Updated: Sep 22, 2022
The phrase “bench to bedside” describes the process by which the results of research done in the lab are directly used to develop new ways to treat patients. This is exactly what we do at Hope Biosciences with our adipose-derived mesenchymal stem cell product, HB-adMSCs.
Our mission in the lab is to develop adult stem cell-based therapeutics that will address unmet clinical needs. Consistent with that mission, we focus on making our technology available for use in FDA-authorized clinical trials to gain the regulatory approvals necessary to make stem cell therapy more broadly available to patients. We also understand that there are seriously ill patients who will not be eligible for clinical trials and may not have other treatment options. In these circumstances, stem cell treatment outside of a clinical trial may be possible through the FDA-regulated Expanded Access Program.
In the United States, there are three ways one can gain access to stem cell therapy. These treatment pathways include Clinical Trials, Expanded Access, and Right-to-Try. So, how do the study types differ?
Clinical trials are controlled studies that measure the safety and effectiveness of new treatments through consenting participants; in other words, they are designed to test whether drugs or treatments are safe and effective in people. Clinical trials are a lengthy, five-stage research process that begins with cellular studies, progresses to animal testing, then moves on to testing in humans if the research appears to have potential. A study must pass the human testing phase before the FDA will approve the treatment for legal prescription by doctors to patients. Clinical trials in stem cell research are strictly regulated and monitored by the FDA because the FDA has classified stem cell-based products as drugs. Patients volunteer for clinical trials to potentially try a new treatment and/or to promote research. Clinical trials include both a treatment and a placebo group, so there is no way to guarantee that a given participant will receive the investigational treatment.
Clinical trials follow strict scientific and quality standards to protect patients and produce reliable study results. Trials have eligibility requirements, including inclusion criteria (things required to participate in the study) and exclusion criteria (factors, conditions, etc. that prevent a person from participating). Every clinical trial has unique requirements.
Expanded Access, otherwise known as “compassionate use”, is an FDA regulated pathway for patients with serious and life-threatening conditions. Studies of this kind allow a doctor to request experimental treatment directly from a drug manufacturer on behalf of a patient who has exhausted all other treatment options and who is not eligible for existing clinical trials. An application must be approved by both the FDA and a third-party Institutional Review Board (IRB). Once approved, the drug manufacturer must agree to make the drug for the patient.
Right-to-Try is an access decision between a patient, their doctor(s), and a drug manufacturer made only for life-threatening diseases and conditions. This option is designed for patients who have exhausted all treatment options and are not eligible for a clinical trial or are too far away from a trial site. Requests are made directly to the drug manufacturer at a physician’s discretion, without FDA approval or oversight.
What option(s) a patient may be eligible for can depend on whether a physician diagnoses a current disease or condition as either “serious” or “life threatening.” A serious disease or condition is defined as one that has a substantial impact on day-to-day functioning. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress in severity. A classification of “immediately life threatening” indicates a stage of disease in which there is reasonable likelihood that death will occur within a matter of months, or in which premature death is likely without early treatment.
Currently, all clinical trials that utilize Hope Biosciences’ HB-adMSCs are managed by the Hope Biosciences Stem Cell Research Foundation (HBSCRF). HBSCRF, headquartered in Sugar Land, Texas, exists to revolutionize medicine by accelerating translational research in regenerative medicine to develop cures for all. HBSCRF is administered through the Greater Houston Community Foundation (GHCF). If you would like to learn more about our clinical trials or how our cells are being used to treat a variety of indications, information can be found at hopebio.org.