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In America, autism is estimated to affect 1 in 54 children, and some parents are using a controversial treatment in an attempt to help their kids -- stem cells.
Stem cells from cord blood are said to decrease inflammation, stimulate the brain, replace damaged neurons, and improve function, but scientific evidence backing up these claims is lacking. Duke University is conducting clinical trials testing cord blood for autism and supervising investigative producer Leslie Marcus sat down with the parents Rich and Ashley, whose son Richie was part of the initial study.
They say they noticed autism markers when their son was 2 years old explaining he had delayed speech, delayed potty training, and the way he interacted and played with others was different. The parents also say he was hit by a bus driver while in preschool and was not able to tell his parents. They had hoped being involved in the clinical trial might help him express himself and become more independent. But around 2 to 3 days after his first infusion, they say their son became much more violent and angry, regressed, began hand flapping and began having recurring fevers.
"It was like having a stranger in our house," mom Ashley says. "It was sad, it was very isolating for him." When they contacted Duke University and explained they believed their son had a negative reaction to treatment, they say a nurse said no one else in the trial was having a similar experience and Rich and Ashley began to think the study might not be related to what was happening to their son. They also tell The Doctors they were never provided the results of the study prior to it being made public, something they tell us Duke assured them they would be given.
To address their son's setbacks, they used multiple types of therapy like ABA therapy (applied behavior analysis), occupational therapy, speech therapy, a self-contained school program, and a team of doctors -- including a psychiatrist and psychologist.
Rich and Ashley are happy to share that their son, who is now 13, "has made a huge amount of progress" and they say there is still work to do, but feel they are headed in the right direction.
The Doctors also speak with Gina and Wade, whose daughter Gracie was involved in the same Duke study. They share that 6 months after she began stem cell therapy they saw remarkable and robust improvement in their child with changes to her verbal and socialization skills. Gracie, who is now in 7th grade, tells us she loves school and is getting good grades.
We also speak with a family, who went abroad to Panama and spent $300,000 dollars on autism stem cell treatments for their son and they tell us the results were a miracle. See their story and their child before and after his infusion, HERE.
Psychiatrist Dr. Ish Major explains it is possible for each case to have dramatically different results and it is nearly impossible to predict the outcome of a clinical trial.
To help parents Rich and Ashley continue with their son's ABA benefits they need an updated autism diagnosis and The Doctors have arranged for an evaluation for son Richie with psychologist and autism expert Dr. Doreen Granpeesheh at no cost to them.
When contacted by The Doctors, the Duke University clinical trial said in a statement:
How is what Duke is doing different from an unproven stem cell clinic?
Duke is conducting multiple phase 1 and phase 2 clinical trials studying the safety and potential efficacy of cord blood- and cord tissue-derived cellular therapies. The clinical trials are conducted under Investigational New Drug (IND) applications through the FDA. The conditions under study include hypoxic ischemic encephalopathy (HIE) in newborns, cerebral palsy (CP) in young children, autism spectrum disorder (ASD) in children and young adults, acute ischemic stroke in adults, certain inherited leukodystrophies in children, primary progressive multiple sclerosis in adults, and COVID-acute respiratory distress syndrome (ARDS) in adults.
All clinical trials are conducted under IND from the FDA and are approved by the Duke Institutional Review Board (IRB). All participants or their parents/legal guardians sign written informed consent to enroll in the clinical trial. The consents are reviewed with the participants and their families several times in advance of the signing.
Cellular products under study include autologous and sibling cord blood, allogeneic unrelated donor cord blood, allogeneic cord tissue mesenchymal stromal cells (CT-MSC), and DUOC- a microglial/macrophage-like cells manufactured from allogeneic cord blood. Manufacturing of CT-MSC and DUOC occur in clean rooms in an FDA registered, FACT accredited, GMP laboratory at Duke under IND. Infusions are prepared and tested in the Duke Stem Cell Laboratory, an FDA registered, FACT and CAP accredited, CLIA certified clinical laboratory of Duke Hospital.
