New treatment approved for rare blood cancer

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Have you ever heard of Erdheim-Chester Disease? It’s a rare but deadly form of blood cancer – but fortunately a new drug to treat this disease was recently approved.

What is Erdheim-Chester disease?

Erdheim-Chester Disease is a slow-growing cancer that originates in bone marrow, causing a spike in a type of white blood cell called a histiocyte. This can spur tumours that develop in the heart, lung, brain and elsewhere, the FDA said in a news release. The cancer only affects about 700 people worldwide, about half of whom have the BRAF V600 mutation.

The disease often affects the long bones (in the arms or legs), but it can also occur in the tissue behind the eyeballs, kidney, skin, brain, lungs, heart or pituitary gland.

What are the symptoms?

The symptoms of Edrheim-Chester Disease are hard to pinpoint, but often involve bone pain, liver failure and kidney failure. The symptoms will present depending on what part of the body is affected.

In some individuals, the pituitary gland can be infiltrated, leading to a metabolic condition called diabetes insipidus, which causes the insufficient distribution of the antidiuretic hormone, causing a person to pass large amounts of urine.

Zelboraf (vemurafenib) has been approved by the US Food and Drug Administration as the first drug to treat Erdheim-Chester Disease, a rare but deadly blood cancer. The approval covers patients who have a genetic mutation called BRAF V600.

Life expectancy is short among patients, the agency said.

What the drug does

“This [drug] was first approved in 2011 to treat certain patients with melanoma [skin cancer] that harbour the BRAF V600 mutation, and we are now bringing the therapy to patients with a rare cancer with no approved therapies,” said Dr Richard Pazdur, director of the agency’s Oncology Center of Excellence.

Zelboraf is a kinase inhibitor that’s designed to inhibit enzymes that spur cancer cell growth. The most common side effects include joint pain, skin rash, hair loss, fatigue and heart problems, the FDA said. Pregnant women shouldn’t take the drug, since it could harm a developing foetus, the FDA said.


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