Multiple Sclerosis: conclusion of the Phase I clinical trial with brain stem cell transplantation

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For the first time ever, all patients recruited in a brain stem cell transplantation trial were treated for the experimental treatment of secondary progressive multiple sclerosis.

Milan, 25 May 2020 - On the occasion of the World Day of Multiple Sclerosis, which is celebrated on 30 May, the Pontifical Academy for Life of the Vatican State and its President Archbishop Vincenzo Paglia, together with the Revert Onlus Association, announce the conclusion of the Phase I clinical trial that involves human brain stem cell transplantation in fifteen patients with secondary progressive multiple sclerosis. The experimentation, coordinated and financed by the Casa Sollievo della Sofferenza Foundation and by Revert Onlus with the patronage of the Stem Cell Foundation of Terni, was carried out thanks to the collaboration among clinical research centers, coordinated by the IRCCS Casa Sollievo della Sofferenza in San Giovanni Rotondo, such as the Hospital of Terni and the Cantonal Hospital of Lugano.

The Phase I trial, authorized by the competent commissions of the National Institute of Health and the Italian Medicines Agency, AIFA, with the protocol number Eudract 2015-004855-37, began in January 2018 with the admission of the first patient and is the first step towards the development of an experimental protocol to treat multiple sclerosis patients based on the transplantation of clinical grade human brain stem cells into the brains of patients suffering from this serious disease. The purpose of the trial is to verify the safety of the treatment. The fifteen patients included in the protocol were divided into four groups and transplanted with increasing doses of cells, the last six patients receiving the highest dosage (24 million cells). All the patients were discharged after 48 hours of observation following the transplant and they did not experience any side effects in the immediate post-operative period or in the months following. The clinical teams will continue the monitoring activities for at least a year after the surgery. Any possible neurological and therapeutic effects are now being evaluated.

«We are proud to announce this important milestone in the ongoing experimentation with brain stem cells» says Angelo Vescovi, Scientific Director of the IRCCS Casa Sollievo della Sofferenza in San Giovanni Rotondo and of the Advisory Board of Revert Onlus, as well as Professor of the University of Milano Bicocca. «We are now waiting for the one-year follow-up and the submission of the Phase II protocol for this disease as soon as possible».

The experimentation is based on scientific data which had a worldwide impact and which were published in 2003 in the journal Nature. This Phase I clinical study for Multiple Sclerosis represents the third stage of a journey that began 12 years ago with the creation of the world human brain stem cell bank - still today unique in the world. The study continued with the first transplant in eighteen patients with ALS in 2012, whose trial ended successfully in 2015, and Phase II will be initiated shortly.

«A flagship of Italian research» says Angelo Vescovi, who adds «we intend to make these cells available for research activities all over the world, in particular to research groups that at the moment cannot implement new experiments just for the lack of appropriate cells. All this will obviously occur in a non-profit system, as was the case in all our studies in which the patients didn’t have to incur any expenses».

This is how Archbishop Vincenzo Paglia comments the important announcement: «I thank the Director of IRCCS Casa Sollievo della Sofferenza, Prof. Angelo Vescovi, who has been working with tenacity and determination for almost thirty years to reach new goals in regenerative medicine and give hope to those suffering from multiple sclerosis and other neurological diseases. Revert's research - continues Archbishop Paglia - is unique in its kind because it is devoid of any ethical and moral issues and this makes us even more proud of the results obtained and makes us look to the future with greater optimism, always with respect for life. The milestone reached also shows that the initiative was not extemporaneous, but it evolved over the years, expanding and including other neurological diseases and the evaluation of desirable standardizable therapeutic effects. Sick people deserve to have answers and treatment options and it is only serious and constant work that can satisfy all of this, free of charge».

(Original text: Italian. Translation by Leonardo Stefanucci)

MS stem-cell transplant in world first. Ground-breaking operation in Rome

(ANSA) - Rome, May 25 - Brain stem cells have been transplanted for the first time in the world into 15 multiple sclerosis patients, the Pontifical Academy for Life, the Associazione Revert Onlus, and the Fondazione Casa Sollievo della Sofferenza said Monday.

    No patient has shown any side effects, they said.

    Possible therapeutic effects of the trial are now being assessed.