A Breakthrough in Duchenne Muscular Dystrophy

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Duchenne Muscular Dystrophy (DMD or Duchenne) is a fatal genetic disorder that affects 1 in every 3,500 boys born globally. Duchenne results in progressive muscle weakness due to a mutation in a gene encoding for dystrophin on the X-chromosome. It first affects boys around age four and by age twelve, most people with Duchenne cannot walk. By age 21, most people cannot move below the waist. Few people with Duchenne live past thirty, due to the serious medical complications that include issues with the heart and lung functioning.
Researchers at the Cedars-Sinai Heart Institute offered hope last week to families of kids with Duchenne, when they released the results of the aptly-named “HOPE Duchenne” trial, a joint Stage I/II-A trial that used cellular therapy to treat Duchenne. In the trial, children with Duchenne were given cardiac progenitor cell infusions through a catheter placed in their three main arteries. Cardiac progenitor cells, are a certain type of stem cell that when signaled, develop into new heart tissue.
This is important for children with Duchenne, as heart tissue is, after all, muscle tissue, and Duchenne can lead to the wasting away of heart tissue just as it leads to the weakening of arms or legs. Later in life, cardiomyopathy is a grave concern for people with Duchenne. “Generally, the primary cause of death in [patients with Duchenne] is heart failure,” said Eduardo Marban, MD, PhD, the lead researcher of the study. “If we can slow or reverse heart failure in Duchenne patients, it will be a step forward.” But research to this point has not addressed this need.
“The need is great because there is no current treatment to address heart failure in these patients,” said Dr. Marban. Dr. Marban has been at the forefront of this area of research, developing the process to make the type of cell that the children were treated with, “cardiosphere-derived cells,” or CDCs. Cardiosphere-derived cells are harvested from donor hearts, so they carry none of the moral ambiguity of embryonic stem cells, and can easily be made available.
In the study, twenty-five children with Duchenne were treated. Twelve were treated with the experimental cellular therapy, while the other thirteen received the standard therapy for a child with Duchenne. The results were striking. The patients treated with cellular therapy had a 7% reduction in scarring from cardiomyopathy compared to when the patients started the therapy. For the children receiving the standard therapy, their scarring continued to worsen.
Muscular regeneration was not confined to the heart; the people that received the treatment had stronger arms, as well. One year after treatment, eight out of nine of the patients that had advanced impairment of the muscles in their arms showed improvements in their arm strength. In contrast, none of the patients receiving the standard course of treatment saw improvements and all continued to get weaker.
This study shows the promise of cellular therapy in helping people with Duchenne to live longer, happier lives. If studies like this can develop methods of treatment that can reverse, rather than just slow, the effects of Duchenne, that can lead to real advances in both length and quality of life.
Dr. Marban and his team are not done. Phase II trials for the CDC-based treatment of Duchenne are scheduled to start in early 2018. Dr. Ronald G. Victor, another primary researcher at Cedars-Sinai, says that in this trial, “instead of a one-time infusion during a cath lab procedure, the patients will receive the CDCs in an intravenous drip.” This would make the treatment even less invasive than in the stage I/II trial, and therefore more accessible to patients. Also, patients “will receive multiple treatments spaced out over several months,” according to Dr. Victor.
For parents of children with Duchenne, both now and in the future, this study offers hope that perhaps a cure is possible. Sometimes, hope makes all the difference in the world.

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Antibiotics: Friend or Foe?

