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News Feature: Gene therapy successes point to better therapies
Thursday, 2019/11/28 | 08:09:56

Elie Dolgin - PNAS November 26, 2019 116 (48) 23866-23870


Despite some data concerns, two treatments for a rare pediatric killer could usher in a new wave of innovative medicines for neurological conditions.


Figure: The drug milasen, tailor-made by Timothy Yu (Left) for a young girl named Mila Makovec (CenterMother 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.


The drug Zolgensma was recently in the news for all the wrong reasons.


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 efficacy of Zolgensma,” terming the miscues an “isolated incident.” And despite the October setback, the original aim for the drug—administered to kids via infusion into the bloodstream—was not affected and continues to find success.


In the eyes of the public and politicians, the reputation of Zolgensma—the first gene replacement therapy to hit the market for a neurological disease—may be tarnished, at least in the near term. But the flurry of media coverage may also have obscured what the drug development community believes to be the more enduring story of Zolgensma: its impact not only on patients but on the entire field of gene therapy.


Zolgensma’s approval in May capped off a landmark run for the biotech industry, coming just 2.5 years after the arrival of Spinraza (jointly developed by Biogen and Ionis Pharmaceuticals), the first antisense drug to substantially alter the course of a life-threatening disease. Thanks to these two cutting-edge medicines, a diagnosis of SMA is no longer a death sentence. Researchers and pharmaceutical companies alike now look to Zolgensma and Spinraza for inspiration as they pursue the next generation of therapies that target the root causes of other devastating neuromuscular diseases—Huntington’s, amyotrophic lateral sclerosis (ALS), and muscular dystrophy chief among them.


“This is the beginning of a coming revolution in the treatment of severe neurological diseases,” says Michael Ehlers, the former head of research and development at Biogen. “And we look forward to a time when we look back at this period and say, ‘SMA paved the way for an entire set of severe neurological diseases that now have treatments.’”


The regulatory process for Zolgensma fell short. And drug prices will continue to prompt outrage among patients and policy makers. But the science behind these drugs suggests that both could prove to be therapeutic trailblazers.


See https://www.pnas.org/content/116/48/23866

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