Today, leadership from Brainstorm Cell Therapeutics announced results from their recently completed U.S. phase II stem cell study of NurOwn® in patients with ALS through a press release and webinar. Below we provide some detail on this study.
The Brainstorm stem cell trial is based off of NurOwn, which is a cell therapy platform centered on mesenchymal stem cells derived from bone marrow samples given by the participants in the trial. They are induced to secrete neurotrophic factors (MSC-NTF), which are a type of nutrient for cells that was previously show to have protective effects in animal models of neurodegenerative disease.
Researchers at Neuralstem Inc. are investigating a potential ALS therapy that involves injecting stem cells into a person’s spinal cord. This week, the results of the phase II study that tested the safety of this type of stem cell transplantation were published in the journal Neurology. Below we give some background on this latest study.
Meet Dr. James Connor, an ALS researcher who leads a team at Penn State Hershey Medical Center in Hershey, PA that recently received an ALS Association Investigator-Initiated grant to move his exciting research forward. The ALS Association spoke with Dr. Connor about his investigations into how a therapeutic iron solution is potentially protective in ALS and how collaboration at Hershey bridges the lab and the ALS clinic.
What has the support from The ALS Association meant to you and how has your Investigator-Initiated grant pushed your project forward?
The support helps us on so many levels. It excites us, invigorates us and validates us. But at same time, there is a strong sense of responsibility because this is money from someone that was most likely touched by this devastating disease. That level of trust means a lot and heightens our work. It is a privilege to work on the brain and ALS and to have some ideas that could potentially help someone that is suffering. When we are working 60 hours a week and are tired and working on a weekend to do an experiment, knowing where that award money came from to do our work invigorates us.
Do you have a message for the donors that helped make this project possible?
We always tell our donors that our job is to provide the hope. When a person goes into the doctor’s office and gets the devastating diagnosis, clinicians are obviously wonderful working with the person to deal with the disease. We want to be the element of hope to come in and say that our study is working and hopefully this will be relevant and helpful to the patient. In return, donors are instilling their hope in us, which is a tremendous gift and honor. Also, we have lots of great ideas, but without the funding, they are just ideas. So donors are part of our implementation team. Donors join our team! When we receive a grant, we get excited to go work to do our experiments. Otherwise, people get frustrated because they cannot afford to do the experiments they want to do.
“We are in a time of great hope in ALS therapy development. The efforts highlighted here are poised to accelerate progress in clinical trials and to bring new treatments to people living with ALS.” – Barb Newhouse, President and CEO, The ALS Association
Collaboration is a cornerstone of The ALS Association’s global research program. That’s why The Association hosts an annual ALS Drug Company Working Group that brings together representatives from pharmaceutical companies and academia to discuss how to move ALS research and therapy development forward.
This year, the Working Group was held at the 68th Annual American Academy of Neurology (AAN) Meeting in Vancouver in April. Below are some of the highlights from the meeting.