Stem Cell Clinical Trials and MS

Transplantation of stem cells from various cell sources may represent a safe and effective therapy for multiple sclerosis (MS).

Autologous Hemopoietic Stem Cell Transplantation in MS

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

How Autologous hemopoietic stem cell transplantation works for treatment of multiple sclerosis

The first step in Autologous hemopoietic stem cell transplantation 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 Autologous hemopoietic stem cell transplantation for MS

Autologous hemopoietic 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-MES, 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. 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 Autologous hemopoietic stem cell transplantation indicate that the stem cell 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.

Mesenchymal Stem Cell (MSCT) Clinical Trials in MS

Autologous mesenchymal stem cells, commonly called MSCs, have been shown to induce immunologic and neurologic effects (neuroregenerative and neuroprotective) in animal models. Autologous transplant means that the cells are taken from then re-injected into the same subject.

Under the right conditions, animal studies have shown that mesenchymal stem cells can mature into myelin-producing cells in MS disease models. Myelin is important because MS is characterized by damage to the myelin sheath, a protective covering that surrounds the nerves of the central nervous system.

Adipose-derived stem cells (ASC) are adult MSCs taken from fat. They can be isolated through enzymatic digestion.  mesenchymal stem cells are also found in bone marrow, skin, and other body parts.

Mesenchymal Stem Cell Studies

A recent study evaluated the safety and tolerability of intrathecal (injected into the spine) autologous transplant of adipose stem cells over one year. The secondary objectives were relapse rate and progression of the disability which were assessed 18 months after the study began. Researchers aimed to achieve local immunomodulation (return) of the participant’s immune system via transplantation of autologous adipose stem cells.

Results of the study suggested that ASC intrathecal therapy for MS is safe and slows the progression of the disease. The method may benefit people with rapidly progressive MS because it is a powerful anti-inflammatory and immunosuppressive treatment. No differences in the Expanded Disability Status Scale (EDSS) score were found in the 18 month-period between groups of relapsing-remitting and slow progressing MS participants. No disease progression occurred among participants by the end of the study follow-up. No serious or severe adverse effects were observed. Time without progression of the disease was 18 months for most participants.

adipose stem cells are promising candidates for cell-based therapies aimed at stopping and reversing the loss of myelin in MS patients, but ACSs should be reserved for aggressive cases, for disease still in the inflammatory phase, and for the rare malignant form of the disease.

Bone marrow-MSCs were intrathecally administered in another study. The study showed that the treatment was safe and well tolerated in people with MS and that no adverse events occurred.

Another recently published study demonstrated that damaging immune system defects seen in patients with multiple sclerosis (MS) may be repaired using umbilical-cord MSCs.

We offer Stem Cell Immunotherapy or Gene Therapy for many diseases and mesenchymal stem cells Immunotherapy Treatments for many disease types now, and the list is growing!

With expanded, manipulated, and activated stem cells we can provide better results than with early non-manipulated stem cell applications.

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