Newswise — On the Fourth of July 75 years ago, in 1939, legendary first baseman Lou Gehrig – afflicted with a nerve disorder called amyotrophic lateral sclerosis, or ALS – stepped to the microphone at Yankee Stadium and said goodbye to baseball.
Barely two months earlier, as his 13-year streak of consecutive games played reached 2,130, that moment would have been unthinkable. But the degenerative effects of ALS, now widely known as Lou Gehrig Disease, were so rapid that in less than two years Gehrig would be dead at age 37.
Since then, researchers have been unable to reverse the downhill course of the disease, but there is hope that new experimental methods may change that.
The National Institute of Neurological Disorders and Stroke estimates that 5,000 Americans are diagnosed with ALS each year, most commonly between the ages of 50 and 65. Most patients die with respiratory failure within three to five years of diagnosis, though roughly 10 percent survive a decade or longer.
Jerry Belsh, a physician and professor of neurology at Rutgers Robert Wood Johnson Medical School, is director of the Neuromuscular and ALS Center of New Jersey, one of just 34 ALS Association-certified centers in the country that care for patients with the disease and the only one in New Jersey. Rutgers Today asked Belsh to describe the effects of ALS, as well as what researchers are doing to improve the outlook for those who suffer from it.
Rutgers Today: What is ALS, and please describe the signs that a patient might have it.
Belsh: ALS is a degenerative disease that affects motor neurons, the nerves that control voluntary muscle function. The main symptoms are muscle weakness and wasting, as well as twitching in muscles. Usually, these abnormalities begin in a hand or a foot, or in muscles controlling speech. Gradually, weakness progresses to involve other limb and trunk muscles, swallowing and respiratory muscles, and eventually the patient will require a feeding tube and ventilator to maintain nutrition and breathing.
ALS is a very specific disease for motor nerves, and does not affect vision, sensation, bladder and bowel, and sexual function. Except for fewer than 10 percent of patients known to have a genetic mutation, its cause is not known. The disease may, in fact, have multiple causes.
Rutgers Today: Are there more effective treatments now than there were 75 years ago, when Lou Gehrig’s case made ALS so widely known?
Belsh: We have learned a lot about this disease, but we still don’t have a treatment that will reverse the downhill course. In fact, there is only one drug on the market that is specifically used to treat ALS. Riluzole, which we have had for nearly 20 years, inhibits the body’s production of a chemical called glutamate, which is believed to be toxic to motor neurons. But the benefit is quite modest. We’re talking about prolonging life by just three to five months, and maybe a slightly slower rate of deterioration.
However, we are able to use medications and therapies that treat many of the symptoms. For instance, certain medications can improve muscle stiffness or excessive drooling, while a walker or customized ankle splint can aid a patient’s ability to walk.
Rutgers Today: The federal government devotes an estimated $40 million per year to research to find effective treatments and a cure. Research funding also comes from private sources such as the ALS Foundation. Does the latest research include anything promising?
Belsh: Our center continues to participate with other academic medical centers across the country in carrying out treatment trials of potential cures. I am optimistic that we are on our way toward finding new treatments that are more effective. For instance, a specific gene is known to produce a faulty protein that contributes to ALS symptoms in certain patients. Human clinical trials on a medication designed to alter the gene in those patients are underway.
There is another promising trial involving stem cell infusions into parts of the spinal cord where the motor nerves originate. Ultimately, we hope that this dramatic stem cell infusion will reduce motor neuron death, produce new healthy motor neurons, and result in a reversal in the downhill course of this disease.
Rutgers Today: What can be done to improve the life of a patient with ALS – or maybe more accurately, to make it less bad?
Belsh: The team at our center is coordinated by our nurse, Mary Ann Mertz. It includes an occupational therapist and physical therapist who deal with weakness in the arms and legs, and offer practical solutions like replacing buttons on a patient’s shirt with Velcro, so getting dressed is easier for the patient to manage. Our speech therapist deals with decreasing ability to speak and swallow, and shows patients how to use modern tools like iPads to be understood when speech fails them. Our nutritionist advises patients concerning diets to help preserve patients’ nutrition and hydration. We have an extremely knowledgeable social worker who helps patient and family cope with the many psychological, social, and financial difficulties that commonly accompany ALS. All of our patients see a pulmonologist, or lung specialist, who closely follows their respiratory function.
Maintaining optimum functioning of life for as long as possible is our overriding goal.