Log out
My profile and settings
My bookmarks
Comment history
Please complete your account verification. Resend verification email.
today
This verification token has expired.
today
Your email address has been verified. Update my profile.
today
Your account has been deactivated. Sign in to re-activate your account.
today
View all newsletters in the newsletter archive
today
You are now unsubscribed from receiving emails.
today
Sorry, we were unable to unsubscribe you at this time.
today
0
0
Back to profile
Comment Items
You have not left any comments yet.
title
you replied to a comment:
name
description
Saved Posts
You haven’t bookmarked any posts yet.

Let’s recommit to funding work on the Global Goals, because it’s saving lives and helping people escape extreme poverty.

Read more
Become a Gates Notes Insider
Sign up
Log out
Personal Information
Title
Mr
Mrs
Ms
Miss
Mx
Dr
Cancel
Save
This email is already registered
Cancel
Save
Please verify email address. Click verification link sent to this email address or resend verification email.
Cancel
Save
Email and Notification Settings
Send me updates from Bill Gates
You must provide an email
On
Off
Send me Gates Notes survey emails
On
Off
Send me the weekly Top of Mind newsletter
On
Off
Email me comment notifications
On
Off
On-screen comment notifications
On
Off
Interests
Select interests to personalize your profile and experience on Gates Notes.
Saving Lives
Energy Innovation
Improving Education
Alzheimer's
Philanthropy
Book Reviews
About Bill Gates
Account Deactivation
Click the link below to begin the account deactivation process.
If you would like to permanently delete your Gates Notes account and remove it’s content, please send us a request here.

Fresh air

The heroes of the cystic fibrosis fight

Breath from Salt gave me new perspective on a disease I’ve been following for two decades.

|
0

In 1999, a Microsoft colleague named Paul Flessner approached me with a personal request: Would I be willing to help fund the development of new drugs for cystic fibrosis, an awful lung disease that affects about 30,000 people—including more than 10,000 children—in the U.S. and about 70,000 people worldwide?

Ten years after the discovery of the gene implicated in CF, researchers still had not produced any medical breakthroughs for CF patients, and Paul was concerned that time was running short for his sons. So he asked me to support an effort by a small startup called Aurora Biosciences to screen up to 10,000 chemical compounds a day in an effort to find ones that might help CF patients; at the time, typical screening methods allowed for testing just a few chemical compounds a day.

The whole project was untried and risky. But my dad, who was helping Melinda and me with our early philanthropic efforts, and I reasoned that philanthropists should be willing to take big risks like this, and so we decided to provide $20 million of the $47 million needed to launch this research project.

This pathbreaking work led, over two decades, to several very effective CF medicines, including drugs marketed with the names Kalydeco, Orkambi, and Trikafta. For many CF patients, these medicines produce the kind of miraculous “Lazarus effect” that I would see when I visited with AIDS patients taking cocktails of antiretroviral medicines. In fact, Paul recently sent me a video of one of his sons, a software engineer in his early 30s, running at an altitude of 7,500 feet. “For both boys, it’s like not having CF anymore,” he wrote. “Since Trikafta, one of the boys has stopped taking all of the antibiotics he used to control infections and stopped all airway-clearance therapy. It is a huge new liberty for him.”

Through Paul, I’ve been able to follow the big milestones in this success story. But now, thanks to a new book called Breath from Salt, I understand a lot more about all the people who made these breakthroughs possible, including many thousands of physicians, scientists, statisticians, engineers, patients, advocates, donors, and entrepreneurs.

Breath from Salt is not for everyone. It’s long and sometimes feels overly detailed; I suspect the author, Bijal P. Trivedi, felt obliged to include all of the people she interviewed, even if they played minor roles. But given my interest in the process of discovery and my connection to this specific effort, I couldn’t put the book down.

The book is reminiscent of Tracy Kidder’s 1981 book Soul of a New Machine, which documents the creation of a powerful new computer, because Trivedi leads you on a tour through the ups and downs of the discovery process. She gives it life-and-death urgency and emotion. In fact, I was surprised to learn that she doesn’t have a family connection to CF.

