What side effects are associated with this type of intervention?

Common treatments for Amyotrophic Lateral Sclerosis (ALS) include riluzole, edaravone, and stem cell therapies. Riluzole can cause side effects such as nausea, weakness, and liver function abnormalities. Edaravone may lead to bruising, gait disturbances, and allergic reactions. Stem cell therapies, particularly those involving neural progenitor cells and neurotrophic factors like GDNF, are still under investigation, but potential side effects include immune reactions, infection, and the risk of tumor formation. The investigational treatment CNS10-NPC-GDNF aims to promote neural cell survival, and while it has shown promise in animal models, its safety profile in humans is still being evaluated.

What prior FDA approvals exist?

The FDA has approved several treatments for Amyotrophic Lateral Sclerosis (ALS). Riluzole, approved in 1995, is the first drug shown to extend survival. Edaravone, approved in 2017, helps slow the decline in daily functioning. Recently, sodium phenylbutyrate-taurursodiol (PB-TURSO) was approved based on its ability to slow functional decline and extend time to tracheostomy and hospitalization. While these treatments do not involve cell transplantation or neurotrophic factors like GDNF, they represent significant advancements in ALS management. The investigational CNS10-NPC-GDNF therapy, which involves transplanting neural progenitor cells engineered to produce GDNF, is a novel approach aimed at promoting neural cell survival and could potentially complement or enhance existing treatments.

What other types of research exist?

Promising novel alternatives to CNS10-NPC-GDNF for ALS treatment include: 1) Insulin-like growth factor-I (IGF-I) therapy, which has shown potential in slowing ALS progression in clinical trials. 2) Vascular endothelial growth factor (VEGF) therapy, which has demonstrated neuroprotective effects in both animal models and early human studies. 3) Mesenchymal stem cell (MSC) therapy, which is being explored for its ability to modulate immune responses and promote neuroregeneration. 4) Gene therapy approaches targeting the reduction of abnormal SOD1 protein, which have shown promise in preclinical models. These alternatives are being actively researched and may offer new hope for ALS patients.

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Stem Cell Therapy

CNS10-NPC-GDNF for ALS

Phase 1

Recruiting

Led By Richard Lewis, MD

Research Sponsored by Cedars-Sinai Medical Center

Eligibility Criteria Checklist

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

Must have

Forced Vital Capacity ≥50% of predicted normal in supine

Age: 18 years or older

Must not have

Persons of childbearing capacity not willing to practice birth control

Donor Specific Antibodies (DSA) ≥ 2500MFI or CPRA ≥ 20%

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 9-hole peg testing will be performed 7 times over 15 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety of placing special cells into the brains of ALS patients. These cells release a protein that helps brain cells stay alive and healthy. The study aims to see if this new treatment is safe for people.

Who is the study for?

This trial is for adults over 18 with ALS symptoms for less than 3 years, who can consent and have a caregiver. They must be able to breathe well on their own (FVC ≥50%), travel to the site, and not be on certain drugs or have used stem cells before. Women must test negative for pregnancy and use birth control.

What is being tested?

The safety of CNS10-NPC-GDNF cell transplants into the brain's motor cortex is being tested. These are engineered stem cells that become neural cells producing GDNF, a protein aiding neural cell survival, previously tested in spinal cords but now in human brains.

What are the potential side effects?

Potential side effects aren't specified here but may include typical risks associated with craniotomy surgery, immunosuppression therapy such as infection risk increase, reaction to foreign cells, or neurological changes due to intervention.

Eligibility Criteria

Inclusion Criteria

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

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My lung function is at least half of what is expected for someone healthy.

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I am 18 years old or older.

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My ALS symptoms started less than 3 years ago.

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I have growing weakness in my arms, confirmed by an EMG test.

Exclusion Criteria

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

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I am not willing to use birth control.

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My antibody levels are high, indicating a possible rejection risk.

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I have no conditions that limit my arm strength or coordination tests.

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I have not had untreated major depression or psychosis in the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 9-hole peg testing will be performed 7 times over 15 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~9-hole peg testing will be performed 7 times over 15 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 9-hole peg testing will be performed 7 times over 15 months for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Compound Motor Action Potential (CMAP)

Force Generation via Accurate Test of Limb Isometric Strength (ATLIS) testing

Functional Hand assessments using 9-hole peg test

+4 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: CNS10-NPC-GDNF - Group CExperimental Treatment1 Intervention

Unilateral Motor Cortex, 0.5x10\^6 cells in 10 µL/site, 21 sites (10.5x10\^6 total cells) - Motor cortex corresponding to the dominant hand

Group II: CNS10-NPC-GDNF - Group BExperimental Treatment1 Intervention

Unilateral, Motor Cortex, 0.5x10\^6 cells in 10 µL/site, 21 sites (10.5x10\^6 total cells) - Motor cortex corresponding to the non-dominant hand

Group III: CNS10-NPC-GDNF - Group AExperimental Treatment1 Intervention

Unilateral, Motor Cortex, 0.25x10\^6 cells in 10 µL/site, 21 sites (5.25x10\^6 total cells) - Motor cortex corresponding to the non-dominant hand

0 / 8Eligibility 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 Amyotrophic Lateral Sclerosis (ALS) focus on protecting motor neurons and supporting their function. One promising approach involves the transplantation of neural progenitor cells engineered to produce glial cell line-derived neurotrophic factor (GDNF), such as in the CNS10-NPC-GDNF trial. GDNF promotes the survival and health of neural cells, which is crucial for ALS patients as the disease leads to the progressive loss of motor neurons. Other treatments aim to reduce excitotoxicity by lowering excessive glutamate levels, modulate inflammation to prevent further neuronal damage, and enhance overall neuroprotection. These mechanisms are vital for slowing disease progression and improving the quality of life for ALS patients.

Transplantation of Neural Progenitor Cells Expressing Glial Cell Line-Derived Neurotrophic Factor into the Motor Cortex as a Strategy to Treat Amyotrophic Lateral Sclerosis.CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unit.