When Is a Drug Worth a Fortune

When is a drug worth more than one hundred forty-five thousand dollars a year? For some, if it can prolong their lives. The new drug, Edaravone is only one of two drugs approved to treat amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig's disease.

ALS is a progressive neurological disorder and is fatal within three to five years after diagnosis. It affects the motor neurons, which are nerve cells that control the voluntary muscles we use to chew, walk, breathe, and talk. In ALS, these motor neurons die and stop sending signals to muscles. Eventually these muscles waste away and people die from not being able to breathe. What makes ALS tragic is that the person is aware because they do not lose any of their higher brain function.

Until Edaravone was recently approved by the FDA, more than twenty years had passed without any significant new treatments for the disease. The federal agency had asked the drug's Japanese manufacturer to apply for approval in the US when it saw the effectiveness of the drug. In the clinical trial in Japan, Edaravone significantly reduced the progression of ALS in patients. It's intravenously infused daily for fourteen days followed by fourteen days of no medication. This cycle is repeated monthly.

Edaravone may be counteracting something called oxidative stress, which may be involved in the death of neurons in ALS. Oxidative stress is generated when cells metabolize and if not eliminated can damage and kill cells. Even though studies show the drug didn't benefit people in late stages of ALS, it worked best on those diagnosed within two years.