Stem cell safety trial gets underway

Stem cells have been injected directly into the spinal cord of an American with MND as part of the first rigorous clinical trial to assess the safety of stem cell treatments for the disease.

The safety trial is being fully funded by a biotechnology company called Neuralstem, which has developed a process for generating large quantities of neural stem cells from the spinal tissue of a single eight week old foetus. (These are not the same as embryonic stem cells, which are derived from embryos at the very earliest stage of development.) The U.S. Food and Drugs Administration (FDA) has given approval for the trial, which is being conducted by neurologists and neurosurgeons at Emory University ALS Center in Georgia, using cells provided by Neuralstem.

Safety, not effectiveness

At least 12 volunteers with MND will undergo a new surgical procedure to receive injections of stem cells into their spinal cords. The trial aims to establish only whether or not this transplant procedure is safe. Participants will embark on the trial one at a time and will be monitored closely for a long time after undergoing the procedure, with results not expected for at least 2 years. It will be left to future trials to reliably establish whether or not the stem cells can actually slow disease progression.

Neural stem cells have the potential to turn into the different types of nerves and support cells normally found in the central nervous system but Neuralstem have stressed that they do not expect their cells to cure ALS or replace damaged motor neurones. Instead, they hope that their cells might protect surviving neurones.


Additional trial receives approval

Another American company known as TCA Cellular Therapy has also recently received FDA approval to go ahead with a safety trial of a different type of stem cell for the treatment of MND. In this case, bone marrow stem cells will be collected from each trial participant and then processed in TCA Cellular Therapy’s labs. They will then be injected back into the participant’s spine via lumbar puncture.


Stem cell therapy remains unproven

These two trials are the first rigorous, FDA-approved safety tests of stem cell treatments for MND and represent the earliest stages of the clinical trials process. Until a treatment has completed all the stages of this process, including tests of effectiveness in large numbers of people with MND, it remains unproven.

The stem cell treatments being offered at various clinics around the world have not undergone clinical trials and their safety has not been reliably tested. Unlike the participants in the trials mentioned above, people with MND who choose to attend these clinics will have to pay for their treatments. They are also unlikely to be monitored by the clinic for any length of time after receiving their treatment. More information on the issues surrounding unproven treatments can be found in Research info sheet U - Unproven Treatments (130 kb) . The International Society for Stem Cell Research has also produced a useful patient information booklet.