Hope Biosciences has reportedly received FDA authorization to conduct a pediatric clinical trial using their adipose(fat)-derived mesenchymal stem cells (HB-adMSCs) to assess their impact on juvenile idiopathic arthritis (JIA) in children aged 2 to 16.
This trial aims to determine whether intravenous (IV) infusion of these stem cells can alleviate symptoms and enhance the quality of life for affected children.
Hope Biosciences reports that their cell therapy has been used in conditions related to JIA, such as rheumatoid arthritis, chronic musculoskeletal pain, severe osteoarthritis, psoriatic arthritis, and lupus. The company states that previous protocols have indicated that HB-adMSCs are safe and may lead to significant improvements in pain levels and functionality.
Juvenile Idiopathic Arthritis (JIA), previously known as Juvenile Rheumatoid Arthritis, is an autoimmune disease with an unknown cause, affecting an estimated 300,000 children in the United States and approximately 3 million globally. The condition is characterized by chronic or cyclical joint pain in both small and large joints, often accompanied by fatigue, rash, blurred vision, fever, and appetite changes. Current treatments primarily focus on symptom management.
The Phase II clinical trial (NCT06623240) is being conducted by Hope Biosciences Research Foundation (HBRF) at their Sugar Land, Texas site and is currently enrolling participants. The company says the trial will use a balanced, randomized, double-blind, crossover design, involving an 8-week active treatment period with three infusions, followed by a 12-week period without treatment, and then another 8-week period with three more treatments.
Approximately half of the participants will receive treatment first, followed by a placebo after the washout period, while the other half will receive the placebo first, followed by treatment.
There is no cost for the treatment, but travel expenses to the site are not covered. For more information, visit hopebio.org, email HBRF at clinical@hopebio.org, or call (346) 900-0340, ext. 101.
Publisher’s take:
Based solely on this press release by Hope Biosciences, this is a very interesting trial. Even though it’s only Phase II, which is par for the course for the industry currently, the study design and the fact that the trial doesn’t cost any money are great. It should be done this way, yet it’s often not. Stem cell companies have a history of selling their treatments without any trials, using very weak trials and putting the cart before the horse, or worse yet, registering a trial as a marketing stunt to get patients in the door who pay full price. In the case of Liveyon, they did the latter, blinding a few people, and the founder is in federal prison now.
Additionally, the use of adipose (fat) stem cells is at a crossroads. It’s shown some promise in trials, such as the Mayo Clinic’s spinal cord injury trial, but it has run into many legal issues because of how it’s made.
In short, you take a patient’s fat which contains stem cells, but you need to process it to isolate them. Part of that process involves enzymes, which could alter the characteristics of the orthobiologic, and the FDA sees that as a risky question mark.
One of the bars the FDA has set is that the product must be “minimally manipulated”, and often using an enzyme is considered more than that. If it crosses that line, the product begins to be regulated like a drug, which requires a lot more regulatory hoops (known as a 351 HCT/P).
This has hit the courts; for instance, in USA v. California Stem Cell Treatment Center, the FDA eventually reversed the initial decision, finding that the enzyme usage is more than minimal manipulation, and put it through stricter regulations.
More recently, Florida Republicans pushed two bills to legalize umbilical stem cells in the Sunshine State, which specifically excluded adipose stem cells. It seems like the goalpost is changing.
“Our technology has safely treated children as young as 18 months with conditions ranging from congenital muscular dystrophy to cerebral palsy, epilepsy, and autoimmune disorders,” said Donna Chang, CEO of Hope Biosciences. “We are confident that the same technological advancements that have delivered consistent, high-volume, and repeatable cell therapy treatments in adult trials will pave the way for effective treatments in pediatric patients. Encouraging results from our adult rheumatoid arthritis trials, where participants showed significant and lasting improvements in joint pain and function, give us great hope for similar success in children with juvenile arthritis.”
