Hope Advances - Radicava™ Approved to Treat ALS

A message from Dr. Terry Heiman-Patterson

On Friday, May 5, Radicava was approved by the FDA for the treatment of ALS. As the first drug approved in more than 20 years, Radicava gives hope to our entire community - clinicians, researchers, and people with ALS - that we are making progress in slowing down this disease. Our dream is to stop it in its tracks.

The trial history of Radicava in Japan (trials narrowed their focus to study PALS early in disease progress) shows us the value of exploring responder groups in studies of new drugs. The fact that testing was focused on a subset of PALS does not mean that the drug does not work in others, but simply allows us to detect an effect that might otherwise have been missed. 

This is fast becoming the age of individualized medicine. It will require that we understand disease mechanisms and individual differences in active disease processes and use this information to target drugs to PALS that we predict will respond. The discovery of ALS biomarkers (measurable, physiological signs that change as the disease progresses) will be critical to this process. This underscores the importance of ongoing efforts in the research community to collect tissue samples and DNA to define individual differences between PALS so that we can enhance our understanding of this complex, heterogenous disease. 

We are on the precipice of major breakthroughs! This is clearly a time for celebration of the approval of Radicava and a new era in ALS therapeutic development.

Both the FDA and MT Pharma America are to be commended for their commitment to ALS and to accelerating the drug approval process. The MDA/ALS Center of Hope at Temple University will be working actively to set up a process for receiving Radicava once it is available.

For more information, click here

For questions about Radicava™, please feel free to contact the following services set up by the company:

Searchlight Support™ at 1-844-SRCHLGT (1-844-772-4548)

You can also sign up to receive updates about Radicava™, including when it will be available for your healthcare provider to prescribe at: https://www.radicava.com/patient/

BREAKING NEWS: Radicava (edaravone) Approved by FDA

The US Food and Drug Administration has approved Radicava (edaravone) to treat people with ALS in the United States. 

This is important news for the ALS community. Radicava (edaravone) was approved today by the FDA to treat people with ALS. “After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”

We at the ALS Hope Foundation would like to congratulate MT Pharma America on this latest development. The neurologists at the MDA/ALS Center of Hope clinic are looking forward to having a new treatment option. For more information on Radicava, please visit their website, www.radicava.com, or read the press release.

We also want to thank YOU for donating and advocating for people with ALS and their families, not only during ALS Awareness Month, but everyday. You helped make this possible! Thank you for your support! 

New Video Series Aims to Provide Visual Aid in Understanding ALS

ALS Hope Foundation is pleased to announce a brand new Educational Video Series! The goal of the series is to provide valuable educational tools for people with ALS (PALS) as well as their caregivers. The nine video series covers practical advice ranging from how to increase computer accessibility to advice for supporting nutrition. In one video, a staff member touts the benefits of using PEG (Percutaneous Endoscopic Gastrostomy) tubes to assist those who have difficulty eating. The video aims to provide a detailed explanation of the process, starting with when to consider a PEG tube to a demonstration of the physical device, followed by best practices for maintenance. The information is presented in an easy to understand format in a concise 8 minute video. The aim of the series is not to provide all possible data on the subject but to deliver easily accessible and relevant facts to help PALS and caregivers make informed decisions.

Other practical videos include splinting and seated leg exercises as well as facts regarding speaking and swallowing difficulties. The information in these videos is paramount to the Foundation’s goal to provide educational support. The videos are free and easily shared so that the public, as well as PALS and caregivers, can learn more about the disease and the steps taken to combat its effects.

To view the videos, follow this link. Remember to subscribe to our YouTube channel by hitting the red Subscribe button.

The beginning of a beneficial partnership: Inaugural Gala of Hope commemorates the ALS Clinic's move to Temple Health

ALS Hope Foundation's first annual Gala of Hope was a resounding success! Thank you to our PALS, friends, families and supporters who came out to the event. The evening began with a cocktail hour where guests chatted with friends old and new while enjoying delicious hors d'oeuvres. The party migrated into the ballroom where shimmering chandeliers and champagne-inspired decorations greeted guests as they took their seats. 

