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Are MPN Clinical Trials Important for New Treatments?

Are MPN Clinical Trials Important for New Treatments?
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Published on August 7, 2020

MPN Trials Help Determine Best Combination Therapies

MPN clinical trials are critical to improving care and outcomes for patients with myeloproliferative neoplasms (MPNs) because of the rarity and complexities of the blood cancers, according to two researchers from the United States and Canada.

However, the outbreak of the novel coronavirus has made participating in clinical trials much more challenging.

“MPNs are very rare, and so folks who are living with MPNs, and were diagnosed 20 or 30 years ago, really didn't have a lot of options for treatments beyond aspirin and phlebotomy and we've been doing phlebotomy in medicine for hundreds of years,” said Dr. Chris Hillis, a researcher, clinician and assistant professor from McMaster University in Hamilton, Ontario. “And so certainly, I think after a few hundred years, it's time that we started testing some new therapies and we really are at an exciting time.”

Clinical Studies Advance MPN Combination Therapy Options

Dr. Hillis, along with Dr. Barbara Klencke, the chief development officer for Sierra Oncology, and MPN patient Marilyn Lidor and her care partner, Dov Lidor, participated in a recent Answers Now program with Patient Power Co-Founder Andrew Schorr about the importance of MPN clinical trials.

During the webinar, Dr. Hillis stressed that MPN trials are crucial for determining the best combination of therapies for specific conditions.

He noted that JAK inhibitors have been a significant improvement in the treatment of MPNs, but as single agents, JAK inibititors aren’t enough.

“And so, I think combination therapies… might be the way of the future,” he said. “But we don't know what the right combination is and so entering a trial, either after ruxolitinib (Jakafi) to say, ‘What's the next best step for me?’, or if ruxolitinib is not working for you… or if you're at the beginning of your MPN journey, thinking about a combination trial just so that we can learn more about what's next or what can make the JAK inhibitors better.”

Myeloproliferative Neoplasm Patients Have Higher COVID-19 Risk

Until recently, the challenge of clinical trials has been convincing patients to endure an extra burden of clinical and hospital visits to monitor their progress. The coronavirus outbreak has added another layer of concern for clinical trial participants.

Patients with MPNs and other underlying conditions are at higher risk for contracting COVID-19, the disease resulting from the coronavirus.

Dr. Klencke told Schorr that most hospitals are limiting their clinical trial activity.

Sierra has launched the MOMENTUM Phase III trial designed to enroll 180 myelofibrosis (MF) patients who are symptomatic and anemic, and who have been treated previously with a JAK inhibitor. The trial is testing the effectiveness of an orally administrated drug called momelotinib.

“These are complicated things for them to do,” she said. “The first thing that some of our sites have done is to prioritize vaccines or other COVID treatments.

“But when you have a cancer population that needs care anyway, and you have a cancer trial that doesn't have a high burden of additional activities — we've actually seen sites be able to prioritize (our study). And so we've been very pleased about that. We're actually really nicely on target. Despite trying to enroll over the last six months.”

Marilyn Lidor, of Toronto, who was diagnosed with essential thrombocythemia (ET) in 2006 and MF in 2014, has chosen to continue participating in the clinical trial she started nine months ago. She is on Jakafi and a new drug called CPI-0610.

“I still go down to the hospital every six weeks, but there's hardly anybody there really, so I really have no concerns, and everybody who I do meet is masked and takes a lot of precautions, so I'm really very comfortable,” she said. “And then, in between the six weeks, I go to a local lab near me which is also…hardly anybody there, so I'm very comfortable.”

“I'm doing great,” she added. “I'm feeling great, big difference since I'm on the trial.”

MPN Observational Studies Offer an Alternative to Clinical Research

For those patients who can’t join a clinical trial, Dr. Hillis recommended they enroll in observational studies or registries.

“In general, for folks who have rare subtypes of what is already a rare disease, there's very few interventional trials or drug trials for those conditions,” he said. “But what there are is a number of registry or observational studies across North America and Europe that I would highly encourage (patients) to enter into.

“It's only once we get a critical mass of five or 10 people with a particular subtype of a subtype (that) we can start to answer questions,” he added. “And even if we can only answer those questions in an observational way, meaning without say, putting them on a drug, we can at least start to generate some ideas of trials we could do in the future.”

Sign up for Patient Power MPN e-newsletters to get the latest myeloproliferative neoplasm information delivered right to your inbox.

If you are a Myelofibrosis patient interested in participating in a clinical trial, use our online tool to start your search. For other MPNs visit the MPN Research Foundation to find open trials.

~Megan Trusdell


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