'I was scared, my belly felt funny and my brain felt weird.'
This episode of the Pulse from NPR opens with the fragile cadence of a young, innocent voice, instantly setting the tone for the podcast entitled 'How Gene Therapy is Offering Hope – Once Again'.
As I leaned in attentively, the host introduces Shelby Campbell, an eight-year-old from Philadelphia suffering from a rare blood disorder called beta-thalassemia. Campbell's voice resonates with vulnerability as she describes her experience following life-changing US Food and Drug Administration-approved gene therapy treatment in June 2023. I use the adjective 'life-changing' genuinely and not for effect, as Campbell's life has transformed from requiring blood iron level management for 12 hours every night and blood transfusions every two-to-three weeks, to not requiring a transfusion or even any blood supplements since the therapy, which is 65 days at the time of the podcast.
Early on in the podcast, gene therapies emerge as the 'golden goose' in today's medicine.
Spanning from blood disorders to alcohol addiction, cancer and even homemade gene therapies for lactose intolerance, the 55-minute podcast offers a handful of concise and easily digestible stories encapsulating the past and present hurdles of gene therapies. As a researcher developing gene therapies for neurological diseases, the podcast pleasantly surprised me with its commitment to integrity and honesty, evident in every segment, offering a nuanced exploration of the world of gene therapies.
After the story of Campbell's gene therapy success is drawn to a close, the narrative takes a somber turn accompanied by moody music. The host explains the first arrival of gene therapies in 1998, recounting the tragic story of Jessie Gelsinger. Merely four days after receiving a gene therapy at the University of Pennsylvania for a metabolic disorder, Gelsinger experienced a fatal inflammatory response. This not only shattered his family's world, but sent 'shockwaves' through the entire field, bringing progress to an almost complete standstill. From this sobering point, the podcast embarks on the uphill journey that gene therapies undertook to regain trust.
The stories alternate between patient or researcher point of view which sustained my engagement throughout the entire 55 minutes. This proves instrumental in providing a balanced and informative story. Throughout the podcast scientific jargon is broken down, often through the use of metaphors. This not only adds an element of entertainment but offers light relief for non-science listeners. A standout moment for me occurred during the discussion of CAR T-cell therapy for leukaemia, which is a therapy that uses the patient's own immune cells and modifies them to improve their cancer-killing abilities. The podcast host whimsically likens the large CAR T-cell manufacturing machinery to a fancy espresso machine. In response, Dr Stephan Kadauke, assistant director of the cell and gene therapy laboratory at Children's Hospital of Philadelphia, proceeds to link the different functions of the CAR T-cell factory to those within a coffee machine. This creates a vivid mental image of a steaming coffee maker ingeniously spewing out engineered patient cells.
The challenges from each story roll into one another, connecting each isolated gene therapy anecdote into an intertwined web of obstacles. A prominent and recurring theme revolves around patient access and the hefty commercial price tag of these therapies. The $3.2 million hold the Duchenne muscular dystrophy gene therapy (see BioNews 1196) had over the life of six-year-old Cash Huber from Delaware strongly resonated with me, on a professional and personal level. If science and medicine is advancing at a rate where it can significantly manage, or even cure, debilitating diseases, then we also need to advance the access and lower the financial boundaries to enable use within the clinic.
The podcast takes a surprising turn when the story of Justin Atkin is told – a trained biologist who crafted and administered his own gene therapy out of desperation to alleviate his debilitating lactose intolerance. Is Atkin among the likes of Albert Hofmann and Werner Fossmann – scientists known for self-experimentation in the name of research? While the story holds an undeniable entertainment factor, there's an underlying need for a *do not try at home* disclaimer. The DIY clinical trial approach will likely appall most researchers. This trial is marked by eating 'the most milk-tastic, cheesey, monstrosity of a pizza we could find' all under the measuring parameters of 'if this doesn't hurt him, nothing will!' Despite the apparent risks, it has prompted me to follow up this unconventional journey with Atkin's video documentary.
I would recommend this podcast as an insightful snapshot of the gene therapy landscape, suitable for all audiences. If you can set aside the monotone, unengaging voice of the podcast host, the podcast demonstrates how gene therapies are offering hope again, while acknowledging the ongoing work that lies ahead. As we turn to mainstream news sources, it seems likely we will see an increasing amount of gene therapy successes in the next coming years.
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