Hss summer 2015 neuromuscular newsletter

Neurology HOSPITAL FOR SPECIAL SURGERY

Neuromuscular News Volume 1, Issue III

July 2015

Letter from the Director

IN THIS ISSUE

Dear Friends,

Special points of interest:

On behalf of our entire neuromuscular team, I are proud to bring you another edition of our newsletter.

● Patient spotlight!

● Clinician Tips

● Research Updates

● Annual Research

This summer is full of new projects and clinical research trials in our Department. We have a number of investigator initiated and pharmaceutical supported trials targeting various Neuromuscular diseases starting. We aim to review each of these trials in this and our following edition, and my staff and I are available to answer any questions you have regarding our work.

Please also enjoy selected photos from our annual gala. It was a fun filled night held to support our neuromuscular research program at Hospital For Special Surgery.

Thank you to all of you who made this possible with your support. Plans for next year’s event have already started.

I hope you will join us.

As always, our mission at HSS is to provide the highest quality of care and improve the quality of life for all our patients while advancing the science of understanding and treating patients with ALS and other disorders of nerve and muscle.

Benefit Dinner

Getting around town...

Wheelchair-Accessible Yellow Taxi Service The New York City Taxi and Limousine Commission manages a program that provides wheelchairaccessible yellow taxi dispatching services.

Wheelchair-accessible yellow taxis will respond to trips originating in Manhattan and ending anywhere in the five boroughs, Westchester and Nassau counties, and the three regional airports.

Passengers pay the normal metered taxi fare in New York City from the point of pick-up to destination.

There are no extra costs to passengers.

Dale J. Lange, M.D. Director, ALS program at Hospital for Special Surgery

There are five ways to request a New York City wheelchair-accessible taxi: ● Call 311 ● Call the dispatch center directly: (646) 599-9999 ● Text a request to: (646) 400-0789 ● Use the mobile app “Wheels on ● Wheels” (WOW), powered by Taxi Magic and available free at the Apple iTunes Store ● Order online at: www.nycaccessibledispatch.org

Share your story!

How to Contact Us:

We are grateful for your continued support and hope you enjoy our newsletter. As always, comments and ideas for content are welcomed.

If interested in writing in our patient blog, sharing your experiences and thoughts, please contact Mona or Shara.

Mona: Shahbazim@hss.edu Shara: HolzbergS@hss.edu Lindsay: KaplanL@hss.edu Lauren: LangfordL@hss.edu

Neuromuscular Research Annual Benefit Mona Shahbazi, NP On May 15th, Department of Neurology held its annual fundraising benefit at The Plaza in New York City.

It was a night full of music, food and dancing, with live music by Don Felder of the Eagles and exciting live and silent auction items! Joe Piscopo started the night with a rendition of New York, New York to a room of nearly 250 people, who had come to support our program.

Money raised from this event will help fund the Neuromuscular research Program here at HSS, providing clinical trials and best available patient care.

Thank you to those who supported us and we look forward to next year’s event!

If interested in participating next year, please contact Mona or Shara.

Healthy Breakfast in a Hurry Jessica Cording MS, RD, CDN

A balanced breakfast can set the tone for your whole day. These quick and easy recipes will make a healthy morning meal a breeze.

OVERNIGHT PB & J OATMEAL

MAKE-IT-A-MEAL CEREAL SMOOTHIE

EGG MUFFINS

This only tastes like it spent a long time simmering on the stove. It’s a great way to add a little sanity to your morning in just minutes. Mix it up the night before and enjoy hot or cold the next morning.

Smoothies make a great portable breakfast. The balance of protein from the milk and yogurt and the fiber from the fruit and cereal will keep you satisfied and energized for hours. The spinach is a great way to sneak in any veggies—you can’t even taste it!

These muffins can be thrown together quickly and make a convenient high-protein breakfast. Customize with your favorite veggies and cheese. Enjoy them right out of the oven or store extras in the fridge to warm up in the microwave when you’re ready to eat. If feeding a larger crowd, feel free to multiply the recipe.

