30,000 Americans have ALS or amyotrophic lateral sclerosis, also known as Lou Gehrig's disease. It's a fatal neurodegenerative disease that takes away the ability to walk, talk and eventually even breathe. That quick decline happens within two to five years. There is no known cure. Now, a revolutionary trial is underway to test a new kind of treatment for ALS using human stem cells.

55 year-old Tom Elliott is not a quitter. He has ALS. He fights to keep up with the daily routines of his life even as the disease makes everything harder.

"Brushing the teeth has become a real chore. Turning and rolling in bed to get comfortable has become an impossibility. This disease is about having to give up and sacrifice a lot,” said Elliott.

As ALS progresses, it destroys the nerve cells in the brain and spinal cord that control muscle movement, until "they cease to be able to move, they become essentially locked in their bodies,” explained Emory University’s Dr. Nicholas Boulis.

Dr. Boulis and his team at Emory University helped develop an experimental approach to treating ALS implanting stem cells called human neuro-progenitors directly into the spinal cord.

"We want to put those cells right next to those dying motor neurons in the hopes that those cells will provide protection and restoration of function, keep those cells alive, make 'em stronger,” said Dr. Boulis.

It's the first ever US clinical trial of its kind.

"I'm optimistic that we can do this safely. I'm optimistic that we'll have opened the door to a world of opportunities,” said Dr. Boulis.

Tom is one of the first to have stem cells injected into his spinal cord, a procedure with high risk and no promises. Doctors say the stem cells won't generate new neurons but may help protect the still functioning motor neurons and slow the progression of the disease. (:14)

"Maybe in the near or far future we'll be able to manage the disease better, and then perhaps one day cure it,” wished Elliott.

If the stem cell transplant technique works for ALS, researchers say it could open the door for new therapies for spinal cord injuries, multiple sclerosis and other disabling illnesses. Human stem cells for this ALS study were developed by a Maryland-based biotech company.

RESEARCH SUMMARY
TOPIC: STEM CELLS FOR ALS: MEDICINE'S NEXT BIG THING?

BACKGROUND: Named after the esteemed baseball player who died of causes unknown in 1941, Lou Gehrig's Disease, or Amyotrophic lateral sclerosis (ALS) is a degenerative neurological disease that weakens muscles, causes physical disability and death. ALS is a relatively rare disease, affecting only one to three people out of 100,000 worldwide. It is seen more commonly in men than in women, often starting in middle age or above (usually between age 40 and 60), although the disease can technically occur at any age. 5%-10% of ALS victims have a genetically inherited variation of the disease, but in most cases doctors are unable to determine its cause. The symptoms result from the gradual death of nerve cells located in the brain and spinal cord. (SOURCE: Mayo Clinic and WebMD.com)

SYMPTOMS: Early symptoms include uncontrollable muscle convulsions, limb weakness, and the slurring of speech. The disease's progression usually begins with a loss of functionality in a specific area, such as one leg, foot, hand, or the tongue. These problems will slowly start to spread to every limb as motor neurons die and are therefore unable to transmit signals to the muscles. One may experience difficulty with "footdrop," or control of operating the front area of the foot. Diminished hand strength, muscle cramping, and general loss of motor coordination also occur, and eventually lead to complete muscle paralysis. Victims in advanced stages often experience oral and respiratory complications, such as difficulty speaking, swallowing, chewing and breathing. (SOURCE: Mayo Clinic and WebMD.com)

TREATMENT: Serious complications such as breathing and eating difficulty are treated with the help of medical technologies and procedures. For patients with breathing problems, a breathing device similar to the one used to treat individuals with sleep apnea is worn at night. Surgically, a tracheostomy and applied respirator can be used to mechanically stimulate the lungs. For patients with eating difficulties, a feeding tube helps lower the risks of food aspiration and lung fluidity, which can lead to pneumonia. There is only one FDA-approved medication used to slow the disease, and it is called riluzole (Rilutek). Riluzole has not been proven to be effective in all users, but is believed to help by lowering the levels of a chemical messenger in the brain called glutamate. Other medications are prescribed to lessen the severity of symptoms such as muscle cramps, constipation, excessive salivation and fatigue. A few therapies exist to help ALS victims cope with the disease. Physical therapists use low-impact muscle strength and range of motion exercises to help the patient to strengthen their movement. Occupational therapists can help patients to effectively use a wheelchair, brace or walker to transport themselves easier. Speech therapists can help patients to employ techniques and technologies, such as speech synthesizers and communication computers, to assist with verbal communication. (SOURCE: Mayo Clinic)

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