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Epacadostat

Epacadostat is an investigational immunotherapy drug being studied in various clinical trials for the treatment of different types of cancer. This article will provide an overview of recent clinical trials using epacadostat, often in combination with other immunotherapy drugs, to treat cancers such as lung cancer, gastric cancer, pancreatic cancer, and head and neck cancer. We’ll explore the potential benefits, side effects, and current state of research for this promising new treatment approach.

Table of Contents

What is Epacadostat?

Epacadostat is an innovative drug being studied for the treatment of various types of cancer. It is also known by its alternative names INCB024360 and IDO1 inhibitor[1]. Epacadostat belongs to a class of drugs called immunotherapy, which works by helping your body’s immune system fight cancer cells[2].

How Epacadostat Works

Epacadostat works by blocking an enzyme called IDO1 (indoleamine 2,3-dioxygenase 1). This enzyme is often overactive in cancer cells and helps them hide from the immune system. By blocking IDO1, epacadostat may allow the immune system to recognize and attack cancer cells more effectively[2][1].

Cancers Treated with Epacadostat

Epacadostat is being studied in clinical trials for various types of cancer, including:

  • Thymic carcinoma: A rare cancer that starts in the thymus gland[3]
  • Non-small cell lung cancer (NSCLC): A common type of lung cancer[4]
  • Urothelial carcinoma: A type of bladder cancer[4]
  • Gastrointestinal stromal tumors (GIST): A type of tumor that occurs in the digestive tract[5]
  • Pancreatic cancer[6]
  • Head and neck squamous cell carcinoma (HNSCC)[7]
  • Nasopharyngeal cancer: A type of cancer that starts in the upper part of the throat behind the nose[8]
  • Myelodysplastic syndromes (MDS): A group of blood disorders where the bone marrow doesn’t produce enough healthy blood cells[1]

Epacadostat in Combination Therapy

Many clinical trials are studying epacadostat in combination with other immunotherapy drugs, particularly those that target PD-1 or PD-L1 proteins. These combinations include:

  • Epacadostat + Pembrolizumab (Keytruda): This combination is being studied in thymic carcinoma, GIST, and head and neck cancer[3][5][7]
  • Epacadostat + Atezolizumab (MPDL3280A): Being studied in NSCLC and urothelial carcinoma[4]
  • Epacadostat + Durvalumab: Being studied in nasopharyngeal cancer[8]
  • Epacadostat + Sirolimus: Being studied in advanced solid tumors[9]

The idea behind these combinations is that they may work better together to stimulate the immune system against cancer than either drug alone.

How Epacadostat is Administered

Epacadostat is typically taken orally (by mouth) in the form of tablets. The dosage and frequency can vary depending on the specific clinical trial and the type of cancer being treated. Some common dosing schedules include:

  • 100 mg twice daily[3]
  • 300 mg twice daily[7]
  • 600 mg twice daily[1]

It’s important to note that the optimal dosage is still being determined through clinical trials.

Potential Side Effects

As with any medication, epacadostat can cause side effects. The full range of side effects is still being studied in clinical trials. Some potential side effects may include fatigue, nausea, and changes in blood cell counts. When used in combination with other immunotherapy drugs, there may be additional side effects related to the immune system becoming overactive[7][8].

Ongoing Research and Future Prospects

Epacadostat is still considered an experimental drug and is not yet approved for general use outside of clinical trials. Researchers are conducting various studies to determine its effectiveness, optimal dosing, and potential side effects. Some areas of ongoing research include:

  • Using epacadostat before surgery (neoadjuvant therapy) in head and neck cancer[7]
  • Studying epacadostat’s effects on the immune system and tumor microenvironment[10]
  • Investigating biomarkers that might predict which patients will respond best to epacadostat treatment[7]

The results of these ongoing clinical trials will help determine the future role of epacadostat in cancer treatment. While early results are promising in some cancers, more research is needed to fully understand its benefits and risks.

