CIRM-funded stem cell clinical trial for spinal cord injury expands patient recruitment

asterias

It’s always great to start the week off with some good news. Today we learned that the Food and Drug Administration (FDA) has given Asterias Biotherapeutics approval to expand the number and type of people with spinal cord injuries that it treats in their CIRM-funded clinical trial.

Up till now, Asterias has been treating people who have injuries at the C5-C7 level, those are the lowest levels of the cervical spine, near the base of the neck. Now they will be able to treat people with injuries at the C4 level, that’s not only higher up the neck but it’s also the second most common form of spinal cord injury.

In a news release Dr. Ed Wirth, Asterias’ Chief Medical Officer, says this is a vote of confidence from the FDA in the company’s AST-OPC1 stem cell therapy:

“FDA’s decision to allow the company to enroll qualified patients with C-4 level injuries is the result of the data supporting the safety of both AST-OPC1 and the procedure to inject the cells and means that the second most common cervical spinal cord injury population can now be eligible to receive AST-OPC1. The overall changes to the study protocol will enhance our ability to enroll qualified patient candidates for our current SCiStar study and we also expect the changes to help enrollment rates in a future, larger clinical study.”

C4 image

Photo courtesy Shepherd Center, Atlanta

People who are injured at the C4 level are typically paralyzed from the neck down and need constant help, while people with C5-C7 injuries typically have some use of their hands and arms. Caring for someone with a C4 injury is expensive, with lifetime costs estimated around $5 million. Anything that could help people recover some movement would not only reduce those costs but would, more importantly, also increase the quality of life for people.

Asterias is not only expanding the patient population they are working with, they are also expanding the window for treating the injury. Currently patients have to be enrolled from 14 to 30 days post injury. In this new C4 group that window has been extended to 21 to 42 days post injury.

The reason for that change is that because C4 is higher up in the neck, newly injured people often need to be placed on a ventilator to help stabilize them. These patients take a little more time to recover from the initial trauma before they are ready to be treated.

We have blogged several times (here, here and here) about the encouraging news from the Asterias trial and how it appears to be helping people with injuries at the C5-C7 level recover some movement in their arms and hands. In some cases, such as with Kris Boesen for example, the improvement has been quite dramatic. Now the hope is that this new patient population will see similar benefits.

kris-boesen

Kris Boesen, CIRM spinal cord injury clinical trial patient.

The study is being conducted at six centers in the U.S., including some here in California,  and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment.

Clearing the first hurdle: spinal cord injury trial passes safety review

Jake 2

Jake Javier, participant in Asterias clinica trial

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 wracking 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 got some good news about one clinical trial we are funding, the Asterias Biotherapeutics spinal cord injury trial. Asterias announced that its Data Monitoring Committee (DMC) has reviewed the safety data from the first two groups of patients treated and found no problems or bad 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 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 are treated between two weeks and one month after the injury was sustained.

The therapy involves transplants of Asterias’ AST-OPC1 cells which were made from human embryonic stem cells. The AST-OPC1 cells have been turned into oligodendrocyte progenitors, which are capable of becoming the kind of cells which 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, that there were 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.

jake and family

For people like Jake Javier this news is not about numbers or data, it’s personal. Earlier this summer Jake broke his neck at a pool party, celebrating graduating from high school. It left him paralyzed from the chest down with extremely limited use of his arms and hands. On July 7th Jake was enrolled in the Asterias trial, and had ten million cells transplanted into his neck.

It could be months, even as much as one year, before we know if those cells are having any beneficial effect on Jake. But at least for now we know they don’t seem to be having any negative effects.

“First do no harm” is the cardinal rule that all budding physicians are taught. This trial seems to be meeting that benchmark. Our hope now is that it will do a lot more, and truly make a difference in the lives of people like Jake.

As Randy Mills, CIRM’s President and CEO, said in a news release:

“I recently met with Jake and heard first-hand what he and his family are going through in the aftermath of his injury. But I also saw a young man with remarkable courage and determination. It is because of Jake, and the others who volunteer to take part in clinical trials, that progress is possible. They are true heroes.”


* On a side note, Roman Reed, a great champion of stem cell research and a patient advocate extraordinaire, helped make much of this story happen. He helped Jake enroll in the Asterias trial ,and the research that led to this therapy was pioneered by Dr. Hans Keirstead who was funded by the Roman Reed Spinal Cord Injury Research Act.

 

Related Links: