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Allogeneic Fetal Mesenchymal Stem Cells

This article discusses an innovative clinical trial investigating the use of allogeneic fetal mesenchymal stem cells, known as BOOST cells, for treating severe Osteogenesis Imperfecta (OI) in children. The trial aims to evaluate the safety and effectiveness of this advanced therapy for a rare genetic condition that causes brittle bones. The study focuses on children with OI type III and severe type IV, exploring both postnatal and prenatal treatment options.

Table of Contents

What are BOOST Cells?

BOOST cells are a new and innovative treatment being studied for children with severe Osteogenesis Imperfecta (OI), also known as brittle bone disease. These cells are officially called “allogeneic fetal mesenchymal stem cells” and are being developed as an Advanced Therapy Investigational Medicinal Product (ATIMP).[1]

The full name of this treatment is “BOOST cells (cryopreserved expanded human first-trimester fetal liver-derived mesenchymal stem cells)”. Let’s break this down:

  • Allogeneic: This means the cells come from a donor, not the patient themselves.
  • Fetal: The cells are derived from fetal tissue, specifically the liver.
  • Mesenchymal stem cells: These are special cells that can develop into various types of tissues, including bone.
  • Cryopreserved: The cells are frozen for storage and later use.
  • Expanded: The number of cells is increased in the laboratory before use.

Understanding Osteogenesis Imperfecta

Osteogenesis Imperfecta (OI) is a rare genetic disorder that affects the body’s ability to produce collagen, a crucial protein for building strong bones. This results in bones that break easily, often from little or no apparent cause.[1]

The clinical trial focuses on two severe types of OI:

  • Type III OI: This is the most severe form in children who survive the newborn period. It’s characterized by very fragile bones that can break even before birth.
  • Severe Type IV OI: This type is moderately severe, with frequent fractures and bone deformities.

How BOOST Cells Work

While the exact mechanism is still being studied, BOOST cells are believed to work by:

  1. Traveling to the bones after being injected into the bloodstream.
  2. Potentially developing into bone-forming cells or supporting existing bone cells.
  3. Possibly reducing inflammation and promoting healing.
  4. Potentially improving the overall structure and strength of the bones.

Current Clinical Trial

A clinical trial is currently underway to test the safety and effectiveness of BOOST cells for treating severe OI in children. This trial is classified as a Phase I/II trial, which means it’s in the early stages of testing in humans.[1]

Key points about the trial:

  • It’s an open-label trial, meaning both doctors and patients know which treatment is being given.
  • Multiple doses of BOOST cells will be administered.
  • The trial is taking place at multiple medical centers.
  • Treatment can be given either after birth (postnatal) or both before and after birth (prenatal and postnatal).
  • Results will be compared to historical data and untreated patients.

Who Can Participate in the Trial?

The trial has specific criteria for who can participate. Some key inclusion criteria are:[1]

  • Children under 18 months of age (adjusted for premature birth)
  • Diagnosed with OI type III or severe type IV
  • Confirmed genetic mutation in either the COL1A1 or COL1A2 gene
  • Parent or guardian over 18 years old who can provide informed consent

There are also several exclusion criteria to ensure the safety of participants. These include other medical conditions that might interfere with treatment, bleeding disorders, and certain allergies.

The Treatment Process

The treatment involves giving four doses of BOOST cells through an intravenous infusion (directly into the bloodstream). Depending on the group, treatment may be given:

  • Only after birth (postnatal)
  • Both before and after birth (prenatal and postnatal)

The exact timing and dosage of the treatments will be determined by the clinical trial protocol.

