ALS and Regenerative Medicine

ALS and Regenerative Medicine

ALS Overview

Better 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. BrainStorm Therapeutics Inc is conducting a Phase IIa trial in Israel for the use of NurOwn, a treatment that uses autologous mesenchymal stem cells present in the patient's own bone marrow to generate NTF (neurotrophic factor)-secreting cells for preservation of neurons and stimulation of their growth. Ultimately their goal is to re-establish nerve-muscle interaction and to slow down or halt progression of the disease.  Another company, Neuralstem Inc. - working in conjunction with Emory University - recently completed a Phase I safety trial of its NSI-566 spinal cord neural stem cells for another treatment of ALS (Neuralstem Inc.). These cells are expected to graft into the region they are transplanted, synapse and form circuits with the patients’ motor neurons, and ultimately halt the further progression of the disease. Neuralstem is planning to commence an ALS Phase I/II trial for its cells later in 2012.  Although these innovative therapies are still years away from becoming commercially available, they could potentially greatly improve the quality of life for ALS sufferers and save families and the healthcare system billions of dollars over time.