Clinical Trials Are Vital to Improving Medical Care

Manish Gupta has over 15 years of experience in developing and executing global clinical trials in cardiac and neurological devices (including those developed to manage essential tremor). He shares how clinical trials work and how the IETF partners with Cala Health to recruit participants. 

 

By Manish Gupta
Vice President of Clinical Affairs
Cala Health, Inc.

Photo of Manish Gupta with Cala HealthClinical trials are research studies that explore whether a medical device, drug or treatment is safe and effective for humans. These studies also may show which medical approaches work best for certain illnesses, diseases, disorders or groups of patients. Clinical trials produce the most reliable data available for health care decision making. They follow strict scientific standards to protect patients and help produce dependable study results.

Clinical trials are one of the final stages of a long and careful research process. The process often begins in a laboratory, where scientists/technologists first develop and test new ideas. If an approach seems promising, the next step for higher risk devices may involve animal testing. This shows how the approach affects a living body and assesses its safety. However, an approach that works well in the lab or animals may not always work well in people. Thus, research in humans is needed.

For safety purposes, clinical trials start with small groups of patients to find out whether a new approach causes any harm. In later phases of clinical trials, researchers learn more about the new approach’s risks and benefits in larger groups of patients.

Quote from Manish Gupta for Research Month blogA clinical trial may find that a new device, drug or treatment

1) improves patient outcomes; or
2) offers no benefit; or
3) causes unexpected harm

All of these results are important because they advance medical knowledge and help improve patient care.

Patients participating in research are generally referred to as “subjects.” During a clinical trial, doctors, nurses, social workers, and other health care providers might be part of the subject’s treatment team. They will monitor the subject’s health very closely, conducting more tests and medical exams than standard care.

Taking part in a clinical trial can have many benefits. If a new treatment is proven to work, subjects are among the first to benefit.  Even if subjects don’t directly benefit from the clinical trial, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many subjects volunteer because they also want to help others.

Many government agencies, companies, patient advocacy groups and other organizations sponsor clinical trials. Collaboration between two or more of such groups/organizations is common in clinical research to create patient awareness about the clinical research and the disease. Cala Health’s collaboration with International Essential Tremor Foundation (IETF) is one great example of a partnership that creates patient awareness throughout the United States about essential tremor (ET) clinical trials. Cala Health, Inc. is actively conducting ET clinical trials of its wrist-worn therapy at leading centers in the US.

IETF is an essential partner to Cala Health informing the ET community of the clinical research opportunities to advance medical knowledge and patient care.

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About Cala Health, Inc.
Cala Health is a medical technology company pioneering a new class of electrical medicine. The company is merging innovations in neuroscience and electronics to deliver individualized, prescription neuromodulation therapies. These therapies treat chronic disease non-invasively by stimulating peripheral nerves with body-worn electronics. The company is headquartered in the San Francisco Bay Area and backed by leading investors in both healthcare and technology, including Johnson & Johnson Innovation – JJDC, Inc., Corp, Lux Capital, Lightstone Ventures, GV, dRx Capital and Action Potential Venture Capital.

The IETF funds research grants, advocates for more research on essential tremor, and works with companies like Cala Health to recruit participants for research studies. Your donations to research are the reason the IETF is able to carry out these initiatives and work toward improving the quality of life for every generation living with essential tremor. Help us keep hope alive.

 

 

 

 

 

 

 

 

 

Research for Essential Tremor Gives Hope to Young People

By Deirdre Maciak
IETF Scholarship Recipient

I was only 16 years old when I was diagnosed with essential tremor. Up until that point, I don’t think I ever really registered how young I was. I had started driving, going out with my friends without supervision, and doing all the things that are expected of teenagers finding their way in the world. It’s an incredibly careless time in one’s life—there is so much ahead of you that the lines between the past, present and future are blurred.

Photo of Deirdre Maciak, IETF Scholarship WinnerMy main goals were always so clear to me. I wanted to get through high school consistently getting better at playing saxophone, study harder and get better grades, get into nursing school, and come out at the end with my dream job. But, being told that you have a chronic condition, one that won’t go away and will probably only progress over time, will bring even a busy-minded teenager to a halt.

