Dr. Morie Gertz, professor of medicine at the Mayo Clinic in Rochester and world renowned expert in amyloidosis, shares his views on the past, present, and future treatments of AL (light chain) amyloidosis. Over his four decades of experience with this disease, he has diagnosed and treated thousands of patients, advanced research, and managed countless clinical trials. This makes him the perfect professor to orate on the dramatic evolution of treating this historically devastating disease to the optimism of today, and the breakthrough world of tomorrow. This is a must-watch video from a legendary expert.
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The Future for Patients with Transthyretin Cardiac Amyloidosis is Looking Brighter
The treatment for patients with Transthyretin Cardiac Amyloidosis has advanced significantly since 2018 when there were no FDA-approved therapies. In this presentation, Dr. Mat Maurer from Columbia University shares how diagnostic imaging techniques have significantly improved, thereby reducing the need for an invasive heart biopsy. In addition, he shares fascinating statistics on how the age and stage of diagnosis has been evolving. Based on today’s clinical trials, providers are optimistic that the expansion of options for patient care will continue.
The future is indeed looking brighter.
Transplant: Inpatient vs Outpatient
There is no cure for Amyloidosis.
There are, however, an increasing number of treatment alternatives that can significantly reduce, if not eliminate, the disease and put the patient into remission. The most aggressive treatment is a stem cell transplant (SCT); sometimes referred to as a bone marrow transplant.
Stem cells are cells in the bone marrow from which all blood cells develop. This treatment aims to eradicate, typically through high-dose chemotherapy (e.g., melphalan), the faulty plasma cells which make the amyloid light chains. Once eradicated, fresh cells, harvested from the patient themselves (autologous), a donor (allogeneic), or an identical twin (syngeneic), are infused into the patient. This will help to recreate a healthy bone marrow and hopefully stop further production of the amyloid protein.
This complex treatment typically takes four to six weeks and is performed on an inpatient, outpatient, or some combination, depending on the hospital. There are meaningful differences that are important to know and incorporate into each patient’s personal situation in order to make an informed decision.
From the Healthcare Perspective
Across the country, there are multiple hospitals that perform SCTs to treat amyloidosis. While hard data is elusive, the tally of transplants at each facility, we know, is not spread evenly. We do know that Mayo Clinic (Mayo) and Boston University (BU) dominate the list and perform the majority of transplants. It may not be a surprise, then, that these two hospitals are considered amyloidosis Centers of Excellence in the U.S. They see a high volume of cases, have extensive depth and breadth of expertise, and have sophisticated diagnostic equipment. They are also the two hospitals who have pioneered performing outpatient transplants. The good news is this is evolving, with more centers across the country expanding their transplant program to treat amyloidosis.
Everyone would agree that hospitals are germ and bacteria magnets, which can be dangerous for transplant patients with low to no immune systems. BU and Mayo, for example, found patients were better able to withstand the everyday germs outside of the hospital better than the more potent ones within hospitals. This provides a strong incentive for hospitals to consider outpatient, or if they choose the inpatient route, must be ever super mindful of this reality.
There are risks with SCT, and patient safety is key. Having a patient in-house during the treatment affords the hospital maximum control during the process, while being outpatient transfers some responsibility to the caregiver, such as monitoring the patient’s temperature, food, and fluid intake. Being inpatient also affords the quickest access to experts, equipment, and drugs in the event things go awry, which does happen. Mayo has found that a meaningful percentage (38% according to Dr. Morie Gertz) of patients never need hospitalization during the SCT process; however, on the occasions where it is necessary the duration averages a handful of days.
