A Patient Advocate and Doctor’s Perspective on Clinical Trials: Update on the Retigabine Phase II Trial

Last week, Dr. Brian Wainger of Massachusetts General Hospital and Stephen Winthrop, Chairman of The ALS Association Board of Trustees, gave their unique clinical trial perspectives during the Northeast ALS Consortium (NEALS) webinar titled, “Retigabine Clinical Trial Update & Discussion with ALS Patient Advocate Stephen Winthrop.” For many years, The ALS Association has proudly partnered with NEALS to run ALS centered webinars to disseminate the most up-to-date information to the ALS community. Today’s post discusses the actively enrolling Retigabine clinical trial, which The ALS Association funds, as well as an honest perspective on what it is like to participate in ALS clinical trials.

 

Patient advocate and a championed NEALS Research Ambassador, Stephen Winthrop (pictured LEFT), joined the discussion to give his thoughtful perspective of clinical trial participation. Stephen was diagnosed with ALS almost four years ago and has been involved in over 12 ALS clinical trials – both observational (does not test a drug) and interventional (tests a drug), including the Retigabine study. The decision to participate in a clinical trial is a complicated question with arguments for and against. Stephen provides honest insights on the pluses and minuses of clinical trial involvement, while giving real life examples of some of his experiences.

“The quality and the experience will vary depending on the test and the individuals you are working with. It is a big question of fit,” Stephen explains.

He goes on to state, “The only way we are going to beat this awful disease is by finding a cure and one small way I can do that is by participating in clinical trials. Yes, it involves a needle stick and yes it takes a little time out of your day, but it is worth it. You are helping.”

Dr. Wainger (pictured RIGHT), one of the Principle Investigators of the Retigabine phase II clinical trial, gave a brief trial overview and update. The Retigabine trial is a double-blinded, placebo-controlled study to test the drug as a potential treatment for people living with ALS. The trial is actively recruiting at 12 sites in the U.S. The primary goals are to measure the effects of Retigabine on upper and lower motor neurons (i.e. the cells that die in ALS) physiology in people with ALS and evaluate safety outcomes.

During the first part of the webinar, Dr. Wainger explains the trial clinical rationale and goes over the study in detail including the goals, inclusion/exclusion criteria, the study timeline and what the study requires from its participants. The goal is to enroll at least 30 more ALS patients into the trial as soon as possible.

Watch the webinar here for all the detailed information.

The trial focuses on hyperexcitability of motor neurons. It was previously shown that people living with ALS have motor neurons (both upper and lower) that fire too many signals, meaning they are hyperexcitable. Too much firing leads to motor neuron damage. Retigabine is designed to reduce the over firing of motor neurons.

To physiologically test motor neuron hyperexcitability in trial participants in real time, the investigators use techniques called transcranial magnetic stimulation (TMS) and nerve conduction studies. These specialized tests are a way to measure the connections between motor neurons and muscles. TMS works by stimulating the motor cortex (part of the brain that controls muscle movement) with a magnet and records the response of the muscles in the hand. Nerve conduction studies evaluate the ability of motor neurons to conduct signals to muscles. An important secondary outcome of this study is to determine the potential for the use of these techniques for future ALS trials.

Stephen explains, “What they were trying to do using TMS, which looks like a ping pong paddle held above my head, was to try to make my right thumb to twitch by increasing the magnetic field. No pain was involved to see if my thumb twitched or did not twitch.”

One unique aspect of the Retigabine clinical trial to highlight is that each trial participant will donate a blood sample to be made into induced pluripotent stem cells (iPSCs). These iPSCs are then made into motor neurons in a dish, which reflect the exact genetic makeup of the person they were derived from.

The effects of Retigabine on patient derived motor neurons will also be tracked and compared to the impact of the drug on the patient. This is the first ALS clinical trial to attempt this type of comparison, which has potential as a prognostic and diagnostic tool. Patient derived iPSCs could even possibly predict how a subgroup of ALS patients respond to a drug, which would improve clinical trial design and recruitment.

Stephen adds, “There are so many aspects of this study that is adding to the knowledge base that Brian and his colleagues around the country and around the world are using that plant seeds for clinical successes in the future.”

“One of the things I have said about participating in a clinical trial is that when you are in a room with someone with ALS, do not forget about the human dimension of what you are looking at here. Don’t forget to say to a potential participant, ‘I am sorry that you have been struck by this awful disease,’ and just let that pause. Don’t forget to say, ‘Thank you or thanks for your small part.’”

