David Fajgenbaum - Every Cure, non-profit for drug repurposing using AI

Andy

Andy

Senior Member (Voting rights)
Dr. David Fajgenbaum has nearly died not once, but five times. The cause each time was a rare disorder called Castleman disease, an affliction on the boundary between cancer and an autoimmune disorder. It caused his entire body to swell up. Previously a muscled college football player, he first became bloated, then very thin.

Fajgenbaum, who was in medical school when he got sick, did something extraordinary. He founded a patient advocacy group, the Castleman Disease Collaborative Network. But more than that, he delved into the science of his disease, and proposed the treatment that, after five relapses, has kept him healthy since. It was an existing drug, sirolimus, that no one had thought to use for Castleman disease. Football, he said, helped him deal with the failure inherent in medical research.

Now 34, Fajgenbaum details his experience in a new book, “Chasing My Cure,” in which he also writes about his mother’s death from brain cancer and the way the disease affected every aspect of his life, including his relationship with his wife. He’s an assistant professor of medicine in the Division of Translational Medicine and Human Genetics at the University of Pennsylvania in Philadelphia.
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https://www.statnews.com/2019/09/12/after-nearly-dying-five-times-doctor-learned-to-treat-himself/
 
Some very familiar scenarios in the article. "Be a patient..."

More like be ridiculously and naively patient, not just a patient.

For most of us, our first, and sometimes only glimpse into the medical infrastructure is with our GP.

If the GP believes "they're working on it" that's the message patients get.

Many are told this about their particular health concerns. I was: "We're just waiting for a cure."

Even taking a more proactive approach and writing to the NIH, and Health and Welfare Canada (what it was called at the time), didn't get me more than a nice "We're working on it."

Essentially, a "go away" message. Don't call us, we'll call you.

Patients often seem to be viewed as annoying, or much worse if they're proactive.

For rare or neglected diseases, or for that matter well-funded ones that still need much work, it is essential that we are involved, and even take the lead.
 
These two quotes sounded familiar:

"I think that, as I said before, if the incentives aren’t in place, sometimes the federal government needs to step in."

"There’s thousands of diseases with no answers, millions of patients. And there’s thousands of drugs that are available today. Can we really mobilize resources in a way to make use of existing drugs? I think that’s a really cool idea."
 
I recently listened to this podcast with the author (a M.D.) of this book. Quite interesting with many parallels to people with a rare or poorly diagnosed illness. The author has Castleman disease. It even includes mention of mTor and sirolimus (rapamycin) as possibly helping these patients, cytokines storms and about the similarity of the storm to covid19's.

It also inculdes a short discussion of negotiating the process of creating patient centered research via a patient organization obtaining their own funding and directing what they want researched, instead of hoping researchers getting funded by a government etc.. There is not any detail in this discussion, but it certainly made me want to read the book and explore their patient organization...

https://www.npr.org/2020/05/13/855285638/doctor-with-rare-disease-decides-to-find-his-own-cure
 
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Wow, that's an amazing talk - I highly recommend watching it. What an impressive and lovely guy! What a fantastic project! (I feel really inadequate now.)

What a pity we know largely naff all about the biology of ME/CFS and are probably not even in the database. But the whole thing about repurposed drugs really looks the way to go.
 
AI Summary for those who can't watch:

General Summary:
David Fajgenbaum nearly died multiple times from Castleman disease, a rare immune disorder with no approved treatments.
Turning hope into action, he pioneered drug repurposing—finding existing FDA-approved drugs for new uses—by studying his own disease and using sirolimus to achieve long-term remission.
He co-founded Every Cure, a nonprofit using AI to rapidly identify repurposable drugs for thousands of diseases lacking treatments.
Despite the potential, repurposing is underused due to lack of profit incentives.
Every Cure advances new and underutilized therapies, saving lives and promoting awareness to unlock hidden cures sitting on pharmacy shelves.
David calls for collective effort to help patients hear "we have something" instead of "we've tried everything."


Transcript Summary:
00:04
David Fajgenbaum shares how in 2010, at age 25, he was critically ill with Castleman disease, a rare and deadly illness that causes the immune system to attack vital organs. His doctors told him there was nothing more they could do, and his family prepared for his death.


00:29
Despite being a medical student and former college quarterback, David had never heard of Castleman disease before he got sick. With no approved treatments, his doctors tried an experimental combination of seven chemotherapies not originally intended for his disease, which saved his life temporarily.