Dr. Terry Heiman-Patterson opened the evening by thanking MT Pharma for their generous sponsorship of the Gala as well as thanking Robert Rosti and Jean Hubble for attending the event on their behalf. She also extended a warm thanks to Temple Health for their new support of the MDA/ALS Clinic of Hope and the ALS laboratory. She stressed the importance of collaboration and teamwork in fighting ALS as working together is the only way to find a cause and cure for this devastating disease.

Dr. Patterson introduced our 2016 Season of Hope ambassador, Dr. Jeffrey Kramer, to speak about his experience with ALS. The speech was marked by Dr. Kramer's quiet strength and dignity in dealing with the disease. It took a great deal of courage to speak about such sensitive issues in front of a room full of people. We thank him again for sharing his story. 

Remarks from Dr. Ausim Azizi, Professor and Chair of the Department of Neurology at Temple University Hospital and Marissa Lozano, Associate Director of Family Support and Clinical Care from the Muscular Dystrophy Association followed. Collaboration and teamwork were the predominant themes; we appreciate their partnership and thank them for their generosity and dedication.

We cannot thank our guests enough for making an effort to attend this year's Gala. It gives us strength to know we have such passionate and thoughtful individuals supporting our PALS and ALS Hope Foundation. Together, we are keeping Hope on the Horizon!

Photos from the event can be viewed here. Keep an eye on our website for information about next year's event!

Dr terry heiman-patterson (left) poses with dr. jeffrey Kramer, 2016 season of hope ambassador.

Dr terry heiman-patterson (left) poses with dr. jeffrey Kramer, 2016 season of hope ambassador.

The 27th International Symposium on ALS/MND

The 27th International Symposium on ALS/MND was held in Dublin, Ireland this year.  Terry Heiman-Patterson, MD and Sara Feldman, DPT, ATP, spent the week with approximately 1200 scientists, clinicians, association members and PALS/CALS attending meetings, sessions, posters and presentations. This symposium is put on every year by the MND Association of England, Wales and Northern Ireland and was hosted this year by the Irish MND Association.

The week of meetings began December 4, 2016 when Chairwoman Carol Birks (MND Australia) opened the International Alliance of ALS/MND Associations meeting. The Alliance brings together ALS/MND organizations from around the globe.  The ALS Hope Foundation has been a member of this international group of ALS/MND organizations since about 2001. We are pleased to announce that Sara Feldman was re-elected to the Board of Directors for another three-year term. Follow this link to the Alliance website to read more about the meeting and the Alliance. 

The Ask the Experts event is held the second day of the meetings. This year’s panel included Dr. Jonathan Glass (Emory), Dr. Jeremy Shefner (Barrows) and Dr. Jan H. Veldink (Utrecht). Click here for the archived session.  Thanks to the ALS Association, this event was lived streamed so people unable to attend the conference were able to participate in the discussion.

This year, during the second day, the final judging of the Prize4Life and ALS Associations AT Challenge took place. Terry Heiman-Patterson was honored to be a member of the judging panel for this prize.  Click here to find out more about this challenge and find out who won!

Next, the Allied Professionals Forum was held on December 6, 2016. This is a day-long session developed for Allied Health Professionals and the ALS Hope Foundation is proud to be a sponsor again this year. Sara Feldman is co-chair along with Steve Bell (MND Association).    The presentations from the APF can be found here.

Last, but not least, the 27th International Symposium on ALS/MND took place on December 7-9, 2016. Sally Light (MND Association) opened the Symposium and IMNDA welcomed everyone in attendance. Dr. Heiman-Patterson was a member of the Scientific Review Board for this year’s conference and was on the abstract selection committee; Sara Feldman was on the judging panel for the clinical poster prize.

Dr. Heiman-Patterson presented 2 posters, “Evaluation of an ALS Staging System in a US Population,” and Understanding the Use of Noninvasive Ventilation in the Treatment of ALS: Results of an International Physician Survey.” Sara Feldman, PT, DPT, ATP, presented a poster with Peggy Allred, PT, DPT (Cedars-Sinai) on “Common Powered Mobility Components for the ALS/MND Population.”

Dr. Heiman-Patterson chaired the final session of the Symposium with Kevin Talbot (UK).

Next year, the 28th International Symposium on ALS/ MND will be held in Boston, Massachusetts and the ALS Hope Foundation will be co-hosting with ALS Therapy Development Institute.

