Other antineoplastic agents

Table of contents

• What is other antineoplastic agents?
• How does other antineoplastic agents work?
• Which conditions is other antineoplastic agents used or studied for?
• How is other antineoplastic agents given?
• What do the clinical trials show?
• What side effects are known?

What is other antineoplastic agents?

Other antineoplastic agents is a category of medicines that includes anticancer drugs which do not fit neatly into other, more specific classes. Antineoplastic medicines, also called anticancer drugs, affect how cells grow and divide, meaning they are antiproliferative. They work through different mechanisms to prevent the development and spread of neoplastic cells, which are abnormal cells that can form tumours.^[3]

One substance that falls under this category is imetelstat, the active substance investigated in the clinical trial described on this page. Imetelstat is being studied for the treatment of myelofibrosis, a rare type of bone marrow cancer in which scar tissue builds up inside the bone marrow and disrupts normal blood cell production.

Other medicines listed in this broad category include isotretinoin, lenalidomide, and abiraterone, which are used for conditions ranging from acne and anaemia to multiple myeloma and prostate cancer.^[3] The category therefore covers a wide range of substances with different chemical structures and clinical uses.

How does other antineoplastic agents work?

Because this is a broad category rather than a single chemical family, the exact mechanism of action depends on the individual substance. In general, antineoplastic agents prevent the growth and spread of cancer cells by interfering with cell division. They also affect rapidly dividing normal cells, which is why they can suppress the bone marrow, slow growth, impair healing, cause sterility, and lead to hair loss.^[3]

The clinical trial data for this substance group concerns imetelstat, which is given intravenously at a dose of 9.4 mg per kilogram of body weight.^[1] The trial record does not describe the molecular target of imetelstat in detail, but it is being compared against best available therapy for people with myelofibrosis whose disease has relapsed after or is refractory to treatment with JAK inhibitors.

Which conditions is other antineoplastic agents used or studied for?

The conditions studied or treated with medicines in this category vary widely depending on the specific substance. The clinical trial data provided here focuses on one condition:

• Myelofibrosis — a rare bone marrow cancer in which scar tissue forms in the bone marrow, leading to problems with normal blood cell production. The trial investigates imetelstat in people with intermediate-2 or high-risk myelofibrosis whose disease is relapsed or refractory to JAK-inhibitor treatment.^[1]

More broadly, medicines classified as miscellaneous antineoplastics are used or studied in many other conditions, including acute myeloid leukaemia, acute lymphoblastic leukaemia, breast cancer, colorectal cancer, multiple myeloma, melanoma, non-small cell lung cancer, ovarian cancer, and prostate cancer, among others.^[3]

How is other antineoplastic agents given?

The way a medicine in this category is given depends on the specific substance and its formulation. In the clinical trial described on this page, imetelstat is given as an intravenous infusion, meaning it is delivered directly into a vein. The dose used in the trial is 9.4 mg per kilogram of body weight.^[1]

Other antineoplastic agents come in different forms. For example, daunorubicin hydrochloride, another medicine with antineoplastic activity, is provided as an injection that must be given into a rapidly flowing intravenous infusion and must never be given by intramuscular or subcutaneous injection because severe local tissue damage can occur if the medicine leaks outside the vein.^[2] This illustrates that the route of administration for antineoplastic agents depends on the specific medicine and its formulation.

What do the clinical trials show?

The clinical trial data available for this substance group includes one Phase 3 trial investigating imetelstat in myelofibrosis. Phase 3 trials are late-stage studies that compare a new treatment against current standard treatments to see whether it works better or is safer.

The trial enrolled 354 participants with intermediate-2 or high-risk myelofibrosis whose disease is relapsed or refractory to JAK-inhibitor treatment.^[1] The study is currently authorised, meaning it has received regulatory approval to proceed.

The primary outcome measured in the trial is overall survival, defined as the time interval from the date of randomisation to the date of death from any cause.^[1] This means the study is looking at whether people treated with imetelstat live longer than people receiving best available therapy. The trial compares imetelstat against best available therapy, which is the current standard treatment for this condition.

What side effects are known?

Side effects vary depending on the specific antineoplastic medicine. For the broad category of miscellaneous antineoplastics, the known effects on rapidly dividing normal cells mean these medicines are likely to suppress the bone marrow, suppress growth, impair healing, cause sterility, and cause hair loss.^[3]

For daunorubicin hydrochloride, a specific medicine with antineoplastic activity, the product label describes several important side effects. Severe myelosuppression occurs when the medicine is used at therapeutic doses, which can lead to infection or haemorrhage. Myocardial toxicity, which in its most severe form can be potentially fatal congestive heart failure, may occur during therapy or months to years after treatment ends. The risk of myocardial toxicity increases after a total cumulative dose exceeding 400 to 550 mg per square metre in adults.^[2]

These side effects are specific to daunorubicin and may not apply to other substances in the antineoplastic category. The clinical trial record for imetelstat does not provide a side effect profile in the data available here.