Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Peer into GEN’s crystal ball for a glimpse of what’s to come.

With all the new developments taking place in biopharma right now, it often feels like we’re living in the future—but the future holds even more promise. GEN has already offered its predictions for what’s to come in biotech this year; below are 10 trends we think are likely to reshape biopharma in the coming years—if not starting in 2013—based on a spot-check of recent journal articles, public announcements, news reports, and commentaries in GEN and elsewhere:

Transplants Without The Waiting List

Nina Tandon, Ph.D., offered a refreshing change from typical celebs-and-sleaze daytime TV fare as a guest on Katie Couric’s talk show on December 1. The senior fellow at Columbia University’s Lab for Stem Cells and Tissue Engineering—an electrical and biochemical engineer whose research focuses on directing cell growth and differentiation through electrical signals—was celebrated as a “Woman Who Should Be Famous.” Dr. Tandon’s work in growing 3D bioprinted heart tissue is expected to lead to the growth and implantation of complex organs, like the heart and liver, into patients. When Katie asked, “How long till an actual fabricated human heart can be transplanted?”, Nina replied, “I think it will be about 15 to 20 years”—the same answer she gave GEN for an October 10 article on the progress and challenges of fabricating tissue for human use, “Tissue Engineering No Soft Cell”.

Preventing Inherited Diseases

Scientists from the New York Stem Cell Foundation (NYSCF) Laboratory and Columbia University Medical Center (CUMC) developed a technique that holds potential for preventing children from inheriting mitochondrial diseases. In a study published online in Nature December 19, researchers detailed how they removed the nucleus of an unfertilized egg cell, replacing it with the nucleus of another donor’s egg cell. The egg cell contained the genome of the donor, but not her mitochondrial DNA. Cell lines were grown for more than a year and generated adult neurons, heart cells and pancreatic beta cells. The donated genome’s original mitochondria were undetectable, preventing inherited diseases from passing down to children. Next steps include production of more mitochondrial disease-free egg cells and the generation of healthy progeny in an animal model. But in their paper, the researchers hinted the technique won’t find wide use quickly: “Before proceeding with human clinical trials on the transfer of the maternal genome, it will be important to publicly discuss patient needs, ethical considerations, and to establish appropriate guidelines for the use of oocyte nuclear genome transfer in assisted reproduction.”

Gene Therapy Comes of Age

Two breakthroughs this year made it possible to talk about gene therapy coming of age, and within a generation, envision its wide use in reversing disease. One was the European Union’s November 2 approval of uniQure’s marketing authorization application for Glybera® (alipogene tiparvovec) for lipoprotein lipase deficiency, the Western world’s first approved gene therapy drug. “The precedent of an approved AAV [adeno-associated virus] product is going to help enormously in the further business development and commercialization of gene therapy,” said James M. Wilson, M.D., Ph.D., of the University of Pennsylvania Perelman School of Medicine, editor-in-chief of Human Gene Therapy, HGT Methods, and HGT Clinical Development, all published by GEN publisher Mary Ann Liebert, Inc. The other milestone was the success by researchers at University of Pennsylvania’s Perelman School of Medicine of a gene therapy that used infusions of patients’ own T cells genetically engineered to attack their tumors. The technique showed nine of 12 leukemia patients in remission, two of them for more than two years.

Remote Monitoring In Clinical Trials

More than a year after FDA urged clinical trial sponsors to use risk-based remote monitoring, AMC Health announced on December 18 that it won agency approval to do so in a Phase IIa trial by Transparency Life Sciences of the blood pressure drug lisinopril in patients with multiple sclerosis. Researchers will collect data on blood pressure and heart rate, mobility, physical and mental function, symptoms, side effects, quality of life, and medication adherence, directly from patients in their homes. It’s not the first clinical trial to use remote monitoring; AMC Health said it is also assisting a Phase I trial by a “major pharmaceutical company” it won’t identify. Pfizer claimed the distinction last year when it announced the first randomized clinical trial entirely using electronic tools, with some 600 patients participating from 10 clinical sites. As biopharmas grow their appetite for cutting R&D costs, telemonitoring can be expected to see much wider clinical use.

Written in Blood

Researchers at Johns Hopkins Kimmel Cancer Center and Howard Hughes Medical Institute earlier this month published proof-of-principle data in Science Translational Medicine showing promise for detecting cancer in late-stage patients through DNA changes seen via noninvasive whole-genome sequencing of blood plasma. Rebecca Leary, Ph.D., the study’s first author and a Johns Hopkins postdoctoral fellow, told the blog of the New York Genome Center she is hopeful the technique “could be applied in some aspect of the clinic within the next five years or so,” since the cost of DNA sequencing continues to fall (this was the year of the much-ballyhooed $1,000 genome). But she added that such sequencing must also be proven to detect early-stage cancers as well, which will require large clinical trials.

