New drug a possible breath of fresh air for CF sufferers
More than two decades after discovering the gene responsible for cystic fibrosis (CF), a cure is knocking on the door. Now, as hope looms, those involved with the Holmes County Great Strides Walk-a-thon, which will be held Sunday, May 15, believe that it is time to support scientific research more than ever.
In a bit of thrilling news, Vertex Pharmaceuticals announced recently that the drug, VX-770, improved lung function in people with cystic fibrosis in a late-stage clinical trial. The drug also reduced the frequency of disease exacerbations that required treatment with antibiotics.
While the news is wonderful for those suffering from CF, VX-770 is designed to improve one of the lesser CF mutations, one which only affects about four percent of CF sufferers. However, according to Erica Matisz, Vertex is working on another drug that could have a direct bearing on the most common CF mutation. However, that one is further behind in its development, but it does speak volumes as to how far CF research has come over recent years.
“We’ve experienced some amazing results,” said Tony Mullet, Holmes County CF coordinator, whose daughter Michaela suffers from CF. “The new application would eventually affect 90 percent of the CF population. If we can crack one, we can crack the whole thing. The impact would be huge. It may be a few years off, but the strides we are making are incredible.”
About 30,000 Americans and 70,000 people worldwide have cystic fibrosis, a disease caused by defects in a gene responsible for the transport of chloride ions across cell membranes. People with the disease tend to have very thick mucus in their lungs, which leads to infections and lung damage. Many do not live past age 40, which is decades older than it was 25 years ago.
While the excitement of a solution to CF is on the horizon, cures don’t just materialize. It takes a long time to develop a drug for cystic fibrosis. It can take years of research to determine how a mutation actually causes a disease and then to design a drug that corrects the problem. Thus the expense of research is great.
Not surprisingly, Vertex received $75 million in financial support from the Cystic Fibrosis Foundation, one of the first nonprofit disease groups to give money to companies, since CF gets no federal aid in its fundraising efforts.
“These results are highly encouraging,” said Matisz. “This is the closest we’ve come yet to finally figuring this thing out. We obviously don’t want to put all of our eggs into one basket, and there are about 30 other drugs out there that are continuing to be tested, but it’s exciting. This could have an effect on tens of thousands of people.
“All of the money that CF walks like Holmes County’s and many others has gone a long way in creating the technology to make this happen.”
“It’s the fruits of our labor,” added Mullet. “We don’t know exactly what is going to happen, but we are extremely thrilled with the direction we’re headed.”
Over the years, Holmes County Great Strides has been instrumental in raising hundreds of thousands of dollars for CF research. The annual walk sees local teams made up of caring families, businesses and individuals who want to help generate funds for research take up the cause by walking a 5-K, during an afternoon of fun, food and fundraising. The group has set a goal of $44,000 this year.
“We have had so many wonderful people get behind this and join in on our efforts to raise money for CF research,” said Matisz. “This walk really has been embraced by the community, and we welcome anyone who wants to help find a cure for CF to get involved.”
The Holmes County Great Strides Walk-a-thon in May will take place at 2:30 p.m. at the Rails to Trails Airport Park. Anyone wishing to get involved with the walk may do so by calling Mullet at 330-893-2255, or by logging on the CF website at: http://greatstrides.cff.org.
In a bit of thrilling news, Vertex Pharmaceuticals announced recently that the drug, VX-770, improved lung function in people with cystic fibrosis in a late-stage clinical trial. The drug also reduced the frequency of disease exacerbations that required treatment with antibiotics.
While the news is wonderful for those suffering from CF, VX-770 is designed to improve one of the lesser CF mutations, one which only affects about four percent of CF sufferers. However, according to Erica Matisz, Vertex is working on another drug that could have a direct bearing on the most common CF mutation. However, that one is further behind in its development, but it does speak volumes as to how far CF research has come over recent years.
“We’ve experienced some amazing results,” said Tony Mullet, Holmes County CF coordinator, whose daughter Michaela suffers from CF. “The new application would eventually affect 90 percent of the CF population. If we can crack one, we can crack the whole thing. The impact would be huge. It may be a few years off, but the strides we are making are incredible.”
About 30,000 Americans and 70,000 people worldwide have cystic fibrosis, a disease caused by defects in a gene responsible for the transport of chloride ions across cell membranes. People with the disease tend to have very thick mucus in their lungs, which leads to infections and lung damage. Many do not live past age 40, which is decades older than it was 25 years ago.
While the excitement of a solution to CF is on the horizon, cures don’t just materialize. It takes a long time to develop a drug for cystic fibrosis. It can take years of research to determine how a mutation actually causes a disease and then to design a drug that corrects the problem. Thus the expense of research is great.
Not surprisingly, Vertex received $75 million in financial support from the Cystic Fibrosis Foundation, one of the first nonprofit disease groups to give money to companies, since CF gets no federal aid in its fundraising efforts.
“These results are highly encouraging,” said Matisz. “This is the closest we’ve come yet to finally figuring this thing out. We obviously don’t want to put all of our eggs into one basket, and there are about 30 other drugs out there that are continuing to be tested, but it’s exciting. This could have an effect on tens of thousands of people.
“All of the money that CF walks like Holmes County’s and many others has gone a long way in creating the technology to make this happen.”
“It’s the fruits of our labor,” added Mullet. “We don’t know exactly what is going to happen, but we are extremely thrilled with the direction we’re headed.”
Over the years, Holmes County Great Strides has been instrumental in raising hundreds of thousands of dollars for CF research. The annual walk sees local teams made up of caring families, businesses and individuals who want to help generate funds for research take up the cause by walking a 5-K, during an afternoon of fun, food and fundraising. The group has set a goal of $44,000 this year.
“We have had so many wonderful people get behind this and join in on our efforts to raise money for CF research,” said Matisz. “This walk really has been embraced by the community, and we welcome anyone who wants to help find a cure for CF to get involved.”
The Holmes County Great Strides Walk-a-thon in May will take place at 2:30 p.m. at the Rails to Trails Airport Park. Anyone wishing to get involved with the walk may do so by calling Mullet at 330-893-2255, or by logging on the CF website at: http://greatstrides.cff.org.
Published: March 10, 2011
