Our fa­ther has ALS; here’s how the U.S. Se­nate can help

The Denver Post - - OPINION - By El­lie Cim­bura and Ai­den Cim­bura El­lie and Ai­den Cim­bura live in High­lands Ranch.

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 high­fives, 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 lame­duck 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.

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