Northwestern Medicine’s immunotherapy and autoimmune diseases program, by all appearances a successful clinical research program involved with the testing of hematopoietic stem cell transplantations (HSCT), will cease to operate. The official reason: its’ chief, Richard Burt, will resign as he takes a research sabbatical to teach the HSCT protocol he developed around the US as well as write a book. Strange, however, that a dynamic and success clinical investigative site focused on some of the most advanced investigational therapies would shut down just because its’ leader is leaving.
Katarina Zimmer writing for The Scientist broke this story which included some exchanges with Professor Burt.
Why Couldn’t the Clinical Research Site Continue?
Ms. Zimmer weaved this important point directly into her story. After all, good systems and processes along with a competent management team in a thriving active and quality-driven clinical investigative site can ensure ongoing operational integrity. Their clinical research department appeared to many to be a program with staying power. The Scientist article included a comment from Mark Freedman, a neurologist directing research for multiple sclerosis at Ottawa Hospital noted: “if this research program is so successful [why] it should suddenly stop if he’s not there is a little bizarre.”
A Successful Study?
Burt had recently reported positive results from a clinical trial targeting multiple sclerosis. This study, one of a handful worldwide, evidenced that HSCT could potentially be a contender treatment for multiple sclerosis drugs in a majority of patients succumbing to relapse post-first-line standard treatment. In fact, Ms. Zimmer reported that actress Selma Bair reported on Instagram that she can “walk much better” because of participation in the HSCT clinical research trial although still recovering from the chemotherapy used to deplete her immune system before the stem cell treatment.
Patients Found out Via Facebook
Facebook, as TrialSite News can attest, is perhaps the most powerful patient group platform worldwide. The Northwestern Chicago HSCT Stem Cell Transplantation—Official Patient Run appeared to be how patients were notified about the closure. This seems a little strange. Clinical investigational sites are governed by the need for stringent FDA-compliant systems and processes—known as “Good Clinical Practices” or GCP. These organizations set up policies and procedures for most everything. Typically there is a process and set of procedures and work instructions prepared for a major event such as the closure of the facility. Would it be in the policy to notify patients by Facebook? Perhaps each and every patient received a formal letter of closure but that wasn’t uncovered by a very thorough journalist.
A Significant Warning from the FDA Just a Few Years ago
Apparently, as Ms. Zimmer for The Scientist dug up, Burt and his team have troubles with the FDA just a few years ago—November 2016—receiving an FDA Warning Letter declaring a failure to ensure that the investigations were conducted following the general investigational plan and protocols contained in the IND. Violations were significant and represented evidenced by an investigator-initiated study that was struggling with quality controls—with the findings it was conceivable that the clinic was not led and conducted with quality principles top of mind. Although Burt ultimately followed protocol to response—e.g. identify root cause analysis and evidence a corrective action plan, clearly, an FDA inspection caught a number of substantial problems that could be perceived by some quality professionals that Burt’s clinic lacked a quality-driven culture.
FDA Warning not Associated with Closure
The Scientist was able to confirm from Burt that the research site’s closure had nothing to do with the FDA warning. He was quoted by Ms. Zimmer in an email “In December 2016, the FDA sent a follow-up letter saying our response was satisfactory and that there were no further actions or questions. The FDA found no treatment-related deaths in any of the studies”
Did some Patients Pay $150,000 to Participate in the Northwestern Stem Cell MS Trial?
Apparently a stem cell biologist at the University of California, Davis raised some concerns about select practices at Burt’s clinic on his blog. Paul Knoepfler is a known cynic of some stem cell treatments and practices. For example, he is quoted in his blog back in 2017 that “Some patients have said on the internet that they are required to pay as much as $150,000 for participation in the Northwestern MS stem cell trial or in parallel off -study experimental administration of experimental stem cells.” Knoepfler noted that it doesn’t violate FDA rules to charge for a clinical trial but it can be considered a questionable practice on ethical and moral grounds—e.g. it precludes huge numbers of patients based on income/economic class, etc. Ms. Zimmer probed local news and found an insurance company denied Northwestern’s clinic HSCT treatment coverage at the clinic (outside of clinical study) to multiple sclerosis patients as it is “experimental.” At least in one case family members took a second mortgage on their home pay for the study reported the article.
Official Position of Northwestern
Christopher King, Director of Media Relations and Communications at Northwestern Memorial Healthcare noted in a statement to The Scientist “Northwestern Medicine is grateful for Dr. Burt’s pioneering achievements in this field and the care he has provided to his patients.” King emphasized all patients will complete their treatment at the center prior to closure.
First, we recommend reading Ms. Zimmer’s entire story in The Scientist, an excellent publication, at the link to source below. Dr. Burt appears to be on a mission to take his research protocols nationwide as well as write a book. More than likely the Northwestern clinic was really driven by Professor Burt—his vision and mission—and he is the propelling force behind all that went on. This can be common in scientific endeavors where the lead visionary is at the helm. Moreover, as stem cell research represents a complex and risky research endeavor clearly if the visionary is moving on then the impetus to keep going may likely diminish. The individual must take control of their healthcare. Patients should carefully scrutinize any clinical research regardless of how prominent an institution. When a clinical investigator is charging to participate in the study the patient should get a second if not a third opinion—speak with physicians in the specific field of the study to get an objective third-party but expert opinion. Do you need help? We are building TrialSite Network concierge services just for that purpose. The goal is to connect patients with the best and more targeted clinical researchers and physicians in their respective fields to empower individuals with relevant, timely and meaningful information that can help them make the best possible decision for their personal health. We are on a mission to raise the bar in health care when it comes to clinical research!Source: The Scientist