Stories about: clinical trials

Safety trial of algal anesthetic kicks off

Green algae
Algae similar to these could be the source of a powerful local anesthetic. (Micropix/Wikimedia Commons)
Two years ago, we told the story of the quest of Charles Berde, MD, PhD, of Boston Children’s Division of Pain Medicine, to turn an algal toxin called neosaxitoxin into a long-lasting local anesthetic.

At that time, Berde—together with Alberto Rodríguez-Navarro, MD, from Padre Hurtado Hospital in Santiago, Chile, and a Chilean company called Proteus SA—already knew that neosaxitoxin, a site 1 sodium channel blocker which in nature is produced by algal blooms, could help patients who had undergone laproscopic surgery recover more quickly and experience less pain compared with the current state -of-the-art local anesthetic called bupivacaine.

The group has now taken a big leap forward. In May, they launched a Phase 1 clinical trial at Boston Children’s in healthy male patients, aimed at showing that neosaxitoxin produced by Proteus from bioreactor-grown algae is safe using clinically relevant doses.

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Mind the gap in drug research for children

Diseases like malaria strike children harder than adults, but clinical trials for these diseases rarely include or focus on children. Why? (WHO/P. Virot)

We’re pretty focused on the safety of the things around us. Our drinking water gets checked for chemicals, bacteria and other things that could make us sick. Kids’ car seats are tested to make sure they’ll keep children safe in an accident.

But there’s one surprising arena where this focus on safety and testing often falls short: the medications we give our children. Not just in the United States, but globally.

There are lots of reasons why fewer drugs get tested for safety and efficacy in children than in adults. It’s time-consuming, expensive and, frankly, risky. The ethics of testing new medications in children are pretty thorny.

And, overall, the market for pediatric drugs is much, much smaller than that for drugs for adults, since children fortunately don’t get sick as often as us grown-ups.

But for some diseases like asthma and diarrheal diseases, children bear a greater burden than adults—one that’s not matched by the amount of research done on drugs for kids.

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Let’s liberate clinical trial data

The Gutenberg press disseminated ideas to a wider society. But in the clinical world, much information is still on "lockdown." (Wikimedia Commons)

The best things in life are free: friends, sunny days, beautiful vistas. Wouldn’t it be nice if knowledge were also free? Historically, libraries promulgated knowledge sharing because it was for the public good. We see this spirit increasingly embraced on the Internet – take the recent announcement of a collaboration between Harvard and MIT to make their courses freely available to users around the world via the edX platform.

But have we made all useful knowledge available in a way that allows for the greatest societal advancement? Not really. According to Ken Mandl, MD, MPH, director of the Intelligent Health Laboratory at the Children’s Hospital Informatics Program (CHIP), one important source of information still on lockdown is clinical trial data. In an article called, “Learning from Hackers: Open-Source Clinical Trials” published this month in Science Translational Medicine (not currently available in full text), Mandl and his coauthors call for making raw, de-identified clinical trial data free to the public.

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From fish to people – first drug ID’d in zebrafish crosses a milestone

A recent clinical trial brings the drug FT1050 one step closer to becoming the first drug identified with the help of zebrafish (above) to make it to patients. (Soulkeeper/Wikimedia Commons)

In 2007, working with zebrafish, Leonard Zon and his team in Children’s Stem Cell Program made an unexpected discovery: That a drug originally developed to treat stomach ulcers could boost the production of blood stem cells, by about four-fold.

That drug – FT1050, a chemical variant of a fatty, hormone-like molecule called prostaglandin E2 (PGE2) – recently crossed a major milestone: the successful conclusion of a Phase I clinical trial. Led by Zon’s colleague Corey Cutler, a clinical researcher at Dana-Farber Cancer Institute, the trial aimed to determine the drug’s safety as a way of helping patients who receive umbilical cord blood stem cell transplants recover their immune function more quickly.

The trial brings the FT1050 one step closer to becoming the first drug identified with the help of zebrafish to make it to patients.

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Newly approved Berlin Heart helps patients waiting for a transplant

On the Berlin Heart, Alina Siman, 4, has regained her energy which will make her a better transplant candidate when a new organ becomes available

Four-year-old Alina Siman is being kept alive on a device that gained approval in the U.S. just two weeks ago. The Berlin Heart Group’s EXCOR, a ventricular assist device manufactured in Berlin, Germany, takes over the normal function of a heart by pumping blood directly to the pulmonary artery and into the lungs.

