Mitsubishi Tanabe Pharma Corporation (MTPC), which is locally based in Jersey City, N.J. with a head office in Osaka, Japan, announced yesterday that a New Drug Application has been submitted to the U.S. Food and Drug Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateral sclerosis (ALS).
Those in the ALS community may have questions about what this means for them. Below we provide some background information on edaravone.
The goal of their project is to accelerate a novel microRNA (miRNA) therapeutic approach to reduce neuro-inflammation, in order to bring it into clinic trials and meet a significant medical need in ALS. Below we provide some background on this exciting work. Continue reading Targeting Neuro-inflammation to Treat ALS
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.
Aquinnah Pharmaceuticals, in partnership with researchers at Boston University, are targeting stress granules to design new therapies for amyotrophic lateral sclerosis (ALS). The goal is to advance promising new drug leads aimed at providing disease-modifying treatments for patients that will slow the clinical progression of this devastating disease.
One of the cornerstones of The ALS Association’s global research program is to fund milestone-driven projects to push research efforts more rapidly toward effective treatments and cures. The Association successfully accomplishes this is through fostering partnerships between academic laboratories and industry, and then funding them through The Association’s TREAT ALS™ Drug Development Contract program grants.
One great example of this type of collaboration is a partnership between Dr. Ben Wolozin, Professor of Pharmacology and Neurology at Boston University School of Medicine and Dr. Glenn Larsen, CEO of Aquinnah Pharmaceuticals based out of Cambridge, Mass. Dr. Wolozin also serves as Aquinnah Pharmaceutical’s Chief Scientific Officer.
When I was diagnosed with ALS, I was hoping to see my sons graduate from high school. As it turns out, I’ve been blessed to have seen them graduate from high school and college.
I know I’m one of the lucky ones. ALS has progressed fairly slowly in me and I don’t take that for granted. From day one of learning I have this disease, I’ve done what I can to advance ALS research and the discovery of new treatments.
All of us living with ALS have to have hope: hope for a treatment and hope for a cure. That’s why I’m excited about a new report that came out today from the Pharmaceutical Research and Manufacturers of America (PhRMA) and The ALS Association, “Medicines in Development for Rare Diseases.”
This new report, which also shares my story, finds that America’s biopharmaceutical research companies are currently developing more than 560 medicines for patients with rare diseases, 38 of which are for people with neurological disorders, including ALS.
Among the new therapies in development for ALS is antisense technology against SOD1. Antisense technology is an important step toward helping patients and their families manage ALS and something The ALS Association has supported since 2003.
There are about 7,000 known rare diseases that affect fewer than 200,000 people in the U.S. every year. This report gives me hope that industry is focusing on these diseases, including ALS, more than ever before.
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. Continue reading What Your Support Gives to Researchers: An Interview with Dr. James Connor
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.