Treating patients on an outpatient basis requires hospitals to alter their process and training, and rely on the patient and caregiver to assume a more engaged role. Without question, hospitals benefit significantly from the experience of performing high volumes of outpatient transplants. Mayo, according to Dr. Morie Gertz, performed their first SCT in March 1996, and their first outpatient SCT in September 1998. In total, they have performed 744 SCTs and currently average about 33 transplants per year. According to Dr. Vaishali Sanchorawala, BU performed their first SCT in July 1994, and their first outpatient SCT in October 1996. In total, they have performed roughly 675 SCTs for AL Amyloidosis, with an annual run rate ranging between 25 and 50. Together, these institutions have over two decades of valuable experience. According to experts, small volume and the resultant lack of experience is likely the key driver behind why hospitals elect to perform SCTs on an inpatient basis.
From the Caregiver Perspective
Caregivers play a critical role in the SCT process, working closely with the healthcare team to ensure the patient is progressing appropriately. They are so critical, in fact, that regardless of inpatient or outpatient, hospitals will not proceed with a SCT unless they are confident the patient has capable and continuous caregiver support.
The role of a caregiver varies greatly between an inpatient and outpatient process. When inpatient, the caregiver provides important emotional support, as being confined to a hospital for weeks on end can be draining and discouraging. This can range from just being present, to chatting, to light activities. Caregivers also assist in the physical need for exercise, helping and encouraging the patient to walk whenever and however many steps possible. The caregiver role may be filled by one or more persons, often impacted by the distance the hospital is from home.
Outpatient SCT procedures are significantly more demanding of caregivers. For the duration of treatment, the hospital will require the patient and caregiver(s) to be proximal to the hospital. Mayo, for example, requires patients to be within ten minutes of the hospital. Fortunately, there are many hotels, motels, inns, and homes for rent (HomeAway, VRBO) that are transplant-friendly and reasonably priced. It is 24/7 support, monitoring the patient’s key indicators, administering and monitoring meds, transporting the patient to/from the hospital daily, securing meds, shopping and preparing food, maintaining the household (e.g., laundry, sanitizing, etc.), and on and on. The list is extensive and exhaustive. Arranging for such intensive support can be a challenge. Some patients assemble a series of caregivers who rotate in/out for periods of time, others are able to secure one dedicated caregiver for the entire time, and in rare instances, the patient is able to have a team of caregivers for the duration.
Whichever caregiver structure is chosen, it is important to also consider self-care for the caregiver. Mini breaks can go a long way to help sustain their ability to meet the needs of the patient and the requirements set forth by the hospital.
From the Patient Perspective
For patients, it is all about getting through this treatment and hopefully arriving at a successful outcome. Time distills down to weeks, then days, and then when things are their most difficult, just getting through the next hour is the focus.
Having a good and capable caregiver(s) in place can help the patient focus only on themselves, knowing the caregiver will take care of everything else.
Side effects of the SCT can be multiple and vary from patient to patient. The list of effects can include fatigue, fever, diarrhea, nausea/vomiting, loss of appetite, mucositis, and hair loss. Fortunately, the healthcare team can be very helpful in mitigating these effects.
Exercise is important to ward off muscular atrophy and does improve recovery. Every step matters. Both Mayo and BU find patients do better and are home quicker if they spend less time in bed and more time moving around. In addition, patients tend to benefit from the required additional movement needed when living away from the hospital.
Emotionally, a SCT is tough. No way around that. But having distractions, whether provided by the caregiver, getting out of bed to exercise or being out and about via outpatient does contribute to an improved psyche. Having any sense of normalcy is welcome.
Cost differs greatly between inpatient and outpatient treatment, with outpatient coming in meaningfully less expensive. Anecdotal information has outpatient transplants at roughly 50% off the cost of inpatient transplants. Yet regardless of the approach, SCTs are extraordinarily expensive, and most likely patients need their insurance to sign off before treatment can begin. One of the considerations by insurance companies is which hospital the patient is proposing for treatment. During our personal experience, where we dealt with two national insurance companies, both informed us that having treatment at a Center of Excellence made a difference.
