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DMTs better than HSCT

While HSCT is regarded as a treatment option with the potential to at least temporarily suspend disease activity and progression, more research is needed. The treatment also needs to be more accessible to patients.

While research is underway all around the world, multiple disease-modifying treatments, known as DMTs, are used to help manage MS symptoms and also slow the progression of disease.

HSCT vs. DMT Treatment

The National Multiple Sclerosis Society highly advises starting DMTs as soon as possible
following diagnosis after reviewing all pertinent information and therapies.2 Continuous
DMT consumption has been linked to:

  • lessen the frequency of fresh relapses
  • Reduce the number of new relapses
  • Slow progression of disability
  • Decrease new inflammation within the central nervous system

DMTs come in one of three ways, either as oral, injectable, or intravenous medications. Conventional DMTs help alleviate some MS symptoms through a variety of mechanisms, including by inhibiting immune cell activation and decreasing the inflammatory response of the immune system.

What Is HSCT Treatment?

While there is currently no cure for multiple sclerosis (MS), there are numerous diseasemodifying therapies, lifestyle modifications, and experimental treatments aimed at adequately managing the disease. Over the last few decades, one such experimental treatment known as Hematopoietic Stem Cell Transplantation (HSCT) has been at the forefront of progressive MS research.

HSCT attempts to reset the immune system to slow or stop MS from progressing, at least temporarily. But it’s only available in clinical trials and not everyone with MS is eligible for it. The goal of HSCT is to potentially “reboot” or reset the body’s immune system. Since MS is an autoimmune disease, the body’s immune system is under attack from itself. HSCT aims to knock out the miscommunication between the brain and the immune system and decrease or completely halt the demyelination that occurs.

In a 2016 study, nearly 70% of people who received HSCT showed no new MS disease activity after three years.1 The study also found throughout the trial and follow-up that recipients of HSCT had a significant recovery in neurological function. This further illustrates that HSCT may not only stop new disease activity, but potentially help repair pre-existing neurological damage.

The study “Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis – A Randomized Clinical Trial” was published in the journal JAMA. https://jamanetwork.com/journals/jama/fullarticle/2720728

HSCT may be a beneficial treatment option for people who have relapsing-remitting MS (periods of stability between periods of symptoms)

  • Have had MS for less than 10 years
  • Are younger than 60 years old
  • Have had new lesions on MRI or relapse in condition despite appropriate diseasemodifying therapies
  • Are unable to take high-efficacy disease-modifying therapies

HSCT has been found to be most effective in people with highly active relapsing MS. While HSCT is regarded as a treatment option with the potential to at least temporarily suspend disease activity and progression, more research is needed. The treatment also needs to be more accessible to patients. At present the total cost of HSCT at major centres like Mexico and Russia cost more than 100,000 US Dollars including travel and stay. At world class Artemis Hospital in India it cost about 30,000 US Dollars all inclusive. Please click this link to know more about world’s most affordable HSCT treatment https://hsctindia.com/hsct-treatment-package/

hsct prosses

Multiple sclerosis (MS), a perplexing and progressive neurological condition, wreaks havoc on the brain, spinal cord, and optic nerves. Its symptoms can range from manageable to devastating, and to date, no cure exists. But, there is a glimmer of hope in the form of a new treatment option, Hematopoietic Stem Cell Transplantation (HSCT).

Although HSCT is making waves for its potential to stop the progression of MS, it’s crucial to comprehend the pros, cons, and outcomes before making a decision. So, let’s delve into the mysteries of HSCT for MS and find out if it truly works or not.

The Science Behind HSCT

Thirty-five years ago, Dr. Richard Burt began a journey to treat chronic autoimmune diseases as they’d never been treated before. Using a treatment originally developed for leukemia but modified to be more gentle—a one-time combination of immune targetingdrugs followed by a transplant of the patient’s blood stem cells—he has documented the successful and often dramatic reversal of multiple sclerosis, systemic sclerosis (scleroderma), chronic inflammatory demyelinating polyradiculoneuropathy (CIPD), neuromyelitis optica, and Crohn’s disease HSCT is a radical treatment that involves destroying the immune system through high- dose chemotherapy or radiotherapy and then infusing new immune-forming stem cells.

