You’ve heard of the Ice Bucket Challenge, which raised more than $100 million for Amyotrophic Lateral Sclerosis (ALS) research in 2014. But what is happening with efforts to find better treatments or a cure for ALS? Massachusetts General Hospital’s ALS Multidisciplinary Clinic offers a comprehensive clinical care approach shown to extend and improve quality of life for people with ALS, also known as Lou Gehrig’s Disease. MGH’s ALS Clinical Research Program is leading the way in discovering important therapies that may one day halt its progression altogether.
After the Ice Bucket Challenge
Mass General runs one of the largest multidisciplinary clinics in the country, with a team that includes physicians and experts in physical therapy, respiratory therapy, nursing and social services.
As anyone who used social media last year knows, the Ice Bucket Challenge was everywhere. Created to draw attention to, and raise money for ALS, the Ice Bucket Challenge raised a remarkable $115 million in 2014 for various nonprofits. From Rihanna to Donald Trump, George W. Bush to Kim Kardashian, it seemed that everyone was participating in the Ice Bucket Challenge. But what was the money raised for? What research is being done on ALS?
At Mass General, the answer is “a lot.”
Many Disciplines, One Sharp Focus
Research indicates that ALS patients who receive care at a multidisciplinary clinic do much better. Mass General runs one of the largest multidisciplinary clinics in the country, with a team that includes physicians and experts in physical therapy, respiratory therapy, nursing and social services.
The Mass General ALS Multidisciplinary Clinic provides individualized care from our team of experienced ALS healthcare providers. Available 24/7, the team sees our patients at Mass General, in the home and through telehealth. All our patients have access to the latest treatments and clinical trials. Our relationship with patients is a partnership to find treatments for ALS.
Merit Cudkowicz, MD, chief of Neurology
In addition, Mass General is a leading ALS research center. Mass General Hospital researchers co-founded and co-directed the Northeast ALS Consortium, a group of over 130 ALS clinic centers throughout the world who work together to find treatments for ALS. They pioneered several approaches to accelerate getting treatments to patients, including the use of a Central IRB and Neurobank and GUID. These systems that ensure that data and biofluids from all studies are combined and a shared resource.
Plus, Mass General has one of the nation’s most active research sites, with more than 20 ALS clinical research studies currently active.
Among them:
Testing retigabine (a drug discovered by Mass General’s Brian Wainger, MD, PhD) that may restore proper function of neurons
Testing RNS60, a drug that can decrease inflammation in the brain and spinal cord
Re-programming stem cells from people’s skin or blood into healthy motor neurons to study the disease causes and targets for therapy
Using gene therapy to shut down genetic mutations that cause inherited forms of ALS
Studying clinical care guidelines; using retrospective patient reviews to see if we can provide better car
A Place for Hope
A great deal has been learned about ALS in recent years. For example, it is now known that there are different types of ALS; researchers are working to find biomarkers of each type, so they can personalize treatment and make it more effective.
Dr. Cudkowicz believes this inflammation is what causes ALS to spread — blocking or dampening it could halt the progression of the disease.
Another important breakthrough involves inflammation. “We are able to see inflammation in the brain, in the motor cortex of people with ALS,” explained Merit Cudkowicz, MD, chief of Neurology and director of the ALS Research Program. Dr. Cudkowicz believes this inflammation is what causes ALS to spread — blocking or dampening it could halt the progression of the disease. She and her team are testing several drugs to treat the inflammation. “The inflammation we see using special PET imaging gives us a target for treatment,” she said.
In the meantime, physicians at the Mass General ALS Multidisciplinary Clinic provide the latest treatment to people with ALS. The clinic’s unit chief, James Berry, MD, believes the Ice Bucket Challenge was “a shot of adrenaline” for patients, families and the entire ALS community. “It has created hope and a feeling of connection among those with the disease,” he said. “We want our clinic to be a place for hope.”