Sunday November 17, 2019

Scientists Hail New Cystic Fibrosis Treatment

"Now, 30 years later, that time has come"

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Scientists, Cystic Fibrosis, Treatment
FILE - A research scientist works in a laboratory at Vertex Pharmaceuticals Inc. in San Diego, March 4, 2015. The company's new cystic fibrosis drug, Trikafta, was approved by the Food and Drug Administration last week. VOA

On Aug. 25, 1989, an 8-year-old girl with cystic fibrosis wrote in her journal that it was “the most best day” because scientists had “found a Jean for Cistik fibrosis.”

On Thursday, the current head of the National Institutes of Health — who was a member of one of the teams that found the gene — wrote in The New England Journal of Medicine that a triple-drug therapy has been found to be highly effective in treating the life-threatening disorder.

“We hoped that the gene discovery would someday lead to effective treatments for children and adults with cystic fibrosis,” Francis Collins wrote. “Now, 30 years later, that time has come.”

The drug, called Trikafta, was approved by the Food and Drug Administration last week.

Scientists, Cystic Fibrosis, Treatment
On Thursday, the current head of the National Institutes of Health — who was a member of one of the teams that found the gene — wrote in The New England Journal of Medicine. Pixabay

Some 30,000 Americans have been diagnosed with cystic fibrosis, which causes thick mucus buildup in the patient’s organs, affecting respiration and digestion. While other drugs have helped lengthen patients’ lives, those born with the disease are expected to live only into their 40s.

Past treatments helped only a small percentage of patients, but Trikafta targets Phe508del, the most common mutation of the cystic fibrosis gene. Collins said this means 90% of those suffering from cystic fibrosis — including Jenny, the 8-year-old journal writer — will be helped by the therapy.

Now 38, Jenny McGlincy told The Washington Post that she cried when she read the drug had been approved.

“To think of my lung function improving or my digestion increasing, or even adding a few years to my life that I could spend with my daughter. … Now that it’s available, I’m a little like, ‘Is this really happening?’ ” she told the Post.

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 Model of collaboration

Cystic fibrosis research has set a standard of how the collaboration between nonprofits and pharmaceutical firms can help develop treatments. Collins points out that the Cystic Fibrosis Foundation, frustrated that gene treatments were slow to be found, decided to invest directly in a small company called Aurora Biosciences, which is now Vertex Pharmaceuticals, the developer of Trikafta.

That collaboration, “now spanning more than two decades, can be seen as an important model for other rare genetic diseases,” Collins wrote.

Scientists, Cystic Fibrosis, Treatment
“We hoped that the gene discovery would someday lead to effective treatments for children and adults with cystic fibrosis,” Francis Collins wrote. Pixabay

After the discovery of the gene, Collins wrote a song, “Dare to Dream.”

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“The lyrics expressed hope that the gene discovery would lead to effective treatments for cystic fibrosis — that someday we would see ‘all our brothers and sisters breathing free.’ It is profoundly gratifying to see that this dream is coming true,” he said. (VOA)

Next Story

Here’s Why Complimentary Cancer Therapies Can Cause More Harm

Doctors need to be more proactive about asking their patients what else they are taking when they are being treated for cancer

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Cancer
It is particularly important that patients always check with their doctors first before trying complementary therapies for Cancer that has spread to the skin. Pixabay

A medical expert has said that Cancer patients should tell doctors treating them about the herbal products they may be taking since some ingredients could affect their treatment.

Maria Joao Cardoso, the head breast surgeon at the Champalimaud Cancer Centre in Lisbon, Portugal, said that there was no evidence that herbal therapies or creams worked.

If in doubt, it is best not to take anything, she added.

Garlic, ginger and ginkgo pills, for example, can delay the healing of skin wounds when breast cancer spreads, she said.

“Doctors need to be more proactive about asking their patients what else they are taking when they are being treated for cancer,” Cardoso told the BBC.

She said that it is particularly important that patients always check with their doctors first before trying complementary therapies for cancer that has spread to the skin. This happens in one in five cases of breast cancer, and less in other cancers.

The danger is that many products can interfere with the hormone therapy or chemotherapy treatments, and certain ones prolong the blood clotting process, which can lead to wounds taking longer time to heal and more scarring.

She said that herbal products like green chiretta, feverfew, garlic, ginkgo, ginseng, hawthorn, horse chestnut and turmeric slow down clotting.

Cancer
A medical expert has said that Cancer patients should tell doctors treating them about the herbal products they may be taking since some ingredients could affect their treatment. Pixabay

Cardoso said that it is not surprising that patients and their carers go searching for complementary or alternative treatments that might make a difference.

But she said people should know that “they could end up doing more harm than good”.

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“The highest goal in medicine is important to remember: Do no harm,” she said.

As per the website of Cancer Research UK, some complementary therapies might stop conventional treatments working as well as they should. (IANS)