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  1. 1. Alliance for Regenerative Medicine - Disease SpotlightDisease Facts:Description: Amyotrophic Lateral Sclerosis (ALS)+ Causes nerve cell deaththat leads to disability, + A Devastating, Debilitating Disease With No Known Cureparalysis, and eventualdeath 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 underlyingPatients: 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 technologiesnew patients diagnoses with approximately 5,600 the healthcare system of more could potentially transform theeach year additional cases being than $6 billion per year. Total current standard of care byCause: 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 evenProgression: 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 aCurrent 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 diagnosedcosts total over $6 billion home with the patient at later company, is conducting a Phase the life expectancy averages 2per 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 usesRM 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 mayactive 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 afteror completed using stem to alleviate the disruptive and diagnosis, the neurodegenera- trophic factors) producing cellscells for treatment or to debilitating side effects of the tive nature of the disease is that stall the degeneration ofcreate disease models to disease. These include reduced considered irreversible with motor neurons. Anotherscreen 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. Theseinvestigating the use of patient. In addition, the University to conduct a Phase I heavily affect the quality of lifemesenchymal (bone psychological effects of the clinical trial of fetal-derived of the patient as they must bemarrow) and fetal cells to disease are devastating, as most stem cells for another addressed with often invasiveslow 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 morepatient 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 oforganizations 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 representagencies 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 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.