February 2020

Physical fitness

Elamipretid Improves Fitness In Barth Syndrome, Analysis Finds

Elamipretid (MTP-131) improves the fitness of people with Barth syndrome, compared to the natural course of the disease, according to a new comparison using data from the TAZPOWER phase 2/3 clinical trial.

Stealth BioTherapeutics, the company that develops elamipretid and funded TAZPOWER, plans to meet with the FDA later this quarter to discuss these results.

Barth syndrome is a rare mitochondrial disease caused by mutations in the gene tafazzine (TAZ). Such mutations lead to low levels of cardiolipin, a lipid (fatty compound) necessary for the production of energy by the mitochondria. As such, common symptoms of Barth syndrome include muscle weakness and fatigue.

Elamipretid can enter mitochondria and protect cardiolipin, which can restore cellular energy production. It is given by subcutaneous injection (under the skin).

The TAZPOWER clinical trial (NCT03098797) enrolled 12 people with genetically confirmed Barth syndrome. The trial was conducted in two parts: in the first, participants received daily injections under the skin of 40 mg of elamipretid for 12 weeks, then 12 weeks of placebo, or vice versa, with a wash- out ”of four weeks. period between. This trial design is called a crossover study, and it allows for greater statistical power because there is both treatment and placebo data for all participants.

Following the crossover portion of the trial, 10 of the participants enrolled in an open-label extension, during which they all received elamipretid and were followed up to 168 weeks (3.2 years ).

Results previously reported by TAZPOWER suggested that elamipretid improved heart function.

In the new analysis, data from the trial was compared to data from 19 untreated people with Barth syndrome, which were collected from 2012 to 2019 by investigators at the Johns Hopkins Kennedy-Krieger Institute in Baltimore, Maryland. These data were used to create a model of the natural course of Barth syndrome.

To minimize bias, Stealth did not have access to this natural evolution data while the TAZPOWER trial was ongoing, and the statistical team that developed the natural evolution model was separate from the team that analyzed the results of TAZPOWER.

The main focus of TAZPOWER was the Six Minute Walk Test (6MWT), which, as the name suggests, measures how far a person can walk in six minutes. This is commonly used to assess physical condition in ambulatory people.

TAZPOWER participants (including open-label extension) receiving elamipretid for up to a year had an average improvement of 6MWT over 80 meters (approximately 260 feet). In contrast, in the natural course of the disease, improvement was less than one meter (less than three feet). This difference was statistically significant, meaning that it was unlikely to be due to chance.

There were also statistically significant improvements, compared to the natural course, in muscle strength tests and in sit-stand assessments (ease of getting up from a seated position).

According to Stealth, more detailed results will be presented at future medical meetings.

“We are grateful to have access to such recent and carefully collected natural history data to serve as a control for our open-label TAZPOWER extension, demonstrating the enormous potential of elamipretid,” said Reenie McCarthy, CEO of Stealth , in a press release. “Thanks to the foresight and commitment of our researchers at Johns Hopkins and the Barth Syndrome Foundation, we were able to show that the effect of elamipretid on important functional parameters associated with Barth is not only striking, but far beyond what one would expect based on the natural course of the disease.

“Our patient community is in desperate need of therapy that addresses the complex, multi-system challenges that arise as a result of Barth Syndrome,” said Emily Milligan, Executive Director of the Barth Syndrome Foundation. “In our meetings with the FDA, the Agency has stressed the importance of natural history data to support therapeutic development in this ultra-rare disease, and we are optimistic that elamipretid will demonstrate such measurable improvements in several key markers of the disease which have also been demonstrated. persist over time.

Marisa holds an MA in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied new genetic drivers of ovarian cancer. She specializes in cancer biology, immunology and genetics. Marisa started working with BioNews in 2018 and has written on science and health for SelfHacked and the Genetics Society of America. She also writes / composes musicals and coaches the University of Pittsburgh Fencing Club.

