A New Hope for Rare Bone Disease

It’s called Hereditary multiple exostoses (HME). Yes, quite the mouthful. Our friendly neighborhood scientists at Children’s Hospital of Philly (CHOP) have been working diligently to study this rare bone disease and have finally uncovered a new hope in treating it.

There’s been a drug candidate hiding in the shadows that blocks abnormal proteins and could possibly eliminate the risk of cancer and avoid patients from having to undergo traumatizing childhood surgeries.

HME is a very painful disease especially toward children. It comes as several bone deformities in the form of lumps or spurs. To learn more about the nitty gritty details of HME, click here.

Maurizio Pacifici, Ph.D., a developmental biologist and director of Orthopaedic Research at CHOP was optimistic about the trials.

“Our study is the first to provide proof-of-principle evidence that the formation of osteochondromas can be inhibited by systemic drug treatment in animal models of HME/MO. This is a first critical step that provides the rationale to conceive and organize a clinical trial in the future.”

There are currently no treatments for HME and victims are suffering from deformed osteochondromas in their skeletons that could potentially turn malignant. In CHOP’s study, they found a savior compound that battles tumors in animal models of the disease. This very compound could be the saving grace for HME, as the first treatment in history.

These animal models were the first to provide cut and dry evidence that osteochondromas can be hindered by drug treatment. Through surgery, the severe osteochondromas are removed but some stay behind, unable to be removed and create long lasting burdens for patients.

People suffering with HME can now hope to participate in exercise and sports without experiencing chronic pain. Time and further tests will tell.
Source: Giphy

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