aHSCT – Autologous Hematopoietic Stem Cell Transplant in MS

Autologous hematopoietic stem cell transplant, also known as “stem cell therapy” or by the acronym aHSCT, is an emerging yet controversial treatment method for multiple sclerosis (MS).

How aHSCT works for treatment of multiple sclerosis

The first step in aHSCT for MS involves collecting stem cells extracted from the patient’s bone marrow, peripheral blood or umbilical cord blood. After the material is collected, the patient’s immune system is either partially or completely ablated (immunoablation), which means the immune system is significantly reduced or destroyed temporarily with combinations of chemotherapy, monoclonal antibodies, and anti-thymocyte globulin. Because multiple sclerosis is an autoimmune disease, immune system suppression is a key step to essentially “reprogramming” it to function properly.

After immunoablation is complete, the collected stem cells are then re-infused to reconstitute the immune system and re-activate it. The intended result of the therapy is to return the immune system to a functional state so that the body’s autoimmune attack on myelin is halted.

Clinical studies involving aHSCT for MS

Autologous hematopoietic stem cell transplantation, an experimental therapeutic approach for multiple sclerosis as well as other diseases, has been tested in clinical settings and reviewed by leading scientists. Below are consolidated reports on three prominent aHSCT studies.

The first study consists of a case series involving a mix of 151 participants with relapsing and progressive MS. All participants underwent the treatment at Northwestern University in Chicago from July 2003 to February 2014.

A significant proportion of the patients (63%) were treated off study protocol on a compassionate basis (having secondary progressive disease, an Expanded Disability Status Scale [EDSS] score of 6.0 or more, or a particularly disabling disease). The remaining 55 participants (37%) were treated on the study protocol and met the criteria. Criteria included relapsing-remitting MS, EDSS between 2.0 and 6.0, received treatment with at least one FDA-approved drug, and had at least two corticosteroid-treated relapses within the last year or one corticoid treated relapse and gadolinium enhancing lesions shown on an MRI.

A relatively low proportion of treatment-related complications (9%) occurred and no deaths were reported. There was significant improvement in the EDSS for the majority of patients, which was the primary objective of the study, and 80% of the participants achieved disease-free survival at two years after treatment.

Another study, HALT-MS, was a single arm, Phase 2 trial of immunoablation followed by autologous transplantation of stem cells. The study included 25 participants (with relapsing-remitting MS, EDSS score between 3.0 and 5.5 and two or more clinical relapses in 18 months despite other treatment). The primary objective was noted time until treatment failed. After two and three years of treatment, the overall event-free survival probability was 83% and 78%, respectively. The EDSS score improved a median of 0.5 after three years.

The third study was a single arm, Phase 2 of immunoablation followed by autologous transplantation of stem cells across three Canadian Hospitals (NCT01099930). The study included 24 participants with aggressive MS. All had multiple early relapses, an EDSS score of at least 3.0 with five years of diagnosis, and evidence of ongoing clinical disease activity despite at least one year of treatment.

All 23 surviving participants were free of clinical relapses and new gadolinium enhancing lesions for the duration of follow-up (median 6.7 years).

Seventeen participants (70%) had no more progression in EDSS scores after treatment and eight patients (35%) had sustained improvement in EDSS scores three years after treatment. The rate of brain atrophy was not substantially different from healthy volunteers.

The three studies offer several positive results for continued investment in the role of aHSCT for MS patients.

More recent studies in aHSCT indicate that the therapy might be more successful if done in the earlier inflammatory stages of MS. Ideal candidates may be patients less than 40 years old with a short disease duration of less than five years, recurring and disabling relapses, presence of inflammatory activity shown on brain MRI scans, and who were unresponsive to approved therapies.

However, a large, randomized clinical study comparing aHSCTs with the best approved therapies is still needed to confirm the role of stem cell transplant in MS management.

Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.


