About HSCT
What You Need to Know
What is HSCT for MS?
HSCT stands for hematopoietic stem cell transplantation. It uses chemotherapy and stem cell transplantation to erase and reboot the immune system. It was actually a treatment originally created back in the 1960s to help treat cancer. In the case of MS, it actually uses the patient's own stem cells for the transplantation.
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It was first used to treat MS in 1995 in Greece. The HSCT protocols in place today were pioneered by Dr. Burt at Northwestern University in Chicago beginning in 1996. The initial efforts used myeloablative chemotherapy. However, Dr. Burt has switched to a non-myeloablative approach in recent years. Research today looks at both approaches. (https://www.msaustralia.org.au/news/real-world-follow-up-study-of-ahsct-for-ms/)
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While HSCT is not a guarantee MS will never come back, it is the most effective long-term treatment for MS. Offering at least 5-10 years of no disease progression, with many patients going much longer without their MS symptoms getting worse.
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It is important to understand that while this is as close to a cure MS patients can find right now, it is not a cure. It does not reverse the damage of MS. The hope is that with current medical advancements that it buys time for a real cure to be found.
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Myeloablative vs Non-Myeloablative
What are myeloablative vs non-myeloablative chemotherapy approaches for HSCT? The words myeloablative and non-myeloablative refer to the amount of chemotherapy and the effects the chemo has on a patient's bone marrow.
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A myeloablative chemotherapy approach uses high doses of chemo to completely wipe out a patient's immune system and destroy the bone marrow. While it comes with higher risks, some researchers feel it may result in longer term effects on stopping MS. The recovery time from a myeloblative chemotherapy approach can take 9-12 months or longer.
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A non-myeloblative chemotherapy approach to HSCT uses lower doses of chemo to erase the immune system, while leaving the bone marrow in place. The risks from chemo are much less, and the immune system bounces back faster since the bone marrow is able to provide some immune memory. While research hasn't been able to show if the lower amounts of chemo are less effective, it still provides at least 5-10 years of remission.
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Why not do this in the US - The Beat MS Trial (Myeloblative)
There are treatment options in the US. In fact, Selma Blair received non-myeloblative HSCT from Dr. Burt at Northwestern about five years ago.
The Beat MS Trial is also underway. It is the largest HSCT trial ever conducted, with nineteen different universities and medical research hospitals participating in it. It is trying to establish myeloablative HSCT as an effective last line treatment protocol for MS.
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However, there are two problems with pursuing treatment in the US. First, it is extremely cost prohibitive. The treatment alone cost $100,000+ and is not covered by insurance.
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Second, both the Beat MS Trial and trials doing non-myeloblative HSCT treat HSCT as a last line of defense. Qualifying for these treatments requires having had at least two disease modifying therapies fail to halt the progression of the disease.
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MS damage is permanent. It cannot be reversed. Waiting until the damage is done is counterintuitive.
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Why Moscow - Dr. Fedorenko's HSCT approach (Non-Myeloblative)
It is for these reasons that MS patients often seek HSCT treatment outside of the US.
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Dr. Fedorenko and his team at the Pirogov National Research Hospital in Moscow have performed over 2,500 HSCT procedures to treat MS patients. They have a very high success rate. In fact, they have a 95% 10 year success rate for patients like Matt who are using HSCT as a first line of defense.
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The best part is Pirogov is a state of the art research facility, and Dr. Fedorenko has streamlined the procedure so that it can be done in a matter of weeks with the patient in the hospital the entire time. This reduces risks while the immune system is at risk while also reducing the time that a patient is away from family and friends.
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