Jatin Shah, MD, PhD, of Sumitomo Pharma, discusses two treatments in Phase 1 trials for myelofibrosis and acute myeloid leukemia.
Jatin Shah, MD, PhD, of Sumitomo Pharma, discusses two treatments in Phase 1 trials for myelofibrosis and acute myeloid leukemia.
Myelofibrosis
Primary myelofibrosis is characterized by the buildup of scar tissue in the bone marrow responsible for producing blood cells. Because of the buildup, the bone marrow is unable to make enough normal blood cells. This leads to anemia, weakness, fatigue, and often, swelling of the liver and spleen. While patients are typically above the age of 60 and there are higher incidences in males, no other established risk factors are present. Traditionally, treatment for this disease has included an allogeneic bone marrow transplant or JAK inhibitors.
As discussed by Dr. Shah, TP-3654 is an oral investigational inhibitor currently in a Phase I clinical trial for the treatment of myelofibrosis. The trial involves patients with intermediate and high-risk myelofibrosis who have previously been treated with or ineligible for a JAK inhibitor.
The treatment has been well tolerated and demonstrates limited drop in blood counts. Side effects have also been low-grade and manageable. Reductions in splenic volume have also been observed, a key measure in myelofibrosis treatment.
Currently, the study is focusing on establishing the best dosage and enrolling more patients. From there, researchers will look into the best combination of therapies utilizing TP-3654 and JAK inhibitors.
Acute Myeloid Leukemia (AML)
Acute myeloid leukemia is a cancer that affects the blood and bone marrow. The signs and symptoms vary but may include:
- easy bruising
- bone pain or tenderness
- fatigue
- fever
- frequent nosebleeds
- bleeding from the gums
- shortness of breath
- weight loss
The cancer is one of the most common types of leukemia among adults and is rarely diagnosed in people under age 40. There are many potential causes such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop acute myeloid leukemia have no identifiable risk factor. Standard of care for AML has traditionally included standard cytotoxic chemotherapy, FLT3 inhibitors, and allogeneic stem cell transplants.
DSP-5336 is a targeted therapy in a Phase I clinical trial. Its focus is on patients with NPM1 mutations or MLLR rearrangements. The drug is currently being evaluated in patients with relapsed AML. DSP-5336 has demonstrated adequate safety, with few low-grade, manageable side effects
As Dr. Shah explains, next steps in this trial include establishing the correct dosage, balancing side effects and efficacy, and enrolling more patients. Researchers will also begin looking at best combinations for treatment with DSP-5336 and other therapies.
For more information on these and other rare cancers, visit https://checkrare.com/diseases/cancers/
