Multiple sclerosis, Muscular Dystrophy, updates

waltky · Dec 22, 2016

A "landmark" drug in treating multiple sclerosis...

Multiple sclerosis drug 'a landmark'
Thu, 22 Dec 2016 - A drug that alerts the immune system is described as a "landmark" in treating multiple sclerosis.

Trials, published in the New England Journal of Medicine, suggest the drug can slow damage to the brain in two forms of MS. Ocrelizumab is the first drug shown to work in the primary progressive form of the disease. The drug is being reviewed for use in the US and Europe. MS is caused by a rogue immune system mistaking part of the brain for a hostile invader and attacking it. It destroys the protective coating that wraps round nerves called the myelin sheath.

The sheath also acts like wire insulation to help electrical signals travel down the nerve. Damage to the sheath prevents nerves from working correctly and means messages struggle to get from the brain to the body. This leads to symptoms like having difficulty walking, fatigue and blurred vision. The disease can either just get worse, known as primary progressive MS, or come in waves of disease and recovery, known as relapsing remitting MS. Both are incurable, although there are treatments for the second state.

Patient with MS

'Change treatment'

Ocrelizumab kills a part of the immune system - called B cells - which are involved in the assault on the myelin sheath. In 732 patients with progressive MS, the percentage of patients that had deteriorated fell from 39% without treatment to 33% with ocrelizumab . Patients taking the drug also scored better on the time needed to walk 25 feet and had less brain loss detected on scans. In 1,656 patients with relapsing remitting, the relapse rate with ocrelizumab was half that of using another drug. Prof Gavin Giovannoni, from Barts and The London School of Medicine and Dentistry, was involved in the trials and said: "The results shown by these studies have the potential to change how we approach treating both relapsing and primary progressive MS." He told the BBC: "It's very significant because this is the first time a phase three trial has been positive in primary progressive MS." More than 100,000 people are diagnosed with MS in the UK, around one-in-five are progressive.

Dr Aisling McMahon, the head of clinical trials at the MS Society, commented: "This is really big news for people with the primary progressive form of multiple sclerosis. "It's the first time a treatment has shown the potential to reduce disability progression for this type of MS, which offers a lot of hope for the future." The drug is being considered by the European Medicines Agency and the US Food and Drug Administration. But Prof Giovannoni warned that patients in the UK may be disappointed as it may be hard for the NHS to fund everyone getting a drug that is likely to be expenseive. He told the BBC: "I would expect a narrow group of people to be eligible." Dr Peter Calabresi, from John Hopkins University in Baltimore, added: "This is the first drug to show a significant effect in slowing disability progression in a phase three trial in primary progressive multiple sclerosis and therefore represents a landmark study in the field." But he warned doctors to "stay vigilant" because of the risk of side-effects. Weakening the immune system increases the risk of infection and of cancer emerging.

http://www.bbc.co.uk/news/health-38392548
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waltky · Nov 2, 2017

Baby Gene Therapy Study Offers Hope for SMA...

Baby Gene Therapy Study Offers Hope for Fatal Muscle Disease
November 01, 2017 | WASHINGTON — A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots' lives, and some began to roll over, sit and stand on their own, researchers reported Wednesday.

Only 15 babies with spinal muscular atrophy received the experimental gene therapy, but researchers in Ohio credited the preliminary and promising results to replacing the infants' defective gene early - in the first few months of life, before the neuromuscular disease destroyed too many key nerve cells. "They all should have died by now, said Dr. Jerry Mendell of Nationwide Children's Hospital, who led the work published by The New England Journal of Medicine. Yet, "those babies are still improving.” Mendell cautioned that much more study is needed to prove the gene therapy works and is safe. Nor is it clear whether the replacement gene's effects would wane over time.

Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia, Oct. 4, 2017. Misa was 4 years old when he received his gene therapy treatment.

Spinal muscular atrophy occurs in about 1 in 10,000 births, and those with the most severe form, called SMA Type 1, rarely reach their second birthday. They can be born looking healthy but rapidly decline. One study found just 8 percent of the most severely affected survived to age 20 months without needing permanent mechanical ventilation to breathe. There is no cure. The first treatment wasn't approved until last December _ a drug named Spinraza that requires spinal injections every few months. The experimental gene therapy approach aims for a one-time fix.

What goes wrong

Spinal muscular atrophy is caused when a mutated gene can't produce a protein crucial for survival of motor neurons, nerve cells in the spinal cord that control muscles. Some children carry extra copies of a backup gene that produces small amounts of the vital protein, and thus have much milder forms of the disease.

Gene replacement

Scientists loaded a healthy version of the gene into a virus modified so it couldn't cause illness. Then 15 babies got a one-time intravenous injection. The virus carried the healthy gene into motor neurons, where it got to work producing the protein those nerve cells require to live. Three babies received a low dose of the gene therapy, as a first-step safety precaution. The remaining 12 got a high dose.

Results
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waltky · Mar 25, 2018

MS drug hope for delaying secondary-progressive stage...

MS drug hope for secondary-progressive stage
23 March 2018 - A study of a new drug to treat advanced cases of multiple sclerosis suggests it may be possible to delay progression of the disease in the short term, although the effects were small.

In a trial of 1,327 people, in The Lancet, 26% saw their disability worsen after three months taking siponimod compared with 32% taking a dummy drug. No drugs currently exist for secondary-progressive multiple sclerosis. An MS expert expressed caution, saying other new treatments were still needed. About 100,000 people in the UK have MS - a lifelong, progressive condition. Most are diagnosed between the ages of 20 and 70.

MS affects the central nervous system and can cause problems with:

* vision
* balance
* fatigue
* stiffness
* memory

Most cases start as relapsing-remitting MS and most of these develop into secondary-progressive MS within 15-20 years.

'Modest effect'

Patients in this trial, which was funded by drug company Novartis, had had MS for an average of 17 years - four years with secondary MS, the advanced stage. Most needed assistance with walking before the trial began. When standard measures of disability were used to track their progress, there was a 21% lower risk of walking or arm movements getting worse for those given the drug, compared with those taking the placebo. But the international research team found the drug had no effect on maintaining patients' walking speed and it had some side-effects, although it was still thought to be safe.

Siponimod is a pill taken once a day

Lead author Prof Ludwig Kappos, from the University of Basel, said: "Although the results are not as good as we wanted to see, it is a very large study, which is robust. "It means siponimod is one option to delay the disease in the advanced stage." Dr Susan Kohlhaas, director of research at the MS Society, said: "These results bring us closer to the first ever treatment for people with secondary-progressive MS - so it's big news. "This trial showed that siponimod had a modest but significant effect in slowing disability progression, which is incredibly encouraging."

'Disappointing'

But Dr Luanne Metz, from the University of Calgary, in Canada, said a second trial was needed to confirm the benefits of the drug and its impact beyond three to six months. She said: "Although siponimod seems to reduce the time to confirmed disability in secondary-progressive MS, the treatment effect was small. "In our opinion... the absence of a significant difference for the key secondary clinical outcome are disappointing results and do not suggest that siponimod is an effective treatment for secondary-progressive MS." She added: "Trials of other novel treatments that target non-inflammatory mechanisms are still needed." Before the drug becomes available on the NHS, it would need to be approved by the European Medicines Agency and then recommended as cost-effective by bodies in the UK.

http://www.bbc.com/news/health-43502845
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Multiple sclerosis, Muscular Dystrophy, updates

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