LOS ANGELES (CBSLA.com)  —   Medical breakthroughs — seemingly every day — are giving unimaginable hope to people suffering with once incurable diseases.

But the treatments are cost prohibitive — and that would be a major understatement. The question facing the medical community — what are these drugs worth and who ought to pay for them?

Moreover, what to do when a patient’s only hope is one of the most expensive drugs ever made .

CBS 2’s Pat Harvey profiles one man and an infant facing just those questions.

Adam expects to start treatment this summer. He and his wife say they will continue to appeal to their insurance company even after being denied.

Adam Beyers has Spinal Muscular Atrophy. He is being treated by Perry Shieh, a UCLA neurologist.

Ethan Cheng is 5-months-old. He’s a medical miracle. In December, he was too weak to cry.

His parents told Harvey he was too weak to move his arms or legs.  Because of a missing gene, his brain lost the ability to send messages to his muscles.

“It’s thought of as an untreatable disease,” says Dr. Shieh, “it’s very devastating.”

At least that’s how the medical community viewed it up until a few short months ago.

A drug recently approved by the FDA is giving people with the disease hope.

In February, Ethan got an injection of Spinraza,

Beyers wants a literal shot, too.

“I’ve been waiting for this drug for 28 years,” Adam says.

He has trouble moving, but he is grateful for his great house, wonderful wife, good job, his dogs.

But the couple also has challenges. With every new day, it gets harder for him to move.

“I will have to hire someone, a health aide, to help me do basic things,” he says.

His doctor asked his insurance company if they would cover the expense or vastly lower the cost of the Spinraza.

“They are standing firm that they are not going to cover it for me,” says Adam.

Those it’s approved for use by all ages, the insurance company says for someone his age it is investigational for him because he wasn’t diagnosed before 6-months of age.

And complicating the situation, the drug’s price — $750,000 for the first year of treatment and then $375,000 a year for the rest of his life.

“I think we all worry about the high cost of health care. But this is a very innovative drug,” says Shieh.

The drug’s company, Biogen, justifies the price saying it’s based on “clinical value, impact to the healthcare system and funding of research and development.”

The company provided the first doses for little Ethan and has agreed to cover the first few doses for Adam, as well.

But if Anthem doesn’t eventually come around, he can’t be sure he will be able to stay on it.

“Without access to this drug,” he says, matter of fact, “I worry that I have an expiration date.”


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