“In my own experience, those seemingly routine personal touches go a long way, because in the end I do think their decision to participate is fueled in part by just a little whisper of a hope that this will maybe help me. The bigger piece is that you, as a participant, are part of an army of people – I truly believe – will bring an end to this disease.”

We are thankful to Stephen for giving his honest perspectives and we value his dedication to the fight against ALS. With passionate, committed physicians, researchers, clinic staff, allied professionals and especially clinical trial participants – both living with ALS and healthy – all working together in clinical trials, we are many steps closer to a cure.

Watch the full webinar here.

For more information about the Retigabine trial visit the NEALS trial siteand clinicaltrials.gov #NCT02450552.

Q&A with Dr. Javier Jara – Novel Neuroinflammation Study Published

Dr Javier Jara 2The ALS Association sat down with Dr. Javier Jara, Research Assistant Professor at Northwestern University Feinberg School of Medicine, who just published groundbreaking work focused on brain inflammation caused by ALS. This work was published in the July issue of the Journal of Neuroinflammation. The ALS Association has proudly supported Dr. Jara since 2010 through both our Milton Safenowitz Postdoctoral Fellowship Program and a recent Investigator-Initiated Grant.

Q: Thank you for joining us today! We were happy to hear the great news that your paper was just published. It is always rewarding to hear of our scientists’ successes. Congratulations! First, our readers and I would love to know why you love working in ALS research.

A: ALS is a very complex disease in which several cellular systems are disturbed. This allows me to tackle the disease from different angles, which could be a more efficient strategy to understand disease pathways.

Q: What are the major findings of your paper?

A: This paper sought to understand the role of inflammation, especially in the motor cortex of ALS (i.e. part of the brain responsible for muscle movement). We were able to study cells that are involved in immune response in the spinal cord and brain using green and red fluorescent protein tags. These cells were increased in numbers early in the disease and we were able to observe them in the vicinity of dying upper motor neurons in the motor cortex. Our observations in human ALS motor cortex also correlated with an increase of activated cells that participate in the immune response, which is important. Our studies are novel and bring a new perspective to the role of the immune response in ALS motor cortex pathology.

“This work would not have been possible without the support of The ALS Association through the Milton Safenowitz Postdoctoral Fellowship Program. Obtaining this fellowship changed my career and I am extremely grateful for the donor’s support. I encourage donors to continue their hard work to support patient care and research.” – Dr. Jara

Q: What is the significance of your research?

A: By developing a novel ALS model to investigate inflammation, we were able to set up a strong foundation for future studies to understand the role of cells involved in the immune response. Because these cells are labeled with a fluorescent tag, we can visualize and isolate them from the brain and spinal cord at different stages of disease initiation and progression. We can also use various models that develop ALS due to different underlying causes.

Q: What are your next steps?

A: We are currently investigating the secreted factors and proteins that are increased during disease in these immune cells with the hope to establish novel molecular markers and identify therapeutic pathways.

Javier Jara_option2

Q: When we first funded your research, you were a Postdoctoral Fellow. Since then you have been promoted to Research Assistant Professor, which is excellent! What are your future career goals?

A: I would like to set up my own line of research in the near future and for this purpose I would like to obtain an independent faculty position. In 2015, I was funded by The ALS Association to set up an independent line of investigation from Dr. Ozdinler’s lab to understand the relationship between brain injury and ALS. With The Association grant and Dr. Ozdinler’s support, I was able to investigate what happens to upper motor neurons after a single mild cortical injury insult. These studies have been fruitful and I am currently writing a manuscript.

Q: What do you enjoy doing outside the lab?

A: I am a runner and I enjoy running marathons. I have done five marathons so far, and I am always looking forward every year to start a new running season!

That is impressive! Thank you for joining us today. We are looking forward to hearing more great things out of your lab and to read your next manuscript!

Read more about Dr. Jara here and here.

Paper citation:

Evidence for an early innate immune response in the motor cortex of ALS.

Jara JH, Genç B, Stanford MJ, Pytel P, Roos RP, Weintraub S, Mesulam MM, Bigio EH, Miller RJ, Özdinler PH.

J Neuroinflammation. 2017 Jun 26;14(1):129. doi: 10.1186/s12974-017-0896-4.

PMID:28651542

Read free article here.

7 Climbers Tackled Mount Kilimanjaro to Raise Awareness for ALS

Kilimanjaro Graphic_AFTER CLIMB-01Nirali Shah recently returned from the journey of a lifetime, climbing Mount Kilimanjaro.

“It was definitely an adventure to say the least a lifetime experience it was more challenging than I had expected,” Nirali said. “Every day was longer than we had thought a bit more grueling.”

