a) Amyotrophic lateral sclerosis (ALS) is a serious debilitating disease with poor life expectancy of typically only 3-4 years from onset. Patients with ALS become rapidly and severely disabled losing the strength in their arms and legs and eventually lose the ability to speak. As these patients are clearly highly vulnerable, there are a number of study design and treatment aspects that a HREC would be concerned about. These are discussed below: • Intervention arms: The trial here wishes to evaluate a new drug for ALS compared to placebo. As there is a drug available to treat ALS (Riluzole)(1), the decision to use placebo over available treatment would need to be clearly justified and evaluated in detail. • That there is sufficient justification that the drug will be as least as effective as current methods of care. • That if the treatment is to be highly effective, would the trial have stopping rules to enable the placebo group to also be treated. • Access to additional drug use/physiotherapy for ALS symptom management: There are several additional medications that can help with the symptoms (e.g. spasticity, pain) of ALS during the course of the treatment (2). Physiotherapy in ALS patients has also shown to help alleviate fatigue and pain and may help to delay muscle weakening (3). Would patients (in both …show more content…
CTNs are used to notify the TGA that a clinical phase I trial is to be undertaken and can only be submitted once a local ethics committee has evaluated all the relevant scientific data related to the drug and its use in treating ALS (e.g. in laboratory experiments and animal studies). More than 98% of Phase I trials in Australia for new drugs use a CTN. A CTX is only used when the local ethics committee is not willing to review the scientific data and the data is then submitted to the TGA. CTXs are avoided where possible as they are more expensive and have longer processing
Amyotrophic Lateral Sclerosis (ALS) is a terminal disease, also known as Motor Neurons Disease, Charchot Disease and Lou Gehrig disease. ALS destroys the Central Nervous System (CNS) and causes damage to the upper and lower motor neurons in the brain. Signs and symptoms are characterized as: muscles weakness, muscle atrophy, twitching and reduced muscle reflexes. Eventually the patient will become paralyzed and rely on a tracheostomy and ventilator for breathing (ALS Association [ALSA], 2010).
There are 20,000 new cases of ALS diagnosed each year in the United States. This yields an incidence of 3 per 100,000 (Brown, 2006). There is no known cause for ALS in 95% of patients; however, 5% have an identifiable genetic mutation (Elman, 2016). The disease can present in individuals less than 30 years of age, but peaks between 40 and 60 years of age. Before the age of 65, more diagnoses are made in men; after the age of 65, gender incidence is equal. There is no clear-cut ethnic or racial predisposition in ALS (Ricks, 2016). The lifespan is approximately 3-4 years after diagnosis. However, in 10 % of
Abramovitz, Melissa. "Lou Gehrig's Disease" p17-61. Treatment for ALS or Lou Gehrig's disease has found that Vitamin E and the drug known as Riluzole can help the progression of the disease. When taking the medication Riluzole it has shown to prolong survival in patients by two to three months and it has also delayed the use for breathing support. There is a few side effects to taking Riluzole, such as headache, dizziness, fatigue, and it has also shown that it can do damage to your liver and kidney's. There is no cure for ALS, but researchers are trying to find out what actually causes this disease and how they can cure it.
Imagine you are a 78-year-old man. A beloved professor from Brandeis University. Day by day, take notice of the increased difficulty of breathing, more so than the usual out-of-breath feeling from climbing a flight of stairs. About two or three weeks later, you start to feel muscles in your body cramping up, even while partaking in slight activity. After various testing, your results come back you have ALS, short for Amyotrophic lateral sclerosis.
