Proceed with Caution as Cell-Based Retinal Disease Therapies Show Promise.

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Summary

Things to consider before participating in a clinical trial:

  1. Has an Investigational New Drug (IND) filing been approved by the U.S. Food and Drug Administration?
    1. Do not participate in any clinical trial that does not have documented FDA authorization.
    2. Seek consultation from your retinal care provider before considering participating in a clinical trial.
  2. What to expect in a well-designed clinical trial:
    1. The intent of the clinical trial should be clearly specified.
    2. There should be no cost or fees associated with the clinical trial.
    3. An informed consent form outlining the details (risks and benefits) of the study should be given to all participants.
    4. Legal language releasing the investigator(s), the sponsor(s), the institution, or its agents from their legal liability for negligence is a red flag.

To members of the Inherited Retinal Disease (IRD) Community:

The Foundation Fighting Blindness is committed to funding research efforts focused on finding treatments and cures for blinding retinal diseases that impact millions of people across the world. This includes stem cell-based therapies that show promise in helping restore or maintain retinal function and vision.

In recent years, the number of stem cell treatments offered by various clinics claiming to benefit retinal eye disease has increased. Of concern, are those clinics administering stem cell-based therapies that do not adhere to the U.S. Food and Drug Administration’s (FDA) standards. Recently, a federal court ruled that the FDA has regulatory authority over stem cell procedures and has the power to halt them, even if the stem cells originate from the participating patient.

The Foundation strongly suggests that patients considering any cell-based therapy, whether stem cell or similar technology, proceed only with FDA-authorized clinical trials or treatments. To confirm whether a study is under the oversight of the FDA, first ask those providing the treatment, or conducting the clinical study, detailed questions such as those found below which were provided under the guidance of the Foundation’s Scientific Advisory Board.

What to Ask

When considering a novel treatment or participation in a clinical trial, ask:

  • Has an Investigational New Drug (IND) filing been approved by the FDA?
    • The FDA carefully reviews all filings for an IND prior to clinical trial authorization. You should NOT participate in any clinical trial that does not have IND approval. The fact that a “trial” is listed on the clinicaltrials.gov website does NOT indicate that the FDA has authorized it. Other review processes, like Institutional Review Board (IRB), are NOT a substitute for FDA authorization.
  • Ask to review documentation showing authorization by the FDA.
  • What is your retinal care provider’s opinion on the novel treatment or clinical trial?
    • Seek consultation from your primary retinal care provider or consider obtaining a second opinion before participating in the trial. Share any information you have on the therapy with your doctors.

What You Should Expect

The following are things you should expect in a well-designed clinical trial:

  • The intent of the clinical trial should be clearly specified.
    • Clinical trials are designed and intended to benefit future patients and may not be effective for participants in the clinical trial.
    • A Phase I clinical trial is designed to test the safety of the treatment in humans.
    • A Phase II clinical trial is designed to establish if a treatment that has been determined to be safe in humans, also demonstrates some benefits to the intended populations.
    • A Phase III clinical trial enables investigators to better gauge the effectiveness and side effects of a therapy in a large population of diverse patients.
    • Clinical studies will have FDA-authorized research protocols that address a disease within a particular human population.
    • Any treatment claiming to treat multiple retinal diseases, or retinal and other diseases, is highly suspect and should be met with caution.
  • There should be no costs or fees associated with the clinical trial.
    • The cost of the clinical trial is paid for by drug developers or governmental agencies. Patients should NEVER PAY for treatment as part of a clinical trial (sometimes insurance will pay for some “standard care” costs).
  • You should be given a detailed “informed consent” form outlining the details of the study, as well as any risks and potential benefits. You can also ask to see the detailed study protocol, which describes the schedule of tests, procedures, medications and dosages, etc. Also, it should describe the eligibility criteria for participation (i.e., age, sex, disease type, etc.).
    • Every clinical trial requires written informed consent before receiving treatment.
    • This document will explain what will be done to the participant, how the research protocol works, what risks or discomforts the participant may experience, and that involvement is a voluntary decision of the participant.
    • If there is any language that releases, or appears to release the investigator, the sponsor, the institution, or its agents from their liability for negligence, or appears to be too legalistic, do NOT sign and seek guidance from your retinal health care provider or the Foundation.

Currently, there are several ongoing stem cell clinical trials focused on inherited retinal diseases and age-related macular degeneration. While the Foundation is excited to see cell-based therapies move from the laboratory to patients, it is critical patients are alert to the therapeutic offerings that are not overseen by the FDA and are able to differentiate those from FDA-authorized studies that have the potential to provide the next treatment or cure.

To contact the Foundation Fighting Blindness with questions about a trial you are considering please email info@FightingBlindness.org or phone (800) 683-5555.

If you would like more information on clinical trials related to blinding retinal diseases, please visit our Clinical Trials page. For information on the status of clinical trials currently in progress, please visit our Clinical Trial Pipeline page.

On behalf of the Foundation Fighting Blindness and its Scientific Advisory Board.