Autologous and sibling cord blood units are obtained from private/family cord blood banks where the family banked the cord blood at birth. Autologous and sibling cord blood units must meet specifications including sufficient cell doses, negative donor screening and testing, negative sterility testing at the time of banking. They are further qualified with identity and potency testing before shipment to Duke. Allogeneic cord blood units are obtained from FDA licensed public cord blood banks including the Carolinas Cord Blood Bank at Duke, which has been FDA licensed since 2012, and is also FACT and CAP accredited. All cord blood units were thawed and washed on an automated device utilizing a standardized operating procedure and tested for sterility, cell count recovery, viability, and viable CD34 cell content after thaw.
Cell infusions are performed in controlled medical settings, specifically, in infusion centers experienced in administering cellular products and monitoring for adverse events. All participants receive infusions in an outpatient infusion center. All participants are given pre-medications to prevent infusion reactions. All participants are monitored with oximetry, blood pressure and temperature during, and for a minimum of 1 hour after, their infusion. If a participant experiences an infusion reaction, it is promptly treated. A physician, nurse practitioner and registered nurse are present during every product infusion.
Duke developed a screening protocol to gather preliminary information from participants or parents who have reached out to the center exploring whether there is a clinical trial that they/their child could participate in. The screening, under an IRB protocol, allows for collection of initial demographic information and then, with informed consent, medical information about the participant and, if applicable, their family banked cord blood unit. The screening protocol also allows for testing, if applicable, to further determine study eligibility. If a participant appears to be potentially eligible for a clinical trial that is actively enrolling, the participant/family is approached with specialized questionnaires and testing to further determine eligibility for that trial. Study team members, clinical research nurse coordinators, physicians, and nurse practitioners interact with the participant/parents during this period by phone and zoom to provide information about the study and to gather additional testing results.
Nineteen clinical trials have been conducted over the past 10 years. Currently, six clinical trials are actively enrolling patients.
All clinical trials collect extensive data regarding safety and efficacy endpoints for two years or more. The phase 2 clinical trials are randomized, placebo-controlled studies with cross-over points so that all patients are assured treatment with investigational product at some point during the study. All trials involve joint participation with cell therapy experts and specialists in the disease under study, thus all study teams are multidisciplinary. The results of all clinical trials are presented at medical conferences and published in peer-reviewed medical publications.
In addition, Duke has an expanded access program under FDA IND to provide access to infusions with autologous or full or partially HLA-matched sibling cord blood to children with CP, HIE or ASD who have eligible cord blood units and who are not eligible for ongoing clinical trials.
Do you and the Panama Stem Cell Institute have ties in terms of receiving funding from the same donor?
Duke has predominantly received funding from the Roberson Foundation and the Marcus Foundation. There are no ties of any kind between the Duke and Panama programs.
Does the Marcus foundation get any of the money generated from the EAP?
No. Also, there are no profits generated from EAP. The charges for EAP cover the costs of determining eligibility for the patient and their cord blood unit, testing potency, and confirming identity of the cord blood unit, thawing, washing, preparing and testing the cord blood unit for infusion, testing of the patient, clinic visits to assess history physical, routine lab tests, and general medical eligibility, pre-medications, IV placement, monitoring during infusion, and follow-up for monitoring of safety and parent-reported efficacy.
How is Duke/Cryo-Cell's plan different than commercialization via an EAP?
There is no plan by Duke to commercialize via EAP. Commercialization will proceed along conventional FDA commercial development pathways with phase 1, 2, 3 clinical trials, and BLA applications. EAP can provide supportive data, predominantly for safety, to support the BLA application(s).
How will recruiting thousands of kids with autism off-study via an EAP impact Duke's ability to run solid clinical trials in this area?
Duke does not recruit children for the EAP. Duke is only providing access to this therapy through EAP for children who are not eligible for ongoing clinical trials and who have qualified autologous or sibling cord blood units, and it is determined they might benefit from this investigational treatment as part of clinical care. In general, this represents <10% of patients/families who reach out to us.
Why do patients have to pay for access to experimental stem cell-related infusions through Duke and Cyro-cell?
Duke’s charge for participation in EAP covers costs of standard care required to screen and test the cord blood unit and the patient for eligibility, perform a history and physical and routine lab testing, place an IV, give pre-medications, thaw, wash, test, and prepare the cord blood for administration, administration of the cord blood in the infusion center, observation of the patient during administration, further observation and IV fluids post cord blood infusion, follow up with the patient/family at 1 day, 2 weeks, 3, 6, 12 and 24 months post-infusion for safety assessments.