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

(CNN)-An experimental stem cell therapy proved effective and safe in patients with a relapsing form of multiple sclerosis , an autoimmune disease that affects the central nervous system, new research finds.

A stem cell transplant using a lower-dose regimen of chemotherapy plus immune system suppressors is more effective at preventing disease progression compared to currently used disease-modifying therapies, according to the new study published Tuesday in the journal JAMA. (The stem cell treatment is known as “autologous hematopoietic stem cell transplantation” or HSCT.)

“I never use the word ‘cure’ — never,” said Dr. Richard K. Burt, lead author of the study and chief of Immunotherapy and Autoimmune Diseases at Northwestern University Feinberg School of Medicine in Chicago. However, only a minority of patients receiving HSCT relapse by the five year mark, he said. “The vast majority don’t.”

How does this experimental stem cell therapy work?

HSCT essentially reboots the immune system.

HSCT India“Out with the old, in with the new” is the goal, said Burt, adding that it is a “one-time treatment. You’re done, you’re off drugs.” First a patient’s blood stem cells are collected and then the patient is treated with chemotherapy drugs. Then, blood stem cells are returned to the patient to jump start the development of a new immune system.

In contrast, disease-modifying therapies work differently. These are a dozen or more drugs designed to be chronic, continuous treatment that targets and modulates the immune system. The new comparison study took place at medical centers in the US, UK, Sweden and Brazil, where 110 patients with relapsing-remitting MS participated in the randomized clinical trial, a gold standard medical test. Patients received either the HSCT protocol or a different class of disease-modifying therapy than they’d previously used.

HSCT proved to be the more effective treatment: Of 55 patients receiving HSCT, only three patients showed disease progression at one year, the study showed. Yet, 34 of 55 patients in the disease-modifying therapy group showed disease progression at one year. Disease progression was measured using the Expanded Disability Status Scale, a method for monitoring changes in symptoms over time.

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Among the HSCT group, the proportion of patients with disease progression was (roughly) 2% up to two years, 5% at three years, and 10% at 4 and 5 years. Meanwhile, the proportion of patients with no evidence of disease — defined as no progression, no relapses, and no new or enlarging lesions on MRI scans — was (nearly) 98% at one year, 93% at two years, 90% at three years, and 78% at four and five years.

By contrast, almost one quarter of the patients in the disease-modifying therapy group showed disease progression at one year, more than half at two years, and just under three-quarters at five years. And the proportion of patients with no evidence of disease was (about) 40% at six months, 21% at one year and 3% by years four and five.

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What are the side effects of HSCT?

Side effects of HSCT can include infertility, said Burt, who noted that women can choose to preserve their eggs before treatment. And some patients developed autoimmune thyroid disease, a treatable condition. He noted that the disease-modifying treatments also have side effects.

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Australian AHSCT Trial Results Released.

– The results of an observational study of 35 people with relapsing-remitting and secondary progressive MS who received the treatment in Sydney have just been released.

– Autologous haematopoietic stem cell therapy (AHSCT) uses a combination of chemotherapy and reinfusion of blood stem cells to rebuild and reset the immune system.

– In this study, 60% showed no evidence of disease activity for three years after the treatment and 13 participants showed improvements in their disability scores. 8 people had continued disability progression during the follow-up period.

– Immune cell analysis showed that after one year, attacking immune cells were still wiped out by AHSCT but cells that regulate the immune system were restored.


A Sydney team have published results from their study of Autologous Haematopoietic Stem Cell Therapy (AHSCT) in people with relapsing-remitting and secondary progressive MS.

AHSCT has been under investigation, worldwide and in Australia, as a treatment option for MS using different protocols and in different types of MS. Good results have been seen for many people with relapsing MS however, it is also a treatment with high risks and international clinical trials and studies have shown that it is not effective or suitable for everyone with MS.

AHSCT aims to reset the immune system using chemotherapy to wipe out most or all the immune cells and then the individual’s own blood stem cells are reinfused to rebuild the immune system. The hope is that the treatment puts MS into remission.

Researchers and clinicians at St Vincent’s Hospital in Sydney have been conducting a study of the treatment since 2010. Their results have been published in the Journal of Neurology, Neurosurgery and Psychiatry. The study included 20 people with relapsing-remitting MS and 15 people with secondary progressive MS, who have now been followed for an average of 3 years following the treatment.

This study used the “BEAM” chemotherapy protocol, which completely removes the immune system and is the one most commonly used in people with MS around the world, but with limited published clinical trial results.

Participants in this study had levels of disability that ranged from low to moderately severe (disability scores between 2 and 7 as measured by the expanded disability status score (EDSS)) and had failed to respond to at least two previous therapies for MS, including two thirds who had failed on natalizumab, a high potency MS medication.

