Watch exciting video of our special guest speaker Dr. Richard Bedlack, who spoke about ALS reversals at the 14th Annual Research Update and Awards Luncheon on May 31! Thank you for joining us to make a difference in the lives of people with ALS and those who care for them. We are educating the community!
Pat Quinn and Pete Frates, the co-founders of the Ice Bucket Challenge, have a message for the ALS community...
Until we find a cure, every August is Ice Bucket Challenge Month. Get ready for August 2015!
The clinic team poses on the day of the luncheon; PHOTO CREDIT: ROBERT E. PATTERSON
On Sunday May 31st, The ALS Hope Foundation hosted its annual Research Update and Awards Luncheon at the Marriot Philadelphia West in Conshohocken. Close to 100 people from the ALS Hope Foundation community were in attendance, including PALS, their families, the clinic team, advocates, and supporters.
Our guest speaker was Dr. Richard Bedlack, Director of the Duke ALS clinic and founder of ALS Untangled. Dr. Bedlack spoke to us about his latest research project on ALS reversals. There are rare instances in which a person with ALS stops progressing or even gets better. His clinic, along with 20 others worldwide have teamed up to understand these ALS reversals and, perhaps, make them happen more often with the help of clinical trials and the social network Patients Like Me. Dr. Terry Heiman-Patterson, President of the ALS Hope Foundation, took the stage after him and explained the ALS Hope Foundation's newest programs and our most recent and exciting research projects.
After her update, Dr. Heiman-Patterson presented the awards. The Sunrise Award was given to Dan Pellegrino for having demonstrated the highest level of commitment to the ALS Hope Foundation by tirelessly supporting its mission and fundraising. Dan tragically lost his battle with ALS on November 21, 2014, but his dedicated family continues to support our efforts. They accepted this award on his behalf.
several honorees pose with dr. heiman-patterson and the mda/als center of hope team; Photo Credit: Robert E. Patterson
The Courageous Heart Award is reserved for those who have taken a proactive role in the fight against ALS, advocating for PALS and supporting the ALS Hope Foundation while courageously battling ALS themselves. Our Courageous Heart winners for 2015 were Dennis Casey, Sharon Corosanite, H. Todd Kelly, Denise Richards, and Karen Gallimore. We would like to again thank you for everything that you do to keep hope on the horizon! Your courage inspires us all.
The Community Hope Award was given to Doug Fowler and to Ram Consulting in recognition of their outstanding support and dedication to the ALS Hope Foundation. Finally, the Extraordinary Honoree Award was given to Bernie Parent in appreciation for his service and dedication to promoting ALS awareness. His efforts and continued support are greatly appreciated. We also acknowledged our Horizon Honors, a title given to individuals or organizations who have helped to keep hope on the horizon by contributing or raising over $1,000 in 2015.
All honorees and speakers inspired everyone in attendance. May we all remember their courage and their dedication as we continue to keep Hope on the horizon!
The ALS Hope Foundation was honored to participate in the Collaboration for a Cure event on April 17, 2015. We at the foundation believe that partnership and cooperation are the only way forward! The statement below is a prepared release about the event shared with all those who were in attendance. Read and enjoy!
Thanks in part to last summer’s ALS Ice Bucket Challenge, the first Collaboration for a Cure took place on April 17, 2015, in Arlington, Virginia. Representatives from 18 organizations who are fighting amyotrophic lateral sclerosis (ALS) gathered to identify key areas where collaboration among their organizations could help drive forward collective efforts to help people living with this disease. One such attendee, Dr. Kevin Horton, Chief of Surveillance and Registries within the Division of Health Studies (pictured above), presented information about the National ALS Registry.