Nirali put together an international team to climb the world’s tallest free standing mountain in honor of her mother Sonal Shah, who passed away from ALS. The goal was twofold, to raise awareness for ALS and to raise funds for the ALS Association Greater New York Chapter. Nirali said when the climb got tough she just thought about her mom and everything she went through since her ALS diagnosis.

“It was really all about my mom, I would think about my mom, and all the ALS patients, it was a physical activity and it’s about pushing yourself to your limit. There were times I felt I couldn’t move and I thought if I feel this way how do ALS patients feel everyday dealing with their challenges, because this is nothing in comparison to the fight ALS patients’ face after their diagnosis. It really put things in perspective,” Nirali said.

5 out of the 7 climbers made it to the peak. Nirali came just shy of the top of the mountain when she got sick from the lack of oxygen.

“I got really close to the top I was about 100 meters short, I got really sick and was vomiting and fainting,” Nirali said. “I really thought about my mom at that moment near the top, all I could hear was my mom telling me to listen to my body and not be stupid.”

Nirali said she feels like this climb has made a difference.

“I think it really got people interested. I noticed even if the donations were small a much larger number of people stepping up to donate. People I followed up with were amazing and it unifies people around a cause. It’s just the way of grabbing attention and it’s what we need to create awareness,” Nirali said.

Nirali set a goal to raise $19,341, the height of Mount Kilimanjaro in feet. So far she is at about 60% of that goal but there is still time to donate and help Nirali and the team meet their goal. You can donate by clicking here.

New York family makes The Walk to Defeat ALS a Bi-coastal affair

For the Abbate family the Walk to Defeat ALS is a bi-coastal family affair. Elan Swanson first formed the walk team Donna Donna Bo Bonna in honor of her mom Donna who lives in Los Angeles, and was diagnosed with ALS in 2016. But just one Walk team wasn’t enough, so Chris Abbate, Donna’s brother, decided their team would have a presence on both coasts and will participate in both the Westchester and LA Walks.

The Abbate family and members of Team Donna Donna Bo Bonna. Donna is pictured in the back row, centered
The Abbate family and members of Team Donna Donna Bo Bonna. Donna is pictured in the back row, centered

“Donna has a big family support group here on the east coast because it is where she lived for years before moving out to LA two years ago for a job opportunity and to be closer to her daughter and granddaughter,” Chris said.

Chris said that for his family, the Walks are all about spreading awareness, raising funds, and keeping Donna’s spirits up.

“We have noticed changes since her diagnosis. Her speech pattern has altered; she speaks a little slower, especially when the discussion becomes lengthy. When it comes to physical work, she does things at her own pace, but she does NOT give up or ask for help. Donna is a fighter, strong and courageous in all aspects of her life.

Chris says the challenges haven’t stopped Donna from living her life to the fullest!

“She has been traveling quite a bit. We met her in Nashville and she also recently went to Hawaii. She’s come back to NY twice, traveled to Vegas, and has a trip planned this year to Arizona,” Chris said.  “Even though the disease is having an affect on her it’s not effecting her joy or her love to live.”

The Abbate family says it’s important for others to step up and get involved now, before it hits close to home.

“Get involved because you never know when it might affect you or your family, or a close friend. If it does, try and support them as best you can. I encourage people to help by walking, and if possible, by donating,” Chris said. “Our entire family is Donna’s support team and we do it all for her.”

To find a Walk to Defeat ALS in your area, head to our website and register today for one of our 3 remaining Fall Walks!

www.ALSWALKS.org or call 1-800-672-8857

NJ Native Plans to Conquer Mount Kilimanjaro to Conquer ALS

Nirali Shah Conquers Mt Kilimanjaro to Conquer ALS

Nirali Shah has never been one to back down from a challenge, and taking on Mount Kilimanjaro, the world’s tallest mountain, is no exception. In honor of her mom, Sonal Shah, who lost her battle to ALS in 2015 Nirali put together an international team to climb to the summit to raise awareness and funds for ALS.

“My mom lived an extraordinary life, she served as a vital ALS advocate by writing a book about her life and experience with ALS called “My Life, Legacy, and ALS”.  She also travelled to Washington DC many times to join ALSA in pushing for federal legislation and funding for research, and now it is my turn to fight and do what I can to bring attention to this devastating disease,” Nirali said.

The goal is to raise $19,341 the exact height of Mount Kilimanjaro in feet. Nirali says the climb will last 7 days and she plans to reach the summit on June 25th. This is no easy task given Mount Kilimanjaro is the tallest free standing mountain in the world.

“I’ve been doing a lot of training, but I think a big component of this will be about staying strong mentally throughout the climb,” Nirali said.