Suddenly I had to reevaluate everything that I wanted in life. My diaphragm was spasming too much to have a good control on my air supply while playing saxophone. My physics class only had stools, and because there was no support, I spent more time trying to control my shaking core than paying attention to the teacher. I had a lot of questions. How am I going to be steady enough to draw blood when I’m a nurse? And, why did this have to happen to me, a 16-year-old girl, before I could achieve any of my dreams?

I’m not the first or last teenager out there whose plans have been derailed in some Quote from Deirdre Maciak about the importance of ET researchcapacity due to essential tremor. But, I am part of a generation of people with the condition who have better access to experimental treatments due to research and new discoveries.

Working with my neurologist, I’ve tried one medicine so far, but the side effects were difficult, so I’m exploring other options and I expect to need some of the new innovations in my lifetime for sure. Knowing that there are options out there to help control the frustrating symptoms has helped me put everything in better perspective.

Today, I follow what’s happening with ET by reading the International Essential Tremor Foundation (IETF) website, and watch what hospitals in my area, such as Brigham and Women’s, are doing with focused ultrasound. I was also excited to learn that a family friend, who is a research scientist, recently starting working for a company that is hoping to release a new drug that would be a big step in helping people with neurological disorders including ET. They hope to know this fall if they receive their next approval – and I am optimistic that it can help me and people of all ages who are dealing with this condition.

I was accepted into nursing school and start this fall! So despite this condition, and maybe also partly because of it, I’ll give it my all with the hope of helping people in general, and possibly those who suffer specifically from lifelong conditions as I do.

I still have a lot of questions. But, the new and ongoing research for ET gives hope to us young people, that even though our conditions may worsen over time, there are also so many ways that modern medicine can help us live our lives normally and we all need to work toward that goal together.

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July is a time with the IETF draws awareness to its many research initiatives: funding research grants, advocating for more research on essential tremor,  recruiting for research studies, and more. Your generosity is the reason the IETF is able to carry out these initiatives and work toward improving the quality of life for every generation living with essential tremor. Help us keep hope alive. Donate today. 

 

 

Partnering for Successful Essential Tremor Clinical Trials

By Spyros Papapetropoulos, MD, PhD
Chief Medical Officer
Cavion, Inc.

Collaboration with the International Essential Tremor Foundation (IETF) is critical for advancing new treatment approaches for essential tremor (ET). Our company, Cavion Inc., has been engaged in the discovery and development of a new class of T-type calcium channel (Cav3) inhibitors for the treatment of neurologic diseases like essential tremor. Last fall we initiated a Phase 2 clinical trial of our lead investigational oral drug, CX-8998.

Photo of Dr. Spryos PapapetropoulosAs a small precision medicine biotechnology company, we needed to recruit for our clinical trial as rapidly as possible. Our trial, called T-CALM (Tremor-CAv3 Modulation Trial), was designed to assess whether CX-8998 decreases the severity of tremors and improves quality of life by reducing abnormal activity in certain regions of the brain. In addition to evaluating a completely new class of therapy, our trial design also incorporated state-of-the-art digital tools to objectively quantify tremor. We needed to recruit more than 90 patients to participate at 25 research centers around the U.S.

While ET is relatively common, many patients are not under the regular care of a physician for the condition and do not seek out clinical trial opportunities. In addition, ET patients often do not understand the role of clinical trials in advancing new treatments. The IETF has built a community of engaged patients across the country and is a well-established source of news and information regarding tremor. Our intention was to reach patients through a trusted channel and we turned to the IETF to partner with us in informing patients and their loved ones about our clinical trial. They featured a story about T-CALM on their website, sent emails and mailed printed flyers to patients that live in our trial site communities. The information we provided explained the value of clinical trials, the design of our trial and set expectations for what patients would experience as participants in the clinical trial.