The invitation to this first meeting was to individuals at many of the leading ALS organizations. Patients who lead some of those organizations were invited. Although, none were able to attend, they were well represented by their chosen delegates. The group is committed to having people living with ALS and caregivers of people with ALS participate in future meetings and task groups. The following organizations were represented: ALS Canada, ALS ETF, ALS Hope Foundation, ALS Therapy Alliance, ALS Therapy Development Institute (ALS TDI), ALS Worldwide, Answer ALS, International Alliance of ALS/MND Associations, Iron Horse Foundation, Kevin Turner Foundation, Les Turner ALS Foundation, Muscular Dystrophy Association (MDA), Prize4Life,Project A.L.S., Target ALS, Team Gleason, The ALS Association, and The Robert Packard Center for ALS Research at Johns Hopkins.
The day began with a welcome from Barbara Newhouse, President and CEO of The ALS Association. While The ALS Association brought together this first meeting, it was acknowledged that future meetings will be convened and driven forward by the community of ALS organizations. Newhouse introduced the moderator for the day, Paul De Morgan, who has 16 years of experience in facilitating and mediating multi-party, consensus-oriented policy dialogues and site-specific dispute resolution processes.
After a full day of discussion (described below), there was near unanimous commitment to continue to support the collaborative into the future. While it was agreed to continue to discuss key issues pertaining to ALS as a group, it was acknowledged that the power of the meeting was in the diverse opinions and levels of experience. It was noted throughout the meeting that the focus must remain on the patient and finding solutions for their urgent needs. It was also unanimously agreed that despite the many diverse opinions, the participants were eager to work together if it meant immediate action.
Many areas need to be addressed, but for this meeting, three areas of focus were identified in advance to be topics of discussion. The first topic involved gaining consensus on the prevalence and incidence of ALS. The goal was to share detailed information of the known and unknown regarding current data and gain alignment in messaging regarding incidence vs prevalence. Individuals and organizations have used a range of estimates when describing the number of people living with ALS in the United States, from as few as 12,000 to as many as 40,000 or more. Representatives from the Agency for Toxic Substances and Disease Registry (ASTDR), including Kevin Horton, DrPH, MSPH, CPH, Chief of Surveillance and Registries within the Division of Health Studies, and Paul P. Mehta, M.D., Medical Epidemiologist, presented information on the National ALS Registry and its efforts to identify people living with the disease to better understand incidences and prevalence.
After a robust discussion among members of the group, it was determined that the data is not sufficient to support currently reported numbers of people living or diagnosed with ALS. The registry’s first report is based on one year’s worth of data. Although it is consistent with published literature, the organizations agreed that the first year data from the registry may have under-reported prevalence. Therefore, the organizations agreed that it was critical to work together to urgently explore options to enhance the registry. A task force made up of designees from the ALS organizations in attendance will join together to help accomplish that goal. Creating a strategy to foster enrollment through clinical experiences and also in patient and caregiver outreach are two areas agreed upon that need immediate improvement. It was ultimately agreed that all people living with ALS need to be counted and their medical voices heard through their critical data.
The second discussion of the day focused on the formidable challenges that exist to bring a new therapy to market and the development of a guidance document to help companies and researchers involved in ALS drug development navigate the regulatory process and speed drug development. Given that the U.S. Food and Drug Administration (FDA) does not have the resources to develop guidance documents for each disease, this group is unified in a commitment to assure the guidance document is not only produced but also reflects the recent community driven efforts. Resources from the ALS Ice Bucket Challenge are dedicated to that assurance. The initiative will involve participation from the entire ALS community: people living with ALS, ALS organizations, clinicians, researchers and partners in the industry. It will be governed by a steering committee comprised of content area experts who will chair individual working groups focused on specific chapters of the guidance document including, biomarkers, benefit-risk, clinical trials and outcome measures, natural history and diagnosis. The steering committee will also be advised by a patient and caregiver advisory committee to ensure patient input throughout the guidance development process. This will be the first patient-focused guidance for ALS product development that would be submitted to the FDA. And, it will be a consensus-based work product that incorporates stakeholder views in areas such as trial design, biomarkers, surrogate endpoints, patient-reported outcomes, benefit-risk and others.
