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ELTANEXOR

Eltanexor is an investigational drug currently being studied in clinical trials for patients with blood cancers that have not responded to previous treatments or have returned after initial improvement. A notable ongoing phase I trial is examining the combination of Eltanexor with another medication called Venetoclax for treating relapsed or refractory myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). This study aims to determine the safety, appropriate dosage, and effectiveness of this drug combination. Eltanexor works by trapping tumor-suppressing proteins within cancer cells, potentially causing these cells to die or stop growing, while Venetoclax blocks a protein called BCL-2 that cancer cells need for survival.

Table of Contents

What is Eltanexor?

Eltanexor, also known by its research name KPT-8602, is an investigational medication being studied for the treatment of certain blood cancers[1]. It belongs to a class of drugs that work by affecting specific proteins in cancer cells, potentially causing these harmful cells to die or stop growing. Eltanexor is currently being evaluated in clinical trials and is not yet approved for general use outside of research settings.

What Conditions Can Eltanexor Treat?

Based on ongoing research, Eltanexor is being studied for the treatment of several serious blood disorders[1]:

  • Myelodysplastic Syndrome (MDS) – A group of disorders where the bone marrow doesn’t produce enough healthy blood cells. MDS can be relapsed (returned after a period of improvement) or refractory (not responding to standard treatments).
  • Acute Myeloid Leukemia (AML) – A fast-growing cancer of the blood and bone marrow that affects the production of normal blood cells. Like MDS, AML can also be recurrent or refractory to treatment.

Specifically, Eltanexor is being investigated in patients whose disease has either come back after initial treatment (relapsed) or has not responded well to previous treatments (refractory)[1]. These patient populations often have limited treatment options, making new therapeutic approaches particularly important.

How Does Eltanexor Work?

Eltanexor works through a unique mechanism that involves trapping certain proteins, known as tumor suppressing proteins, within cancer cells[1]. These proteins normally help to control cell growth and prevent cancer. By keeping these beneficial proteins inside the cancer cells, Eltanexor may cause the cancer cells to die or stop dividing.

This approach differs from many traditional cancer treatments and represents a targeted strategy for addressing these difficult-to-treat blood cancers.

Current Clinical Research

Eltanexor is currently being studied in a Phase Ib clinical trial in combination with another medication called Venetoclax[1]. This study aims to:

  • Establish the safe and biologically effective dose of Eltanexor when used together with Venetoclax
  • Estimate how many patients achieve complete remission (disappearance of all signs of cancer)
  • Assess the overall response rate (percentage of patients whose cancer shrinks or disappears)
  • Measure how long patients survive overall
  • Determine how long patients live without their disease getting worse (progression-free survival)
  • Assess how long responses to treatment last

The research also includes exploratory objectives to better understand which patients might benefit most from this treatment combination[1].

How is Eltanexor Administered?

In the current clinical trial, Eltanexor is given as an oral medication (taken by mouth) once per day for 5 days per week[1]. The treatment schedule may vary, with the medication being given for 14, 21, or 28 days each cycle, depending on the study protocol. Each complete cycle lasts 28 days (about 4 weeks).

When combined with Venetoclax, the treatment regimen involves:

  • Eltanexor taken orally once daily for 5 days per week
  • Venetoclax taken orally once daily on days 1-14 of each cycle

Patients continue receiving treatment cycles every 28 days as long as their disease doesn’t worsen and they don’t experience unacceptable side effects[1].

Potential Side Effects

As with any medication, especially cancer treatments, Eltanexor may cause side effects. The current clinical trial is specifically designed to monitor and assess adverse events (side effects) that participants may experience[1]. These events are categorized and graded according to standardized criteria called the Common Terminology Criteria for Adverse Events (CTCAE).

While the specific side effect profile of Eltanexor is still being established through ongoing research, patients in the trial undergo regular monitoring to detect and manage any adverse effects that may occur.

Combination Therapy with Venetoclax

Eltanexor is being studied in combination with another medication called Venetoclax[1]. Venetoclax is a B-cell lymphoma-2 (BCL-2) inhibitor, which means it blocks a protein called Bcl-2 that helps cancer cells survive.

The rationale for combining these two medications is that they work through different mechanisms:

  • Eltanexor traps tumor suppressing proteins within cancer cells
  • Venetoclax blocks the Bcl-2 protein that cancer cells need for survival

By targeting cancer cells in two different ways, researchers hope this combination may be more effective than either medication alone for patients with relapsed or refractory MDS or AML[1].

Patient Monitoring During Treatment

Patients receiving Eltanexor in clinical trials undergo comprehensive monitoring to track both their response to treatment and any potential side effects[1]. This monitoring includes:

  • Bone marrow aspiration and biopsy – A procedure to collect and examine bone marrow samples to assess how the disease is responding to treatment
  • Blood sample collection – Regular blood tests to monitor blood cell counts, liver and kidney function, and other important health parameters

After completing treatment, patients in the current study are followed for up to 24 months (2 years) with check-ups approximately every 3 months[1]. This long-term follow-up helps researchers understand the lasting effects of treatment and how durable the responses are.

