Researchers at NYU Langone Health have pulled out of a trial investigating the use of an old tuberculosis vaccine to treat children with Type 1 diabetes only months after they began enrolling participants on Long Island.
The vaccine, called Bacillus-Calmette-Guerin, or BCG, has generated intense interest among various patient advocacy groups, including those focused on Alzheimer’s disease and cancer, as well as diabetes. Some recent research suggests that the vaccine, first administered in 1921, also may protect against Covid-19 and respiratory diseases because of its broad effects on the immune system.
A large trial of the vaccine’s effects on blood sugar in adults with Type 1 diabetes is nearing completion. Scientists had hoped to test the BCG vaccines on children as well, since good management of Type 1 diabetes leads to fewer complications related to diabetes.
The lead investigators of the pediatric trial, who are at Massachusetts General Hospital in Boston, are proceeding with the study, but NYU Langone’s abrupt withdrawal could potentially jeopardize its viability if they are unable to collect data on the children at the NYU site.
A total of 150 children were to be included in the study, and the NYU team had already given the vaccine to at least 18 youngsters. They were to be followed for five years; each was to receive eight safety-check visits in the first year after their inoculations.
“Who’s monitoring our daughter medically now, and who’s monitoring the injection site?” said Kevin Miller of Smithtown, NY, whose 14-year-old daughter enrolled in the study last year.
“I’m sure there is something in the fine print of all the papers we signed that says they have a legal right to do this,” Mr. Miller added, “but I don’t necessarily know that means it’s right to leave kids like my daughter and others hung out to dry.”
NYU Langone officials said in a statement that an institutional review board, which approved the study in December 2020, decided to end participation after reviewing more research about the BCG vaccine.
A statement said that the university “determined that withdrawal from the study will not affect the safety of enrolled participants,” and said that the children could continue to see NYU physicians for their usual diabetes care.
An earlier study by the scientists at Massachusetts General found that two doses of BCG vaccine reduced blood sugar levels to near normal in a very small group of adults with Type 1 diabetes. Other studies of BCG, using different versions of the vaccine and following Type 1 diabetes subjects for varying periods of time, have yielded mixed results.
The new trial is Phase II, aimed at evaluating the efficacy of the vaccine treatment. Usually when such trials are stopped, it is because of a safety concern. Enrollment of new participants ceases, but subjects who have already received the intervention continue to be followed.
NYU has made no mention of a safety problem, however, and will no longer follow the participants for purposes of the study.
The lead investigator of the trial, Dr. Denise Faustman, director of the immunobiology laboratory at Massachusetts General Hospital, said that NYU’s withdrawal was a serious deviation from trial protocol that may call into question the final results.
“The Food and Drug Administration cares most about safety in children and the protection of children, and to miss any safety visit is a serious violation of the protocol,” she said.
Controlling blood sugar in patients with diabetes is critical in preventing serious long-term complications such as vision loss, nerve damage, kidney disease and amputations. One hypothesis is that BCG may tamp down the production of abnormal white blood cells that attack the body’s own tissues, including insulin-generating cells in the pancreas.
The idea has long been controversial, and Dr. Faustman has struggled to raise money to support her studies. In 2018, when she published the research suggesting that BCG lowered blood sugar levels, the JDRF — formerly the Juvenile Diabetes Research Foundation, a major funder of research — and the American Diabetes Association took the unusual step of issuing a joint public statement expressing skepticism about the findings.
In response to questions about his position on Dr. Faustman’s research into the BCG vaccine, Chelsea-Lyn Rudder, a spokeswoman for JDRF, said that the foundation’s position “remains unchanged.”
“JDRF prioritizes research funding that has the highest likelihood of accelerating the delivery of therapies to cure and prevent Type 1 diabetes,” Ms. Rudder added, “while maintaining strategic funding in research that improves the lives of those with the disease.”
The BCG vaccine is more than a century old and generic, and drug makers have shown little interest in investing in research and development related to it. Newer treatments often carry eye-popping prices. A newly approved drug, teplizumab, may delay the onset of Type 1 diabetes by a few years but costs nearly $200,000 for a two-week treatment course.
Though NYU officials did not explain the review board’s decision to halt the trial, a letter from Massachusetts General Hospital responding to the NYU action references two scientific papers, both of which reported ambiguous results and called for carrying out more randomized controlled trials on BCG vaccines in people with type 1 diabetes.
The review board at Massachusetts General said that the two papers that were brought to their attention did not raise any concerns about an “unanticipated problem” and that there was no reason to stop Dr. Faustman’s trial.
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