The researchers tracked evidence of disease activity via relapses, brain lesions on magnetic resonance imaging (MRI) and disability progression. In this group of people with MS, 60% showed no evidence of disease activity for up to three years after the treatment. In the people with relapsing-remitting MS, the result was higher, with 70% showing no disease activity. 73% of participants had no disability progression during the follow-up period. In fact, 13 participants had improvements in their EDSS disability score of an average of 1.4 points – all but one of these had relapsing-remitting MS. However, eight participants had their disease continue to progress during the follow-up period, two with relapsing-remitting MS and six with secondary progressive MS.

83% of participants had no new or enlarging lesions at their last MRI scan and 96% showed no active lesions.

Participants experienced the expected side effects of high dose chemotherapy, including ulcers in the digestive tract, nausea and hair loss. One third required red blood cell transfusions and a half required platelet transfusions during their hospital stay. No-one died as a result of the treatment in this study which is in keeping with recent international AHSCT outcomes for people with MS receiving medium intensity chemotherapy where the treatment is provided at an experienced hospital centre

An important part of this study was the work conducted by MS Research Australia-funded PhD Student and clinician, Dr Jennifer Massey, who looked at the profile of immune cells in patients following the treatment. This work showed that the attacking types of T immune cell were still wiped out and that there was a sustained rise in other immune cells that work to calm and regulate the immune system. By comparison, these changes were not seen in patients who received the same AHSCT for lymphoma. This suggests that in people with MS, AHSCT may work by turning off the attacks as well as restoring the immune system’s ability to regulate itself. These results may help us work out how to better treat MS without needing to use intensive chemotherapy treatments.

This study shows that AHSCT can provide good outcomes for people with relapsing MS who do not respond to other types of MS treatments. The results are similar to those seen in other international studies and confirm that people with relapsing-remitting MS respond better to AHSCT than those with secondary progressive MS.

Each person’s situation is unique and decisions about any MS treatments, taking into consideration the potential benefits, risks and side effects, should be made in careful consultation with your medical team. A small number of Australian hospitals are currently providing this treatment for people with MS, but can only do so for patients who have failed to respond to other MS therapies and with a referral from a neurologist.

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Stem Cell Transplant Enabled This Wheelchair-bound MS Patient To Walk Again

 

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.

Roy Palmer, 49, was confined to a wheelchair for several years. He was an MS patient and suffered from multiple sclerosis many years ago due to which he couldn’t walk again. When the radical stem cell treatment emerged, he decided to give it a shot. Palmer went through a hematopoietic stem cell transplant (HSCT) after seeing a documentary about it on a program called Panorama on BBC. He said in an interview, “If they can have that done, on a trial, why can’t I have it done?” HSCT is performed by taking a patient’s own stem cells and destroying the immune system with radiation or chemotherapy. After performing all these steps, hematopoietic stem cells are transplanted back into the body.

The National Multiple Sclerosis Society has listed the treatment as risky and is still in the experimental stages. The procedure is yet to be approved by the Food and Drug Administration. The long-term side effects of the procedure include infections and infertility in both genders. Palmer decided to take the risks as they were worth a shot. He said that two days after the surgery he regained the feeling in both his legs and learned how to walk again. He said, “I’ve been given a second chance at life and I started volunteering at my local police station. We went on holiday not so long ago to Turkey and I walked on the beach. Little things like that, people do not realize what it means to me.”

Multiple sclerosis cripples millions of patients all over the world. It is also known as MS. The disease tricks the body to attack the protective coverings of nerves. This causes extensive damage to the spinal cord and the brain. Often the mobility of patient degrades from walking to vision loss, paralysis, and reduced brain functions. The issue is that the treatment of the disease has not been discovered yet. The Mayo Clinic says, “We went on holiday not so long ago to Turkey and I walked on the beach. Little things like that, people do not realize what it means to me.”

It is currently treated with corticosteroids like oral prednisone or with plasma exchanges if the steroids are not working on the patient. The disease was first highlighted in 2015 when a team from Chicago’s Northwestern Memorial Hospital performed first HSCT for an MS patient. The success of the treatment brought hope into the research for a cure to MS. HSCT is a life-saving treatment as well as cost effective. Most estimates show that MSCT treatments cost around $125,000. The FDA-approved drug for the treatment of MS cost around $5000 per month. MS patients are treated with one or more of those drugs.

The researchers from Northwestern Memorial Hospital has also advised MS patients that MSCT is not for everyone. Dr. Richard Burt explained, “If you’re doing well on first-line therapies, interferons or Copaxone, good, that’s where you should stay. But if you’re having frequent relapses, two or more a year despite those therapies … I think that’s the group that, rather than going to Tysabri or Fingolimod, should be given this therapy because it’s so much more beneficial. Plus, if you wait until you’ve had all those other [DMTs] then you increase the risk of this treatment.”

However, Palmer has huge gratitude for the procedure. In an August opinion piece, raising money for the Imperial Healthy Charity’s Blood Fund he wrote, “It might not be a cure for my MS, but this stem cell transplant has changed my life. I’m now able to walk unaided again. It can still be very painful, so I’m continuing to have physiotherapy to strengthen my legs. Some of the exercises are really hard but I’m hoping it improves.”

