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Stem Cell Transplant Stops MS in its Tracks – In-Depth Doctor’s Interview

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UCI Health neurologist, Dr. Michael Sy, MD, talks about a new procedure to stop MS symptoms.

Interview conducted by Ivanhoe Broadcast News in July 2022.

Can you give some background on MS?

SY: Yeah. So, MS affects almost a million Americans. It is one of the leading causes of disability amongst young people in the United States.

What are the main symptoms that these people are living with?

SY: MS patients live with a variety of symptoms. Sometimes it’s problems with vision, sometimes it’s loss of strength, loss of sensation. They can also have cognitive impairments. A lot of MS patients have extreme fatigue that they deal with. A lot of patients also have mood disorders or bowel and bladder dysfunction.

And do these symptoms get any better?

SY: Sometimes, symptoms can come and go, but part of MS is a progressive disability accumulation whereby some of these symptoms continue to get worse over time. And our current treatments are not able to stop that from happening.

What are current treatments? Is it all medication based?

SY: Yeah. Medications, right now, there’s more than 20 FDA approved treatments for MS. It is a success of modern medical science. And they are highly effective in reducing the inflammatory attacks that occur with MS. So most common form of MS is what we call relapsing remitting. It’s characterized by inflammatory attacks called relapses. And these medications, some of them are very effective in reducing the number of relapses that do occur. Some of them to the point where patients don’t have any more relapses. However, some patients continue to have relapses even on these highly effective medications. And in addition, some patients will have a progressive decline in function independent of relapses and our medications are not so great at preventing that progressive decline.

As a physician, it’s probably frustrating for you not to be able to offer more, right?

SY: Yeah, for sure. Obviously, there are limitations to the medications that we have currently. And it is frustrating when we see patients declining and can’t do much more for them.

But now, there’s a new hope, right?

SY: Yeah. Bone marrow transplant, autologous bone marrow transplant in this case, offers the next step in terms of treatment. So most of our current treatments are immunosuppressive medications already. They suppress the immune system either by modifying how the immune system is acting or by destroying part of the immune system. And bone marrow transplant offers the opportunity to just completely reset the immune system and offering the biggest gun, the biggest tool that we have in treating MS.

How does it work?

SY: Autologous bone marrow transplant involves first mobilizing patient’s own stem cells, collecting those stem cells, and then wiping out the immune system using chemotherapeutic agents. And then allowing the body to recover by giving back their initial stem cells from before.

How successful has this been?

SY: Bone marrow transplant has been studied across the U.S., particularly mostly in Northwestern, in Canada and in the U.K. And the studies are pretty much in line and showing that about 80 percent of the time patients no longer have relapses after a bone marrow transplant even though they’re not on medication anymore. So this is being compared to patients who are on medications. So that means they’re chronically immunosuppressed. So after bone marrow transplant, patients no longer have to be chronically immunosuppressed to prevent relapses most of the time. In terms of reducing this all important disability progression to indolent decline in function, it’s not quite as successful, but about 65 percent of the time patients don’t experience any continued further progression of disability. This compares to even our most efficacious medications it’s only about 50 percent of the time patients don’t experience any disability decline. Bone marrow transplants still has to be further study. So there are a couple of randomized controlled trials to prove out how much more effective it really is both in preventing future relapses as well as preventing this disability progression.

Is this the first time that you’ve seen anything work like this?

SY: Yeah. It’s unique in the sense that it is a final solution. Most of our treatments are patients who are on them chronically. For example, our most effective medications the patient may start in their 20s because MS is often diagnosed at a very young age and they will remain on these medications until they’re 50 or 60 years old. So, 30 plus years of exposure to medications. And honestly we don’t have great data on especially our highest efficacy medications and what that- what happens after 30 years of use. So, bone marrow transplant is extremely unique in the sense that it collapses all of that experience into a very short and extremely intense three-month period after which they’re done. And at that point they can go about their life and not worry about being on medication and being immunosuppressed long-term.

Everything in medicine, a lot of it comes down to money, and you would think that bone marrow transplants aren’t cheap, but it’d be a lot less expensive in a lifetime, right?

SY: Correct. And our medications for MS are very expensive medications. They can cost anywhere from $50,000 to $100,000 a year. You just look at that over again another 30 years. Bone marrow transplant itself ranges from $150,000 to $300,000. So. the cost of the bone marrow transplant is covered in about three or four years.

Can you talk about one of your patients, Kathy?

SY: Yeah. She’s responded very well to the transplant. Once you tolerated the whole procedure very well, again, it’s a very intense procedure. You do have to be hospitalized for a significant period of time. But she actually did very well throughout the whole process and has bounced back very quickly as well. So that’s really great that she has done that. And I think our younger patients like her tend to do that. And the earlier you attempt bone marrow transplant, the better you’re going to deal with the whole process essentially. On top of that she’s not only recovered very quickly from the transplant itself, but she feels much better after transplant compared to before.

Do you wipe away all the symptoms then for some of these patients?

SY: No. So, what bone marrow transplant doesn’t do is, it doesn’t repair the damage that has already been done. So again, this is a therapy that’s focused on the immune system and stopping the immune system from causing further damage to the brain. And again, it’s a definitive solution for doing that. Unfortunately, the damage has already occurred and it has already accrued is still there. The hope is that by stopping the inflammatory attack, it allows the body a little bit of reprieve and allows the body a chance to heal itself. And sometimes that does happen. In fact, the data suggests that at least in the first year after bone marrow transplant most patients will actually improve and will do better for a period of time.

Is Kathy part of a clinical trial?

SY: She is not part of a clinical trial.

Is that a next step here in the United States?

SY: Yeah. There are ongoing clinical trials with stem cell transplant. There are one major one here in the U.S. and another one being conducted I believe in Europe. And so we’ll see the results of those trials in the coming years. But at this time it is something that is offered and it’s still something that can be done. And it’s a matter of financing the cost for it. And thankfully, I think a lot of insurance companies are realizing that it’s cheaper to do this than to keep a patient on inexpensive drug for decades essentially. And so they’ve become more willing to proceed with bone marrow transplant.

END OF INTERVIEW 

This information is intended for additional research purposes only. It is not to be used as a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or accuracy of physician statements. Procedures or medicines apply to different people and medical factors; always consult your physician on medical matters.

If you would like more information, please contact:

John Murray

jdmurray@hs.uci.edu

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