The response to the IETF’s targeted outreach was very positive, with many patients visiting our trial website to learn about the trial and contacting the sites to inquire about participating. Thanks in part to the IETF, we were able to complete our study recruitment in time. As ET research continues, I am hopeful that the IETF will continue playing an invaluable role in educating patients and their families about clinical trial opportunities for emerging therapies targeting the treatment of essential tremor.

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July is a time with the IETF draws awareness to its many research initiatives: funding research grants, advocating for more research on essential tremor,  recruiting for research studies, and more. Your generosity is the reason the IETF is able to carry out these initiatives and work toward improving the quality of life for every generation living with essential tremor. Help us keep hope alive. Donate today. 

Comprehensive Meeting Focuses on Tremor Disorders

By Shari Finsilver
IETF Board Vice President

Calling all tremor patients … I have some great news. We are in very good hands!

I had the privilege of attending the 1st International Tremor Congress in New York City on May 11 & 12. I must say, I was totally blown away . . . by the level of research that is currently being conducted around the world, by the wide age range represented, and by the organizational excellence of the entire meeting.

The goals of the conference were:

  • Formulate an evidence-based approach to optimize the treatment for tremor disorders.
  • Develop evidence-based scientific knowledge for the future clinical study design in tremor disorders.
  • Describe the up-do-date clinical diagnosis and treatment in each tremor disorder.
  • Indicate the cutting-edge scientific discovery for tremor, and also current tremor therapy development.

Among the approximately 200 people attending this inaugural conference, there were the experts present . . . those who have created the science of tremor research, whose names grace hundreds of medical journal articles, as well as the textbooks used by the numerous students whom they have all mentored. Then there was the next generation of tremor researchers . . . those who are conducting novel, original studies, leading the way with their brilliant ideas. Also present were students including students in the fields of neurology, movement disorders, public health, etc. They were there to learn, collaborate, and be challenged to continue this very important work. Since much of the successful research is a collaboration between academia and industry, many of those industry representatives were also present, ready to learn with the rest of us.

The first meeting day was devoted to science. This included research projects involving the circuitry of the brain, a focus on Purkinje cells, on climbing fiber synapses, and on neuroimaging, just to name a few areas of interest. The second day focused on the current and emerging therapies, ranging from medications under development for tremor control, to surgical interventions (both invasive and non-invasive,) to wearable devices that can improve tremor control.

The IETF was proud to be one of the many sponsors of this first Tremor Congress. A huge thank-you goes out to the course directors and their committee:  Sheng-Han Kuo, M.D. (Assistant Professor of Neurology, Columbia University), Elan D. Louis, M.D., M.S. (Professor of Neurology and of Epidemiology: Chief, Division of Movement Disorders, Yale University), and Ming-Kai Pan, M.D., Ph.D. (Assistant Professor of Medical Research, National Taiwan University). And, also to the faculty, whose presentations were outstanding.

Raising Awareness About ET and the IETF

Patrick McCartney
Executive Director

I recently attended the 70th American Academy of Neurology (AAN) Conference in Los Angeles, CA along with our board president Dr. Kelly Lyons. This annual conference offered more than 300 top-quality programs covering the spectrum of neurology; exciting networking opportunities; and expanded Experiential Learning Areas that allow attendees to interact, explore, and learn outside of the classroom. This year’s announced attendance was more than 14,000.

AAN offers non-profits free booth space and the IETF has exhibited at the conference for several years. This is a great opportunity for us to raise awareness and educate doctors, medical students, and pharmaceutical and medical device companies about the resources available on essential tremor from the IETF.

I’m always amazed at how many doctors and other exhibitors I talk with at these conference who either have ET or have family members or friends with ET. Talking to these people affirms why we attend these type of conferences because most of them have never heard of the IETF and they’re excited to hear there is a reliable source for accurate and objective information on ET.

Making Connections 
This conference also gave me a chance to catch up with several of our IETF Medical Advisory Board members. I enjoyed seeing Dr. Elan Louis from Yale University School of Medicine, Dr. Mark Hallett, a senior investigator with NINDS and Dr. Keith Coffman from Children’s Mercy Hospital right here in our backyard in Kansas City.

