What side effects are associated with this type of intervention?

The most common side effects of intravenous allogeneic mesenchymal stem cell (MSC) therapy, which is being studied for adrenoleukodystrophy (ALD), include mild to moderate adverse reactions such as hypersensitivity reactions, mild lymphadenopathy, and potential immune responses. Other regenerative and immunomodulatory treatments may also carry risks of infection, graft-versus-host disease, and complications related to the administration procedure. Overall, while MSC therapy shows promise, careful monitoring for these side effects is essential.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Adrenoleukodystrophy (ALD) that utilize intravenous allogeneic mesenchymal stem cells (MSCs) with regenerative and immunomodulatory properties. The primary treatment approach for ALD has been hematopoietic stem cell transplantation (HSCT), which aims to halt the progression of the disease. The use of MSCs is still under investigation in clinical trials and has not yet received FDA approval for this indication.

What other types of research exist?

Promising alternatives to IV-MSC for Adrenoleukodystrophy patients include gene therapy and hematopoietic stem cell transplant (HSCT). Gene therapy aims to correct the genetic defect causing ALD, while HSCT can replace the defective cells with healthy ones. Lorenzo's oil is also used to manage ALD, particularly in asymptomatic patients or those with early-stage disease.

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Stem Cell Therapy

MSC Therapy for Adrenoleukodystrophy

Phase 1

Waitlist Available

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation

Age ≥ 3 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

No Placebo-Only Group

Summary

This trial involves injecting donor stem cells into patients with active CALD to help manage symptoms and stabilize their condition while they wait for a more permanent treatment.

Who is the study for?

This trial is for patients with active cerebral adrenoleukodystrophy (CALD) who are over 3 years old, have not had gene therapy or a transplant yet, and are expected to live more than 6 months. They must have certain MRI findings and blood tests showing proper organ function.

What is being tested?

The study is testing the use of mesenchymal stem cells given through an IV as a temporary treatment for CALD while waiting for a stem cell transplant or gene therapy. This intervention fills the gap between CALD diagnosis and these longer-term treatments.

What are the potential side effects?

Potential side effects may include reactions related to immune response, such as inflammation, or issues due to sedation required for administering the treatment. Specific side effects will depend on individual patient responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with ALD based on specific tests.

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I am at least 3 years old.

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I have never had gene therapy or a transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).

Secondary study objectives

Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: IV-MSC for cALD (Early Disease/Bridge Therapy)Experimental Treatment1 Intervention

Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.

Group II: IV-MSC for cALD (Advanced Disease)Experimental Treatment1 Intervention

Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Adrenoleukodystrophy (ALD) include Lorenzo's oil, hematopoietic stem cell transplantation (HSCT), and emerging therapies like intravenous allogeneic mesenchymal stem cells (IV-MSC). Lorenzo's oil works by reducing the levels of very long-chain fatty acids (VLCFAs) in the blood, which are toxic to the brain. HSCT aims to replace the defective hematopoietic cells with healthy ones, potentially halting the progression of cerebral ALD. IV-MSC therapy, currently under study, leverages the regenerative and immunomodulatory properties of mesenchymal stem cells to repair damaged tissues and modulate the immune response, potentially providing a bridge to more definitive treatments like HSCT or gene therapy. These treatments are crucial for ALD patients as they target the underlying pathophysiology of the disease, aiming to slow or halt its progression and improve quality of life.

"Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: rationale and current assessment of efficacy.