With FDA approval granted on December 16, the U.S. joins Europe and Canada in offering the device for children of all ages with end stage heart failure.

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Magnetic brain stimulation advances to clinical trial for epilepsy

Transcranial magnetic stimulation (TMS) for epilepsyPatients with severe epilepsy can have seizures every day – sometimes waking up on the floor, not knowing what happened. For about 1 in 3 epilepsy patients, drugs are of no help. An implanted vagus nerve stimulator can sometimes control seizures, but often not. Surgically removing the excitable brain tissue can be curative, but often, too many areas of the brain are involved to effectively remove the entire seizure focus. Or the area causing the seizures is too close to a vital brain area – say, a memory or motor area – making surgery too risky.

Alex Rotenberg, a neurologist in Children’s Hospital Boston’s epilepsy program, has been having success with an experimental technique for this kind of disabling, treatment-resistant epilepsy. Known as repetitive transcranial magnetic stimulation (rTMS), it has helped a small number of patients with no other good options for controlling their seizures.

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Reducing unnecessary care: The SCAMPs manifesto

Can we reduce health care costs without rationing? (Image: Fibonacci Blue via Flickr)

We all know the problem: The cost of health care needs to come down. About five years ago, pediatric cardiologists at Children’s Hospital Boston realized it was critical to practice more cost-effectively. “Most of the money that is going to be removed from the federal budget to reduce budgetary deficits is going to come from health care in one fashion or another,” cardiologist-in-chief James Lock told an audience of senior Children’s physicians last month. “There’s no question we were under a tremendous amount of pressure.”

Seeking to eliminate unnecessary care and testing, Lock’s team first turned to clinical practice guidelines, or CPGs, a tool meant to standardize “best practices.” But it soon became clear that CPGs were ineffective, giving no insight into how to improve care or how to deal with unexpected findings. Even worse, over time, many mandated CPGs have been shown to be wrong by subsequent data.

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From kittens to Fragile X: Do all autisms share a common thread?

(AmberStrocel/Flickr)

Mark Bear’s research interests have taken him from studying vision in kittens to learning and memory in mouse models, and more recently, to the study of Fragile X syndrome, one of the leading genetic causes of autism and intellectual disability in humans. Along the way, he has made several ground-breaking contributions to neuroscience – one of which he described as one of MIT’s presenters at this week’s inaugural CHB-MIT Research Enterprise Symposium, which kicked off an exciting new scientific collaboration between MIT and Children’s.

I have followed Mark Bear’s work since I was an undergraduate at Brown University, where he used to teach the Introduction to Neuroscience course. That’s where I first learned about the seminal experiments in kittens (see this PDF), showing that covering one eye at birth rewires their brains not to “see” out of that eye, work that Bear was continuing to refine. Our paths crossed again more recently due to our common interest in studying autism.

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A new start for gene therapy for “bubble boy” disease: First U.S.-treated patient doing well

The first U.S.-treated patient with his parents. Photo: Patrick Bibbins

Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns.

After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. Jeremías had to stop attending nursery school, for fear he’d bring home an infection his baby brother might catch. When Agustín’s relatives came to help out, they had to change their clothes and wash their hands, and couldn’t enter Agustín’s room.

Agustín, born in Argentina, has a form of X-linked Severe Combined Immunodeficiency, or SCID-X1, better known as “bubble boy disease.”

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New opportunities for Angelman

Chromosome 15. Image: Wikimedia Commons

Angelman syndrome (AS) is a rare, neurogenetic condition characterized by severe developmental delay, movement disorder, speech impairment (often with a complete lack of speech) and an unusually happy demeanor. Nearly every individual with AS faces at least two major challenges in their daily life: cognitive or intellectual disability, and movement disorder, usually in the form of ataxic (uncoordinated) gait, unsteadiness, jerky movements or tremors. Seizures are also common, and present a daunting health challenge.

Arising in one out of every 10,000 to 20,000 children from the loss of an enzyme on chromosome 15 called Ube3A, AS falls in the category of orphan diseases: ones that affect fewer than one in 200,000 Americans.  There is no cure for AS, but there are therapies and medications that can help the symptoms. Seizures can be controlled with the right medications, physical therapy can improve ataxia, and speech therapy helps improve communication skills.

Like nearly all orphan diseases, research on AS has historically not been well-funded, but orphan diseases have lately gained growing attention, especially at Children’s Hospital Boston.

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