Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurological (nervous system) disease. Sometimes called Lou Gehrig's disease, ALS belongs to a group of disorders known as motor neuron diseases.
ALS attacks nerve cells, called motor neurons, that control voluntary muscles (the muscles we are able to control). This leads to progressive weakness and disability. When these cells die, voluntary muscle control and movement are lost. People with ALS eventually lose their strength, ability to move their arms, legs, and body, and the ability to breathe on their own. In most cases, their minds remain sharp and alert.
The average life expectancy for people with ALS is 2 to 5 years. However, ALS is a variable disease that progresses at different rates for different people.
If you think you or your loved one has ALS, or you have already been diagnosed with ALS, it is important to talk to your healthcare provider (HCP) and ask about your treatment options as soon as possible.
While ALS is a rare disease, it is one of the most common neuromuscular conditions. Sometimes ALS is inherited, but in most cases, there is no known cause. ALS is not contagious.
ALS more commonly affects white men, non-Hispanics, and people aged 60 to 69. But younger and older people can develop ALS. Men are more likely to develop ALS than women.
The ALS Association reports that:
Not all people experience the same symptoms or progression with ALS. Some early signs and symptoms of ALS include:
There is currently no cure for ALS. However, there are many medications that are used to help relieve symptoms, such as muscle cramps and fatigue, although they are not indicated to treat the disease. There are also 2 prescription medications that are FDA-approved for the treatment of ALS:
The stem cell therapy involves extracting mesenchymal stem cells from the bone marrow of ALS patients. These stem cells are then manipulated in culture into cells that secrete a growth factor called NeuroTrophic Factor (NTF), which helps keep nerve cells in the brain and spinal cord healthy and alive. The NTF-secreting stem cells (called NurOwn cells) are then transplanted back into the same ALS patient (making this an autologous stem cell therapy) by injection into either the spinal fluid or the muscles.
The NurOwn method was developed by BrainStorm Cell Therapeutics, a biotech company based in the US and Israel. Results from the clinical trial showed that a majority of the patients benefitted from the NurOwn stem cell therapy.
HMO Principle scientist and senior author on the study, Dr. Dimitrios Karussis, explained: " Close to 90% of patients who were injected intrathecally through the spinal cord fluid were regarded as responders to the treatment either in terms of their respiratory function or their motor disability. Almost all of the patients injected in this way showed less progression and some even improved in their respiratory functions or their motor functions.”
A PRNPresswire Release covering this study called the stem cell therapy the “first-of-its-kind treatment for treating neurodegenerative diseases.”
Not a cure just yet
This stem cell therapy will need to be tested in more patients before the it can be determined truly effective in slowing progression of ALS. And Dr. Karussis was quick to note that the NurOwn stem cell therapy isn’t a cure for ALS, but rather an early-stage therapy that will provide significant benefit to patients by slowing disease progression.
“I am optimistic that within the foreseeable future, we may provide a treatment to ALS patients that can slow down or stop the progression. I believe we are in the early stages of something new and revolutionary with this harvested stem cell infusion therapy. While this is absolutely by no means a cure, it is the first step in a long process in that direction. I see this treatment as being potentially one of the major future tools to treat degenerative diseases of the brain and spinal cord, in general.”
If you are experiencing early signs and symptoms or you think you may have ALS, it's important to talk to your healthcare provider (HCP) as soon as possible. Your HCP can help:
Receiving a diagnosis of ALS can be overwhelming. But getting an answer as early as you can may allow you to start treatment as soon as possible.
To assess the progression of ALS, your HCP may ask questions about your daily functioning and any problems with speech, swallowing, or breathing. One tool your HCP may use is the ALS Functional Rating Scale–Revised (ALSFRS-R).
The ALSFRS-R measures the loss of functional ability in people with ALS. The scale consists of 12 items: speech, salivation, swallowing, handwriting, cutting food, dressing and hygiene, turning in bed, walking, climbing stairs, dyspnea (difficulty breathing or shortness of breath), orthopnea (trouble breathing when lying down), and respiratory insufficiency.
Each item of the ALSFRS-R is scored between 4 and 0. Higher scores represent greater functional ability. A maximum total score is 48 and the minimum total score is 0.
The information and advice provided here are general in nature and are not intended to be a substitute for professional medical advice, diagnosis, or treatment. You are strongly encouraged to seek the advice of your doctor or other qualified healthcare provider with any questions regarding a medical condition.