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Margarida holds a BS in Health Sciences from the University of Lisbon and an MSc in Biotechnology from the Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge, UK-based biotechnology company that discovers and develops fully human therapeutic monoclonal antibodies.

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Fitness exercise

Exercise helps kids excel in school

Physical activity is known to stimulate both the body andbrain functions, so it’s no wonder that exercise can also help children do better in school. However, there are not enough children who practice the minimum requirement of one hour of physical activity per day, as foreseen by the US Department of Health and Human Services (HHS). In reality, it is estimated that only 21.6% of children aged 6 to 19 met these requirements in 2015.

Exercise can be added to a child’s routine in a number of ways before, during and after school. Find out how you can help your child be more active despite a busy school schedule.

Encouraging children to be active is essential for proper growth and development. However, it is important to recommend activities that are safe and appropriate to their abilities. Exercise should be fun, so it’s something they’ll want to do.

Most physical activities for a child should include aerobic exercise of moderate to vigorous intensity, such as:

  • ride a bike
  • functioning
  • dancing
  • play active games and exercise

Play activities and sports that help children of all ages build strong bones, including:

Young children tend to prefer short periods of activity with brief periods of rest, while older teens may participate in longer, more structured activities.

The HHS recommends that children aged 3 to 5 years participate in physical activity throughout the day. Variety is the key here: you can decide to take your child to the playground, or you can play ball in the backyard.

Younger children enjoy active games, such as gymnastics or games in the jungle. You can also find clubs and teams suitable for young children in your local park to add variety.

Older children and adolescents are better equipped for weight-bearing activities. These include aerobic activities, such as soccer or lacrosse. They can also do strength training exercises, such as:

  • push ups
  • pull-ups
  • mountain climbing
  • burpees

While it’s important to engage older children in the right kinds of exercise for their age, it’s just as crucial that they get the right amount of physical activity. In 2018, the HHS released more specific guidelines for children aged 6 to 17.

The recommendations, as described in the Physical activity guidelines for Americans include:


Children in this age group need 60 minutes of aerobic activity per day. Most days should consist of moderate intensity activities, such as walking and swimming. HHS also recommends three days a week of more vigorous activities, such as biking and playing contact sports, such as basketball.

Muscle building

Children also need three days of muscle activity per week. Ideas include weight-bearing exercises, such as push-ups and gymnastics.

Strengthening bones

Your child also needs three days of bone building activity per week. Bodyweight exercises, such as burpees and running, as well as yoga and skipping rope, can help strengthen your bones.

You can duplicate certain activities. For example, running can be both an aerobic activity and a bone building activity. Swimming can help build muscle while providing effective aerobic workouts. The key is to keep moving as often as possible, selecting the activities that you enjoy and want to do again.

One way to make sure your child is getting enough physical activity is to lead by example. Try to adopt an active lifestyle yourself and make it part of the family’s daily routine.

Here are some ideas to encourage your child to be more active:

  • Make physical activity part of time spent with family.
  • Enjoy public parks, baseball fields and basketball courts in your community.
  • Keep an eye out for upcoming events that promote physical activity at school or in your child’s community spaces.
  • Challenge your child to stay away from electronic devices and play with their friends.
  • Team up with other parents in your neighborhood to create a safe environment for birthdays or the holidays.

The most comprehensive approach to improving a child’s health involves home, school and community. Parent-teacher associations can further promote these ideas by defending:

Yet the ideas above are not foolproof. Schools are increasingly burdened with testing requirements, which can reduce physical education. It is estimated that 51.6% of high school students have taken physical education classes in 2015. Only 29.8 percent went there each day.

Besides the time constraints to complete academic requirements, some children may also have other obligations, such as clubs and work. Others may have transportation issues that would otherwise help them get to safe places to play sports. Staying active requires some planning and consistency.

Physical activity is one of the best ways for children to improve their health. Aim for at least an hour of activity per day, including aerobic, muscle-building, and bone-building exercises. Health benefits aside, your kids will likely do better in school too.

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