Our dad has ALS; the Senate could help by reforming FDA drug approval

Originally published by Aiden Cimbura and Ellie Cimbura for The Denver Post

Twenty months ago our dad was diagnosed with ALS. Once very athletic, he can no longer dress himself, brush his teeth, breathe without the use of a machine, speak without the help of a communication device, shower on his own, eat a meal, walk, give us hugs or high-fives, or tell us he’s proud of us or that he loves us in his own voice. Our dad can no longer work and our mom is a schoolteacher who doesn’t make a whole lot, so this is a huge expense that is really hard for our family to afford.

ALS, short for amyotrophic lateral sclerosis, is a progressively degenerative disease that affects nerve cells in the brain and the spinal cord. Its sufferers gradually lose the ability to control voluntary functions, like taking a walk, or just using a phone.

There is no cure for ALS and it is fatal 100 percent of the time.

There are promising new treatments in clinical trials in the U.S. and drugs being safely used around the world, and our dad needs access to them now. We can’t wait any longer.

Our dad was fortunate to be one of the lucky 48 patients chosen to participate in BrainStorm’s NurOwn stem-cell trial. He saw immediate improvement that lasted for months, but then the gains began to fade. He needs more doses of the treatment, which we immediately asked for, but we were told no because it was just a trial. In Israel, where the treatment is already approved, patients are allowed multiple doses. The U.S. trial ended 18 months ago and the favorable data for all participants has been given to the Food and Drug Administration. All we can do is sit and wait.

There’s a drug called Radicut/Edaravone that was approved in Japan for ALS two years ago. They have 10-plus years of data in their trials, and it’s safe, effective, and now being used also in South Korea. The company has applied for approval with the FDA, but we won’t get an answer until June 2017.

We lose more than 500 Americans each month to this disease; so why is the FDA waiting? There are promising drugs and therapies in the FDA queue, but the patients who need them most can’t get to them. The FDA system isn’t working like it should.

Our U.S. senators will have the chance to give us the hope we need while they are in the lame-duck session. They will be asked to support Senate Bill 2912, or the Trickett Wendler Right to Try Act. This bill gives people with terminal diseases the opportunity to try drugs and therapies that have passed phase 1 in the FDA’s trials. These are the drugs that are determined safe and are already showing promise as they move onto the next phase. Traditionally it takes 12-15 years and more than $1 billion to make it through phase 3 of the FDA trial, but terminally ill patients like our dad don’t have that long to wait.

We really hope Colorado Sens. Michael Bennet and Cory Gardner will see the urgency, support this law and also push the FDA to approve drugs for all terminally ill patients more quickly. Our dad is dying and this is nothing kids should have to watch. Sadly, there are many more kids like us who do have to watch a parent suffer from this horrible disease. You can help to end it — just take action.

Ellie and Aiden Cimbura live in Highlands Ranch.