As the year comes to a close, it's traditional to reflect on the happenings of the past year and consider the possibilities of the coming one. Here's an overview of developments and what's in store for six areas of biotech:
A Genetically Modified Animal Is Coming to Dinner
It looks like the first genetically modified (GM) animal will be approved as a food source in 2013. AquaAdvantage Salmon, the GM salmon developed by Canadian AquaBounty Industries has been languishing in FDA limbo although the FDA concluded the fish was safe for human consumption in September 2010. Since then, it was waiting on an environmental assessment, which it turns out was completed in April of 2012 but was held up for what appears to be political reasons. The FDA finally took action on December 26 to open up public comment on this final hurdle for approval. The fish should be approved in the first quarter of 2013.
For more background on GMO food, see Genetically-Modified-Food-How-Did-We-Get-Here
Gene Therapy Becomes Just Another Drug
Back in July, the European Medicines Agency recommended for the first time approval of a gene therapy drug. Glybera, made by Dutch company uniQure, was formally approved in November and should be available for treatment of a rare genetic deficiency of the lipoprotein lipase (LPL) gene.
Although China and Russia have approved a few gene therapy treatments for cancer and cardiovascular disease, their efficacy is not as well established. Glybera is the first gene therapy approval in an independently regulated healthcare market.
While uniQure has applied for Food and Drug Adminstration (FDA) approval for Glybera, there are a couple other gene therapy treatments that are working through both the US and European approval processes. For example, treatments for immune deficiency ADA-SCID ("bubble boy" syndrome), inherited blood disorder beta-thalassemia, and Leber's congenital amaurosis, an inherited eye disease leading to blindness. In 2013, the FDA should approve its first gene therapy treatment and the European Medicines Agency approve additional applications of this unique genetic approach to treat diseases.
For more details on the progress in gene therapy, check out Gene Therapy Is Finally Becoming a Medical Reality.
The Supremes May Finally Decide if Companies Can Own Genes
In August, a federal appeals court upheld its previous decision that the company Myriad had a valid patent on the isolated BRCA1 and BRCA2 genes that contain mutations linked to about 10% of breast cancers. Approximately, 60% of women that have one of these mutations develop breast cancer. As a result of the ruling, Myriad owns the technology to test for these mutations and patient samples tested for these gene variants needs to be sent to Myriad's labs in Salt Lake city.
The case originally started in 2009 when the ACLU sued Myriad over its enforcement of BRCA patents. Just this past month, the Supreme Court agreed to review the gene patent issue involved in lawsuit. Basically, it looks as though they will decide whether companies can patent human genes or not.
The current review occurs as sort of a follow up to their ruling in April 2012 against Prometheus Labs patents on a blood test that assesses a patient's ability to metabolize the drug thiopurine. The ruling in that case suggested that the Court believes that merely correlating certain gene variations with physiological conditions does not qualify as an invention, just an observation, and so, is not patentable.
Following its decision in the Prometheus case, the Supreme Court instructed the federal appeals court to review its original decision upholding the Myriad BRCA tests. As mentioned above, the appeals court ruled again in favor of Myriad in August. However, the ruling seems in conflict with the Prometheus ruling. This may be the reason the Supreme Court has decided to consider the gene patent issue in the Myriad dispute—to clarify the situation and finally settle the question of whether a company can hold a patent on a gene.
In the spirit of a New Year forecast, I'll make a prediction that the Court will rule that a gene sequence is not patentable. Of course, my training is in biochemistry, not jurisprudence, so this is not a well thought out legal assessment. The prediction reflects more my feeling that the information content of DNA sequences should be in the public domain. I am in agreement with the American Medical Association, Association of Human Genetics, and Dr. James Watson, co-discoverer of the DNA structure. However, frankly, the idea of patenting genes is a bit of a misnomer as pointed out by the Biotechnology Industry Organization, and there are certainly important commercial impacts, as this organization describes, if the Court's decision is not sufficiently nuanced to protect investment in biotech R&D.
