Acute Myelogenic Leukemia (AML) is a type of cancer that starts from the bone marrow and affects the blood cells. AML progresses rapidly, and it is the most common type of acute leukemia affecting adults.

Although there have been some advances in the treatment of AML, the prognosis remains poor, with only a 30-40% survival rate among patients. However, researchers have recently discovered new hope for AML patients with the development of gene-targeted medicines.

Understanding Gene-Targeted Medicines

Gene-targeted medicines are a type of precision medicine that specifically target genetic abnormalities associated with a particular cancer.

These medicines aim to block the growth and spread of cancer by interfering with the function of specific genes or proteins involved in the development and progression of the disease. The use of gene-targeted medicines has revolutionized cancer treatment, as they have shown to be more effective and better tolerated than conventional chemotherapy.

The Discovery of Gene-Targeted Medicines for AML

AML is caused by mutations in various genes, which alter the normal function of blood cell production and differentiation. Researchers have discovered specific gene mutations that are commonly found in AML patients, including FLT3, IDH1, IDH2, and NPM1.

These mutations lead to the overproduction of immature cells, which impair the production of normal blood cells, leading to anemia, infection, and bleeding.

Researchers have identified gene-targeted medicines that can block the function of these mutated genes, thereby inhibiting the growth and spread of cancer cells.

For instance, midostaurin is a gene-targeted medicine that blocks the FLT3 gene, which is found in around 30% of AML patients. Midostaurin has shown to improve survival rates among AML patients when combined with standard chemotherapy.

Similarly, ivosidenib and enasidenib are gene-targeted medicines that block the IDH1 and IDH2 genes, respectively, which are found in about 20% of AML patients. These medicines have shown to reduce the abnormal accumulation of immature cells and promote the production of healthy blood cells.

Clinical Trials of Gene-Targeted Medicines for AML

The development of gene-targeted medicines for AML is still in the early stages, and more clinical trials are needed to evaluate the safety and efficacy of these medicines.

However, the results from the initial phase II clinical trials of these medicines are promising, and they have shown to provide better outcomes for AML patients.

In a phase II clinical trial of ivosidenib, AML patients with IDH1 mutations were given the medicine once a day for 28-day cycles. About 41.6% of patients showed a complete remission of cancer, and 21.6% showed a partial remission.

The progression-free survival rate was 9.3 months, and the 6-month overall survival rate was 86.6%. Similarly, in a phase II clinical trial of enasidenib, AML patients with IDH2 mutations were given the medicine once daily. About 40.3% of patients showed a complete remission, and 19.3% showed a partial remission.

The median duration of response was 8.2 months, and the overall survival rate was 34.8% at 12 months.

Challenges Ahead for Gene-Targeted Medicines for AML

Although gene-targeted medicines have shown promising results in the treatment of AML, there are still some challenges that need to be addressed before they can become widely available.

One challenge is the high cost of these medicines, which may limit access to those who need them, especially in low- and middle-income countries. Another challenge is the development of resistance to these medicines, which may limit their long-term effectiveness. Therefore, more research and development are needed to improve the safety and efficacy of gene-targeted medicines for AML.

Conclusion

The development of gene-targeted medicines for AML has brought new hope for patients suffering from this aggressive form of leukemia.

Although these medicines are still in the early stages of development, they have shown promising results in clinical trials and provide a more effective and better-tolerated alternative to conventional chemotherapy. Further research and development are needed to address the challenges of cost and resistance and to make these medicines widely available to patients in need.