Athersys, Inc. is a US-based biotech company developing stem cell treatments for neurological, inflammatory and immune and cardiovascular diseases.
Its patented MultiStem candidate can be delivered to patients up to 36 hours after ischemic stroke – significantly opening up the window to treatment.
Dr Robert William Mays is Head of Neurosciences and Vice President of Regenerative Medicine at Athersys. SR Times learns how the treatment works.
When did you start looking at stem cell therapy?
There were a group of us out at Stanford in the mid-90s that had some ideas about starting a biotech company. In the early 2000s, we acquired the rights to this adult cellular therapeutic and we’ve spent the last almost 20 years realising what therapeutic indications we can apply the cells to to see meaningful benefit.
But also equally important is understanding clinical indications where the cells may not work. Once you have a hammer, everything looks like a nail. We started going after acute neurologic indications because of the data we were getting in animal models.
What makes stroke such an unmet clinical need and why is MultiStem a promising treatment?
Stroke is the number one cause of disability throughout the world and there is a very small bucket of therapeutic interventions. Some of the clot dissolving drugs have to be given in a fairly short timeframe after the onset of stroke, before you increase the risk.
The mechanical thrombectomy devices have proven to be effective. But they only get utilised in a small percentage of patients who have large vessel proximal type of occlusions because of the risk you get when you start chasing clots further up in the brain.
In our animal models, we saw that we could administer cells and see therapeutic benefit later than the sub-acute timeframes that the TPA or other thrombectomy devices are utilised.
They are now investigating increasingly longer times with both clot busters and mechanical reperfusion devices, but we saw that we could do even better and go out to later time points.
And we could piggyback. We could still use our cells after those other therapies have been tried and maybe not been effective. They weren’t mutually exclusive.
How does MultiStem work?
The cells that MultiStem encompass are sort of like the firemen for the body. They’re there to resolve inflammation after the onset of acute, severe injury.
We have some of these cells in our bodies. We isolate them from bone marrow, but it’s believed that they are in other organs and tissues as well. But the older we get, the fewer of them we have.
Strokes are occurring mostly in an ageing population. So if you come in with a large dose of the cells in the appropriate timeframe after the onset of a stroke, you actually turn down the pro-inflammatory processes, which seems to lead to more severe inflammation.
It appears that the migration of the cells into the brain can do more harm than good. So if you can turn off the bad guys, going up to the site of the injury faster, and more quickly up-regulate the reparative phenotypes to other immune cells and other reparative responses, you can resolve things more quickly and you’ll have less potential scarring.
So the rehabilitation and repair can occur more naturally and more quickly in the stroke patients.
What challenges have you faced in developing stem cell treatments and actually bringing them to the market? It’s obviously been quite a long road.
We started doing animal studies in 2005, so we’re 16 years in. It’s hard with cellular therapeutics. It’s easy to make a lot of the compound and be able to understand all of the chemical properties of a traditional small molecule drug. With cells, these are living organisms, living medicine, that is responding to the body once it’s placed in.
It’s one thing to put them into an animal after stroke and be able to sacrifice the animal, examine all the tissues or the changes the cells impart to that animal. Humans, not so much.
It’s the translational aspect of it. The whole field of cell therapy and the translational acceptance by regulators is a delicate tightrope at this particular time.
How far along the ischemic stroke pathway are you?
We’re in a pivotal Phase III study right now in the United States. But it’s going to be a global study. We have sites in Asia Pacific and in the UK. Our Phase II was the United States and at six sites in the UK.
And also, for this pivotal Phase III will have other sites in Western Europe as well. We’ve been cranking at this for a while and we’re looking to complete this pivotal study by the end of 2022.
Our partner in Japan, Helios, announced that they’ve completed enrolment in an almost identical study. It’s a pivotal registrational study using the same exact cells in acute ischemic stroke in the Japanese Asian population. We expect top line data from our partners in the not too distant future.
What are your goals for 2022?
Getting the MASTERS-2 stroke trial completed here in the United States. We have ongoing preclinical work a number of indications, we’ve got the most of them continued down the line, including great hemorrhagic stroke data and we hope to be submitting that paper in 2022.
What are some of the other non-stroke indications that you’re looking at?
We are actively enrolling in acute respiratory distress syndrome (ARDS), which affects a lot of COVID-19 patients. We completed a Phase I safety study and read out the data on that maybe three years ago. And we are conducting a Phase II ARDS study here in the United States, looking at both pneumonia and COVID-19.
Healios has completed a smaller Phase I/Phase II study in looking at ARDS patients in Japan. And they have top line data on their first time point and the results look very encouraging.
