Illustration of superhero battling neuroblastoma with AZD8055 molecules.

Can AZD8055 Stop Neuroblastoma? Promising Research on a Novel Inhibitor

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Kai Mendoza in Health & Wellness December 2025 • 4 min read.

"A New Study Explores How AZD8055 Targets mTOR to Combat Neuroblastoma Cell Growth and Improve Outcomes."


Neuroblastoma (NB) remains a formidable challenge in pediatric oncology, with high-risk cases often proving lethal despite aggressive treatments. Researchers are constantly seeking new ways to improve outcomes for these young patients, and one promising area of exploration involves targeting specific pathways within cancer cells that fuel their growth and survival.

A critical pathway in NB is the PI3K/Akt/mTOR signaling cascade. This pathway is often hyperactive in neuroblastoma, contributing to oncogenesis, treatment resistance, and poor prognosis. The mTOR (mammalian target of rapamycin) protein, a central component of this pathway, plays a vital role in cell growth and metabolism, making it an attractive therapeutic target.

Recent research has focused on AZD8055, a potent dual mTORC1/mTORC2 inhibitor. This article explores the potential of AZD8055 in combating neuroblastoma, drawing upon a study that investigated its effects on NB cells both in vitro (in the lab) and in vivo (in animal models). We'll break down the science and discuss what this could mean for future neuroblastoma treatment.

How AZD8055 Targets and Disrupts Neuroblastoma Cell Growth

Illustration of superhero battling neuroblastoma with AZD8055 molecules.

The study revealed that mTOR signaling is extensively activated in neuroblastoma cells. When these cells were treated with AZD8055, the activity of mTOR and its downstream molecules was significantly reduced. This disruption had a profound impact on the cancer cells' behavior.

Specifically, AZD8055 was shown to:

  • Inhibit cell growth effectively.
  • Induce cell cycle arrest, preventing the cancer cells from dividing and multiplying.
  • Promote autophagy, a process where cells essentially "self-eat" damaged components, and apoptosis, or programmed cell death.
These findings suggest that AZD8055 can effectively halt the progression of neuroblastoma by interfering with its fundamental growth processes. The research team also tested AZD8055 in mice with neuroblastoma tumors (xenograft model). The results were encouraging: AZD8055 significantly reduced tumor growth without causing apparent toxicity to the animals. This is a crucial finding, as many cancer treatments come with harsh side effects.

The Future of AZD8055 in Neuroblastoma Therapy

This research provides a strong rationale for further investigating AZD8055 as a potential treatment for neuroblastoma. Its ability to target mTOR, disrupt cancer cell growth, and reduce tumor size in preclinical models is highly promising.

While these findings are encouraging, it's important to remember that this research is still in its early stages. Clinical trials in humans are needed to confirm the safety and efficacy of AZD8055 in treating neuroblastoma. These trials will help determine the optimal dosage, identify potential side effects, and assess how well AZD8055 works in combination with other cancer therapies.

AZD8055 represents a significant step forward in the ongoing quest to find more effective and less toxic treatments for neuroblastoma. By targeting a key pathway that drives cancer cell growth, this novel inhibitor holds the potential to improve outcomes and offer new hope to children and families affected by this challenging disease.

About this Article -

This article was crafted using a human-AI hybrid and collaborative approach. AI assisted our team with initial drafting, research insights, identifying key questions, and image generation. Our human editors guided topic selection, defined the angle, structured the content, ensured factual accuracy and relevance, refined the tone, and conducted thorough editing to deliver helpful, high-quality information.See our About page for more information.

Everything You Need To Know

1

What is AZD8055, and what does it do in relation to neuroblastoma?

AZD8055 is a dual mTOR inhibitor, a type of drug that targets the mTOR protein. This protein is a key part of the PI3K/Akt/mTOR signaling cascade, which is often overactive in Neuroblastoma (NB). By inhibiting mTOR, AZD8055 disrupts the pathway, which is critical for cancer cell growth and survival. This disruption makes AZD8055 a promising therapeutic option.

2

Why is mTOR important in the context of neuroblastoma, and how does AZD8055 interact with it?

mTOR, or mammalian target of rapamycin, is a protein that plays a central role in cell growth and metabolism. In the context of neuroblastoma, the PI3K/Akt/mTOR signaling cascade, including mTOR, is often hyperactive. This hyperactivity drives cancer cell growth, making mTOR an attractive target for therapeutic intervention. Targeting mTOR with AZD8055 helps to halt the progression of neuroblastoma by interfering with fundamental growth processes.

3

What is the significance of the PI3K/Akt/mTOR signaling cascade in neuroblastoma, and how does AZD8055 affect it?

The PI3K/Akt/mTOR signaling cascade is a critical pathway in neuroblastoma. When this pathway is hyperactive, it contributes to oncogenesis (the formation of tumors), treatment resistance, and a poor prognosis for patients. AZD8055 targets this pathway by inhibiting mTOR, a key component. This inhibition leads to the disruption of cancer cell growth, cell cycle arrest, promotion of autophagy, and apoptosis.

4

What specific effects did AZD8055 have on neuroblastoma cells and tumors in the study?

AZD8055's potential is demonstrated by its ability to inhibit cell growth, induce cell cycle arrest (stopping cell division), and promote autophagy and apoptosis in neuroblastoma cells. These effects were observed both in laboratory (in vitro) settings and in animal models (in vivo). The reduction of tumor growth in animal models, without apparent toxicity, suggests AZD8055 could be a safe and effective treatment.

5

What are the implications of this research for the future of neuroblastoma treatment?

The study's findings provide a strong basis for further research on AZD8055 as a neuroblastoma treatment. It shows that AZD8055 can effectively reduce tumor growth and interfere with the fundamental processes driving cancer cell growth. The success of AZD8055 in preclinical models supports further investigation through clinical trials to determine its efficacy and safety in humans. This research signifies a step forward in pediatric oncology.

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