Vertex's New Cystic Fibrosis Drug Treats the Cause, Not Just the Symptoms

Vertex Pharmaceutical (VRTX) shares soared 15% Wednesday as Wall Street cheered the results of a late-stage study of its new cystic fibrosis drug, an experimental treatment that targets the underlying cause of the disease rather than just its symptoms.

In patients with cystic fibrosis, a defective gene and the protein it creates cause the body to produce unusually thick, sticky mucus that builds up in the lungs and the pancreas, leading to life-threatening lung infections and an impairment of the body's ability to properly break down and absorb food.

The drug, VX-770, is a pill that targets the defective protein. It showed sustained improvements in lung function of over 10% in patients with a specific gene mutation. The caveat, though, is that the specific mutation being targeted by the drug, G551D, affects just 4% of the CF population. But it's possible that the results of this trial will provide a basis for the use of VX-770 to treat other mutations. VX-770 is currently being tested in combination with another drug, VX-809, in people with two copies of the more common Delta F508 mutation.

In the trial, patients also showed improvement across all key secondary goals of the study, Vertex said, including decreased pulmonary and respiratory symptoms and weight gain (seven pounds on average). In addition, average sweat chloride (salt) levels, a key clinical indicator of CF, dropped toward normal levels, indicating that the drug is having an effect on the underlying defect.

VX-770 helps "open the gate" at the cell surface -- enabling the protein to pass through, as it would in healthy cells. This restores a proper flow of salt and fluids on the surface of the lung, the company explained.

"Treating the underlying cause of cystic fibrosis with VX-770 led to clinical improvements that were far beyond our expectations, providing support for an entirely new approach to the treatment of this disease," said Peter Mueller, executive VP of global research and development, and chief scientific officer for Vertex.

A Long Time Between Genetic Discovery and New Medicine

"These results are highly encouraging," said Robert Beall, president and CEO of the Cystic Fibrosis Foundation, which collaborated with Vertex on the drug development, including an approximately $75 million investment. "They provide scientific evidence that support our long-standing belief that targeting the underlying defect of CF may have a profound effect on the disease."

Cystic fibrosis is an inherited genetic disease that affects about 30,000 people in the U.S. and 70,000 worldwide, according to the Cystic Fibrosis Foundation. In the 1950s, few children with cystic fibrosis lived to attend elementary school. Today, patients can expect to live into their 30s, 40s and beyond. Still, very few treatments are available to CF patients, and those that are generally target the disease's symptoms.

But while this drug trial is being hailed a victory, it also highlights the problems of targeted therapy, as Mathew Herper of Forbes writes. "Cystic fibrosis is a symbol of how difficult it is to move from genetic discoveries to new medicines. The gene for the disorder, which causes patients' lungs to fill with thick mucus, was discovered in 1989 by Francis Collins, now the director of the National Institutes of Health. Yet treatments have been slow in coming."

Vertex said it will file for U.S. approval of the drug in the second half of this year. Analysts estimate VX-770 could, if approved, generate peak annual sales of some $600 million.

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To all out there I would like to prays this new med and the strives taken to beat this beast of a disease . I would like to extend hope to all you parents out there . I have CF I was diagnosed a 14 months and not expected to see 2 yrs.
I guess I proved them wrong . Here is the hope I extend at this time I just celebrated my 40th birthday on Nov. 11. For the parents out there that listen to and dread all the restictions and complications in life that the doctors tell you your child will not be able to do things or have to be protected from everything I and my mother will tell you It's BULL !!! I'm here to tell you nothing can stop them from doing what they want to with their lives , you will hear doctors contradict this . I live in florida and the Doctors here think the same way until I put them in their place they get frustrated because I prove them wrong at every turn and your children can to. Let them know they can be a Cop,Doctor,Teacher,or Firefighter. I know I've been a Firefighter for over 20+ years and only now between the CF and the abusive environment of the job am I stating to have problems . Let them be strong and free and best wishes and GOD bless them all.

February 26 2011 at 10:51 AM Report abuse rate up rate down Reply

sgard206 If you or your child had CF, you would not be complaining about the cost of the drug! Do you have any idea how much money is spent every month to keep anyone with CF healthy? 75K is spent every six months! In some cases, it is spent every three mohths on drugs & treatments to keep patients healthy!!!
Besides that, the fact that they may be able to use the basis of this drug to treat patients with other strands fo the gene is phenomenol!

February 26 2011 at 9:33 AM Report abuse rate up rate down Reply

I too have 2 daughters with Cystic Fibrosis. My girls were diagnosed at ages 5 & 8. My other children are fine except my youngest son who is a carrier. My girls had all the signs and symptoms. The eight year old had stomach pain all the time and everytime she tried to eat she would stop because as soon as she had the first bite she would begin to hurt. She was always coughing up stuff and clearing her throat. My youngest was constantly having upper respetory infection and would have to go to the hospital. Their explanation was she was suseptable to infection but not knowing why. It took a doctor who had never seen her to recognize the symptoms to get a diagnosis. They are 17 and 20 and doing okay. The 17 yr old did gymnastics which help keep her lungs clear. The 20 yr old did competetive cheerleading for several yrs. She is now in college but is having breathing problems and has to go on enzymes to eat. She also has to have a special vitamin but I can't afford them. They are about 75.oo and my insurance doesn't cover them. They had genetic testing and was to have the same CF gene of which there were only 3 or 4 people had also. Johns Hopkins hopes to identify this gene. I hope this medicine will help them. To all the other parents out there: Good Luck!

