Starting a clinical trial is like taking a step into the unknown. It’s moving a potential therapy out of the lab and testing it in people. To reach this point, the researchers have done a lot of work trying to ensure the therapy is safe. But that work was done in the lab, and on mice or other animals. Now it’s time to see what happens when you try it in the real world.
It can be quite nerve-racking for everyone involved: both the researchers, because years of hard work are at stake, and the patients, because they’re getting something that has never been tested in humans before; something that could, potentially, change their lives.
Today we have good news about one clinical trial we are funding: the Asterias Biotherapeutics spinal cord injury trial. Asterias announced that its Data Monitoring Committee (DMC) reviewed safety data from the first two groups of patients treated and found no problems or serious side effects.
That’s an important first step in any clinical trial because it shows that, at the very least, the therapy is not going to make the patient’s condition any worse.
The big question now is: will it make their condition better? That’s something we’ll come back to at a later date when we have a better idea of how the people treated in the trial are doing. But for now, let’s take a deeper dive into the safety data.
Asterias – by the numbers
This current trial is a Phase 1/2a trial. The people enrolled have all experienced injuries in the C5-C7 vertebrae – that’s high up in the neck – and have essentially lost all feeling and movement below the injury site. All were treated between two weeks and one month after the injury.
The therapy involves transplants of Asterias’ AST-OPC1 cells, which were made from human embryonic stem cells. The AST-OPC1 cells were turned into oligodendrocyte progenitors, which can become the cells that help protect nerve cells in the central nervous system, the area damaged in spinal cord injury.
The first group of three patients in the Asterias trial was given 2 million cells. The second group of five patients received 10 million cells. The DMC said the safety data from those patients looked fine and showed no signs of problems.
As Dr. Edward Wirth, the Chief Medical Officer at Asterias, said in a news release, this means the company can plan for its next phase:
“The positive safety data in the previous phase 1 study and in the ongoing phase 1/2a study gives us the confidence to now proceed to administration of 20 million cells, which based on our significant pre-clinical research is likely well within the dosing range where we would expect to see clinically meaningful improvement in these patients.”
Asterias is now looking to enroll 5-8 patients for this 20 million cell phase.
First, do no harm
For people like Jake Javier, this news is personal, not just data. Earlier this summer, Jake broke his neck at a pool party the day before high‑school graduation, leaving him paralyzed from the chest down with limited arm and hand movement. On July 7, he enrolled in the Asterias trial and received ten million stem cells in his neck.
It may take months, even a year, to know whether the cells help. For now, we know they have not caused harm. “First, do no harm” is medicine’s core rule, and this trial appears to meet that standard. We hope it will do much more for people like Jake.
“I recently met with Jake and heard what he and his family are going through,” said Randy Mills, CIRM’s President and CEO. “I also saw a young man with remarkable courage and determination. Because of Jake and others who volunteer for clinical trials, progress is possible. They are true heroes.”
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* On a side note, Roman Reed—a powerful advocate for stem cell research—helped make this story possible. He assisted Jake in enrolling in the Asterias trial, and Dr. Hans Keirstead pioneered the research that led to this therapy, funded by the Roman Reed Spinal Cord Injury Research Act.
Hello, I’m wondering if this therapy would be beneficial to someone with a C4 injury? Injury happened 1 year and 9 months ago. Thank you
Dear Olya, thanks for the question. This clinical trial is only focusing on C5-C7 injuries that have happened within 30 days of treatment. The hope is that if this proves successful they may be able to extend it to treat other people with older injuries. However, that is likely to be several years in the future. This clinical trial is still in its early stages so we need to be mindful that there is a long way to go, and a lot of work still to be done, before we know if this works.
Hello,
After receiving radiation treatment from being diagnosed with cancer, I then developed Spinal Stenosis, & at times the pain of walking, standing or bending is totally unbearable.
Is there any hope for me, as from leading a normal life, I am now a cripple, and house-bound and in constant pain.
If you can help me, it would give me back some of my life.
Anne-lucinda Connor
Hi Anne-Lucinda. We are very sorry to hear about what happened to you. The clinical trial we are funding is currently testing a stem cell transplant in patients with recent spinal cord injuries to their neck. They first have to make sure the therapy is safe before they can address whether it helps patients get some of their motor abilities back. In the future if this type of therapy is successful in helping spinal cord injury patients, there is a possibility that a similar treatment could be tested for other spinal cord conditions. If you would like to keep track of the ongoing stem cell clinical trials, you should check out the clinicaltrials.gov webpage.
My brother had accident last month 8-8-2016. Fracture in c6. After that he operated by acdf plate in his neck. Can you help him. His hand working.
My daughter is 21 years old and suffered a C-5 spinal injury in July 15, 2017- she has gone through inpatient rehab form 08/11 to 09/29- she is currently in outpatient rehab- we are very interested in trying different approaches to her recovery- Stem Cells sounds like it is very promising and we are indeed enthusiastic about the possibility of getting more help. If possible we would love to speak with someone about a clinical trial.
Hi Kathy. Thanks for your comment and for reading our blog. I am sending you an email in response to your question.
Any hope for an ischemic paralysis patient (level T10-12 with sacral sparing) with damaged peripheral nerves resulting in flaccid paralysis?
There is always hope John, but right now I don’t know of any clinical trial that would be appropriate for you.