INGREDIENTS: ● 1/3 c rolled or instant oats ● 1 tbsp ground flax ● 1 tsp cinnamon ● 1/4 tsp vanilla extract ● 1/2 c berries or ½ a banana, mashed ● 1 cup milk of choice ● 1 scoop protein powder (optional) ● 1/2 -1 tbsp peanut butter ● 1 tsp jelly DIRECTIONS: Mix oats, flax, cinnamon, protein powder vanilla, and fruit in a bowl. Add milk and stir well. Cover and allow to sit in the fridge overnight until oats have absorbed the liquid.

Enjoy cold or heat up in the microwave in 1-minutes intervals until desired temperature. Top with peanut butter and jelly.

INGREDIENTS: (serves 1) ● 6 oz plain Greek yogurt ● ½ c milk of choice ● 1 cup ice (optional) ● ½ banana, fresh/frozen ● ½ c frozen berries ● 1 tbsp cocoa powder ● ½ cup high-fiber cereal of choice ● 1 large handful baby spinach DIRECTIONS: Starting with liquid and ending with spinach, layer ingredients in blender. Cover and blend on HIGH until smooth. Pour into a glass or to-go cup and enjoy!

Notes: If using non-dairy milk and/or yogurt, adding protein powder will ensure you’re getting enough. add nut butter, dry milk powder, or additional milk or yogurt if desired. Try swapping another fruit in place of the berries and banana.

INGREDIENTS: ● 6 eggs or 1.25 cups liquid egg whites ● 1.5 cups chopped veggies (such as spinach, broccoli, or peppers) ● salt & pepper to taste ● 2 tbsp cheese (such as grated cheddar or goat cheese crumbles) DIRECTIONS: Preheat oven to 350 degrees F. Grease a 6-muffin tin.

Whisk together eggs/whites, spices, and cheese.

Divide egg mixture amongst muffin cups. Add vegetables.

Bake 15-20 minutes or until set.

(Makes 6 muffins)

What’s the Buzz on Research We have four very exciting new studies that we will be recruiting for over the next few months!

Phase 2 Pharmacodynamic Study of Ezogabine on Neuronal Excitability in Amyotrophic Lateral Sclerosis Nickname:

Retigabine

Study Drug: Ezogabine Study Design:

Phase II multicenter, randomized, double-blind, placebo-controlled, 14-week study.

Study Population:

PALS who are 18 years or older, with a Slow Vital Capacity (SVC) score greater than or equal to 60%.

Purpose: Past research has shown that the motor neurons of ALS patients may be producing more

electrical activity than motor neurons of people without ALS and that this extra electrical activity tires the neurons and contributes to their death. We are doing this research study to find out whether the drug ezogabine will lower motor neuron activity in people with ALS. During this study we will also find out whether the drug is tolerable and safe for patients with ALS.

Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group Study to Evaluate the Safety, Tolerability and Effect of Tirasemtiv in Patients with Amyotrophic Lateral Sclerosis Nickname:

Cytokinetics

Study Drug: Tirasemtiv Study Design:

Phase III multi-national double-blind randomized, placebo controlled 56-week study.

Study Population:

PALS who are 18 years or older, with a Slow Vital Capacity (SVC) score greater than or equal to 70%.

Purpose: The study drug, Tirasemtiv, is being investigated as a potential new therapy for the

improvement of breathing, muscle weakness, and muscle fatigue in patients with ALS. The purpose of this study is to evaluate Tirasemtiv’s safety and effectiveness for patients with ALS.

What’s the Buzz on Research Safety of Caprylic Triglycerides in ALS: A Pilot Study Nickname: Study Drug:

Axona

AC-1204

Study Design:

This is a pilot, 16-week study.

Study Population:

ALS patients who are 18 years or older, with a Forced Vital Capacity (FVC) greater than 40%.

