By Donald R. Hopkins

The recent global campaign launched against a select number of neglected tropical diseases (NTDs) is a welcome development. But we should be as careful about measuring progress toward the control or elimination of these diseases as we are about choosing which ones to target.

The World Health Organization (WHO) has done the world a great service by raising awareness of the need and opportunity to reduce poverty by addressing certain NTDs that are associated with poverty. In October, it issued its first report on the extent of NTDs and set out a plan for collective action against 17 of them, from Chagas disease to leprosy to Guinea worm disease (https://www.who.int/neglected_diseases/2010report/en/index.html/).

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Image by The Carter Center/Louise Gubb

By Elie Dolgin

In the US, many of the nearly one in ten people suffering from depression rely on so-called ‘third generation’ antidepressants, which typically alter the reuptake of the signaling molecule serotonin. It’s no secret, though, that these drugs can take weeks to kick in, and sometimes carry serious side effects.

However, in the past few months alone, researchers have discovered a suite of potential drug targets for depression. Unlike the previous targets, such as serotonin, dopamine and norepinephrine, which act at the synapse between neurons, the newer targets act within the brain cells.

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By Elie Dolgin

Since antiretroviral therapies came onto the market more than two decades ago, the level of investment into research to treat HIV in most countries around the world has plateaued. But in South Africa, where nearly one in five adults is infected with HIV, researchers have ramped up experiments to develop drugs against the deadly disease. In fact, a report published recently in the journal Scientometrics (doi:10.1007/s11192-010-0277-6, 2010) shows that the proportion of the Rainbow Nation’s scientific output focused on HIV now soars above that of any other country (see chart).

Despite South Africa’s internal dedication to HIV research, its relatively modest size and resources mean that the country produces around 3% of the world’s scientific literature on the topic, notes study co-author Anastassios Pouris, director of the University of Pretoria’s Institute for Technological Innovation. “South Africa is putting a lot of effort into the field,” he says, “but it cannot resolve the issue by itself.” As such, Pouris argues that the country should reduce its focus on science as a means to curb the AIDS epidemic and instead turn to diplomacy to persuade richer nations to invest more in research into treatments for HIV and other diseases that disproportionately affect the African continent.

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Among the many institutions devoted to defeating diseases, there are a handful of heavyweight nonprofits that set the agenda. When one key player, the Global Fund to Fight AIDS, Tuberculosis and Malaria, launched in 2002, it ushered in an era of improved drug distribution for these infections. Now, as the board of the Global Fund meets in Sofia, Bulgaria, on 13 December, it will have to decide how to proceed with a predicted $1.3 billion dollar shortfall in funding needed to meet a growing battle against its target diseases.

Earlier this year, the Global Fund predicted that it would need $13 billion in funding for the next three years—$8.8 billion of which would go to maintaining current programs and $4.2 billion of which would go to expanding programs to implement new efforts in areas with a growing disease burden.

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Image of Global Fund director Michel Kazatchkine by US Mission Geneva206 via Flickr Creative Commons

To truly know whether a stem cell has the developmental flexibility to give rise to new, therapeutic tissue types, the cells must undergo a definitive test in which they are injected into mice and observed. Yet many say this measure is too slow, too expensive and too unreliable. Elie Dolgin goes in search of a replacement.

Earlier this year, doctors in Atlanta injected millions of cells derived from embryonic stem (ES) cells into the spinal cord of a partially paralyzed patient. The trial, run by the California-based company Geron Corporation, promises to usher in a new era of regenerative medicine in which millions of people are treated each year with the products of so-called ‘pluripotent cells’, defined by their ability to morph into any type of tissue.

The stakes for developing a reliable test for true pluripotency are high. The US National Institutes of Health (NIH) has invested more than $500 million in human ES cell research and plunged hundreds of millions more into induced pluripotent stem (iPS) cells. Once the therapies hit the clinic, pluripotent stem cells could be worth more still.

“This is easily a field that has the market potential to be in the hundreds of billions of dollars,” says Reni Benjamin, a senior biotechnology analyst at Rodman & Renshaw in New York.

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Here’s the skinny on the weight loss drugs that disappointed, the hepatitis drugs that matched the hype and others as we look back on the major headlines relating to medications in 2010.

