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Autologous Muscle Precursor Cells

A groundbreaking clinical trial is underway to investigate the potential of autologous muscle precursor cells, specifically mesoangioblasts, in treating mitochondrial myopathy caused by the m.3243A>G mutation. This innovative approach aims to improve muscle strength and function in patients with this rare genetic condition. The study focuses on the safety and effectiveness of intra-arterial administration of these cells to the upper arm, offering hope for those affected by this debilitating disorder.

Table of Contents

What are Autologous Muscle Precursor Cells?

Autologous muscle precursor cells, also known as autologous mesoangioblasts or MABS06, are a type of cell therapy being studied for the treatment of certain muscle disorders[1]. These cells are derived from the patient’s own body, specifically from skeletal muscle tissue. The term “autologous” means that the cells come from the same individual who will receive the treatment, which can help reduce the risk of rejection by the immune system.

Target Condition: Mitochondrial Myopathy

The primary condition being targeted in this clinical trial is mitochondrial myopathy caused by a specific genetic mutation called m.3243A>G[1]. Mitochondrial myopathy is a group of muscle diseases caused by damage to the mitochondria, which are the energy-producing structures in our cells. This condition can lead to muscle weakness, fatigue, and other symptoms that can significantly impact a person’s quality of life.

How the Treatment Works

The treatment involves the following steps:

  1. Cells are collected from the patient’s own muscle tissue.
  2. These cells are processed and grown in a laboratory to create a cell suspension for injection.
  3. The prepared cells are then administered back to the patient through intra-arterial injection. This means the cells are injected directly into an artery[1].

In this specific clinical trial, the treatment focuses on the biceps brachii (BB) muscle in the upper arm. Patients receive three separate injections of their own muscle precursor cells into the left arm.

Clinical Trial Objectives

The main goals of the clinical trial are:

  • To assess the effect of the cell therapy on muscle strength and fatigue in the treated arm compared to the untreated arm[1].
  • To evaluate the safety of the treatment, including monitoring for any serious side effects or complications[1].
  • To measure changes in muscle mass, structure, and function before and after treatment[1].

Eligibility Criteria

To participate in this clinical trial, patients must meet certain criteria[1]:

  • Age: 18-64 years old
  • Diagnosed with the m.3243A>G mutation causing mitochondrial myopathy
  • Able to provide informed consent

There are also several factors that would exclude a person from participating, such as certain medical conditions, medications, or lifestyle factors. It’s important to discuss these with a healthcare provider or the research team to determine eligibility.

Safety Considerations

The clinical trial places a strong emphasis on patient safety[1]. Some key safety measures include:

  • Monitoring for any serious adverse events (SAEs)
  • Checking for potential vascular obstructions (blockages in blood vessels)
  • Observing changes in neurological vital signs
  • Performing angiography (a type of X-ray that looks at blood vessels)
  • Close monitoring for 8 hours after each procedure

Potential Benefits

While the effectiveness of this treatment is still being studied, the researchers hope to see improvements in[1]:

  • Muscle strength and reduced fatigue in the treated arm
  • Increased muscle mass
  • Improved mitochondrial function in the treated muscle
  • Reduced mutation load (the amount of abnormal mitochondrial DNA) in the treated muscle

It’s important to note that this is an early-phase clinical trial (Phase I/II), which means that while the treatment shows promise, its full effects and long-term outcomes are still being investigated.

Aspect Details
Trial Name 2024-515129-27-00
Condition Mitochondrial myopathy due to m.3243A>G mutation
Treatment Intra-arterial autologous mesoangioblasts (muscle precursor cells)
Primary Objective Assess effect and safety of three administrations to the biceps brachii muscle
Key Measurements Muscle strength, fatigue, mass, morphology, mutation load, mitochondrial respiratory capacity
Eligibility Adults 18-64 with heteroplasmic m.3243A>G mutation
Safety Monitoring Angiography, neurological vital signs, adverse events
Follow-up Period 4-6 weeks after third administration

FAQ

  • What is the main purpose of this clinical trial? The main purpose is to assess the effect and safety of three intra-arterial administrations of autologous mesoangioblasts (muscle precursor cells) to the biceps brachii muscle of patients with the m.3243A>G mutation, which causes mitochondrial myopathy. The trial aims to evaluate changes in muscle strength and fatigue before and after treatment.
  • Who is eligible to participate in this trial? Eligible participants are adults aged 18-64, both male and female, who have the heteroplasmic m.3243A>G mutation. They must provide written informed consent and meet other specific criteria. Some exclusion factors include the use of certain anticoagulants, excessive alcohol intake, and significant concurrent illnesses.
  • How will the treatment be administered? The treatment involves three intra-arterial administrations of autologous mesoangioblasts (the patient's own muscle precursor cells) to the upper left arm, specifically targeting the biceps brachii muscle.
  • What are the main outcomes being measured in this trial? The primary outcomes include safety parameters such as angiography results and monitoring of neurological vital signs. The trial will also assess changes in muscle strength and fatigue of both the treated and untreated biceps brachii muscles using a Biodex dynamometer. Secondary outcomes include evaluating muscle mass, morphology, mutation load, and mitochondrial respiratory capacity through muscle biopsies.
  • How long will participants be monitored after treatment? The main assessments will be conducted at baseline and 4-6 weeks after the third administration of the autologous muscle precursor cells. However, safety monitoring will begin immediately after each treatment, with an 8-hour post-procedure monitoring of neurological vital signs.

Ongoing Clinical Trials on Autologous Muscle Precursor Cells

  • Study on the Effect and Safety of Autologous Mesoangioblasts for Patients with Mitochondrial Myopathy Due to m.3243A>G Mutation

    Not yet recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

Glossary

  • Autologous Muscle Precursor Cells: These are cells taken from a patient's own body, specifically from muscle tissue, that have the potential to develop into new muscle cells. In this trial, they are being used as a potential treatment for mitochondrial myopathy.
  • Mesoangioblasts: A type of muscle precursor cell that can develop into various types of muscle tissue. In this trial, they are being used as the primary treatment for mitochondrial myopathy.
  • Mitochondrial Myopathy: A group of muscle diseases caused by damage to the mitochondria, which are structures within cells that produce energy. These conditions typically result in muscle weakness and fatigue.
  • m.3243A>G Mutation: A specific genetic mutation in mitochondrial DNA that can cause mitochondrial myopathy. It affects the body's ability to produce energy efficiently in cells.
  • Intra-arterial Administration: A method of delivering treatment directly into an artery. In this trial, the muscle precursor cells are administered into the artery supplying the biceps brachii muscle.
  • Biceps Brachii Muscle: The large muscle on the front part of the upper arm, responsible for flexing the elbow and rotating the forearm.
  • Heteroplasmic: In genetics, this refers to the presence of both normal and mutated DNA within the same cell or individual. In this case, it relates to the m.3243A>G mutation.
  • Angiography: A medical imaging technique used to visualize the inside of blood vessels and organs, often used to check for blockages or abnormalities.
  • Biodex Dynamometer: A device used to measure muscle strength and fatigue by assessing the force produced during muscle contractions.
  • Mitochondrial Respiratory Capacity: The ability of mitochondria to produce energy through cellular respiration. This is often impaired in mitochondrial myopathy.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effect-and-safety-of-autologous-mesoangioblasts-for-patients-with-mitochondrial-myopathy-due-to-m-3243ag-mutation/