Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic Lateral Sclerosis (ALS)

Also known as Lou Gehrig’s disease, ALS (amyotrophic lateral sclerosis) afflicts an estimated 30,000 Americans with approximately 5,600 additional cases being diagnosed each year. ALS is the progressive degeneration and death of the motor neurons that control the movement of muscles, leaving patients progressively weak and resulting in worsening disability and eventual death. Once diagnosed the life expectancy averages 2 to 5 years, though a very small percentage of people will live 20 years or more with the disease.  While people with ALS may have a reasonable quality of life for a year or more after diagnosis, the neurodegenerative nature of the disease is considered irreversible with current treatments and invariably results in the death of the patient. In addition, the psychological effects of the   disease are devastating, as most patients become functionally quadriplegic and bedridden in the end stages of the disease while maintaining high levels of awareness and sensory function.

Currently the only available treatments do not actually treat ALS, but instead attempt to alleviate the disruptive and debilitating side effects of the disease. These include reduced mobility, impaired speech, breathing problems, and dietary challenges. These heavily affect the quality of life of the patient as they must be addressed with often invasive or expensive devices like ventilators, feeding tubes, lifts, and digital speech aids. Treating these secondary conditions creates not only incredibly high costs of care for the disease, but also has a huge impact on the quality of life for both patients and care givers.

Economic Impact

At late stages, the disease is incredibly costly, as patients typically require intensive care and long hospitalizations. In advanced cases treatment expenses can be as high $200,000 annually, representing an aggregate direct cost to the healthcare system of more than $6 billion per year.  Total economic impact is difficult to measure because — in addition to the cost of medicine, devices, and hospital visits — most of the care involved in ALS is provided at home by family members. Many individuals quit their jobs in order to stay at home with the patient at later stages of the disease or must pay for hospice care.

Regenerative Medicine Solutions

While the traditional approach to drug development for ALS do not address the underlying causes of the disease, regenerative medicine technologies could potentially transform the current standard of care through a reversal or halt to the progression of the disease, by slowing motor neuron death. These technologies may even have the potential to replace or regenerate lost cells over time, allowing patients to regain a normal quality of life.

Regenerative medicine / advanced therapy companies active in developing treatments for ALS include:

BrainStorm Therapeutics Inc: BrainStorm's ALS program is currently testing MSC-NTF cells ALS patients after it has shown the cells can have a benefit in the human DOD1 transgenic mouse model of ALS. The company has completed two single-arm studies in ALS patients, conducted in Israel at Hadassah Medical Center, and have also completed a 48 patient randomized, double-blind, placebo-controlled phase 2 trial at three prestigious medical centers in the United States at Mayo clinic, MGH (Massachusetts General Hospital) and UMass (University of Massachusetts). More about their ALS products here