Drugs in line


Although there is no apparent cure for CF there is a fair amount of research going towards finding a maintenance drug.
The recent arrival of Kalydeco from Vertex Pharma into market  is giving good hope to CFers all over the world.Kalydeco- Ivacaftor : Kalydeco is the only drug in market for Class  3 and 4 gating and residual Mutations produced by Vertex Pharm..  The missing amino acid glycine at position 551 is replaced by aspartic acid. This is being termed as a miracle drug by CFer around. But miracles are costly.

Lumacaftor (Vx-809): Lumacaftor is yet an other product for Class  2  (mainly for DDf508 or Df508 )transfer and trafficking mutation which on in-vitro and in in  – vivo  is showing good results. Clinical trials phase III have been a success and is expected to go for drug approvals end of year(2014). It might hit the market anytime next year 2015. http://www.reuters.com/article/2014/05/02/vertex-idUSL2N0NN1TX20140502. Lumacaftor in combination with kalydeco is the next wonder drug. Check out the below figure to understand what Lumacaftor can make.


VX – 661 : Another next generation corrector from vertex aiming the most common mutation homozygous and heterozygous DDF508.

N30 Pharma : With their amazing products  such as N91115 , Lead compound and N6022, N30pharma  are talking about Lung repair CFTR  correction and and Anti- inflammation. Their site is a must browse for on going events. http://www.n30pharma.com/.
Their new press release states about discovery of a efficacious CFTR corrector. Interesting part of it is that Ramakrishna B an Indian Origin
is part of the team.

Gene Therapy :  CF community has lots of hopes pinned on to Gene Therapy which involves correcting the faulty gene by replacing it with therapeutic one. The carriers for the therapeutic DNA are called vectors. A full fledged research is on, to find out a best vector, viral or non viral.

PTC : PTC therapatics aims at Class I CF mutations, Non Sensical muations. Their Product  Atalauren is now going through Phase 3 clinical trials . This product can be used for many other problems caused by non sensical mutation.  Find more details at http://ir.ptcbio.com/releasedetail.cfm ?ReleaseID=848453

Advanced Inhalation Therapies (AIT) : The Product NOxCureCF from  AIT targets the Pseudomonas and the Staphylococcus aureus bacteria, using a specialized form  of inhaled Nitric Oxide (NO) . Relief from Antibiotics is definitely a news worth mention.

Pulmozyme Dornase Alfa: Genentech which manufactures Pumozyme from Chinese Hamster Ovary. Pulmzyme over years has shown great results and is marketed by Roche Pharamceticals