Technological advancements have revolutionized nearly every field of medicine from orthopedics to genetic testing.
Sabrina Paganoni, MD, PhD, a clinician and researcher in the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital, has seen firsthand the potential power and impact technology could have for amyotrophic lateral sclerosis (ALS).
Paganoni is using technology to find new treatments and improve the care and quality of life for patients with this progressive neurodegenerative disease with no known cure.
Using technology to improve research and care
Traditionally, the goal of a clinical trial has been to test a drug’s efficacy. However, Paganoni sees a larger learning opportunity.
“There’s so much that you can learn from clinical trials,” says Paganoni. “I’m really interested in how to develop and use emerging technology to improve clinical trials and learn more about ALS as we test drugs. That way, regardless of the trial outcome, we can learn something about the disease to advance our knowledge.”
Paganoni is grateful to fellow NCRI researcher James Berry, MD, MPH who spearheaded several technology projects and first tested the use of a smartphone app to monitor ALS disease progression.
“Dr. Berry first applied the app to monitor disease progression in a group of ALS clinic patients so we decided to collaborate and test the app in the context of an ALS trial,” says Paganoni. “Apps have been used for other conditions like Parkinson’s disease, but this is the first app that is being tested in an ALS drug trial.”
Paganoni also recognizes that technology can help improve patient care in the short term as researchers await the results from clinical trials. Her background in physical medicine and rehabilitation and her work at Spaulding Rehabilitation Hospital has made her aware of new technology’s potential to improve the care and quality of life of patients who have disabling diseases.
“As you develop new treatments, there’s always going to be a delay or some time that it takes to finish the research. But in the meanwhile, we can address the pressing needs of patients.”
Paganoni uses assisted technology to support patients she sees in her clinical work. For example, she is collaborating with Conor Walsh, PhD, from the Wyss Institute on a wearable robotic globe that can assist with motor functions such as helping patients open and close their hand.
She feels optimistic that such devices can improve patient quality of life.
Collaboration and clinical trials
Paganoni and her colleagues at the NCRI collaborate with basic scientists both at Mass General and at other institutions to carry drugs through the developmental pipeline and efficiently design, conduct, and manage clinical trials on-site.
One such collaboration is with the MassGeneral Institute for Neurodegenerative Disease (MIND) which works to translate laboratory discoveries into prevention, treatment and cures for ALS as well as Alzheimer’s, Huntington’s, Parkinson’s and other neurodegenerative diseases.
Paganoni is currently working with MIND investigators Michael Schwarzschild, MD, PhD, and Ghazaleh Sadri-Vakili, PhD, to test a drug known as inosine that they successfully tested in pre-clinical models of Parkinson’s and ALS.
Paganoni explains that inosine raises levels of urate, a natural antioxidant present in the body that may help counteract oxidative stress, an imbalance between the production of free radicals and the ability of the body to detoxify their harmful effects.
“We and other researchers have analyzed ALS databases and found that people with ALS who have higher urate levels live longer,” says Paganoni. “However, we don’t know if drugs like inosine result in improved outcomes, such as better quality of life or longer lifespan. This trial is our first step in answering this question.”
She is hopeful that among the clinical trials she is currently involved in — including the aforementioned inosine trial — at least one will result in a drug that could be made available to patients.
A unique setting for research
Paganoni says the ability to conduct ALS research from bench to bedside and in collaboration with researchers working on other neurological diseases is made possible by both the NCRI and Mass General as a whole.
She credits NCRI founder and Co-Director and Chief of Neurology, Merit Cudkowicz, MD, MSc with developing the center’s unique infrastructure.
“Dr. Cudkowicz’s leadership and vision is the reason why this place is so prominent in ALS research.”
Paganoni also says the collaborative spirit at Mass General helps researchers studying ALS work in parallel with investigators looking at other neurological diseases.
“Mass General is a unique place in that researchers are encouraged to work collaboratively and there is such a breadth of expertise. By sharing our knowledge and working together, we can make progress faster and more efficiently.”