When you hear the term “disease-modifying therapies,” you may well ask, “Don’t all therapies attempt to modify disease?” The short answer is that many do, but not all. Some treatments, for example, aim only to improve symptoms (symptomatic therapies). This is how Aaron Ritter, MD, explains the difference:

“There are four approved Alzheimer’s disease (AD) medications. All four have symptomatic benefits in that they help the brain work more efficiently, but there is little data to suggest they stop the processes causing the disease. Taking a symptomatic medication is like pouring oil into your car’s engine; it helps the car run better, but it doesn’t necessarily fix the underlying problem.”

Disease-modifying therapies, on the other hand, offer hope of preventing or halting the progression of disease.

“With recent advances in our understanding of how AD causes the brain to malfunction, we may now be able to target some of these disease processes,” says Dr. Ritter, Director of Clinical Trials for Cleveland Clinic Lou Ruvo Center for Brain Health. “This is a very exciting time because a number of promising disease-modifying therapies are being tested at our center.”

He cautions, however, that challenges persist: “Changes tend to occur slowly in neurodegenerative diseases. Showing that a medication has an impact on disease course may take years to demonstrate.”

A Brief Guide to Clinical Trials
Thanks to the selflessness of clinical trial volunteers, researchers gain valuable insights on how the brain works ­and why sometimes it doesn’t. Clinical trials have varying aims, but with the exception of observational studies, which contribute to our understanding of brain changes but do not test new medications, most trials conducted at the Lou Ruvo Center for Brain Health fall in the disease-modifying therapy category.

These studies are also termed “interventional” because new treatments (or placebos) are administered to participants. Here’s a brief rundown of only a few of the ongoing interventional trials in the center’s three major areas of focus:

Cognitive
TANGO: Asks whether an antibody against the tau protein, given as an IV infusion, can slow the decline of cognitive and functional impairment in people with mild cognitive impairment (MCI) or mild Alzheimer’s.

EMERGE: Tests whether an antibody directed against amyloid protein and given as an IV infusion can slow cognitive and functional impairment in early Alzheimer’s disease.

GRADUATE II: Tests whether an antibody directed against amyloid and given as a subcutaneous injection can slow cognitive and functional impairment in early Alzheimer’s disease.

Movement Disorders
Nilo-PD: Asks whether the anti-cancer drug nilotinib can be repurposed to slow the progression of Parkinson’s disease.

Multiple Sclerosis
ARISE for MS: Investigates whether starting treatment promptly with the drug Tecfidera®, used to treat relapsing forms of multiple sclerosis (MS), can delay or prevent onset of the disease in people whose brain scans indicate that MS may be developing.

You can view a full list of ongoing trials, many of which are currently recruiting volunteers, at clevelandclinic.org/brainhealthtrials.

The Case for Early Intervention
The quest to find therapies that will attack the causes of brain disorders argues strongly for intervention before disease becomes severe. The lessons of the past decade are clear: the earlier we can test disease-modifying therapies, the better, says Dr. Ritter: “This has led us to encourage patients and families to pursue early testing, diagnosis and treatment.”

If early intervention could delay the onset of Alzheimer’s disease by five years, he adds, we could decrease its prevalence by 50 percent – and save countless patients and families the heartbreak of a disorder for which we currently have no cure.

 “Ancient writings tell us that brain diseases have plagued people for thousands of years,” says Dr. Ritter. “Conquering them would be a global achievement with enormous political, social and economic consequences for all humanity.”

A Global Cause Needs Grassroots Support
Of course, the benefits of a breakthrough in halting the wreckage from the morbidity and mortality of neurodegenerative disease extend far beyond the family circle.

You can enlist in the fight. We have made significant strides in the first 10 years of the Lou Ruvo Center for Brain Health’s existence, but we need you more than ever to accelerate the tempo in the next decade. Together, we can make advances we only dream of today.

To find out if you, a loved one or a friend meets the qualifications for one of our clinical trials, call 855.LOU.RUVO or email healthybrains@ccf.org.

Photo: Chart – Based on Kennedy GJ. Primary Psychiatry, Vol. 14, No. 11, 2007. Provided by Cleveland Clinic Nevada