Research trials will provide many patients access to an investigational medication that might slow the progression of Multiple System Atrophy (MSA).
Benefits of participating in clinical trials at no cost to you or your family may include:
- Lab Work
- MRI Scans
- Neurological Exams
- More Access to Physicians Investigational Medications (Placebo Controlled)
- Lodging and Meals
- Travel (Within US for caregiver & patient)
Investigational Treatment for Multiple System Atrophy (MSA)
CenExel RMCR is conducting clinical studies that will provide many patients with a new investigational medication. The study seeks to determine if the investigational treatment will safely slow the progression of MSA.
Multiple system atrophy is a rare disease that affects the nervous system and impacts an individual’s motor function, balance and coordination, blood pressure, heart rate, sexual function, digestion, and elimination.
If you have been diagnosed with multiple system atrophy, you may be experiencing the difficulties outlined above. Daily activities of self-care may be more challenging for you and your loved ones. Symptoms may cause you to feel as though you are losing your independence.
Multiple Systems Atrophy (MSA) is commonly misdiagnosed especially early on in the course of the disease. It usually presents with Parkinsonism (MSA-P) or with incoordination of the limbs and gait (MSA-C). In North America, Parkinsonism is the more common presentation. Although often indistinguishable from Parkinson’s disease early on in the disease, patients with MSA often have more symmetric features of Parkinsonism and may have less tremor compared to patients with Parkinson’s disease. The progression of Parkinsonism is usually more rapid in patients with MSA-P than with patients with Parkinson’s disease. Many patients with MSA-C fail to respond to levodopa therapy or may respond poorly. Of those patients who initially respond to levodopa, most tend to gradually lose their levodopa response over a few years.
Patients with MSA-C have to be distinguished from other causes of incoordination, especially other neurodegenerative diseases affecting the cerebellum such as Spinocerebellar Ataxia.
Parkinsonism in patients with MSA is usually treated with standard antiparkinson’ s medications such as levodopa. For patients that develop involuntary movements (dyskinesia) associated with levodopa, amantadine may be used to reduce these abnormal involuntary movements. In addition, some patients who fail to respond to levodopa or who respond poorly may have additional benefit for bradykinesia (slowness of movement) with amantadine therapy. Amantadine may also be used for incoordination associated with MSA-C though it is likely that less than 25% of patients have a clinically significant improvement for incoordination. Botulinum toxin (Botox) injections may be used for abnormal posturing (dystonia) or muscle spasms. Blood pressure drops causing lightheadedness or fainting is often treated with lifestyle measures (increased fluids and salt, compression stockings, abdominal binder) and medications to increase blood pressure (e.g. midodrine, fludrocortisone, droxidopa, and pyridostigmine). Bladder dysfunction may be treated with standard medications to reduce urinary urgency (e.g. trospium, tolterodine, etc.), but when bladder control becomes increasingly poor, use of a catheter or diaper may be indicated. Constipation is often problematic for MSA patients and taking extra fluids and fiber may be helpful in addition to a combination of laxatives (e.g. MiraLAX, Senokot, etc.).
Most patients with MSA can benefit from physical therapy, predominantly focusing on gait and balance to prevent falls as well as a stretching and home exercise program to maintain fitness and prevent deconditioning and the development of contractures (reduced range of motion of joints). As the disease progresses, use of a walking aid such as a cane or walker is typically needed. Manual dexterity may be affected both by Parkinsonism and incoordination so occupational therapy intervention to help with a use of adaptive devices may help to maintain independence for activities of daily living. Speech and swallowing are often affected and may result in difficulties with eating and drinking. Speech therapy focusing on loudness of speech may be helpful for some patients with softness usually associated with MSA-P. As MSA progresses, patients may develop choking or coughing, more commonly when drinking than when eating. Swallowing evaluation, with the help of a speech therapist, is often helpful to guide instruction on compensatory techniques to reduce the risk of pneumonia caused by food or liquid entering the lungs. When the risk of pneumonia becomes high, some patients benefit from dietary alterations including thickening of liquids and change in food textures. Some patients elect to undergo feeding tube placement to enable maintenance of nutrition.
There is currently extensive research ongoing to better understand the cause and progression of MSA.
Medical professionals are evaluating an investigational medication designed to suppress an enzyme called myeloperoxidase (MPO), believed to cause inflammation and damage to neurons in the brains of patients with multiple system atrophy. The main purpose of the study is to see if it helps slow disease progression. The investigational medication tablets are taken twice daily.
Patients who are already taking stable doses of medications used to treat MSA symptoms may be able to continue their current medications while participating in the study. After determining eligibility, study participation lasts for about 11 months and requires participation from a patient caregiver who will need to attend seven study visits spread out over that time with the patient.
Do you or a loved one have MSA?
Please fill out the survey to help us determine if you, or your loved one, may qualify for an investigational study aiming to potentially slow disease progression.