Despite the viral success of the Ice Bucket Challenge, the cure for Amyotrophic Lateral Sclerosis (ALS) may still be far away.
In the meantime, a therapeutic regimen recently tested at USF on lab mice genetically modified to display symptoms of the disease, could stall its progression.
Dominic D’Agostino, an assistant professor of molecular pharmacology and physiology at USF Health, said the results were promising.
“We put together a project that is a combination therapy that utilizes things readily available to patients,” he said.
USF researchers and students are currently studying the effectiveness of “the Deanna Protocol,” a new regimen of nutritional supplements combined with moderate exercise to combat muscle atrophy and motor neuron degradation caused by ALS.
“What’s unique about the Deanna Protocol is that it hits multiple mechanisms working synergistically together to prevent the cells of the body from dying,” D’Agostino said.
ALS, also known as Lou Gehrig’s Disease, for the famous 1940s baseball player who developed it, attacks the nerve cells responsible for muscle control. As the motor neurons degenerate, patients progressively lose control of their body until the lungs fatally give into paralysis.
“ALS will impair your ability to speak, to swallow and to breathe,” D’Agostino said. “It’s a terminal illness. When someone gets ALS, they’re going to die from it — they know that.”
The life expectancy for the average patient is two to five years, according to the ALS Association.
“Steven Hawking has ALS and has lived with it for quite some time,” D’Agostino said. “He’s lived with it for decades, but that’s extremely rare.”
The disease can develop at anytime, without warning or reason, to people of all ethnic and socioeconomic statuses. According to the Florida Hospital, it affects up to 30,000 people in the U.S.
Though ALS is the world’s most common neuromuscular disorder, not much is known about its cause or treatment, and while the Deanna Protocol is no cure, it could slow the progression by supplying energy to decaying motor neurons.
Dr. Vincent Tedone, a retired Tampa orthopedic surgeon, created the regimen after his daughter was diagnosed with the disease. With little available treatment, Tedone began experimenting with alternatives.
Through trial and error, Tedone found Alpha-Ketoglutarate (AKG) was an ingredient that played a vital role in slowing her deterioration. Commonly marketed as a bodybuilding supplement in health stores such as GNC, the compound aids in protein synthesis.
“When the father ran out of AKG supplements to give his daughter, she froze and couldn’t move. She became ataxic with tremors,” he said. “When they got her another source of AKG, her symptoms subsided. The attack on her speech and her motor function went away.”
All other elements of the regimen are commercially available, such as zinc, magnesium and vitamin B complex, and are meant to aid the cells in breaking down energy for use.
Another source of ALS research on campus is the USF ALS Clinic, a large clinic founded in 2010 that treats 150 to 200 patients. It employs physical therapists, speech pathologists, dieticians, psychiatrists and respiratory therapists to help patients manage ALS.
Because of its unorthodox approach to testing, Dr. Lara Katzin, an assistant professor of neuromuscular medicine and electromyography, said the USF ALS Clinic could not officially support the Deanna Protocol.
“It’s just not done in the way that regular clinical trials are done,” she said. “It’s a whole list of different compounds being taken without individual ones being studied.”
The clinic also conducts clinic trials that patients may enroll in, such as research into bone marrow-derived stem cell treatment and the effect of strength training on respiratory function.
Katzin cautioned that patients must understand the Deanna Protocol is not widely-tested enough to be approved with confidence by the medical community, and it is still not at the point to justify a clinical level.
“Patients can be vulnerable to anything put online that says it can make them better,” she said.
D’Agostino said it is too difficult to get FDA approval for supplemental regimens because of the ethics of a human trial where one group is given placebo.
“There’s a sense of (patients) being completely abandoned by the scientific community and the medical community because they’re given no options,” he said.
Nonetheless, he said funding from the recent Ice Bucket Challenge would help continue hope of a better future for ALS patients.
“Anything that brings out more awareness to ALS is a good thing, no matter how you do it,” he said.
According to the ALS Association, over three million donors raised more than $100 million this summer due to the Ice Bucket Challenge, compared to last summer’s $2.8 million.
The USF ALS Clinic recently took the Ice Bucket Challenge in a string of challenges across USF Health. Katzin said the clinic was proud to the support awareness for the patients it treats.
“These are some of the most amazing patients,” she said. “They are wonderful, wonderful patients that have a wonderful spirit.”