New Drug Fingolimod (Gilenya) Could Help Kids With MS

In continuation of my update on Fingolimod

Researchers say the first drug for children with multiple sclerosis vastly outperformed another common MS medication in a new clinical trial.

Fingolimod(Gilenya) reduced relapse rates by 82 percent in patients aged 10 to 17 compared with interferon beta-1a, a drug commonly used to slow the progression of the degenerative nerve disease.

Nearly 86 percent of children on fingolimod remained relapse-free after two years of treatment, compared with only 39 percent of children taking interferon beta-1a, researchers reported.

"I do recommend doctors consider using fingolimod as first-line treatment in pediatric MS," said lead researcher Dr. Tanuja Chitnis, director of the Partners Pediatric MS Center at the Massachusetts General Hospital for Children.

Based on results from this clinical trial, the U.S. Food and Drug Administration in May approved the use of fingolimod in children, Chitnis said.

That makes fingolimod "the first drug approved in the U.S. for pediatric MS," Chitnis said.

Other drugs like interferon beta-1a are used in children, but their use is considered "off-label," said Bruce Bebo, executive vice president of research for the National MS Society.

"We consider this a major development, a major milestone in the MS treatment landscape," Bebo said of fingolimod's approval for use in children.

Multiple sclerosis occurs when the immune system turns on the nervous system and attacks the protective sheath that covers nerve fibers, disrupting communication between the brain and the rest of the body.

MS causes vision problems, numbness or tingling, tremors, slurred speech and fatigue in patients. If left unchecked, it eventually will make it difficult for the person to walk.

Fingolimod is believed to treat MS by suppressing blood levels of lymphocytes, white blood cells that promote the immune system attack on nerve fibers, said Bebo, who was not involved with the trial.

"It slows down dramatically the pathways that lead to relapsing MS," Bebo explained.

The FDA approved fingolimod for use in adults in 2010, and the new trial is part of agency requirements to test new drugs in pediatric patients, Bebo said.

The peak age of MS onset is the 30s and 40s, but about 3 to 5 percent of patients develop symptoms of the disease in childhood, researchers said in background information.

For the clinical trial, 215 young patients were randomly assigned to take either fingolimod, which is an oral drug, or interferon beta-1a, which is injected.

Fingolimod kept MS from progressing in more than 8 out of 10 children taking the drug for two years, more than double the percentage of kids taking interferon beta-1a.

Fingolimod also slowed the development of lesions on the brain and spinal cord, a hallmark of MS. The rate of new or newly enlarged lesions discovered through MRI was 4.4 with fingolimod and 9.3 with interferon beta-1a.

Both drugs carried a high risk of adverse events, 89 percent with fingolimod and 95 percent with interferon beta-1a.

Serious adverse events occurred in about 17 percent of patients taking fingolimod, and included seizures, infection and low white blood cell counts.

"The ultimate decision is based on a clear benefit-risk discussion between the physician and family-patient. A careful review of potential side effects and monitoring is required," Chitnis said.

The findings were published Sept. 12 in the New England Journal of Medicine.

https://en.wikipedia.org/wiki/Fingolimod

FDA approves fingolimod drug for multiple sclerosis...

Fingolimod (see structure), a drug modified from a fungus  (Isaria sinclairii), a structural analogue of sphingosine and gets phosphorylated by sphingosine kinases in the cell originally found in Asian wasps, prevents autoimmune attacks by trapping white blood cells in the body's lymph nodes. Two large Phase III clinical studies published in February found that fingolimod was at least twice as effective in preventing MS attacks when compared to placebo or current treatments. 

Research on additional uses for fingolimod continues at the University of Chicago, including a new clinical trial in patients with progressive MS, for which there are no available treatments. With fingolimod adding to the recent boom of new MS therapies, and with a number of clinical trials for new therapies in progress, patients should be sure to seek out an experienced MS center for their care.

As per the claim by the lead researcher, Anthony Reder, MD, Professor of Neurology at the University of Chicago Medical Center,  fingolimod is first oral medication for multiple sclerosis was approved  by the Food & Drug Administration. He also claims that'

"We have six drugs right now, and they all involve injections. So the convenience alone of a pill is a major change in how we treat MS."

Hope people suffering from MS, (A chronic, neurologic disorder, which affects roughly 400,000 Americans and 2.5 million people around the world.  MS can cause issues with walking and movement, fatigue, weakness, pain, and loss of vision. Patients with relapsing-remitting MS suffer from intermittent and unpredictable immune system attacks that can damage the brain, spinal cord, and eyes) breathe a sigh of relief..

More..

