standard and more-frequently-dosed baclofen (brand name Lioresal), Phase 3 clinical trials show. Latest trial data were presented in two posters during the 33rd Annual Meeting…

June 27, 2019 News by Vijaya Iyer, PhD

MS Patients with Low Physical Disability Incur High Economic Burden, Study Shows

Even at a low level of disability, people with multiple sclerosis (MS) have substantial indirect and informal caregiving costs due to disease progression — with unemployment markedly adding to the economic burden, a Spanish study reports. The study, “Economic burden of multiple sclerosis in a population with low physical disability,” was published in the journal BMC Public Health. MS has significant impact on health-related quality of life, with disability and fatigue — two hallmarks of the disease — hindering patients' ability to work or study. This often results in early unemployment. As MS progresses, many individuals often need the support of caregivers to perform daily life activities. Limited information is available on MS-associated economic burden imposed on patients by the costs of informal care and loss of employment. To fill this gap, Spanish researchers evaluated the cost burden of indirect and informal care for 199 people with MS. Participants (mean age 43.9 years, and 60% females) were followed at 19 MS clinical units across Spain. Among the participants, 172 (86.4%) had relapsing-remitting MS, and 27 (13.6%) had primary progressive MS. The Expanded Disability Status Scale (EDDS) was used to determine each individual’s level of disability. EDDS scoring is done by a trained neurologist, who quantifies a patient’s disability on a scale of zero to 10, in 0.5 increments.  EDDS also is used to monitor changes in disability levels through the course of MS. The greater the EDDS score, the higher the disability level. The team used the 23-item MS Work difficulties questionnaire (MSWDQ-23) to assess the level of hardships participants experienced at their workplace. A patient-reported survey, the questionnaire highlights the extent of psychological and cognitive, physical, and other external difficulties experienced at work by people with the disease. MSWDQ-23 scores range from zero to 100, with a higher score correlating to more significant workplace difficulties. The study population had a median EDDS score of 2, and a median MSWDQ-23 score of 31.5, indicating an overall low level of physical disability. Despite this, MS was found to have a marked impact on the individuals’ work and academic activity. At the time of MS diagnosis, 70.6% of the participants were employed. However, at the start of this study, 9.6 years later, the employment rate among the participants had dropped to 47.2%. The retirement rate increased from zero at the time of diagnosis to 23.6% at the time of study visit. Most participants retired at a mean age of 43.6 years — and 95.7% of them cited MS progression as the reason for retirement. Among the student population, 90.9% reported absenteeism in the year before the study visit. During the same time period, 30.9% of employed participants were absent from work. Overall, 10.1% of all participants took sick leaves during the prior year. Being absent from work, and early retirement, impose several indirect costs in this study population, the researchers found. Sick leave due to MS resulted in a mean annual cost of €416.6 (US$473.59), while work absenteeism accounted for €763.4 (US$867.83) yearly. Early retirement due to MS added an additional mean annual cost burden of €5,810.1 (US$6,604.92). The annual costs per patient due to premature work disability or pension increased to €1,816.8 (US$2,065.34). Caregivers accompanied 72% of the patients for the study visit. The researchers noted that participants' spouses reportedly spent a minimum of more than 200 hours annually in providing care — more than a full-time job. A total 28.1% of participants required paid professional support for their daily activities, such as person to do housework and a physiotherapist. Patients also reported the use of assistive devices and adaptations. Crutch or walking stick use was reported by 10.6% of participants, while 8% had some home adaptation to help in their daily activities. Use of non-reimbursable devices was reported by 21.6%. The mean annual cost of informal caregiving, including activities by professional staff, was €1,328.7 (US$1,510.46). Use of assistive devices added an additional €736.6 (US$ 837.37) yearly. “MS is responsible for a substantial economic burden due to indirect and informal care costs, even in a population with low physical disability,” the researchers said. “Effective therapeutic interventions to improve the management of early symptoms as well as implementing workplace strategies focused on job retention may be essential to decrease the high economic burden of MS,” they concluded.

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