STUDY: Stem cell treatment in progressive MS—repair and regeneration possible

[ActiveMSers]

Multiple Intrathecal Dosing of Neural Progenitors Administered to Progressive MS Patients with Disability Is Safe and Improves Disability Scores

Authors

Saud Sadiq, Leslie Blackshear, Gloria Joo, Tamara Vyshkina, Violaine Harris
New York, NY, USA

Abstract

OBJECTIVE: To evaluate safety, tolerability, and preliminary efficacy of three intrathecal (IT) administrations of autologous mesenchymal stem cell-derived neural progenitors (MSC-NPs) in progressive multiple sclerosis (MS).

BACKGROUND: MSC-NPs are an autologous bone marrow-derived population with regenerative potential. Preclinical studies in mouse EAE resulted in improved neurological function, suppression of local inflammatory response, and trophic support at the lesion site. Pilot clinical experience with IT MSC-NPs in six MS patients also supported the dosing, safety, and potential efficacy of this therapy.

DESIGN/METHODS: The study is an FDA-approved phase I clinical trial of autologous MSC-NPs administered IT in three doses of up to 10 million cells per injection, spaced three months apart. Enrollment includes 20 MS patients with established disability (range 3.5 to 8.5) and relatively stable disease as evidenced by less than 1.0 point change in EDSS in the last year, and stable MRI disease burden with no enhancing lesions in the last six months. Primary safety outcomes include adverse event assessments. Secondary efficacy outcomes include EDSS, timed 25 foot walk, PEG test, PASAT, evoked potentials, and urodynamics testing. Patients are followed 3 months and 6 months after treatment.

RESULTS: IT-MSC-NPs have been administered to all 20 study participants. Thirteen out of 20 have received all 3 doses and 3-month follow up. There were no serious adverse events, and minor adverse events included transient headache and/or fever in approximately 65% of patients. Neurological improvements were observed in a subset of patients after treatment, which included improved EDSS, improved PEG tests, and better bladder function clinically and on urodynamic testing.

CONCLUSIONS: The MSC-NP trial is the first of its kind to test IT administration of neural progenitors as a regenerative MS therapy and appears safe and well tolerated. Based on the promising efficacy trends, a phase II trial is warranted.

[ActiveMSers]

Here is the press release. A long way to go, but promising results... - D

Stem Cells Show Reversal of Disability in Multiple Sclerosis at the American Academy of Neurology (AAN) 2016 Annual Meeting

MS Patients Take Steps without Cane as Damage is Repaired

Dr. Saud A. Sadiq, Director and Chief Research Scientist at the Tisch MS Research Center of New York (Tisch MSRCNY) will be presenting on Tuesday, April 19, 2016, the extraordinary results from his FDA-approved stem cell clinical trial for MS at the American Academy of Neurology’s 68th Annual Meeting in Vancouver, BC, Canada. Dr. Sadiq will deliver the highly anticipated news during a session titled “Remyelination and Repair in Multiple Sclerosis (MS) Data Blitz Presentations.”

The presentation reveals data from the Phase I trial showing that the novel stem cell treatment was safe and well-tolerated with no serious adverse events reported. What’s more, the cutting-edge protocol of this trial created at Tisch MSRCNY allowed for the delivery of brain-like neural cells within 30 minutes of harvesting, a technique not seen anywhere else in the world.

“Repair and regeneration is possible. We have a patient who no longer needs her cane, one who has transitioned from a motorized scooter to taking steps with a walker and another who has discontinued their bladder medication as those symptoms have dramatically improved. This is the first treatment that improves established disability in patients with progressive MS and shows us there is hope that a future treatment is possible,” stated Dr. Saud A. Sadiq.

Due to these unprecedented results, the FDA has already advised Tisch MSRCNY to begin preparations for Phase II of this important trial to establish efficacy of stem cells as a reparative therapy. This is expected to commence at the end of 2016, once funding is secured.

About Tisch MS Research Center of New York
The mission of the Tisch Multiple Sclerosis Research Center of New York is to conduct groundbreaking medical research to ensure unparalleled care and positive outcomes for MS patients. Its integrated relationship with the International MS Management Practice (IMSMP) accelerates the pace at which research discoveries translate from lab bench to bedside. The Center aims to identify the cause of MS, understand disease mechanisms, optimize therapies, and repair the damage caused by MS while offering patients access to the best and most advanced treatments possible.