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This site is dedicated to scientific community working on ALS. Our aim is to optimize researchers time and efforts by providing updated, well organized information on novel findings, available resources and research support.
AriSLA - The Foundation for research on ALS - has been set up to make ALS research investments more effective and efficient, to speed up the clinical research impact e and to provide patients with better care, improved conditions and life expectancy. Its aim is to boost Italian excellencies in basic, clinical and technological research. The Foundation founders are Fondazione Cariplo, Fondazione Telethon, Fondazione Vialli and Mauro and AISLA.

 

 

Update about the Italian and American ALS Neural Cell therapy trials

Results of Phase I Italian clinical trial on amyotrophic lateral sclerosis (ALS) with transplant of human neural stem cells

 

The Revert Onlus Association and the IRCCS Casa Sollievo della Sofferenza, in collaboration with the Stem Cell Foundation presented on September 29, 2015 in Rome, the results of the Phase I clinical trial on amyotrophic lateral sclerosis, an advanced stem cell experimental therapy, conducted using GMP-grade cells, in compliance with the European Medicines Agency rules, and certified by AIFA.

The Phase I trial on ALS patients began on June 25, 2012 with transplant in the spinal cord (lumbar and cervical) of human neural stem cells from biopsies of naturally aborted fetuses, and ended on June 5, 2015, with the transplant of the eighteenth patient. Phase I trials are the first stage of testing in human subjects on small group of volunteers. This phase is designed to assess the safety (pharmacovigilance), tolerability, pharmacokinetics, and pharmacodynamics of a drug.

During the meeting the trial coordinator Prof. Angelo Vescovi, MD, Professor of Cell Biology at the University of Milan Bicocca, Scientific Director of IRCCS Casa Sollievo della Sofferenza (San Giovanni Rotondo), stated that no important adverse events attributable to the surgical procedure or the transplanted cells have been reported. This result has opened the way for the phase II clinical trial, which will involve 60-80 ALS patients, and will allow you to assess dosing requirements and verifying the efficacy of the neural stem cells treatment.

 

For further information http://www.revertonlus.org/index.php/progetti/la-sperimentazione-sulla-sla/

 

Previous article about this trial are available here:

http://www.alscience.it/?view=cnews362#c362

 

 

Neuralstem Investigator Provides Phase II Update on ALS Cell Therapy at American Neurological Association Annual Meeting

 

The encouraging results coming from a nine-month Phase II trial of NSI-566 human spinal cord stem cells (HSSCs) in Amyotrophic Lateral Sclerosis were recently presented at the American Neurological Association Annual Meeting (Chicago, September 27-29th) by Principal Investigator Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute, Director of Research of the ALS Clinic at the University of Michigan Health and Neuralstem unpaid Consultant.

Neuralstem concluded final surgeries in the company's NSI-566/ALS Phase II trial for ALS in January 2010, at Emory University. This Phase I safety trial, to evaluate the safety of the NSI-566 stem cells and surgical technique, was designed to enrol up to 18 patients.

Neuralstem concluded in the company's NSI-566/ALS Phase II trial, primarily evaluating safety, in July 2014. After a six month patient follow-up period, this phase of the study concluded in the first quarter of 2015.

The Phase II open-label, dose-escalating trial of NSI-566 evaluated 15 ambulatory patients with ALS, averaging a mean duration of disease of 15.5 months. Participants were divided into five dosing cohorts with three patients in each, who received increasing quantities of cells in the cervical region of the spinal cord via bilateral intraspinal injections ranging from two million to eight million cells. The fifth cohort received an additional eight million cells in the lumbar region.

The data were combined from those coming from Phase I trial and showed that the intraspinal transplantation of the cells was safe and well-tolerated throughout the escalating doses, reaching a maximum tolerated dose of 16 million cells via 20 bilateral injections. There appeared to be no acceleration in disease progression due to the therapeutic intervention. Secondary efficacy endpoints included stabilization of ALS Functional Rating Scale-revised (ALSFRSr) scores, and assessment of respiratory functioning, grip strength and muscle strength. Researchers calculated a 95% confidence limit around the slopes of decline of ALSFRSr scores, forced vital capacity (FVC) and grip strength of the ProAct historical database subjects, and evaluated if trial subjects fell within or outside those limits. 73% of Phase II patients, and 79% of combined Phase I and II patients, fell above the upper confidence limit of the ALSFRSr score. 50% of Phase I and II combined, and 40% of Phase II patients' forced vital capacity percent predicted fell above the upper confidence limit, compared to the ProAct database. For grip strength control, researchers used the Ceftriaxone (CEF) study database, since grip strength data was not available in the ProAct database. 67% of Phase I and II combined, and 60% of Phase II patients, all at nine months post-intervention, fell above the 95% upper confidence limit. 

The most common adverse event (AE) was transient post-operative pain due to surgery.  One serious adverse event due to the surgical procedure was observed, but was not attributed to the cells themselves. The patient's motor function was initially weakened and then recovered to the patient's ALS baseline. 

"Based on this encouraging safety and clinical effect, we look forward to moving to a  registration-directed trial in 2016," said Karl Johe, PhD, Chief Scientific Officer.

A larger control NSI-566/ALS Phase II trial is expected to commence in 2016.

See more at: http://www.neuralstem.com/cell-therapy-for-als#sthash.z0MjSTTQ.dpuf

 

Previous articles about this trial are available here :

http://www.alscience.it/?view=cnews267#c267; http://www.alscience.it/?view=cnews125#c125; http://www.alscience.it/?view=indice#c119

Here you can find ALS Stem cells clinical trials currently available on www.clinicaltrials.gov   http://www.alscience.it/#c351

 

 




     
     
     
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