Seeking Alternative Therapies for COPD? Get the Facts!

Part 1: Considerations before trying an unproven treatment for COPD

Photo Credit: Lee Edwards/CaiaImages.

Personal stories are powerful.  Few people can understand the struggles of a patient suffering from COPD quite like another person who is experiencing the same thing. When patients share their stories, camaraderie, hope, and even healing often result.

Many blogs and websites exist that share anecdotes of success stories of patients who have tried new and exciting therapies for COPD.  Many of these stories appropriately inspire hope, encouragement, and empowerment.

  It is important to remember, however, that one person’s story does not always represent scientific evidence, and that there are innumerable factors that contribute to each patient’s personalized experience.  Therefore, readers should approach such anecdotes with a healthy skepticism. 

For example, patients may read that “Therapy X” is the holy grail of COPD therapy, and on the same website hear others denounce the very same therapy.  What’s worse is that there are countless medical ‘scams’ that prey upon patients with incurable and irreversible diseases, including COPD and many others, with false promises of dramatic disease regression or even cure.  Sorting through it all is confusing and hard for patients (and even clinicians!) to know who to trust, when to try new therapies, and when to steer clear of so-called ‘miracle cures.’

In the upcoming weeks, we will share a series of articles that will help patients and their families make educated and informed decisions about how to approach alternative therapies for COPD.

  We will describe what questions you should ask and who you should ask them of.  In the series, we’ll discuss how patients and families can sort through the enormous volume of online material about new therapies, including research studies, and how to evaluate researchers and clinicians who provide yet unproven therapies.

So, let’s get started. 

1) FDA Approval

First, it’s important to understand the basics of how medications, devices and other therapies are regulated in the United States.  The main regulatory body, the Food and Drug Administration (FDA), is a government-run agency whose mission is to “assure the safety, efficacy, and security of human veterinary drugs, biological products (such as blood products or stem cells), medical devices (such as pacemakers), our nation’s food supply, cosmetics and products that emit radiation.” 

In order for a medication or therapy to become widely available to the public, FDA approval is required.  Patients should take precautions when considering therapies that are not FDA approved.  That said, clinicians may often prescribe medications or treatments that are “off-label”, meaning that the FDA has labeled the drug as safe for Disease X, but the clinician is providing the medication to treat Disease Y.  This is commonly done in practice, especially if published research has established that a therapy is effective but has not yet received its final approval from the FDA for that situation (a process which can take many years.) 

2) Clinical Trials

Another way that alternative therapies may be delivered safely to patients is through a clinical research study, or ‘clinical trial.’  The FDA requires that therapies undergo 4 phases of clinical trial testing prior to receiving approval, and this is a process that can take up to 20 years.  Most of the time, medications and therapies must first be tested in animal models, such as rats, monkeys or sheep prior to initiation in a human.  However, once human trials begin, therapies must progress through the following four phases of research:

  • Phase I: Researchers test a new drug or treatment in a small group of people (dozens) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. These trials are not designed to determine if a drug is effective or not, but only to establish safety.
  • Phase II: The drug or treatment is given to a larger group of people (hundreds) to see if it is effective and to further evaluate its safety. In other words, these studies determine if the therapy works in the body the way it is expected to and/or to figure out optimal dosing.
  • Phase III: The drug or treatment is given to large groups (thousands) of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.  These trial designs are called ‘randomized controlled trials’ and usually compare the new treatment to a ‘placebo’ or to a standard treatment.  Relatively few therapies make it through to Phase 3 testing.
  • Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

3) Unproven Therapies

Perhaps the greatest risk to patients, however, comes from clinicians who offer therapies that have not been FDA approved and are NOT part of clinical research studies.  Patients should be very suspicious of therapies that have not gone through (or are in the process of going through) the four phases of clinical trials.

For example, stem cells therapies are NOT regulated by the FDA and have yet to go through the phases of clinical research trials.  Despite this, however, some organizations offer stem cell therapies to patients at with very high out-of-pocket costs.  This practice is not only unethical but exceedingly dangerous for patients who may not realize the risks associated with these therapies. There are no professional medical societies that recommend this treatment, yet commercial (for profit) organizations (often disguised as medical centers) offer this therapy anyway.  Staying informed about  the evidence behind new therapies is an extremely important first step prior to trying new therapies, including research studies.

Stay tuned for Parts 2 and 3 of the series, which is meant guide patients in information gathering, investigation, and informed decision-making about alternative therapies for COPD.

Part 1: Seeking Alternative Therapies for COPD? Get the facts!

Part 2:  How can I participate in a clinical research study and what should I know before starting? 

Part 3: What are some examples of new therapies and ongoing research studies for patients with COPD?

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