I also had the opportunity to meet with some of our partners who work (or are working on) a variety of ET specific medications or medical devices for the ET community. I was able to spend time with folks from Liftware, Abbott, Cavion Pharmaceutical, Boston Scientific and Medtronic. I also met with one of our newest partners the Alliance for Patient Access.

Another Advocacy Opportunity
In October, we will attend the American Academy of Family Physicians Annual Meeting in New Orleans. We think it’s important to attend this meeting to raise awareness with family physicians who quite often are the first doctors to diagnose ET in many patients. Again, almost all the doctors I talk with at this meeting see ET patients, but have no idea the IETF exists.

I know sometimes it seems like there is nothing new on the horizon to help ET patients. After attending AAN I’m excited about the new projects being worked on including ET specific medications, improvements in DBS and Focused Ultrasound treatments, and new medical devices that will help improve the quality of life for ET patients around the world.

ET Will Not Prevent Me from Achieving My Goals

Each semester, the International Essential Tremor Foundation presents four college scholarships to students with essential tremor. The scholarships represent hope for the future, and provide support to these students during a pivotal time in their lives. As part of the scholarship application process, each applicant is asked to write an essay that answers the question, “How has essential tremor affected my life?” The following essay is from one of our spring 2018 scholarship recipients.

By Kelley Cordeiro
Doctoral Student at Molloy College
Rockville Centre, NY

How has essential tremor affected my life? I could answer this question by describing the challenges it creates trying to control my trembling hands with even the smallest tasks, like threading a needle, counting out change, applying makeup, typing, or measuring ingredients, just to name a few. I could talk about how much I notice my tremor at the end of a long day, when I try to unwind by reading a book or watching television, but I have to concentrate on trying to keep my head still. This becomes even more difficult when my head trembles on my pillow at night, making relaxation and sleep a conscious effort, and often an elusive goal.

I might answer that having ET has caused me to answer questions for my children about why I am shaking, why grandma shakes too, or the most difficult question, “will I shake like that when I grow up?”

There are so many possible ways to answer the question of how ET has affected my life. My favorite answer is that is has NOT affected my life. I am the mother of three wonderful children. I chose a mid-life career change to afford me the opportunity to be a stay-at-home mom, but also involved returning to school at an advanced age. I completed my master’s degree in a new field with a 4.0 GPA. I am now pursuing my doctorate degree in the field of education, with the goal of being an agent of change in diverse learning communities.

I take on extra teaching opportunities to help cover the costs of my tuition, which increases my stress level, which increases my tremor, but which is not an excuse I am willing to let stand in my way, or prevent me from achieving my goals.

Will my children develop a tremor as they grow up? What is the best way to answer this question for my children? Research indicates that there is a strong chance that they may develop this familial condition. So, when I respond to my children I want to give them an honest answer, supported by the evidence of my example: Yes, you may develop essential tremor, but it doesn’t have to affect your life!

I want to thank the IETF for the opportunity to be considered for a scholarship, which can help me achieve my educational goals. I would like to say that ET has not affected my life, but the IETF has!

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The deadline for fall 2018 scholarship applications is May 1, 2018. Application information is available on the IETF website. Interested in supporting students with ET during their educational journey? You can donate online.

 

Are Essential Tremor and Parkinson’s Connected?

It’s a common misdiagnosis. Someone notices a tremor in their hands or body and automatically they think they have Parkinson’s disease. Some have said their doctor prescribed a Parkinson’s disease medication for them until they discovered the correct diagnosis was essential tremor (ET). And sometimes a person is diagnosed with ET when they are actually in the early stages of Parkinson’s.

Parkinson’s disease and ET will always be connected because of their similar physical symptoms. There is more public awareness about Parkinson’s, but ET is eight times more common. At the International Essential Tremor Foundation, we hear from individuals who have both conditions.

