Clinical trials offer a unique treatment experience when you live with myeloproliferative disorders. Lower cost of care, direct access to a specialized MDS medical team, and the opportunity to try new and emerging treatments are all potential benefits.
Myeloproliferative disorders, now referred to as myeloproliferative neoplasms (MPNs), include a group of blood cancers that develop when bone marrow produces too many red blood cells, white blood cells, or platelets.
Some forms of MPNs, such as essential thrombocythemia (ET)
Depending on the specific diagnosis you received, your healthcare team may recommend participating in an MPN clinical trial to expand your treatment possibilities.
Clinical trials are research studies testing the safety and efficacy of emerging medical therapies.
They consist of up to 5 phases that start with exploratory evaluations and end with a post-approval study once the therapy has proven safe, effective, and has passed regulatory agencies like the Food and Drug Administration (FDA).
Clinical trial phases include:
- Phase 0: micro-dosing phase, aims to understand dose tolerance
- Phase 1: explores dosing safety and potential adverse effects
- Phase 2: looks at how the new treatment works against cancer
- Phase 3: compares the new treatment against a current standard treatment
- Phase 4: evaluates long-term safety and effectiveness following FDA approval and public use
Clinical trials can offer several benefits related to medical care costs, level of care, and treatment opportunities.
“Participation in clinical trials has many advantages,” explains Dr. Jan Philipp Bewersdorf, hematologist at Yale Cancer Center. “Firstly, patients get access to the most promising, novel therapies that are not yet available outside of clinical trials…” Secondly says Bewersdorf, participating in a clinical trial is a “generous gift” that benefits others with an MPN diagnosis “by helping researchers learn more about novel therapies and the underlying disease.”
When you participate in a clinical trial, you’re placed under the care of a medical team who specializes in your diagnosis. And because you’re assisting in research, many of the costs associated with that specialized care may be significantly reduced or removed.
Typically, the study coordinator or research nurse organizes examinations, tests, and follow-ups to ensure the clinical trial runs smoothly.
Why might a clinical trial be right for MPNs?
For MPNs, clinical trials may hold appeal because of treatment challenges some people face with these disorders.
Dr. Daniel Landau, an oncologist and hematologist with The Mesothelioma Center says it’s important to understand the nature of MPNs when looking at the value of clinical trials.
“I think, first off, we need to understand a bit about myeloproliferative disorder. While we try to separate MPNs into different categories, like polycythemia vera, essential thrombocytosis, or myelofibrosis, the reality is that they overlap with one another.”
Landau points out that over the last several years, the treatment options for MPNs have been extremely limited.
“But as time has progressed and we’ve learned more about the etiologies of these diseases, we have been able to adapt more targeted therapies to treat them,” he says.
Clinical trials do have a potential downside, indicates Dr. Derek Janssens, lead investigator at the Van Andel Institute’s Janssens Laboratory and assistant professor in the Van Andel Department of Epigenetics.
“By their nature, clinical trials are experimental,” he says. “There isn’t a guarantee that a potential new treatment will work. Participants also may experience side effects.”
Janssens explains prior to agreeing to participate in a trial, medical staff share information about the trial and its potential risks through a process called informed consent. This ensures that participants understand possible risks and benefits.
Each clinical trial will have its own set of eligibility criteria based on the study’s goals and the trial phase.
“Ongoing clinical trials for MPNs seek to pair patients with the best treatment options to manage and potentially cure their conditions,” Janssens says. “For this reason, participation in Phase I and Phase II clinical trials is typically limited to a subset of patients that are most likely to benefit.”
Even if you aren’t a candidate for the early phase clinical trials, there may be another place for participation. Bewersdorf states almost everyone is a candidate for MPN clinical trials, and he discusses it as an option with all his patients.
“There are differences between individual trials in terms of their inclusion and exclusion criteria, but at Smilow Cancer Center, we are fortunate to have a large number of trials so that we can offer a trial to almost all of our MPN patients.”
Your healthcare team can help you connect with clinical trials that fit your diagnosis, but you can also search for open opportunities by visiting:
When selecting a clinical trial, several factors in the title can help you understand what the trial is about and if you qualify:
- trial phase
- medication
- MPD type
In the on-going clinical trial for MPD titled A phase 3 study of the hepcidin mimetic rusfertide (PTG-300) in patients with polycythemia vera, you can see right away from the title it’s a phase 3 clinical trial, which means the focus will be comparing the new treatment against a current standard therapy.
You can see the medication you may be taking is called hepcidin mimetic rusfertide (PTG-300). The details also indicate researchers are specifically asking people who have received a confirmed diagnosis of polycythemia vera to apply for participation in the clinical trial.
It’s important to discuss clinical trial participation with your healthcare professional to ensure it’s the best choice for your diagnosis.
Factors that can influence your decision include:
- costs
- time commitment
- travel
- underlying health conditions
- types of required testing and procedures
- side effects
“I tell all my patients that enrolling in a clinical trial is a very personal decision and that there is no right or wrong decision as trial enrollment is always optional,” says Bewersdorf. “It is important for patients to understand what is involved when they sign up for a trial in terms of logistics (e.g., number of study visits, study related procedures), possible side effects, and expectations for how well the treatment is likely going to work.”
Bewersdorf adds that patients can decide to stop a trial at any time without providing a specific reason.
If you want to participate in a clinical trial and you’ve discussed the decision with your care team, you can contact the clinical trial administrator through their details on the clinical trial listing.
Clinical trials offer a way to receive expert, specialized medical care and novel treatments for conditions like MPN. By participating in a clinical trial, you’re helping improve future outcomes for others with these conditions.
If you’ve received a confirmed MPN diagnosis, your healthcare team can help you navigate which clinical trial suits your needs best.
You can stop participation in a clinical trial at any time, for any reason.