Star Trek Diagnostics

The communicators envisioned by Star Trek have come to life, thanks to today’s smartphones, so why not also the medical tricorders used by Drs. “Bones” McCoy and Beverly Crusher? In October, a research team led by Tony Jun Huang, Ph.D., of Pennsylvania State University published proof-of-concept results in the journal Lab on a Chip showing success for the technology behind the tricorders: The team’s miniature device uses two sound waves as acoustic tweezers to help sort a continuous flow of cells into five outlets on a dime-sized microfluidic chip. Researchers tested their device on human white blood cells affected by leukemia by first focusing them into the main channel, then separating them into five channels. Dr. Huang’s team is among several researchers developing tricorders. Peter Jansen, Ph.D., now at the University of Arizona, developed an open-source version while a grad student in the Cognitive Science Laboratory at McMasters University. Qualcomm hopes to advance the technology with its Tricorder X-Prize Contest, which promises a $10 million top prize for the winning tricorder. Launched in January 2012, the contest is set to run about 42 months, leaving the winner to be decided by mid-2015.

AIDS Vaccine

The Texas Biomedical Research Institute has applied for a U.S. patent for a once-in-a-lifetime HIV vaccine designed to stop transmission of the virus during sex. It’s not an AIDS cure but the next best thing, since 90% of HIV infection cases occur during intercourse, when the virus passes through the body’s epithelial cells. In January, according to the San Antonio Express-News, researchers will begin inoculating rhesus monkeys at the institute with various dosages of the vaccine at the vagina, rectum, mouth or skin—monitoring their immune response and then infecting them with different HIV strains to test their ability to protect against infection. “By about 2015 we should know if the works, or if it was just a great dream,” said Claire Gauduin, a researcher in Texas Biomed’s department of virology and immunology who invented the vaccine along with Philippe Blancou of France’s University of Nice-Sophia Antipolis.

Health Data Commons

Decades of segregated collections, paper and electronic, of human health information vital to drug discovery may give way in the coming decade or so to a single “health commons” or pool of publicly available health data, depending on the success of an initiative taking shape. Consent to Research is looking to collect data on 100,000 people during the commons’ first year, and on 1 million people during the effort’s first five years, CTR head John Wilbanks told attendees at this year’s TED Global conference in Edinburgh, Scotland last June. The more data collected, the easier it will be to apply modeling techniques leading to new hypotheses about connections between human health, DNA, and the choices people make in life. One early bellwether will be how many of the 100,000 people whose data will be returned to them by the Athena Breast Health Network opt to contribute that information to the commons.

Six-Figure Drugs

More drugs are being approved with annual costs that exceed $100,000 a year, and more will be in coming years. Among recent example is Ariad Pharmaceuticals’ drug Iclusig (ponatinib), approved by FDA December 14 for adults with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia. Iclusig is being priced at $115,000 per year’s supply; it is Ariad’s first approved branded drug product. Another recently approved drug, Cometriq (cabozantinib), is an inhibitor of multiple receptor tyrosine kinases approved November 29 for treating progressive, metastatic medullary thyroid cancer (MTC). Drug developer Exelixis plans to price Cometriq at $9,900 for a 28-day supply, or more than $119,880 annually. All the same, biopharma drug developers will want to make up from somewhere the money they will lose as generic competition grows, and as Washington reduces reimbursements as part of the Affordable Care Act. For what it’s worth, Forbes’ Matt Herper made the same prediction last year. Things surely have not changed since then.

Costlier Generics, Too

Generic drug makers will be subject this year to new fees enacted as part of the Generic Drug User Fee Amendments of 2012 (GDUFA), part of the Food and Drug Administration Safety and Innovation Act enacted in July by President Obama. Companies will have to pay for applications, as well as Drug Master Files (DMFs) that will cost them as much as $20,000 to $30,000 each. While FDA says the fees will amount to less than ten cents for the average generic prescription, PCI Synthesis says in its predictions for 2013 that the extra costs “could have an adverse effect on the generic drug industry,” especially smaller companies. “Could”? More like “will,” and you can bet generic drug makers “will” try to recoup that cost with higher prices. So much for generics as the low-cost alternative to brand-name drugs.

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