Regenerative medicine’s cutting edge

An Oct. 20 gala for the Cedars-Sinai Board of Governors Regenerative Medicine Institute promises to be full of big names: Hosted by Jay Leno, it will honor philanthropic leaders Adele and Beny Alagem and actor Sylvester Stallone. 

But not to be overlooked regarding the event — which is projected to generate approximately $2 million — is the institute’s director, Clive Svendsen. The Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine is a leader in research on ALS (Lou Gehrig’s disease), which affects nerve cells in the brain and spinal cord and eventually leads to paralysis and death. 

The Journal spoke with Svendsen about how regenerative medicine is revolutionizing his field and others, and holding out promise of made-to-order cells that could rejuvenate defective or ailing body parts. An edited transcript of that interview follows.

JEWISH JOURNAL: What is regenerative medicine? 

CLIVE SVENDSEN: Regenerative medicine is the idea of … making things grow again within your body. We can regenerate tissues that have been dying over time or are getting aged and worn out either by putting new cells in … or by encouraging your own cells to regenerate. 

JJ: What is the focus of the Cedars-Sinai Board of Governors Regenerative Medicine Institute?

CS: The focus … is on induced pluripotent stem cells, what we call IPS cells. … In simple terms, you can take any tissue in the human body … put it in a petri dish, and using one … protein, you can take that cell back in time to an embryonic state. Once you have the cell back in time to this pluripotent [state], you can make any cell of the human body and … grow those cells in incubators.

JJ: Sounds very futuristic.

CS: It is. … I have my own cell line in the incubator. And from that line, we can make neurons, we can make beating heart cells. So I can actually regenerate any of my tissues in the lab through this technology. And they are actually my tissues. If I transplanted them back into myself — which I don’t have approval to do — those cells theoretically should not reject, which is one of the biggest problems with organ transplant. 

JJ: So is that the goal, to be able to give ourselves back our own cells?

CS: That’s one of the goals: to learn how to create these cells, go through the process of creating, let’s say, new heart cells, and inject those cells back into the heart. We’re not clever enough yet to make a whole organ from these cells … but we can take those cells and inject them … to rejuvenate specific areas. 

JJ: How are you applying this concept to ALS?

CS: In ALS, you get paralyzed over time. There’s a certain kind of cell, called a motor neuron, which basically goes from the spinal cord to your muscles and [causes muscle contraction]. That’s how movement occurs, through the motor neurons firing signals to the muscles. 

What we’ve found is that the cells around the motor neurons — the support cells — are sick. The motor neuron is dying because it has no support cells. Our therapy is taking these IPS cells, making them into the support cells of the brain and spinal cord … and then injecting [them] back into the spinal cord of the patient. We did [another thing] which is really exciting: We engineered the cells … to make a therapeutic protein — a drug. So when you put the cells back into the spinal cord, they release a drug that normally can’t get across the blood-brain barrier. 

That’s [how we hope to use] regenerative medicine to treat ALS. We’ve filed for approval of the clinical trial with the FDA and we’re waiting for approval to move forward into patients. … It’s really a safety trial to see if we can do this in 18 patients.

JJ: What would it mean if this approach works?

CS: That’s a huge if. … Delivering a drug this way to the spinal cord has not been done before. … We don’t know what causes ALS. … [so] it’s very hard to design a treatment …

We’re doing an amazing study [to try to understand the causes]. It’s a $20 million study, in collaboration with Johns Hopkins and Massachusetts General Hospital. We’re making a thousand of these IPS lines from ALS patients across the country. They have their blood drawn, the blood comes to Cedars-Sinai, and we make these IPS cells. …

We’re trying to model ALS in the dish using IPS cells. … And then I think we’ll start to understand why motor neurons in ALS patients are different.

There’s some amazing technology that’s coming [called Organ-on-Chip technology, which uses living human cells in micro-engineered environments as a way to simulate the workings of the body]. The company [that developed this technology] is called Emulate. They’re trying to emulate human biology. … What I want to do is, if a patient has lung disease, I want to have their lung tissue on a chip, so that we can assess them in the lab as well as in the clinic.

JJ: Looking forward for the next five to 10 years, what might you predict?

CS: I think instead of having a family history, patients will have their genome sequenced. Here at Cedars-Sinai, we would love to have a patient’s IPS line made. … Once we’ve made your line, we could have it on standby at the hospital. If you have a problem — for example, you get diabetes — we have a way of making your pancreas cells. If you get heart problems, we may be able to produce a cell that we can inject into your heart. If you have an arrhythmia … we could create beating heart cells in the dish that have an arrhythmia … and test 100 drugs on the cells in the dish. When one of them works, it’ll work for you because [it will have been tested using] your beating heart cells.