Intraspinal Transplantation of Autologous ADRC in ALS Patients

Study Overview
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Brief Summary
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The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).

Detailed Description
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Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a short time leads to significant impairment of motor functions and death. The frequency of ALS is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a younger age. Since the symptom onset is most frequently in the fifth or sixth decade, ALS is a huge economic burden for the society. There are many studies conducted to treat the disease and prevent it, but currently the medicine offers only one drug that can slow the appearance of disease symptoms but could not stop the progression. Yet, improvements in medical management, including nutrition and breathing, regularly increase patient survival - 50% of affected patients live at least 3 or more years after diagnosis; 20% live 5 years or more; and up to 10% will survive more than 10 years. The stem-cell-based therapies could be therefore a new waited strategy for ALS clinical treatment. Since the clinical course of ALS may vary substantially between patients, we are planning to qualify them with a primarily established clinical course based on a detailed anamnesis and clinical assessment. The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. In order to select a group of 30 ALS patients for the ADRC treatment, approximately 50 ALS patients will be examined. All patients enrolled will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).

Official Title
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Safety and Efficacy of Intraspinal Transplantation of Autologous ADRC in ALS Patients

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: clinically definite or probable ALS according to El Escorial criteria life expectancy of more than 1 year INR ≤2 before liposuction lack of treatment with immune-suppressants and/or corticosteroids within min. 20 days prior to recruitment constant riluzole treatment (50 mg/bid) throughout the study period compliance with treatment regimen e.g. will and possibility to attend check-up visits Polish citizens Exclusion Criteria: primary haematological disease, including hypercoagulable states Presence of comorbidity that would stand in the way of neurosurgical treatment- previous history of a spinal-cord surgery at the clinically affected level previous/current history of neoplasm or comorbidity that could impact upon patient's survival PEG pregnancy /lactation noninvasive/invasive mechanical ventilation at time of recruitment alcohol abuse, cocaine amphetamine, etc.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Autologous ADRC injection<br> | Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients<br>* 3 injections of ADRC: 1 intraspinal and 2 intrathecal<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Functional progression rate | Delta ALS Functional Rating Scale (ALSFRS-R)/month | 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Electrophisiological progression rate | electromyography (EMG)/ Motor unit number index (MUNIX) | 3 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, ADRC, adipose derived regenerative cells, intraspinal transplantation, mesenchymal cells