Man diagnosed with ALS gets new treatment

Squeezing by cameras and reporters in the cramped Allegheny General Hospital exam room, the nurse reached to get her hands on a box she had spent months waiting for.

“This is the gold,” said Carol Chieffe as she opened the box, revealing two bags of clear liquid. As she examined the instructions and prepared an IV, ALS patient Rene Fogarty sat waiting for the needle prick.

Mr. Fogarty, 51, of Turtle Creek was diagnosed with amyotrophic lateral sclerosis in May. Since January, he has been undergoing tests and trials. Doctors have explained how the progressive neurodegenerative disease causes its victims to lose the ability to move and control muscles and eventually leads to paralysis and death.

But on Friday he was the first patient to receive edaravone — branded as Radicava — a new drug aimed at fighting the disease and which could prolong his life. The drug was approved for use in the United States in May by the U.S. Food and Drug Administration.

Edaravone was tested in a small clinical trial in Japan. In the sixmonth study of 137 participants, some received the drug and some were given a placebo. “At Week 24, individuals receiving edaravone

declined less on a clinical assessment of daily functioning compared to those receiving a placebo,” according to a statement from the FDA.

The hope is that it can increase the lifespan and quality of life of ALS patients.

“It’s taken a lot for me not to cry,” said Ms. Chieffe on Friday morning. It’s the first glimpse of hope she’s seen since 1995, she said, when the FDA approved Riluzole, a drug that reduces the effects of the disease long enough to buy patients three to six months.

The Centers for Disease Control estimate that 12,000 to 15,000 people in the U.S. live with the disease — often known as Lou Gehrig’s disease — with 5,000 to 6,000 diagnosed every year. The majority die within two to five years of diagnosis. But Edaravone has demonstrated it can slow the decline of physical deterioration in ALS patients by 33 percent.

Nurses and doctors told Mr. Fogarty on Thursday that the new drug had arrived in the United States and he could begin treatment. He jumped at the idea.

“Why not?” he said. “I’m doing it for all those out there that have yet to be diagnosed. Whatever good experience and good data scientists can take out of this, that’s my main motive.”

For 14 days Mr. Fogarty will sit in a chair for an hour and receive the intravenous treatment. After that he will be drug-free for 14 days, and then begin a cycle of 10 treatments out of 14 days, and 14 days drug-free. He will repeat this for the rest of his life.

Edaravone is not a cure, said Merritt Spier, executive director of the ALS association’s Western Pennsylvania chapter. And there are other hurdles before the drug— which seems to be effective only on early-diagnosed patients — can be administered to more people. Just one infusion costs more than $1,000 and with the treatment continuing for the rest of the patient’s life, it could add up to more than $140,000every year.

Another issue is insurance coverage. Many companies have yet to include the treatment in their plans, because the drug hit America’s shores only on Aug. 1. Also, because the drug works only on patients who were recently diagnosed, the treatment will be at the physician’s — and the insurance company’s— discretion.

“We’re proceeding with cautious optimism,” Ms. Spier said. “There are currently several research projects and drug trials coming down the pipeline. [Edaravone] is the first of several to come, and it may be that the treatment of ALS or the cure for ALS will be a cocktail of drugs, not just one.”

Research for treatments was slow before 2014, when the Ice Bucket Challenge — a social media campaign in which people soak themselves in ice-cold water, donate to the ALS Association and challenge others to do the same — raised $115 million. Ms. Spier said the money has allowed the organization to put more into research, more than doubling commitments from $7 million to $17 million.

The large cost of bringing a drug to market in the United States still makes things difficult, Ms. Spier said, so fundraising efforts cannot stop.

“We’re not done,” she said “We could use about 20 more ice bucket challenges.”

For Linda Fogarty, Mr. Fogarty’s wife, the process has been exhausting. She clutched at her husband’s arm throughout the treatment, every once in a while reaching for a tissue as tears of joy streamed down her face.

“We’ve been wanting to do this treatment for such a long time and we were just waiting and waiting for August to come,” she said. “We are very happy.”