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.

Advertisements

First spinal cord injury trial patient gets maximum stem cell dose

kris-boesen

Kris Boesen, CIRM spinal cord injury clinical trial patient.

There comes a pivotal point in every experiment where you say “ok, now we are going to see if this really works.” We may be at that point in the clinical trial we are funding to see if stem cells can help people with spinal cord injuries.

Today Asterias Biotherapeutics announced they have given the first patient in the clinical trial the highest dose of 20 million cells. The therapy was administered at Santa Clara Valley Medical Center (SCVMC) in San Jose, California where Jake Javier – a young man who was treated at an earlier stage of the trial – was treated. You can read Jake’s story here.

The goal of the trial is to test the safety of transplanting three escalating doses of AST-OPC1 cells. These are a form of cell called oligodendrocyte progenitors, which are capable of becoming several different kinds of nerve cells, some of which play a supporting role and help protect nerve cells in the central nervous system – the area damaged in spinal cord injury.

In a news release, Dr. Edward Wirth, Asterias’ Chief Medical Officer, says this could be a crucial phase in the trial:

“We have been very encouraged by the early clinical efficacy and safety data for AST-OPC1, and we now look forward to evaluating the 20 million cell dose in complete cervical spinal cord injury patients. Based on extensive pre-clinical research, this is in the dosing range where we would expect to see optimal clinical improvement in these patients.”

To be eligible, individuals have to have experienced a severe neck injury in the last 30 days, one that has left them with no sensation or movement below the level of their injury, and that means they have typically lost all lower limb function and most hand and arm function.

In the first phase individuals were given 2 million cells. This was primarily to make sure that this approach was safe and wouldn’t cause any problems for the patients. The second phase boosted that dose to ten million cells. That was thought to be about half the therapeutic dose but it seemed to help all those enrolled. By 90 days after the transplant all five patients treated with ten million cells had shown some level of recovery of at least one motor level, meaning they had regained some use of their arms and/or hands on at least one side of their body. Two of the patients experienced an improvement of two motor levels. Perhaps the most impressive was Kris Boesen, who regained movement and strength in both his arms and hands. He says he is even experiencing some movement in his legs.

All this is, of course, tremendously encouraging, but we also have to sound a note of caution. Sometimes individuals experience spontaneous recovery after an accident like this. The fact that all five patients in the 10 million cell group did well suggests that this may be more than just a coincidence. That’s why this next group, the 20 million cell cohort, is so important.

As Steve McKenna, Chief of the Trauma Center at SCVMC, says; if we are truly going to see an improvement in people’s condition because of the stem cell transplant, this is when we would expect to see it:

“The early efficacy results presented in September from the 10 million cell AIS-A cohort were quite encouraging, and we’re looking forward to seeing if those meaningful functional improvements are maintained through six months and beyond. We are also looking forward to seeing the results in patients from the higher 20 million cell AST-OPC1 dose, as well as results in the first AIS-B patients.”

For more information about the Asterias clinical trial, including locations and eligibility requirements, go here: www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

We can never talk about this clinical trial without paying tribute to a tremendous patient advocate and a great champion of stem cell research, Roman Reed. He’s the driving force behind the Roman Reed Spinal Cord Injury Research Act  which helped fund the pioneering research of Dr. Hans Keirstead that laid the groundwork for this clinical trial.