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While many ancient cultures used molds and other plants to treat infections, it was Alexander Fleming who received the credit for the discovery of penicillin in 1928. The discovery of antibiotics is considered one of the most important advances in 20th-century medicine saving countless lives. So what is the catch? Antibiotics can disrupt our microbiome which helps perform many functions needed to keep us healthy, as well as can cause drug resistance. According to the WHO, if the necessary steps aren’t taken to counter antibiotic resistance, humankind is on its way to a future where minor injuries and infections can be a significant cause death.
Actually, according to the CDC, approximately 80 million antibiotic prescriptions per year have been deemed unnecessary and in the United States alone, approximately 2 million individuals are infected annually with bacteria that show antibiotic resistance causing 23,000 deaths. U.S. Antibiotic Awareness Week coming to a close for 2018 is a yearly national effort to help the general public be aware of antibiotic resistance as well as come up with improved guidelines for antibiotic use and prescription writing. The CDC is working to improve antibiotic use in health facilities as well as farms and this week-long effort coincides with the European Antibiotic Awareness Day, Canada’s Antibiotic Awareness Week, Australia’s Antibiotic Awareness Week, and the World Antibiotic Awareness Week showing this is truly an international problem.
Being aware of antibiotic resistance and improving prescription guidelines has become an international priority. So how do we fix this? Education is a good start. Like many lessons, education is key as many people use antibiotics when the pathogen is a virus, not bacteria. Some share their antibiotics with others and others use more than the recommended dose thinking the bacteria will die faster. According to the WHO, if the necessary steps are not taken to counter antibiotic resistance, in the future minor wounds and infections may cause death and the common strep throat or ear infection will become a parent’s worst nightmare.
Additionally, we must take into consideration the deleterious effects antibiotics may have on homeostasis, the equilibrium of the collective microbes that are within each of us composed of bacteria, bacteriophage, fungi, protozoa and viruses. This equilibrium can be altered when we take antibiotics or immunomodulators. Most of the microbes in the microbiome do not cause disease and in fact act to perform many important functions that we cannot perform ourselves. They digest food to generate nutrients for host cells, synthesize vitamins, detoxify carcinogens, metabolize drugs, stimulate the renewal of cells in the gut lining and activate and support the immune system. Microbes can adapt to exploit new habitats and carbohydrate composition seems to drive colonization by those microbes that have the ability to metabolize these body-site specific carbohydrates. This is important as high or low microbial diversity can have different implications for health or disease depending on body location. For example, it has been shown that low microbial diversity in the gut is associated with obesity, inflammatory bowel disease and Crohn’s disease, whereas high microbial diversity in the vagina is often associated with bacterial vaginosis. Loss of metabolic capabilities of certain microbiomes has been seen in particular diseases such as Crohn’s Disease, ulcerative colitis or neonatal necrotizing enterocolitis.
While these pathogens can cause disease in those individuals with weakened immune systems, these microbes are responsible for maintaining cell function and other properties needed for maintaining the body site ecosystem in healthy individuals. We have learned that the bacteria living in each of us are “friend” not “foe”, and we must stop and think before taking antibiotics which can destroy the equilibrium, which we so heavily rely on to stay healthy.
Cells Are the New Cure: The Cutting-Edge Medical Breakthroughs That Are Transforming Our Health by Robin L. Smith, M.D. and Max Gomez, Ph.D.

Cells are the New Cure

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The future of medicine is happening now. Revolutionary new science is providing cures that were considered science fiction five years ago – and not with pills, surgery or radiation– but with human cells.


Promising treatments now in more than 35,000 clinical trials could have dramatic impacts on cancer, autoimmune diseases, organ replacement, heart disease, and even aging itself. These treatments, which are saving the lives of patients in the trials, will soon be rolled to the medical community at large.


The key to these breakthroughs is the use of living cells as medicine instead of traditional drugs. Research has found that our bodies are virtual treasure troves of adult cells that act as agents of remarkable healing and repair. These cells have been called adult stem cells because they resemble embryonic cells in their ability to transform into many other cell types but without the ethical and moral concerns of embryonic cells.


Adult cells can reprogram cells to work more efficiently. They can also carry edited genetic code to repair mistakes and deliver targeted therapies, allowing them to address and even cure many diseases affecting children and adults.


The applications of these cellular therapies are broad and growing. Regenerative medicine allows doctors to repair injured and aging tissues and, incredibly, to create artificial body parts and organs in the lab. New technologies make it possible to harness the immune system to fight cancer and reverse autoimmune diseases like multiple sclerosis, type 1 diabetes, and rheumatoid arthritis. We’re also seeing advances to alleviate the effects of strokes, Alzheimer’s disease, and even allergies. CRISPR, a new technology for targeted gene editing, promises to eradicate genetic diseases, allowing us to live longer lives–possibly beyond age 100 in good health.


Cells Are the New Cure takes you on a tour of the most exciting and leading-edge developments in medicine. You’ll meet the doctors performing the lifesaving research and the patients who have survived illnesses that, before today, were considered a death sentence. The content inside these pages could save your life or the life of someone you love.



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