Broad Institute Embarks on Holy Grail Quest to Harness Old Compounds for Rare Diseases

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It is an understatement to say there are a lot of different types of diseases in the world. Drug researchers and manufacturers focus their resources on many of these illnesses, but some of the rarest diseases can slip by without much in the way of developed treatments.

The Broad Institute of MIT and Harvard is looking to address that shortfall of research. The nonprofit organization is taking bold steps to acquire samples of every drug ever developed in order to test whether or not they could be used off-label to treat other diseases than the ones for which they were manufactured, the Boston Globe reported. Off-label use of medicines is nothing new, but this intense focus could be life-changing for individuals with the rarer diseases. The Broad Institute is making an effort to collect between 10,000 and 11,000 different compounds that have been discovered since the end of the 19th century, the Globe said. Many, or perhaps the majority of these, were never commercialized due to safety issues or general lack of efficacy.

Some companies, such as those founded by biotech entrepreneur Vivek Ramaswamy, are based on attempting to give new life to older or sometimes failed medications. The Broad Institute told the Boston Globe that the Drug Repurposing Hub “will be home to the largest collection of medicines ever assembled to find new potential uses.” The pathway to regulatory approval could be shorter for some of the drugs that have already been approved by the U.S. Food and Drug Administration (FDA).

Currently, the Broad Institute has acquired approximately 4,700 different compounds for its Drug Repurposing Hub, the Globe said. Of those, about 2,000 have been approved by the FDA.

Not only has the Broad Institute collected the drugs, it has also created an online index that includes detailed summaries of the medications for researchers to use. So far dozens of researchers across the globe have expressed interest in the compounds, the Globe said.

Todd Golub, a cancer specialist and the Broad Institute’s chief scientific officer, told the Globe that the researchers are not smart enough to know which drug will work for which disease, so they will test them all. The researchers will test the compounds against a “variety of disease cells, including hundreds of strains of cancer at once.” Many of those strains of cancers are rare, so little work has been done regarding advancing therapeutics for them.

While the work will take some time, so far scientists at the Institute believe they have already identified some older medicines that can help treat more common serious diseases, including breast cancer and colorectal cancer, the Globe said. Still, the researchers are hoping for a “Holy Grail” compound that will prove to be the cure for a rare disease.

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