For more information on this case, check out Genomic's Law Reports.
Slow Progress in Slowing Alzheimer’s Progression
It's been a disappointing year for new Alzheimer's treatments with the failure of phase 3 trial drugs from both Pfizer and Eli Lilly to significantly affect progression of the disease. These drugs targeted beta-amyloid plaques which build up in the brain of Alzheimer's patients. Merck is starting trials with another beta-amyloid targeting drug. While some are skeptical whether this approach can be effective, others see progress in these results as it might suggest approaches need to target beta-amyloid formation earlier, before plaques begin. For instance, early stage trials of a vaccine targeting beta-amyloid showed some promise.
While Alzheimer's therapies saw little progress, advances were made in earlier diagnosis of Alzheimer's. In April 2012, a positron emission tomography (PET) test using a new Eli Lilly's new chemical probe to detect amyloid plaque formation in patients was approved. Also, researchers at Emory in 2012 and the previous year at McGill University identified several blood markers for Alzheimer's. ScienceDaily noted a number of other recent findings that may provide tools for the early detection of Alzheimer's.
We will likely see more advances in the detection Alzheimer's disease in 2013, and possibly even a diagnostic blood test. While there still may be little that can be done in terms of treatment, hopefully, better diagnosis will expedite treatment breakthroughs for the disease in the near future.
Stem Cells Continues Their Advance
In 2012, we saw Drs. Gurdon and Yamanaka receive the Nobel Prize in Medicine for their work on reprogramming normal body cells to make stem cells, known as induced pluripotent stem cells (iPSC). Promising stem cell research has been moving forward too, such as the study working out how to use stem cells to regenerate neurons, and the recently published work using iPSC to correct amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, in mice. Also, preliminary human trials, such as UCSF and StemCells, Inc. collaboration treating 4 children affected with a genetic neural degenerative disease, and another trial correcting spinal injury are showing real promise.
As a result of this progress, it might be reasonable to expect big advances in the stem cell field in 2013. The title of a recent article in the Atlantic, 2013: Year of the Stem Cell, seems to suggest this but actually concludes on a more realistic assessment, stating, "2013 probably won't be the year that the big miracles anticipated from stem cell research will come to pass. But it does promise to be one full of more small, but significant advances toward that future goal." This is my prediction also.
Stem cell research as been gaining some significant momentum and, in 2013, I expect to see promising new results that will likely lead to therapeutic trials in subsequent years. For a perspective on the potential, listen to UC San Diego's Dr. Larry Goldstein comments on the California Institute of Regenerative Medicine's website. As he notes, "If the public continues to adequately fund research with stem cells we will see breakthroughs that are absolutely unexpected and that will change the way that we deliver medicine."
Also, you might take a look at The Range of Stem Cell Technology and Experts Assess the Current Status of Stem Cell Research for a general overview of Stem Cell technology.
The Complexities of Cancer Become Clearer
Of course, there will be advances in cancer research. While difficult to predict, genomic research is leading to a much more sophisticated understanding the different mechanisms regulating progression of this disease, such as the work done by the Buck Institute and University of Santa Cruz in characterizing breast cancer.
Actually, though, diseases is a more accurate term to use with cancer, since it is not just one disease process. Cancer is really a general term to describe a loss of cell regulatory control that produces multiple types of abnormalities that lead to uncontrolled cell growth—a tumor. It seems clear that there will never be a single cure for all cancers. However, while it is difficult to predict any specific advances with such a complex disease, advanced DNA analysis of individual tumors appears on the verge of yielding real breakthroughs in the understanding and treatment of specific cancers in the next several years.
For an glimpse of the abnormalities that produce cancer cells, take a look at The Biological Hallmarks of Cancer Cells and for the sort of approaches researchers are taking to beat cancer, see Seven Areas of Focus for Research on New Cancer Therapies.