Ricki Lewis

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). Available in Italy for three years, Lenmeldy (atidarsagene autotemcel), from Orchard Therapeutics, is groundbreaking, but comes at quite a cost – the $4.25 million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

 

An Ultrarare Neurological Condition

 

MLD affects the white matter in the brain, causing progressive loss of mobility and sensation, as well as intellectual decline and, ultimately, unresponsiveness.

 

To continue reading, go to DNA Science, where this post first appeared.

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It, I predicted that the technology would soon expand well beyond the rare disease world.

 

I was overoptimistic. Gene therapy clearly hasn't had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases. We're still learning possible outcomes of sending millions of altered viruses into a human body. Can they deliver healing genes without triggering an overactive immune response?

 

A report in the September 28, 2023 The New England Journal of Medicine describes a young man with Duchenne Muscular Dystrophy (DMD) who died just days after receiving gene therapy. The details are disturbingly reminiscent of the famous case of Jesse Gelsinger, who died from a ferocious immune response to experimental gene therapy in September 1999.

 

To continue reading, go to DNA Science, where this post first appeared.

The last thing the field of gene therapy needs is another setback. Two studies, not yet peer-reviewed, point to adeno-associated virus (AAV) as a suspect behind the unusual hepatitis that emerged in children in April 2022.

AAV has been critical to the development of gene therapy, as carriers of human genes in the single strand of DNA that is the viral genome.

 

AAV has been considered relatively harmless, as viruses go. It was discovered in 1965 as a tiny tag-along that will only replicate in human cells if adenovirus is also there at some point – hence the "adeno-associated." AAV infection can also accompany certain herpes infections. Several subtypes of AAV have since been identified; AAV2 and AAV9 are gene therapy favorites. And they're common. Eighty percent of us harbor AAV2.

 

To continue reading, go to DNA Science, where this post first appeared. 

I can't believe it's been a decade since I researched and wrote The Forever Fix: Gene Therapy and the Boy Who Saved It. Since then I've shared stories of other families doing amazing things to help researchers develop treatments for their loved ones' rare diseases. The need is all the more compelling in these days of the pandemic.

 

Now entering its fourth decade, gene therapy continues along what seems at times a never-ending rocky road, riding the waves of fantastic success and plunging setbacks.

 

A Slow Start

 

The US has approved just two gene therapies. Luxturna has provided vision to patients with a form of retinal blindness (the basis of The Forever Fix), while Zolgemsa treats spinal muscular atrophy, a disease typically lethal in young children.

 

The latest in a series of setbacks, beginning in 1999 with the death of 18-year-old Jesse Gelsinger, came just yesterday. The FDA placed a clinical hold on two gene therapy trials for sickle cell disease, following reports of blood cancer in two trial participants. But that's not all.

 

To continue reading, go to DNA Science, where this post first appeared.

In late 2011, creation of a lab strain of of H5N1 influenza capable of spreading easily among ferrets, and presumably us, sparked heated debate about whether and when to publish scientific research that could do harm. The same could be said for gene doping.

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