So what are the differences? How can you learn more? One way is to take part in the IETF’s upcoming FREE teleconference, “ET vs. Parkinson’s: How Do They Differ?” Mark your calendar to join us:

WEDNESDAY, APRIL 18, 2018
12 p.m. pacific time
1 p.m. mountain time
2 p.m. central time
3 p.m. eastern time

Teleconference Speaker
Dr. Holly Shill

Dr. Holly Shill, chair of the IETF medical advisory board, will be the teleconference speaker. Dr. Shill is the director of the Muhammad Ali Parkinson Center at Barrow Neurological Institute in Phoenix. Her expertise includes the diagnosis and treatment of involuntary movements, essential tremor, Parkinson’s disease, Huntington’s disease, dystonia, and ataxia. IETF Executive Director Patrick McCartney will be the teleconference moderator.

How Can You Participate in the Teleconference?
If you’ve never participated in a teleconference before, don’t worry, it’s easy! The entire conference is conducted over the phone; no internet or computer is required. You can listen in by yourself, or host a teleconference party in your home and put us on speaker phone. (Then you can have your own group discussion afterward!)

But first, give us a call, 1-888-387-3667, or go online to register. Reservations are required because our capacity for callers is limited. When you register, you’ll receive a call in number and conference code. And, you’ll have the opportunity to give us a question or two that you would like to have answered during the teleconference. Here are a few questions that have already been submitted:

  • Is Parkinson’s more in the feet and ET in the hands?
  • Does ET and/or Parkinson’s affect your memory?
  • What percentage of people with ET develop Parkinson’s?

Our Past Teleconferences Are Online
The IETF conducts several teleconferences each year as part of its educational offering to people with essential tremor. All teleconferences are recorded and posted on our website so anyone can log on anytime to listen to past ones.

We also host educational forums several times a year in cities throughout the U.S. Our next forum is scheduled for June 23, 2018 in Minneapolis. You can read more about this online.

The IETF’s goal is to provide hope to the essential tremor community through awareness, education, support, and research. These teleconferences are one way we carry out the “education” part of our mission.

We know that by providing educational programs, we can continue to communicate the latest information about essential tremor. Join us on April 18!

ET Has Affected 5 Generations in My Family

By Shari Finsilver,
VP of the IETF Board of Directors

Increasing awareness about essential tremor (ET) has been my passion for almost 20 years.  But, I must confess … I was not very open about my tremors prior to this. In fact, I did everything imaginable to hide them, even from my parents!

My courage came from all the members of my support group. It is often said that when we volunteer, we get much more back than we give. Well, that was so true in my case when I decided to start a support group in Michigan in 2000. I marveled at the candor, courage, and self-pride in many of those members and, eventually, decided to follow their lead to become more open about my own ET.

Joining the IETF Board
When I was invited to join the IETF Board of Directors in 2001, I found another platform that I could use to further raise awareness, while also getting involved in education programs, fundraising, and research. I am committed to expanding the work of the IETF, the most recognized organization helping ET patients and families.

ET began challenging my life at 11 years old. I’ve heard it said that those of us with ET are much more intelligent and creative. Well, I’m not sure I would go so far as to say that … but I did become very creative in developing ways to cope with these troubling tremors! When the IETF compiled a list of coping tips, I was able to share all of mine with all of you.

The Choice to Have DBS Surgery
After having lived with increasingly challenging ET for almost 40 years, I decided to have deep-brain stimulation (DBS) surgery in 1999. DBS changed my life.

ET has, so far as I know, affected five generations in my family. I will continue to do everything in my power to help those of us with ET get more effective medical treatments, increase awareness so the public doesn’t misjudge and label us, better educate the medical community, and fund research that will discover a cure.

Join Us to Help Raise ET Awareness
I hope I can count on each of you to do something that helps the ET community. So many great ideas are listed on the IETF website. If all of us raise our voices about ET, we can educate the world.

 

 

Being a Child with ET is Like Being a Tiny Sapling in a Windstorm

By Allison Dyke,
Student at Kuyper College

Allison Dyke

Having essential tremor is like being a tree with leaves shaking uncontrollably in the wind. Even though your roots are strong, you are unable to control the movement of your branches.
However, being a child with essential tremor is like being a tiny sapling stuck in a powerful windstorm. You are not fully developed and your roots have not been completely planted.