The group fervently agreed to coordinate efforts to create a needed momentum for this important guidance. As part of this initiative, a communications plan will be included in order to provide the ALS community with timely input, updates and milestones.
Finally, expanded access was a topic of discussion and how ALS organizations can better communicate information about treatments in development. Because there is currently only one approved drug to treat ALS and given the devastating nature of the disease, there are often calls to support widespread access to therapies that have shown some promise in early stages of development. The FDA has a number of existing authorities that are designed to expedite the development and approval of new treatments and also provide access to those treatments, including outside of a clinical trial and prior to FDA approval. Those authorities include: expanded access; accelerated approval; fast track; priority review; breakthrough therapy designation; and orphan drug designation.
The conversation centered on educating attendees about these authorities as well as other related issues, such as “Right to Try” laws that have been introduced and enacted in states across the country. “Right to Try” laws seek to provide access to investigational therapies outside of FDA governed expanded access programs. The session also focused on how ALS organizations can communicate information about promising investigational treatments to people with ALS and how organizations can respond to calls for widespread access to those potential treatments.
The group recognized that ALS organizations can do more to explain the differences between the various FDA authorities mentioned above. Furthermore, the group acknowledged that people living with ALS have unique perspectives as it relates to the above options and should help guide organizations when they choose how to communicate opinions of a new therapy or treatment concept. It was recommended that a task force convene to discuss expanded access issues.
Those in attendance agreed that there will be future meetings for the Collaboration for a Cure and will immediately begin to work together to follow up on the various action items that were identified by the group.
One final note: the group was shown a video and letter from Pete Frates and Pat Quinn, co-founders of the ALS Ice Bucket Challenge, asking all the organizations to work together and follow their lead in order to make the event a success again in 2015 and the years to follow. ALS Association Chief of Staff Brian Frederick outlined for the group a collaborative Ice Bucket strategy that Pete and Pat are spearheading, which will be for the benefit of the entire ALS community. The message was well received and all agreed we are a greater force when working together.
Dr. Heiman Patterson and her collaborators at Massachusetts General Hospital have been awarded a Clinical Management Grant from The ALS Association to study strategies that help caregivers deal with cognitive changes in ALS. While ALS primarily affects the motor system, up to 40% of PALS experience cognitive impairment, and approximately 20% PALS show behavioral changes, predominantly apathy and disinhibition. These cognitive and behavioral changes add significantly to the burden and distress of caregivers who have now to face the challenges of managing behavioral changes in addition to the physical disability. The overall goal of our study is to characterize the relationship of caregiver burden to cognitive and behavioral impairment in ALS and to develop and perform a feasibility study of a pilot individualized intervention as a way to reduce caregiver burden. Ultimately, this study will provide the necessary background to develop, inform, and guide future targeted and larger scale interventions directed to both PALS and caregivers.
We are pleased to announce that the MDA/ALS Center of Hope has received a Christopher and Dana Reeve Foundation Quality of Life Grant, which will be used to upgrade our Assistive Technology Resource Center. The Resource Center was started in 2006 to provide individuals with ALS and their families with education and access to communication and other forms of technology in order to increase independence and quality of life. This new initiative is specifically targeted to meet the needs that our PALS at the Center identified as most important, specifically cell phone access, computer and iPad access, and environmental control. These grant funds will help with the purchase of programmatic equipment.
Rachel Patterson, General Manager of the International Alliance, and Jacquellyne Hengst, Director of Development for the ALS Hope Foundation in Philadelphia, sit down for a chat with Liz Tucker, producer of the new ALS/MND documentary Filming My Father.
Press play to listen to the interview!
Watch the Filming My Father: In Life and Death trailer on YouTube:
Make sure to like and follow Filming My Father: In Life and Death on Facebook!
Thank you to everyone involved for spreading awareness!
