Fighting to Start a Clinical Trial

For over a decade, our founder and leader, Ryan Benton, was dedicated to advocating for the start of a Mesenchymal stem cells (MSCs) clinical trial for the treatment of Duchenne muscular dystrophy (DMD). He knew the only way to make this therapy available for others with his terrible disease was for the therapy to gain FDA approval following a well-constructed clinical trial. Clinical trials are vital to the continued advancement of treatments and medical knowledge to help improve patient care. While we have years of firsthand experience witnessing the benefits of MSCs for DMD in numerous cases, this evidence is considered anecdotal, meaning it can not be considered scientific evidence that can be verified objectively. To allow this therapy to be taken seriously, an FDA-approved clinical trial is necessary, and starting a clinical trial can be a complex and challenging process. Unfortunately, Ryan passed before we could help start a clinical trial, but we will not stop our pursuit to make stem-cell therapy accessible and affordable to everyone. 

If clinical trials are so beneficial, why are they so hard to start? The United States Food and Drug Administration (FDA) oversees clinical trials to ensure patient safety and the efficacy of new treatments adhere to all of the regulatory requirements. The regulations are extensive and demanding making it difficult to navigate the complex framework of guidelines, protocols, and documentation to gain FDA approval which makes the process very time-consuming and lengthy. Below are some of the factors that need to be in place before the FDA will approve the trial.

Selecting a site for a clinical trial can be a major challenge. If the site selection is rushed, it can give inaccurate site information like staff workload, patient population, facilities and equipment at the site, and qualifications that are vital to ensuring the site has the capabilities to perform the research efficiently. This step is typically started very early in the start-up process to ensure there is enough time to fully asses each site and choose the one that is best suited to house the trial. Once a site has been chosen, they are required to schedule specific staff training before patient enrollment in the trial. It’s imperative that staff are properly trained, as inadequate training can lead to improper participant enrollment, poor data, or noncompliance which can lead to delays in the study. The sponsor also has to deliver the clinical supplies to the site prior to patient enrollment, so if there are shipping delays that can not only affect the timeline but also increase overall costs.

Internal review boards (IRBs), can also affect the timelines of trials. IRBs, also known as ethics review committees, are responsible for making sure the studies comply with applicable regulations, follow institutional policies, meet ethical standards, and protect research participants. If the review board finds issues with the trial during the review process, it can push back the start time. There are local and central IRBs. Local IRBs don’t meet as frequently as central IRBs, and if utilizing a central review board it can require less lead times and a review can sometimes happen quickly. All factors reviewed by these boards are crucial to allow for the successful execution of a clinical trial.

One of the most challenging factors in starting a clinical trial is the negotiations for contracts and budgets. Negotiating between sites and sponsors can take a considerable amount of time. To help expedite negotiations, sometimes organizations will hire a contract research organization (CRO) that has experience with these types of contracts. Sometimes CROs have worked with research sites in the past, and they can refer to the previous contract and budget terms which will help reduce the time spent negotiating.

So, while we worked tirelessly to help start the first large MSC clinical trial in the US for DMD while Ryan was still with us, these factors and many others make it extremely difficult to start a clinical trial. Ryan was passionate about having a trial here in the US based on his personal experience with the dramatic improvements in his health following his 19 treatments and feedback from others with DMD who have received stem cells. Knowing the impact these treatments had on Ryan’s health and quality of life strengthens our drive to continue to push for stem cell therapy to become part of the standard of care for others like Ryan.

While a trial hasn’t been started yet, we have made large strides in helping tackle many of the complex factors involved, and we are hopeful that soon we will be able to show just how much this form of therapy can truly change the lives of not only individuals battling DMD, but also change the lives of participants family, friends, and caregivers. If you want to follow along our journey of working to help start the first large MSC clinical trial for DMD, visit our website, sign up for our newsletters, and follow us on social media.