  1. http://link.springer.com/article/10.1007/s00415-016-8284-z
  2. http://www.tandfonline.com/doi/full/10.1586/14737175.2016.1158648
  3. http://jamanetwork.com/journals/jama/fullarticle/2091305

NurOwn Cell Therapy Found Safe, Effective for Progressive MS in Phase 2 Trial

NurOwn cell therapy led to significant improvements in the physical abilities, vision, and cognition of people with progressive multiple sclerosis (MS) in a Phase 2 clinical trial, top-line data show. In addition to these positive efficacy results, BrainStorm Cell Therapeutics, NurOwn’s developer, announced that its cell-based therapy showed a good overall safety profile. Trial participants received […]

MS News That Caught My Eye Last Week: Plegridy, Telemedicine, AI for MS, Myelin Repair

FDA Approves Plegridy as Intramuscular Injection for Relapsing MS A common patient complaint about Plegridy has been that its subcutaneous injection procedure results in injection site reactions. This new formulation is delivered into the muscle, rather than under the skin. The needle is longer, but Biogen pharmaceuticals thinks the reactions should be fewer. The U.S. […]

Potential for Stem Cells to Repair Myelin Focus of University Toronto Team

Teams of scientists at the University of Toronto are sharing a nearly CA$21 million (about $16.36 million) award into research that might lead to self-repair treatments for multiple sclerosis (MS) and other neurological disorders, the university announced in a press release. Eleven teams at the university and its partner hospitals will share the three-year grant […]

#MSVirtual2020 – MRI Changes Can Reflect Function in Progressive MS, Study Says

Certain MRI measures of the brain and spinal cord directly associate with functional improvements in people with progressive multiple sclerosis (MS), a new study reports. According to BrainStorm Cell Therapeutics, these data will help in determining the benefits of NurOwn, the company’s stem cell-based therapy, in progressive MS patients participating in its ongoing Phase 2 trial […]

Ability to ‘Create’ Astrocytes Supports Their Damaging Role in MS, Like Diseases

An inflammatory environment can turn astrocytes, key supportive cells for neurons, into their killers, fostering the progression of neurodegenerative diseases like multiple sclerosis (MS), a new study shows. This work, led by researchers at the New York Stem Cell Foundation (NYSCF), created for a first time astrocytes derived from human induced pluripotent stem cells (hIPSCs). The […]

‘Transcient’ Damage to CNS Seen with Chemotherapy Used in Stem Cell Transplants for MS

A high-dose chemotherapy combination given to wipe out the immune system before its rescue with autologous hematopoietic stem cell transplant (aHSCT) can cause “transient” damage to neurons and supporting cells of the central nervous system in people with aggressive multiple sclerosis (MS), a Canadian study reports. Nonetheless, its researchers believe that the risk of such neuronal toxicity […]

NurOwn May Curb Damaging Neuroinflammation in MS, Study Finds

NurOwn, believed to have neuroprotective and repairing effects, may also be able to curb the damaging immune responses that contribute to multiple sclerosis (MS) progression, a recent study found. This newly identified potential may extend the benefits of this cell-based therapy, its researchers believe. The findings were to be presented at the 2020 American Academy of Neurology (AAN) annual […]

Study of Stem Cell Therapy for Highly Active RRMS Honored by CR Forum

The MIST Phase 2 clinical trial, supporting the potential of hematopoietic (blood cell-producing) stem cell transplant (HSCT) to significantly slow disability progression in highly active relapsing-remitting multiple sclerosis (RRMS) patients, has received a Distinguished Clinical Research Achievement Award from the Clinical Research (CR) Forum. Five years after the transplant, most treated patients showed no further disease progression or activity, […]

ImStem Biotechnology to Launch Phase 1 Trial Testing IMS001 for MS

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold and cleared the investigational new drug (IND) application to assess IMS001 in the treatment of multiple sclerosis (MS), ImStem Biotechnology announced. The company plans to start a Phase 1 study in the U.S. this year to evaluate the stem cell therapy in several […]

The Multiple Sclerosis Podcast