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Old 02-06-2018, 06:37 PM
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Exclamation Researchers at Tisch show stem cell therapy may reverse disability in MS

Encouraging results from this Phase 1 trial. Next step: Phase 2. This is the press release. The abstract and more are in additional posts below. - D

RESEARCHERS SHOW STEM CELL THERAPY MAY REVERSE DISABILITY IN MULTIPLE SCLEROSIS

New York, NY – February 6, 2018 – Researchers at Tisch MS Research Center of New York (Tisch MSRCNY) have shown a stem cell-based treatment may reverse disability in progressive multiple sclerosis (MS). Results of the study add to the existing evidence demonstrating the safety and tolerability of intrathecal (injected into the spinal canal) administration of autologous mesenchymal stem cell-derived neural progenitors (MSC-NPs). The study has been published in EBioMedicine, a partnership between Cell Press and The Lancet.

MS is an immune-mediated demyelinating disease of the central nervous system and is one of the leading causes of disability in young adults. Cell therapy is emerging as a therapeutic strategy to promote repair and regeneration in patients with disability associated with progressive MS.

“The results from our study justify the initiation of a planned FDA-approved Phase II trial in a larger group of patients,” said Dr. Saud A. Sadiq, Director and Chief Research Scientist at Tisch MSRCNY. “We anticipate that these novel studies may form the therapeutic basis of reversing disability in patients with MS and if successful, these studies could have positive implications for treating other neurological diseases,” he added.

In the post-treatment efficacy analysis, study participants demonstrated a reversal of disability as determined by improved median expanded disability status scale (EDSS). In addition, of the 20 subjects, 70% had greater muscle strength and 50% exhibited improved bladder function.

“Our study was uniquely associated with repeated administrations of fresh, not cryopreserved cells, which may have contributed to the observed efficacy of the treatment,” said Dr. Violaine Harris, Senior Research Scientist at Tisch MSRCNY. “The protocol we created allowed for delivery of these stem cells within thirty minutes of harvesting the cells,” she stated.

This stem cell research has been conducted by Dr. Sadiq and his team since 2001. The study entitled, “Phase I Trial of Intrathecal Mesenchymal Stem Cell-Derived Neural Progenitors in Progressive Multiple Sclerosis,” was a culmination of over a decade of work. As a result of these promising developments, the FDA-approved Phase II study will commence to establish efficacy. Tisch MSRCNY has invested $5 million to build a new Regenerative Medicine Laboratory where all aspects of this innovative research and treatment will take place.

About Tisch MS Research Center of New York

Tisch MS Research Center of New York is an independent nonprofit organization dedicated to finding the cause and a cure for multiple sclerosis by conducting groundbreaking medical research to accelerate the pace at which research discoveries translate from lab bench to bedside. The Center aims to identify the cause of MS, understand disease mechanism, optimize therapies, and repair the damage caused by MS while offering patient access to the best and most advanced treatment possible.
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Old 02-06-2018, 06:39 PM
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Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis

Violaine K. Harrisa, James Starka, Tamara Vyshkinaa, Leslie Blacksheara, Gloria Jooa, Valentina Stefanovaa, Gabriel Sarab, Saud A. Sadiqa

Highlights

Repeated intrathecal dosing of autologous MSC-NPs in patients with progressive MS was safe and well tolerated.

The treatment was associated with functional neurological improvement, particularly in ambulatory patients.
Multiple sclerosis is one of the leading causes of disability in young adults. Our study is the first evidence suggesting that a cell-based therapeutic approach is capable of reversing disability in multiple sclerosis. Results of our study add to existing evidence demonstrating the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitors. Our study was uniquely associated with repeated administrations of cells freshly harvested from culture, as opposed to cryopreserved cells thawed at the bedside, which may have contributed to the observed efficacy of the treatment. The continued development of this therapeutic approach will likely have implications for treating other neurological diseases.

Abstract
Background
Multiple sclerosis (MS) is an immune-mediated demyelinating disease of the central nervous system and is one of the leading causes of disability in young adults. Cell therapy is emerging as a therapeutic strategy to promote repair and regeneration in patients with disability associated with progressive MS.

Methods
We conducted a phase I open-label clinical trial investigating the safety and tolerability of autologous bone marrow mesenchymal stem cell-derived neural progenitor (MSC-NP) treatment in 20 patients with progressive MS. MSC-NPs were administered intrathecally (IT) in three separate doses of up to 1 × 107 cells per dose, spaced three months apart. The primary endpoint was to assess safety and tolerability of the treatment. Expanded disability status scale (EDSS), timed 25-ft walk (T25FW), muscle strength, and urodynamic testing were used to evaluate treatment response. This trial is registered with ClinicalTrials.gov, number NCT01933802.

Findings
IT MSC-NP treatment was safe and well tolerated. The 20 enrolled subjects completed all 60 planned treatments without serious adverse effects. Minor adverse events included transient fever and mild headaches usually resolving in <24 h. Post-treatment disability score analysis demonstrated improved median EDSS suggesting possible efficacy. Positive trends were more frequently observed in the subset of SPMS patients and in ambulatory subjects (EDSS ≤ 6.5). In addition, 70% and 50% of the subjects demonstrated improved muscle strength and bladder function, respectively, following IT MSC-NP treatment.

Interpretation
The possible reversal of disability that was observed in a subset of patients warrants a larger phase II placebo-controlled study to establish efficacy of IT MSC-NP treatment in patients with MS.
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Old 02-06-2018, 06:40 PM
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FULL STUDY (Free as of this posting):

https://www.sciencedirect.com/scienc...52396418300513
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Old 02-06-2018, 06:54 PM
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Phase 2 Study Details (max EDSS 6.5):

Brief Summary:
This is a Phase II, Double-Blind, Placebo-Controlled, Randomized, Cross-over Study designed to determine efficacy of multiple intrathecal administrations of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) compared to placebo in patients with progressive multiple sclerosis. Efficacy will be measured through assessment of disability outcomes. Study participants will receive six intrathecal injections of culture-expanded autologous MSC-NPs at two month intervals in one year and six lumbar punctures as placebo treatment in a second year.

Full details including all eligibility criteria:
https://clinicaltrials.gov/ct2/show/NCT03355365
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Old 02-08-2018, 12:12 PM
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Just got this. Currently they are ONLY recruiting from their center. - D

Quote:
Thank you so much for sharing our news, it is truly appreciated.

We are not actively recruiting patients for our study outside of the Tisch MS Research Center at this time. Our goal is for the trial to lead to an FDA approved treatment for MS and one day be available to people with MS from Centers all over the world.

Please let me know if you need any additional info.
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