chemistry

Targeted molecular therapy for neuroblastoma

Targeted molecular therapy for neuroblastoma is a chemistry topic covered in the lgStudy science library. This page brings together a partial reference excerpt, illustrations, worked examples, real-world applications and a short study plan, so you can understand Targeted molecular therapy for neuroblastoma rather than just read about it. In short: Targeted molecular therapy for neuroblastoma involves treatment aimed at molecular targets that have a unique expression in this form of cancer. Neuroblastoma, the second most common pediatric malignant tumor, often involves treatment through intensive chemotherapy.

Key takeaways

Targeted molecular therapy for neuroblastoma belongs to chemistry; place it in that map before memorising details.
Learn the definition first, then one example that makes the definition concrete.
Connect Targeted molecular therapy for neuroblastoma to a quantity you can measure, compute or draw — that is where exam questions come from.
Reproduce the core statement of Targeted molecular therapy for neuroblastoma from memory before moving on to harder problems.

Reference excerpt

Targeted molecular therapy for neuroblastoma involves treatment aimed at molecular targets that have a unique expression in this form of cancer. Neuroblastoma, the second most common pediatric malignant tumor, often involves treatment through intensive chemotherapy. A number of molecular targets have been identified for the treatment of high-risk forms of this disease. Aiming treatment in this way provides a more selective way to treat the disease, decreasing the risk for toxicities that are associated with the typical treatment regimen. Treatment using these targets can supplement or replace some of the intensive chemotherapy that is used for neuroblastoma. These molecular targets of this disease include GD2, ALK, and CD133. GD2 is a target of immunotherapy, and is the most fully developed of these treatment methods, but is also associated with toxicities. ALK has more recently been discovered, and drugs in development for this target are proving to be successful in neuroblastoma treatment. The role of CD133 in neuroblastoma has also been more recently discovered and is an effective target for treatment of this disease.

Identifying High-Risk Patients High-risk cases of neuroblastoma are difficult to treat, even through intensive chemotherapy. For this reason, molecular targets have been identified and are being developed for treatment in patients who have more difficulty responding to treatment. There are a number of genetic factors that can be used to identify high-risk patients. In neuroblastoma cells, there can be amplification of genomic DNA regions, loss of genomic DNA regions, and genetic abnormalities. All of these factors can contribute to an advanced disease state in high-risk patients. Amplification occurs within a protein called the MYCN oncogene. This protein is amplified in approximately 20% of primary neuroblastoma tumors and is associated with advanced disease state and treatment failure. Loss of genomic regions by deletion can occur at chromosomes 1p and 11q. Loss at 1p is correlated with MYCN amplification and advanced disease state. The loss at 11q is not related to MYCN, but is correlated with adverse patient outcomes. Genetic abnormalities frequently occur in a tumor-suppressor gene called caspase 8. Inactivation of this gene will result in tumor cell survival. Table 1 summarizes the genomic factors used to identify high-risk patients.

Treatment Using Molecular Targets

Anti-GD2 Immunotherapy GD2 is a glycolipid that is expressed on the surface of neuroblastoma cells. It is targeted through immunotherapy in neuroblastoma treatment using monoclonal antibodies. These monoclonal antibodies are used to block GD2 expression, and are thus referred to as anti-GD2 agents. They can be used for tumor-specific therapy because GD2 expression is weak and limited to certain areas in normal human tissue. Therefore, its expression can be easily targeted in tumor cells. While anti-GD2 antibodies are effective in clearing the remaining tumors in neuroblastoma patients, there have also been major toxicities associated with the use of this form of treatment. These toxicities include neuropathic pain, capillary leak syndrome, and hypersensitivity reaction. Anti-GD2 antibodies have been developed for immunotherapy treatment of neuroblastoma and can be grouped into first-generation and second-generation antibodies.