Among the heroes Trivedi profiles is the O’Donnell family, who live near Boston. In 1974, Kathy and Joe O’Donnell had their first child, Joey. Unfortunately, Joey inherited CF mutations from both of his parents, which meant he got the disease. Like all CF patients, Joey could not produce healthy copies of the protein we all need to maintain the balance of salt and water on surfaces in our bodies, such as the surface of the lung. That meant his tiny lungs would fill with thick, sticky mucus, reducing his lung capacity and making him highly susceptible to dangerous infections.

Joe and Kathy knew that many CF patients didn’t make it past adolescence. But they simply would not give up on Joey. They found outstanding healthcare providers, spent months on end in hospitals, and dedicated hours of every day to clapping on Joey’s chest to help free mucus from his airway. Even with Kathy and Joe’s significant resources, their son died at age 12.

Joe, who had grown up in a tough neighborhood but had gone on to become a very successful businessman, found purpose in leading huge fundraising campaigns to spur CF drug discovery. Without his own financial contributions, fundraising prowess, and business acumen, there’s no way that CF patients would have the treatments they have today. Joe helped raise money for drugs that would have worked for his son, but the book makes it clear that that wasn’t his motivation. He did it for all children with CF.

The second hero of this story is the Cystic Fibrosis Foundation and its three generations of leaders. Given that CF is an orphan disease that affects only a small slice of the population, it’s remarkable to me that this organization grew to become one of the biggest and most powerful players among disease-research charities. I was impressed by the courage of the foundation leaders who made the controversial decision to place almost all their chips on scientific research and to build sophisticated patient registries to facilitate clinical trials. I also admired the creative “venture philanthropy” approaches the CF Foundation used to fund drug discovery.

The third group of heroes is the CF researchers who kept building the knowledge necessary to produce these drugs, such as the San Diego-based team that started with Aurora and now work for a company called Vertex. Breath from Salt shows us their passion for their work, the ways they fought to protect it even when it wasn’t paying financial dividends, and the remarkable scientific breakthroughs they pioneered.

It was touching to learn about emotional moments in the process, such as when Fred Van Goor, one of the leaders of the San Diego team, heard the results of the clinical trial on the drug combination he’d helped to create. “Hearing how the patients’ lungs responded to this new medicine, how dramatically it opened them up, made [him weep]. Van Goor was unprepared for the pent-up emotions that had been swirling in him for the past 18 years, and for how desperately he’d wanted those treatments to work.”

The final group of heroes are compassionate caregivers, including parents, who fought to keep CF children alive when they had few tools for doing so. This list includes not just senior physicians like Joey’s doctor at Massachusetts General Hospital but also unsung heroes like a physical therapist who grew very close to Joey and so many other children while working with them every day to help them keep their lungs as healthy as possible.

Breath from Salt is an inspiring book. It’s a testament to what’s possible when passionate leaders help to harness the unique strengths of philanthropy, nonprofits, government, academia, biotechs, big pharma, and medical providers. Given the remarkable pace of innovation in the biosciences, I know there will be more success stories like this in the coming years.

Discussion
Thank you for being part of the Gates Notes Insider community.
Not seeing your comment? You can read our policy on moderating comments here and learn about our Gates Notes badges here.
Badge
📌
Pinned by
Gates Notes
Badge
ʼʼ
0 responses
Sort by
all
all
most
top
old
Comments loading...
CTW
Thanks for visiting the Gates Notes. We'd like your feedback.
Become a Gates Notes Insider
Join the Gates Notes community to access exclusive content, comment on stories, participate in giveaways, and more.
SIGN UP
Already have an account?
Log in here
Logout:


Become a Gates Notes Insider
Become a Gates Notes Insider
Join the Gates Notes community to get regular updates from Bill on key topics like global health and climate change, to access exclusive content, comment on stories, participate in giveaways, and more.
Already joined? Log in
Please send me updates from Breakthrough Energy on efforts to combat climate change.
On
Off
LOG IN
SIGN UP
Title
Mr
Mrs
Ms
Miss
Mx
Dr
This email is already registered. Enter a new email, try signing in or retrieve your password
Why are we collecting this information? Gates Notes may send a welcome note or other exclusive Insider mail from time to time. Additionally, some campaigns and content may only be available to users in certain areas. Gates Notes will never share and distribute your information with external parties.
Bill may send you a welcome note or other exclusive Insider mail from time to time. We will never share your information.
Sign up
We will never share or spam your email address. For more information see our Sign Up FAQ. By clicking "Sign Up" you agree to the Gates Notes Terms of Use / Privacy Policy.
Street address
City
postal_town
State Zip code
administrative_area_level_2
Country
Data
Gates Notes Insider Sign Up FAQ

Q. How do I create a Gates Notes account?

A. There are three ways you can create a Gates Notes account:

  • Sign up with Facebook. We’ll never post to your Facebook account without your permission.
  • Sign up with Twitter. We’ll never post to your Twitter account without your permission.
  • Sign up with your email. Enter your email address during sign up. We’ll email you a link for verification.

Q. Will you ever post to my Facebook or Twitter accounts without my permission?

A. No, never.

Q. How do I sign up to receive email communications from my Gates Notes account?

A. In Account Settings, click the toggle switch next to “Send me updates from Bill Gates.”

Q. How will you use the Interests I select in Account Settings?

A. We will use them to choose the Suggested Reads that appear on your profile page.

BACK
Forgot your password?
Enter the email you used to sign up and a reset password link will be sent to you.
This email is already registered. Enter a new email, try signing in or retrieve your password
Reset Password
Reset your password.
Set New Password
Your password has been reset. Please continue to the log in page.
Log in
Get emails from Bill Gates
Send me updates from Bill Gates
You must provide an email
On
Off
Email me comment notifications
On
Off
On-screen comment notifications
On
Off
This email is already registered
Finish
We will never share or spam your email address. For more information see our Sign up FAQ. By clicking "Continue" you agree to the Gates Notes Terms of Use / Privacy Policy.
You're in!
You're in!
Please check your email and click the link provided to verify your account.
Didn't get an email from us? Resend verification
Upload a profile picture
Choose image to upload
Uploading...
Uh Oh!
The image you are trying to upload is either too big or is an unacceptable format. Please upload a .jpg or .png image that is under 25MB.
Ok
Title
Mr
Mrs
Ms
Miss
Mx
Dr
Cancel
Save
This email is already registered
Cancel
Save
Please verify email address. Click verification link sent to this email address or resend verification email.
Email and notification settings
Send me updates from Bill Gates
You must provide an email
On
Off
Email me comment notifications
On
Off
On-screen comment notifications
On
Off
Select your interests
Saving Lives
Energy Innovation
Improving Education
Alzheimer's
Philanthropy
Book Reviews
About Bill Gates
Finish
Confirm Account Deactivation
Are you sure you want to deactivate your account?
Deactivating your account will unsubscribe you from Gates Notes emails, and will remove your profile and account information from public view on the Gates Notes. Please allow for 24 hours for the deactivation to fully process. You can sign back in at any time to reactivate your account and restore its content.
Deactivate My Acccount
Go Back
Your Gates Notes account has been deactivated.
Come back anytime.
Welcome back
In order to unsubscribe you will need to sign-in to your Gates Notes Insider account
Once signed in just go to your Account Settings page and set your subscription options as desired.
Sign In
Request account deletion
We’re sorry to see you go. Your request may take a few days to process; we want to double check things before hitting the big red button. Requesting an account deletion will permanently remove all of your profile content. If you’ve changed your mind about deleting your account, you can always hit cancel and deactivate instead.
Submit
Cancel
Thank You! Your request has been sent
Page https://www.gatesnotes.com:443/Jim-Grants-Child-Survival-Revolution secs = 0.0308479