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.
“We are in a time of great hope in ALS therapy development. The efforts highlighted here are poised to accelerate progress in clinical trials and to bring new treatments to people living with ALS.” – Barb Newhouse, President and CEO, The ALS Association
Collaboration is a cornerstone of The ALS Association’s global research program. That’s why The Association hosts an annual ALS Drug Company Working Group that brings together representatives from pharmaceutical companies and academia to discuss how to move ALS research and therapy development forward.
Every May, The ALS Association joins the ALS community in celebrating ALS Awareness Month, a time to bring attention and understanding to this disease. As part of this year’s Awareness Month, we are happy to be launching the ALS Association Blog!
We know that people living with ALS – including family members, caregivers, and friends – form a tight-knit community. Because of this, we want to make our blog a place for you to share your stories and talk with each other. Posts will include stories and creative works submitted by people in the ALS community, and visitors to the blog will be able to leave comments and share their favorite articles on social media.
In 2014, The ALS Association Golden West Chapter and patient advocate Jim Barber partnered to build the Neuro Collaborative concept. That year, following the amazing outpouring of support from the ALS Ice Bucket Challenge, The ALS Association committed $5 million dollars to the project. With additional help from The ALS Association Orange County and Wisconsin Chapters, the Neuro Collaborative has become an engine for ALS therapeutics. Learn more about the progress of each partner in the Neuro Collaborative below.
Early development of potential therapeutics is a major bottleneck in ALS therapy development. Eliminating this bottleneck is a significant opportunity for accelerating new treatments. That’s where the Neuro Collaborative comes in.
The Neuro Collaborative is a partnership between three leading laboratories in California: Clive Svendsen, Ph.D. at Cedars Sinai in Los Angeles; Steven Finkbeiner, M.D., Ph.D. at Gladstone Institutes, affiliated with University of California San Francisco; and Don Cleveland, Ph.D. at University of California San Diego.
The goal of the Collaborative is to establish and invest in a leading team of experts to efficiently advance ALS drug development together with industry partners. This complements other programs including the Drug Development Contract Program in the TREAT™ ALS portfolio investing in academic and industry partnerships. This synergetic model leverages open dialogue and the scientific expertise of leading researchers to achieve therapeutic milestones as quickly as possible. It also reduces risk in the drug discovery process and attracts pharmaceutical companies to invest in drug development and clinical trials for ALS. With success, potential therapies for ALS will move more quickly than ever toward FDA approval and the open market. Continue reading The Neuro Collaborative: An Engine for ALS Therapeutics
“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! Continue reading Researcher Spotlight: Dr. Javier Jara