Sara Feldman, Clinical Liaison for the ALS Hope Foundation and Physical Therapist at the MDA/ALS Center of Hope, presents during the 2014 ALS/MND Meetings in Brussels
The 25th Annual International Symposium on ALS/MND was held in Brussels, Belgium, December 2014. Terry Heiman-Patterson, MD, and Sara Feldman, PT, DPT, attended the conference, which was put on by the Motor Neuron Disease Association in cooperation with the International Alliance of ALS/MND Associations. ALS Liga Belgium was the fantastic host of the event.
The ALS Hope Foundation is a member of the International Alliance of ALS/MND Associations, and Dr. Feldman is on its Board of Directors. The Symposium began with the 22nd Annual Meeting of the Alliance of ALS/MND Associations December 2 and 3, 2014. This meeting is a chance for organizations from around the world to come together and share ideas. The day began with presentations by various members on fundraising, advocacy and awareness and, of course, the discussion began with the Ice Bucket Challenge. Every organization was impacted by the donations and the awareness the #IBC raised. Efrat Carmi (IsrALS) reported the top link clicked on the IsrALS website was “What is ALS?” Rob Goldstein (ALS TDI) led the inital discussion, but everyone was eager to share his or her own organization’s story. We then heard from several other members on their activities this past year. The discussions then turned to challenges to providing services and national ALS/MND registries. We heard an update on the partnerships supported by the Alliance then adjourned for the day. The evening dinner was highlighted by a musical performance by Alper Kaya (Turkey MND Association) and his daughter. The presentations continued on the second day with several reports on services and programs offered by member organizations and then ended with a scientific and research update by Dr. Leonard Van Den Berg (ALS Netherlands), including information on Project MinE. In addition to presentations, working groups were formed to address a variety of issues related to the Alliance.
Click here to scroll through the archives of the Alliance Meeting and download each presentation.
Click here to read a full synopsis of the Alliance Meeting from ALS TDI.
The Ask the Experts session followed the Alliance Meeting and brings together people living with ALS/MND and invited speakers for a question and answer session. Individuals who were unable to attend the meeting could also submit questions by email. The speakers this year were Dr. Orla Hardiman (Ireland), Professor Leonard Van Den Berg (Netherlands), and Dr. Lucie Bruijn (USA).
Click here to watch video of the Ask The Experts Session.
The 12th Annual Allied Professionals Forum brought together more than 180 health care professionals from around the globe for a day to share information and network on December 4, 2014. The ALS Hope Foundation has supported the APF for more than 10 years, and Dr. Feldman is a member of the Program Committee. Presentations covered a range of topics including ideas for clinical management of symptoms and FTD, helping staff deal with compassion fatigue, end-of-life issues and electronic options for communication and education. Dr. Feldman presented "Equipment Utilization in the ALS/MND Population: Trends and Timing" (click for video).
Click here to watch video of the 2014 Allied Professionals Forum, click here.
Click here to read more about the Allied Professionals Forum.
The 25th International Symposium on ALS/MND then ran from December 5-7, 2014 and brought together more than 900 scientists, people living with ALS/MND, clinicians and association members. The symposium runs two sessions simultaneously, the scientific and the clinic tracts, and at times a third tract on biomarkers. The scientific themes included RNA processing and dysregulation, protein misfolding and toxicity, autophagy, in vitro modeling, cell biology and pathology, genetics and genomics, murine models, modulating SOD1 toxicity and non-neuronal cells. The clinical sessions included the themes of diagnosis/prognosis, trials and trial design, assistive technology, care practice, epidemiology, end of life decisions, respiratory management, nutritional assessment and management, and cognitive changes.
During the poster sessions, Dr. Terry Heiman-Patterson presented her poster “Application of a Staging System to the ProACT Database Population” with co-authors Anahita Deboo, MD, Saugat Dey, MD, and Guillermo Alexander, PhD. Dr. Feldman presented a poster on “Equipment Utilization in the ALS/MND Population: Trends and Timing.” Her co-authors include Peggy Allred, PT, DPT, from Cedars-Sinai in Los Angeles and Jillian Moraveck, a Biomedical Engineering student at Drexel University.
