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. This article lists 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 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 until 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.