N.Y.U. Langone Health researchers withdrew from a study examining the use of an outdated TB vaccination to treat Type 1 diabetes in children just a few months after recruiting patients on Long Island.
The Bacillus-Calmette-Guerin, or B.C.G., vaccination has piqued the attention of several patient advocacy organizations, including those concerned with Alzheimer’s disease, cancer, and diabetes. A recent study indicates that the vaccination, which was originally given in 1921, may also protect against Covid-19 and respiratory infections because of its wide immune-boosting properties.
A big study examining the impact of vaccination on blood sugar levels in persons with Type 1 diabetes is approaching a conclusion. Scientists had wanted to test the B.C.G. vaccinations on youngsters, since proper treatment of Type 1 diabetes results in fewer diabetes-related problems.
The principal investigators of the pediatric experiment, who are located at Massachusetts General Hospital in Boston, are continuing with the study, but if they are unable to gather data on the children at the N.Y.U. site, the sudden departure of N.Y.U. Langone might threaten the trial’s sustainability.
The trial was to involve a total of 150 children and the N.Y.U. team had already administered the vaccination to at least 18 children. They were to be tracked for five years, with each participant receiving eight safety-check visits in the first year after vaccination.
Who is now medically monitoring our kid, and who is monitoring the injection site? Last year, the daughter of Kevin Miller of Smithtown, New York, whose daughter is 14 years old, joined in the research.
After examining more studies regarding the B.C.G. vaccination, the institutional review board that authorized the trial in December 2020 chose to discontinue participation, according to a statement from N.Y.U. Langone authorities.
The institution said in a statement that withdrawing from the trial would not compromise the safety of enrolled participants and that the children may continue to visit N.Y.U. doctors for their routine diabetic treatment.
In a previous trial, scientists at Massachusetts General Hospital discovered that two doses of the BCG vaccine decreased blood sugar levels to near normal in a small sample of individuals with Type 1 diabetes. Other trials using B.C.G utilizing various forms of the vaccine and tracking people with Type 1 diabetes for differing amounts of time have generated contradictory findings.
The new Phase II study aims to evaluate the effectiveness of vaccination therapy. Typically, when such studies are discontinued, it is due to safety concerns. No new participants will be enrolled, although patients who have previously received the intervention will continue to be monitored.
However, N.Y.U. has made no mention of a safety issue and will no longer monitor the participants for the research.
Dr. Denise Faustman, head of the immunobiology laboratory at Massachusetts General Hospital and the study’s principal investigator, said that N.Y.U.’s withdrawal constituted a severe divergence from the trial protocol that might cast doubt on the final findings.
“The Food and Drug Administration is especially concerned with children’s safety and protection, and missing any safety visit is a major protocol violation,” she added.
To avoid catastrophic long-term problems, such as blindness, nerve damage, kidney disease, and amputations, diabetic patients need to control their blood sugar levels. B.C.G. may inhibit the generation of aberrant white blood cells that target the body’s tissues, including insulin-producing cells in the pancreas, according to one theory.
The concept has long been contentious, and Dr. Faustman has struggled to get funding for her research. In 2018, when she published research indicating that B.C.G. reduced blood sugar levels, the J.D.R.F. — formerly the Juvenile Diabetes Research Foundation, a major research funder — and the American Diabetes Association took the unusual step of issuing a joint public statement expressing skepticism regarding the findings.
Chelsea-Lyn Rudder, a spokesperson for the J.D.R.F., said that the foundation’s stance on Dr. Faustman’s study into the B.C.G. vaccination “remains unaltered” in response to concerns concerning the foundation’s attitude.
Ms. Rudder continued, “J.D.R.F. emphasizes research financing with the best probability of speeding the delivery of treatments to cure and prevent Type 1 diabetes while preserving strategic support in research that improves the lives of individuals affected by the condition.”
The BCG vaccination is almost a century old and generic, and pharmaceutical companies have shown little interest in investing in its research and development. Oftentimes, expenses for modern procedures are exorbitant. Teplizumab, a recently licensed medicine, may postpone the development of Type 1 diabetes by a few years, but a two-week treatment session costs about $200,000.
The review board at Massachusetts General Hospital said that the two papers brought to their notice did not raise any worries about an “unexpected issue” and that there was no need to halt the trial of Dr. Faustman.
