Everything to Know About Experimental Lung Cancer Treatment

Table of Contents
View All
Table of Contents

Many people who have lung cancer consider experimental treatments at some point during their journey,

This article will look at what it means to participate in a clinical trial, the potential benefits and risks, and the types of experimental treatments available for both early-stage and late-stage lung cancer. It also explores questions you may wish to consider before deciding if one of these treatments is right for you.

An illustration with Benefits vs. Risks of Clinical Trials

Illustration by Jessica Olah for Verywell Health

Success Rates of Experimental Treatment

It's difficult to give a percentage for the success rates of experimental treatments for lung cancer, as these vary tremendously. In some cases, a treatment is studied primarily to see if it causes fewer side effects than a known effective treatment.

What Is Experimental Treatment?

An experimental treatment is any type of treatment that has not yet received approval from the Food and Drug Administration (FDA) for general use among the public.

Experimental treatments for cancer may include:

  • Experimental medications (also called investigational drugs)
  • Different types of radiation therapy
  • Surgical approaches

It's important to note that before a medication is offered as an experimental treatment for humans, it goes through an extensive preclinical evaluation. This includes laboratory studies (such as studying the drug on cancer cells grown in a dish) and, in some cases, animal studies. After these preclinical studies are completed, a new medication must undergo FDA approval to be studied as an experimental medication in humans.

The three ways that people with cancer may have access to experimental cancer drugs are through:

  • Clinical trials are by far the most common way in which these treatments are used. These are carefully designed and controlled research studies that include increasing numbers of people depending on the phase of the trial.
  • Expanded access (compassionate use) refers to the use of an investigational drug outside of a clinical trial. Expanded access is regulated by the FDA and may be used only in certain settings. These include having a life-threatening medical condition for which no standard treatment is available and being ineligible to participate in a clinical trial evaluating the drug.
  • The Right to Try law allows people access to experimental treatments outside of a clinical trial if certain criteria are met (life-threatening condition, no standard treatment available, and ineligibility for a clinical trial), but is not regulated by the FDA. In order for a treatment to be used via Right to Try, it must have been previously studied in a phase 1 clinical trial.

If you are feeling anxious about the term "experimental treatment," it may be helpful to keep in mind that every treatment currently used for people with lung cancer was once considered an experimental treatment. In 1938, President Franklin Roosevelt signed the Food, Drug, and Cosmetic Act. It required new drugs to be tested for safety (as an experimental drug) and results submitted to the FDA for review for a new drug application.

Clinical Trials

Clinical trials are research studies that span the time from when a drug (or other treatment) is first used for humans until the treatment receives FDA approval. The three main phases of clinical trials, which are:

  • Phase 1 trials include small numbers of people (with lung cancer, this may include 10 to 30 people) and are designed to test the safety of a treatment (as well as dose), though information may be gathered about effectiveness.
  • Phase 2 trials often have a few hundred people and are designed to determine whether the treatment is effective. Further information is also gained about safety.
  • Phase 3 trials often compare a new treatment to the best available treatment (standard of care) and often have thousands of participants. They are designed to answer questions such as, "Is this treatment better than what we currently have? and "Does it have fewer side effects?"

If a treatment makes it through the three phases of clinical trials, the data can then be submitted to the FDA for approval.

Finding Clinical Trials for Lung Cancer

If you are considering taking part in a clinical trial, talk to your oncologist. You can also visit one of these websites:


There are risks and benefits associated with any treatment for cancer, but some may be unique in the setting of a clinical trial.

Since most lung cancers will continue to grow or spread if not treated, it's important to weigh these potential risks and benefits against the risks and benefits of other treatment options available (standard of care) as well as not treating cancer.

Some potential benefits of experimental treatment include:

  • The chance to use new treatments: With advanced lung cancer, a clinical trial may sometimes be the only way to receive a therapy that directly targets the cancer based on its genetic characteristics.
  • To add additional treatment options: In some cases, a clinical trial may add an additional option for treatment that is preferable in some way. For example, a new treatment may be thought to work better or have fewer side effects than standard treatment options.
  • Increased monitoring of your cancer: Participation in a clinical trial usually requires more frequent visits, as well as lab work and imaging studies.
  • You can change your mind: People who are in a clinical trial are free to stop participating at any time (and for any reason) and instead receive the current standard of care treatment.
  • Feeling empowered: Many people find it empowering to take an active role in their treatment.
  • To help others with lung cancer: Participating in a clinical trial may help advance the science of lung cancer treatment in a way that could benefit other patients in the future.

