Ketamine Earns Orphan Drug Status for ALS


In 1970, ketamine was used to treat injured soldiers on the tail end of the Vietnam War. The treatment helps relieve pain, but doesn’t affect cardiac or respiratory function. Now, in 2021, ketamine is sometimes used to treat depression or suicidal ideation in humans, used as an anesthetic for some animals, and even used recreationally (although not legally). According to ALS News Today, however, ketamine may now have a new use: a potential treatment option for patients with amyotrophic lateral sclerosis (ALS). In fact, an investigational treatment developed by PharmaTher recently received Orphan Drug designation from the FDA.


According to Medical News Today, ketamine is a class III scheduled drug that:

belongs to a class of drugs known as dissociative anesthetics. These types of drugs can make a person feel detached from sensations and surroundings, as if they are floating outside their body.

Normally, in medical settings, ketamine is given either intravenously or as an injectable. Ketamine “trips” can cause feelings of euphoria, relaxation, and happiness. However, it may also cause feelings of nausea or confusion.

Recently, ketamine received Orphan Drug designation from the FDA for the treatment of ALS. Orphan Drug designation is granted to expedite the development and review of drugs and biologics intended to treat rare or chronic conditions (impacting under 200,000 Americans). In patients with ALS, there are heightened glutamate levels in the brain and spinal cord. An excess of this neurotransmitter causes a toxic and harmful over-activation of nerve cells. Ketamine blocks NMDA receptors, which play a role in this process, and prevent an excess of glutamate. At the same time, it raises dopamine levels (as low dopamine levels are linked to ALS). Altogether, researchers believe that ketamine could act as a neuroprotective element and prevent ALS-associated damage.

Additionally, preclinical research from a team at the University of Kansas highlighted how ketamine improved survival rate and muscle function in animal models of ALS. Although this research is not yet published, it did help the FDA decide to grant Orphan Drug status. As a result of receiving this designation, PharmaTher earns a variety of benefits, from fee waivers and tax credits to increased regulatory assistance, as well as 7 years of market exclusivity (upon approval).

Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS) is a rare and progressive neurodegenerative disease which damages nerve cells in the brain and spinal cord. While doctors are not exactly sure what causes ALS, some posit that environmental toxin exposure could play a role. Additionally, there are a small portion of patients (5-10%) with familial ALS who inherit a specific genetic mutation. For the sporadic form (90-95%), risk factors include smoking cigarettes, serving in the military, being male, or age (40s to mid-60s).

In ALS, nerve cells in the brain and spinal cord die, causing muscle weakness and a loss of voluntary movement and control. Unfortunately, ALS is often fatal due to chest muscle wasting and an inability to breathe. Symptoms include:

  • Frequent tripping and falling
  • Muscle spasticity
  • Psychological distress
  • Changes in speech, such as slurring or slowed speech
  • Difficulty chewing, swallowing, speaking, performing small movements, and walking
  • Muscle twitching, particularly in the arms, tongue, legs, or shoulders
  • Difficulty maintaining good posture
  • Muscle weakness
  • Unintended but rapid weight loss
  • Inappropriate crying, laughing, or yawning
  • Inability to move muscles which gradually affects the entire body
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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