January 17th 2017

Propranolol granted Orphan Drug Designation by the European Commission.

A drug commonly used to reducehigh blood pressurereceived Orphan Drug Designation by the European Commission (EC) to treat soft tissue sarcoma.

The ability of propranolol to treat angiosarcoma—–a lethal form of soft tissue sarcoma––was first discovered by Brad Bryan, PhD, in a study that used cell lines and animal models. The findings, published inPLOS Onein 2013, showed that propranolol could fight angiosarcoma and significantly reduce tumor growth.

In another study published inJAMA Dermatologyin 2015, Bryan treated a patient with terminal angiosarcoma, and found that propranolol brought the tumor down to undetectable levels. Furthermore, there were little to no adverse events.

Since then, some scientists around the world have tested propranolol on their own patients with angiosarcoma, and they have reported similar results.

In a study conducted by Dr Shripad Banavali, MD, and investigator Eddy Pasquier, PhD, a patient with metastatic angiosarcoma was treated with propranolol and made a full recovery. A year later, in a study published inEBioMedicine, investigators successfully treated 7 patients with inoperable angiosarcoma.

“What surprised us the most about this new treatment is the fact that we got 100% clinical response, which is defined as either tumor regression or stabilization of the disease,” Pasquier said. “This is not a cure in the sense that most patients will eventually see their disease progression, but this level of response is still very impressive, especially in this patient population with a very bleak prognosis; we’re talking patients whose prognosis was roughly 1 year, give or take a few months.”

The results helped push the nonprofit foundation Anticancer Fund, to gather research on propranolol’s effects, and it applied to have the drug approved as an orphan drug in Europe.

“Our ultimate objective is to have propranolol, if proven effective, fully licensed as a standard of care treatment for angiosarcoma,” said Pan Pantziarka, PhD, oncology research at the Anticancer Fund. “Bryan’s research was important because not only did it demonstrate the validity of this approach in animal models, but it also illustrated the effect of treatment in a patient for whom existing treatments are unlikely to be effective. These 2 elements were important in making the case for the clinical potential of propranolol in this rare and hard-to-treat cancer.”

As the high cost of drugs continues to stir criticism, propranolol could potentially offer a cheaper treatment option for patients with angiosarcoma. Propranolol is a generic drug that costs approximately $4 per month.

“Treating soft tissue sarcoma can easily top $100,000 to $200,000,” Bryan said. “While propranolol will certainly not replace these treatments, our data show it improves the ability of the treatments to work—–all at the cost of a generic copay.”

Recently, the Anticancer Fund formed the Propranolol for Angiosarcoma Task Force to bring together scientists and clinicians who are interested in further developing propranolol treatment options.

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