Finally, what is it really like? While situations vary widely from patient to patient, as may treatments and outcomes, hearing about a SCT straight from a patient who has been there is helpful. Having had an outpatient stem cell transplant in July 2017, hear Mackenzie’s perspective while fresh post-Mayo. Additionally, preparing for an outpatient SCT is more involved for the patient and caregiver; we have provided SCT and Post-Chemo Tips on the Resources page of our website which others may find helpful.
Closing Thoughts
There is strong evidence over many years and many transplants that patient outcomes are better when performed on an outpatient basis. There are, however, notable implications for the healthcare providers, patients and caregivers, depending on which approach is chosen. Inpatient, outpatient and hybrid approaches can provide successful outcomes, but knowing these differences in advance is helpful to the decision-making process.
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Special Thanks
Morie A Gertz, M.D., M.A.C.P.
Consultant | Division of Hematology | Roland Seidler Jr. Professor Department of Medicine | College of Medicine | Mayo Distinguished Clinician
Mayo Clinic
Vaishali Sanchorawala, M.D.
Professor of Medicine | Director, Autologous Stem Cell Transplant Program | Director, Amyloidosis Center
Boston Medical Center and Boston University School of Medicine
Helping Patients Find Their Voice
In the words of Dr. Rodney Falk, “Amyloidosis is a paradigm for thinking outside the box.” The disease can be challenging to diagnose, even for the most seasoned physicians. In the case of the patient, the unknown, with all of its potential disease symptomatic variables, can make for a daunting situation. Developing one’s “patient voice” can seem unduly intimidating. Advocating one’s position may come natural to some, but terrifying for others, especially when the physician is viewed as the expert. Additionally, many patients may find it difficult to translate their feelings without having a medical vocabulary.
With regard to effective collaboration between physician and amyloidosis patient, there have been many success stories, but unfortunately numerous failures as well. One hereditary amyloidosis patient shares a story where after several years of increasing symptoms, and failed attempts with multiple physicians, he finally saw a cardiologist who went out of his way to put the patient at ease, telling him that he wanted to make it perfectly clear that the patient was of utmost importance and that he was being listened to. This was effective in allowing that patient to “find their voice,” with a resulting diagnosis being made shortly thereafter.
Another amyloidosis patient, a woman with AL amyloidosis, had to endure a nine-year journey of increasing symptoms prior to her diagnosis. “I was seen by nine doctors of various specialties, including hand surgeons who performed multiple carpal tunnel release surgeries, an electrophysiologist who diagnosed an unexplained autonomic dysfunction after a tilt table test, gastroenterologists who addressed three separate gastro-intestinal bleeds, multiple cardiological specialists who addressed atrial fibrillation, enlarged heart and diastolic dysfunction, and then finally a cardiologist who listened to my long list of symptoms and reviewed my relevant test results and medical history. This cardiologist maintained a sense of curiosity, resulting in him ordering a “free light chain” assay, leading to additional tests that ultimately confirmed a diagnosis of AL amyloidosis.” The patient states that “the cardiologist listened to me, was not dismissive of my complaints, and did not give up on me. It was this man’s persistence and curiosity that led to my diagnosis and saved my life.”
These two patient stories illustrate how imperative it is that the physician fosters an open and supportive avenue of communication such that the patient can “find their voice” and effectively function as their own advocate. Per Dr. Falk, “…you have to listen to the patient, because the patient is telling you an awful lot.” He goes on to say “…when that person comes through the door, they’re the most important person in the room and not the physician.”
There are several straightforward but effective catalysts that the physician can use to help the patient find their voice:
- Empower your patients. Some patients are intimidated by physicians and feel the power is entirely in their hands, so in order to foster better communication, give your patients a greater sense of participation in their care. This means making the patient feel important. Again, per the words of Dr. Falk, “…when that person comes through the door, they’re the most important person in the room and not the physician.”
