#1 Clinical Trials Search in Europe

Autologous Cd34+ Haematopoietic Stem And Progenitor Cells Transduced With A Lentiviral Vector Encoding The Interferon Alpha-2 Gene

This article explores the use of Temferon, an innovative gene therapy drug, in clinical trials for treating metastatic renal cell carcinoma (RCC) and glioblastoma multiforme (GBM). Temferon consists of autologous CD34+ hematopoietic stem and progenitor cells that have been genetically modified to produce interferon alpha-2. These trials aim to evaluate the safety, biological response, and efficacy of this groundbreaking treatment approach for patients with advanced cancers who have exhausted standard treatment options.

Table of Contents

What is Temferon?

Temferon is an innovative gene therapy medication designed to treat certain types of cancer. It is classified as an Advanced Therapy Medicinal Product (ATMP) for gene therapy[1]. The active substance in Temferon is called autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector encoding the interferon alpha-2 gene[1].

To break this down into simpler terms:

  • Autologous: This means the cells used in the treatment come from the patient’s own body.
  • CD34+ haematopoietic stem and progenitor cells: These are special cells from the bone marrow that can develop into different types of blood cells.
  • Transduced: This means the cells have been modified genetically.
  • Lentiviral vector: This is a tool used to introduce new genetic material into cells.
  • Interferon alpha-2 gene: This gene produces a protein that can help fight cancer.

How Does Temferon Work?

Temferon works by using the patient’s own stem cells as a vehicle to deliver a cancer-fighting gene to the body. Here’s a simplified explanation of the process:

  1. Stem cells are collected from the patient’s bone marrow.
  2. These cells are genetically modified in a laboratory to carry the interferon alpha-2 gene.
  3. The modified cells are then infused back into the patient’s bloodstream.
  4. These cells travel to the bone marrow and develop into different types of blood cells, including certain immune cells.
  5. The modified immune cells can then produce interferon alpha-2, a protein that helps fight cancer.

This approach is designed to provide a continuous, targeted delivery of the cancer-fighting protein directly to the tumor site[1].

Medical Conditions Treated

Temferon is being studied for the treatment of two types of cancer:

  1. Metastatic Renal Cell Carcinoma (RCC): This is a type of kidney cancer that has spread to other parts of the body. Specifically, the trials are focusing on clear cell RCC, which is the most common type of kidney cancer[1].
  2. Glioblastoma Multiforme (GBM): This is an aggressive type of brain cancer[2].

These conditions are typically treated when they have progressed despite standard treatments, indicating that Temferon is being studied as a potential option for patients who have limited treatment choices.

Clinical Trials

Temferon is currently being evaluated in clinical trials. Two main studies are underway:

  1. Study for Metastatic Renal Cell Carcinoma: This is an open-label phase 1/2 study to evaluate the safety, biological response, and efficacy of a single dose of Temferon in patients with metastatic renal cell carcinoma[1].
  2. Long-term Safety Study for Glioblastoma Multiforme: This is a follow-up study evaluating the long-term safety of Temferon in patients with glioblastoma multiforme who have previously received the treatment[2].

These trials are designed to assess how well Temferon works and to monitor any side effects or safety concerns over time.

Eligibility Criteria

For the metastatic renal cell carcinoma study, some key eligibility criteria include[1]:

  • Age between 18 and 70 years
  • Confirmed diagnosis of metastatic clear cell renal cell carcinoma
  • Disease progression following standard treatments
  • Adequate organ function
  • No active infections or certain other medical conditions

For the glioblastoma multiforme follow-up study, the main criterion is that patients must have previously received Temferon treatment in an earlier study[2].

Safety and Efficacy

The clinical trials are designed to assess both the safety and effectiveness of Temferon. Key aspects being evaluated include[1][2]:

  • Tolerability and safety of the treatment
  • Biological activity of Temferon in the tumor
  • Overall response rate and survival rates
  • Long-term safety, including any potential for developing other cancers
  • Presence of modified cells in the blood and bone marrow over time

It’s important to note that as Temferon is still in clinical trials, its full safety profile and effectiveness are not yet fully known.

Administration

Temferon is administered as a solution for infusion, which means it’s given directly into the bloodstream through a vein (intravenously)[1]. The treatment is given as a single dose, but patients are monitored for an extended period afterward to assess its effects and safety.

