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Paxalisib

Paxalisib is an investigational drug being studied in clinical trials for the treatment of various types of brain cancer, including glioblastoma and brain metastases. This article summarizes key information about ongoing clinical trials evaluating paxalisib’s safety and efficacy in patients with brain tumors.

Table of Contents

What is Paxalisib?

Paxalisib is a new drug being studied for the treatment of various brain cancers. It’s also known by several other names, including GDC-0084, GDC0084, and PI3K Inhibitor GDC-0084[1]. This medication is part of a class of drugs called PI3K/mTOR inhibitors, which means it works by blocking certain proteins that cancer cells need to grow[2].

How Paxalisib Works

Paxalisib is designed to target a specific pathway in cancer cells called the PI3K/AKT/mTOR pathway[2]. This pathway is like a communication system that tells cancer cells to grow and divide. By blocking this pathway, Paxalisib aims to slow down or stop the growth of cancer cells. What makes Paxalisib special is its ability to cross the blood-brain barrier, which is a protective layer that prevents many drugs from reaching the brain[3]. This means Paxalisib can potentially treat brain cancers more effectively than some other medications.

Conditions Treated with Paxalisib

Paxalisib is being studied for several types of brain cancers, including:

  • Glioblastoma (GBM): This is an aggressive type of brain cancer that Paxalisib is being tested on in both newly diagnosed and recurrent cases[2].
  • Diffuse Intrinsic Pontine Glioma (DIPG): A rare and aggressive brain tumor that typically affects children[5].
  • Diffuse Midline Glioma (DMG): Another type of brain tumor that is often difficult to treat[5].
  • Brain Metastases: These are cancer cells that have spread to the brain from other parts of the body[4].

Current Clinical Trials

Paxalisib is currently being studied in several clinical trials to determine its effectiveness and safety. Some of these trials include:

  • A study for newly diagnosed glioblastoma patients, testing Paxalisib in combination with radiation therapy and chemotherapy[2].
  • A trial for patients with brain metastases from various types of cancer[4].
  • A study combining Paxalisib with other treatments for diffuse midline gliomas in children and young adults[5].
  • A trial testing Paxalisib along with a special diet (ketogenic diet) and another medication called metformin for glioblastoma patients[6].

Dosage and Administration

Paxalisib is taken orally, usually in the form of tablets. The dosage can vary depending on the specific trial and condition being treated. Some common dosing regimens include:

  • 45 mg taken orally once daily for 28 days, potentially increasing to 60 mg if well-tolerated[2].
  • 21 mg/m² taken orally daily for 28-day cycles[5].

It’s important to note that these dosages are still being studied, and the optimal dose may change as more research is conducted.

Potential Side Effects

As with any medication, Paxalisib may cause side effects. While the full range of side effects is still being studied, some potential effects that researchers are monitoring include:

  • Changes in blood cell counts
  • Alterations in liver function
  • Effects on heart function
  • Changes in blood sugar levels

These potential side effects are being closely monitored in clinical trials to ensure patient safety[2][6].

Future Research and Combinations

Researchers are exploring various ways to use Paxalisib most effectively. This includes:

  • Combining Paxalisib with other cancer treatments like radiation therapy and chemotherapy[2].
  • Testing Paxalisib alongside dietary changes, such as a ketogenic diet (high-fat, low-carbohydrate diet)[6].
  • Using Paxalisib in combination with other targeted therapies based on a patient’s specific tumor characteristics[5].

These ongoing studies aim to find the most effective ways to use Paxalisib to help patients with various types of brain cancers.

Trial Name/ID Phase Cancer Type Key Details
NCT03522298 2 Newly diagnosed glioblastoma Evaluating safety, pharmacokinetics, and efficacy of paxalisib after surgery and chemoradiation
NCT05183204 2 Newly diagnosed and recurrent glioblastoma Combining paxalisib with ketogenic diet and metformin
NCT03994796 2 Brain metastases Genetically-guided treatment including paxalisib for PI3K mutations
NCT05009992 2 Diffuse midline gliomas Evaluating paxalisib in combination with ONC201
NCT03970447 2/3 Newly diagnosed and recurrent glioblastoma Adaptive platform trial including paxalisib as one treatment arm

FAQ

  • What type of drug is paxalisib? Paxalisib is an inhibitor of the PI3K/AKT/mTOR pathway. It is designed to cross the blood-brain barrier and target cancer cells in the brain.
  • What types of brain cancer is paxalisib being studied for? Paxalisib is being investigated for glioblastoma (including newly diagnosed and recurrent), diffuse midline gliomas, and brain metastases from other cancers.
  • How is paxalisib administered? Paxalisib is given orally, typically as a daily dose in tablet form. The exact dosing may vary between trials.
  • What are some of the potential side effects of paxalisib? As with any investigational drug, the full side effect profile is still being determined. Trials are monitoring for adverse events and toxicities to evaluate paxalisib's safety.
  • How can patients learn if they may be eligible for a paxalisib clinical trial? Patients should discuss clinical trial options with their oncologist. Eligibility criteria vary between trials but may depend on factors like cancer type, prior treatments, and overall health status.

Ongoing Clinical Trials on Paxalisib

  • Study on ONC201 and Paxalisib for Children and Young Adults with Diffuse Midline Gliomas, Including Diffuse Intrinsic Pontine Gliomas

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

Glossary

  • Glioblastoma: An aggressive type of cancer that occurs in the brain or spinal cord. It forms from cells called astrocytes that support nerve cells.
  • PI3K/AKT/mTOR pathway: A signaling pathway inside cells that regulates cell growth and survival. It is often overactive in cancer cells.
  • Blood-brain barrier: A border of cells that prevents many substances from passing from the bloodstream into the brain tissues.
  • Brain metastases: Cancer that has spread to the brain from another location in the body.
  • MGMT promoter methylation: A genetic marker that can indicate how well a glioblastoma may respond to certain treatments.
  • Progression-free survival: The length of time during and after treatment that a patient lives without the cancer progressing or worsening.
  • Overall survival: The length of time from the start of treatment or diagnosis that patients are still alive.
  • Dose-limiting toxicity: Side effects of a drug that are severe enough to prevent increasing the dose or continuing treatment.
  • Pharmacokinetics: The study of how a drug moves through the body, including how it is absorbed, distributed, metabolized and excreted.
  • Response rate: The percentage of patients whose cancer shrinks or disappears after treatment.

References

  1. https://clinicaltrials.gov/study/NCT05012670
  2. https://clinicaltrials.gov/study/NCT03522298
  3. https://clinicaltrials.gov/study/NCT04906096
  4. https://clinicaltrials.gov/study/NCT03994796
  5. https://clinicaltrials.eu/trial/study-on-onc201-and-paxalisib-for-children-and-young-adults-with-diffuse-midline-gliomas-including-diffuse-intrinsic-pontine-gliomas/
  6. https://clinicaltrials.gov/study/NCT05183204