Thursday, July 22, 2010

Pill head

Drew takes over 300 pills a month. Thats of 1 medication - Creon. We will go through 150ml of Prevacid, 90 vials of Albuterol, 30 vials of Pulmozyme, 30ml of Vitamax, 6ml of Iron, and 150ml of Sodium in the next 30 days. We have also been on 2 different antibiotics, steroids, and inhaled saline in the past 3 months. And it doesn't look like its going to get any better. I know he needs all of these, but its still a little scary to think of someone so little being on so much medicine.

I wish he didn't need any of this. There is some hope with 2 new drugs and a gene therapy currently in the pipeline with the CFF. I think I wrote about this before, but I'll talk about it again. Here's a little bit of info from the CFF website on these two promising treatments.

GENE THERAPY (This would be a dream come true!!!)

Because a faulty gene causes cystic fibrosis (CF), adding normal copies of the gene to cells could correct these cells and ultimately cure the disease. This approach is exploring ways to introduce normal copies of the gene into CF airways.

  • Compacted DNA (PLASmin™): Copernicus Therapeutics, supported by a CFFT TDA and the TDN. Using compacted DNA (non-viral) to introduce normal copies of the gene into CF airways. A Phase 1a trial demonstrated chloride current changes in the noses of CF patients, but no evidence of gene expression. The gene therapy product is being reformulated prior to additional clinical trials in an attempt to improve the amount and duration of gene expression.

CFTR MODULATION (CFTR - Cystic Fibrosis Transmembrane Conductance Regulator)

(This is a little further down the pipeline and has been showing promising results in the clinical trials. This would essentially turn CF from a life-shortening disease to a chronic disease, like asthma)

These therapies are designed to correct the function of the defective CFTR protein made by the CF gene, allowing chloride and sodium (salt) to move properly in and out of cells lining the lungs and other organs. (no more thick mucus = no more nasty bacteria!)

  • VX-770: Vertex Pharmaceuticals – VX-770 is supported by CFFT and is a new compound called a "potentiator" that may act upon the CFTR protein and help to open the chloride channel in CF cells. Phase 1 dosing has been completed in healthy volunteers and CF patients. A Phase 2 trial in CF patients with at least one copy of the G551D mutation in their CF gene demonstrated improvements in biological measures of CFTR function (nasal potential difference and sweat chloride) and clinical measures of pulmonary health (FEV1). Two Phase 3 studies (one for pediatric and one for adolescent/adult patients) began in Summer 2009.

  • Ataluren (formerly known as PTC124): PTC Therapeutics – A novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It aims to treat CF patients who have what is known as a "nonsense mutation." It has been demonstrated to be safe, orally available and well tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results. The Phase 3 trial began in Summer 2009, which is a 48-week study of ataluren in people with CF age 6 and older. The main goal of the trial is to find out if ataluren can improve lung function in people with the disease.

  • VX-809: Vertex Pharmaceuticals – VX-809 is supported by CFFT and is a new compound called a “corrector” that helps move the defective CFTR protein to the proper place in the airway cell membrane and improve its function as a chloride channel. A Phase 2a trial began in Spring 2009.
I've spent the past few days on the phone, with I can't tell you how many different people, trying to get all of the drugs we need. Insurance doesn't want to cover some of them, and others are only available to us through mail order at this point. I'll fight the good fight with these people and get what we need. Its just been on my mind so I thought I'd share.

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