One of the great advances in medicine is regenerative therapy and stem cell treatments. They are relevant numbers of products and therapies, several of them have been approved by the FDA but many more are in the trial phase.

Stem cell unapproved therapies allowed

The American government has approved the law “Right to Try” after a long bioethical and medical debate (read HERE). Opponents have argued that this law could “provide false hope to patients” and undermine the F.D.A.’s authority. Indeed several regenerative clinics have been fined and sanctioned for medical fraud in their marketing campaigns read more HERE and FDA wins major victories in the campaign against stem cell clinics that marketing bogus and potentially hazardous products.

The Right to Try law establishes several conditions for a patient to opt for still not approved drugs (in trial phase) one of them is the fail or intolerance of approved treatments.

The issue continues to raise ethical and medical concerns. In this respect, a study published by PEW (June 1, 2021) – The official site of the prestigious Pew Charitable Trusts entitled Harms Linked to Unapproved Stem Cell Interventions gives an overview of problems related to the use of therapies and drugs that have not been approved.

We excerpt what, in our opinion, is of more interest from a bioethical point of view. The study begins by reporting the number of stem cell clinics in the US. “More than 700 clinics in the U.S. offer unapproved stem cell and regenerative medicine interventions (SCRIs) for conditions such as Alzheimer’s disease, muscular dystrophy, autism, spinal cord injuries, and, most recently, COVID-19. The products made and sold by these businesses typically use cells from the patient’s own body or from donated amniotic fluid, placental tissue, or umbilical cord blood, among other birth-derived cells and tissues. FDA has given manufacturers and marketers of SCRIs until May 31, 2021, to come into compliance with the agency’s regulations governing human cell and tissue products, including submitting those products for FDA review when necessary.”

The article continues showing a certain control of FDA in these experimental therapies but also reports the difficulties to know all the adverse effects of them. “FDA uses reports of adverse events—undesirable patient experiences associated with the use of medical treatments—to help identify potentially dangerous products meriting further investigation. However, adverse events associated with any medical product are generally underreported, and providers of unapproved treatments are particularly unlikely to report patient harm to regulators or disclose such cases through public reporting— making it more difficult for the agency to prioritize its enforcement actions, and also leading policymakers and the public to underestimate the risks of these interventions.”

Reports of adverse events in the experimental treatments between 2004 and September 2020

For the reasons reported above,  the recent field investigations data has made PEW’s researchers have a particular interest. The report gives first the methodology used in the investigation. The author says, “To create a clearer picture of the risks that these interventions pose and underscore the need for increased FDA oversight, The Pew Charitable Trusts gathered reports of adverse events (AEs) linked to unapproved SCRIs administered outside of clinical trials. In total, we identified reports of 360 people who had AEs that occurred between 2004 and September 2020, including:

  • 334 cases documented in peer-reviewed journals, government and news media reports, and other literature.
  • 5 cases reported in FDA’s adverse event reporting system (known as FAERS), a public database of reports submitted to the agency.
  •  21 cases described in consumer reviews of stem cell businesses posted to Google, Yelp, and Facebook.”

From the 334 AEs, the total number of subsequent deaths was 20.

The study described the kind of adverse events – AEs reported “Many of the AEs identified involved serious bacterial infection, including at least two cases of septicemia, a life-threatening blood infection. Others included serious and even lifelong disabilities such as partial or complete blindness (9); paraplegia (1); pulmonary embolism (6); cardiac arrest (5); tumors, lesions, or other growths (16); and organ damage or failure in several cases that resulted in death. Many of these AEs required hospitalization (104) and caused acute or worsening pain (55). The most common type of interventions linked to these AEs was autologous (that is, the stem cells were obtained from the patient’s body) or donor stem cells administered by injection into the eye, spine, hip, shoulder, or knee.” The researcher continues with valuable findings and considerations that our readers could read in the full report, click HERE.

Conclusions of stem cell unapproved therapies current offered in the US

Following we cite the full conclusions of the report, “As FDA’s enforcement discretion period draws to a close, the agency should maintain pressure on businesses offering unapproved products and ensure that patients are protected from SCRIs that have caused harm or have the potential to cause harm. Encouraging patients and clinicians to report AEs and devising effective strategies to collect more real-world evidence of harm can help the agency in its efforts to curb the growth of this unregulated market and ensure that the regenerative medicine field develops into one that clinicians and patients can trust and safely access.”

Our Observatory statement is that Right to Try laws should be maintained, they support the patient rights but as says the aforementioned article the control of FDA of stem cell clinics and related business should be incremented to reduce AEs and in certain cases forbid some treatments that show more incidence in serious adverse effects.

 

 

 

 

 

 

 

 

 

Harms Linked to Unapproved Stem Cell Interventions Highlight Need for Greater FDA Enforcement

https://www.pewtrusts.org/en/research-and-analysis/issue-briefs/2021/06/harms-linked-to-unapproved-stem-cell-interventions-highlight-need-for-greater-fda-enforcement

https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try#:~:text=The%20Right%20to%20Try%20Act%20permits%2Fallows%20eligible%20patients%20to,is%20a%20patient%20who%20has%3A&text=And%20has%20provided%2C%20or%20their,drug%20to%20the%20treating%20physician