My name is Rick Bedlack. I am a neurologist at Duke University in Durham North Carolina, and I started the Duke ALS Clinic 16 years ago. I am working to empower people with this disease to live longer and better lives and to have a greater role in research. I currently run the ALSUntangled Program (www.alsuntangled.org) and The Northeast ALS Consortium (NEALS) ALS Clinical Research Learning Institute. This is the story of a new program I recently started called ALS Reversals.
ALS is a degenerative disease of motor neurons, typically characterized by progressive muscle weakness, increasing disability and shortened survivals. It is widely recognized that ALS progression can be variable. It can be variable between patients, with some folks progressing much more slowly than others. It can also be variable within a given patient, with periods where the disease seems to speed up or slow down for a while. Less appreciated is the fact that ALS progression can stop (plateau) or even reverse with significant recovery of lost motor functions.
On June 10, 2016, The ALS Association brought together the current and past awardees of its Milton Safenowitz Postdoctoral Fellowship to participate in a day-long research symposium and awards ceremony. The workshop was funded by The Greater New York Chapter of The ALS Association.
About the Award
The award was founded by the Safenowitz family through The Greater New York Chapter of The ALS Association. It is in memory of Mr. Safenowitz, who died of ALS in 1998. These awards are to encourage and facilitate promising young scientists to enter the ALS field. Fellows work with a senior mentor and receive extensive exposure to the ALS research community through meetings and presentations, like this workshop. After completing this fellowship, approximately 90 percent of the awardees stay in ALS research. They go on to establish their own laboratories to continue studying ALS and mentoring more ALS researchers along the way.
Dr. Andrew Geronimo is a talented young investigator using brain computer interface (BCI) technology to improve the lives of people living with ALS by enhancing their ability to communicate. He and his mentor Dr. Zachary Simmons, also at Penn
State Hershey Medical Center, have received a grant from The ALS Association to develop new opportunities for BCI technology. Together they work to train people living with ALS and their caregivers on how to setup and best use BCI in their homes through a telemedicine program. Through this work, they are continuously optimizing the BCI system.
“The ultimate goal for our research is make a difference now for people living with ALS. By listening to their needs and engineering a BCI to meet those needs,” stated Dr. Geronimo.
What is Brain Computer Interface (BCI)?
BCI is exactly what it sounds like: a direct connection between the brain and the computer. Normally, connections are made between the brain and muscles via motor neurons. Motor neurons in the brain relay a signal to the muscles to move.
In ALS, motor neurons are damaged and those connections break. This results in muscle weakness and eventually paralysis. BCI is revolutionary in that it makes up for that lost connection.
Meet Dr. James Connor, an ALS researcher who leads a team at Penn State Hershey Medical Center in Hershey, PA that recently received an ALS Association Investigator-Initiated grant to move his exciting research forward. The ALS Association spoke with Dr. Connor about his investigations into how a therapeutic iron solution is potentially protective in ALS and how collaboration at Hershey bridges the lab and the ALS clinic.
What has the support from The ALS Association meant to you and how has your Investigator-Initiated grant pushed your project forward?
The support helps us on so many levels. It excites us, invigorates us and validates us. But at same time, there is a strong sense of responsibility because this is money from someone that was most likely touched by this devastating disease. That level of trust means a lot and heightens our work. It is a privilege to work on the brain and ALS and to have some ideas that could potentially help someone that is suffering. When we are working 60 hours a week and are tired and working on a weekend to do an experiment, knowing where that award money came from to do our work invigorates us.
Do you have a message for the donors that helped make this project possible?
We always tell our donors that our job is to provide the hope. When a person goes into the doctor’s office and gets the devastating diagnosis, clinicians are obviously wonderful working with the person to deal with the disease. We want to be the element of hope to come in and say that our study is working and hopefully this will be relevant and helpful to the patient. In return, donors are instilling their hope in us, which is a tremendous gift and honor. Also, we have lots of great ideas, but without the funding, they are just ideas. So donors are part of our implementation team. Donors join our team! When we receive a grant, we get excited to go work to do our experiments. Otherwise, people get frustrated because they cannot afford to do the experiments they want to do.
Since 1996, the Sheila Essey Award for ALS Research has been presented by Dick Essey at the American Academy of Neurology Annual Meeting, in memory of his wife Sheila, who battled ALS for 10 years and passed in 2004. This year, the award was presented to Ammar Al-Chalabi, Ph.D., FRCP, DipStat from King’s College London. Find out how Dr. Al-Chalabi’s work is driving progress in ALS research forward.
This year, The ALS Association and the American Academy of Neurology (AAN) were very pleased to honor Dr. Ammar Al-Chalabi with the Sheila Essey Award for ALS Research for his major contributions to the understanding of ALS and the search for new therapies to treat the disease. Dr. Al-Chalabi was presented the 2016 Sheila Essey Award by Dick Essey at the 68th AAN Annual Meeting in Vancouver, B.C. on April 20, 2016. The $50,000 prize was given to fuel his promising ALS research projects and to fund talented young scientists on his research team.
Dr. Al-Chalabi is currently Professor of Neurology and Complex Disease Genetics at King’s College London and Director of King’s MND Care and Research Center. He has made significant contributions to the ALS field. Dr. Al-Chalabi helped identify many known genetic causes of ALS, including C9orf72; developed the ALSoD database, an important tool for researchers funded by The ALS Association, MNDA UK and Therapy Alliance; and has made significant contributions to understanding disease staging, which impacts ALS care and clinical trial design. For more information regarding his illustrious research career click here.
“Our ability to make progress in ALS depends so much on attracting the best young scientists into the field. The ALS Association’s Milton Safenowitz Post-Doctoral Fellowship program is a critical part of that effort. Almost 90 percent of our Fellows stay in ALS research, making up a significant fraction of the younger generation of ALS researchers.”
– Dr. Lucie Bruijn, Ph.D., M.B.A., Chief Scientist, The ALS Association
Javier Jara, Ph.D., is a Research Assistant Professor in Dr. Hande Ozdinler’s laboratory in the Department of Neurology at the Northwestern University Feinberg School of Medicine in Chicago. He was funded by The ALS Association’s Milton Safenowitz Post-Doctoral Fellowship for ALS Research from 2010-2012 and recently was awarded his own Investigator-Initiated grant by The Association. These awards helped support his research including a project that focuses on how upper motor neurons die in ALS and how to intervene to prevent their death. The results of this project were featured in the January 21, 2016, issue of Gene Therapy. Recently, we sat down with Dr. Jara to learn more about his exciting research project and to get to know the person behind the science.
What has the support from The ALS Association meant to you? Do you have a message for donors who helped make your projects possible?
I want to give a big thank you to the donors for their support and trust. Without their generous contribution, I would not be where I am now. I am extremely grateful!