- Listen without interrupting. According to the Journal of General Internal Medicine, patients on average have 11 seconds to explain the reasons for their visit before a physician interrupts. However, if the doctor lets them speak longer, they will tell you what brought them into the doctor’s office.
- Seek and provide information. Knowledge is power, both for the patient and the physician. Patients need to be able to effectively explain their situation to the physician and also have their situation explained back to them in terms that they can understand. This may include asking clarifying questions and allowing the patients to write down any questions that they may have, or any information that they may feel is important.
In summary, with a disease such as amyloidosis, it is imperative that a diagnosis be made in a timely manner, making “the patient voice” all the more critical.
Sources
The Power of the Patient/Physician Collaboration, https://mm713.org/the-power-of-the-patient-physician-collaboration/, Dec. 7, 2021`
6 Ways to Improve Patient Communication, https://www.jotform.com/blog/patient-communication/, March 23, 2023
Support Groups: Can They Play A Role In Your Treatment?
Have you recently been diagnosed with Amyloidosis? Wondering if joining a support group, whether online or in person, would be right for you?
When I was diagnosed with AL Amyloidosis, I thought that it wouldn’t be for me. It took me over a year to join. I had a picture in my mind of what it would be like. I envisioned a group of people gathered together to complain about how sick they were. I was afraid to get a glimpse of my future. I pictured a giant pity party.
What I experienced was extremely different. These groups are full of survivors! They are handling this disease with the most positive attitude possible and are making the best of the hand they have been dealt. I have learned so much from these people.
In these groups, you will find people who have been where you are, recently diagnosed, scared, and overwhelmed. You will find people willing to share the tips and tricks that helped them face some of their darkest moments and most difficult times. You will find people to help support and encourage you through your journey.
To find out more about the role of the support group, I reached out to Muriel Finkel from the Amyloidosis Support Groups (ASG) and Marsha McWhinnie from the Canadian Amyloidosis Support Network (CASN).
WHO IS THE SUPPORT GROUP FOR?
Support groups are for patients with all types of Amyloidosis and their caregivers. Their websites are full of information and resources. Online support groups such as the Amyloidosis Support Groups on Facebook, CASN, Smart Patients, and One Amyloidosis Voice are private and require access permission by the administrators. Such security for access offers comfort for the participants to reveal more personal information. In person support groups provided by Amyloidosis Support Groups, CASN, and the Amyloidosis Foundation, offer additional services and support for patients, as well as access to expert medical professionals.
WHAT IS THE GOAL OF THE SUPPORT GROUP?
Support groups, in general, are concerned with awareness and education. Their goal is to educate and empower patients, promote awareness, support medical research, and to improve the quality of life for those with Amyloidosis.
WHAT SERVICES DO SUPPORT GROUPS PROVIDE?
- Both ASG and CASN operate a toll – free number, the primary goal of which is to provide a compassionate, understanding ear to those recently diagnosed. If you have received this life-altering and scary diagnosis, this call can let you know you are not alone and that there is hope.
- Their websites provide educational videos and articles, medical referrals, patient stories, links to Amyloidosis support resources, and lists of upcoming meetings.
- Both groups host support group meetings, which provide an opportunity to meet other patients and caregivers. During these meetings, an Amyloidosis expert presents, and there is an opportunity to ask questions and share your story. The ASG hosts meetings throughout the United States and the CASN has meetings in Toronto and Quebec.
- The Amyloidosis Support Group also holds a special meeting for ATTR in Chicago every two years, with the top experts in the country participating and presenting.
- ASG sponsors multiple groups on Facebook so patients and caregivers can chat with each other, share stories, and ask questions. These groups are mediated by wonderful volunteers who are up to date with the latest treatments and advances in the field, with assistance from medical advisors. The groups on social media allow patients from all over the world to connect.
- Smart Patients is an online forum with conversations among the amyloidosis community, with topics ranging the full gamut from symptoms to treatment.