This treatment is only indicated for specific conditions, including the most severe types of MS. The objective of HSCT is to halt the progression of MS by controlling the disease- causing inflammatory response of the immune system. Ideally, the new immune system created by the infused stem cells would have no memory of MS-caused autoimmune response and would therefore remain tolerant of the body, avoiding another immune attack.

Potential Rewards of HSCT

HSCT has the potential to bring numerous benefits to MS patients. It has the ability to stop MS progression, reduce or eliminate symptoms, and prevent the immune system from attacking the central nervous system, optic nerves, or brain. If an HSCT treatment is successful, it could be a lifelong treatment, targeting the root cause of MS – the immune system. Although HSCT wouldn’t cure the immune system, it would suppress it enough to prevent it from attacking the central nervous system, optic nerves, and/or brain.

Patient Experiences with HSCT

It’s difficult to determine the long-term effectiveness of HSCT for MS since the treatment is relatively new and has only shown promise in clinical trials. But, there have been a a good number of HSCT cases in the news, and patient forums like https://www.facebook.com/hsctindia that have provided patients with an opportunity to share their experiences. Some patients with aggressive forms of MS who underwent HSCT reported immediate results after the procedure, including the cessation of progression of the disease, regained mobility, and reduced or eliminated dependence on other treatments. Other patients with moderate forms of MS who underwent HSCT reported that the procedure brought the disease into remission, reducing or eliminating dependence on other treatments and regaining mobility and function.

The Cost of HSCT: A Burst of Reality

One of the crucial aspects to understand about HSCT is its cost and the fact that it’s not covered by insurance. HSCT is an extremely expensive treatment that can cost tens of thousands of dollars out-of-pocket. The exact cost of HSCT will vary based on the individual, insurance coverage, and chosen doctor. HSCT is only performed at specialized centres with 30 days stay in isolated hospital rooms equipped with HEPA Filter with Triple Level Air Filtration to avoid any risk of infection, 24 x 7 nursing care and best medical attention. Popular centres in Mexico and Russia cost more than 100,000 US Dollars for HSCT, while in India the cost is much reasonable at about 30,000 US Dollars with comparable facilities. For more details https://hsctindia.com/hsct-treatment-package/

Making an Informed Decision about HSCT

Before making a decision about HSCT for MS, you must consider the following:

    1. The current state of your MS
    2. Available treatment options and potential outcomes
    3. The science behind HSCT treatment
    4. Potential risks of undergoing HSCT
    5. The cost of the treatment
    6. Availability of a support system

Once you have all the facts, you’ll be equipped to make an informed decision about your
health and future.

The outcomes of HSCT as of now has helped thousands of patients with autoimmune
diseases has no doubt proven it to be a revolutionary approach that could convert their
life sentence into a one-time reversible illness.

In multiple sclerosis (MS), a disease that strips away the sheaths that insulate nerve cells, the body’s immune cells come to see the nervous system as an enemy. Some drugs try to slow the disease by keeping immune cells in check, or by keeping them away from the brain. But for decades, some researchers have been exploring an alternative: wiping out those immune cells and starting over.

The approach, called hematopoietic stem cell transplantation (HSCT), has long been part of certain cancer treatments. A round of chemotherapy knocks out the immune system and an infusion of stem cells—either from a patient’s own blood or, in some cases, that of a donor—rebuilds it. The procedure is already in use for MS and other autoimmune diseases at several clinical centers around the world, but it has serious risks and is far from routine. Now, new results from a randomized clinical trial suggest it can be more effective than some currently approved MS drugs.

“A side-by-side comparison of this magnitude had never been done,” says Paolo Muraro, a neurologist at Imperial College London who has also studied HSCT for MS. “It illustrates really the power of this treatment—the level of efficacy—in a way that’s very eloquent.”