Green Light

Gilenya (fingolomod)

This drug from Novartis was the first drug for multiple sclerosis in pill form to gain US Food and Drug Administration (FDA) approval. As a sphingosine-1-phosphate modulator, it prevents white blood cells from attacking the nervous system and has proved more effective at reducing relapse rates than interferon-beta, an existing multiple sclerosis drug.

GRNOPC1

Geron’s much-anticipated phase 1 clinical trial of human embryonic stem cell-derived neural precursors for spinal cord injury finally got off the ground when doctors in Atlanta injected these cells, GRNOPC1, into a human subject. The company plans to implant the cells into up to ten people, who will be followed for up to two years, to assess the safety of the treatment.

Tenofovir

A vaginal gel containing this antiretroviral compound reduced the risk of HIV transmission to women by 39% in the CAPRISA 004 clinical trial, which encouraged the FDA to grant fast-track status to the product.

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We look back on some of the key insights into biomedicine published this year.

Development: Sticking to thalidomide

The sedative and morning-sickness drug thalidomide was withdrawn in 1961 after physicians observed that it caused multiple birth defects, including limb, ear, heart and gut malformations. In recent years, the drug has been reintroduced under strict control for different indications, such as cancer. Yet despite nearly half a century of research, many of the molecular targets underlying the developmental defects caused by thalidomide remain unclear.

This March, Japanese scientists identified a protein called cereblon to which thalidomide binds. In zebrafish and chicken embryos, the researchers showed that thalidomide inhibits the ubiquitin ligase activity of a cereblon-containing complex, stunting limb and fin growth (Science 327, 1345–1350, 2010).

Today, thalidomide is used clinically to treat individuals with multiple myeloma and leprosy, so understanding the molecular targets of thalidomide and their roles in limb formation may help produce safer derivatives of this drug. —AF

Immunology: Injury prone

For years, doctors have puzzled over the molecular similarities between how the body’s immune system reacts to both internal tissue trauma and external microbial infections. Now, scientists have chalked up the common inflammatory response to the shared evolutionary origin of mitochondria and bacteria.

Researchers from the Beth Israel Deaconess Medical Center in Boston tested the blood plasma of 15 trauma patients and found circulating components of mitochondrial DNA and proteins known as formyl peptides. Further dissecting the immune response, the researchers showed these components bind to formyl peptide receptor-1 and Toll-like receptor 9 on the surface of immune cells known as neutrophils, promoting their activation and attracting them to the site of injury (Nature 464, 104–108, 2010).

Notably, these same receptors and signaling pathways are also triggered by invading bacteria, leading to a robust inflammatory response resembling sepsis. —KDS

Metabolism: PPARadigm shift

Earlier this year, after drug regulators severely restricted people’s access to the antidiabetic drug rosiglitazone (Avandia), many experts advised against pursuing similar compounds that activate the nuclear receptor peroxisome proliferator and activator of transcription-γ (PPAR-γ) (Nat. Med. 16, 614, 2010). But a new mechanistic link between obesity and diabetes could reignite the research community to develop the next generation of PPAR-γ–targeting drugs. (Click here to continue reading)

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Sometimes the retraction of a paper can be as noteworthy as the publication of one. Here we list some of the take-backs of 2010 that left the biomedical community most taken aback.

In February, days after the UK General Medical Council censured the unethical behavior of gastroenterologist Andrew Wakefield, The Lancet retracted his notorious 1998 paper that first linked autism with the measles, mumps and rubella vaccine.

In August, less than a month after Virology Journal first published an opinion paper speculating that a biblical woman supposedly healed by Jesus actually had influenza, the journal’s editor apologized for running the article, noting the authors did not provide “robust supporting data” for their hypothesis. Researchers from the Chinese University of Hong Kong had used symptoms described in the gospels of Matthew, Mark and Luke to diagnose the mother-in-law of the apostle Simon Peter, who lay sick in bed before being miraculously “cured by our Lord Jesus Christ.”

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Image by dingler1109 via Flickr Creative Commons

China

750 – number of new jobs added to Roche’s Chinese workforce this year, representing an increase of 25%.

900 – number of additional reps Pfizer wants to bring to China.

6,000 – number of layoffs worldwide announced by Pfizer in 2010

$1.6 billion – price paid by Charles River Laboratories International for one of China’s largest drug-research contractors, WuXi AppTec Co.

$50 billion – estimated value of the Chinese drug market in 2011, according to market research firm IMS Health.

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Image by Leonid Mamchenkov via Flickr Creative Commons