The experimentation underway in the US and Israel on ALS patients. The hypothesis is that stem cells act by releasing protective factors that can favour the survival of neurons and therefore slow down the progression of the disease.
We publish the question sent by a reader to the ALS forum and the answer given by two experts: Dr. Christian Lunetta of the NeMO Clinical Center, Milan, and member of the AISLA medical-scientific commission and professor Piera Pasinelli, professor at the Thomas Jefferson University of Philadelphia and the Robert Packard Center for ALS Research in Baltimore (USA).
Hi, what do you think of the Brainstorm method, currently used in the United States and Israel as a trial for patients with ALS, based on stem cells? Can it be a road to finding a cure cure? Thank you. Andrew
Dear Mr. Andrea, the “Brainstorm” experiment, conceived by the company BrainStorm Cellular Therapeutics (Israel), is based on the repeated intrathecal administration of modified mesenchymal cells, according to the technique called NurOwn®, so that they release certain protective factors for the motor neurons. Mesenchymal stem cells are collected through a bone marrow sample from the patient himself; the hypothesis is that these cells act by releasing protective factors able to favor the survival of neurons and in this way they are able to slow down the progression of the disease, alleviating the symptoms. Currently the phase III study is active in 6 centers in the United States with the aim of evaluating the effectiveness of the treatment. The study includes 200 patients with ALS who will be divided into two groups: the first (100 patients) will receive modified mesenchymal cells, while the second control group (100 patients) will receive a placebo injection that does not contain the cells. This treatment will be carried out every 2 months for 3 cycles.
THE CONCLUSION OF THE STUDY
The previous phase I and II trials verified the safety and tolerability of the treatment and surgical intervention necessary for the transplantation of modified stem cells. On the basis of these results, the phase III trial was started which should close the enrollment of patients by mid-2019.
The conclusion of the study is expected, except for further delays secondary to patient enrollment, by mid-2020. At the end of the study, the researchers engaged in conducting the trial will communicate the results obtained to the scientific community and the authorities in charge. Only then can we really understand whether this therapeutic strategy will or will not be useful in the treatment of ALS. We hope that the phase III study can be completed soon so as to be able to give more complete information to the whole community of patients which obviously needs an effective therapy to combat the evolution of the disease.
THE SAFEST AND MOST EFFECTIVE TREATMENTS
We reiterate the need to wait for the trial to be completed in order to really understand if the treatment works and to determine the best intervention strategy for patients. Unfortunately there is a lot of information (and disinformation) online and on the sites / social media, but it is important to remember that clinical studies are the only way to establish whether a treatment is effective, what its possible side effects are and to know what is the best possible treatment in different situations. This is to ensure, as far as possible, the safest and most effective care for each case. As a result, each clinical study must be very rigorous and conducted on the basis of a trial protocol approved by the competent authorities.
Dr. Christian Lunetta , Clinical Center NeMO, Milan, Medical-Scientific Commission AISLA
Prof. Piera Pasinelli , Thomas Jefferson University, Philadelphia and The Robert Packard Center for ALS Research, Baltimore (USA)