Nearly 30 years ago, when hematologist Richard Burt saw how HSCT worked in patients with leukemia and lymphoma, he was struck by a curious effect: After those patients rebuilt their immune systems, their childhood vaccines no longer protected them, recalls Burt, now at Northwestern University’s Feinberg School of Medicine in Evanston, Illinois. Without a new vaccination, the new immune cells wouldn’t recognize viruses such as measles and mumps and launch a prompt counterattack. That suggested that in the case of an autoimmune disease, reseeding the immune system might help the body “forget” that its own cells were the enemy.

Burt and others have since used HSCT for a variety of autoimmune diseases, including rheumatoid arthritis and lupus. In the past few years, several teams have reported encouraging results in MS. But only one study—which evaluated just 17 patients—directly compared HSCT to other available drug treatments.

In the new trial, Burt and his colleagues recruited 110 people with the most common form of MS, known as relapsing-remitting. In that form of the disease, patients can go long periods without symptoms—which include muscle weakness and vision problems—before inflammation flares up. Trial participants had at least two such relapses in the previous year, despite being on one of several approved MS drugs.

Half the participants continued with drug treatment but switched from a drug that wasn’t working for them to a drug of a different class. The other half underwent HSCT. First, the researchers collected their blood to reinfuse later. Then, they gave patients a combination of drugs to kill most of their immune cells. In this trial, the patients would have regenerated their own immune systems with stem cells in bone marrow that were spared annihilation, Burt notes. But they received the reinfusion of their own stem cell-rich blood to help speed recovery by several days.

A year later, the researchers evaluated how far the disease had progressed in each of the patients. According to a zero-to-10 scale of disability that includes measures of strength, coordination, and speech, roughly 25% of those in the drug treatment group showed at least a one-point worsening in their score, compared with just 2% of those in the transplant group, the researchers report online today in The Journal of the American Medical Association (JAMA).

MRI scans also revealed less extensive brain lesions in the transplant group and improvements in a patient survey about quality of life. (Survey scores worsened slightly in the drug-treated group.) Five years after treatment, about 15% of people in the transplant group had had a relapse, versus about 85% of the control group.

The two groups diverged “in a massive way,” Muraro says. But he adds that two of the most effective MS drugs weren’t included in the control group. One, ocrelizumab, had not been approved when the study was enrolling participants. Another, alemtuzumab, was excluded because it also depletes immune cells…

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stem-cell12

Progress for Stem Cell Transplant in MS

A small, preliminary study in JAMA has found that hematopoetic stem cell transplant (HSCT) may be more effective than disease modifying drugs in patients with highly active relapsing-remitting multiple sclerosis (MS).1 The study is the first randomized trial of HSCT in relapsing-remitting MS. Findings were published online on January 15, 2019 in JAMA.

Researchers also found that patients treated with HSCT had less progression, and more of them had no signs of disease activity than those treated with disease modifying drugs. “This degree of improvement has not been demonstrated in pharmaceutical trials even with more intensive DMT [disease modifying treatments] such as alemtuzumab,” wrote first author Richard Burt, MD, of Northwestern University Feinberg School of Medicine (Chicago, IL), and colleagues.

Relapsing-remitting MS affects about 85% of patients with MS.2 Treatment with disease modifying agents, such as interferons, glatirameracetate, fingolimod, natalizumab, or dimethyl fumarate, carries a high price tag—up to $60,000 per year per patient.3 But these drugs may only slow disease progression rather than halting it. Most patients eventually develop irreversible damage and progressive disability, for which no effective therapies exist.

HSCT may offer the chance get rid of the immune cells that cause MS, basically giving the immune system a fresh start to improve remission rates.

To test the technique, researchers conducted an open-label study, called the Multiple Sclerosis International Stem Cell Transplant (MIST) trial. The study included 110 individuals with relapsing-remitting MS, moderate disability, and highly active MS (at least two relapses in the past year despite disease modifying therapy). Participants were recruited from four centers in the US, Europe, and South America between 2005 and 2016. The trial was composed of 66% women, with a mean age of 36 years.