- A new online community was recently launched called ‘One Amyloidosis Voice,’ which has a message board, social wall, trusted resources, a news and meetings section, and a diagnosis educator.
- Through online forums and participation in local events, Support Group administrators and volunteers spread the word about Amyloidosis, with the goal of increased awareness.
- Both groups are interested in supporting medical research by sharing information about clinical trials with patients. Pharmaceutical reps are sometimes present at meetings to answer questions, and to help connect patients with trials that might be right for them.
WHAT A TYPICAL SUPPORT GROUP MEETING LOOKS LIKE
Although each meeting varies and ends up with a flow of its own, administrators do follow a basic outline which includes the following:
- New Business – A discussion of what is new in the field of Amyloidosis and what the Support Group has been working on
- Guest Speaker – An interactive presentation from an Amyloidosis expert (usually a doctor or other medical professional)
- Question and Answer Period – Questions are encouraged, and one on one time with the presenter may be possible
- Meal or snack is provided (depending on the timing of the meeting)
The atmosphere is laid back and comfortable, and patient confidentiality is paramount. It is absolutely your choice as to whether you share your story and ask questions.
BENEFITS OF THE SUPPORT GROUP
- You hear inspiring stories which can provide comfort and hope for the future.
- You are kept up to date with the treatments, clinical trials, and medical breakthroughs.
- You can share your story with people who genuinely get it.
- You can ask for help if you have questions. Facing symptoms and looking for relief? Concerned about a new symptom or side effect? Chances are there is someone in the support group who has faced it and can offer help and support.
- You can find information on what to expect from treatment. There are people in these groups who have endured stem cell transplants, chemotherapy, and have participated in clinical trials. In some cases, patients have tried various types of treatments and can offer suggestions of what to expect, and how to deal with side effects.
- You can also find information on the leading Amyloidosis Specialists and Centres of Excellence. Because Amyloidosis is such a rare disease, it is vital to get the experts on your team.
- Need to vent? We do that too. Sometimes it just becomes too much. The weight of it all hits and you feel overwhelmed and lost. There’s someone here who can provide a listening ear.
I am so glad that I decided to join these support groups. Not only do I get to hear the inspiring stories of those who are making life happen despite the challenges they face, but I have learned SO MUCH. Amyloidosis patients who have been living with this disease for years have a wealth of information. The administrators of the groups are also up to date with current treatments, clinical trials, and medical breakthroughs happening in the field of Amyloidosis.
I have met people whose advice has helped me through challenging times. There is something special about talking with someone who has been where you are now. Someone who has received the same diagnosis and faced the same overwhelming feelings of fear and uncertainty. To know that others have been where you are and have come out the other side is comforting and inspiring.
Thanks to these support groups I know I am never alone.
How to contact a support group near you
Amyloidosis Support Group
http://www.amyloidosissupport.org/
1-866-404-7539
Canadian Amyloidosis Support Network
1-877-303-4999
Amyloidosis Foundation
http://amyloidosis.org/resources/#websites-and-support-groups
One Amyloidosis Voice
https://www.oneamyloidosisvoice.com/
Smart Patients
https://www.smartpatients.com/
Lori Grover is a guest blogger for Mackenzie’s Mission. She was diagnosed with AL Amyloidosis in 2016 and writes to share experiences and lessons learned during her journey. More wonderful blogs by Lori can be found on her page Amyloid Assassin. When not writing, she is mostly a stay at home mom, florist, crafter, lover of books and food. Enjoy!
Clinical Trials 101
Clinical research is simply medical research involving people. There are two types, clinical studies (aka observational studies) and clinical trials. In this blog, we explore clinical trials and the basics of what you need to know.
WHAT ARE CLINICAL TRIALS?
