Mass. has biggest research projects going
The biggest boons in ALS research have been seen in work on genes and biomarkers — ways to identify the disease and its progress within the body — according to local advocates and researchers.
“The areas of genetic discovery and biomarkers have been really hot,” said Dr. Sabrina Paganoni, chief of neurology at Massachusetts General Hospital. “It’s really a new frontier. Genes and biomarkers hold a key for curing the disease.”
ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease, affects about 12,000 to 15,000 Americans. Patients with ALS are robbed of the ability to walk, talk, chew and eventually, breathe. Most of those who suffer from the disease die within three to five years.
Two new genes have been discovered by Dr. John Landers at UMass Medical School.
Last year, an intravenous medication called Radicava was approved — the first treatment for ALS to get federal approval in 22 years — to help slow the disease’s progression. Biotech company Treeway is working on an oral form of the medication.
There are still numerous projects underway, both locally and nationwide, funded by money from the Ice Bucket Challenge.
One includes an ongoing study at Brigham and Women’s Hospital focusing on identifying biomarkers in the bodies of those suffering from the disease.
“Right now there are no reliable biomarkers to diagnose. It is very hard to predict,” said Dr. Oleg Butovsky, neuroimmunologist at the Ann Romney Center for Neurologic Diseases at the Brigham. “The idea is to be able to detect the onset of the disease.”
The study is based on blood samples taken from ALS patients from the day of diagnosis every three or four months until they die.
“We will be able to identify a combination of different immune cell types which are affected and show how the disease is progressing,” Butovsky said.
Some of the biggest projects are happening within the Bay State, said Lynn Aaronson, executive director of the ALS Association Massachusetts Chapter.
“I think we’ve made a lot of progress and different researchers are working on different aspects of the disease,” Aaronson said. “I think from the ALS Association’s point of view, Massachusetts has the greatest number of ALS research projects in the country.”