Hepatocellular Carcinoma (HCC)

Liver cancer is the second leading cause of cancer-related deaths worldwide, with HCC accounting for most liver cancers. The highest incidence of HCC occurs in regions with endemic hepatitis B virus, including Southeast Asia and sub-Saharan Africa. In the United States, HCC is the fastest rising cause of cancer-related death; over the past two decades, the incidence of HCC has tripled while the five-year survival rate has remained below 12 percent. Cirrhosis of the liver, often caused by hepatitis B or hepatitis C infection or alcohol consumption, is a key risk factor for HCC. While patients diagnosed at an early stage can receive potentially curative liver transplants, surgery or ablation therapies, patients with advanced HCC face a poor prognosis, and there is a clear need for more effective treatments.

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Gastrointestinal Stromal Tumors (GIST)

GIST is the most common sarcoma of the gastrointestinal tract, or GI tract. Tumors arise within cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50-80 typically triggered by GI bleeding, incidental findings during surgery or imaging, and in rare cases, tumor rupture or GI obstruction. Symptoms include pain or discomfort in the abdomen, nausea, vomiting, anemia and fatigue. Patients diagnosed at an early stage may undergo surgery. For patients with metastatic disease, although treatment options have improved dramatically in recent years, a meaningful percentage of patients either do not respond to treatment or relapse after treatment, and there is a clear need for effective treatments for these patients.

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Systemic Mastocytosis (SM)

SM is characterized by the abnormal buildup of mast cells – immune cells that produce histamine and other mediators of the body’s inflammatory and allergic responses. In patients with SM, mast cells produce high levels of these mediators, causing symptoms that range from mild to life-threatening, including pain, nausea, hives, bleeding, fever and anaphylaxis. There are several forms of SM – indolent SM and more advanced forms of the disease, including aggressive SM. In the advanced cases, mast cells accumulate in organs such as the bone marrow, spleen and liver, and result in compromised organ function. There are no approved treatments that target the primary driver of disease in most patients, and there is a clear need for new therapies for people with advanced SM and for those with the indolent form of the disease who have a heavy symptom burden.

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Last Updated: November 13, 2015