Even as a medical student, Dr. David Fajgenbaum took the “physician, heal thyself” adage literally. It saved his life.
“I became critically ill with a disease I never heard of,” Fajgenbaum said. “I went from being completely healthy, no sort of medical challenges, to all of a sudden dying.”
Fajgenbaum, author of the book “Chasing My Cure” and founder of the nonprofit Every Cure, is associate professor at the Perelman School of Medicine at the University of Pennsylvania, where he was a medical student at the time he was first hospitalized with Castleman disease.
He spoke Sunday at the Purdue Northwest Sinai Forum lecture series at PNW’s Westville campus.
Fajgenbaum didn’t give up. “I got seven chemotherapy drugs that weren’t meant for my disease,” he said.
“They didn’t just save my life. They also opened my eyes” to alternate uses for diseases, Fajgenbaum said.
“My liver and my kidneys shut down when I had these relapses,” losing over 100 pounds of fluids all over his body. Fajgenbaum had been a college football player. He showed dramatic before-and-after pictures.
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Fajgenbaum wanted to devote his life to helping patients after his mother died in 2004 of glioblastoma, a form of brain cancer for which there is no known cure. It is nearly always fatal.
Fajgenbaum was a sophomore at Georgetown when his mother died. He started the national Students of AMF Support Network, which spread to other campuses as well. His mother’s initials were AMF, but the initials now represent Actively Moving Forward.
He later cofounded the Castleman Disease Collaborative Network, the Center for Cytokine Storm Treatment & Laboratory and, four years ago, Every Cure.
“To this date, thousands of patients are alive because of drugs we’ve repurposed,” he said.
“It’s just that no one is looking for these things,” Fajgenbaum said. “There’s just never been an entity that’s tried this before.”
“How many more drugs are sitting in the pharmacy that can treat more diseases than we even thought of?”
There are 14,000 diseases without a single approved therapy. They’re rare, but they add up to about 30 million Americans.
“This is a perfect use for artificial intelligence, but there’s no money to be made,” he said. That’s where the nonprofit Every Cure comes in.
It took a year to raise the first $1 million so the new nonprofit could begin its research.
Rather than trying to find treatments for specific patients, Every Cure is using AI to match up existing drugs with known diseases to see which drugs are most likely to work with which diseases. Then the work begins to find patients with that disease to see if a clinical trial can be done.
“We’re looking for every bread crumb that exists. We’re looking at all the low-hanging fruit,” he said. “We use cheap old medicines that no company will ever do the marketing for.”
For some fraction of women with breast cancer, putting lidocaine around the tumor before surgery makes it harder for cancer cells to escape, he said.
“We’re already up to 19 active programs,” repurposing meds for new uses. One or two new tests are added every quarter.
“All of us can benefit from drugs that already exist, and none of us can benefit unless we all get behind things,” Fajgenbaum said.
His own journey with Castleman disease is dramatic.
When he was hospitalized for the first time for Castleman disease, while he was in med school, he spent six months in critical condition. “My dad dropped everything to be with me,” Fajgenbaum said. Dad slept on a pullout bed in his son’s hospital room every night.
Fajgenbaum told of walking with his father on New Year’s Eve 2010, belly bloated because of Castleman disease attacking his autoimmune system. A drunk saw them and thought Fajgenbaum was his dad’s pregnant wife.
He recovered and went back to med school, having missed his entire third year.
That was the first near-death experience but not his last.
“Out of nowhere, it came back, and I almost died a fourth time,” Fajgenbaum said, advancing the narrative.
His doctor explained that not only were there no more options other than chemo, but he also was nearing his lifetime dosage of chemo drugs before they destroyed his heart.
“We can’t keep giving you more of these. You’re out of options,” Fajgenbaum said.
“I had so many things that I wanted to do,” he said. He wanted to get married, have a family, invent a new drug in his mom’s memory and more.
He wanted to invent a new drug that would cure himself. “Of course, I didn’t have $15 billion and 15 years to do it.”
“If you’re going for something, if you’re going to wish for something, if you’re going to pray for something,” ask what you can do to make it happen, he told the audience.
So Fajgenbaum turned to repurposing existing drugs for new uses.
Pharmaceutical companies typically don’t look for new uses for existing drugs because there isn’t a financial incentive to do so, he said. New uses often are found during clinical trials, when drugs are being tested and haven’t received full Food and Drug Administration approval.
Viagra was developed to treat heart disease, increasing the blood flow, before it became a popular erectile dysfunction drug. It also has been used to treat a rare pediatric lung disease. “It happens very randomly,” Fajgenbaum said, that doctors trying to treat a rare condition try another drug to see if it will work.
Thalidomide was developed and marketed as a sleep aid, then used to treat morning sickness before birth defects were linked to its use among pregnant women. The FDA didn’t approve its use for that purpose in the United States, although it was popular overseas. Now thalidomide is being used to treat leprosy.
“This concept that drugs are made for one disease and can’t help any others is just wrong,” Fajgenbaum said.
There are about 4,000 drugs and about 18,000 diseases, he said.
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Castleman disease is rare. “Why is my immune system attacking my heart, my lungs, my kidneys, my liver, my bone marrow?” he wanted to know.
After completing medical school, Fajgenbaum went to business school at Wharton. He realized finding a cure wasn’t simply a medical issue. “It had to do with can we get the right people to work together,” getting the money in place and other organizational issues that a business degree would help him accomplish.
Fajgenbaum got permission to use a lab to examine a lymph node – “basically your home base for your immune system” – and other tissue samples and fluids from his own body to see what might be going on.
Fortunately, he had free time while he was in business school to work in the lab in search of a drug to save his life.
It was during this time that he asked his girlfriend to marry him. Then, he said, “I nearly died for the fifth time.”
“I was receiving chemo weekly,” Fajgenbaum said, hoping he could go into remission before his planned May 24, 2014, wedding.
“I remember lying in the hospital bed and being so disappointed,” he said. “We tried these two drugs. They didn’t work.”
“I remember just lying there, and I was so heartbroken that I wasn’t going to spend any more time with them,” he said. “I had this deep grief for all the things I knew I was never going to experience.”
“I remember closing my eyes, saying goodbye,” before going into a coma.
He was getting chemo treatment. “I’m really getting close to my lifetime maximum; I’ve probably exceeded some of these lifesaving chemo drugs,” he said.
A day and a half later, he woke up, greeted his loved ones in the room and quickly started organizing how to get blood and tissue samples to the labs where they would be analyzed.
“In overtime, every second counts,” and Fajgenbaum felt like he was on his fifth overtime.
From the research, he realized his mTOR protein, which turns the immune system on or off, was in overdrive. That came from studying his cells and comparing them to normal ones.
He tried a drug that hits the off switch and has been in remission more than 12.6 years since the new treatment began.
“We’ve got two sweet little ones. We’ve got a third on the way,” he said, after making it to the wedding date.
Doug Ross is a freelance reporter for the Post-Tribune.