What is the mechanism of action of this treatment?

Hurler Syndrome treatments primarily include Enzyme Replacement Therapy (ERT) and Gene Therapy. ERT involves administering a synthetic version of the deficient enzyme alpha-L-iduronidase, which helps break down accumulated glycosaminoglycans (GAGs) in tissues. Gene Therapy introduces a functional copy of the alpha-L-iduronidase gene into the patient's cells, enabling natural enzyme production. These treatments are vital as they target the root cause of Hurler Syndrome, improving symptoms and preventing further tissue damage.

What side effects are associated with this type of intervention?

The most common treatments for Hurler Syndrome include enzyme replacement therapy (ERT) with laronidase and gene therapy. Known side effects of ERT with laronidase can include infusion-related reactions such as fever, rash, and respiratory issues, as well as potential antibody formation against the enzyme. Gene therapy, while still under investigation, may present risks such as immune responses to the viral vectors used for gene delivery, insertional mutagenesis, and other off-target effects. Both treatments aim to address the deficiency of alpha-L-iduronidase, but careful monitoring for adverse effects is essential.

What prior FDA approvals exist?

The FDA has approved enzyme replacement therapy for the treatment of Hurler Syndrome (Mucopolysaccharidosis Type I). Laronidase (Aldurazyme) is an enzyme replacement therapy that has been approved to treat patients with MPS I by supplementing the deficient alpha-L-iduronidase enzyme. This treatment helps reduce the accumulation of glycosaminoglycans, which are responsible for the symptoms of the disease. Gene therapy approaches are also being explored, but as of now, enzyme replacement therapy remains a primary FDA-approved treatment for addressing alpha-L-iduronidase deficiency in Hurler Syndrome.

What other types of research exist?

Promising novel alternatives to ISP-001 for Hurler Syndrome patients include: 1) Gene therapy approaches such as hematopoietic stem cell gene therapy, which involves modifying the patient's own stem cells to produce alpha-L-iduronidase. 2) Advanced enzyme replacement therapies that may offer improved delivery and efficacy, such as those utilizing fusion proteins or nanoparticles to enhance enzyme uptake by cells. 3) Substrate reduction therapies that aim to decrease the accumulation of glycosaminoglycans, thereby reducing the burden on deficient enzymes. These alternatives are currently being explored in clinical trials and research studies, showing potential for improved outcomes in treating Hurler Syndrome.

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Virus Therapy

Engineered B Cell Therapy for Mucopolysaccharidosis I

Phase 1

Recruiting

Research Sponsored by Immusoft Corporation

Eligibility Criteria Checklist

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

Must have

Must commit to traveling to the study site for the necessary follow-up evaluations.

Age ≥ 18 years at time of study registration.

Must not have

History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.

Requirement for systemic immune suppression.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1year

Awards & highlights

No Placebo-Only Group

Summary

This trial tests ISP-001, which uses modified immune cells to help adults with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie by making a missing enzyme. The goal is to see if this approach is safe and tolerable.

Who is the study for?

Adults over 18 with Mucopolysaccharidosis type I Hurler-Scheie or Scheie can join this trial. They must live close to the study site, have good kidney function and heart health, and be able to attend all follow-ups. Those with certain medical conditions, oxygen needs, immune suppression, past stem cell transplants or B cell cancers cannot participate.

What is being tested?

The trial is testing a new treatment using patients' own modified B cells (ISP-001) for adults with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie. It's a first-of-its-kind study focusing on safety and how well it works in humans.

What are the potential side effects?

As this is a first-in-human study of ISP-001, specific side effects are not yet known but may include typical risks associated with cellular therapies such as immune reactions, infusion-related symptoms, and potential impacts on blood counts.

Eligibility Criteria

Inclusion Criteria

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

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I can travel to the study site for follow-up visits.

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I am 18 years old or older.

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I have been diagnosed with a form of Mucopolysaccharidosis (MPS I).

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My kidneys are functioning well.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of B cell cancer, EBV disease, or autoimmune disorders.

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I need medication to suppress my immune system.

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I am experiencing symptoms of graft-vs-host disease.

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I have had a stem cell transplant before.

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I need extra oxygen all the time.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1year for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Analysis of PBMCs

Assessment of Storage Material (glycosaminoglycan, or GAG)

Concentration of IDUA

+3 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Autologous Plasmablasts (B cells)Experimental Treatment1 Intervention

Dose Level: 5 x 10e7 cells/kg on Day 0

0 / 9Eligibility 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

Hurler Syndrome treatments primarily include Enzyme Replacement Therapy (ERT) and Gene Therapy. ERT involves administering a synthetic version of the deficient enzyme alpha-L-iduronidase, which helps break down accumulated glycosaminoglycans (GAGs) in tissues. Gene Therapy introduces a functional copy of the alpha-L-iduronidase gene into the patient's cells, enabling natural enzyme production. These treatments are vital as they target the root cause of Hurler Syndrome, improving symptoms and preventing further tissue damage.