Histiocytosis Clinical Trials & Research

Histiocytosis Clinical Trials & Research

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MSK Medical Oncologist, David Hyman, examining female patient's hand for Erdheim-Chester disease.

Medical oncologist David Hyman has overseen clinical trials for Erdheim-Chester disease.

Some of the advances in histiocytosis treatment have been led by doctors and researchers at MSK. This includes a trial led by MSK that resulted in the US Food and Drug Administration’s approval of the drug vemurafenib (Zelboraf®) for the treatment of Erdheim-Chester disease. Vemurafenib is in a class of drugs called BRAF inhibitors.

MSK researchers have identified many other gene mutations beyond BRAF in histiocytosis tumors. These findings have provided a new understanding of these conditions — and led to new potential treatments.

You can see a list of clinical trials for histiocytosis that are currently open at MSK here.

In addition to clinical trials, there is other research on histiocytosis ongoing at MSK.

The genetics of histiocytosis

MSK is the leading center for research about the genetics of histiocytosis. We are applying these discoveries to develop new treatments. Our team has several ongoing projects to learn more about the gene mutations that cause histiocytosis and other blood cancers. We are also studying new noninvasive methods to look for histiocytosis-related mutations. These include examining blood and urine.

Learn more about treatments that are available for histiocytosis.

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Erdheim-Chester Disease Registry

For people with Erdheim-Chester disease (ECD), MSK leads a registry study that follows people over time. This allows us to examine what treatments have been effective for different forms of ECD. The registry also investigates issues that are important to people with the disease, such as symptoms, side effects of treatment, and quality of life. Participants complete surveys about their experiences from time to time.

Read an article about ECD symptoms based on this registry.

See the study description or join the study online. People with ECD who are not MSK patients are able to join as well.

Brain imaging and function in histiocytosis

Many people with histiocytosis experience problems with thinking and memory. We have a study that examines brain function in people with histiocytosis, in order to learn more about these symptoms. The study involves MRI scans of the brain and written tests that look at concentration, language, attention, and memory.

Caring for someone with histiocytosis

Caregivers may be friends or family members who provide unpaid help and support to people with cancer and other illnesses. Because histiocytosis is a rare and difficult disease, being a caregiver for a someone with histiocytosis brings unique challenges. At MSK, we are trying to understand the experience of being a histiocytosis caregiver so we can learn how to better support caregivers’ needs. If you would like more information about this study, email neuhistiores@mskcc.org.

For more information about ongoing laboratory research on histiocytosis at MSK, visit the laboratory web pages for Omar Abdel-Wahab and Frederic Geissmann.