From the financial side, participation in a clinical trial may be both a benefit and a risk.

In a clinical trial, the treatment medication is usually provided free of charge and the extra monitoring tests are covered by insurance. On the other hand, it may be more costly to participate in a clinical trial if travel (air fare and lodging) is required and co-pays for additional lab and imaging tests add to costs.

New Treatments May Only Be Available Through a Clinical Trial

Many of the newer treatments for lung cancer, especially advanced lung cancer, may only be available under a clinical trial.


Potential risks of a clinical trial for lung cancer include:

  • Unknown effectiveness: Even if a treatment is thought to be superior to the standard of care, the true effectiveness is largely unknown until studied in a group of people.
  • Unknown side effects: Investigators are not fully aware of the potential side effects of treatment.
  • Frequent visits: Clinical trials usually require more frequent care both for visits and lab/imaging studies. Some people find this time-consuming or challenging in other ways.
  • Travel: Some clinical trials are only available at a single or a few cancer centers in the country. This may be challenging for some people.

Stopping a Clinical Trial

Some people are hesitant to participate in a clinical trial for fear they'll eventually change their mind and want to stop treatment. It's important to note that people can stop taking part in a clinical trial at any point (and at any time) they wish.

Types of Experimental Treatment for Lung Cancer

Experimental treatments for lung cancer may include new approaches to

  • Surgery
  • Radiation therapy
  • Medications/combinations of medications

Clinical trials are designed with different goals, and it's very helpful to understand these goals before considering a trial.

For example, the study may be evaluating whether:

  • The treatment may cure an early-stage lung cancer
  • The treatment may reduce the chance that early-stage cancer will come back (recur)
  • The treatment may extend life with cancer beyond the average amount of time a person is expected to live on the best available treatment to date (standard of care)
  • The treatment has similar effectiveness but has fewer side effects or is better tolerated than standard treatment
  • The treatment may improve quality of life with lung cancer

Should I Consider Experimental Treatment?

Everyone is different when it comes to the desire to pursue a clinical trial. Some people are willing to tolerate more risks if it means they have an opportunity to try a new and potentially better treatment option. Other people may be more concerned about unknown risks or side effects and may prefer the standard treatments available.

When considering a clinical trial, there are several questions you may wish to ask to help you make the best decision for your situation. These may include:

  • What is the success rate of the standard of care treatment? Do you think the experimental treatment will improve survival, reduce recurrence, or give me a better quality of life than the standard?
  • What side effects are suspected, and how do these compare with the side effects of standard treatment?
  • What is the goal of the trial? For example, is it to cure the cancer, improve survival, or improve quality of life?
  • What have earlier studies shown with this treatment (if they've been done)?
  • How have people done thus far in the trial (if known)?
  • Will I need to travel to take part in the trial? If yes, can some of the follow-up testing, such as lab tests, be done at home?
  • If you had my cancer, would you consider this clinical trial? Why, or why not?
  • What is the endpoint of the trial?
  • If the trial is discontinued but I'm responding well to the medication, will I continue to be able to use it?


Experimental treatments are those that have not yet been FDA approved for general use but have undergone extensive preclinical testing prior to human use.

As with approved treatment, there are both benefits and risks to consider.

A Word From Verywell

Deciding whether to receive an experimental treatment can be an anxiety-provoking decision, and it's important to talk about your feelings with your healthcare provider.

For those who work with people with lung cancer, clinical trials are almost the norm in quality care. But for the general public, they can be fraught with myths and stigma. No, you are not a guinea pig, but you should ask whether the clinical study you're considering is a placebo study—one that gives patients either the active treatment to be studied or a treatment with no therapeutic value in order to test the effectiveness of the experimental drug. If so, find out what your chances are of being enrolled in the placebo arm.

As a bottom line, it's helpful to keep in mind that you will only qualify for a clinical trial if the results of that trial are thought to be every bit as good (and hopefully better) than the current best standard of care.

4 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
  1. American Society of Clinical Oncology. Cancer.Net. Phases of clinical trials.

  2. Lung Cancer Research Foundation. Clinical trials.

  3. American Society of Clinical Oncology. Finding a Clinical Trial.

  4. National Institutes of Health. NIH clinical research trials and you.

By Lynne Eldridge, MD
 Lynne Eldrige, MD, is a lung cancer physician, patient advocate, and award-winning author of "Avoiding Cancer One Day at a Time."