Purpose: The purpose of this study is to determine if a diet inclusive of caprylic triglyceride is safe and well tolerated in patients with ALS. The study hypothesis is that caprylic triglycerides will enhance mitochondrial function and cellular energy regulation and increase the presence of ketone bodies in the blood which will be able to slow or stop the progression of ALS in a dose dependent manner.

Double-blind, randomized, multicenter, placebo controlled, parallel-group study to evaluate the efficacy and safety of fingolimod 0.5 mg administered orally once daily versus placebo in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) Nickname: Study Drug:

Fingolimod or Novartis

Fingolimod 0.5 mg

Study Design:

This study is a double-blind, randomized, placebo-controlled Phase II/III, that will continue for three years of follow up.

Study Population:

The study population will consist of male and female patients 18 years and older with CIDP who are on IVIg and/or corticosteroids treatments prior to the study.

Purpose: The purpose of this study is to evaluate the efficacy and safety of fingolimod in the treatment of CIDP compared with placebo. Data from this study will be used to support the registration of fingolimod in the indication of CIDP.

Trials 101: Research Lingo Phases of Clinical Trials:

There are different phases of clinical trial development (phase I, II, and III). Phase I represents the earliest stages of clinical development. Phase III suggests that a drug has already completed earlier phase testing requirements. Typically a drug must complete a phase III trial before reaching approval by the FDA, except if statistically significant treatment in earlier phases.

Randomized:

The terms randomized or randomization suggests that the treatment groups will be assorted randomly. For example, for a Phase II study looking at different dosages of a particular drug, some people will receive the higher dose, some will receive a lower dose, and some will be assigned to placebo. And all of this is done by random assignment.

Placebo-Controlled:

Many Phase II or Phase III studies use a placebo or “sugar pill� as a control agent to make sure the study reduces bias. By blindly and randomly allocating similar patients a control group that receives a placebo, investigators can ensure that any possible placebo effect will be minimized in the final statistical analysis.

Get involved now!

Online survey studies for both patients and their caregivers!

It is our goal at HSS to not only deliver optimal care to our patients, but also to ensure that your needs, as a patient or caregiver, are being met as well. Please take our multidisciplinary clinic survey: https://cs.createsurvey.com/publish/survey?a=yIzCjK about your experiences at our clinic.

While sexual function may not be directly affected by ALS, this study was designed to determine if you believe intimacy and/or your sexual relationships are affected by a diagnosis and symptoms of ALS. Surveys will take approximately 10 minutes each. Thank you in advance for your participation. Your feedback is very important!

PALS: https://www.surveymonkey.comspatientperspectiveintimacyquestionnaire PARTNERS: https://www.surveymonkey.com/s/partnerperspectiveintimacyquestionnaire All responses to these questionnaires are kept confidential. There will be nothing identifying your responses.

Contact Lindsay, Shara or Mona with any questions/concerns

Research Patient Spotlight The DPS Clinical Trial

PATH Clinical Trial

As I enter my 317th day of the DPS Clinical Trial I’d like to reflect on why I opted for this clinical trial. My reasoning starts with how I could encourage others to participate in a clinical trial. This DPS Clinical Trial was a no brainer for me, encourage others and at the same time reap the benefits both physically and mentally. The DPS exercises one’s diaphragm. The mental aspect is encouraged by Dr. Lange’s Clinic Team. They provide constant assistance to me and the positive vibes I take away give me hope and determination to make a difference.

Before deciding to enter the Hizentra clinical trial, I had many fears and anxieties I am sure many share. I worried about losing my routine of IVIG and the hours spent with the infusion staff every two weeks, a very therapeutic event in itself. Hizentra promised ‘freedom’ to administer the drug at home, but I feared losing contact with my team, displaced from the office. I worried about what my insurance would react when I came back off trial, as they never seemed too eager about covering IVIG. I vainly worried if the subcutaneous injections would scar my already rapidly aging stomach. Most of all, what kept me and my wife up late at night talking, was the fear of relapse.

