The first known report of a brain tumor resulting directly from neural stem cell therapy was published yesterday. The report is a fresh reality check against worldwide hopes for life saving stem cell therapies that seem just on the horizon; a stark reminder that success will not be easy. It is also an example of the dangers faced by patients when they venture off to countries all over the world to receive treatments that are untested, unproven, and unregulated. In spite of this, stem cell therapy offers hope for major medical breakthroughs and research of the therapy must continue unabated.
The patient in question was a boy suffering from a rare genetic disorder, ataxia telangiectasia (AT), characterized by severe degeneration of the brain region that controls movement and speech. Beginning in 2001, the boy went to Moscow for several treatments in which fetal neural stem cells were injected into the brain and the fluid surrounding it. The hope was that these neural stem cells would somehow help the boy to regenerate neurons that had been destroyed by AT.
In 2005, four years after the treatment, tumors were found in the boy’s brain and spine. A biopsy of tumor cells from the boy’s spine confirmed that their origin was from the fetal neural stem cells from the treatment in Moscow.
Critics of stem cell therapy, especially embryonic based therapy, will be quick to point at this case as an example of the dangers posed by the therapy, but this sort of thinking is flawed. Stem cell therapy offers the hope of treatment for several of today’s most debilitating diseases and it should be pursued with our greatest urgency and effort. As this incident clearly demonstrates, however, the path toward success is not without risk and there is still much we have to learn.
This story serves as yet another example of the current battle being waged in the world between regulated and unregulated medical discovery. On the one hand we have places such as the United States, where regulations are strict and experiments such as those performed on this boy would not be permitted. On the other hand we have most of the rest of the world, including Moscow in this case, where humans are literally used as guinea pigs to “see what happens” when various medications or even stem cells are given to a patient with little or no scientific rigor to back it up.
It is our view that both the regulated and the unregulated camps have their place in the world. Highly regulated medical discovery gives medicine the discipline and rigor required for detailed understanding and reproducibility. Meanwhile, relatively unregulated medical discovery opens the doors to experimentation that are unique and potentially game changing.
Typically at the Hub we report on positive breakthroughs, but we cannot be credible if we don’t also shine the spotlight on related setbacks. Stem cell therapies undoubtedly offer great promise and we look forward to reporting on its progress even as setbacks like today will occur.