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410: A father’s mission to save his daughter, and allegations on telehealth GLP-1 prescribing

The Readout Loud1h 4m

This STAT News podcast episode covers three major biotech stories: an investigation into telehealth company LifeMD's questionable GLP-1 prescribing practices, a lawsuit between Novo Nordisk and Eli Lilly over deceptive advertising, and an in-depth feature on Matt Wilsey's mission to develop a gene therapy for his daughter Grace's ultra-rare NGLY1 deficiency disease.

Summary

The episode opens with discussion of a federal arrest of Ronald Fisher, a fugitive anesthesiologist who disappeared during a sexual assault trial in 2005 and may have been working as a biotech executive under an assumed name at MX Biopharma without proper background checks. The hosts then shift to Elaine Chen's investigation into LifeMD, a telemedicine company partnered with Novo Nordisk to prescribe GLP-1 medications. Chen interviewed five former employees and reviewed lawsuits filed by two top leaders, revealing allegations that LifeMD pressured providers to see 25 patients per hour, discouraged clinically relevant questions, and provided inadequate patient follow-up and screening. One former COO alleged inappropriate prescribing by the chief medical officer caused him severe side effects including inner ear damage, weight loss-related complications, and ongoing neurological decline. While LifeMD denies these allegations, the investigation raises broader questions about pharmaceutical companies like Novo Nordisk and Eli Lilly partnering with potentially problematic telehealth providers while publicly criticizing compounded GLP-1 alternatives. Chen also reports that Novo is suing Eli Lilly for deceptive advertising because Lilly's head-to-head trial comparisons use Novo's previous 2.4mg dose rather than the newly launched 7.2mg dose, though Lilly argues head-to-head trials are the gold standard and they haven't studied the new dose in comparison. The episode then features an extended interview with Matt Wilsey, founder of Grace Science, about his decade-long journey to develop a gene therapy for his 16-year-old daughter Grace, who has NGLY1 deficiency—a devastating ultra-rare genetic disease affecting every cell in the body with a median lifespan of 14.5 years. Wilsey describes the disease's complexity, his early rejection of end-of-life advice, and his decision to start his own company rather than rely on existing pharmaceutical or academic entities. He explains his pivot from small molecule approaches to gene therapy, the challenging clinical development process including dosing decisions where preclinical animal studies showed potential neurotoxicity at high doses, and the decision to dose Mason Stevens, another patient, first to prevent him from aging out before FDA approval. Grace experienced severe complications after receiving a high dose (3E15 total VGs) including hospitalization, loss of ambulatory function, blood clots, and ICU admission, but has since recovered physical function and shown cognitive improvements. The FDA review team has been collaborative despite manufacturing data concerns, and Wilsey advocates for new regulatory pathways to handle the 20,000+ genes with potential rare disease treatments. He emphasizes that parents of affected children should be at the regulatory table because they understand the risk-benefit calculation differently—comparing drug risks against certain death rather than against placebo. The episode concludes with a sponsored segment featuring Jared Baton from Gilead Sciences discussing the 15-year anniversary of the HPTN 052 study that established the U=U (Undetectable=Untransmittable) concept, which transformed HIV from uniformly fatal to a manageable chronic disease and helped destigmatize the condition globally.

About this episode

What does it take to develop a therapy for an ultra-rare disease? What are the allegations against a telehealth company that’s partnered with Novo Nordisk? And has a wanted fugitive been posing as a biotech executive? We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. We speak with our colleagues Jason Mast and Matthew Herper about their impactful look at one father’s mission to develop a treatment for his daughter’s ultra-rare disease. The subject of that feature, Matt Wilsey, also joins us.

Key Insights

  • LifeMD allegedly pressured providers to review 25 patients per hour including new patients and dose escalations, practices former employees considered clinically irresponsible and potentially unsafe.
  • A former LifeMD executive alleged that rapid, unmonitored dose escalation caused him severe neurological complications including inner ear damage, malnutrition, falls, fainting, speech impairment, and memory loss that persist years later.
  • Novo Nordisk and Eli Lilly publicly criticized telehealth companies for offering compounded GLP-1 alternatives citing safety concerns, yet both companies now actively partner with and recommend telehealth providers using potentially questionable practices.
  • Novo Nordisk's lawsuit against Eli Lilly over advertising claims that comparisons using older dosing are deceptive reflects the intensely competitive GLP-1 market where every claim and advertisement is contested.
  • Matt Wilsey rejected offers from multiple companies to develop a single-patient treatment, insisting that 'Grace is not going to be an outlier. It's all of us or none of us' in commitment to treating the entire NGLY1 community.
  • Grace Wilsey experienced severe complications from her gene therapy including ICU admission, loss of ambulatory function, and blood clots, yet her father continued to frame this as potentially worth the long-term benefit despite nearly breaking his family during the recovery period.
  • The FDA completely shut down communication with Matt Wilsey and Grace Science during the Macari-Prasad leadership era, preventing access to senior leadership despite active engagement from the review team.
  • FDA regulators explicitly told Wilsey not to dose-escalate below 1E15 total VGs despite safety concerns because they believed it was necessary for maximum benefit, but Wilsey refused and the lower dose ultimately proved effective.
  • Wilsey argues that requiring a second manufacturing run for a drug treating 10 patients that demonstrates safety and efficacy represents an outdated 1960s regulatory interpretation rather than appropriate modern standards for ultra-rare diseases.
  • The FDA's RMAT designation for Grace Science's therapy required treating older adolescents (ages 14-16) to facilitate faster development, meaning any improvements in this age group directly attribute to the drug rather than natural disease progression.
  • Wilsey contends that parents of affected children, not just objective observers, should be 'at the head of the table' in regulatory decisions because they uniquely understand that the risk-benefit calculation is risk versus certain death, not risk versus placebo.
  • Gilead's U=U (Undetectable=Untransmittable) movement transformed HIV from a death sentence to a manageable chronic disease while serving a destigmatization function, demonstrating how scientific evidence can simultaneously address both medical outcomes and social discrimination.

Topics

GLP-1 telehealth prescribing practices and patient safety concernsPharmaceutical company partnerships with telemedicine providersNovo Nordisk vs. Eli Lilly advertising lawsuitUltra-rare disease gene therapy developmentRegulatory pathways for N-of-1 and ultra-rare disease treatmentsParent-led drug development and conflicts of interestGene therapy dosing and safety in human trialsFDA regulatory flexibility and communicationHIV treatment destigmatization and U=U movementClinical trial design for rare diseases

Transcript

Welcome to this week's episode of The Read Out Loud, a weekly biotech podcast from STAT. I'm Alison DeAngelis. And I'm Elaine Chen. Adam Forrestein is out this week. It's Thursday, July 23rd, and on this week's episode, I speak with our colleagues Jason Mast and Matthew Herper about their impactful look at one father's mission to develop a treatment for his daughter's ultra-rare disease. The subject of that feature, a man named Matt Wilsey, also joins us to discuss. We also discuss the latest news in life sciences, including a lawsuit between two major pharmaceutical companies and my investigation into a telemedicine provider's questionable GLP-1 prescribing practices. All that's coming after a word from our sponsor. I'm Jesse McWhorters,…

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