ALS Amyotrophic Lateral Sclerosis Clinical Trials Innovation Platform Trial Research

First Platform Trial in ALS Brings Hope for Patients

There is no known treatment or cure for the well-known disease, Lou Gehrig’s disease. The disease, commonly known as ALS (Amyotrophic Lateral Sclerosis) causes the death of neurons controlling voluntary muscles.

There is no known treatment or cure for the well-known disease, Lou Gehrig’s disease. The disease, commonly known as ALS (Amyotrophic Lateral Sclerosis) causes the death of neurons controlling voluntary muscles. The disease can affect a person of any age. Most of the ALS patients die from the disease, and about 10% survive for more than ten years. As deadly as this disease sounds, there is still no cure.

Background:

Sandy Morris, a fifty-three-year-old mother, was diagnosed with ALS. She thought that if other fatal diseases have found a cure by the help of gene therapy, there has to be some cure for ALS as well. The optimistic woman claims that there is a ray of hope for ALS patients. 

Several trails have been conducted to accelerate the research pace of ALS. Five drug makers are working on such drugs. These drugs will be tested in a platform trail. The research done will also turn out to be helpful for other rare diseases as well. 

Advancement of research:

Merit Cudcodkwicz has twenty-five years specializing in ALS along with Sean Healey, launched a research institute in Massachusetts General Hospital. Healey was diagnosed with ALS in May 2018, and it was after this that she decided to change her asset management firm into a research center to carry out work in the hope of finding a cure for ALS. The Healey center recently announced five drugs that are almost ready to be tested in a clinical trail.

About 160 patients will be needed for testing of each of these drugs. The drugs results will be seen within a year or so. It is expected that these drugs will bring the treatment one step closer to the actual cause; finding a cure for ALS. The research work will also be helpful for further improvements in the field and also for finding cures for other similar and rare diseases.

The selection of five Bio-Techs by Healey center was an easy task. Cudkcowiz selected these five applicants from a pool of thirty. Most of the applicants were small companies with low funding, although they had great ideas. The main problems faced by the companies was identifying and distinguishing the effects of drugs. The experiments were complex, making it difficult to predict the results.

Summary:

Although this is just the start in the direction to find a cure for ALS, it has motivated many patients suffering from the disease. The research work that is being done is extraordinary and is being conducted at a very fast pace. Even if the research doesn’t prove to be of help in ALS, it can surely be fruitful for other similar diseases. As a matter of fact, trails have been started in breast cancer, Alzheimer’s disease, and glioblastoma. The researchers are also focusing on carrying out further work in these rare diseases. The optimistic patient, Morris, who is also a leader of the patient group “I Am ALS” is determined to help the researcher in finding a treatment and has helped in several tests. She dreams of finding a cure for the next generation patients of ALS.

Source:

https://www.massgeneral.org/about/pressrelease.aspx?id=2454

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