Welcome to the Age of One-Shot Miracle Cures That Can Cost Millions

Kristin Simpson with her son, Omarion Jordan, whose gene therapy treatment Bloomberg | James Alexander Michie

Kristin Simpson with her son, Omarion Jordan, whose gene therapy treatment has allowed him to leave hospital isolation. PHOTOGRAPHER: TAMARA REYNOLDS FOR BLOOMBERG BUSINESSWEEK

Gene therapy seems to successfully treat patients who were once-incurable as patients like “bubble boy” Omarion Jordan. But progress like this is not cheap. As a result, we welcome the miraculous healing of Age of One-Shot which can cost millions.

Omarion Jordan spent most of his first year of life in hospital isolation rooms. The nightmare began with what seemed at first a diaper rash, a chain of red marks that quickly spread through his body when he was just 3 months old. The creams and ointments failed, as did the treatment with eczema shampoo prescribed by a doctor in the emergency room. Last July, hours after Omarion’s pediatrician injected the three-month shots into his thighs, the boy’s scalp began to weep green pus that hardened and peeled away, taking away his faint brown curls. His head remained crunchy, cracked and bleeding, and his mother, Kristin Simpson, began to panic. “Their screams sounded terrible”, she recalls. “I thought I was going to lose it”.

Disorder with alternative

It should be noted that it was a battery of tests that revealed a rare genetic disorder called severe combined immunodeficiency syndrome (SCID), better known as the “bubble boy” disease, which causes between 40 and 100 newly born in the United States are extremely vulnerable to infections. Omarion, transferred to an Ohio hospital three hours away, was confined to an isolation room with special air filters.

If left untreated, SCID kills most children before they turn 2 years old. Even so, there is the talk of an alternative: an experimental gene therapy that could cure in an absolute way.

There is no doubt that this is the tempting promise of genetic therapies, the potential cures for dozens of diseases that were previously incurable. The US Food and Drug Administration issued his first approval of systemic gene therapy, a Novartis AG treatment for spinal muscular atrophy, on May 24 and says he expects to approve 10 to 20 therapies a year starting in 2025. There are more than 800 trials. Likewise, in course they are directed to diseases that include rare metabolic disorders, sickle cell anemia, hemophilia and Parkinson’s. As the list grows, such treatments have the potential to fundamentally remake the health care system at all levels.

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Source: Michelle Cortez | Bloomberg

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