SAN ANTONIO, Texas (Ivanhoe Newswire) – A new drug, one of a few fast tracked by the FDA last year, may help thousands of people live with leukemia.
Fast track – it’s when the FDA approves a drug months, sometimes even a year, faster than the normal process. In fact, some fast tracks take just days to receive approval. Last year, the FDA approved 50 new drugs and 18 of them received fast track status. One of those drugs is being used for the first time to treat patients suffering from a rare cancer that has very few treatment options.
“Myelofibrosis is a chronic leukemia. It affects about 20,000 patients. It can progress to acute leukemia and sadly can be a fatal disease,” explains hematologist at UT Health San Antonio MD Anderson Cancer Center, Dr. Ruben Mesa.
Myelofibrosis is one of 40 chronic leukemias. It’s an uncommon type of bone marrow cancer that causes an enlarged spleen and disrupts a body’s normal production of blood cells.
Dr. Mesa played a key role in the fast track approval by the FDA for Pacritinib. The oral capsule is the first therapy approved for myelofibrosis patients with low platelet counts.
“People could have improvements in spleen and symptoms and two, that the blood counts stayed the same or improved, which was a significant benefit over our other historical therapies,” Dr. Mesa further explains.
And although the new drug has been fast-tracked and approved quickly by the FDA, the process to get here has been anything but fast. Dr. Mesa has been studying it for more than a decade.
Traditionally, aggressive treatment for myelofibrosis included bone marrow transplants but that can only be done in 10 percent of the cases. That’s why it’s so important to find new medicines that will help patients.
Contributors to this news report include: Marsha Lewis, Producer; Roque Correa, Videographer, Editor.
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TOPIC: FAST TRACKED LEUKEMIA DRUG SAVES LIVES
REPORT: MB #5075
BACKGROUND: Leukemia is cancer of the body’s blood-forming tissues, including the bone marrow and the lymphatic system. In 2022, The American Cancer Society has predicted that around 61,000 people will be diagnosed with leukemia and an estimated 24,000 will die from it. Many types of it exist. Some forms of it are more common in children. Other forms of leukemia occur mostly in adults. Treatment for leukemia can be complex — depending on the type of leukemia and other factors. But there are strategies and resources that can help make your treatment successful.
DIAGNOSING: Different types of leukemia can cause different problems. You might not notice any signs in the early stages of some forms. When you do have symptoms, they may include weakness, bruising or bleeding easily, weight loss, night sweats, or swollen lymph nodes. A diagnosis of leukemia is usually made by analyzing a patient’s blood sample through a complete blood count (CBC) or microscopic evaluation of the blood, or by using flow cytometry.
NEW THERAPY: CAR T-cell therapy has recently generated great excitement for the treatment of children with relapsed ALL. One CAR T-cell therapy, tisagenlecleucel (Kymriah), was approved in 2017 for some children with relapsed acute lymphoblastic leukemia, or ALL. CAR T-cells are currently only approved for use in leukemia that has relapsed or proved resistant to standard treatment. A clinical trial from COG is now testing tisagenlecleucel as part of first-line therapy in children with ALL at high risk of relapse.
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