It’s a difficult, but very important road to conclusively prove that an investigational stem cell therapy is both safe and effective in order to get final approval FDA or equivalents in other countries. For this reason, it is beneficial to highlight even small, but positive steps forward including encouraging pre-clinical data.
Today I’m doing a review of stem cell therapy good news in the clinical path with a focus on biotechs in particular (in alphabetical order), but starting first with news from here at UC Davis.
UC Davis Veterinary and Medical Schools team up in working to develop a new stem cell treatment for spina bifida with promising preclinical results in bulldogs. This great team includes veterinary surgeon Beverly Sturges, Department of Surgery Chair Diana Farmer, and stem cell researchers Aijun Wang and Dori Borjesson. See remarkable video below of the dogs and then I dive into the biotech news below that.
Asterias is expanding its SCI trial further. It announced “that Washington University School of Medicine in St. Louis, MO, has been added as a clinical site in the company’s ongoing SCiStar Phase 1/2a clinical trial of AST-OPC1, a stem-cell derived investigational therapy, in patients with severe cervical spinal cord injury (SCI).” See my recent posts on the company here.
Cynata has some stem cell good news. It reported encouraging, positive pre-clinical data in mice indicating that their Cymerus MSCs have efficacy for asthma in that model. This second pre-clinical study is a nice step forward toward future clinical trials in humans on this indication. I asked CEO Ross Macdonald about this development and here’s what I heard back:
“These preclinical data demonstrate that Cymerus™ MSCs alone or in combination with corticosteroids could provide an improved treatment option for asthma patients, without the side effects and resistance associated with steroid therapy,” said Ross Macdonald, Ph.D., Managing Director and Chief Executive Officer of Cynata Therapeutics. “We look forward to working with the Monash Lung Biology Network to complete preclinical analyses in other features of asthma, like inflammation and airway remodeling, to support a comprehensive preclinical data package and the anticipated advancement of Cymerus MSCs into clinical trials in asthma.”
Also, see my interesting chat with Macdonald from last month.
Mesoblast tells us via a PR that it sees an opportunity ahead for accelerated entry of its MPC-150-IM product for heart disease. I also asked CEO Silviu Itescu about how trials are going at the company and got this quote:
“Mesoblast is very encouraged by the progress being made in both of the Phase 2b/3 randomized controlled clinical trials using our allogeneic Stro-1/Stro-3 positive immunoselected mesenchymal precursor cells in patients with moderate/severe and end-stage heart failure. These trials build on a foundation of scientific rigor, and we hope that they will confirm the positive clinical signals seen with our technology in previously published controlled Phase 2 trials.”
See also my past posts on the company here.
ViaCyte has been getting some good press on its early trial in humans for pluripotent stem cell-based encapsulated pancreatic progenitors for diabetes. See my 2015 interview with CEO Paul Laikind. I’m showing the pic above of the ViaCyte product since I think it is so cool.
Overall, the stem cell and regenerative medicine arena remains a tough one for companies to flourish within, but these and other biotechs as well as universities like my own UC Davis are making progress in the marathon. I hope that new, in a sense experimental policy initiatives such as the FDA RMAT designation may help and not have too many downsides.
What are your favorite recent examples of stem cell good news and why?
Disclosure: I have no financial interest in any stem cell biotech and nothing here should be considered financial or medical advice.