Aspect Details
Drug Name Epacadostat (also known as INCB024360)
Mechanism of Action IDO1 enzyme inhibitor
Administration Oral, typically twice daily
Common Dosages 100mg to 300mg twice daily
Combination Therapies Often combined with pembrolizumab, durvalumab, or other immunotherapy drugs
Cancer Types Studied NSCLC, gastric cancer, pancreatic cancer, head and neck cancer, nasopharyngeal cancer
Primary Outcomes Measured Overall response rate, progression-free survival, overall survival
Treatment Duration Varies by trial; often up to 24 months or until disease progression
Key Potential Benefits Enhanced immune response against cancer, potential for improved outcomes when combined with other immunotherapies
Current Status Investigational; being studied in multiple phase I and II clinical trials

FAQ

  • What is epacadostat and how does it work? Epacadostat is an oral medication that blocks an enzyme called IDO1. By blocking this enzyme, epacadostat may allow for a stronger immune response against cancer cells. It is often used in combination with other immunotherapy drugs to potentially enhance their effectiveness.
  • What types of cancer are being studied with epacadostat? Clinical trials are investigating epacadostat for various cancers, including non-small cell lung cancer (NSCLC), gastric cancer, pancreatic cancer, head and neck squamous cell carcinoma, and nasopharyngeal cancer. It is being studied both alone and in combination with other immunotherapy drugs.
  • How is epacadostat administered in clinical trials? In most trials, epacadostat is taken orally, usually twice daily. The dosage can vary, with common doses ranging from 100mg to 300mg twice a day. It is often given in combination with intravenous immunotherapy drugs like pembrolizumab or durvalumab.
  • What are some potential side effects of epacadostat? As with any medication, epacadostat can cause side effects. The specific side effects and their severity can vary. Common side effects observed in clinical trials include fatigue, nausea, and decreased appetite. However, the full range of potential side effects is still being studied in ongoing clinical trials.
  • How long do patients typically receive epacadostat in clinical trials? The duration of treatment varies depending on the specific trial design. In some studies, patients receive epacadostat for up to 24 months, while in others, treatment continues until disease progression or unacceptable toxicity occurs. Some trials also include options for extended treatment if patients are benefiting from the therapy.

Ongoing Clinical Trials on Epacadostat

  • Study of Pembrolizumab, Epacadostat, and Drug Combination for Patients with Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

    Not recruiting

    3 1 1 1
    Investigated drugs:
    Portugal Spain

Glossary

  • IDO1: Indoleamine 2,3-dioxygenase 1, an enzyme that epacadostat blocks. Inhibiting IDO1 may help boost the immune system's response against cancer cells.
  • Immunotherapy: A type of cancer treatment that helps the body's immune system fight cancer. Epacadostat is considered an immunotherapy drug.
  • PD-1/PD-L1: Proteins found on immune cells (PD-1) and some cancer cells (PD-L1). Blocking these proteins can help the immune system recognize and attack cancer cells.
  • Pembrolizumab: An immunotherapy drug that targets PD-1. It is often used in combination with epacadostat in clinical trials.
  • RECIST: Response Evaluation Criteria in Solid Tumors, a standard way to measure how well cancer patients respond to treatment based on changes in tumor size.
  • Progression-free survival (PFS): The length of time during and after treatment that a patient lives with cancer without it worsening.
  • Overall survival (OS): The length of time from the start of treatment that patients diagnosed with the disease are still alive.
  • Adverse events: Any unfavorable and unintended sign, symptom, or disease associated with the use of a medical treatment.
  • Neoadjuvant therapy: Treatment given before the main treatment, usually before surgery, to shrink a tumor or prepare the body for the primary treatment.
  • Metastatic cancer: Cancer that has spread from its original site to other parts of the body.

References

  1. https://clinicaltrials.gov/study/NCT01822691
  2. https://clinicaltrials.gov/study/NCT03196232
  3. https://clinicaltrials.gov/study/NCT02364076
  4. https://clinicaltrials.gov/study/NCT02298153
  5. https://clinicaltrials.gov/study/NCT03291054
  6. https://clinicaltrials.gov/study/NCT03432676
  7. https://clinicaltrials.gov/study/NCT03325465
  8. https://clinicaltrials.gov/study/NCT04231864
  9. https://clinicaltrials.gov/study/NCT03217669
  10. https://clinicaltrials.gov/study/NCT03471286