Dear Friend of the Moser Center,

COVID-19 has brought unprecedented challenges to us all, including those of us working at the Moser Center for Leukodystrophies at Kennedy Krieger Institute. Following the example set by The Johns Hopkins University, Kennedy Krieger has rolled out a series of protocols to protect patients, students, employees and the general public from infection by the new coronavirus. These measures include a reduction of all lab work not directly related to COVID-19, and a prioritization of lab functions deemed “essential” to our work. We have done our part to donate personal protection equipment and other resources to COVID-19 research labs and medical staff members at Kennedy Krieger and Johns Hopkins, and to limit our own work in the lab to necessary duties only.

Effects of COVID-19 on LBSL Research:

With these changes, we thought now would be a good time to update you all on how this has affected our LBSL research on leukoencephalopathy with brain stem and spinal cord involvement and lactate elevation (LBSL). In prioritizing lab duties, several projects and experiments are temporarily on hold. Of course, certain lab functions must continue: Veterinarians and technicians from Johns Hopkins Research Animal Resources maintain our mice, providing clean cages and food as normal, and once a week, we tend to litters, mating pairs and the general health of our mice.

In terms of cell work, and as many of you know, our LBSL cell models require months of regular care until they are ready for evaluation. To prevent the loss of many months’ work, we have deemed these processes “essential,” and we’re maintaining these cell models until they reach a point at which they can be safely frozen. This includes neuron cell models and the growth and maintenance of cerebral organoids. Because many Johns Hopkins labs have reduced their operations, we are exploring the option of sending frozen samples to be analyzed by companies that are still operational during this time. These companies are reputable and offer comparable pricing, and while we have used some of these companies in the past, we most often choose to support our own labs for these types of experiments, when possible.

Working from Home:

Although we can’t currently do as much work in the lab as we usually do, we’ve adjusted to working from home. The COVID-19 crisis has, at least, given us an opportunity to get caught up on reading scientific journals and protocols, and on trouble-shooting recent issues, planning future experiments, searching and applying for funding opportunities, and planning and writing papers. We’re also getting caught up on our data analysis, which can be very time consuming.

For example, the IncuCyte, a high-resolution microscope inside a cell incubator available to us through a Johns Hopkins lab, captures images of cells at set frequencies. In March, we placed LBSL cells in an environment that allows the cells to develop, and set the IncuCyte to take pictures of the cells every hour for two days, and then every eight hours for the next five days. This produced just over 12,000 images. Software to analyze data this size is quite expensive, so we’ve been designing our own in-house coding programs to organize these images, identify cells within them, and track each individual cell over the one-week period to learn how these cells are changing over time.

Ongoing Clinical Research:

Regarding clinical research, we’re continuing to gather information on individuals with LBSL every six months through remote visits in participants’ homes to understand more about the progression of LBSL. In particular, we’re using wearable accelerometers (devices that measure movement) to determine how walking and balance changes over time in individuals with LBSL. Our research team is analyzing this data from home, using software programs on laptop computers.

So far, data on LBSL participants and healthy volunteers of matching age and sex have shown that gathering data with the wearable accelerometers at home is just as good as doing this testing in person or with gold-standard equipment in the physical therapy lab at Kennedy Krieger. It also appears that in LBSL participants, as opposed to in healthy volunteers, standing balance does change over time, and that change is easily detected by the wearable accelerometers. These devices also show signs of uncoordinated gait when worn by LBSL participants while walking. We are following these gait and balance signs to determine how quickly the signs change over months and years. Once a treatment has been developed, these gait and balance signs will be the crucial metrics we’ll follow in a clinical trial of that treatment, to understand if the treatment is preventing or reversing motor disability in LBSL.

We are very grateful and pleased that our participants have continued to work with us during this difficult time. Thank you!! We also hope that many more will join our study. The main limitation to progress is that we need at least 5–10 more participants for us to interpret the information accurately and publish the next phase of our work. Currently, we’re writing a paper about the results obtained during the first study visit. We have also formed a collaboration with Professor Marjo van der Knaap and Dr. Marc Engelen in Amsterdam to begin recruiting European study participants. The process by which they will participate has been approved. Using funds from donations, we recently purchased the same wearable devices for them to use as we are using in North America. Recruitment for the European study is on hold due to COVID-19, but we will resume our work together as soon as it is safe to do so.

Data Science and Artificial Intelligence Update:

We’ve also continued to expand our capabilities in data science and artificial intelligence, developing new algorithms to analyze MRIs and the clinical data previously referred to in this letter. We’ve been developing much-needed methods to use artificial intelligence to understand disease progression. As this work is entirely based in computer coding, our imaging and artificial intelligence team has been least affected by the pandemic, and encouragingly, the team has maintained its progress at a normal pace. In addition to continuing work on these models, we meet with collaborators once a week through teleconferencing, and we’re always on the lookout for new opportunities to present papers and apply for grants.

We hope this newsletter finds you safe and healthy during this time. We thank you for your support and interest in our research, and we look forward to returning to the labs, once the COVID-19 crisis has passed.

Dr. Ali Fatemi's headshot.

All the best,

Ali Fatemi, MD, MBA
Director, Moser Center for Leukodystrophies 
Chief Medical Officer, Kennedy Krieger Institute 


Please help us to continue vital research to improve the lives of those affected by LBSL. Financial support from individuals like you has helped us to increase our efforts, in order to discover treatments and possibly a cure. Please consider making a gift by visiting this link.