ALS Ice Bucket Challenge Progress

 

To keep you abreast of available options for trial and research participation in our region, The ALS Association Greater Philadelphia Chapter compiles and distributes information on local opportunities. For information about ALS research trials and studies across the U.S., click here.

Category: Drug Trial

Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum. The drug is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has exhibited strong efficacy in several cellular and animal models of ALS. Furthermore, PB and TUDCA have been individually tested in clinical trials of ALS and both showed safety, tolerability, and preliminary signs of efficacy. The trial will test the safety and tolerability of AMX0035, as well as functional outcomes. Analysis of biomarkers of cell function, neuronal damage, and inflammation will be included as a major part of the trial, along with a new measure of muscle strength that has been shown to be tightly correlated with disease progression.

Contact Information

Temple University School of Medicine
Kathleen Hatala, RN, BSN
215-707-4171
kathleen.hatala@tuhs.temple.edu

Category: Drug Trial

Open Label Extension Study of AMX0035 in Patients with ALS (CENTAUR-OLE) Subjects that successfully Complete AMX0035 will be rolled over to the Open Label Extension

*This is only available to patients that have completed AMX0035.

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Contact Information

The Penn Comprehensive ALS Center
Kelly Almasy, RN
215-829-5041
kathleen.hatala@tuhs.temple.edu

Temple University School of Medicine
Kathleen Hatala, RN, BSN
215-707-4171
kathleen.hatala@tuhs.temple.edu

Category: Drug Trial

A Study to Assess the Efficacy and Safety of H.P. ActharŽ Gel in the Treatment of Subjects with ALS

This is a multicenter, multiple dose study to examine the effect of H.P. ActharŽ (Acthar) on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS).

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Contact Information

Penn State Hershey Medical Center
Heidi Runk, CCRC
717-531-0003 ext. 287177
hrunk@pennstatehealth.psu.edu

Category: Drug Trial

Orion (Levosimendan) REFALS

This is a Phase 3 clinic trial of levosimendan (ODM-109) and will look at respiratory function. According to preclinical research, Orion believes that levosimendan helps muscles contract more easily by sensitizing the skeletal muscle’s contractile apparatus to calcium signaling.

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Contact Information

The Penn Comprehensive ALS Center
Kelly Almasy, RN
215-829-5041
kathleen.hatala@tuhs.temple.edu

Temple University School of Medicine
Kathleen Hatala, RN, BSN
215-707-4171
kathleen.hatala@tuhs.temple.edu

Category: Drug Trial

Arimoclomol in Amyotrophic Lateral Sclerosis

A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotrophic lateral sclerosis (ALS).

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Contact Information

The Penn Comprehensive ALS Center
Kelly Almasy, RN
215-829-5041
kelly.almasy@uphs.upenn.edu

Category: Biomarker Study

This biomarker study is collecting biofluids (blood and urine) from people with motor neuron disease and control participants to identify specific biomarkers, which can be used to track disease progression as well as identify a specific sub-group of patients for diagnosis and development of tailored (sub-group specific) therapies.

Contact Information

The Jefferson Weinberg ALS Center
Piera Pasinelli, PhD at
piera.pasinelli@jefferson.edu or
215-955-8394
or
Katelyn Russell at katelyn.russell@jefferson.edu

Category: Biomarker Study

Phenotype, Genotype & Biomarkers in ALS and Related Disorders Currently Enrolling

The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.

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Contact Information

The Penn Comprehensive ALS Center
Kelly Almasy, RN
215-829-5041
kelly.almasy@uphs.upenn.edu

Category: Biomarker Study

Validation of ALS Biomarkers (ALSA BIO3)

The purpose of this study is to learn more about the underlying cause of ALS, as well as find unique biological markers which could be used to diagnose ALS and monitor disease progression. This is done by collecting and comparing blood samples from healthy subjects, and both blood and cerebrospinal fluid (CSF) samples from people with ALS, and other forms of neurodegenerative diseases, over the course of their illness. This project will provide scientists with a wide range of samples and information to help in identifying biomarkers associated with ALS (what changes are unique to ALS) and assess therapeutic targets. The samples and clinical information will be made available to other investigators via the NEALS Biorepository.

Contact Information

Temple University School of Medicine
Kathleen Hatala, RN, BSN
215-707-4171
kathleen.hatala@tuhs.temple.edu

Category: Genetics Research
Name of Study

Genomic Translation for Amyotrophic Lateral Sclerosis Care (GTAC)

The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms present and progress with a particular pattern. This study is only available to patients who are seen at the Hershey Clinic for their ALS care.

