Gene therapy is coming. Indeed, it's already in Dallas.
Millions of North Texas-area patients with previously intractable diseases will benefit from gene therapies' medical miracles. That's in part because UT Southwestern Medical Center is playing a prominent role in gene-therapy development.
Boston-based Vertex Pharmaceuticals recently announced it is merging with Exonics Therapeutics, a biotech company co-founded by Dr. Eric Olsen, who chairs UT Southwestern's molecular biology department.
Gene therapy targets defective or missing genes and repairs or replaces them. The most common method is by using viral vectors, that is, modified viruses that insert new genetic code into defective human cells. UT Southwestern's gene therapy center is one of a small number of such centers that produces the virus that transports genes to the defective cells.
Exonics will focus on Duchenne muscular dystrophy, which leads to muscular degeneration and, along with Becker muscular dystrophy, affects 1 in every 5,600 to 7,700 males ages 5 to 24, according to the National Institutes of Health.
But there's even more good news. The U.S. Food and Drug Administration has just approved a gene therapy treatment for spinal muscular atrophy (SMA) in children. Also known as floppy baby syndrome, SMA is a genetic disorder that affects the nerves and causes a progressive loss of muscle function. It affects between 10,000 and 25,000 U.S. children and adults, according to the SMA Foundation, and often proves fatal for infants before their second birthday.
The new treatment is Zolgensma, made by Swiss drug company Novartis and AveXis, a unit of Novartis. The innovative, one-time treatment appears to be a miracle cure for most of the SMA patients who participated in clinical trials. But it's expensive: $2.1 million.
And last year, the U.S. Food and Drug Administration approved Luxturna, which is used to treat inherited sight loss from retinal diseases. The virus infects the retina and produces a normal protein that restores eyesight. It costs about $850,000 a patient.
When considering cost versus value, there are several points to keep in mind.
First, gene and cell therapies are in their very early stages. "First adopters" of new technology generally pay a much higher price, whether we're talking about TVs, computers, robots, medical devices or medical treatments. Time, economies of scale and competition put downward pressure on those prices.
For example, Zolgensma's primary competitor is Spinraza, sold by Biogen, which requires an annual spinal infusion that costs $750,000 the first year and $375,000 every year thereafter for life. Thus, while Zolgensma's $2.1 million price tag may seem like more, patients will be paying more for Spinraza after five years.
In addition, comparing gene therapy to typical prescription drugs is misleading. A better comparison would be to surgery.
Pediatric heart transplants can run upwards of $2 million, and the child may continue to take anti-rejection and other medications for life — not to mention a likely second heart transplant later.
By contrast, gene therapies are minimally invasive and may actually solve the problem by directly countering the faulty DNA that caused the condition.
Pharmaceutical companies are well aware that prices can be a challenge for many patients, although it's important to remember that insurance generally covers the bulk of these costs.
Importantly, private insurers are looking for creative new ways to pay for these miracle cures. For example, some companies are discussing ways to correlate a treatment's price with its outcome. Patients who benefit would pay, those who don't would pay little or nothing.
Developing these remarkable cures is the first step, finding a way to pay for them will be the second.
And we do need to find innovative ways to pay for them. Because we want innovators to press on with their research and development.
Gene therapies may eventually be able to cure a host of debilitating and deadly diseases, such as cancer, Alzheimer's and arthritis. It likely won't be cheap, but miracles often aren't.