According to the National Institutes of Health (NIH), clinical trials are research studies performed on people that are aimed at evaluating a medical, surgical, or behavioral intervention. Clinical trials are the primary way that researchers find out if a new treatment, like a new drug or medical device (e.g., a pacemaker) is safe and effective in people. Often a clinical trial is used to learn if a new treatment is more effective and/or has less harmful side effects than the standard treatment. Other clinical trials test ways to find a disease early, sometimes before there are symptoms. Still, others test ways to prevent a health problem before it begins. A clinical trial may also look at how to make life better for people living with a life-threatening disease or a chronic health problem.
WHY CLINICAL TRIALS ARE IMPORTANT
Clinical trials permit researchers to test the safety and effectiveness of new therapies. They also allow for a rigorous evaluation through patient participation. Bottom line: it is only after the extensive evaluation and testing from a clinical trial that the FDA will approve the widespread use of any new therapy.
According to Dr. Morie A. Gertz at the Mayo Clinic:
Advancing the medical care for all patients requires participation in clinical trials. Only through clinical trials can we further improve the available therapy options for current and all future patients. Clinical trials seek to answer questions about the natural history and biology of the disease as well as important questions regarding outcomes was all available new therapies. All current treatments received by our amyloid community were derived through others participation in clinical trials. Clinical trials are not only an opportunity to get the cutting edge therapy but is a way to “pay it forward“ for future generations of Patients.
WHAT ARE THE PHASES OF CLINICAL TRIALS?
All clinical trials must be approved by the U.S. Food and Drug Administration (FDA) before they can begin. Prior to that decision, scientists perform laboratory tests and studies in animals to test a potential therapy’s safety and efficacy. Assuming favorable outcomes, the FDA then gives approval for a clinical trial involving humans.
Clinical trials are comprised of four phases to test a treatment, find appropriate dosages, and detect side effects. If following the completion of the first three phases, researchers find the drug or intervention to be safe and effective, the FDA approves it for clinical use and continues to monitor its effects. Overall, the duration of a clinical trial spans years.
- A Phase I trial tests an experimental treatment on a small group of often healthy people (20 to 80) to judge its safety and side effects and to find the correct drug dosage.
- A Phase II trial uses more people (100 to 300). While the emphasis in Phase I is on safety, the emphasis in Phase II is on effectiveness. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. These trials also continue to study safety, including short-term side effects. This phase can last several years.
- A Phase III trial gathers more information about safety and effectiveness, studying different populations and different dosages, using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people. If the FDA agrees that the trial results are positive, it will approve the experimental drug or device.
- A Phase IV trial for drugs or devices takes place after the FDA approves their use. A device or drug’s effectiveness and safety are monitored in large, diverse populations. Sometimes, the side effects of a drug may not become clear until more people have taken it over a longer period of time.
WHY PEOPLE CHOOSE TO JOIN A CLINICAL TRIAL
There are many reasons why people choose to join a clinical trial. Some join a trial because the treatments they have tried for their health problem did not work. Others participate because there is no treatment for their health problem. Some studies are designed for, or include, people who are healthy but want to help find ways to prevent a disease that may be common in their family. By being part of a clinical trial, participants may access new treatments before they are widely available. Especially with a rare disease such as amyloidosis, clinical trials may offer a meaningful impact to a patient’s quality of life.
In addition, people may feel that participating in a clinical trial allows them to play a more active role in their own health care. Participants may receive more frequent health check-ups and closer monitoring through the clinical trial. Other people say they want to help researchers learn more about certain health problems. Whatever the motivation, when choosing to participate in a clinical trial, one becomes a partner in scientific discovery. This can also help future generations lead healthier lives. Major medical breakthroughs could not happen without the generosity of clinical trial participants—young and old.
In the words of Dr. Vaishali Sanchorawala at The Amyloidosis Center at Boston University School of Medicine and Boston Medical Center:
Clinical trials allow researchers and physicians to test the safety and effectiveness of new, promising drugs. Before any drug can be approved, it must be rigorously tested in clinical trials. Without the participation of patients, new treatments and cures will never happen. In addition, participating in a clinical trial may be a great way for patients to access new treatments before they become available. Especially in a rare disease such as amyloidosis, clinical trials can be a vital resource for the care of patients.
