Clinical Trial Will Test Combination Treatment for Erdheim-Chester Disease

A new clinical trial which will test a two-part combination treatment for Erdheim-Chester disease was recently added to the clinicaltrials.gov website. This trial will be specifically for patients with BRAF V600 gene mutations. The two drugs that will be administered are dabrafenib and trametinib. This Phase II clinical trial will be sponsored by the National Cancer Institute (NCI).

About Erdheim-Chester disease

Erdheim-Chester disease, which is also known as polyostotic sclerosing histiocytosis, is a rare disease which is characterized by the abnormal production and accumulation of histiocytes, a type of white blood cell. These cells infiltrate the bone marrow, which can cause the long bones of the body to stiffen, replacing normal organ tissue with connective tissue. The cause of Erdheim-Chester disease remains unknown; only about 500 cases have been recorded so far. Symptoms of the illness include liver failure, kidney failure, bone pain, hypopituitarism (reduction in secretion of hormones), shortness of breath, diabetes insipidus, bulging eyes, and ataxia. Treatment for this disease may include corticosteroids, surgical debulking, ciclosporin, interferon, radiation therapy, chemotherapy, and the drug vemurafenib. The five year survival rate for Erdheim-Chester disease around 68 percent. Survival rates have been improving in recent years. To learn more about Erdheim-Chester disease, click here.

About The Clinical Trial

This approximately year-long study will involve a total of 18 patients who have Erdheim-Chester disease with BRAF V600 mutations. The trial will monitor the safety and effectiveness of dabrafenib and trametinib when used in combination, and will assess the overall clinical response rate, duration of response, survival without disease progression, and overall survival. Patients will receive dabrafenib twice daily and trametinib once per day during twelve 28 day treatment periods. 

About Dabrafenib and Trametinib

Dabrafenib is an inhibitor of the enzyme B-Raf, which is associated with the BRAF gene. This enzyme plays a role in cell growth, and the drug was first approved for the treatment of melanoma with this mutation. Trametinib, and inhibitor of MEK, is also used to treat melanoma. However, melanoma develops resistance to both of these drugs in about six months. As a result it is now standard to use these drugs together to achieve a more durable and long-lasting disease response. Hopefully this combination will be able to make an impact in patients with BRAF mutated Erdheim-Chester disease as well.


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