New Use for Old Drugs Brings Hope to Rare Disease Patients

 

The new nonprofit Every Cure has been created on the basis that every drug currently on the market effects multiple areas in the body. This premise has been proven many times by the number of drugs that are being used beyond their original purpose and treat multiple diseases. Every Cure hopes to expand on that concept to help people with rare diseases.

David Fajgenbaum, M.D. a co-founder of Every Cure, will announce the launch of the organization this week at the annual meeting of the Clinton Global Initiative (CGI). The theme will be Turning Hope Into Action. Leaders from countries around the world are expected to be in attendance.

According to an article in USA News Today, Dr. Fajgenbaum commented that to date, no one has accepted the responsibility to ensure that drugs now on the market are being fully utilized. He said that Every Cure is going to fulfill that role. Every time a patient is cured, an opportunity is created for a repurposed drug to open a new pathway of hope for rare disease patients.

A simple definition of repurposing is that it involves FDA approved drugs that have the potential to be effective in treating diseases for which they are not approved currently.

The Clinton Global Initiative backs Every Cure in its endeavor to find generic drugs that show promise in treating patients with rare diseases. Another goal of the non-profit is to raise $55 million towards coordinating drug data and investigating those drugs in clinical trials.

Many people can still remember the severe birth defects suffered by newborn babies in the 1960s. Thalidomide had been given to pregnant women to treat nausea. However, it surfaced again as a safe and effective therapy to treat inflammation in leprosy and even multiple myeloma, a rare cancer.

Another drug, Tocilizumab, was initially FDA-approved to treat rheumatoid arthritis in 2010. Tocilizumab was then rediscovered in the treatment of severe cases of COVID-19.

Sildenafil citrate (Viagra) was first created not for erectile dysfunction but to treat children who had a rare type of lung disease. It was later found useful for reducing high blood pressure and treating erectile dysfunction.

Dr. Fajgenbaum is personally familiar with the concept of repurposing drugs. Eight years ago he and the current CEO of Every Cure, John Paul Mitchel, repurposed sirolimus, a kidney transplant drug that saved Dr. Fajgenbaum’s life. The medicine was able to successfully treat his Castleman’s disease.

In his capacity as a consultant, Mitchell used machine learning to find new uses for already approved drugs. Although he was only identifying one drug at a time, he understood what can be accomplished through technology. Both men attended the Wharton business school and both received an award for their drug repurposing business plan.

That was where it ended until they realized their concept had a better chance of success if they worked as a non-profit targeting diseases and drugs that other companies were neglecting.

Dr. Fajgenbaum has a type of Castleman’s Disease whereby the body’s vital organs are attacked by his own immune system. Although sirolimus had not been studied in connection with his specific disease, using his own samples and information from medical literature, he decided to try the drug.

An Oversimplification

It may sound easy to say researchers are going to repurpose drugs that already have FDA approval. However, Dr. Fajgenbaum readily acknowledges that there are a few road blocks that must first be overcome if they intend to repurpose any of the three thousand approved drugs.

The first understandable roadblock is that there is not much financial incentive for a company to conduct the appropriate studies while shouldering the inherent risk.

The second roadblock is the lack of data about the drug’s activity. There is no central, accessible database.

Data on a drug’s activity, even if it’s publicly available, isn’t in a central, readily accessible database.

Dr. Fajgenbaum further stated that about ninety percent of FDA-approved drugs have a generic status, but no person or organization has taken the responsibility of identifying other potential uses.

It is estimated that there are approximately 9,000 rare diseases. Every Cure has moved ahead and, facing a mountain of rare diseases, began its enormous task by identifying 106 drugs with the potential to treat 147 rare diseases.

The problem, as Dr. Fajgenbaum explains, is not finding the links between rare diseases and drugs but how to determine which drugs to (a) investigate in clinical trials, (b) how best to cover costs, and most of all (c) to ensure the patients with rare diseases derive benefit from the drugs now on pharmacy shelves.

With cost looming as a critical factor, Every Cure is seeking partners who can bring about cost reduction and help to facilitate the process. Its goal is to raise about ten million dollars in order to create an open-access algorithm (calculations) that will identify repurposing opportunities. They will need another forty-five million to fund clinical trials.

However, the future looks promising as the partners believe they can change the system by fulfilling a tremendous need.

 

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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