Researchers randomized 55 participants to nonmyeloablative autologous HSCT, which is also called “mini-transplant” and uses a patient’s own stem cells with less aggressive pre-treatment to decrease toxicity. The HSCT group received pre-transplant conditioning with cyclophosphamide (200 mg/kg) and antithymocyte globulin (6 mg/kg). The study also assigned 55 participants to disease modifying therapy (DMT) with higher efficacy or from a different class than drugs taken in the last year. Median followup was 2 years.

Results showed that three patients in the HSCT experienced disease progression, while 34 in the DMT group did so. The HSCT group also had significantly longer median time to disease progression compared to DMT (HR, 0.07; 95%CI, 0.02-0.24; P < .001). Median time to clinically significant progression was 24 months in the DMT group and could not be calculated in the HSCT group because of too few events.

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stop-ms

Stem cell therapy for relapsing MS proves effective and safe, study finds

 

A new Intense Stem Cell Therapy known as HSCT has shown promising results during a clinical trial. However, experts give words of caution, saying that it might not work for everyone.

Stem cells might prove to be an important breakthrough for the treatment of multiple sclerosis, but the road to there might be a long and painful one.

A new clinical trial tried to explain this. During the trial, participants with multiple sclerosis (MS)
that were using nonmyeloablative hematopoietic stem cell transplantation (HSCT) showed better results than the participants on disease-modifying treatments (DMTs).

This randomized clinical trial is the first one of its kind, comparing HSCT to DMTs.

Researchers involved in this trial stated that a significant portion of participants on HSCT treatment was successful in slowing down the progression of the disease, in comparison with the participants that were using DMTs.

Out of 103 participants, only three (on HSCT) showed disease progression when compared to the 34 that were on DMTs. Because of this, the research team made a conclusion that HSCT is better at slowing down progression than DMTs, but pointed out that it is important to come up with the right type of patients that will be subjected to what has been shown to be a rigorous and somewhat dangerous therapy.

This study was led by Dr. Richard Burt, chief of immunotherapy and autoimmune diseases at the Northwestern Feinberg School of Medicine in Illinois.

Dr. Barbara Geisser, professor of clinical neurology at the David Geffen School of Medicine at the University of California Los Angeles (UCLA) and clinical director of the UCLA MS program had this to say on the matter: “This important study is the first randomized trial of HSCT vs. DMT, and demonstrated that HSCT was superior to conventional DMT in stabilizing persons with relapsing MS with respect to relapses and progression and improving some functions.”

A rigorous treatment

HSCT works by using chemotherapy in order to aid cells to “forget” they have MS. By doing this, the immune system is brought down to some quite vulnerable levels. The patient has to be hospitalized and treated with the greatest care.

Bruce Bebo, PhD, an executive vice president of research at the National Multiple Sclerosis Society said: “HSCT is not one therapy but a range of therapies from a more mild type of chemo to a stronger type. Dr. Burt practices a more mild form of chemotherapy — more mild form of immunosuppression — so the risks are lower.”

The participants of the trial had relapsing MS, characterized with mild to moderate disabilities, with a range of 2 to 6 on the Expanded Disability Status Scale (EDSS).

Bebo said: “This study stands above the others — first controlled randomized autologous stem cell transplant. Burt and his team should be applauded for taking this on. It’s what everyone was asking for,” adding that “A lot of groups are experimenting with this type of treatment — case studies and evidence is accumulating for its positive benefit and accumulating info for the best patient and approach for success.”

He further continued to explain the difficulty of this study in performance, and elaborated on how Burt and his team were able to create an unbiased way to make a comparison between HSCT and DMTs, by way of excluding ocrelizumab and alemtuzumab.

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stem-cell (2)

Greater Benefits for Relapsing-Remitting MS Found with HSCT Therapy

Lesion load, disability improved for relapsing patients with highly active MS.