Study Aspect Details
Drug Name Eltanexor (KPT-8602)
Combined With Venetoclax
Study Type Phase Ib clinical trial (dose-escalation study)
Conditions Treated Relapsed or refractory myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML)
Mechanism of Action Eltanexor: Traps tumor suppressing proteins within cancer cells
Venetoclax: Blocks BCL-2, a protein needed for cancer cell survival
Administration Eltanexor: Oral, once daily for 5 days per week for 14-28 days per cycle
Venetoclax: Oral, once daily on days 1-14 of each cycle
Cycle Length 28 days
Primary Objectives Establish safe and biologically effective dose
Monitor adverse events
Secondary Objectives Measure complete remission rate
Assess overall response rate
Evaluate overall survival
Determine progression-free survival and duration of response
Procedures Bone marrow aspiration and biopsy
Blood sample collection
Follow-up Period Every 3 months for up to 24 months after treatment completion

FAQ

  • What is Eltanexor and how does it work? Eltanexor (also known as KPT-8602) is an investigational drug that works by trapping 'tumor suppressing proteins' within cancer cells. These proteins normally help control cell growth, but cancer cells often find ways to remove them from the cell nucleus. By keeping these proteins inside the cells, Eltanexor can cause cancer cells to die or stop growing. In the current clinical trial, it's taken orally once per day for 5 days per week.
  • Which conditions is Eltanexor being tested for in this clinical trial? In this clinical trial, Eltanexor is being tested in combination with Venetoclax for patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has either relapsed (come back after a period of improvement) or is refractory (has not responded to previous treatment).
  • How is Eltanexor administered in this clinical trial? In this trial, patients receive Eltanexor orally (by mouth) once per day for 5 days per week. Depending on the study phase, this may continue for 14, 21, or 28 days of each cycle. Each cycle is 28 days long, and treatment continues until either the disease progresses or side effects become unacceptable.
  • What is Venetoclax and why is it being combined with Eltanexor? Venetoclax is a medication known as a B-cell lymphoma-2 (BCL-2) inhibitor. It works by blocking BCL-2, a protein that cancer cells need to survive. It's being combined with Eltanexor because researchers believe the two drugs might work together effectively to combat cancer cells. In the trial, Venetoclax is taken orally once daily on days 1-14 of each 28-day cycle.
  • What are the main goals of this Eltanexor clinical trial? The primary goal of this phase I trial is to establish the safe and biologically effective dose of Eltanexor when combined with Venetoclax. Secondary goals include measuring how many patients achieve complete remission, assessing the overall response rate, and determining survival outcomes (overall survival, progression-free survival, and duration of response). The trial also has exploratory objectives like comparing responses between MDS and AML patients and studying how genetic mutations might affect treatment response.

Ongoing Clinical Trials on ELTANEXOR

  • Study on the Safety and Effectiveness of Eltanexor for Patients with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

    Recruiting

    1 1
    Investigated drugs:
    Italy

Glossary

  • Acute Myeloid Leukemia (AML): A type of cancer that affects the blood and bone marrow. It's characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with normal blood cell production.
  • Myelodysplastic Syndrome (MDS): A group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the blood-forming cells in the bone marrow become abnormal, leading to low numbers of one or more types of blood cells.
  • Relapsed: When a disease returns or worsens after a period of improvement or remission.
  • Refractory: When a disease does not respond to treatment; resistant to treatment.
  • Phase I Trial: The first stage of clinical testing in humans, primarily focused on evaluating a drug's safety, determining appropriate dosage, and identifying side effects.
  • Biologically Effective Dose (BED): The amount of a drug that produces the desired biological effect with acceptable side effects.
  • Complete Remission (CR): The disappearance of all signs of cancer in response to treatment.
  • Overall Response Rate (ORR): The percentage of patients whose cancer shrinks or disappears after treatment.
  • Progression Free Survival (PFS): The length of time during and after treatment that a patient lives with the disease but it does not get worse.
  • Overall Survival: The length of time from either the date of diagnosis or the start of treatment that patients are still alive.
  • Duration of Response (DOR): The time from first documented evidence of response (such as tumor shrinkage) until disease progression or death.
  • Bone Marrow Aspiration and Biopsy: A procedure to collect and examine bone marrow (the spongy tissue inside bones where blood cells are made) to diagnose and monitor blood and marrow diseases.
  • Measurable Residual Disease: Small numbers of cancer cells that remain in the body during or after treatment, often below the level detectable through standard tests.
  • BCL-2 Inhibitor: A type of drug that blocks B-cell lymphoma-2 (BCL-2), a protein that helps cancer cells survive by preventing cell death.
  • Tumor Suppressing Proteins: Proteins that regulate cell growth and division, preventing cells from growing and dividing too rapidly or in an uncontrolled way.
  • Common Terminology Criteria for Adverse Events (CTCAE): A standardized system for classifying the severity of side effects experienced by patients during treatment in clinical trials.
  • Biospecimen Collection: The process of gathering biological samples such as blood, tissue, or other bodily fluids for research purposes.
  • Kaplan and Meier Method: A statistical technique used to estimate and graph survival probabilities as a function of time.

References

  1. https://clinicaltrials.gov/study/NCT06399640