A-WAVES Clinical Trial I came to HSS after a long journey of seeking answers for my ongoing hand problems which at that time had been an issue for seven years. I had seen numerous neurologists and other medical professionals and had undergone surgery of the elbow as well which turned out not to be the solution for my seemingly inexplicable issue. I was referred to the neurology department of HSS where I met with Dr. Lange in May 2014. I remember him smilingly saying that it was obscure cases like mine that would end up at HSS which at that time was very comforting to hear.

He mentioned this research study and suggested that I take part to which I happily and hopefully consented. Two weeks after the first of two weeklong infusions I felt a newborn energy and easiness in walking which I hadn’t experienced in years since it had been so burdensome. My hope grew that I may indeed get better or at least not get worse and that there was an answer after all to my medical condition. At the conclusion of the study it was selfevident that I would continue with the treatment of IVIG. This has been going on for half a year now and I would not want to miss it for anything! Dr. Lange and his fantastic study team consisting of Lauren, Leah, Lindsay, Lorene, Mona and Shara have been my steady companions throughout this journey and have made this process a delightful and easy treatment. I can’t thank them enough!

At the time, I was living in a sort of unstable peace with my CIDP. The medication regimen was keeping me within a certain range of ‘normal’ most of the time, but occasional significant flare-ups could require weeks to fully recover. Hizentra theoretically promised to narrow the range of variations in my symptoms by providing a more stable level of immunoglobulin. The doctor in me can comprehend that and believe it fully, but the patient that I am is ever skeptical and resistant to change. What sealed my decision to participate, however, was the trust and communication I had developed with Dr. Lange and his staff over the years. I don’t mean to say this allayed my fears completely; they were as real as ever. But I was confident that at the slightest tingle of paresthesia or weakness, either Dr. Lange or the research team would be in touch within hours.

Of course, upon starting the trial, I had a mild flare up. Within days, I was in the office, we were able to manage it before it became significant, and I made a speedy recovery.

My other fears proved unfounded as well. While I no longer go in for multi hour infusions, I spend quality time at home with my wife administering my own care in our own home. Also, far from losing contact with my team, I seem to have an ever growing cadre of research and clinical staff in constant contact regarding my well being. Without a doubt, the medication I receive is effective. But, whether in a clinical trial or not, I feel it is physician patient communication that forms the cornerstone of chronic disease care.

Neurology HOSPITAL FOR SPECIAL SURGERY

Thank you for reading! Feedback is welcomed & appreciated!

Sincerely, Your Team

If you are interested in more information about our ALS Research Program, or supporting our work, please contact Dr. Dale Lange at 646-797-8917 Clinic Corner What has been the hardest part of your diagnosis so far?

“Needing help in the bathroom has been the hardest. But my home health aid was professional and it made something frustrating and uncomfortable as tolerable as could be.�

What do you think has been the hardest part?

Send us your comments...

Meet Your Clinic Team Dale J. Lange, MD, Attending Neurologist Program Director

Shara Holzberg, MS CCC-SLP Speech-Language Pathologist and Research Coordinator

Mona Shahbazi, NP, MSN Assistant Program Director

Devany Tiedeman, MS, PT Physical Therapist

Alex Shtilbans, MD, PhD Attending Neurologist

Laura Manfredo, MS, PT Physical Therapist

Dora Leung, MD Attending Neurologist

Gioia Ciani, CMT, MS, OTR/L Occupational Therapist

Lauren Langford, LCSW Social Worker Clinic Coordinator

Lorene Janowski, DPS, OTR/L, MS, C/NDT Occupational Therapist

Leah Pilossoph, RN Clinical Nurse Manager,Infusion therapy

Jessica Cording, RD, MS, RD, CDN Dietician

Lindsay Kaplan, MA Clinical Research Coordinator Neuromuscular Program at Hospital for Special Surgery Department of Neurology, Belaire Building 5th floor 525 East 71st Street, New York, NY 10021