Results from a preliminary study indicate that among patients with relapsing-remitting multiple sclerosis (MS), treatment with nonmyeloablative hematopoietic stem cell transplantation (low intensity stem cell transplantation) was associated with improvement in measures of disability and quality of life, according to a study in JAMA.
Fifty percent of patients with MS are unable to continue employment by 10 years from diagnosis or are unable to walk by 25 years. Despite an annual cost of approximately $47,000 per patient to treat MS, no therapy approved by the U.S. Food and Drug Administration has been shown to significantly reverse neurological disability or improve quality of life, according to background information in the article.
Multiple sclerosis is thought to be an immunemediated disorder of the central nervous system. Autologous (the use of one’s own cells) hematopoietic (blood) stem cell transplantation (HSCT) is a form of immune suppression but unlike standard immune-based drugs, autologous HSCT is designed to reset rather than suppress the immune system.
A team of researchers from Northwestern University studied the association of nonmyeloablative HSCT with neurological disability and other clinical outcomes in patients with relapsing-remitting MS (defined as acute relapses followed by partial or complete recovery and stable clinical manifestations between relapses; n = 123) or secondary-progressive MS (defined as a gradual progression of disability with or without superimposed relapses; n = 28) treated between 2003 and 2014.
Outcome analysis was available for 145 patients with an average follow-up of 2.5 years. On a measure of the Expanded Disability Status Scale (EDSS), there was significant improvement in 41 patients (50 percent of patients tested at 2 years) and in 23 patients (64 percent of patients tested at 4 years). The team state that this is the first report of significant and sustained improvement in the EDSS score following any treatment for MS.
Receipt of HSCT was also associated with improvement in physical function, cognitive function and quality of life. There was also a reduction on another measure of clinical disease severity, volume of brain lesions associated with MS seen on magnetic resonance imaging (MRI). Four-year relapse-free survival was 80 percent and progression-free survival was 87 percent.
Patient selection is important in determining outcome, the researchers state. In the post hoc analysis, the EDSS score did not improve in patients with secondary-progressive MS or in those with disease duration longer than 10 years.
The team summise that the results are limited because this was an observational study without a control group, adding that definitive conclusions will require a randomized trial. However, this analysis provides the rationale, appropriate patient selection, and therapeutic approach for a randomized study.
Source: The JAMA Network
Michelle Petersen is the founder of Healthinnovations, having worked in the health and science industry for over 21 years, which includes tenure within the NHS and Oxford University. Healthinnovations is a publication that has reported on, influenced, and researched current and future innovations in health for the past decade.
Michelle has been picked up as an expert writer for Informa publisher’s Clinical Trials community, as well as being listed as a blog source by the world’s leading medical journals, including the acclaimed Nature-Springer journal series.
Healthinnovations is currently indexed by the trusted Altmetric and PlumX metrics systems, respectively, as a blog source for published research globally. Healthinnovations is also featured in the world-renowned BioPortfolio, BioPortfolio.com, the life science, pharmaceutical and healthcare portal.
Most recently the Texas A&M University covered The Top 10 Healthinnovations series on their site with distinguished Professor Stephen Maren calling the inclusion of himself and his team on the list a reflection of “the hard work and dedication of my students and trainees”.
Michelle Petersen’s copy was used in the highly successful marketing campaign for the mega-hit film ‘Jumanji: The Next Level, starring Jack Black, Karen Gilian, Kevin Hart and Dwayne ‘The Rock’ Johnson. Michelle Petersen’s copywriting was part of the film’s coverage by the Republic TV network. Republic TV is the most-watched English language TV channel in India since its inception in 2017.
An avid campaigner in the fight against child sex abuse and trafficking, Michelle is a passionate humanist striving for a better quality of life for all humans by helping to provide traction for new technologies and techniques within healthcare.