ALS Biologics

The ALScell™ (aka JadiCell™) is unique in that it possesses features of mesenchymal stem cells, however, ALScells outperform these cells in terms of a) enhanced growth factor production; b) augmented ability to secrete exosomes; and c) superior angiogenic
and neurogenic ability.

According to the National Institutes of Health (NIH), amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a neurological disorder that affects motor neurons, the nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing. As motor neurons degenerate and die, they stop sending messages to the muscles, which causes the muscles to weaken, start to twitch (fasciculations), and waste away (atrophy). Eventually, in people with ALS, the brain loses its ability to initiate and control voluntary movements such as walking, talking, chewing and other functions, as well as breathing. ALS is progressive, meaning the symptoms get worse over time.

The U.S. Food and Drug Administration has approved several drugs for ALS that may prolong survival, reduce the rate of decline, or help manage symptoms. However, there is currently no known treatment that stops or reverses the progression of ALS.

Early symptoms include:

  • Muscle twitches in the arm, leg, shoulder, or tongue
  • Muscle cramps
  • Tight and stiff muscles (spasticity)
  • Muscle weakness affecting an arm, a leg, or the neck
  • Slurred and nasal speech
  • Difficulty chewing or swallowing
  • As the disease progresses, muscle weakness and atrophy spread to other parts of your body.

People with ALS may develop problems with:

  • Chewing food and swallowing (dysphagia)
  • Drooling (sialorrhea)
  • Speaking or forming words (dysarthria)
  • Breathing (dyspnea)
  • Unintended crying, laughing, or other emotional displays (pseudobulbar symptoms)
    Constipation
  • Maintaining weight and getting enough nutrients

Eventually, people with ALS will not be able to stand or walk, get in or out of bed on their own, use their hands and arms, or breathe on their own. Because they usually remain able to reason, remember, and understand, they are aware of their progressive loss of function. This can cause anxiety and depression in the person with ALS and their loved ones. Although not as common, people with ALS also may experience problems with language or decision-making. Some also develop a form of dementia known as FTD-ALS.

Most people with ALS die from being unable to breathe on their own (known as respiratory failure,) usually within three to five years from when the symptoms first appear. However, about 10% survive for a decade or more.

Diagnosing ALS

It is important to get an accurate ALS diagnosis as soon as possible. ALS treatments may be most effective early in the course of the disease. A neurologist familiar with ALS can help a person get diagnosed early after symptom onset.

There is no single test that can definitely diagnose ALS. A healthcare provider will conduct a physical exam and review the person’s full medical history. A neurologic examination will test reflexes, muscle strength, and other responses. These tests should be performed at regular intervals to assess whether symptoms are getting worse over time.

A healthcare provider may conduct muscle and imaging tests to rule out other diseases. This can help support an ALS diagnosis. These tests include:

Electromyography (EMG)—evaluates how well nerves and muscles are functioning. This test can include:

  • A nerve conduction study (NCS) — measures the electrical activity of nerves and muscles by assessing the nerve’s ability to send a signal along the nerve or to the muscle.
  • A needle exam — a recording technique that detects electrical activity in muscle fibers using a needle electrode.
  • Magnetic resonance imaging (MRI) — uses a magnetic field and radio waves to produce detailed images of the brain and spinal cord.

Treating ALS
There is no treatment to reverse damage to motor neurons or cure ALS at this time. However, some treatments may slow progression of the disease, improve quality of life, and extend survival. New treatments have become available in the past several years, and researchers continue to explore diverse avenues to slow or stop progression of ALS.

Supportive health care is best provided by integrated, multi-disciplinary teams of professionals that may include physicians, pharmacists, physical, occupational, speech, and respiratory therapists, nutritionists, social workers, clinical psychologists, and home care and hospice nurses. These teams can design an individualized treatment plan and provide special equipment aimed at keeping people as mobile, comfortable, and independent as possible.

A doctor may prescribe other medications or treatments to help manage symptoms, including muscle cramps and stiffness, excessive saliva and phlegm, and unwanted episodes of crying and/or laughing, or other emotional displays. Medications may also help with any pain, depression, sleep disturbances, or constipation.

Doctors may use the following medications approved by the U.S. Food and Drug Administration (FDA) to support a treatment plan for ALS:

 

  • Riluzole (Rilutek) is an oral medication believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. Clinical trials in people with ALS showed that riluzole may prolong survival by a few months. The thickened liquid form (Tiglutik) or the tablet (Exservan) that dissolves on the tongue may be preferred if the person has swallowing difficulties.
  • Edaravone (Radicava) is an antioxidant given either orally or intravenously and has been shown to slow functional decline in some people with ALS. RADICAVA ORS is a form of edaravone that can be taken orally or via feeding tube.
  • Sodium phenylbutyrate/taurursodiol (Relyvrio) is an oral medication that is believed to prevent nerve cell death by blocking stress signals in cells. It has been shown to slow functional decline and extend survival in some individuals with ALS.
  • Tofersen (Qalsody) is given through a spinal injection to ALS patients who have been determined to have a mutation in the SOD1 gene. While the benefits of this drug are still under study, it may work by decreasing one of the markers of damage to neurons.