Nirali knows her mom will be there in spirit giving her that extra push to make the climb.

“My mom would have loved this. Growing up my parents and I travelled quite a bit and one of the things we always did was go to the national parks and hike there. She always said she wanted to go to Tanzania, but she didn’t make it. So this climb is for her and for all of the people whose lives are cut short by an ALS diagnosis,” Shah said.

You can support Nirali’s Kilimanjaro climb by donating at https://www.crowdrise.com/niralishah5, all proceeds will go to the ALS Association Greater New York Chapter.

Greater New York Chapter Expands Patients Services with the Introduction of 2 New Support Groups

The Greater New York Chapter is proud to announce the addition of support group services in the Bronx and Queens.

The newly formed support Group in the Bronx will be held at the VA Hospital. Both Veterans and non-veterans, family, and friends are all welcome. The group will be lead by a member of the VA staff and a member of the Greater New York Chapter and will take place on the second Wednesday, monthly from 1-3 PM at the James J. Peters Medical Center at 130 W. Kingsbridge Rd, Bronx, NY 10468.

The Greater New York Chapter will also be holding the first Queens Support Group on June 21st from 6-8 PM at  SilverCrest Center for Nursing and Rehabilitation 144-45 87th Ave., Briarwood, NY 11435. Friends, family and PALS are all welcome to attend. This support group will be held every third Wednesday of the month from 6-8 PM.

Five Boroughs NYC Map with new ALS Support Groups highlighted in Bronx and Queens

“The addition of these two support groups provides a needed service that allows both PALS and family to access support in their borough, and closer to home. This will help fill a gap especially for our families in Queens. Travel is not easy for many of our PALS and their caretakers and families. We want everyone in our area to have access to the support and care they deserve,” Michelle McKenzie, Patient Services Coordinator said.

For questions regarding the new support groups you can contact Michelle McKenzie at MMcKenzie@als-ny.org or 212.720.3045.

Hospital for Special Surgery Program Expanding from Half to Full Day Clinic

The ALS Association Greater New York Chapter and the Hospital for Special Surgery are proud to announce additional clinic sessions at the Association’s Certified Treatment Center. The expansion of services will ensure that more ALS patients in our region have access to comprehensive care.

“We are excited to expand our multidisciplinary care ALS clinic to an additional day. Our clinics strive to provide high quality care, support and information for those living with ALS and their caregivers with the support of the Greater NY Chapter,” Dr. Dale Lange said.

One clinic will be reserved for new patients, the other for established patients. The current clinic is held on Wednesday afternoons. The additional HSS clinic will be held on Tuesdays at 12:30PM. For more information and/or to schedule an appointment you can call 646.797.8917 or 212.774.2361.

Resist and Persist in the Fight Against ALS

Resist and Persist T-Shirt for ALS Awareness

Resist and Persist is the mantra Jon Selikoff lives by. Whether it be to fight for a political cause — or to fight against his disease — Jon wants it to inspire people to stand up for something.

“The phrase ‘Resist and Persist’ just kept popping into my head and I felt I needed to express it visually,” Jon said.

Jon decided to use that visual expression to make T-shirts and use the proceeds for a fundraiser. Since Jon was diagnosed with ALS in 2015 he said picking the ALS Association Greater New York Chapter to donate the profits was a no brainer.

“When I was in the process of being diagnosed, we really struggled to find a doctor that was covered by my insurance. My mom reached out to the ALS Association Greater New York Chapter and was connected with Chapter nurse Kayvan Freeman, who in turn recommended Dr. Daniel MacGowan at The ALS Association Center of Excellence at Mt. Sinai Beth Israel. I’m indebted to Kayvan, because she led me to Dr. MacGowan who was finally able to give me a diagnosis in December 2015,” Jon said. “I had been seeing different neurologists all throughout 2015, so it was a very long year and to finally have it resolved was just such a relief. And I’m sure as my disease progresses, I’ll come to rely on the Association Greater New York Chapter more and more.”

Jon says he was shocked by the amount of support his fundraiser received. In part, he believes it’s because of the versatility of the phrase ‘Resist and Persist,’ which can pertain to everyone’s everyday struggles.  The fundraiser brought awareness to his fight against ALS and in the process raised more than $4,500 for the Chapter.

“The success of the fundraiser far surpassed any goals I had. I was hoping to raise at least $500, maybe $1,000. But to my surprise in the first run, I sold 133 shirts. When the shirts arrived and people started posting pictures, more people wanted them, so I opened it up again. Frankly, the support from my friends just blew me away,” Jon said.

Jon says it’s important to continue the fight to raise awareness and funds for ALS.

