Acute myeloid leukemia (AML) is the most common acute leukemia in older adults. It is associated with chromosome abnormalities and gene mutations. Only 3% of AML patients do not have some form of mutation. Genetic rearrangements are extremely common.

These abnormalities make AML difficult to treat. If relapse occurs, the prognosis is not favorable. Oftentimes, drugs that are initially successful against AML are ineffective when it returns. In recent years, researchers have discovered that relapsed AML usually expresses high levels of a protein called STAT3.

What Is STAT3

STAT3 is an active protein throughout the body. It is involved in cell growth, division, and death. Studies have shown that it can contribute to cancer cell survival. STAT3 can inhibit diseased cells from dying. After initial treatment, STAT3 can survive on stem cells and eventually help AML to relapse.

A New Drug To Target STAT3

Danvatirsen is a drug compound that specifically targets STAT3 and decreases leukemic growth. A new study is set to investigate the combination of danvatirsen and venetoclax, as well as danvatirsen monotherapy. “After conducting preclinical tests indicating that danvatirsen shows promise against AML, Dr. Shastri is investigating the drug in a phase 1 clinical trial.”

The study (NCT05986240) will begin in August 2023, with primary completion in March 2025. It will test the safety of danvatirsen and the optimal dosage for treatment. "We will investigate the effect of danvatirsen, alone and in combination with venetoclax, on leukemic stem cells."

Side Effects

Danvatirsen binds to STAT3, which promotes antitumor activity. By suppressing the STAT3 protein, tumor cell growth is decreased. This contributes to cancer cell death. Common side effects are:

• Elevated levels of liver enzymes. This can indicate the liver is inflamed or damaged. 
• Low blood platelet count.
• Fatigue.

Conclusion

“High relapse rate of acute myeloid leukemia (AML) is still a crucial problem despite considerable advances in anti-cancer therapies. One crucial cause of relapse is the existence of leukemia stem cells (LSCs) with self-renewal ability, which contribute to repeated treatment resistance and recurrence.”

With the success of this Phase 1 study, danvatirsen may become an effective standard of therapy treatment for AML patients.

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