Sightlines

Decoder: Gene Therapy Breakthrough

Since 1989, labs around the world have initiated more than 2,200 clinical trials testing gene therapies, but only a few studies have resulted in government-approved applications. The United States has led the way in this research to treat or prevent disease, but Washington has authorized not one therapy for commercial development. In October, Philadelphia-based Spark ...

CHANGSHA, HUNAN - JUNE 19:  Chinese scientists work at the national laboratory of medical genetics of China in Central South University June 19, 2006 in Changsha city, Hunan province of China. The lab was founded in 1972. In 1991, the laboratory was selected and founded by the national government as the national laboratory of medical genetics.The research in the lab focuses on mapping, identification, and functional analysis of the hereditary disease linked genes. Its research has recently been extended to gene therapy and human cell. Scientists in the lab have contributed more than 300 scientific publications since its establishment.  (Photo by Guang Niu/Getty Images)
CHANGSHA, HUNAN - JUNE 19: Chinese scientists work at the national laboratory of medical genetics of China in Central South University June 19, 2006 in Changsha city, Hunan province of China. The lab was founded in 1972. In 1991, the laboratory was selected and founded by the national government as the national laboratory of medical genetics.The research in the lab focuses on mapping, identification, and functional analysis of the hereditary disease linked genes. Its research has recently been extended to gene therapy and human cell. Scientists in the lab have contributed more than 300 scientific publications since its establishment. (Photo by Guang Niu/Getty Images) Credit: Guang Niu / Staff

Since 1989, labs around the world have initiated more than 2,200 clinical trials testing gene therapies, but only a few studies have resulted in government-approved applications. The United States has led the way in this research to treat or prevent disease, but Washington has authorized not one therapy for commercial development.

In October, Philadelphia-based Spark Therapeutics announced that it was applying for U.S. regulatory approval after it treated an inherited eye disease that causes blindness. If given the green light, the treatment will join the likes of China-approved Gendicine, the world’s first gene therapy ever to see the commercial light of day (in 2003); Neovasculgen, approved by Russia in 2011; and Glybera, which the European Commission approved in 2012. And that’s where the list seems to stop.

Why are there so few approved therapies? Developing them is extremely complicated; their application also presents serious risks. (U.S. trials nearly halted after 18-year-old Jesse Gelsinger died in 1999 during gene therapy treatment for a rare liver disorder.) Moreover, there are endless ethical questions: How can a push for “designer babies” be avoided? What will happen if genetic changes are inherited by generations to come?

These questions may never get firm answers, but that won’t stop people from dreaming of a future free from a slew of genetic disorders — and thus a healthier world.

Who’s Treating What? 

The target of a whopping 64 percent of gene therapy studies since 1989, cancer has consistently been the focus of clinical trials, with North America leading the charge. But the continent is also responsible for the majority of all clinical studies across each category (save infectious diseases and healthy volunteers). It has exclusively conducted 64 percent of them and dwarfs Europe, which has initiated only 27 percent.

 

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Boom and Bust

Prior to the year in which China approved the world’s first gene therapy, only 13 trials had been conducted in Asia. Since then, the continent has picked up its pace: Of the 104 trials it has ever conducted (with known initiation dates), more than half were launched after 2011—and 36 percent between 2014 and July 2015. In contrast, the figures for North America and Europe have stayed relatively unchanged since the late 1990s.

Data were collected on Feb. 3, 2016, from the Journal of Gene Medicine, which included a total of 2,210 clinical trials in the database. Data range from 1989, when scientists conducted the first human gene therapy trial, to July 2015, when it was last updated. A version of this article originally appeared in the March/April issue of FP.

Photo credit: Getty/Guang Niu

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