Long-Term Monitoring

Given the nature of gene therapy, long-term monitoring is crucial. The follow-up study for glioblastoma patients aims to assess the long-term safety of Temferon, including[2]:

  • Any potential for causing other cancers (mutagenic potential)
  • General long-term safety
  • Disease progression and survival rates
  • Presence of modified cells in the blood over time

This long-term monitoring is essential to fully understand the safety and effectiveness of Temferon over time.

Aspect Details
Drug Name Temferon (Autologous CD34+ hematopoietic stem and progenitor cells transduced with a lentiviral vector encoding the interferon alpha-2 gene)
Cancer Types Studied Metastatic renal cell carcinoma (RCC), Glioblastoma multiforme (GBM)
Trial Phases Integrated Phase 1 and 2
Primary Objectives Assess safety, tolerability, and biological activity of Temferon
Secondary Objectives Evaluate long-term safety, efficacy, and survival outcomes
Administration Single intravenous infusion
Key Eligibility Criteria Adults with advanced cancer, disease progression after standard treatments
Follow-up Period Up to 1 year for initial studies, with long-term follow-up for safety monitoring

FAQ

  • What is Temferon and how does it work? Temferon is a gene therapy drug made from a patient's own blood stem cells (CD34+ cells). These cells are genetically modified to produce interferon alpha-2, a protein that can help fight cancer. The modified cells are then infused back into the patient, where they can potentially target and attack cancer cells.
  • What types of cancer are being studied with Temferon? The clinical trials are focusing on two types of cancer: metastatic renal cell carcinoma (a type of kidney cancer that has spread to other parts of the body) and glioblastoma multiforme (an aggressive brain cancer).
  • Who is eligible for these Temferon clinical trials? Eligibility varies, but generally includes adult patients with advanced cancer who have already tried standard treatments. For the RCC trial, patients must have metastatic clear cell renal cell carcinoma that has progressed after standard treatments. The GBM trial is a follow-up study for patients who have already received Temferon in a previous trial.
  • What are the main goals of these clinical trials? The primary goals are to assess the safety and tolerability of Temferon, evaluate its biological activity in the body, and measure its effectiveness in treating the cancer. The studies also aim to monitor long-term safety and potential side effects of the treatment.
  • How is Temferon administered to patients? Temferon is given as a single dose through intravenous infusion. Before receiving Temferon, patients undergo a conditioning regimen to prepare their body for the treatment. After infusion, patients are closely monitored for side effects and treatment response.

Ongoing Clinical Trials on Autologous Cd34+ Haematopoietic Stem And Progenitor Cells Transduced With A Lentiviral Vector Encoding The Interferon Alpha-2 Gene

  • Long-term safety study of Temferon (modified stem cells with interferon-α2) in patients previously treated for glioblastoma multiforme

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on the Safety and Effects of Temferon for Patients with Metastatic Renal Cell Carcinoma Using a Drug Combination

    Not recruiting

    1 1 1 1
    Investigated drugs:
    Italy

Glossary

  • Autologous: Using cells or tissues obtained from the same individual. In this case, the patient's own blood stem cells are used to create the treatment.
  • CD34+ cells: A type of blood stem cell that can develop into different types of blood cells. These cells are collected from the patient and used to create Temferon.
  • Hematopoietic stem and progenitor cells: Immature cells in the bone marrow that can develop into various types of blood cells.
  • Lentiviral vector: A tool used in gene therapy to introduce new genetic material into cells. It's derived from a type of virus but modified to be safe for use in treatment.
  • Interferon alpha-2: A protein naturally produced by the body that helps fight viruses and may also have anti-cancer properties.
  • Metastatic renal cell carcinoma: A type of kidney cancer that has spread beyond the kidneys to other parts of the body.
  • Glioblastoma multiforme (GBM): An aggressive type of brain cancer that forms from glial cells (cells that support and protect nerve cells).
  • ECOG PS: Eastern Cooperative Oncology Group Performance Status, a scale used to assess how a patient's disease is progressing and how it affects daily living abilities.
  • RECIST: Response Evaluation Criteria in Solid Tumors, a set of rules used to measure if tumors are responding to treatment.
  • Progression-free survival: The length of time during and after treatment that a patient lives without the cancer getting worse.
  • Overall survival: The length of time from either the date of diagnosis or the start of treatment that patients are still alive.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-temferon-for-patients-with-metastatic-renal-cell-carcinoma-using-a-drug-combination/
  2. http://clinicaltrials.eu/trial/long-term-safety-study-of-temferon-in-patients-with-glioblastoma-multiforme/