Concerns over stem cell therapy
for SMA type 1 children

The TREAT-NMD Project Ethics Council (PEC) has recently been made aware of the debate regarding the use of stem cells in the treatment of SMA type 1.

The members of the PEC have asked that the following statement from the president of SMA Europe be published on the TREAT-NMD website. The PEC's stance on the use of stem cells was initially published in January 2010 can also be found here.



It has recently come to our attention that there appears to be some confusion surrounding the subject of stem cell treatments for SMA Type 1 children in Italy.  As President of SMA Europe (a body representing the SMA organisations of 7 European countries: UK, France, Spain, Germany, The Netherlands, Switzerland and Italy), I thought it worth explaining our point of view, in the hope that it might help to clarify the reasons why clinicians and SMA organisations throughout Europe do not feel able to support stem cell treatments for SMA patients at this time.

SMA research is at a critical and exciting stage, with a whole range of possible therapies under exploration, the first of which are now just coming into or approaching clinical trials.  Whilst there are many frustrations associated with the length of time it takes to get new drugs and treatments licenced, the principles behind the different stages are necessary to ensure that we end up with treatments that not only result in conclusively proven benefits, but also do not cause any harm in the process.

Stem cell therapy has not satisfactorily passed any of these stages and we must therefore conclude that, based on our current understanding, there is not a role for it at this point in time.  The investigation by the Italian Health Minister in August 2012 came to the same conclusion, followed by the finding by AIFA (Italian Association for Medicinal Products) that the culture conditions and the handling of the cells to be transplanted did not meet the highest standards for therapeutic applications in humans, which was why the investigators pursuing this experimental avenue had their licence withdrawn with immediate effect.

We obviously deeply sympathise  with families who have a child affected by SMA Type 1, who are understandably attracted to the 'last hope' offered by stem cell treatment.  What parent would not do anything to try and save the life of their child, when faced with the prospect of losing them?

However, it is every clinician's responsibility to put their patients' interests first and, in these cases, it is the child that is the patient, and a very young one at that.  These unlicensed stem cell treatments are not only scientifically unproven in terms of their benefits, but are also potentially causing harm to children and compromising their quality of life.  It is up to clinicians to take all the facts into consideration and advise parents how to make the best and safest choices for their children, based on the scientific evidence available.

Stem cell therapy is a dynamic area of research for many conditions, with exciting results in some cases, and encouraging progress in others.  However, that does not make it a universal panacea and, although it is currently being explored for possible SMA applications in the future, it is only one of several avenues, with others much further ahead in development.

If and when stem cell therapy scientifically proves itself, then SMA Europe and all clinicians involved in the treatment of SMA will, of course, happily consider it within the range of experimental treatments available.  There may also be opportunities for children to participate in future clinical trials, but it is important that these are conducted within the framework of European law, rather than unlicensed within a rogue institution outside it.

Hope is a powerful emotion and an incredibly positive one, but it should never be used as an excuse for shortcuts in the pursuit of good quality research for the benefit of children now  and for the field to advance in the future.  SMA Europe and all the countries involved (including Italy) are working hard, along with other organisations in the US and all over the world, to fund the BEST research into this devastating condition, leading to the BEST treatments and the BEST outcomes which might, hopefully, one day mean a cure for Spinal Muscular Atrophy.

Casimir Knight

President, SMA Europe

12 Apr 2017