Finally a treatment for CF page 1
nostrum
31st January 2012, 07:08 PM
http://www.nytimes.com/2012/02/01/business/fda-approves-cystic-fibrosis-drug.html?_r=1&partner=rss&emc=rss
Amazing that there is now a therapy to counter the actual defective gene.
Long long time coming. RIP Jacinta who couldn't wait that long.
Amazing that there is now a therapy to counter the actual defective gene.
Long long time coming. RIP Jacinta who couldn't wait that long.
maiforpeace
31st January 2012, 09:17 PM
What a breakthrough. Finally.
Yes, and sadly too late for many.
Was Jacinto a friend of yours?
I lost a young friend to CF...she was my next door neighbor. One day I was playing Joan Osbourne's "One of Us" and she knocked on my door...she was interested in what I thought about the song and during the course of doing so I told her I was an atheist. That began a friendship between us, sharing music, and supporting her through the shedding of born again beliefs that her mother had forced on her all her life.
She was 22 when she died. :no:
Yes, and sadly too late for many.
Was Jacinto a friend of yours?
I lost a young friend to CF...she was my next door neighbor. One day I was playing Joan Osbourne's "One of Us" and she knocked on my door...she was interested in what I thought about the song and during the course of doing so I told her I was an atheist. That began a friendship between us, sharing music, and supporting her through the shedding of born again beliefs that her mother had forced on her all her life.
She was 22 when she died. :no:
maiforpeace
31st January 2012, 09:19 PM
Double post
Adenosine
31st January 2012, 11:32 PM
That's great. Unfortunately there are over 1300 mutations in the CFTR protein that cause CF, ranging from deletions of entire sections to single nucleotide polymorphisms like the one this drug treats. It is unlikely that drug therapy will be made for all of them.
The best, and least likely to happen, treatment will be true gene therapy where the defective gene is replaced in it's entirety. The problem is delivering the new gene to the chromosome. The only true gene therapy available was developed by a Chinese biotech company. It's called Gendicine and replaces a protein called p53 in head and neck squamous cell carcinomas. p53 is a tumour suppressor. Some 70% of people with cancer have a defective p53 gene. The treatment uses an adenovirus to insert the wild type gene into the cancer cells. Unfortunately for most cancer patients it only works on easily accessible cancers, like skin cancers.
That's the current holy grail in medical research, how to replace defective genes in a targeted manner.
The best, and least likely to happen, treatment will be true gene therapy where the defective gene is replaced in it's entirety. The problem is delivering the new gene to the chromosome. The only true gene therapy available was developed by a Chinese biotech company. It's called Gendicine and replaces a protein called p53 in head and neck squamous cell carcinomas. p53 is a tumour suppressor. Some 70% of people with cancer have a defective p53 gene. The treatment uses an adenovirus to insert the wild type gene into the cancer cells. Unfortunately for most cancer patients it only works on easily accessible cancers, like skin cancers.
That's the current holy grail in medical research, how to replace defective genes in a targeted manner.
Hermit
1st February 2012, 02:14 AM
Another downside:
"Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year"
Who can afford that?
"Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year"
Who can afford that?
Adenosine
1st February 2012, 02:17 AM
Another downside:
"Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year"
Who can afford that?
I'm sure it will be on the PBS which will drastically reduce the cost.
"Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year"
Who can afford that?
I'm sure it will be on the PBS which will drastically reduce the cost.
Hermit
1st February 2012, 03:16 AM
Another downside:
"Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year"
Who can afford that?
I'm sure it will be on the PBS which will drastically reduce the cost.
I was thinking of the US, where about 30,000 people suffer from this rare disease. Do they have a PBS, or do they need to rely on private insurance? If the latter, don't health insurance policies cover their arses by including an annual as well as a per classification refund cap?
"Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year"
Who can afford that?
I'm sure it will be on the PBS which will drastically reduce the cost.
I was thinking of the US, where about 30,000 people suffer from this rare disease. Do they have a PBS, or do they need to rely on private insurance? If the latter, don't health insurance policies cover their arses by including an annual as well as a per classification refund cap?
Mantisdreamz
1st February 2012, 08:40 AM
Goddamn. This is amazing... I'm skeptical that it'll work (probably due to tiredness).
My best friend, since grade 3 has CF. She's a huge fighter, doesn't even like to talk about the fact that she has CF. But she's been through hell and back. She had a tube implanted into her stomach when we were 12 years old, in order to make sure she was getting tons of nutrients. She's been through countless lung infections.
And personally, I don't want to see my best friend who is an amazing person die young. *words can't express*
I have, and she too, have always thought that in this day and age there would eventually be a cure.
My best friend, since grade 3 has CF. She's a huge fighter, doesn't even like to talk about the fact that she has CF. But she's been through hell and back. She had a tube implanted into her stomach when we were 12 years old, in order to make sure she was getting tons of nutrients. She's been through countless lung infections.
And personally, I don't want to see my best friend who is an amazing person die young. *words can't express*
I have, and she too, have always thought that in this day and age there would eventually be a cure.
nostrum
1st February 2012, 08:51 AM
mantis, fingers and toes crossed that your friend has the mutation that is treated by this drug :hug:
Mai, yes Jacinta was in primary school with me. I didn't know she had CF. She died at just age 19 :(
Mai, yes Jacinta was in primary school with me. I didn't know she had CF. She died at just age 19 :(
ksen
1st February 2012, 10:34 PM
This is good news!
eta: I thought this was the start of another Christian Forums thread.
eta: I thought this was the start of another Christian Forums thread.
oblivion
2nd February 2012, 12:43 AM
This is good news!
eta: I thought this was the start of another Christian Forums thread.
:fistbump:
eta: I thought this was the start of another Christian Forums thread.
:fistbump:
Jerome
2nd February 2012, 02:16 AM
:thumbsup:
nostrum
2nd February 2012, 12:18 PM
This is good news!
eta: I thought this was the start of another Christian Forums thread.
:fistbump:
Double :colbert:
eta: I thought this was the start of another Christian Forums thread.
:fistbump:
Double :colbert:
Nhận xét
Đăng nhận xét