Stem cell transplantation was more effective than disease-modifying therapy (DMT) for patients with highly active relapsing-remitting multiple sclerosis (MS), a randomized clinical trial showed.

Over a median follow-up of 2 years, only three of 55 patients treated with non-myeloablative autologous hematopoietic stem cell transplantation (HSCT) had disease progression, compared with 34 of 55 patients on medication, according to Richard Burt, MD, of the Northwestern University Feinberg School of Medicine in Chicago, and colleagues.

Mean disability level improved in the HCST group during the first year, but worsened in the disease-modifying therapy (DMT) group, they reported in JAMA.

“This is the first randomized trial in relapsing-remitting MS patients of hematopoietic stem cell transplantation versus best-available DMT,” Burt told MedPage Today. “You need to look at the group we treated — they had aggressively relapsing MS with moderate disability — and the results were much better than standard drug therapies.”

Two newer drugs were not included in the study, Burt noted: ocrelizumab (Ocrevus), which had not been approved during the study enrollment period, and alemtuzumab (Lemtrada), due to the autoimmune effects it has been associated with. “Because we had a built-in cross-over in the study, we didn’t know what effect alemtuzumab might have if these patients crossed over to transplant,” he said.

The trial included 110 patients with relapsing-remitting MS who had at least two relapses while receiving drug treatment in the prior year. These patients had an Expanded Disability Status Scale (EDSS) score of 2.0 to 6.0 (EDSS scores range from 0-10, with 10=worst neurologic disability) and were randomized at 4 U.S., European, and South American centers from 2005 to 2016. Mean age was 36 years and 66% were female.

Patients in the stem cell transplantation group had a non-myeloablative regimen — a lower-dose, short course of more tolerable immune-specific chemotherapy and antibodies to suppress the immune system — and discontinued DMTs prior to the trial, with variable washout periods. Patients in the DMT group were treated with a mean of 1.3 different DMTs: 21 patients received natalizumab (Tysabri), 14 received dimethyl fumarate (Tecfidera), 14 received fingolimod (Gilenya), nine received glatiramer acetate (Copaxone), seven received interferon beta-1a (Rebif), six received mitoxantrone, and one received teriflunomide (Aubagio). Patients whose EDSS worsened with continued DMT treatment could cross over to the HSCT group.

The randomized clinical trial is the first to compare HSCT to DMTs.

During the first year, mean EDSS scores improved from 3.38 to 2.36 in the HSCT group and worsened from 3.31 to 3.98 in the DMT group (between-group mean difference -1.7, 95% CI -2.03 to -1.29, P<0.001).

Clinically significant disease progression also occurred much less frequently in the HSCT-treated group than the DMT-treated group (HR 0.07, 95% CI 0.02-0.24). Rates of disease progression at 1 year were 1.92% in the HSCT group and 24.5% in the DMT group. At 5 years, they were 9.71% in the HSCT group and 75.3% in the DMT group.

The HSCT group also had fewer relapses, improved MRI lesion load, a higher proportion of patients maintaining no evidence of disease activity, and a better quality of life. There were no deaths, and no patients who received HSCT developed non-hematopoietic grade 4 toxicities, such as myocardial infarction, sepsis, or other disabling or potential life-threatening events.

“Most comparative trials between DMTs measure benefit as a slowing of MS manifestations; this trial showed a global improvement over time in the mean level of disability and MRI lesion load in the HSCT-treated group while deterioration occurred in the DMT-treated group,” noted Harold Atkins, MD, of the University of Ottawa, in an accompanying editorial.

“Despite the positive results, several aspects of this trial deserve comment,” Atkins observed. While chemotherapy doses used in this study resulted in less severe toxicity than doses used for other malignant diseases, many patients experienced moderate to severe acute toxicity soon after HSCT, he stated. No treatment-related mortality was reported here or in other published cohorts of HSCT for MS performed in this manner, but “caution in patient selection remains warranted, as deaths have been reported using the same HSCT procedure in other autoimmune diseases,” he wrote.

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