ET Diagnosis
Since the age of four, I have battled with essential tremor. My shaky hands and legs made tasks such as eating, tying shoes, and riding bikes difficult to complete. I was just learning how to do these activities, which can be challenging without having a movement disorder. While these tasks were strenuous, the hardest part about being a child with essential tremor was other children and adults jumped to conclusions without understanding what essential tremor is.

“She’s just stubborn,” was often coldly stated to my parents as an explanation of why I was failing at making a perfect bow with my shoelaces. If I wasn’t being “stubborn” I must be either nervous or cold. Fine motor skills were frustrating to master and I became withdrawn from participating in physical activities. It was hard to understand why I couldn’t do what other children my age could do. With supportive parents, I was encouraged to never give up. The saying at my house is, “Where there is a will, there is a way.”

Soon I found an activity where shaking didn’t matter, swimming. It helped rebuild the confidence that this disorder had taken away. I found ways around difficult tasks, such as using assistive devices to improve my penmanship or supporting my elbow when applying makeup. It was clear that the physical and emotional effects of essential tremor were not going to dictate my future.

Connecting with the IETF
Then at sixteen, I discovered the International Essential Tremor Foundation, and wish I would have found them sooner. Their website provided informational downloads that I gave to my teachers and school administrators, so they could better understand this disorder. I connected with other kids my age who were going through the same experiences. IETF encouraged me to reach out to my community by organizing awareness walks and starting a support group. And they provided me with a college scholarship. With their support, I have met wonderful people who understand what having tremors is all about and I know I am not alone.

Now I’m nineteen and people still ask if I’m cold or nervous. But, I’ve learned that sharing my experience and educating others about essential tremor gives me an opportunity to explain that this condition is not just for older adults, but impacts young people as well. An estimated 10 million people in the United States are affected by this condition, yet very few have heard of it or understand it’s impact. Through awareness, I’m no longer a tiny sapling; I am strong oak supported by a dense forest away from the wind.

Editor’s Note
Allison is one of thousands of children and young adults around the world who struggle to live normal lives with essential tremor. She is a former IETF Scholarship Recipient. The scholarship program is one of many support programs conducted by the IETF. Your donations help support these programs and people with essential tremor. Donate today. 

New Year, New Opportunities

Patrick McCartney
Executive Director

As we kick off 2018, we are excited about the New Year and the opportunities to promote our mission to provide hope to the essential tremor (ET) community worldwide through awareness, education, support, and research.

The IETF is celebrating its 30th anniversary in 2018. We will kick off the celebration in March, which also happens to be National Essential Tremor Awareness month. There are a variety of ways for you to advocate for more ET awareness and education in your community. To learn more about how you can get involved here.

When we ask ET patients what they would like to see the IETF focus its efforts on, one subject comes up consistently: research to help find a dedicated treatment(s) for ET and, hopefully, someday a cure.

With the generous support of our donors,
we have been able to fund more than
$750,000 in ET research.

 

If you would like to learn more about the studies your donations have funded and the results they have achieved, you can find more information here.

Also, there are several clinical trials currently taking place that focus on ET-specific medications and wearable devices. If you are interested in participating in one of these trials check out the list of programs on the IETF website. You can also visit www.clinicaltrials.gov for easy access to free information on clinical studies for a wide range of conditions including ET.

Another great way to get involved is through a local support group. The IETF currently has more than 60 support groups around the world. We have a listing of all the supports groups on our website here. Or take part in our online support group through Facebook.

If you can’t find a support group in your area and Facebook isn’t your thing, we encourage you to consider starting a support group. We have all the tools to get you started and help you promote the meetings.

As always, we thank you for your support of the IETF and the ET community. The IETF has so many resources to share with patients, doctors, family members, and caregivers. We hope you will spread the word and help us continue to educate people on the most common movement disorder in the world.