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A large clinical trial to test the first drug specifically designed to treat autism is under way at 12 sites across the United States.
Curemark, a 10-person drug research company based in Rye, New York, says the drug, CM-AT, helps children with autism digest proteins. This in turn allows the children to ingest essential amino acids from the proteins, and ultimately produce key brain signaling molecules, company officials say.
For decades, gastrointestinal (GI) issues in children with autism have been a hot topic of debate. Up to 70 percent of children with autism report having GI problems such as stomach pain or constipation, and 60 percent have food selectivity, according to a 2006 study. A highly publicized report last summer, however, found that most of those issues are no more common in children with autism than in healthy children.
Because of this conflicting evidence, as well as the mystery surrounding how CM-AT works, some autism experts are skeptical of Curemark’s claims. Seven independent scientists contacted for this article declined to comment on the trial, citing the dearth of published data and the controversial nature of the therapy’s premise.
Nevertheless, the U.S. Food and Drug Administration has approved the Phase III trial, intended to show that CM-AT improves both the core features of autism — communicative problems, social deficits and repetitive behaviors — as well as less common symptoms, such as digestive problems. Phase III trials are randomized and placebo-controlled, and are the final stage in the long road to regulatory approval.
Ten of the 12 trial sites have begun recruiting 3- to 8-year-old children with autism, and the company says it plans to enroll 170 children by July.
“This is a very big deal,” says pediatric chiropractor Joan Fallon, Curemark’s founder and chief executive officer. “It’s been many, many years of preparing and preparing and preparing, and now we’re there.”
The face of the Mona Lisa is probably the most iconic in all of art. The coy, thin smile, the smooth cheeks, the fatty folds underneath squinty eyes.
This week — in what must have been an extremely entertaining lecture — an Italian doctor said that Mona’s features reveal that she didn’t eat too well. From the BBC (via Spoonful of Medicine):
For Dr Vito Franco, from Palermo University, she shows clear signs of a build-up of fatty acids under the skin, caused by too much cholesterol. He also suggests there seems to be a lipoma, or benign fatty-tissue tumour, in her right eye.
Dr Franco says his medical examinations reveal more than artistic viewings… “The people depicted in art reveal their physicality, tell us of their vulnerable humanity, regardless of the artist’s awareness of it,” he [said].
Children with autism have gastrointestinal problems, and special diets can help resolve these issues.
That idea is all but accepted as fact by parents, advocates and scientists alike. Many parents of children with autism report that the children have frequent stomachaches, constipation or acid reflux, and some attribute these to problems digesting gluten or dairy proteins.
In a review published Monday in Pediatrics, a large and diverse group of pediatric experts states unequivocally that there is no evidence to support the idea of autism-specific gut problems or of a so-called ‘leaky gut’ that doesn’t allow children with the disorder to properly digest nutrients.
The journal periodically publishes these consensus reports to try to address controversial issues in an objective way, and to help guide physicians in making good diagnoses and treatment plans. For this one, the panel included 28 clinicians with expertise in, among other things, child psychiatry, epidemiology, allergies, nutrition and pain.
The group’s ultimate recommendation is to treat gut problems in children with autism as you would treat them in any child. The only difference between the two groups may be in how the child responds: a stomachache may make a typical child a bit cranky, but provoke more severe outbursts in a child with autism.
The panel doesn’t rule out the possibility that future research might uncover autism-specific gut problems, pointing out that studies to date are somewhat difficult to interpret. For instance, one study estimates that more than 70 percent of children with autism have gastrointestinal troubles, whereas another pegs that number at a paltry 9 percent.
The panel recommends that studies focus on more precisely calculating this prevalence, rather than on investigating gut issues as root causes of the disorder rigorously designed studies to more precisely calculate this prevalence and to investigate gut issues as root causes of the disorder.
In the meantime, the experts say it’s best to avoid the special diets pushed by the alternative medicine community. They have shown no benefit to children with autism, they note, and, if taken to extremes, could result in harmful malnutrition.
(Edited 1/9, thanks to comment from MJ)
In 1987, Robert Getzenberg was beginning his doctorate at the Johns Hopkins School of Medicine urology department, the oldest in the country. Founded in 1915, the James Buchanan Brady Urological Institute’s historical roots seep from its every corner. Its current location boasts a cozy library showcasing the original chair and desk of the department’s founder. Exquisite medical illustrations—including the first to document prostate surgery—line the hallways. Here, under the eye of advisor Donald Coffey, a powerhouse in prostate cancer biology, the energetic young Getzenberg started hunting for distinctive molecular signatures of the disease in rat tumor tissue.
Prostate cancer biomarkers had recently become an enticing line of research. In 1986, a San Diego biotech, Hybritech, Inc., unveiled the first US Food and Drug Administration (FDA)-approved blood test for one such marker, called prostate specific antigen (PSA)—a protein that is leaked by damaged prostate cells, including cancer cells, into the blood.
But PSA was far from perfect. As the name implies, the protein is specific to the prostate, not to prostate cancer. Early tests found that most men with common (and benign) prostate inflammation also score high for PSA. So Coffey and Getzenberg were looking for a marker with fewer false positives.
For 50 years, microbiologists have known that the nucleus of a cancer cell looks drastically different from that of a normal cell: instead of forming a smooth circle, it typically has pinches in the membrane that make it look more like a lumpy snowman or a clover. In the 1970s, Coffey discovered the nuclear matrix—the three-dimensional mesh of proteins supporting a cell’s DNA—and suggested that this structure plays a part in the life cycle of the cell.
