Gene therapy successes point to better therapies

Abstract

Despite some data concerns, two treatments for a rare pediatric killer could usher in a new wave of innovative medicines for neurological conditions . The drug Zolgensma was recently in the news for all the wrong reasons. The drug milasen, tailor-made by Timothy Yu ( Left ) for a young girl named Mila Makovec ( Center ; Mother on Right ) who has Batten disease, was based on the drug Spinraza, which is helping pave the way for future gene therapies. Image credit: Boston Children’s Hospital. In August, the US Food and Drug Administration (FDA) gave drug manufacturer AveXis, Inc, a subsidiary of Novartis AG, a major slap on the wrist for violations related to the approval of Zolgensma, a new treatment for spinal muscular atrophy (SMA). The agency said the company had failed to promptly report to the proper regulatory authorities issues of data manipulation in some product testing. Ominous newspaper headlines followed. It didn’t help that the drug is extremely expensive—a record-setting $2.1 million for a single dose. In part, the need for only limited doses drove the price up; pharmaceutical companies typically develop drugs that patients need to take for a lifetime. Even so, the price tag sparked debates anew about drug affordability. Adding to Zolgensma’s woes: In October, Novartis and AveXis halted a study of the drug’s use in adults, citing safety concerns observed in monkey studies when the therapy was administered directly into the spinal fluid. But some of the regulatory concerns, at least, seem to be overblown. According to the FDA’s inspection report, the initial data discrepancies were limited to a handful of mouse experiments, and importantly, the human clinical results look sound. In an August 28 webinar hosted by Cure SMA, senior FDA official Peter Marks said the agency continues to “remain confident in the safety and …