New Hope for Cystic Fibrosis Patients

On October 18, 2010, the Cystic Fibrosis Foundation announced the beginning of a drug trial that I have been waiting for. It marked the start of what all of my fundraising efforts have been for – the two drugs that could give Maggie a normal, healthier lifespan. These two drugs could mean that no parent has to think about burying their child due to CF again. They could mean that people with CF will die with it, not from it. This could be the beginning of the end for CF.

Vertex Pharmaceuticals has been in partnership with the CF Foundation to find better treatments and a cure for CF. The amount of money it will take to make this happen is astronomical and this latest trial has been years in the making. But here we are. VX-770 and VX-809 are two oral drugs that target the underlying cause of CF – defective or missing proteins in cells. Until now, the drugs have only been tested separately, but it is thought that the two drugs together will bring great benefit to people who have the most common CF mutation, Delta F508. 50% of people with CF have two copies of this mutation. Nearly 90% of people with CF have at least one copy. So it stands to reason that if these drugs work, thousands of lives will be saved.

Maggie has two copies of the Delta F508 mutation, one from me and one from my husband. You must have two mutations to have CF. Delta F508 is considered a class 2 mutation, class 1 being thought of as presenting the worst symptoms. Everyone remains hopeful, but this is a cloud constantly over your head. Every move you make is one to protect your child’s life as you watch 20-year-olds with CF die or suffer while waiting for a lung transplant that may only buy them two more years. The start of this drug trial means the beginning of new hope.

The frustrating part is that it will still be years before these drugs are proven safe and effective. In that time young lives will continue to be lost to CF. But if we keep our hopes up and are fundraising efforts strong, and we continue to take every precaution to keep our children healthy, we could finally see our dreams realized. We could finally be on the other side with CF in the rearview mirror.

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About Nancy

I am a freelance writer focused on parenting children with special needs. My articles have been featured in numerous parenting publications and on www.parentingspecialneeds.org. I am the former editor and publisher of Vermont HomeStyle Magazine. I am a wife and mom to a two daughters, one with cystic fibrosis and one who is a carrier for cystic fibrosis.