AI discovers existing drugs work for rare diseases
A patient’s life was saved after researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata, and Every Cure, used artificial intelligence to use existing drugs to treat idiopathic multicentric Castleman disease (iMCD).
Every Cure and partners are developing medical databases and AI algorithms to unlock new uses for existing medicines for all diseases, and a recent assessment identified adalimumab as the most promising treatment for iMCD out of all 3,000 approved drugs.
iMCD is a rare and life-threatening disorder involving hyperactivation of the immune system, leading to uncontrolled dysfunction of organs. The only FDA-approved treatment works in some of those diagnosed with iMCD, leaving most patients with limited options.
Albert, a 50-year-old iMCD patient on all known medications, was prescribed adalimumab by Luke Chen of the University of British Columbia after consulting with David Fajgenbaum and his team from Every Cure, who have identified potential benefits of this treatment using a guided proteomics approach. AI.
“After a two year battle with iMCD, I am sadly running out of options, causing my family and I to prepare for hospital treatment,” said Albert.
The Every Cure team identified the medication when Albert started exhibiting the worst symptoms in his two year battle with the iMCD. Within days of taking adalimumab, his organs were functioning again, his symptoms subsided, and he was in remission. The use of adalimumab to treat iMCD will be a new application.
Monoclonal antibodies
Adalimumab is a monoclonal antibody that is typically used to treat severe and often life-debilitating inflammatory conditions such as rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, and Crohn’s disease, among others. It is given by injection under the skin.
Adalimumab was originally approved by the US Food and Drug Administration (FDA) in 2002. Treating iMCD with adalimumab is not an approved use at this time. With this groundbreaking discovery, Every Cure and its research partners are starting to lay the groundwork for conducting clinical trials to further validate its use for iMCD.
Take note of existing drugs
To help scale this case study for other potential uses, Every Cure, a nonprofit dedicated to unlocking the full potential of approved drugs to treat additional diseases, has partnered with researchers at the University of Pennsylvania, Penn State University, and the University of North Carolina, including David Koslicki, Alex Tropsha and Chris Bizon for developing the AI-powered Every Cure LinkMap. The algorithm scores each existing drug potency to treat every known disease based on world knowledge from publications and databases. The Every Cure LinkMap originally ranked all 3,000 drugs that are FDA-approved to treat all 12,000 human diseases, resulting in 36 million evaluations.
Every Cure is currently analyzing the top results from its initial findings on Every Cure LinkMap, in addition to consulting industry experts, to identify the most promising candidates for reuse, including opportunities such as the use of metreleptine for anorexia, folinic acid for autism spectrum disorder, anakinra for sepsis, and bosutinib for ALS. These and other candidates will be further evaluated and prioritized for future clinical trials.
Every Cure is a non-profit organization seeking urgent funding to further optimize its algorithms and evaluate its highest ranking results to take directly to patients. It is also seeking partnerships for data contributions.
Potential future impact
“We are very encouraged by the progress we have made with limited resources that have saved the lives of patients diagnosed with Castleman disease, angiosarcoma, and COVID-19 by reusing existing medicines,” said Every Cure co-founder and CEO, Give Mitchell.
“However, we are even more excited about the potential future impact as we integrate more data, optimize algorithms and investigate our most promising prospects. Our mission is to offer options to patients who have run out of hope. We do so by taking responsibility for ensuring that every drug is used to treat every possible disease. It is a huge responsibility but much needed and we are positioned to do it.”
Each Cure founder and Center for Cytokine Storm Treatment & Laboratory (CSTL) director, Fajgenbaum, announced this momentous breakthrough at the STAT Summit in San Francisco.
“I live thanks to recycled medicine that I found when I was dying of a rare disease during medical school. Now, I am on a mission to unlock every drug so that no patient is told that we have tried everything when a life-saving drug is on the pharmacy shelf,” said Fajgenbaum.
