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Genetic Linkage

A Tale of Two Clinical Trials: Gene Therapy for a Rare Disease and a Vaccine for COVID-19

Encouraging preliminary findings in a phase I clinical trial for a COVID-19 vaccine were widely reported as soon as the paper appeared in The New England Journal of Medicine July 14. Coverage of the recent deaths of two boys in a clinical trial to test a gene therapy for a rare, devastating muscle disease were more under-the-radar.

Comparing the two very different scenarios illuminates the scientific rigor behind the clinical trial process.

 

The boys had X-linked myotubular myopathy. MTM affects 1 in 50,000 male births. Seventy-five percent of boys die in weeks or months of respiratory failure; average life expectancy is 29 months. Given that prognosis, taking the risk of an experimental treatment in a clinical trial makes sense. Parents of participants as well as physicians know that children can die during the trial, due to the disease or to toxicity at higher doses of a treatment.

 

In contrast, volunteers in a clinical trial to evaluate a vaccine are healthy.

 

Although comparing MTM gene therapy and a COVID-19 vaccine is a bit of apples and oranges, those fruits are in the same bin in terms of progression through the three phases of development of a new treatment or preventative:

 

To continue reading go to my blog DNA Science, where this post first appeared. 

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