Regulation Kills: The FDA’s War on Gene Therapy

In April 2012, 7-year-old Emily Whitehead was on the brink of death. In April 2013, Emily spoke at an American Association for Cancer Research conference – cancer-free and with a full head of hair.

“Thank you everyone for giving us prayers and helping the doctors to make other kids better,” Emily told the room full of CEOs, “so cancer doesn’t take away any more of my friends.”

Emily suffered from acute lymphoblastic leukemia for more than a year before her parents enrolled her in a gene therapy trial at the Children’s Hospital of Philadelphia. In less than a month, Emily’s cancer was gone.

Reflected in Emily’s victory, however, is a national tragedy. According to a May 2012 Centre Daily Times article, Emily was the first child in the U.S. to receive this sort of treatment. Oncologists in China, however, have been successfully using gene therapy for several years.

Nearly a decade before Emily’s treatment, another American scored a similar victory against cancer. Suffering from a tumor “the size of a fist”, Adam Winiarski had been given months to live. Winiarski was interested in gene therapy and had nothing to lose – but the treatment he needed was inaccessible in the United States.

Determined to defeat his cancer, Winiarski traveled to China – where, according to American experts, gene therapy regulations are “too lax”. At Beijing’s elite Tongren hospital, Harvard-trained Dr. Niu Qi quickly sent Winiarski’s cancer into complete remission.

By denying Winiarski the treatment he needed, our government failed both an American citizen and our nation’s economy. In this instance alone, America seems to have lost a victory against cancer, Winiarski’s business, and even the business of a skilled Harvard alum. “Safety” – in whatever arcane way that concept is understood by government regulators – apparently takes precedent over all these concerns.

In 1999, 18-year-old Jesse Gelsinger became the first American to die while in a gene therapy trial. An FDA investigation concluded that Gelsinger’s death was caused by carelessness on the part of researchers. Yet months later, the FDA began a sweeping crackdown on gene therapy that has frightened many researchers away from thoroughly pursuing life-saving areas of study.

Gendicine, the drug that cured Winiarski, has been found to be three times more effective than radiotherapy in a clinical trial of 120 patients. The only recorded side effect was mild fever. Yet, while China’s FDA approved the drug for commercial production years ago, our own FDA has yet to approve a single gene therapy product.

In the nine years between Winiarski’s treatment and Emily Whitehead’s, our burdensome government has failed countless cancer patients as surely as Jesse Gelsinger’s doctors failed him. Thanks to obstructive regulatory practices, much-needed tools are languishing in a bureaucratic abyss – while Americans who could be benefiting from them are dying (or, as in Winiarski’s case, taking their business overseas).