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Pralsetinib

Pralsetinib is an emerging targeted therapy being investigated in clinical trials for the treatment of various cancers with RET gene alterations. This article summarizes key information about ongoing clinical trials evaluating pralsetinib’s safety and efficacy in conditions like non-small cell lung cancer, thyroid cancer, and other RET-altered solid tumors. The trials aim to assess pralsetinib as a potential treatment option for patients with limited alternatives.

Table of Contents

What is Pralsetinib?

Pralsetinib is a targeted cancer therapy drug. It is also known by the brand names Gavreto in the United States[1] and BLU-667[2]. Pralsetinib belongs to a class of medications called tyrosine kinase inhibitors (TKIs). These drugs work by targeting specific proteins in cancer cells that help them grow and spread.

How Pralsetinib Works

Pralsetinib is designed to target a specific genetic alteration called RET fusion or mutation. RET stands for “rearranged during transfection,” which refers to a gene that can become altered in some cancers. When the RET gene fuses with another gene or develops mutations, it can lead to uncontrolled cell growth and cancer.

Pralsetinib works by blocking the activity of the altered RET protein, which can help slow or stop the growth of cancer cells with these specific genetic changes[2]. This targeted approach means that pralsetinib may be effective against cancers with RET alterations, regardless of where in the body the cancer originated.

Conditions Treated with Pralsetinib

Pralsetinib is being studied and used to treat several types of cancer that have RET gene alterations, including:

  • Non-Small Cell Lung Cancer (NSCLC): This is the most common type of lung cancer. Pralsetinib is being used to treat advanced or metastatic NSCLC with RET fusion[3].
  • Medullary Thyroid Cancer (MTC): This is a rare type of thyroid cancer that starts in the C cells of the thyroid gland. Pralsetinib is being studied for use in MTC with RET mutations[4].
  • Other RET-altered Solid Tumors: Pralsetinib is also being investigated for use in other types of solid tumors that have RET fusions or mutations[2].

How Pralsetinib is Administered

Pralsetinib is taken orally (by mouth) in the form of capsules. The typical dose is 400 mg once daily[5]. However, the exact dosage may vary depending on the specific condition being treated and the patient’s individual factors. It’s important to take pralsetinib exactly as prescribed by your healthcare provider.

Ongoing Clinical Trials

Several clinical trials are currently underway to further study the effectiveness and safety of pralsetinib in various settings:

  • First-line Treatment: A study is comparing pralsetinib to standard chemotherapy as the first treatment for patients with advanced RET fusion-positive NSCLC[3].
  • Medullary Thyroid Cancer: A trial is evaluating pralsetinib compared to standard treatments (cabozantinib or vandetanib) in patients with RET-mutated MTC[4].
  • Neoadjuvant Treatment: Pralsetinib is being studied as a treatment before surgery (neoadjuvant therapy) in patients with early-stage NSCLC that has RET fusions[6].
  • Basket Trial: A large study called TAPISTRY is investigating pralsetinib and other targeted therapies in patients with various types of solid tumors that have specific genetic alterations, including RET fusions[1].

Potential Side Effects

As with all medications, pralsetinib can cause side effects. Common side effects may include:

  • Fatigue
  • Constipation
  • Muscle and joint pain
  • High blood pressure
  • Decreased white blood cell counts

It’s important to discuss potential side effects with your healthcare provider. They can provide more detailed information and help manage any side effects that may occur[4].

Aspect Details
Drug Name Pralsetinib (BLU-667)
Target Conditions RET fusion-positive NSCLC, Medullary Thyroid Cancer, RET-altered solid tumors
Administration Oral, 400 mg once daily in 28-day cycles
Trial Phases Phase 1/2, Phase 2, Phase 3
Key Outcomes Measured Objective response rate, progression-free survival, overall survival, safety profile
Patient Populations Adults and select adolescents with RET-altered cancers, including treatment-naive and previously treated patients
Notable Trial Designs Basket trials for multiple tumor types, neoadjuvant/adjuvant studies, comparisons to standard of care

FAQ

  • What types of cancers is pralsetinib being studied for? Pralsetinib is being studied for various cancers with RET gene alterations, including non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC), and other RET-altered solid tumors. Clinical trials are evaluating its efficacy in both early-stage resectable cancers and advanced metastatic disease.
  • How is pralsetinib administered? Pralsetinib is typically administered orally (by mouth) once daily. The standard dose in most trials is 400 mg taken daily in continuous 28-day cycles.
  • What are the main goals of the pralsetinib clinical trials? The main goals include evaluating pralsetinib's safety profile, determining its effectiveness in shrinking tumors or slowing disease progression, assessing quality of life impacts, and measuring survival outcomes like progression-free survival and overall survival in patients with RET-altered cancers.
  • Are there any notable side effects of pralsetinib? While specific side effects are not detailed in the trial summaries provided, the studies are monitoring for adverse events. As with most cancer therapies, pralsetinib may cause side effects that will be carefully tracked and managed during the trials.
  • How can patients potentially access pralsetinib treatment? Patients may be able to access pralsetinib by enrolling in an appropriate clinical trial if they meet the eligibility criteria. Some trials also have pre-approval access programs to provide the drug to patients who cannot participate in trials. Consulting with an oncologist is the best way to determine potential access options.

Ongoing Clinical Trials on Pralsetinib

  • A Study Testing How Pralsetinib Affects Other Medications in Patients with Advanced or Metastatic Solid Tumors

    Not yet recruiting

    3 1 1
    Investigated drugs:
    Spain

  • Study Comparing Pralsetinib and Standard Drug Combination for Patients with Advanced RET Fusion-Positive Non-Small Cell Lung Cancer

    Not recruiting

    3 1 1 1
    Investigated drugs:
    Belgium France Germany Ireland Italy The Netherlands +4

  • Study on the Effectiveness of Inavolisib, Atezolizumab, and Entrectinib in Patients with Advanced or Metastatic Solid Tumors

    Not recruiting

    2 1 1 1
    Investigated drugs:
    Belgium Denmark France Germany Italy Poland +2

Glossary

  • RET: A gene that, when altered, can drive the growth of certain cancers. RET stands for 'rearranged during transfection'.
  • Non-small cell lung cancer (NSCLC): The most common type of lung cancer, accounting for about 80-85% of all cases.
  • Medullary thyroid cancer (MTC): A rare type of thyroid cancer that develops in the C cells of the thyroid gland.
  • Neoadjuvant treatment: Treatment given before the main treatment, usually to shrink a tumor before surgery.
  • Adjuvant treatment: Additional treatment given after the primary treatment to lower the risk of cancer coming back.
  • 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 or diagnosis that patients are still alive.
  • Objective response rate (ORR): The proportion of patients whose cancer shrinks or disappears after treatment.
  • RECIST: Response Evaluation Criteria in Solid Tumors – a standard way to measure how well a cancer patient responds to treatment.
  • Adverse event (AE): Any unfavorable and unintended sign, symptom, or disease associated with the use of a medical treatment.
  • Biomarker: A biological molecule found in blood, other body fluids, or tissues that is a sign of a normal or abnormal process, or of a condition or disease.
  • Next-generation sequencing (NGS): Advanced technology used to sequence large amounts of DNA quickly, often used to identify genetic alterations in tumors.

References

  1. https://clinicaltrials.gov/study/NCT04589845
  2. https://clinicaltrials.gov/study/NCT03037385
  3. https://clinicaltrials.gov/study/NCT04222972
  4. https://clinicaltrials.gov/study/NCT04760288
  5. https://clinicaltrials.gov/study/NCT04204928
  6. https://clinicaltrials.gov/study/NCT04302025