Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a fatal motor neuron disorder. This group of rare neurological disease mainly involve the dysfunction of nerve cells (neurons), responsible for controlling voluntary muscle movement. Voluntary muscles movements include chewing, breathing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. ALS belongs to a broader group of disorders caused by gradual deterioration and death of motor neurons, known as motor neuron diseases. These neurons initiate and provide vital communication links between the brain and the voluntary muscles by extending from the brain to the spinal cord and to muscles throughout the body.
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According to the National Institute of Neurological Disorders and Stroke, the respiratory failure is major cause death in ALS cases, most people die within 3–5 years from when the symptoms first appear. However, a lesser percentage of ALS patients, approximately 10% survive for 10 or more years. People with ALS lose their strength and to hold the body upright. Some individuals even find it difficult to breathe on their own. Several studies show, some people with ALS may develop cognitive problems. Despite many studies conducted to find the cause of ALS, only a small percentage of cases are found to be inherited and others have showed no known cause.
No cure has yet been found for ALS. However, few drugs have been approved by the Food and Drug Administration (FDA) to treat ALS. The drugs only prolong life by few months or slow down the clinical decline but does not relieve symptoms. Other treatments are designed to relieve symptoms and improve the quality of life for people with ALS. Drugs are available to help individuals with pain, panic attacks, depression and anxiety.
Amyotrophic Lateral Sclerosis: Epidemiology Insights
Report Ocean will focus on epidemiology segmentation at global level depending on the following characteristics:
- Total Prevalent Population of Amyotrophic Lateral Sclerosis
- Total Diagnosed Prevalent Population of Amyotrophic Lateral Sclerosis
- Type-specific Diagnosed Prevalent Population of Amyotrophic Lateral Sclerosis
- Age-specific Diagnosed Prevalent Population of Amyotrophic Lateral Sclerosis
- Gender-specific Diagnosed Prevalent Population of Amyotrophic Lateral Sclerosis
According to population-based studies, the prevalence of ALS is between 4.1 and 8.4 per 100 000 persons. In the United States, a prevalence of 5.2 per 100 000 was reported in 2015 as per the US National ALS Registry, which was similar to the prevalence of 5.0 per 100 000 as reported in 2014.
Maximum cases of ALS are sporadic, however, only 5–10% of the cases are familial ALS. Both types of ALS are associated with degeneration of cortical and spinal motor neurons. Even though the etiology of ALS remains unknown, the mutations of superoxide dismutase 1 have been known as the most common cause of FALS. Sporadic means the condition arises without a clear cause while, Familial ALS (FALS) is the case of inheritance.
As per the studies, the mean age of ALS onset is between 51 and 66 years. However, the condition is mostly diagnosed between 54 and 69 years of age. Type of ALS is one the major predictor for age at the time of disease onset. Patients with familial ALS tend to have an earlier age of onset compared with patients with sporadic ALS. The prevalence of this neurological disorder is greater in males than in females. In sporadic ALS men and women has different clinical features, there is greater likelihood of ALS onset in the spinal regions in males while in bulbar region in females. In case of FALS, sex does affect the clinical course of disease, but shows earlier onset in males.
Market for Amyotrophic Lateral Sclerosis
The market is accelerated by the increment observed in ALS cases. According to the National Organization for Rare Disorders (NORD), ALS affects approximately 30,000 people and almost 5,000 new cases are diagnosed each year in the United States. The worldwide market for ALS can be segmented by type, by treatment type and by end user.
- Type
- Sporadic ALS
- Familial ALS
- Treatment Type
- Stem cell therapy
- Physical therapy
- Speech therapy
- Respiratory therapy
- Medications
- Riluzole
- Nuedexta
- Radicava
- Others
- End user
- Hospitals
- Specialty Centers
- Research and Academic Institutes
- Others
Rising geriatric population along with increasing awareness about ALS and rise in disease occurrence rate supports the growth of this market. Moreover, the high number of unmet needs made the, manufacturers to focus on innovation and technological advances in treatment options which further the drive the market forward. On the other hand, the high cost associated with ALS along with difficulties arising in clinical trials due to the rarity of the disease may pose an impediment to the market during the forecast period.
In between types of ALS, sporadic segment has major market share due to increased prevalence of the condition. The medication segment is expected to hold the major share in the global ALS treatment market. The FDA has approved only two drugs for the treatment of ALS, Rilutek (riluzole), and Radicava (edaravone) while, Nuedexta (dextromethorphan/quinidine) has received the approval for the treatment pseudobulbar affect only which is a comorbidity of ALS. The increasing demand for new approaches and advanced treatment options has increased the share of stem cell therapy in the market. The requirement to visit and stay in the hospital during ALS treatment facilitates the growth of this segment among the end users. While, the research of innovative medication for ALS taking place in the various Research and Academic Institutes also supports the growth of market.
Companies across the globe are thoroughly working toward the development of new treatment therapies for ALS. Some of the key players in the therapeutic market of ALS at a global level are Cytokinetics, Orphazyme, Orion, Brainstorm Cell Therapeutics, Biogen, Anelixis Therapeutics, Implicit Bioscience, Clene Nanomedicine, Neurosense therapeutics, AMYLYX, Immunity pharma, MediciNova, Biohaven Pharmaceuticals, and others. While some of the companies have recently shifted their focus toward this indication, yet others are expected to create a significant influence on the market size during the forecast period.
Marketed Drugs
Rilutek (Riluzole; Covis Pharmaceuticals) is indicated for the treatment of ALS and mechanism by which riluzole exerts its therapeutic effects in patients with ALS is still unknown. This drug acts to slow the disease’s progression or perhaps reduces levels of a chemical messenger (glutamate) in the brain, which is often present in higher levels in people with ALS. This is administered orally in the form of a tablet. Many ALS patients will develop dysphagia (trouble swallowing) as their disease progresses, and this can lead to difficulty taking pills such as Rilutek.
Tiglutik (Riluzole; oral suspension; ITF Pharma) is the oral suspension, with the similar proposed mechanism as Rilutek, because both contain riluzole. However, this is administered via injection. There are various side effects like cause liver injury, neutropenia, interstitial lung disease, including hypersensitivity pneumoniti, etc.
Radicava (Edaravone; Mitsubishi Tanabe Pharma) is the treatment option for the ALS. In clinical trials, this drug has shown to decrease the decline of the physical functions. It is administered via intravenous infusion. There might be adverse effects after the intake because this contains sodium bisulfite, which might cause a type of allergic reaction that can be serious and life- threatening.
Emerging Drugs
ODM-109 (Orion) is an oral levosimendan in the Phase III clinical trial that is under evaluation for the treatment of the symptoms of the amyotrophic lateral sclerosis (ALS). It has been granted orphan drug designation in ALS by the US Food and Drug Administration (FDA).
Arimoclomol (Orphazyme) is in Phase III clinical trial. Arimoclomol amplifies and prolongs the activated, HSP-producing state of Heat shock factor 1 (HSF1). This leads to an amplification in the production of cell-protective HSPs, but only in physiologically stressed cells. This drug has also been tested for Niemann–Pick type C, Gaucher disease and others as well.
Biohaven Pharmaceuticals is also evaluating the safety and efficacy of Verdiperstat (BHV-3241; AZD3241) for the treatment of ALS in phase II/III clinical trial (NCT04436510). It is a first-in-class, potent, selective, brain- penetrant irreversible myeloperoxidase (MPO) enzyme inhibitor which has been Licensed from AstraZeneca in September2018.
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