Clinical trials can expand your access to new MPN treatments. To learn more about MPN studies, myMPNteam spoke to Dr. Gabriela Hobbs, the clinical director of leukemia services at Massachusetts General Hospital. Dr. Hobbs has designed and directed multiple studies for people with MPNs, both at Massachusetts General and with researchers at other institutions. Here’s what you should know before looking into a clinical trial.
Clinical trials are research studies that test new ways of treating disease, managing symptoms, or improving quality of life. In a clinical trial, the researchers (also called investigators) recruit a group of participants who agree to follow testing protocol and monitoring to evaluate the effects of an experimental treatment. Clinical trials may involve lifestyle interventions, such as a specific diet, or a new medication, procedure, or device.
Clinical trials are led by a principal investigator (typically a medical doctor) and may be sponsored by academic organizations, the government, or private companies. Because new medications must undergo a series of trial phases before approval, drug manufacturers frequently sponsor clinical trials. You may also find clinical trials at a large hospital or university. Often, people with MPNs learn about studies through their MPN specialist.
Dr. Hobbs is a big proponent of clinical trials. However, she emphasized being comfortable with your decision and never feeling pressured to participate (or continue participating) in a study.
According to Dr. Hobbs, “When deciding if you want to participate in a trial, it’s important to talk openly with your physician about whether or not this is the right thing for you and understand what the potential benefits and risks are.”
She said, “I tell my patients that as a clinical investigator, my bias is that you should always consider participating in a clinical trial. But I also understand clinical trials may not be the right thing for every patient, every single time. You should feel comfortable asking questions about what the trial is, including the risks and benefits. No matter how excited your doctor may be about a clinical trial, participation is always voluntary. The relationship that you have with your doctor shouldn’t change whether or not you participate.”
Dr. Hobbs encouraged people to take their time going through the paperwork of a clinical trial so they know what they’re signing up for. “Having that communication with the care team is so important to be able to say, ‘OK, I read this document, which is so long and overwhelming with all of these potential side effects, but tell me what this really means.’”
Dr. Hobbs also noted that there is a wide range of clinical trials, some with greater risks than others. “There are clinical trials that directly affect how the patient is treated. And those trials are very varied. In some trials, we’re trying a new medication for the very first time. In others, we’re trying a medication that’s been used (perhaps for many years for other blood cancers) and has already been FDA approved. We may already know a lot about it, but we're just trying it for the first time in MPN patients, for example.”
She explained, “For studies where we’re trying a drug for the very first time, there may be a higher risk and potentially unknown benefits. Studies that have used a drug for many different trials, like a later-stage trial where the drug is almost close to being approved, may have more of a known benefit or a potential known benefit. But not every patient is in the right place in their treatment to be considered for a clinical trial.”
Sometimes, people turn to clinical trials when other treatments aren’t working for them. However, it’s a common misconception that clinical trials are only for those with no other options or in the late stages of a disease. Dr. Hobbs explained why clinical trials are designed for people with MPNs at any stage: “I try to have trials for patients in different stages of their disease so that I can offer something to each of my patients.”
There’s no harm in asking your MPN specialist about a clinical trial, even if you’re newly diagnosed.
It’s normal to worry that a clinical trial may take you off course from progressing with treatment. However, several measures protect the best interest of participants:
Ideally, a clinical trial provides access to better treatments before they hit the market. Not only can clinical trials potentially help you but also others who are diagnosed with MPNs in the future.
You can ask the trial investigators and your doctor about continued access to a treatment that improves your quality of life or slows disease progression. It may be difficult to get insurance coverage for an experimental treatment before it is FDA approved, but every situation is different. If it’s in your best interest to keep going with an experimental treatment, you should pursue the opportunity.
Clinical trials for cancer are usually designed so one group (the control) receives standard treatment and the other group receives standard treatment plus supplementary or experimental treatment. It would be unethical to deny participants baseline medical care, so you shouldn’t worry that you’ll be in a placebo group left with no treatment. If you are concerned about being given a placebo, make sure to ask whether placebos are part of the study you’re considering.
As a trial participant, you have the right to leave a clinical trial at any time. Sometimes, people opt out of a study because they are experiencing negative side effects, their MPN is getting worse, or they don’t feel like keeping up with the additional testing and medical visits required by the study.
Dr. Hobbs explained, “Our priority when doing a clinical trial is to make sure that we are not putting the participants at risk. If there’s a side effect or even the hint of a side effect, we are monitoring patients really carefully. A patient is always able to say, ‘I don’t want to participate in this study anymore.’”
Participants may need to move or travel to a large university or cancer center for some clinical trials. Other times, your oncologist can monitor you while you receive experimental treatments. Dr. Hobbs emphasized that participants in clinical trials are monitored by an attentive health care team who either takes over the treatment plan or collaborates with their previous provider.
“I definitely try to work closely with patients’ primary care doctors, making sure that a patient with an MPN is healthy in general. I make sure that all the notes that I write get sent to the primary care doctor. Managing cardiovascular comorbidities with MPNs is important for preventing blood clots, a common MPN complication. If a patient needs something else specifically, and we’re working in a large hospital and have the ability, we will refer patients within our same hospital to other health care providers or social work. We work with social workers very closely, so there are lots of people who help out.”
“You’re very closely monitored, and there are additional people taking care of you.”
There may be costs associated with clinical trial participation. Some costs are considered patient care costs, while other expenses are research-related. Your health insurance should continue to cover most patient care costs, such as hospital stays, standard treatment, follow-up visits, labs, and imaging tests.
If you require additional doctor’s visits, testing, or if there’s a cost for the experimental drug or procedure, you’ll need to determine who is responsible for the bill. Ask questions about costs before signing up for a research study to avoid surprises. Study participation may even save you money if the treatment is covered by the trial.
Dr. Hobbs shared that various ongoing trials may lead to approvals for new myelofibrosis treatments. She said, “We are in an exciting time for myelofibrosis. There are going to be several phase 3 clinical trials. When drugs get to phase 3, they're close to seeking FDA approval. We’ve seen some really exciting and promising results from these new drugs.”
If you’re interested in finding out more about clinical trials, start by asking your MPN specialist. You can also search for upcoming trials online from these resources:
On myMPNteam, the social network for people with MPNs, you can connect with other people living, working, and thriving with MPNs. Members come together to ask questions, give advice, and share their stories with others who understand life with MPNs.
Have you thought about participating in a clinical trial? Do you still have questions or concerns after reading this article? Share your thoughts in the comments below, or start a conversation by posting on myMPNteam.
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