February 25 2011 at 8:12 PM Report abuse rate up rate down Reply

We have four beautiful daughters age 16,15,14 and 10. Two of them (the oldest and the youngest) have CF. For us every research and new medication is a victory. We have the hardest race ahead of us - to keep our children healthy until the day comes when they all will be cured. Because we keep the faith that that day would come. It hurts when people question the percentage of people that can be helped or amount of money that spend on research.What is a minimal percentage for you sgard206 is 200% for my family - a 100 % for each of my kids! The CF foundation is very active to raise money for our researches and honestly if we have millions of dollars in the budget for ant research I would not question any amount that would make healthy humans and give future to our children.

February 25 2011 at 7:06 PM Report abuse +1 rate up rate down Reply

I agree tgutie5136. My friend who past away from CF always talked about getting married, and having children and was so optimistic that something would come her way. If this can help even just one person or one child get to do the normal things all of us healthier people take for granted everyday, or one person or one child to have a long lifetime with their family and friends, well then I think this is PRICELESS! I know my friend Stacey is very happy in heaven today smiling on such a great breakthrough in CF research. Keep it coming CFF and Vertex!!

February 25 2011 at 5:17 PM Report abuse +4 rate up rate down Reply

I just had an amniocentisis yesterday to determine if my unborn son has CF and while both my husband and I have the Deltaf508 mutation artlicles like this encourage me. I pray everyday that my son is not affected by this disease but if he does this is just another set of challenges we will face as his parents its good to know some scientists are on our side in this battle.

February 25 2011 at 4:09 PM Report abuse +1 rate up rate down Reply
1 reply to Natasha's comment

my prayers are with you and your family

February 25 2011 at 5:19 PM Report abuse rate up rate down Reply

The article is incorrect. VX-770 does not open a "gate" to allow the PROTEIN to pass through. CFTR is the defective/mutated cell surface protein responsible for Fibrosis. This drug restores a mutated "gate" to functionality, so that CHLORIDE IONS can pass through. While proteins can be ions, the ions involved in cell function in Cystic Fibrosis and any other ion channel defect are all very small, ranging from a single ionized atom to a single ionized amino acid. None of these are anywhere near as large as a protein.

I'm glad there will be a treatment for even one form of Cystic Fibrosis.

February 25 2011 at 3:47 PM Report abuse +2 rate up rate down Reply

sgard206, this breakthrough has been long coming and may lead to treatments for those not compatible with this med. you are a KILL-JOY! Even if it only helped a few patients, it would be worth the money. I hope you never have an illness that needs this kind of research. I am a Breast Cancer survivor and awaiting new treatments that could put a stop to this disease also. We are so close to solving the puzzle of so many illnesses. Take a deep breath and be thankful your apparently healthy and send well wishes to those who aren't. My Grandmothers neighbor had six children in the 50's and they all died of CF. My daughter had a sweat chloride test when she was very young, and was found to have Celiac Disease, but we were very worried it was CF. Good luck to all people fighting any disease that is difficult to overcome.

February 25 2011 at 3:06 PM Report abuse +3 rate up rate down Reply
1 reply to tgutie5136's comment

my prayers are with you

February 25 2011 at 5:19 PM Report abuse rate up rate down Reply

I don't mean to be a kill-joy, as I know people with CF and it's heartbreaking. However, I did a little math. 30,000 people in the U.S. with CF. This drug helps the 4% with the specific gene mutation. That's 1200 people. Then the drug showed improvement in only 10% of that group--120 people. At a $75 million investment, that's $625,000 per person. With research dollars tight and getting tighter (Thank Congress), that seems like a lot of money for relatively few people helped. Surely, there are other avenues of CF research that would help more.

February 25 2011 at 2:48 PM Report abuse -3 rate up rate down Reply
1 reply to sgard206's comment

sgard206, read it more carefully. It had a 10% LUNG IMPROVEMENT in those 4% who had the gene mutation. Rome wasn't built in a day. This baby step helps 4% of CF patients today, and is another piece of the puzzle. Are you saying that they should stop trying to solve the puzzle because it is too expensive?? Ludicrous!!

February 25 2011 at 3:36 PM Report abuse +2 rate up rate down Reply

This is such great news. I have only known one person with this in my life, a child just diagnosed. Such a sad thing to be told, your 8 year old son has Cystic Fibrosis. I am so glad to think he is probably still alive at 26, and that a new treatment might even add to his life span. There is a God he heard my prayers.

February 25 2011 at 2:20 PM Report abuse +5 rate up rate down Reply