“As there’s no cure and barely any effective treatments, clearly not enough is being done. I have found that not everyone even knows what ALS is. Sometimes Lou Gehrig’s Disease clues them in, but not always. It’s a challenge because it’s not lung cancer. It’s not a common disease and it’s not something you can just say, “Don’t do this and you won’t get ALS.” I wish I had the answer,” Jon said.

But he believes if people continue to ‘Resist and Persist’ in the fight against ALS there will be a day when there is an effective treatment and a cure.

If you are interested in starting your own fundraiser to help fight ALS, click here or contact Vivian Jung at 212.720.3043 or vjung@als-ny.org.

Emily Moles Tackles New Role as President of the Young Professionals Group

Emily Moles is all too familiar with the devastating effects of ALS. Emily’s mom, Bethanne Moles was diagnosed with ALS in 2010. Three years ago Emily decided to join the ALS Association Greater New York Chapter Young Professionals Group (YPG), and was recently elected YPG President.

“I wanted to get involved, because living out of state, away from my mom, I wanted to find a way to continue to help out,” Emily said. “My mom is part of a really great family and has so much support around her, but not everybody has that. Why wouldn’t you want to help if you have the time and resources?”

Emily said it’s important to recognize that while strides have been made there is still a long way to go when it comes to both awareness and ultimately finding a cure.

“In terms of awareness there has obviously been a lot of progress since my mom was diagnosed,” Emily said. “With ALS it’s still all about making someone as comfortable as possible, that’s all you can do. It’s heartbreaking because it’s such a progressive disease and the decline is hard because people are all there mentally, and to be fully aware that you are deteriorating is terrifying.”

Which is why Emily has lofty goals for the YPG this year, including increasing membership, fundraising, and event attendance.

“I want to raise a ton of money, I want to fundraise because money is what got us the research discoveries, and in the end money is what helps patients. There is very little that I can do in terms of finding a cure, but I can definitely sponsor people to do so,” Emily said. “I also want more patient outreach by our members and volunteers. People with families who live out of state need us, and it’s important to show our core community that we really care.”

If you are interested in joining the YPG, their next meeting is on May 25th at the ALS Greater New York Chapter office at 42 Broadway Suite 1724. For more information or to be added to their mailing list for meetings and fundraising events, you can email YPG@als-ny.org or call Brett Murphy at 212.720.3052.

“Team We Are Carol Moeller” Takes on the 2017 Long Island Marathon to Help Fight ALS

We Are Carol ImageIn an amazing demonstration of strength and courage, three months after her ALS diagnosis Carol Moeller finished her final 10k race at the 2016 Long Island Marathon.

“She couldn’t speak, was having trouble walking, couldn’t swallow, and she delayed her feeding tube operation so she could compete in the 10k race. She did it and she didn’t give up. She was a week away from having a feeding tube inserted into her body, but still pushed herself into completing a 10k,” Carol’s son, Kevin Moeller said.

Carol’s other son Dennis Moeller added, “Mom was in the middle and we surrounded her like secret service. She fell twice during the race.  A mile before the finish line, we helped her up and my brother and I each had an arm around her.  We told her to slow down and she just moaned the word, RUN! She finished the race and it was really special.”

This year the Moeller family will be running the Long Island Marathon, on May 7th, in honor of their mother Carol who passed away last October.

“I know it would make her feel good that I’ve been training the way she always did. We owe it to her. We saw her run with very limited abilities and I said if my mom can run 6 miles while already losing her ability to smile, stand up straight, or to cry, there is no way I can’t run the 26 miles. There is no quit in any of us,” Dennis said.

Running was always a part of Carol’s life, so the tribute is more than fitting.

“She would wake up at 4:30 in the morning and run 6 to 10 miles, 6 days a week, and be home in time to make breakfast, get us to school, and go to work. That was her routine for 30 years,” Dennis said. “Running is the only way to honor her, in the way she truly deserves.”

The Moeller family is hoping this run will bring with it support, because their goal now is to raise awareness, and bring in funds for research. So far “Team We Are Carol Moeller” is closing in on its $20,000 dollar initial goal.

“My mom kept asking for something to help her, some magic pill that would take this all away. And you know, hopefully through these efforts we are going to help someone, somewhere, come up with that magic pill.” Kevin said.

But until that day the Moeller family says they will continue to run for ALS and continue to fight for a cure. Dennis said when he needs motivation to keep on going he just hears the words his son told him during one of his training sessions, “Daddy, I think Grandma is cheering for you from the sky.”

If you would like to show your support and donate to Team We Are Carol Moeller head to tinyurl.com/carolmoeller