Charity Cases: When Venture Capital Meets Philanthropy

There’s a familiar adage that money can’t buy happiness. Generally, I hold this to be true. But I believe there is one exception and as counterintuitive as it may sound, Money can buy happiness—when you give it away! Many people would scoff at such a notion and balk at attempting this experiment with their own hard earned capital. In fact, you are probably laughing at this point or have concluded that I’ve taken leave of my senses. But I want to challenge our readers to consider this notion.

There is a great sense of empowerment when one unshackles themselves from the powerful lure of money. All throughout recorded history, humans have been obsessed with money in all of its forms. We seek more of it—at any cost. Many of us spend a lifetime trying to accumulate it; with varying degrees of success. Some of us will lie, cheat, steal and borrow to get more of it. Others save, invest or hoard it. On occasion some of us (myself included) spend it on needless goods that will likely end up in a landfill. (I once read that 67% of our entire GDP eventually ends up in a landfill!) A few will abandon spouses and families for it. And, as the evening news proclaims all too often, some will go so far as to kill for it.

Personally, I have found a great sense of happiness in giving money away. It’s empowering, and if done in certain contexts, the results can change or even save lives.

Many years ago, I was visiting my dad and my stepmother. My stepmom’s nieces—two adorable little girls named Regan and Randy—also happened to be visiting that day. Regan and Randy were about eight years old at the time. After pouring all morning, the rain finally stopped and the two girls wanted to play outdoors. A bit later I went out to see what they were up to. Little Regan was playing with the worms that tend to surface after a downpour in Chicago. I had a riot explaining a little biology to her and discovered that a curious child provides an enormous amount of entertainment value. Being childless, I got to be a parent for a few hours. I later found out after everyone went home, that Regan and Randy had a hereditary disease by the name of Cystic Fibrosis. Both parents can carry the gene but do not have the disease themselves. However, when both parents carry the gene and they have children, in a most terrible twist of fate, they stand a chance of their children having CF. The suffering this disease causes is immeasurable to both children and their parents.

But In 1989 the first obstacle toward a treatment/cure was scaled when a group led by Francis Collins identified the gene for Cystic Fibrosis. Later, a small protein was discovered called Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). In CF, the gene responsible for making the CFTR protein is defective. This protein functions as a channel across the membrane of cells that produce mucus, sweat, saliva, tears, and digestive enzymes. The channel transports negatively charged particles called chloride ions into and out of cells. The transport of chloride ions helps control the movement of water in tissues, which is necessary for the production of thin, freely flowing mucus. Mucus is a slippery substance that lubricates and protects the lining of the airways, digestive system, reproductive system, and other organs and tissues. When CFTR is defective, the cells that line the passageways of the lungs, pancreas, and other organs produce mucus that is abnormally thick and sticky. The abnormal mucus obstructs the airways and glands, leading to the characteristic signs and symptoms of cystic fibrosis—extreme difficulty in breathing and in digesting food. The CFTR protein also regulates the function of other channels, such as those that transport positively charged particles called sodium ions across cell membranes. These channels are necessary for the normal function of organs such as the lungs and pancreas and the gastrointestinal tract.

The Volume of air inhaled by the lung with cystic fibrosis is not even close to normal. Moreover, it creates a perfect environment for infection both in the lungs, the pancreas and other organs. A few decades ago, patients afflicted with CF rarely made it into their twenties. About 30,000 people in the United States have Cystic Fibrosis.

Soon after the experience with Regan and Randy, I wrote a check to the Cystic Fibrosis foundation. It wasn’t a lot of money (I didn’t have a lot back then), but I remember hoping that they’d eventually find a treatment or even a cure so these little girls wouldn’t literally die from drowning in their own fluids. After sending the check, I often wondered what happens when you give money to charity. Does it do any good? Does it make an impact? Will the money lead to groundbreaking research? Twenty years later, I received my answer.

The Cystic Fibrosis Foundation was founded in 1955 by parents whose children were afflicted by the disease. In the 1950’s and 60’s, patients born with CF usually died in their teenage years or younger. The foundation (CFF) over the years has taken donations of over $600 million dollars to advance the cause and try to find a treatment or perhaps a cure. They had some successes, and medical advances extended the lives of CF sufferers into their thirties. The treatments however, were mostly successful in warding off infections, improving respiratory capacity, and ameliorating symptoms.

