Clinical Activities

Clinical Care at the MGH: Caring Expertise

The purpose of the Neuromuscular Clinic at the MGH is to provide expert diagnostic services as well as access to innovative research studies and trials while the patient remains under the care of their primary care physician. This clinic, sponsored in part by the Muscular Dystrophy Association, uses a multi-disciplinary approach to the diagnosis and treatment of neuromuscular disorders. Within one visit, patients are able to schedule appointments with their MGH neurologist while also using the expertise of MGH pulmonary specialists, physical, occupational and speech therapists. At the end of each clinic, neurologists hold a collaborative review and group discussion of individual patient treatments and therapies. This approach is invaluable to each patient's care.

Clinical Trials: Offering Access to Potential Therapies

One of the functions of the neuromuscular clinic is to conduct clinical trials. These are studies that test the efficacy of potential ALS treatments. These trials are traditionally defined in three stages, or phases. Each phase is designed to further our understanding of ALS treatment by answering specific questions.

The MGH has been leading studies of promising new therapies since 1994. Dr. Merit Cudkowicz established the Neurology Clinical Trials Unit. This unit is dedicated to rapid, accurate execution of drug trials centered on the ALS Clinic and the Partners Neurology department. In addition, Drs. Cudkowicz and Brown have founded the Northeast ALS group (NEALS) to facilitate rapid drug trials.

MGH is currently active in the following ALS clinical trials:

BrainGate Pilot Clinical Study
ALS Participants Needed For Research Study
Patients who have motor neuron disease such as amyotrophic lateral sclerosis (ALS) and have no or limited use of their hands are needed for an FDA regulated research study to evaluate a new technology which may allow an individual with motor neuron disease to control a computer cursor by thought. This study is invasive and requires surgery. You must live within 2 hours of Boston, Massachusetts to participate. The study requires a commitment of 13 months. This study is being conducted by Dr. Leigh Hochberg at Massachusetts General Hospital.

If you would like to learn more about this study please contact Dr. Leigh Hochberg at (617) 726-4218.

Topiramate
MGH was the Coordination Center for this 20 center clinical trial involving 288 subjects. This is the first NIH funded clinical trial in ALS. Enrollment for this study was completed in 12 months; results available in  August 2001 did not confirm that topirimate is beneficial in ALS.

Creatine
MGH is the Co-Principal Investigator with Dr. Shefner. This is an 18 center clinical trial. All data management and analysis are at the MGH. The trial started 11/2000 and was completed in 2003.  No benefit of creatine in ALS was detected. This study was sponsored by the Muscular Dystrophy Association (MDA).

Buspar
This small study was conducted by MGH and Johns Hopkins University with funding from ProjectALS to test the hypothesis that Buspar can produce transient improvements in pulmonary function in ALS.

Dextramethorphan/quinidine
This compound was tested and found to be beneficial in a phase III study as a treatment for emotional liability ("pseudobulbar affect") in ALS.  The compound is manufactured by Avanir, Inc. which sponsored this study.

Celebrex
MGH was the Coordination and Data Management Center for the clinical trial of Celebrex in ALS.  As of October, 2004, the Northeast ALS consortium completed this study in 300 people with ALS treated for one year.  At 800 mg per day, Celebrex was safe and well tolerated.  Celebrex did not have any demonstrated beneficial effects on ALS disease course.  Studies are underway to assess whether Celebrex as used in this study had the predicted pharmacologic effects in the treated participants.  The study results will be presented at the International Motor Neuron Disease Association on December 3, 2004.

Ceftriaxone

The MGH and the Northeast ALS consortium have been awarded a NIH grant to test the safety and efficacy of ceftriaxone in patients with ALS.  Enrollment is anticipated to begin in the first quarter of 2005.

Sodium Phenylbutyrate

In late 2004 or early 2005, the MGH and the Bedford VA Hospital will begin a safety and tolerability study of sodium phenylbutyrate in patients with ALS.

Additional Clinic Referrals and Clinical Trials Information

To obtain additional information on clinic appointments, call the neuromuscular secretary at 617.724.3914. For additional information on clinical trials, please contact a clinical trials coordinator at 617-724-4246.