Gaining Access to Stem Cells
A Quick Guide to Clinical Trials, Expanded Access Studies, and Right to Try
Hope Biosciences Stem Cell Research Foundation (HBSCRF) is currently carrying 16 FDA-authorized studies across 10 disease indications. Of these, five are clinical trials (COVID-19 prevention and treatment, Alzheimer’s disease, and traumatic brain injury) and eight are expanded access studies (amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), severe osteoarthritis, Parkinson’s disease, spinal cord injury, cerebral palsy, COVID-19 longhaulers and chronic musculoskeletal pain). How do the study types differ?
In the United States there are three ways to gain access to stem cells, called “treatment pathways.” The three treatment pathways are clinical trials, expanded access, and Right-to-Try.
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 tests, then moves to humans if the research appears to have potential. A study must pass through the thrid 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 treatment and placebo groups, so there is no way to guarantee that a given participant will receive the trial treatment.
Clinical trials follow strict scientific and quality standards to protect patients and product 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 studies are designed 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.
There are four types of Expanded Access studies. In emergency cases for a single patient, a request can be made to and approved by the FDA over the phone, provided physician agrees to submit a written application within 15 days of phone authorization (see FDA 21 CFR 312.305 and 312.310). In non-emergency cases for a single patient, the study request proceeds through written FDA approval channels. If several individual patients request access to the same treatment, an expanded access study can pursued with what is called an “intermediate patient population.” Finally, a “large patient population” study entails investigation in a controlled trial.
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. HBSCRF does not currently offer the Right-to-Try option; check out this blog article for more information from our Founder.
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.
HBSCRF is proud of our work to offer both clinical trials and expanded access studies to patients in need. Since the Foundation’s inception in March 2020, HBSCRF has administered nearly 100 billion stem cells through FDA-authorized studies, at no cost to patients.
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). As one of the leading philanthropic grant makers, GHCF is proud to partner with donors to create a meaningful and long-lasting impact in our community. Learn more at hopebio.org.
