Cell therapy weekly: First FDA-monitored clinical trial for the use of stem cells in hypoplastic left heart syndrome patients
This week: Japanese pharma company offers AUS$202 million for Cynata Therapeutics (Australia) and US$3 million grant for research into mesenchymal stem cell-based Alzheimer’s therapy.
The news highlights:
First FDA-monitored clinical trial for the use of stem cells in hypoplastic left heart syndrome patients
Japanese pharma company offers AUS$202 million for Cynata Therapeutics
US$3 million grant for research into mesenchymal stem cell-based Alzheimer’s therapy
First FDA-monitored clinical trial for the use of stem cells in hypoplastic left heart syndrome patients
A first-of-its kind, FDA-monitored Phase I clinical trial has demonstrated the potential for autologous cord blood as a treatment for hypoplastic left heart syndrome (HLHS) in children. HLHS is a severe, congenital heart condition resulting from maldevelopment of the left-hand side of the heart, often requiring multiple reconstructive surgeries. The unique trial involved ten patients diagnosed with HLHS in utero. The blood was processed by the Mayo Clinic’s Human Cellular Therapy Laboratories and ReGen Theranostics (both MN, USA) to yield a personalized, concentrated stem cell product that was injected back into the patients at the time of their second reconstructive surgery. No deaths or adverse health events were observed in the patients during the 6-month post-surgery follow-up period. Planning for a Phase IIb clinical trial is now underway to assess the efficacy of the therapy for improving patients’ heart strength.
Harold Burkhart, first study author and a Pediatric Cardiothoracic Surgeon at OU Medicine (OK, USA), commented: “We now have a reproducible protocol to utilize stem cell therapy in babies with HLHS. Our hopes are that this groundbreaking research will lead to stem cell therapy strengthening these babies’ hearts while delaying or even preventing the need for a heart transplant in some.”
Japanese pharma company offers AUS$202 million for Cynata Therapeutics
Sumitomo Dainippon Pharma (Osaka, Japan) has offered to buy the Australian stem cell therapy biotechnology company Cynata Therapeutics (Victoria, Australia) for AUS$202 million.
The offer appears to be an attempt by Sumitomo to expand their advanced therapy business following from their 2018 financial statement in which they referred to the regenerative and cell therapy fields as critical areas of future research and development.
US$3 million grant for research into mesenchymal stem cell-based Alzheimer’s therapy
Longeveron (FL, USA), an age-related cellular therapies biopharmaceutical company, has been granted US$3 million by the Alzheimer’s Association (IL, USA) for their mesenchymal stem cell (MSC) Alzheimer’s research. The prestigious, competitive grant aims to support, and accelerate the production of, promising therapies for the treatment and/or prevention of Alzheimer’s disease. Longeveron’s therapy utilizes adult MSCs to target neuroinflammation in patients with mild Alzheimer’s disease. Longeveron is currently recruiting patients for its Phase I clinical trial of their MSC therapy.
Geoff Green, President of Longeveron, commented: “We have seen in our Aging Frailty clinical trials the potential of stem cells to reduce inflammation, which has been identified as a potential factor in the progression and acuity of Alzheimer’s disease. We are honored to be named as a recipient and would not be as far along in our Alzheimer’s stem cell clinical research without this major support from the Alzheimer’s Association.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.