01:08
David faced multiple relapses—five times in three years—and nearly died repeatedly. He recalls one relapse when he was overwhelmed with grief over the life experiences and contributions he might lose, including the family he dreamed of and the patients he wanted to treat.


01:45
He realized that hoping for a cure was not enough. To survive, he needed to take action himself. However, developing a new drug would take 15 years and cost a billion dollars, which he could not afford or wait for.


02:10
David explored repurposing existing drugs from other diseases to treat his condition, inspired by examples like Viagra and thalidomide, which were repurposed successfully to treat different diseases. This is possible because different diseases may share underlying mechanisms treatable by the same drugs.


02:52
Doctors can prescribe FDA-approved drugs for new uses off-label if the benefits outweigh risks, and this practice is common in the US, with about 25% of daily prescriptions written off-label.


03:08
By studying his own blood, David identified an overactive immune signaling pathway. He found that sirolimus, a transplant drug never used for Castleman disease before, could turn it off. Testing it on himself, he achieved remission that has lasted over 11 years.


03:42
During his remission, David married his girlfriend Caitlin, had two children, wrote a book titled "Chasing My Cure," and joined the University of Pennsylvania faculty to continue researching cures for rare diseases.


04:05
In 2022, David co-founded Every Cure, a nonprofit aiming to unlock the full potential of existing drugs to treat various diseases. The organization has advanced 14 repurposed treatments that have helped thousands of patients.


04:24
David gives examples like Kylo, who started nursing school after being saved by a repurposed cancer drug, and Michael, who survived rare cancer with a melanoma drug now used globally for that condition.


04:45
David reflects on the thousands of drugs available that could treat many of the 14,000 diseases without approved therapies, many affecting 1 in 10 people. Repurposing drugs can be much cheaper and faster than developing new ones.


05:29
However, drug repurposing is not profitable, especially for rare diseases, because most repurposed drugs are generic, and clinical trials cost a lot. Thus, drug companies focus on profitable new drugs, and no major institutions have taken systematic responsibility for repurposing—until Every Cure.


06:04
Every Cure uses artificial intelligence (AI) to scan global knowledge on 4,000 drugs and 18,000 diseases to find promising drug-disease matches rapidly, similar to how Netflix recommends movies using data science.


06:39
AI helps prioritize the best opportunities to reduce suffering and maximize impact. Every Cure studies these drug candidates in labs and clinical trials or directly promotes ones already proven effective.


07:01
David and his cofounders were uncertain how to fund this work until they discovered The Audacious Project, which supports innovative nonprofits tackling big challenges. After an initial rejection, they received transformative funding from Audacious and the US federal agency ARPA-H.


08:18
With funding, Every Cure plans to repurpose 15 to 25 treatments by 2030 and aims to expand to dozens or hundreds more. Their AI pipeline is already helping patients, including a Castleman disease patient saved by a high-scoring drug from their platform.


08:58
They sometimes find drugs that are effective but underutilized, like leucovorin, a cheap vitamin derivative shown to improve speech in children with specific antibodies. Examples include Mason, nonverbal for three years, who began speaking in three days, and Ryan, nonverbal for five years, who spoke to his father for the first time.


10:10
Though a blood test exists to identify patients who could benefit from leucovorin, it remains underused, illustrating the critical need for awareness and accessibility that Every Cure seeks to address.


10:21
The organization has also helped Joseph, diagnosed with a rare cancer called POEMS syndrome, who was saved through repurposed drugs originally for multiple myeloma, turning a farewell into planning a wedding.


11:12
David emphasizes how many patients and their loved ones hear "we've tried everything," but often not all options have been explored. He calls for collective effort to find and deliver these hidden cures.


11:39
David invites the audience to join Every Cure's mission to unlock life-saving treatments sitting on pharmacy shelves, transforming despair into hope for patients worldwide.


12:06
In a brief dialogue with host Latif Nasser, David explains repurposed drugs could be affordable cures available at local pharmacies, but profit incentives currently hinder their use.


12:38
They discuss historical medicine shifts from broad cure-alls to targeted therapies, and how Every Cure aims to broaden therapeutic horizons again by uncovering overlooked drug uses.


13:23
David invites people to share experiences of off-label drug use at everycure.org/ideas, donate to fund trials, and help raise awareness to amplify these treatment opportunities.


13:56
The talk concludes with thanks and applause for David Fajgenbaum and his work to transform rare disease treatment and drug repurposing efforts.
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"By studying his own blood, David identified an overactive immune signaling pathway." We're still waiting for that identification for ME. We can't look for a drug to fix a pathway we can't find. Once that pathway is found, this sort of organization could help.
 
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