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Cancer treatment for MS patients gives ‘remarkable’ results

UK doctors in Sheffield say patients with multiple sclerosis are showing “remarkable” improvements after receiving a treatment usually used for cancer.

About 20 patients have received bone marrow transplants using their own stem cells. Some patients who were paralysed have been able to walk again. Prof Basil Sharrack, of Sheffield’s Royal Hallamshire Hospital, said: “To have a treatment which can potentially reverse disability is really a major achievement.” Around 100,000 people in the UK have MS, an incurable neurological condition. Most patients are diagnosed in their 20s and 30s. The disease causes the immune system to attack the lining of nerves in the brain and spinal cord.

Immune system ‘rebooted’

The treatment – known as an autologous haematopoietic stem cell transplant (HSCT) – aims to destroy the faulty immune system using chemotherapy. It is then rebuilt with stem cells harvested from the patient’s own blood. These cells are at such an early stage they’ve not developed the flaws that trigger MS. Prof John Snowden, consultant haematologist at Royal Hallamshire Hospital, said: “The immune system is being reset or rebooted back to a time point before it caused MS.” About 20 MS patients have been treated in Sheffield in the past three years. Prof Snowden added: “It’s clear we have made a big impact on patients’ lives, which is gratifying.”

Multiple Sclerosis

In MS the protective layer surrounding nerve fibres in the brain and spinal cord – known as myelin – becomes damaged. The immune system mistakenly attacks the myelin, causing scarring or sclerosis. The damaged myelin disrupts the nerve signals – rather like the short circuit caused by a frayed electrical cable. If the process of inflammation and scarring is not treated then eventually the condition can cause permanent neurodegeneration.

The BBC’s Panorama programme was given exclusive access to several patients who have undergone the stem cell transplant. Steven Storey was diagnosed with MS in 2013 and, within a year, went from being an able-bodied athlete to needing a wheelchair and losing sensation in much of his body. BBC’s panoramn programmeHe said: “I went from running marathons to needing 24-hour acute care. At one point I couldn’t even hold a spoon and feed myself.” Within a few days of the transplant he was able to move his toes, and after four months he could stand unaided. Steven still needs a wheelchair but is astounded at his progress: “It’s been incredible. I was in a dire place, but now I can swim and cycle and I am determined to walk.” Holly Drewry was just 21 when she was diagnosed with MS and her condition deteriorated after she gave birth to her daughter Isla.

She said “Within a couple of months I got worse and worse. I couldn’t dress or wash myself; I didn’t even have the strength to carry my daughter.” Holly needed a wheelchair before her transplant, but after the treatment she walked out of hospital. She said: “It’s been a miracle. I got my life and my independence back and the future is bright again in terms of being a mum and doing everything with Isla.” Two years on she has suffered no relapses and there is no evidence of active disease on her scans. Doctors describe her MS as dormant, but there is hope that the transplant might be a permanent fix.

Cost-Effective

United statesThe Royal Hallamshire Hospital – together with hospitals in the United States, Sweden and Brazil – is part of an international trial, MIST, which is assessing the long-term benefits of the stem cell transplant. All those on the trial have relapsing remitting MS, where patients experience attacks – or relapses – followed by periods of remission. The treatment involves intensive chemotherapy, so patients are warned that there are side-effects such as nausea and hair loss. Paul Kirkham, another MS patient, said he was glad to have had the transplant but added: “It does knock you. I’d rather have done 10 rounds with Mike Tyson.” The transplant involves a one-off cost of around £30,000, which is comparable to the yearly cost of some MS treatments.

Because the procedure involves no new and instead re-purposes an existing therapy using the patient’s own cells, there is little profit incentive for companies to get involved. Prof Richard Burt, Northwestern University, Chicago carried out the first HSCT for MS as long ago as 1995 and is coordinating the international trial which began in 2006. He said: “There has been resistance to this in the pharma and academic world. This is not a technology you can patent and we have achieved this without industry backing.” A study published last year involving MS patients in Chicago showed significant reductions in neurological disability, and for some the improvements persisted for at least four years, although there was no comparative control group.

The outcome of the more detailed MIST trial – which will report in a couple of years – could determine whether the stem cell transplant becomes a standard NHS treatment for many MS patients. Dr Emma Gray, head of clinical trials at UK’s MS Society , said: “Ongoing research suggests stem cell treatments such as HSCT could offer hope, and it’s clear that in the cases highlighted by Panorama they’ve had a life-changing impact. “However, trials have found that while HSCT may be able to stabilise or improve disability in some people with MS it may not be effective for all types of the condition.” Dr Gray said people should be aware it was an “aggressive treatment that comes with significant risks”, but called for more research into HSCT so there could be greater understanding of its safety and long term effectiveness.

Panorama is broadcast on BBC One at 20:30 on Monday 18 January 2016.…

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