The week-long meetings ended with an ongoing commitment to the ALS/MND community and knowledge that there is further work to be done.
Dr. Terry Heiman-Patterson, photographed by Robert X. Fogarty
The Ice Bucket Challenge underscores how just a few people can make a huge difference! Thanks to Pete Frates and Pat Quinn, this challenge—and the ALS cause—has gone viral, and now this “orphan” disease is recognized worldwide.
Some have criticized the Ice Bucket Challenge for being frivolous. But here it is, $100 million later, still going strong, with donations being made to worthy ALS organizations all over the country. Through the generosity of our supporters, the ALS Hope Foundation has raised $60,000, and the influence of the ALS awareness that has been raised through celebrity and media participation is incalculable.
The new surge in funds generated by the Ice Bucket Challenge has the potential to turn the tide toward real treatment and a cure for ALS. But how should the funds be spent? How can we use this money responsibly and effectively, with the most impact?
It would seem to me that we have to think about how to help people living with the disease today while also preparing for a better tomorrow through research. In the here and now, we have a moral obligation to optimize the care and treatment of people already living with ALS (PALS), and to provide much-needed support to their families and caregivers.
The best way to provide that help to PALS today is through the support of multidisciplinary clinics—making it easier for committed physicians to run their centers and to deliver state-of-the-art care and technology. We also need to support the additional functions related to research that clinics provide, including collection of clinical data and tissue specimens and the establishment of dedicated ALS research labs at local centers. This will pave the way to real progress in research. This is why raising funds for local centers through organizations like the ALS Hope Foundation as well as donating to national organizations is so important. People with ALS should have access to the best care and research opportunities out there, no matter where they live.
We also need to spend funds to apply the technologies already available to the physical and communication needs of people with ALS so that they can be more independent and remain connected to their families, friends and communities. We need to advocate for insurance reimbursement for commercially available devices, and we need to support the use and development of technologies not yet commercially available but effective. These technologies need to be expanded and new technologies developed to open the world not only for PALS, but all people with physical disabilities.
We need to continue to support networks of clinical ALS centers that are uniformly trained and experienced in ALS clinical trials and clinical research like the network built through the Northeast ALS Consortium (NEALS). NEALS has the infrastructure to design and implement efficient, well designed trials for any promising treatment that may come to light through research with widely distributed access for PALS in the US through more than 100 member ALS centers. We need to work to accelerate the time it takes to get an idea from the laboratory bench into the clinic since clinical research is critical to progress in solving ALS. This clinical research must be done with the utmost care and accuracy.
We also need to educate PALS and keep PALS and families engaged in the process as the stakeholders. We need to ensure that we consider the needs and wishes of PALS and join with them in advocacy to mandate the best care, research and the rapid deployment of potentially efficacious treatments to the clinic.
Finally, we need to invest in research. What we do NOW will affect all that is to come. We need to better understand all the causes of motor neuron death in ALS and the complex genetics of the disease so that we can design treatment strategies that will make a real and tangible difference to people affected by ALS. This means funding not only established investigators, but new ideas, collaborative research, and innovative thinkers. We need to make sure that everyone with an idea has the opportunity to pursue it, that funds are used judiciously but also across many labs that work together, building on each other’s strength, sharing results and ideas, and allowing discourse and reduces redundancy. The research community must be accountable not only to funding agencies, but most importantly to PALS.
The urgent need of people living with ALS is palpable and THE TIME IS NOW . . . to advocate, to spread awareness, to continue raising funds to support clinical care and research, and to find treatments that lead to a cure . . . so that people living with ALS can look to the future!!!
Sincerely,
Dr. Terry Heiman-Patterson, President
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