#ECTRIMS2019 – Promises and Warnings About Stem Cell Therapy

#ECTRIMS2019 – Promises and Warnings About Stem Cell Therapy

Publication date: Sep 12, 2019

Stem cell therapy, or stem cell transplant, is an emerging yet controversial treatment approach for multiple sclerosis (MS). While some data uphold it as one of the most efficacious MS treatments, to date there have been no controlled studies comparing it to conventional medicines and providing more robust evidence regarding its safety and clinical benefit. Under the topic -HSCT and stem cell treatment in MS,” a group of researchers discussed the promise and current challenges of stem cell transplant at the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), being held Sept. 11-13 in Stockholm. To date, the only proven stem cell treatment for MS is autologous HSC transplant (AHSCT), particularly in people with highly active relapsing disease. AHSCT basically consists of a bone marrow transplant where HSCs are collected from the patient (aka autologous) and given back after the immune system is partially or completely wiped out by a conditioning treatment, usually chemotherapy. Greater expectations for AHSCT have been raised after recent studies indicated that the therapy might be more successful if done in the earlier (inflammatory) stages of MS. Despite these results, most clinicians are reluctant about the risks and the lack of data regarding AHSCT in MS patients. In addition, the majority of patients (68-70%) achieve -no evidence of disease activity” (NEDA) – no relapses, no increase in disability (as measured by EDSS), and no new or active lesions on MRI – at five years after treatment, suggesting a superior efficacy compared to conventional disease-modifying therapies (DMTs). The researcher also presented a case study of a young patient with MS, and used it to emphasize that -in a situation of clinical equipoise” between choosing a DMT or HSCT, clinicians should -subject patients to the least possible risk!” This was the focus of the oral presentation -Mesenchymal stem cell treatment in MS-, given by Antonio Uccelli, MD, professor at the University of Genova, Italy. As more robust data is needed, researchers are conducting a Phase 1/2 trial called MESEMS (mesenchymal stem cells for multiple sclerosis) in MS patients to assess the safety and efficacy of MSC treatment compared to placebo. Overall, Uccelli concluded that, although the trial did not meet its primary endpoint, it -demonstrated an important trend toward decreasing the annualized relapse rate over 24 weeks” with MSC treatment, and suggested that -analysis of secondary and exploratory outcomes will reveal whether MSC are effective on other MRI and/or clinical parameters in MS, as well as on any defined metrics suggesting repair. “

Concepts Keywords
Autoimmune Diseases Chemotherapy
Autologous MRI
Blood Neural network
Bone Marrow Transplantation
Bone Marrow Transplant Stem-cell therapy
California Stephen L. Hauser
Central Nervous System Primary immunodeficiency
Chemotherapy Multiple sclerosis
Death Rate Transplantation medicine
Disability Lymphology
DMT Hematology
Europe Branches of biology
Fat Stem cells
Fluid Medical specialties
Genova Medicine
Hematopoietic Bone marrow transplant
Immune Response Chemotherapy
Immune System Immune autoreactivity inflammation
Immunosuppressive Uncontrolled inflammation
Inflammation Conventional disease
Intrathecal Injection Diseases
Italy Deranged neural network
Mesenchymal G comparison
MRI Control groups
Multiple Sclerosis
Myelin
Nerve
Nervous System
Neural Network
Neurodegenerative Diseases
Neurons
PhD
Placebo
Primary Endpoint
Prime Time
Progressive
Relapse
San Francisco
Spinal Cord
Statistical Significance
Stockholm
Sweden
UCSF

Semantics

Type Source Name
gene UNIPROT LAT2
pathway BSID Neurodegenerative Diseases
disease MESH neurodegenerative diseases
disease MESH death
disease MESH inflammation
disease MESH uncertainty
disease MESH autoimmune diseases
gene UNIPROT DMTN
drug DRUGBANK Dimethyltryptamine
gene UNIPROT CLNK
gene UNIPROT CYREN
gene UNIPROT MAGEE1
gene UNIPROT ARID1A
disease DOID multiple sclerosis
disease MESH multiple sclerosis
disease MESH relapses
disease DOID relapsing-remitting MS
gene UNIPROT PDC
disease MESH community
gene UNIPROT MAP6
gene UNIPROT FUT1
drug DRUGBANK Mesenchymal Stem Cells
pathway BSID Immune System
disease MESH experimental autoimmune encephalomyelitis
gene UNIPROT MSC

Original Article

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