So that’s where the duo began their search, in rat models of prostate cancer tissue called ‘Dunning tumors’. Using gels that separate proteins on the basis of weight and charge, Coffey and Getzenberg and their colleagues found several proteins, including one called D-2, expressed in the Dunning tissue but not in controls.
This story falls into the category of ‘probably more amusing than it should be’. From the Wall Street Journal:
The list of things to avoid during flu season includes crowded buses, hospitals and handshakes. Consider adding this: your doctor’s necktie.
Neckties are rarely, if ever, cleaned. When a patient is seated on the examining table, doctors’ ties often dangle perilously close to sneeze level. In recent years, a debate has emerged in the medical community over whether they harbor dangerous germs.
Several hospitals have proposed banning them outright. Some veteran doctors suspect the antinecktie campaign has more to do with younger physicians’ desire to dress casually than it does with modern medicine. At least one tie maker is pushing a compromise solution: neckwear with an antimicrobial coating.
Meet ‘The Novack Experience’, a Philadelphia cover band of earnest rockers who, oh yeah, just happen to be doctors and medical students. The energetic lead is Dennis Novack, 63, an internist and an associate dean at Drexel University College of Medicine. All of the bands featured in the clip competed recently in a med school ‘battle of the bands’ concert, covered by The Scientist.
How do they find time to practice?? Here’s what members of one of the other bands, ‘Freaks of Nurture’, said:
Despite the intense pressure of PhD programs and demands of med school, the band members have found support for their music among their academic mentors. After hearing the band play, “the chair said I should spend more time on my music,” laughed [band leader Alec] Schmaier. Being known as the scientist or resident who plays in a band has its benefits, say band members. In the sea of medical school students, having a unique hobby allows you to stand out in the crowd, said singer Ehimare Akhabue. The dual identity can have other benefits, added bass player Rob Fenning. Rather than getting grilled on the difficult questions during med school interviews, questions invariably veer to what it’s like to play in a band, he said.
…is that doctors routinely call for unnecessary medical procedures, particularly expensive imaging like CTs or MRIs. From a commentary in this week’s New England Journal of Medicine:
Between 1993 and 2001, the number of myocardial perfusion scans increased by more than 6% per year, with no justification for their use based on disease rates, health care disparities, or newly published, definitive randomized trials. Since 1992, the number of CT scans obtained has quadrupled. Physicians are referring their patients for so many imaging tests that as many as 2% of cancers may be attributable to radiation exposure during CT scanning.
Eric Goosby, new PEPFAR head
Since its inception in 2003, the United States President’s Emergency Plan for AIDS Relief—PEPFAR—has spent $18.8 billion curbing the HIV epidemic in developing countries. But after providing antiretroviral drugs to more than 2.1 million people, the massive program is now shifting its focus from treatment to prevention.
In May, as part of his federal budget request, US President Barack Obama moved PEPFAR under a new administrative umbrella—the ‘Global Health Initiative’—and suggested a six-year commitment of $51 billion for PEPFAR and malaria programs.
More recently, on 19 June, the US Senate approved the president’s choice of a new PEPFAR leader: Eric Goosby, a physician and former chief executive officer of Pangaea Global AIDS Foundation.
Since his confirmation, Goosby has repeatedly stressed that big changes are in store for PEPFAR—most notably in handing over management responsibilities to local governments overseas and in scaling up prevention efforts. “Prevention is an essential component of PEPFAR and is becoming an increasingly critical priority in the absence of an HIV vaccine or cure,” he told Nature Medicine in an email.
This new focus comes as welcome news to many public health experts. “[PEPFAR] badly needs some new directions,” says Joanne Csete, associate professor of population and family health at Columbia University.
The oft-mocked organ finally gets some respect!
From Monday’s New York Times:
Scientists have discovered that the spleen, long consigned to the B-list of abdominal organs and known as much for its metaphoric as its physiological value, plays a more important role in the body’s defense system than anyone suspected.
Reporting in the current issue of the journal Science, researchers from Massachusetts General Hospital and Harvard Medical School describe studies showing that the spleen is a reservoir for huge numbers of immune cells called monocytes, and that in the event of a serious trauma to the body like a heart attack, gashing wound or microbial invasion, the spleen will disgorge those monocyte multitudes into the bloodstream to tackle the crisis.
Yes, he most certainly will, according to a passionate essay by immunologist Neil Greenspan.
Greenspan’s talking about Francis Collins, Obama’s recent pick for leading the National Institutes of Health (NIH). The NIH is one of the world’s largest scientific funding agencies, and its director is responsible for managing 27 institutes, 18,000 employees and a $31 billion annual budget.
Although some scientists have raised eyebrows at Collins’ outspoken evangelical Christian beliefs, few doubt his research cred. Over the past two decades, his research team has discovered faulty genes in cystic fibrosis, Huntington’s disease, neurofibromatosis, type 2 diabetes and several other conditions.
From 1993 to 2008, as head of the National Human Genome Research Institute, he successfully led the government’s effort to sequence the 6 billion letters of the human DNA code. (He reportedly plays a guitar inscribed with a DNA double helix.)
But Greenspan is not worried about Collins’ research qualifications, nor his nonsensical ideas about reconciling faith and science. Greenspan’s worried about Collins’ track record of hype:
