Clinical trial to test efficacy of stem cell transplants in stopping ALS muscle deterioration

Once funding is obtained, researchers can perform a clinical trial to test the efficacy of transplanting bone marrow cells into muscles to stop deterioration caused by the illness. The method has already yielded positive results on mice.

The Neuroscience Institute at the Universidad Miguel Hernández (UMH) in Elche, Alicante, and the Spanish National Research Council (CSIC) are actively searching for €492,800 to be able to develop a worldwide pioneering test on 100 patients suffering from amyotrophic lateral sclerosis (ALS) which could, by using adult stem cells, stop the progressive deterioration of the locomotor apparatus - which eventually leads to patients' death. This therapy is spearheaded by the director of the Neuroscience Institute, Salvador Martínez, and has been successfully tested on mice.

The clinical trial would be undertaken over a three year period in the Virgen de la Arrixaca (Murcia), Hospital General (Alicante) and Sant Joan d'Alacant (Alicante) hospitals in eastern Spain and, if successful, would make it possible to then treat, for example, the 900 patients that are diagnosed with ALS every year in Spain, stopping the progressive and inevitable paralysis of their muscles. Neuroscience Institute manager Salvador Martínez has been looking into therapies to fight ALS for two decades, and as the head of a group of a dozen scientists, has been able to verify in a laboratory that transplanting bone marrow cells to the muscles stops the deadly progressive muscular deterioration caused by this illness.

This is possible because adult stem cells from the bone marrow act as guards and prevent the death of motor neurons (named as such because they are in charge of muscle movement), with the added benefit that there is no cell rejection because the transplanted cells are from the same patient. However, a lack of public resources has so far kept testing on humans from being a possibility due to the associated costs. Therefore, the hospitals from Murcia and Alicante have requested a financing project to the Carlos III Health Institute in Madrid, the national entity which covers clinical trials with no commercial purpose.

The objective is to test the transplant of bone marrow cells in the tibialis anterior muscle (located near the shin and which dorsiflexes, or lifts, the foot) as well as in the hand muscles, where the loss of strength due to the illness if clinically very noticeable. The trial would be double-blind and would consist of inoculating the muscles of one hand with bone marrow cells and those in the other with a placebo, without the doctor or the patient knowing which is which.

Once the financing is covered, the study will have to be verified by Spanish Medicine Agency AEMPS before it can be worked on as a potential therapeutic option, which would then make it possible to test other muscles commonly affected by the disease, such as the diaphragm. Martínez explains that 'this method of transplant keeps our hopes of being much more effective alive, because we can introduce many cells into a muscle, as many as we need, and the amount of times necessary (depending on the size of the muscle). Furthermore, the transplant is performed without surgery and only requires local anaesthetic.'

He explains that public funds are needed to proceed with this line of research against ALS, as this type of therapy 'isn't interesting for pharmaceutical laboratories' because it uses 'the patient's own cells' instead of marketable products.

As many as 100 patients from anywhere in the country would take part in the trial, as long as they have the green light to be recruited by their neurologist. Once the initial transplant is performed in the Arrixaca hospital, electromyography tests would be performed every three months for at least a year in the Hospital General and the Hospital de Sant Joan d'Alacant in order to objectively measure the evolution of the muscle's strength and range of movement.

Groups from Italy and Norway have already shown interest in this therapy and have asked the Neuroscience Institute for information in order to perform similar tests in their countries. Basic research has been performed in the Institute, located in the Sant Joan d'Alacant campus of the UMH, and has been financed for years by programs of the Spanish Government, the EU and the Spanish Network for Cellular Treatment (TerCel), as well as donations from trusts such as Diógenes and Rotary Illice.

On average, more than two people are diagnosed with ALS every day in Spain, an illness with no cure which only has low-efficiency palliative care treatment available, as it only prolongs a patient's life by about six months within a three to five year life expectancy period. A genetic origin has been established for 10% of cases, but it remains unknown for the rest, leading to the appearance of theories related to genetic and environmental predisposing factors.

Source: http://ruvid.org/ri-world/stem-cell-transplants-could-stop-als-muscle-deterioration/