Warning: Exosome Treatment in the US for SCI

Posted by Amanda Zimmerman in Research News on March 17, 2020 # Research

While there is great promise on the therapeutic usage of exosomes (vesicles that can hold growth factors or other biological Scientistsignaling molecules) in SCI and other neurological disorders, it has come to the research team’s attention that some in the US and abroad are promoting these as approved therapies. Exosomes (particularly those derived from certain stem cells) are currently being explored in clinical trials, but there are NO approved exosome treatments in the US currently.

I want to caution that an “exosome” in itself, like a “stem cell”, means nothing if we don’t know what type and what conditions were used to make them (think of an exosome as a biological carrier, the benefit is not in the exosome but in what it contains). Going to a clinic peddling unapproved treatments may not only be a waste of money for a placebo, they may actually be dangerous. In December of 2019, the FDA actually put out an advisory on exosomes based on reported adverse events. An excerpt is below:

The clinics currently offering these products outside of FDA’s review process are taking advantage of patients and flouting federal statutes and FDA regulations. This ultimately puts at risk the very patients that these clinics claim to want to help, by either delaying treatment with legitimate and scientifically sound treatment options, or worse, posing harm to patients, as evidenced by these recent reports of adverse events.

The FDA also has some good advice for any person with an SCI to consider with all “experimental treatments.” Within the US, patients and their families should:

  • Ask if the FDA has reviewed the treatment. You also can ask the clinical investigator to give you the FDA-issued Investigational New Drug Application (IND) number and the chance to review the FDA communication acknowledging the IND. Ask for this information before getting treatment and follow up with your personal health care provider to confirm this information.
  • Request the facts and ask questions if you don’t understand. To participate in a clinical trial that requires an IND application, you must sign a consent form that explains the experimental procedure. The consent form also identifies the Institutional Review Board (IRB) that assures the protection of the rights and welfare of human subjects. Make sure you understand the entire process and known risks before you sign. You also can ask the study sponsor for the clinical investigator’s brochure, which includes a short description of the product and information about its safety and effectiveness.

Based on all the evidence I’ve seen to date, exosomes engineered to carry the right ratio of growth factors (not simply derived from mesenchymal stem cells) seems most likely to lead to therapeutic benefit in SCI. Yet huge barriers still exist for scalability, safety, dose, and determining what the ideal ratio of growth factors will actually lead to functional improvements. While we all want these barriers to be resolved ASAP, participating in unapproved therapies are unlikely to result in any functional improvements, and likely risk more serious complications. We urge all caution in approaching experimental treatments that are not part of a regulated and monitored clinical trial.