Understanding Clinical Trials for Blood Cancer: What Patients Should Know

When you or someone you love is diagnosed with a blood cancer, the conversation quickly turns to treatment options. Chemotherapy, immunotherapy, targeted therapy, stem cell transplants — the list can feel endless. But there is one option that often goes unmentioned in those early conversations: clinical trials for blood cancer. Clinical trials are carefully designed research studies that test new treatments or new combinations of existing treatments, and for many patients, they represent a real and sometimes life-changing path forward.

Despite this, fewer than eight percent of adult cancer patients in the United States participate in clinical trials. That number is not low because patients are unwilling — research shows that when patients are actually offered a trial, more than half agree to participate. The gap exists because many patients are never told about trials, never referred, or simply do not know where to start. This guide is meant to close that gap.

What Are Clinical Trials and Why Do They Matter?

A clinical trial is a research study in which volunteers receive a new treatment under close medical supervision. Virtually every standard cancer treatment in use today — every chemotherapy drug, every immunotherapy, every targeted therapy — was once tested through a clinical trial. Without these studies, medical progress would stop. For blood cancer patients specifically, clinical trials have led to major breakthroughs in recent years, including CAR-T cell therapy and several targeted drugs that have transformed the outlook for conditions that were once considered untreatable.

Participating in a clinical trial does not mean you are a guinea pig or that you will receive unproven care. Trials go through multiple phases of testing before they ever reach patients, and every trial follows a strict protocol reviewed by ethics boards and federal regulators. Patients in clinical trials often receive more frequent monitoring and closer attention from their medical team than those receiving standard care.

Phases of Blood Cancer Clinical Trials

Clinical trials are conducted in phases, each with a specific purpose. Understanding these phases can help you make a more informed decision about whether a particular trial is right for you.

Phase I trials test a new treatment for the first time in a small group of people, focusing on safety and dosage. These trials typically enroll 15 to 30 patients.
Phase II trials expand the study to a larger group and focus on whether the treatment works for a specific type of cancer. Researchers also continue to monitor safety.
Phase III trials compare the new treatment against the current standard of care, often enrolling hundreds or thousands of patients across multiple centers. These are the trials that can lead to FDA approval.
Phase IV trials take place after a treatment has been approved and monitor its long-term effectiveness and side effects in the broader population.

A common concern is that you might receive a placebo instead of real treatment. In blood cancer trials, placebos are rarely used alone. Most trials compare a new treatment against the current best available treatment, so every participant receives active care.

How to Find a Clinical Trial for Blood Cancer

Finding the right clinical trial can feel overwhelming, but several free resources exist to help you navigate the process. You do not have to do this alone, and you do not need to be a researcher to find a trial that fits your situation.

Talk to your oncologist or hematologist. Your doctor can review your diagnosis, subtype, and treatment history and identify trials that match your clinical profile. If your doctor does not mention trials, it is completely appropriate to ask.
Contact the Leukemia and Lymphoma Society Clinical Trial Support Center. Their nurse navigators specialize in blood cancers and can help you identify appropriate trials, understand eligibility requirements, and connect you with enrollment teams — all at no cost.
Search ClinicalTrials.gov, the federal database of all registered clinical trials. You can filter by disease type, location, and phase to find studies near you.
Ask about trials at academic medical centers. Many of the most promising blood cancer trials are based at university-affiliated cancer centers, and some offer remote participation options or travel assistance.

What to Expect as a Clinical Trial Participant

Every clinical trial has a detailed plan called a protocol, which outlines who is eligible, what treatments are given, what tests are required, and how long the study lasts. Before you enroll, the research team will walk you through the protocol in a process called informed consent. You will have the opportunity to ask questions, take time to think, and discuss the decision with your family and care team.

Once enrolled, you can generally expect more frequent visits and monitoring than standard treatment. This can include extra blood draws, imaging scans, and check-ins with your medical team. While this requires more of your time, many patients find the closer monitoring reassuring. It is also important to know that you can withdraw from a clinical trial at any time and for any reason — participation is always voluntary.

Understanding the Costs

One of the most common concerns about clinical trials is cost. In most trials, the study drug and related tests are provided at no charge by the trial sponsor. Routine care costs — such as doctor visits and standard lab work — are typically covered by insurance. However, there can be additional costs related to travel, lodging, childcare, and time away from work that catch patients off guard. Understanding which costs are covered before you enroll is essential.

Ask the research team for a detailed breakdown of what the trial sponsor covers versus what falls to you or your insurance. Many trials also offer stipends or reimbursement for travel and related expenses. Organizations like the Leukemia and Lymphoma Society and the Patient Advocate Foundation can help you find financial assistance for trial-related costs.

Questions to Ask Before Joining a Clinical Trial

Before enrolling, make sure you feel confident in your understanding of the trial. These questions can help guide the conversation with the research team.

What is the purpose of this trial, and what phase is it in?
What treatment will I receive, and how does it compare to the current standard of care?
What are the known risks and potential side effects?
How long does the trial last, and how often will I need to visit the study site?
Will I have to pay for any part of my care during the trial?
What happens if the treatment does not work or I want to stop participating?
Will I still be able to receive standard treatment if I leave the trial?

For more guidance on communicating with your medical team, our guide on questions to ask your oncologist covers additional topics every blood cancer patient should discuss with their doctor.

This article is for informational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always consult your healthcare provider with questions about your specific medical condition.

Every Patient Deserves to Know Their Options

Clinical trials are not a last resort. They are a legitimate treatment option that can be considered at any stage — from first diagnosis through relapse and beyond. The treatments that save lives today were made possible by patients who chose to participate in trials before those treatments were widely available. If a clinical trial might be right for you, the most important step is simply asking your doctor about it.

At the Live Like Brent Foundation, we know that pursuing the best possible treatment means managing the everyday burdens that come with a cancer diagnosis — transportation, bills, meals, and more. Our comfort fund program provides direct financial support to blood cancer patients and their families so they can focus on their health instead of their expenses. If you or someone you love is exploring treatment options for blood cancer, apply for a comfort fund today, or make a donation to help another family get the support they need.