The meetings were stimulating and inspiring; motivating and thought-provoking; encouraging and promising. Scientists, clinicians, associations, PALS, CALS, everyone was present and everyone participated in the conversation. We know that for PALS, time is limited and precious and we all feel the urgency. Know that at any given moment, someone, somewhere around the globe is working on better treatment, improved care, and to find a cure for ALS/MND. 


The Temple University MDA/ALS Center of Hope


Things are changing for the MDA/ALS Center of Hope! 

We are pleased to announce that on December 1, 2016 we will be moving to Temple University at the Lewis Katz School of Medicine. Temple University is committed to helping us to continue to provide the best care possible while raising the bar even higher.  To this end, Temple is helping to provide additional breadth to our program with clinic pulmonary services, genetic services, case management and counseling, and wellness programs to supplement our state of the art care. Of course we will continue to have physical and occupational therapy, speech therapy, nutrition, respiratory therapy, nursing, and DME. 

Our MDA colleagues and the ALS Hope Foundation staff will also be there to help support our clinic.  Everyone at Temple, as well as our team members, are excited about the move as we are certain it will enhance our ability to improve the care that we are able to deliver while continuing the friendly and warm feeling that we try to create during clinic visits. The MDA/ALS Center of Hope will be the first step in the development of a Neurodegenerative Disease Institute and our new partners at Temple are engaged and working hard to make it a success. We are looking forward to welcoming you to our new facility and will rely on your input to make our center jump to the next level.

The Temple University MDA/ALS Center of Hope will be located at:
Boyer Pavilion, 5th Floor
3509 N Broad St
Philadelphia, PA 19140
Convenient Valet Parking is available at the lobby of the Boyer Pavilion. 

The appointment number will be 215-707-3040


The discovery of two new ALS genes


Everyone is excited about the recent news that two new genes, NEK1 and C21orf2, were identified as susceptibility genes in ALS. This news adds to the growing sentiment among researchers that our genetic make-up plays an important role in who is at risk to develop ALS and that implicated genes can provide clues about what causes ALS as well as provide targets for treatment.

Familial ALS (FALS) cases comprise between 5-10% of all ALS and is generally a dominantly inherited disease, meaning that one of the individual’s parents passed on the abnormal gene. Remember that a gene contains the code for producing a particular protein and that the code is in the form of a series or sequence of building blocks called nucleotides.  A change in that sequence is called a polymorphism and some changes are not deleterious while other changes result in a damaged protein and are termed mutations.

Almost 20 % of people with FALS have a mutation in the gene that codes for the protein Cu/Zn superoxide dismutase located on chromosome 21. To date scientists have now identified more than 30 different genes that when mutated can either cause or increase the risk of developing ALS. In fact, the most common genetic cause of ALS is the abnormal extra chromosomal material in the C9orf72 gene, called a hexanucleotide repeat. This abnormality has been found not only in 23% of families with ALS but also in 5-7% of people with ALS and no family history -i.e. people with sporadic ALS.

NEK1 is the latest gene identified to show a specific variation from the normal sequence in upwards of 3% of people living with ALS. What is most exciting is that this abnormality was identified through a world-wide collaboration of scientists collecting genetic material (DNA) and sequencing the entire genome (all of the genetic material from each individual) from more than 1000 familial cases of ALS. Additional examination of more than 13,000 sporadic ALS individuals also revealed an increased percentage of people with the same specific variation in the NEK1 sequence when compared to normal controls.   This variation in the sequence would result in a possible loss of function in the NEK1 gene. NEK1 plays many roles in the cell, some of which have been implicated as a cause of neuronal damage in ALS including a role in the cytoskeleton which is important to transport in the nerve cells, regulation of membranes of mitochondria important in energy production, and a role in DNA repair.

It is clear that as we identify more and more of these variations in “susceptibility” genes, we will gain a better understanding about the pathways that are important for disease. Even more importantly we may be able to use these variations to stratify populations for individualizing therapies directed at particular pathways implicated by the genetic makeup of individuals with either sporadic or familial ALS. 

Read more about the newly discovered NEK1 and C21orf2 FALS genes from ProjectMinE, and find out more about this initiative started by PALS!        https://www.projectmine.com/news/