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That's why it is more important now than ever before to raise
as much money as we can! It's like racing against a ticking clock!
The Cystic Fibrosis Foundation has built a dynamic "pipeline" for the development of more new
potential CF therapies than ever before. To treat a complex disease like cystic fibrosis (CF), therapies must target problems
in the airways and the digestive system. In the CF pipeline, there are also promising new therapies designed to rectify the
cause of CF—a faulty gene and/or its faulty protein product.
Denufosol (Inspire Pharmaceuticals, Inc.) –
Denufosol is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect
of drug on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed
enrollment. The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.
My daughter may be participating in the phase 3 trial for this
one! It is so extremely promising and will be starting over the next couple of months! Keep your fingers crossed!!!
GENE THERAPY
Because a faulty gene causes cystic fibrosis (CF), adding normal copies of the gene to cells should correct
these cells and ultimately cure the disease.
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Compacted DNA (Copernicus Therapeutics) – Non-viral
compacted DNA (PLASmin™) is being used 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.
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PROTEIN ASSIST/REPAIR
This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow
chloride and sodium (salt) to move properly in cells lining the lungs and other organs.
- PTC 124 (PTC Therapeutics) – PTC 124 is a novel, small molecule compound, that promotes
the read-through of premature truncation codons in the CFTR mRNA. 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.
- VX-770 (Vertex Pharmaceuticals) – VX-770 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
patients. Researchers are evaluating results and a Phase 2 trial has begun in CF patients.
- Curcumin (Seer Pharmaceuticals) – Curcumin is seeking to correct
the abnormal processing of CFTR in the cell. It appears to work in some strains of CF mice but not all. A previous Phase 1
trial in CF patients did not show correction of CFTR. A follow-on study at a higher dose is ongoing to confirm these findings.
- GSNO (Nitrox, LLC) – S-Nitrosoglutathione (GSNO) levels are low in
the lungs of CF patients. GSNO has been shown to promote trafficking of the Delta-F508 CFTR in some, but not all, tests on
cultured cells. Nitrox LLC is developing inhaled GSNO for CF and is presently carrying out required preclinical safety studies,
as well as formulation studies.
- VX-809 (Vertex Pharmaceuticals) – VX-809 is a "corrector" that helps
to move the defective CFTR protein to the proper place in the airway cell membrane and to improve its function as a chloride
channel. A Phase 1 dosing trial has begun in healthy volunteers.
RESTORE SALT TRANSPORT
The goal of this approach is to hydrate thick CF mucus in the lungs by correcting the amount of salt (sodium
& chloride) along the cell surface.
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Denufosol (Inspire Pharmaceuticals, Inc.) – Denufosol
is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect of drug
on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed enrollment.
The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.
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SPI-8811 (Sucampo Pharmaceuticals, Inc.) – This oral
agent is believed to bypass transport defect of chloride ions. Initial Phase 2a trial evaluating safety and efficacy. Thirty
patients have been recruited.
- Moli (Lantibio, Inc.) – Moli is believed to affect the
ion transport defect in CF patients. A Phase 1 trial has demonstrated safety and a placebo-controlled, multi-dose, dose-ranging
Phase 2 trial in Europe demonstrated positive changes in pulmonary function with highest dose.
- Gilead GS9411 (now Gilead Sciences; formerly Parion ) –
An earlier compound (Parion 552) has been used to develop proof-of-concept. Gilead GS9411 acts by blocking sodium absorption.
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ANTI-INFECTIVE
The following drugs are being studied for their effectiveness in fighting lung infections for people with
CF.
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TOBI (Novartis Pharmaceuticals) – This CFF/Children’s
Hospital, Seattle-developed aerosol antibiotic was licensed to Chiron and received FDA approval in 1998. Currently being used
by more than 15,000 patients worldwide. The benefit at first sign(s) of Pseudomonas infection is being evaluated in a Phase
4 EPIC study which began in the 4th quarter of 2004.
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Azithromycin (Pfizer, Inc.) – In patients with chronic
PA, this oral antibiotic improved lung function and weight gain, and decreased hospitalization rate. A large-scale trial was
completed in 2002. Two follow up studies are in progress.
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Gilead AZLI (Gilead Sciences) – Multiple Phase 3 trials
of the aerosolized form of aztreonam, a widely used IV antibiotic in CF, have been completed and the FDA is reviewing all
data. This antiobiotic may be ready for market in 2008.
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TIP (TOBI Inhaled Powder) (Novartis Pharmaceuticals) –
Novartis is developing TOBI as a powder to enable a faster, more convenient dosing regimen. Dosing of TIP will take a fraction
of the time of liquid TOBI. A Phase 3 trial began in 2006.
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Bayer-Inhaled Ciprofloxacin (Bayer Schering Pharma) –
An inhaled version of the antibiotic ciprofloxacin is being developed for treatment of airway infections. A small Phase 2
study in Germany is underway and trials in the United States are planned.
- SLIT-amikacin (Transave, Inc.) – This is a liposomal
formulation of the antibiotic amikacin. Animal model studies have shown it to decrease the Pseudomonas aeruginosa
burden in the lung. A Phase 1/2 trial in Europe has completed enrollment. A Phase 2 trial began in the Foundation's Therapeutics
Development Network in 2007.
- Mpex MP-376 – MP-376 is a new formulation of levofloxacin
being developed for aerosol administration to CF patients for management of chronic pulmonary infections due to Pseudomonas
aeruginosa and other bacteria. A Phase 2 trial is scheduled to begin in 2008.
- Kalobios KB001 (Kalobios Pharmaceuticals) – A Phase
I clinical trial has been initiated to test the safety of this antibody approach for the treatment of Pseudomonas aeruginosa
lung infections.
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Pseudomonas Vaccines – Several companies are in preclinical
development of pseudomonas vaccines. The development of Berna Biotech’s product, which was in Phase 2 testing, has been
halted due to lack of efficacy.
There are still many others, too! The thing is, none of these things can help if there is not
enough money to research and produce them!
I won't stop raising money until the cure is here! |
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