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. 

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. 

No Boundaries, No Favorites. Essential Tremor Has No Familiar Face.

By Tammy Dodderidge, IETF Marketing and Communications Manager

Who do you picture in your mind when you think about someone who has essential tremor? Chances are, it’s a senior adult or a grandparent. The reality is essential tremor has no familiar face. It afflicts people all ages and races. While senior adults struggle with it, so do middle-aged adults, teens and even toddlers. It doesn’t discriminate based on the color of your hair, or eyes or skin.

This is our message during National Essential Tremor Awareness (NETA) Month in March. We want people to understand that ET has no boundaries. It doesn’t play favorites. It is a serious, debilitating condition for more than 10 million people around the world.

Background on NETA Month
Thanks to a three-year effort by the International Essential Tremor Foundation (IETF), in 2010 Congress passed a House resolution that officially designates March as National Essential Tremor Awareness Month. This month gives ET a platform. Most people have heard of Parkinson’s disease and the physical tremors associated with it, but not many have heard of essential tremor. We need to let them know it exists and then educate them about the needs of people with ET.

The next time you are in a crowd, look around and chances are, someone there has ET. You won’t know it because it’s something that people work hard to hide. You won’t know it because people are embarrassed by it. You won’t know it because people fight it and deny it. You won’t know it because many people who have ET suffer depression and isolate themselves so they don’t even leave their houses.

Take Action
What can you do to take part in NETA Month? Many, many things!

  • The IETF has created some special NETA Month social media banners specifically for Facebook and Twitter. Download them from our website and use them on your social media sites during the month of March (as the cover art or just share them with your friends and followers).
  • Order one of our NETA Month posters and hang it in a prominent area where people will notice it: your church, the grocery store, the library, your office. When someone asks, “What’s this poster? What is essential tremor?” you will have done your job in raising awareness!
  • Watch this Tremor Talk blog site during March. We will be featuring some guest writers who have ET, work in the ET community and advocate for people with ET. Their first-hand stories promise to be insightful and educational – and heartfelt. Share these blogs on your social media sites, or send the links to your friends by email. RAISE AWARENESS!
  • Hold a Do It Yourself (DIY) Fundraiser to support the work the IETF does toward education, research and advocacy. We have a list of DIY fund raising ideas on our website, and we also have special DIY  fund raising posters you can order from us for free to advertise your events.
  • Watch your mail for a letter from the IETF and make a donation to receive an NETA Month t-shirt and/or tote bag. These items are one more way to get ET in the public eye. Wear your shirt proudly and use your tote when shopping, or for carrying items to and from work. If you are not on our mailing list, you can join our mailing list online.

Once we all begin to see ET as a condition that impacts millions of people all over the world, we will begin to realize the need for more research that will lead to new treatments, and hopefully someday, a way to prevent or cure it for everyone.

Speak up, speak out and let’s rock this year’s NETA Month!

 

Share Your ET Stories with Me

Hello to everyone in the essential tremor community!

I am new to the International Essential Tremor Foundation and wanted to introduce myself. I am the new marketing and communications manager. My role involves development of messages and stories to educate the public about essential tremor and the impact it has on more than 10 million people nationwide. I serve as editor of our magazine, Tremor Talk, and our Tremor Gram enewsletter. I oversee our social media sites, Facebook and Twitter, and manage our website, among other responsibilities.

I must confess I had never heard of ET before I applied for this position. How could that be? It impacts so many people and yet I was not aware. But I am catching up. Each day I read and hear stories from people of all ages who are living with ET. Just this week I was reviewing the applications from our scholarship recipients and was in awe of their positive outlooks, and also saddened by what they have had to endure. One student was diagnosed at age six, so he has had little experience of NOT having ET. One is a mother of three who said her biggest challenge has been answering her children when they ask, “Will I shake like that when I grow up?”

I am proud to work for an organization that is serving the ET community with awareness and education initiatives, and support, like scholarships. And last year we donated $75,000 toward research initiatives to further better treatment options for ET.

The learning will continue for me, and I am asking all of you for your support in this area. I would not be able to do my work without the ET community at large who share their stories of hope, frustration and, sometimes despair. No, some stories are not happy ones, but these are the ones that may resonate with others who are battling each day. I will do my best to share these stories and keep the lines of communication open. And, I look forward to the day when I can write that story about the breakthrough in research that will help everyone. I know it’s coming . . . keep the faith. . . and keep in touch with me.

I look forward to hearing from all of you out there. Write to me at tammy@essentialtremor.org.

 

 

The IETF awards four scholarships to exceptional students with ET

IETF Scholarship Recipient Karissa Hartzell

The International Essential Tremor Foundation is pleased to award four students who are affected by essential tremor with $500 scholarships for the 2015-2016 academic year:  Tyler Criswell from Altoona, Ala.; Karissa Hartzell from Andover, Ohio (pictured left); Katie Merrill from Arvada, Colo.; and Hannah Spence from Colchester, Vt.

“These four students exhibit impressive qualifications based on academic and extracurricular activities in their communities and will become outstanding, future leaders in helping create more awareness about essential tremor,” said Catherine Rice, Executive Director of the IETF.

People with essential tremor have to be adaptable. Daily activities are often difficult, if not impossible, to accomplish without some type of assistance. It takes a special type of person to look obstacles in the eye and continue to push forward, regardless of the amount of time or effort it takes. It takes drive, passion, and a strong sense of self, and these four scholarship recipients truly display what it takes to be successful in their college careers while coping with ET. Read more about their stories here.

IETF scholarships are awarded to qualified students of all ages who have been diagnosed with ET, to lessen the burden of higher education. The scholarship can be used for supplies, books or tuition at licensed, accredited institutions of higher education (including trade schools) and are paid directly to the educational institution. For more information about the IETF scholarship program, please visit www.essentialtremor.org/about-the-ietf/scholarships.