Thank you, Liam Davenport, for your February 25th article, “Unproven Therapies are ‘Muddying’ Cell and Gene Advances” in Medscape. Although it was published several months ago, the content is well worth sharing with our readers. Your article shines a light on yet another example of the “cart before the horse,” where treatments are marketed and products are sold in the absence of research to ensure their effectiveness and safety.

Like you, we are concerned about the accelerating proliferation and marketing of novel cell and gene-based therapies for a wide array of conditions.  As you mention, this trend overshadows groundbreaking and encouraging advances in biomedical research, disrespects the hard work or those exploring new treatments properly, and can easily divert scant resources.

The points raised in this piece resonate with us on myriad levels. As is the case for so many parents of children with autism, individuals are spending thousands of dollars of hard-earned money for treatments that lack any evidence of scientific support.  In some cases, individuals are even relying on crowdfunding to garner the monies needed to access these treatments. The fact that there is so much interest in biomedical treatments that lack even an iota of scientific support is a sad reflection of the status of our current healthcare system and captures precisely what is happening within the autism community.

As per this article, the FDA has approved “breakthrough therapy” designations. We agree that such fast tracks may serve to protect companies from lawsuits and to remove the limit they can be charged to consumers. This creates a rich environment for exploitation and harm not just within the autism community but across a wide array of conditions.

We also appreciate the article’s reference to the continuum of clinical evidence as this is such an important point of discussion when evaluating the scientific support underlying new treatments. Sadly, such a continuum will likely be misrepresented by some “treatment” marketers who are more eager to gain profit than to help individuals with their respective conditions.

Thank you for your well written article and for sharing this information which in many ways mirrors the work of ASAT.  Consumer education remains essential during these challenging times and your article helps consumers better appreciate that the marketing of novel cell and gene-based therapies is far outpacing the scientific research that validates and guides its use, as well as to exercise caution when considering treatments as many lack scientific support.



David Celiberti, PhD, BCBA-D, Executive Director

Association for Science in Autism Treatment


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