Saturday, January 24, 2009

Stem Cell–Derived Therapy Approved for Clinical Trial

Back in 1990 the first human somatic gene therapy trial began (see this). The subjects of the earliest gene therapy experiments were children who suffered from a rare genetic disease called adenosine deaminase deficiency (ADA), a form of SCID (also known as the " bubble boy" disease-- see here).

Today, 19 years later, there are over 1400 clinical trials for gene therapy worldwide (see here) for a variety of diseases ranging from cancer, single gene disorders, infectious diseases and neurological disorders. Most of these clinical trials are in the first phase, which means they have yet to be shown to be safe and effective therapeutic interventions. But there is hope, as this story and this story indicate. And there has also been serious setbacks.

It is against the background of the noble aspirations and yet complex challenges facing gene therapy that I think we should embrace the good news that the first embryonic stem cell derived therapy has been approved for clinical trial.

Contrary to what the media reports, the real story here is not that that these stem cells are derived from human embryos and that this raises profound questions about the ethics of improving the life of some at the cost of others-- for these are not embryos that anyone intends to implant in a uterus and (possibly) grow into a human. The real story is how dogma and ignorance (and politics!) threatens to hold science and medicine back, and thus delay therapeutic benefits to people who have suffered spinal cord injuries (and countless other possible diseases).

So this announcement is an important move forward for medical science. But we should appreciate the complex challenges that lay ahead, and adopt a realistic standard for measuring the success of these novel interventions. One that measures things in terms of decades rather than a year or two.