POTENTIAL RISKS OF A CLINICAL TRIAL
There are no guarantees of success from a clinical trial, and there are risks. For starters, there may be serious side effects. Also, the therapy may not improve upon current treatment, or may not even work at all. Finally, as a clinical trial participant, you may be part of the control group, which means either standard treatment or no-treatment placebo. In other words, there are no assurances you would receive the new therapy.
FINDING A CLINICAL TRIAL
Thanks to the internet, folks can find lots of information regarding the wide array of open clinical trials. So much so that it may be overwhelming. Particularly with regards to amyloidosis, casting such a wide net may not be the most productive approach. Since finding an appropriate clinical trial is not as easy for rare diseases such as amyloidosis, here are a few excellent places to start.
- My Amyloidosis Pathfinder (MAP). Developed by the Amyloidosis Research Consortium (ARC), MAP helps patients discover and learn about amyloidosis-related clinical trials. After answering a short questionnaire, MAP matches patients to trials specific to their condition and ones for which they may be eligible.
- Boston University / Boston Medical Center. A recognized Center of Excellence for amyloidosis, BU has an ongoing robust array of clinical trials for different types of amyloidosis.
- Mayo Clinic. A recognized Center of Excellence for amyloidosis, Mayo Clinic has an extensive clinical trial program in the area of amyloidosis.
- ClinicalTrials.gov. This resource, provided by the U.S. National Library of Medicine, is a database of over 250,000 privately and publicly funded clinical studies conducted around the world (in all 50 states in the U.S. and 204 countries).
INFORMED CONSENT PROCESS
The informed consent process is a key part of the safeguard of a clinical trial. Before joining a clinical trial, each participant will be told what to expect (e.g., treatments, tests) and what might happen (e.g., benefits and risks, including possible side effects). It is also the point where participants should ask ample questions about the trial, which the clinical trial coordinator should be more than willing to answer.
Below is a list of questions compiled from sources, including the NIH and The Clinical Study Center, recommending what patients should consider asking before consenting to participation in a clinical trial.
- What is the purpose of the study?
- Who is sponsoring the study, and who has reviewed and approved it?
- Who will be in charge of my care?
- What treatment or tests will I have? Will they hurt?
- What are the chances I will get the experimental treatment?
- What are the possible risks, side effects, and benefits of the study treatment compared to my current treatment?
- How will I know if the treatment is working?
- How will you protect my health while I am in the study?
- What happens if my health problem gets worse during the study?
- How will the study affect my everyday life?
- How long will the clinical trial last?
- What will happen after the conclusion of the study?
- Where will the study take place? Will I have to stay in the hospital?
- Will you provide a way for me to get to the study site if I need it?
- Will being in the study cost me anything (e.g., treatment, tests, travel)? If so, will I be reimbursed for all expenses (including other charges such as child care)? Will my insurance cover my costs?
- Can I take my regular medicines while in the trial?
- Who will be in charge of my care while I am in the study? Will I be able to see my own doctor?
- Will you follow up on my health after the end of the study?
- Will you tell me the results of the study?
- Whom do I call if I have more questions?
- How will you keep my doctor informed about my participation in the trial?
- Does the study compare standard and experimental treatments?
- If I withdraw, will this affect my normal care?
- What are the chances that I will receive a placebo?
- What steps ensure my privacy?
Taking part in a clinical trial is solely the decision of the participant, although they may want to discuss it with their medical team prior to finalizing a decision. If one decides to join the trial, they will be required to sign an informed consent form that presents the key facts of the study and indicates they have been told all of the details and want to be part of the study. Importantly, the informed consent form is NOT a contract. Participants can leave the trial at any time and for any reason without being judged or put in a difficult position regarding medical care. Researchers much keep health and personal information private. Also, during the trial, participants have the right to learn about new risks or findings that emerge.If researchers learn that a treatment harms any of the participants, they’ll be removed from the study.
