1) ALS is a progressive neurodegenerative disease that causes motor neuron death leading to muscle paralysis and death, usually within 2-5 years of diagnosis. 2) Current treatments only manage symptoms and do not stop the underlying progression of the disease. Regenerative medicine approaches aim to regenerate lost motor neurons to halt progression. 3) Several regenerative medicine companies are investigating stem cell therapies for ALS, with the goal of slowing motor neuron death and preserving function. These innovative approaches could potentially improve quality of life and reduce healthcare costs compared to current symptom management only treatments.

1. Alliance for Regenerative Medicine - Disease Spotlight
Disease Facts:
Description: Amyotrophic Lateral Sclerosis (ALS)
+ Causes nerve cell death
that leads to disability, + A Devastating, Debilitating Disease With No Known Cure
paralysis, and eventual
death Better known as Lou Gehrig’s advanced cases treatment to drug development for ALS do
disease, ALS (amyotrophic expenses can be as high not address the underlying
Patients:
lateral sclerosis) afflicts an $200,000 annually, representing causes of the disease, regenera-
+ 30,000 in the US; 5,000
estimated 30,000 Americans an aggregate direct cost to tive medicine technologies
new patients diagnoses
with approximately 5,600 the healthcare system of more could potentially transform the
each year
additional cases being than $6 billion per year. Total current standard of care by
Cause: diagnosed each year. ALS economic impact is difficult to halting or reversing progression
+ Unknown causes the motor neurons that measure because — in addition by slowing motor neuron death.
control the movement of to the cost of medicine, devices, These technologies may even
Progression:
muscles to begin to deteriorate and hospital visits — most of have the potential to replace or
+ Life expectancy is 2-5
and die, leaving patients the care involved in ALS is regenerate lost cells over time,
years after diagnosis
progressively weaker resulting provided at home by family allowing patients to maintain a
Current Cost of Care: in worsening disability and members. Many individuals quit normal quality of life. Brain-
+ Direct and indirect their jobs in order to stay at Storm Therapeutics Inc., a RM
eventual death. Once diagnosed
costs total over $6 billion home with the patient at later company, is conducting a Phase
the life expectancy averages 2
per year stages of the disease or must I/II trial in Israel for the use of
to 5 years, though a very small
percentage of people will live 20 pay for hospice care. NurOwn, a treatment that uses
RM and ALS: autologous mesenchymal stem
years or more with the disease.
+ 4 RM companies with Currently the only available cells present in the patient’s
While people with ALS may
active ALS programs treatments do not actually own bone marrow to create
have a reasonable quality of life
+ 6 clinical trials ongoing treat ALS, but instead attempt dopamine and NTF (neuro-
for a year or more after
or completed using stem to alleviate the disruptive and
diagnosis, the neurodegenera- trophic factors) producing cells
cells for treatment or to debilitating side effects of the
tive nature of the disease is that stall the degeneration of
create disease models to disease. These include reduced
considered irreversible with motor neurons. Another
screen for new drugs mobility, impaired speech,
current treatments and invaria- company, Neuralstem Inc., is
breathing problems, and
+ Current programs are bly results in the death of the currently working with Emory
dietary challenges. These
investigating the use of patient. In addition, the University to conduct a Phase I
heavily affect the quality of life
mesenchymal (bone psychological effects of the clinical trial of fetal-derived
of the patient as they must be
marrow) and fetal cells to disease are devastating, as most stem cells for another
addressed with often invasive
slow disease progression patients are quadriplegic in the treatment of ALS. Although
or expensive devices like
end stages of the disease, but these innovative therapies are
ventilators, feeding tubes, lifts,
still maintain high levels still years away from becoming
and digital speech aids.
awareness and their basic commercially available, they
Treating these secondary
senses as their body functions could potentially greatly
The Alliance for conditions creates not only
progressively shut down. improve the quality of life for
Regenerative Medicine incredibly high costs of care for
ALS sufferers and save families
(ARM) was formed to At late stages, the disease is the disease but also has a huge
and the healthcare system
advance regenerative incredibly costly, as patients impact on the quality of life for
billions of dollars over time.
medicine by representing typically require intensive care both patients and care givers.
and supporting the and long hospitalizations. In While the traditional approach
community of
companies, academic + Regenerative Medicine—In Brief
research institutions,
What is regenerative medicine? tissue and renew biologic products addressing many more
patient advocacy groups,
Regenerative medicine (RM) is a function in the body is what conditions advancing in clinical
foundations, and other rapidly evolving interdisciplinary distinguishes RM from other trials. RM holds the promise of
organizations before the field in health care that trans- types of treatments and brings treating a broad range of dis-
Congress, federal lates fundamental knowledge in new hope for dramatically eases that collectively represent
agencies and the general biology, chemistry and physics improving clinical outcomes and a substantial burden to our
public. into materials, devices, systems curing disease. healthcare system, including
and therapeutic strategies, What diseases can RM treat? acute and chronic conditions
For more information, including cell-based therapies, such as diabetes, congestive
RM products are currently on
please visit us online at: which augment, repair, replace the market for the treatment of heart failure, Parkinson’s
www.alliancerm.org or regenerate organs and wounds, cartilage defects, and disease, stroke, renal disease,
tissues. The ability to repair diabetic foot ulcers, with spinal cord injury, and ALS.