Chronic myeloid leukemia (CML) is a type of blood cancer that affects the bone marrow and disrupts the production of healthy blood cells.

There have been several recent advances in the treatment of CML that have improved patient outcomes and quality of life. In this article, we will discuss the latest developments in CML treatment and the progress made towards a cure.

Why is CML difficult to treat?

CML is caused by a genetic mutation that results in the overproduction of white blood cells. These excessive white blood cells can accumulate in the bone marrow and other organs, leading to various complications such as anemia, fatigue, and infections.

CML is classified into three phases: chronic, accelerated, and blast. The chronic phase is the earliest and most common phase, and patients can often live with CML for many years without experiencing symptoms.

However, if left untreated, CML may progress to the accelerated or blast phase, which are more difficult to treat and have a worse prognosis.

Because CML is caused by a genetic abnormality, it is not responsive to traditional chemotherapy agents. Instead, CML treatment targets the specific genetic mutation that causes the disease.

The advent of targeted therapy

In the early 2000s, a breakthrough in CML treatment occurred with the development of targeted therapy. Targeted therapy drugs work by inhibiting the activity of a specific protein called BCR-ABL, which is created by the genetic mutation that causes CML.

By inhibiting BCR-ABL, targeted therapy drugs can reduce the overproduction of white blood cells and effectively treat CML.

The first targeted therapy drug approved for CML treatment was imatinib, which was shown to be highly effective in treating CML patients in the chronic phase.

Imatinib has been widely adopted as the first-line treatment for CML and has significantly improved patient outcomes.

New targeted therapy drugs

Since the introduction of imatinib, several new targeted therapy drugs have been developed that have improved upon its efficacy and safety:.

Dasatinib

Dasatinib is a second-generation targeted therapy drug that inhibits both BCR-ABL and other related proteins. It is effective in treating patients who are resistant to imatinib or who have progressed to the accelerated or blast phase of CML.

Dasatinib also has a better safety profile than imatinib and is associated with fewer side effects.

Nilotinib

Nilotinib is another second-generation targeted therapy drug that is highly effective in treating CML patients.

It is similar to dasatinib in that it inhibits both BCR-ABL and related proteins, but it has a different chemical structure and mechanism of action. Nilotinib is associated with fewer side effects than imatinib and is also effective in patients who are resistant to imatinib.

Bosutinib

Bosutinib is a third-generation targeted therapy drug that is effective in treating CML patients who are resistant to imatinib, dasatinib, or nilotinib.

It has a different chemical structure than the other targeted therapy drugs and is able to inhibit BCR-ABL even when it mutates and becomes resistant to other treatments. Bosutinib is associated with a higher incidence of gastrointestinal side effects than the other targeted therapy drugs.

Combination therapy

Combination therapy involves using more than one drug to treat CML in order to improve efficacy and prevent the development of drug resistance.

Combination therapy has been shown to be effective in treating CML patients who are resistant to imatinib or who have progressed to the accelerated or blast phase of the disease.

Several studies have investigated the use of combination therapy with the different targeted therapy drugs. One study found that combination therapy with dasatinib and interferon alpha was more effective than dasatinib alone in treating CML patients.

Another study found that combination therapy with imatinib and the targeted therapy drug omacetaxine was effective in treating CML patients who were resistant to both imatinib and other targeted therapy drugs.

Bone marrow transplantation

Bone marrow transplantation is a type of treatment that involves replacing a patient’s diseased bone marrow with healthy bone marrow from a donor.

Bone marrow transplantation is often used in CML patients who have progressed to the accelerated or blast phase of the disease or who are resistant to targeted therapy drugs.

Since bone marrow transplantation involves significant risks and complications, it is typically reserved for patients with advanced CML who have exhausted other treatment options.

However, bone marrow transplantation can be curative in some cases and can provide long-term remission from CML.

Conclusion

CML treatment has significantly progressed in the last two decades with the development of targeted therapy drugs. These drugs have improved patient outcomes and quality of life and have revolutionized CML treatment.

The ongoing research and development in CML treatment offer hope for a cure for this challenging disease.