A recent clinical trial recruited participants early in their ALS progression, with milder symptoms and a larger vital capacity. According to Mitsubishi
Following the diagnosis of ALS the main goal of treatment is focused at relieving the symptoms. The most important matter in managing ALS patients is that they continually sustain the highest amount of movement and activity as possible and remain comfortable while doing so. Although a cure for ALS has not yet been developed, effective treatments do exist to assist the patients in their fight with the disease. Physicians urge the patients to carry on with their everyday lives doing all the activities and physically exerting themselves as they would normally do. But, they also recommend the patients to limit their amount of physical exertion
People first need to know what ALS is. ALS is very difficult to diagnose. So far there is no
Typically, there is a small number of people used in these Phase I trials, between 20 and 80. Phase II trials have more participants(100-300) who have the condition or disease that the product may be able to treat. Researchers want to gather further safety data and preliminary evidence of the drug’s beneficial effects, and they develop and refine research methods for future trials with this drug. If the drug is indicated to possibly be effective during Phase II, given the observed severity of the disease, the drug will progress to Phase III. In Phase III, the drug is studied in a larger number of people with the disease, between 1,000-3,000 usually. The phase further tests the product’s effectiveness, monitors side effects and, in can compare the product’s effects to a standard treatment, if one is available already. Having more participants reveals the less common side effects. Phase II and Phase III clinical trials typically involve a “control” standard. One group is given the drug and the control group is given either a standard treatment for the illness or a placebo. Phase IV is the part of the trial that is sometimes conducted after a product is already approved and on the market. The purpose is to find out more about the treatment’s long-term risks, optimal use, and benefits, or to test the product in different demographics, such as children. Informed consent is the process by which potential participants for a study are given complete information about the study. The informed consent process provides an opportunity for the researcher and patient to exchange information and ask questions. Patients are invited to enter a trial but are not forced to do so. They can consent to participate if they find the potential risks and benefits acceptable. A participant must sign a consent form prior to enrolling in a study before
I. Take a moment, and point at the computer screen. Notice that your index finger is extended and your other fingers are curled into your palm. How did your hand know to do that? It all began in your brain, the starting point for an important chain of communication. Your brain ordered nerve cells, to activate
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that causes devastating debilitation and raises many ethical questions. Many of these questions revolve around quality of life and end of life issues. ALS, specifically, complicates theses issues because of the natural progression of the disease. Patients gradually loose their ability to move, breath and communicate, while retaining, for the most part, their cognitive function. There is currently no cure for the disease and very little in the forms of treatment options, with the average life span after an ALS diagnosis only about three to five years with a small portion of the population living beyond 10 years. As a result, caring an ASL patient can put a lot of stress on their caregivers. Consequently, there is also the risk to the patients, who wishes can be ignored due to their inability to advocate for themselves.
The manifestations of ALS are caused by the location of motor neuron death. When upper motor neurons die, the symptoms include problems controlling fine movements, spasms, dysphagia, dysphonia and dysarthria (Porth & Matfin, 2009). “Manifestations of lower motor neuron destruction include fasciculations, weakness, muscle atrophy, and hyporeflexia” (Porth & Matfin, 2009, p. 1284). Patients with early signs of ALS usually complain of feeling weak on one side, which is due to the slowing of electrical impulses to that group of muscles (Ignatavicius & Workman, 2010). Since the impulses are slowed, they are not receiving adequate electrical stimulation to move and the person feels weak. As the disease progresses, all the motor neurons die and are not regenerated so, the patient is left paralyzed, losing the ability to speak, swallow and breathe (Ignatavicius & Workman, 2010).
I started being able to pick up objects that weighed more than seven pounds. My muscles were not as strong as they were before I got diagnosed, but I believed that I could regain all the frail muscle I had into strong, beautiful muscles. Tomorrow would be the second day of school for me. I took off almost three months of school and all my sports were postponed until I started getting better. “Bri, you are making a spectacular recovery, this is the first case this whole hospital has seen where only one dose of Riluzole has almost cured you from ALS!” he exclaimed to me. He told me to take it slow, only pick up items less than five pounds and be careful with cuts or sores getting infected. I went home that day with a smile on my face and a hop in my step. It was satisfied to know that I was the first person to have this type of recovery. Even though I still have the diagnosis of ALS, I go back to doing my normal things. Now I play for my school soccer team and this past season I got over $2,000 in donations for me and my condition, I donated all the donations I received to the ALS Association for people that are going through this disease that can’t afford the medication
Introduction: A long-term degenerative neurological condition (LTDNC) is a term used to describe diseases that affect the nervous system leading to its degeneration. Conditions such as Parkinson’s disease, amyotrophic lateral sclerosis, multiple sclerosis etc. are all categorized by impairment in brain, nerve and spinal cord pathway cells (Canadian Institute for Health Information, 2007). Due to such disruptions, the nerve signals between the brain and the body are affected which result in problems with walking, controlling movement, balance, full or partial paralysis, breathing and talking problems, occurrence of seizures, lack of bladder and bowel control at later stages, and even problems with the heart (Canadian Institute for Health Information, 2007). In 2011, the Canadian Institute for Health Information reported that Canada has one of the highest incidences of multiple sclerosis (MS) in the world, where approximately 93,500 individuals are currently affected. Despite the prevalence of the disease, there is still much that remains unknown. According to the 2012 statistics from the ALS Society of Canada, the number of new diagnoses per year is
ALS was commonly known for the ice bucket challenge, as thousands of people across the world prompted efforts to find a cure for this fatal disease. However, the harsh fact is that there is no cure, which is why Euthanasia should be considered a viable choice of treatment for ALS patients. According to the National ALS Association “Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord.” The motor neurons progressively degenerate and die, as communication is inhibited between the brain, spinal cord, and muscles in the body. Gradually muscles lose control over movement. Consequently, treatment options are only available to reduce symptoms of the disease. For muscle weakness, one can do physical therapy.
Have you ever heard of ALS, better known as Lou Gehrig’s disease? For many people, ALS is a disorder that they may not know much about. I never heard of it either until my father was diagnosed with this disease in 2006. Because there is no known cure, it is important to detect this disease early, so that proper treatments and preparation can be done before it’s too late.