PARTICIPANT PROTECTION & SAFETY
Before committing to participate in a clinical trial, it is important to understand participant safety. Congress has put laws in place to protect against abuses, and today every clinical investigator is required to monitor and make sure that every participant is safe. These safeguards are enforced by the Federal Government. Every clinical trial follows a protocol that describes what the researchers will do. The principal investigator, or head researcher, is responsible for making sure that the protocol is followed.
In addition, there are multiple scientific oversight groups to aid in the control of clinical trials.
- Institutional Review Board (IRB): Comprised of doctors, scientists, statisticians, and lay people, IRBs provide scientific oversight for all clinical trials in the United States. IRB members regularly review studies and their results, making sure risks (or potential harm) are minimized.
- Office for Human Research Protections (OHRP): The U.S. Department of Health and Human Services’ (HHS) Office for Human Research Protections (OHRP) oversees all research done or supported by HHS. The OHRP helps protect the rights, welfare, and well-being of research participants. They provide guidance and oversight to the IRBs, develop educational programs and materials, and offer advice on research-related issues.
- Data Safety Monitoring Board (DSMB): Comprised of research and study topic experts, this board is required for every NIH phase III clinical trial. Their role is to review data from a clinical trial for safety problems or differences in results among different groups of relevant studies. If they find that the experimental therapy is not working or is harming participants, they will halt the trial right away.
- Food and Drug Administration (FDA): In the United States, the FDA provides oversight for clinical trials that are testing new medicines or medical devices. They review applications before any testing on humans is done, checking to ensure a proposed clinical trial has proper informed consent (see earlier) and protection for human subjects. In addition, the FDA provides oversight and guidance at various stages throughout the trial.
Scientific oversight informs decisions about a trial while it’s underway. For example, some trials are stopped early if benefits from a strategy or treatment are obvious. That way, wider access to the new strategy can occur sooner. Sponsors also may stop a trial, or part of a trial, early if the strategy or treatment is having harmful effects. Protecting the safety of people who take part in clinical trials is a high priority for all involved. Each trial has scientific oversight, and patients also have rights that help protect them.
DECIDING WHO PARTICIPATES IN CLINICAL TRIALS
After signing the informed consent form, the clinical staff will screen the candidate against the clinical trial criteria. The screening may involve cognitive and physical tests. Inclusion criteria for a trial might include age, stage of disease, gender, genetic profile, family history, and whether or not the candidate has a study partner who can accompany them to future visits. Exclusion criteria might include factors such as specific health conditions or medications that could interfere with the treatment being tested. Generally, individuals can participate in only one trial or study at a time. Different trials have different criteria, so being excluded from one trial does not necessarily mean exclusion from another.
Clinical trials need numbers … many volunteers must be screened to find enough people for a study, and with rare diseases such as amyloidosis, important trials are often significantly delayed due to a lack of participants. This can seriously slow down the rate at which new drugs are discovered, tested, and made available to patients.
CONCLUSION
Not all clinical trials have successful outcomes. However, every disease-related drug and therapy treatment prescribed today is the result of clinical research. Clinical trials are absolutely necessary to determine that a treatment is safe and that it has a real positive effect on a particular disease, better than that observed by a placebo or the current standard of care.
Final thoughts from Isabelle Lousada, founder and CEO of Amyloidosis Research Consortium:
Clinical trials play a critical role in evaluating novel therapies, establishing the best treatment pathways, and increasing our knowledge about amyloidosis.
SOURCES
Amyloidosis Research Consortium
Boston University / Boston Medical Center, Amyloidosis Center
The Clinical Study Center
Mayo Clinic
National Institute on Aging
National Institutes of Health
U.S. National Library of Medicine