FDA Expands Approval of Gilenya (fingolimod) to Treat Multiple Sclerosis in Pediatric Patients

In continuation of my update on fingolimod

The U.S. Food and Drug Administration today approved Gilenya (fingolimod) to treat relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older. This is the first FDA approval of a drug to treat MS in pediatric patients.

“For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “Multiple sclerosis can have a profound impact on a child’s life. This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis.”

Gilenya was first approved by the FDA in 2010 to treat adults with relapsing MS.

MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body. It is among the most common causes of neurological disability in young adults and occurs more frequently in women than men. For most people with MS, episodes of worsening function and appearance of new symptoms, called relapses or flare-ups, are initially followed by recovery periods (remissions). Over time, recovery may be incomplete, leading to progressive decline in function and increased disability. Most people with MS experience their first symptoms, like vision problems or muscle weakness, between the ages of 20 to 40. Two to five percent of people with MS have symptom onset before age 18 and estimates suggest that 8,000 to 10,000 children and adolescents in the U.S. have MS.

The clinical trial evaluating the effectiveness of Gilenya in treating pediatric patients with MS included 214 evaluated patients aged 10 to 17 and compared Gilenya to another MS drug, interferon beta-1a. In the study, 86 percent of patients receiving Gilenya remained relapse-free after 24 months of treatment, compared to 46 percent of those receiving interferon beta-1a.

The side effects of Gilenya in pediatric trial participants were similar to those seen in adults. The most common side effects were headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities.

Gilenya must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks. Serious risks include slowing of the heart rate, especially after the first dose. Gilenya may increase the risk of serious infections. Patients should be monitored for infection during treatment and for two months after discontinuation of treatment. A rare brain infection that usually leads to death or severe disability, called progressive multifocal leukoencephalopathy (PML) has been reported in patients being treated with Gilenya. PML cases usually occur in patients with weakened immune systems. Gilenya can cause vision problems. Gilenya may increase the risk for swelling and narrowing of the blood vessels in the brain (posterior reversible encephalopathy syndrome). Other serious risks include respiratory problems, liver injury, increased blood pressure and skin cancer. Gilenya can cause harm to a developing fetus; women of child-bearing age should be advised of the potential risk to the fetus and to use effective contraception.

Cladribine-the first oral disease-modifying multiple sclerosis therapy ?

In my earlier blog, I have mentioned about the NDA (new drug application) of this drug Cladribine as drug to treat Multiple Sclerosis.  Now as per the report by Decision Resources, one of the world's leading research and advisory firms for pharmaceutical and healthcare issues, finds that a recent "refuse to file" (RTF) letter issued by the FDA regarding Merck Serono's oral cladribine has heightened the competition between oral cladribine and its primary competitor, Novartis/Mitsubishi Tanabe's FTY-720 (fingolimod), to be the first oral disease-modifying multiple sclerosis therapy to reach the market in the United States.

Although oral cladribine's first-to-market advantage over FTY-720 will be reduced as a result of a likely delay to market caused by the FDA's action, oral cladribine is still expected to launch in the U.S. in 2010 while FTY-720 remains on track to launch in early 2011. .....

Ref : http://www.decisionresources.com/News-and-Events/Press-Releases/Multiple-Sclerosis-010510

Multiple sclerosis drug shows promise for preventing heart failure

A drug already approved to treat multiple sclerosis may also hold promise for treating cardiac hypertrophy, or thickening of the cardiac muscle-a disorder that often leads to heart failure, researchers at the University of Illinois at Chicago College of Medicine report. 

Using an experimental mouse model of cardiac hypertrophy, Solaro and his team found that FTY-720 (Fingolimod, see structure) significantly reduced heart mass; lessened fibrosis, or stiffening of the heart muscle; and improved overall cardiac function in the mice that received the drug.

The researchers also showed that the drug inhibits expression of several genes involved in cardiac hypertrophy.

"We saw that FTY-720 blocked the activity of a protein we know is involved in causing heart-cell thickening," said Solaro. When that protein is blocked, he said, collagen and other proteins involved in heart-cell thickening are also down-regulated.

Collagen, a fibrous protein found between heart cells, causes the heart muscle to become stiff. Collagen is often overabundant in people with cardiac hypertrophy.

"When the heart muscle is stiff, it actually takes effort to relax the heart and allow blood to flow into the ventricles, so this is another way this disease causes the heart to work harder than it should have to," Solaro said.

"FTY-720 is a potential therapy to treat this disease and prevent heart failure for people where the disease is acquired through high blood pressure, and possibly inherited hypertrophy as well," he said.

Ref : http://circheartfailure.ahajournals.org/content/6/4/833

Multiple sclerosis drug shows promise for preventing heart failure