First-Generation: 14G2a ch14.18 3F8 Second-Generation: Hu14.18-IL-2 Hu14.18K332A mAb1A7 All of these antibodies are going through clinical trial processes for the treatment of neuroblastoma. The most extensively studied of these antibodies is ch14.18. Through randomized trials, it has been found that treatment with ch14.18 is most effective when combined with cytokines, such as granulocyte macrophage colony-stimulating factor (GM-CSF) and interleukin-2 (IL-2). This combination therapy improves the outcome of high-risk neuroblastoma, but does not decrease the risk of toxicities. For this reason, the second-generation antibodies have been developed, which have fewer associated toxicities but are continuing trials to determine their therapeutic efficacy.

ALK in Familial Neuroblastoma Mutations in the anaplastic lymphoma kinase (ALK) oncogene can be inherited and are a major cause of neuroblastoma. These mutations occur in approximately 5-15% of neuroblastoma cases. ALK has recently been discovered as a molecular target of chemotherapy in the treatment of neuroblastoma patients. Drugs that target ALK are referred to as ALK inhibitors. ALK is expressed on the surface of neuroblastoma tumor cells, making it easily accessible as a target for cancer treatment. In neuroblastoma patients who do not possess a mutated form of ALK, targeting the non-mutated form of ALK on a tumor cell can also be beneficial. This will cause the tumor to undergo apoptosis, which is programmed cell death. ALK inhibitors can also be used to treat another cause of neuroblastoma referred to as MYCN gene amplification. Amplification of the MYCN protein is a genetic mutation associated with neuroblastoma tumors. MYCN amplification is correlated with a specific mutation in ALK, referred to as the F1174L mutation. ALK inhibitors can target this mutation and suppress the MYCN protein in the tumor cell. The following is a list of ALK inhibitors currently in clinical trials for treatment of neuroblastoma:

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Worked examples

Example 1 — a first encounter with Targeted molecular therapy for neuroblastoma

Start with the simplest possible case. Write down what Targeted molecular therapy for neuroblastoma claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In chemistry, the smallest case is usually a single object, a single equation or a single measurement. Check that every symbol or term in your sentence has a meaning in that case.

Example 2 — changing one variable

Take the situation from Example 1 and change exactly one quantity: double it, halve it, or set it to zero. Predict what should happen to Targeted molecular therapy for neuroblastoma before you calculate. Comparing your prediction with the result is the fastest way to find out whether you understand the idea or only the words.

Example 3 — an exam-style question

Typical questions about Targeted molecular therapy for neuroblastoma ask you to (a) state it precisely, (b) apply it to given data, and (c) explain a limitation. Practise writing all three answers in under five minutes; the third part is what separates a full-mark answer from an average one.

Applications of Targeted molecular therapy for neuroblastoma

In research: Targeted molecular therapy for neuroblastoma appears in chemistry research whenever the underlying quantities have to be modelled precisely. Papers usually cite it as a starting assumption and then explore where it breaks down.
In technology and industry: Engineering practice reuses Targeted molecular therapy for neuroblastoma in design rules, simulations and safety margins. Knowing the idea lets you read a specification sheet and understand why the numbers look the way they do.
In the classroom: Targeted molecular therapy for neuroblastoma is common in secondary-school and first-year university syllabi. It links to neighbouring topics Targeted therapy, so understanding it makes those chapters shorter.
In everyday life: Look for Targeted molecular therapy for neuroblastoma outside the textbook — in sport, cooking, traffic, electronics or the sky above you. An example you found yourself is remembered far longer than one you were given.

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How to study Targeted molecular therapy for neuroblastoma in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Targeted molecular therapy for neuroblastoma means in your own words.
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Frequently asked questions

What is Targeted molecular therapy for neuroblastoma in simple terms?

Why does Targeted molecular therapy for neuroblastoma matter?

Because it connects several chemistry ideas at once: it gives you a definition you can apply, a quantity you can calculate, and a way to check whether a result is plausible.

How should I study Targeted molecular therapy for neuroblastoma?

Read the excerpt, restate it from memory, then work through the examples and applications listed on this page. The five-step study plan above takes about twenty minutes.

What does this page cover?

It gives you a compact reference excerpt plus original lgStudy explanations, examples, applications and study material on Targeted molecular therapy for neuroblastoma.