ALS: New Treatment Extends Lives


BOSTON, Mass. (Ivanhoe Newswire) – Every year in the United States, about 5,000 people are diagnosed with ALS, also known as Lou Gehrig’s disease. There is no cure, and patients often die within two years of receiving a diagnosis. A social media movement several years ago brought it to the forefront while  raising millions of dollars for research. Now, one of the drugs developed as a result of that funding has just been FDA approved for use.

In 2014, the “Ice Bucket Challenge” was all over social media. People challenged their friends to dump ice water over their heads and then make a donation to the ALS Association. A portion of the $115 million raised went to research and contributed to the development of a new drug.

Dr. Sabrina Paganoni, MD, PhD, at Massachusetts General Hospital, was the lead investigator on the clinical trial that led to approval of this new therapy.

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“The new drug is called AMX 35, and it’s a combination of two different drugs.” Dr. Paganoni explains.

Now marketed as Relyvrio, the drug comes in a powder form that patients can drink or take by feeding tube. It works by slowing the  loss of spinal and brain nerve cells.

Dr. Paganoni adds, “Things that we do every day that we take for granted, like talking or swallowing or walking or climbing stairs, people who took the drug did better on those functions for longer.”

Patients taking the drug also lived, on average, five months longer. Giving some valuable time.

“People want to see their kids graduate from school or they want to see their first child get married. So, really five or six months could be the difference between making it to that event or not,” Dr. Paganoni emphasizes.

One drawback that has been a major concern for patient advocates is the cost of the medication – around $158,000 a year. The company that developed the drug says the cost to patients will be lower because they anticipate insurance will cover it. Relyvrio is the third FDA-approved drug for ALS and its symptoms.

Contributors to this news report include: Cyndy McGrath, Producer; Kirk Manson, Videographer; Roque Correa, Editor.

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REPORT:       MB #5151 

BACKGROUND: Amyotrophic Lateral Sclerosis  is a neurological disease that impacts nerve cells in the brain and spine. ALS is also commonly known as Lou Gehrig’s disease. The disease consists of the muscles receiving no nourishment and as result, wasting away. As the disease progresses it leads to hardening and scarring in the spinal chord region. The desintigration of of motor neurons that ALS causes eventually leads to their quietus. It affects nerve cells working in both the upper and lower parts of the body. Ninety percent of cases do not tie a family history or genetic mutation of the illness. There is however a 50 percent chance orf offspring contracting the genetic mutation from family members.


DIAGNOSING: The symptoms tend to be moderate. They can range from trouble gripping a water bottle to changes in vocal frequency when holding conversation. The progression of symptoms varies depending on the patient as well. Patients are often given an estimated survival rate of two to five years ago upon diagnosis. People have be known to live ten years or longer as well. Symptoms typically begin in muscles that control speaking and swallowing, in the hands, arms, legs, and feet. Not all symptoms are the same, but globally muscle weakening and paralysis are inevitable. Sight, touch, hearing, smell, and taste are not affected by ALS because the disease only attacks motor neuorns.


NEW TECHNOLOGY: Relyvrio is a new oral drug prescription therapy for adults. For those living with Amyotrophic Lateral Sclerosis, the drug is designed to help slow the disease progression. The prescription drug comes in a powder that can be injested with a feeding tube. It works by slowing down the pace of death of spinal and bvrain nerve cells. Common side effects of the drug are changes in bile and acid levels and sodium retention. Even more common side effects are diarrhea, stomach pains, naseuea, and upper respiratory tract infections.



Katie Marquedant

If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at

Doctor Q and A

Read the entire Doctor Q&A for Dr. Sabrina Paganoni, MD, PhD

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