But then a major breakthrough surfaced in the Cystic Fibrosis world, and over the course of the decades, new hope arrived for those afflicted with CF. A small biological testing company which performed large- scale high-throughput screening for compounds had found a molecule that might re-activate the ion channel defect and allow the healthy exchange of water and chloride through the channel. This action would dilute the thick mucus buildup and allow the natural action of the lungs to expel the fluids, giving CF patients a shot at a more normal life.

The small testing company was San Diego based Aurora Biosciences. Earlier, the Cystic Fibrosis Foundation found out about the CFTR research that Aurora was carrying out and joined forces with them by pledging $47 million to continue the promising research on CFTR and ion channeling. The pledge to Aurora was one of the largest donations from a non-profit organization to a for-profit entity ever. The catalyst for this huge pledge was Bob Beall, the CEO of the Cystic Fibrosis Foundation. The man was on a mission, and that mission was to find a cure for this dreaded disease. Frustrated by the slow progress made by academic institutions that could only screen a couple of compounds, he realized Aurora Biosciences and its high-throughput screening was the best chance (to date) of getting to the source of the CF problem. And their early CFTR studies suggested advancements beyond just treating symptoms.

Then, in 2001, Vertex Pharmaceuticals bought Aurora Biosciences to add to its business portfolio and to have in-house expertise in high-speed screening to assist in discovering new drugs to bring to market. In doing so, Vertex retained the CFTR/Ion channel research. Despite some tense moments between Beall and the Vertex senior management, Beall possessed an Olympic Gold medal in persistence and pressed on. (Vertex had some issues convincing shareholders to invest in developing a drug with limited appeal to only a small subset of the CF population, especially with drug development costs so high, and other targets with wider patient need and greater potential revenues.) However, Beall prevailed and the CFF/Vertex partnership flourished. In fact, the foundation, starting in the late 1990’s began what was to become a $150 million investment over the next few years. Vertex received much needed money to develop a drug, and the CFF attained royalty rights on any commercial drug that resulted from Vertex’s labs. Thus, a new way of fighting disease was born – venture philanthropy.

How did the investment do? Glad you asked. CFF’s investment achieved returns that would be the envy of anyone on Wall Street. Vertex went on to develop Kalydeco (which treated the more rare CF mutation that afflicted only a few thousand people in the United States) and followed it up with Orkambi (which treats the more common CF gene variant). Bob Beall’s efforts paid off more than anyone ever imagined. Those royalty rights that cost CFF $150 million became quite valuable. Just last year, the Cystic Fibrosis Foundation sold off those royalty rights to Royalty Pharma, an investment firm that funds late stage clinical trials in exchange for royalty rights. The proceeds: $3.3 billion, making the foundation one of the largest in the U.S. in terms of assets.

In the grand scheme of life, the money is less important than the lives that were changed. Aaron Stocks, a CF patient, broke down crying in his driveway one day following a workout. He was enrolled in the Kalydeco placebo-controlled clinical trials and knew he was getting the drug because he didn’t become winded like he usually did. He was quoted in a Bloomberg article saying, “This is what it feels like to be a human.”

So, my $20 check written to the CFF years ago, bought only a few test tubes perhaps. No big deal. Or so I thought at the time. But when you add a few million more donors, an ocean full of persistence, and time, a little deal becomes a BIG deal. And I think Regan, Randy and Aaron would all agree with me since all three are still enjoying life. Regan and Randy are married and in their mid -thirties. Aaron actually works for the Cystic Fibrosis Foundation.

So go ahead, take a leap of faith, and give some money away to a truly great scientific/medical (or other) cause. It will bring you the greatest joy possible: a life WELL lived by helping those live life WELL who otherwise couldn’t… because down the road, some curious worm-loving kid who didn’t have much of a chance at life, will grow up and be able to dance at her wedding with her father. I’d make that trade any day.

In the spirit of venture philanthropy...
In the spirit of venture philanthropy, In Sickness and Wealth will donate a portion of its profits to worthy medical causes. It’s time for us to leverage up on Beall’s efforts and join the fight against other diseases – either by investing in companies like Vertex that engage in these endeavors, or by donating to charity. We welcome readers to share their stories on this matter.

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