Archive by date | February 2012

Chip promises better diagnosis for common blood disorder

Chip promises better diagnosis for common blood disorder

For more than fifty years, blood smear tests for sickle cell disease have been the standard diagnostic tool for physicians. But the tests, which show whether the patient’s red blood cells have an abnormal form of the iron-carrying protein hemoglobin that will cause them to take on a crescent shape in response to low oxygen levels in the blood, fails to predict the severity of symptoms. It is a large diagnostic loophole, considering the symptoms of sickle cell disease, which affects more than 13 million people worldwide, can range from tiredness to life-threatening blood vessel clogs. A new microfluidic chip promises to change that by providing a way to measure the risk of dangerous vascular clogging before it happens.  Read more

Small biotechs raring to cash in on the orphan disease market

Small biotechs raring to cash in on the orphan disease market

The high sticker price paid last year by the French drug giant Sanofi for Genzyme, the preeminent rare disease company, was widely seen as a ringing endorsement for the Cambridge, Massachusetts–based biotech’s innovative business model. Although Genzyme had started to diversify—much to some analysts’ chagrin—at its core lay a deep pipeline of enzyme replacement therapies (ERTs) that served small patient populations but with steep reimbursement rates. “Genzyme leveraged their enzyme replacement therapies across multiple rare diseases and showed it could be profitable,” says Matthew Riordan, an analyst at Putnam Associates, a biopharma consulting firm based in Burlington, Massachusetts.  Read more

VIDEO: Stem cell discovery puts women’s reproduction on fertile ground

Researchers have discovered a population of human ovarian stem cells with the potential of forming new eggs during a woman’s reproductive years. The findings, reported online today in Nature Medicine, could lead to new therapies that might help extend female fertility into late middle age and beyond.  Read more

Open innovation drug discovery looks to the masses for insight

Open innovation drug discovery looks to the masses for insight

Companies these days use crowdsourcing for everything from striking gold to marketing facial moisturizer. Now a new startup, Transparency Life Sciences, hopes to harness that collaborative power to make clinical trials more effective and efficient by asking the opinions of doctors as well as patients and their families. The company, launched last month and based in New York, is the latest in a string of so-called ‘open innovation’ drug development initiatives. But until now most of these efforts have only crowdsourced from a limited group: researchers.  Read more

FDA moves to fix drug shortages, but warns that the problem is not over

FDA moves to fix drug shortages, but warns that the problem is not over

The latest twist in the saga of US drug shortages came today when the country’s Food and Drug Administration (FDA) announced that it had found alternative sources for the cancer drugs methotrexate and doxorubicin, two of the 220 drugs currently in short supply in the US.  Read more

The price of failure: New estimate puts drug R&D in the billions per agent

The analytical fisticuff over how expensive research and development is for pharmaceutical companies flared up again today with a new estimate that pegs the cost of inventing a new medicine at well over the $1 billion price tag often tossed around in the industry. After factoring in money spent on drug failures, Bernard Munos of the InnoThink Center for Research In Biomedical Innovation says that the average cost to bring a drug to market closer to a staggering $4 billion.  Read more

Ubiquitin pioneer awarded 2012 prize in biomedicine

Ubiquitin pioneer awarded 2012 prize in biomedicine

The researcher who revealed that ubiquitins act like labels, tagging other proteins for destruction, received this year’s Frontiers of Knowledge Award in biomedicine from the BBVA Foundation in Spain. The award—which includes an unrestricted cash prize of €400,000 ($525,800)—went to Alexander Varshavsky, a molecular biologist at the California Institute of Technology, the foundation announced on Monday. Varshavsky’s work on ubiquitin advanced understanding of immune system disorders, cancers and neurodegenerative diseases.  Read more

A whole clot of hope for new hemophilia therapies

A whole clot of hope for new hemophilia therapies

People with hemophilia have a lot of reasons to be hopeful these days. A December gene therapy study showed early success, and yesterday the Dublin-based pharmaceutical company Shire announced a partnership with the Richmond, California-based biotech Sangamo BioSciences. Together, the companies plan to develop hemophilia treatments that target defects in four clotting factor genes with the zinc finger DNA-binding protein technology developed by Sangamo. Shire will provide financial support to bring these therapies through clinical testing.  Read more