Expected Outcomes and Measurements

The trial will measure several outcomes to determine if BOOST cells are safe and effective. These include:[1]

  • Safety: Monitoring for any side effects or adverse reactions
  • Fracture frequency: How often bones break
  • Time to first fracture after treatment
  • Number of fractures at birth (for those treated before birth)
  • Bone mineral density: A measure of bone strength
  • Growth in height and weight
  • Overall clinical status of OI
  • Biochemical markers of bone turnover: Substances in the blood that indicate bone formation and breakdown

Safety Considerations

As with any new treatment, safety is a top priority. The trial will closely monitor for any side effects or adverse reactions. Some potential risks that will be watched for include:[1]

  • Allergic reactions to the treatment or components used in its preparation
  • Increased risk of blood clots
  • Potential interactions with the immune system

It’s important to note that BOOST cells are still in the experimental stage. While they show promise, more research is needed to fully understand their effectiveness and safety for treating severe Osteogenesis Imperfecta in children.

Aspect Details
Trial Name Treatment of severe congenital Brittle bone disease after or before and after birth
Condition Studied Osteogenesis Imperfecta (OI) type III and severe type IV
Treatment BOOST cells (allogeneic fetal mesenchymal stem cells)
Administration Intravenous infusion, four doses
Primary Objective Assess safety and tolerability in children, fetuses, and pregnant women
Key Secondary Objectives Fracture frequency, bone mineral density, growth, clinical status of OI
Eligibility Age Less than 18 months (corrected age)
Key Inclusion Criteria Clinical diagnosis of OI type III or IV, specific genetic mutation
Key Exclusion Criteria Other interfering disorders, contraindications for invasive procedures, clotting risk factors
Trial Category Category 2 (safety and efficacy trial for new product development)

FAQ

  • What is Osteogenesis Imperfecta (OI)? Osteogenesis Imperfecta, also known as brittle bone disease, is a rare genetic condition that affects bone strength. In this trial, they focus on type III and severe type IV OI, which are more serious forms of the condition.
  • What are BOOST cells? BOOST cells are allogeneic fetal mesenchymal stem cells. These are special cells taken from fetal liver tissue that can potentially help in treating OI. They are given to patients through an intravenous infusion.
  • Who can participate in this clinical trial? The trial is for children under 18 months of age (corrected for gestational age) who have been diagnosed with OI type III or severe type IV. They must also have a specific genetic mutation in either the COL1A1 or COL1A2 gene.
  • What are the main goals of this clinical trial? The primary goal is to assess the safety and tolerability of BOOST cells in children, fetuses, and pregnant women. Secondary goals include measuring the frequency of bone fractures, bone density, growth, and overall clinical status of OI.
  • How is the treatment administered? The treatment involves four doses of BOOST cells given through intravenous infusion. Some participants may receive treatment before birth (prenatally), while others will receive it after birth (postnatally).

Ongoing Clinical Trials on Allogeneic Fetal Mesenchymal Stem Cells

  • Study on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands Sweden

Glossary

  • Osteogenesis Imperfecta (OI): A genetic disorder characterized by fragile bones that break easily, also known as brittle bone disease. It is caused by defects in genes that produce collagen, an important protein in bone structure.
  • Allogeneic: Refers to cells or tissues that are taken from a genetically different donor of the same species. In this case, the stem cells come from a donor, not the patient themselves.
  • Mesenchymal Stem Cells: A type of stem cell that can develop into various cell types, including bone, cartilage, muscle, and fat cells. They have potential therapeutic applications in many diseases.
  • Intravenous Administration: A method of giving medications or fluids directly into a vein using a needle or tube.
  • Bone Mineral Density: A measure of how much calcium and other minerals are present in a section of bone. It is used to diagnose osteoporosis and assess the risk of bone fractures.
  • Prenatal: Referring to the period before birth, during pregnancy.
  • Postnatal: Referring to the period after birth.
  • Clinical Trial: A research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease.
  • Phase I/II Trial: A clinical trial that combines both phase I (testing safety and side effects) and phase II (testing effectiveness) of a new treatment.
  • Donor Specific Antibody-test: A blood test that checks if a recipient has antibodies against a potential donor's cells, which could lead to rejection of transplanted cells or tissues.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-boost-cells-for-treating-severe-osteogenesis-imperfecta-in-children-before-and-after-birth/