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Contact Information

Penn State Hershey Medical Center
Matthew Bankert
717-531-0003 ext. 323351
mbankert1@pennstatehealth.psu.edu

Category: Tissue Bank Program

This program aims to further the understanding of how ALS and other neurological disorders develop, by establishing a repository of postmortem brain, spinal cord, and other tissues that will be used to advance research. The tissue bank will be built through donations from ALS, other neurological diseases and non-neurologic control patients. Paired with thorough case profiles, this banking program will facilitate efforts to identify pathology, biomarkers, molecular targets, and mechanisms of ALS. The study is open to both Jefferson and non-Jefferson patients.

Contact Information

The Jefferson Weinberg ALS Center
Piera Pasinelli, PhD
piera.pasinelli@jefferson.edu
or
215-955-8394
or
Katelyn Russell katelyn.russell@jefferson.edu

Category: Tissue Bank Program

Tissue Donation at Temple Hospital

In a disease like ALS in which the cause is unknown, no animal models of ALS can substitute for understanding how it affects humans. We are currently collecting blood, urine, cerebrospinal fluid (CSF) and autopsy materials from people with ALS and other motor neuron diseases to look for clues in the human tissue. All samples are tied to de-identified clinical information in a database to help maximize the usefulness of this precious resource. This includes demographic information, environmental exposures, and medical history. This will increase availability of human tissue for research with pertinent corresponding clinical information.

Contact Information

Temple University School of Medicine
Terry Heiman-Patterson, MD
terryheimanpatterson@gmail.com
or
Kathleen Hatala, RN, BSN
215-707- 4171
kathleen.hatala@tuhs.temple.edu

Category: Improving Trial Design and Delivery of ALS Drugs

Improving Delivery of ALS Drugs

Despite the progress in research, ALS remains a disease with no effective treatment. Riluzole and Edaravone slow down the patients’ functional decline, but do not stop or reverse the disease. One explanation for our inability to stop ALS in its first signs stems from a phenomenon that occurs at the blood-brain and spinal cord barrier that is called pharmaco-resistance. This phenomenon is driven by several proteins that function as pumps. These pumps work to protect the brain and the spinal cord from a toxic environment that comes from outside the nervous system and/or is created by the disease. Unfortunately, these pumps are not very selective and in their attempt to pump out toxins; they also pump out drugs and medications. In ALS it is the disease itself that activates these pumps and drives the patients’ response to therapies, which varies from patient to patient. With this study, we are using patients’ derived cells (induced pluripotent stem cells [iPSCs] to re-create in vitro the patients’ blood-brain barrier so we can measure the activity of these pumps and study how the pumps get activated. Because each patient (or group of patients) is different, the ultimate goal of this study is to identify groups of patients with highly sensitive pumps (drug-resistant patients) and patients with moderately or not sensitive pumps (drug-responsive patients). Using the same in vitro blood-brain barrier systems, we are also testing medications that can slow or block the function of these pumps. Ultimately, this study helps us design better clinical trials grouping patients in drug-sensitive vs drug-resistant patients. In the drug-resistant patients, researchers can use the pump-blockers to allow the ALS drug to remain in the brain and the spinal cord longer and to provide its beneficial effect.

Contact Information

The Jefferson Weinberg ALS Center
Piera Pasinelli, PhD at
piera.pasinelli@jefferson.edu or
215-955-8394
or
Katelyn Russell at katelyn.russell@jefferson.edu

Category: Technology

EEG-Based Brain-Computer Interface

By using EEG (brain wave) signals from the scalp to create a signal, the Brain-Computer Interface (BCI) allows people to make selections from the computer screen. The study is intended to evaluate both the complexity of the system and the degree to which each participant will be able to communicate. Trials will consist of asking the subject to follow a series of simple instructions and to complete certain tasks while using the BCI. We now have a home-based study underway.

Contact Information

Temple University School of Medicine
Terry Heiman-Patterson, MD
terryheimanpatterson@gmail.com
or
Kathleen Hatala, RN, BSN
215-707- 4171
kathleen.hatala@tuhs.temple.edu

Category: Caregivers

Caregiver Burden in ALS with Frontotemporal Dementia

While ALS primarily affects the motor system, approximately 40% of people with ALS have cognitive impairment predominantly with executive dysfunction. Approximately 20% show behavioral changes, predominantly apathy and disinhibition. These cognitive and behavioral changes add significantly to the burden and distress of caregivers who have to face the challenges of managing behavioral changes in addition to the physical disability. The overall goal of this study is to characterize the relationship of caregiver burden to cognitive and behavioral impairment in ALS and to develop and perform a feasibility study of a pilot individualized intervention as a way to reduce caregiver burden. Ultimately, this study will provide the necessary background to develop, inform and guide future targeted and larger scale interventions directed to both patients and caregivers.

Contact Information

Temple University School of Medicine
Terry Heiman-Patterson, MD
terryheimanpatterson@gmail.com
or
Kathleen Hatala, RN, BSN 215-707- 4171
kathleen.hatala@tuhs.temple.edu