How DNA Sequencing Is Transforming the Hunt for New Drugs

DNA Sequenced – For Development of Medicines

Manufacturers of drug have commenced amassing hugetroves of human DNA, hoping to shorten the time it would take in identifying new drug candidates which according to some could be a way of transforming the development of medicines. These attempts would help researchers in recognizing rare genetic mutation through scanning of large databases of volunteers who tend to agree in having their DNA sequenced, providing access to thorough medical records.

This is made possible by the lower cost of genetic sequencing which took the government funded scientist around $3 billion and 13 years in sequencing the first human genome by 2003. The cost was nearly $1,500 per genome as of last year which is down from $20,000 five years back.A deal signed by Regeneron Pharmaceuticals Inc. with Pennyslvania’s Geisinger Health System in January 2014 to sequence partial genomes of around 250,000 volunteers, is laying claims to the discoveries based on the new approach.

Company executives have informed Reuters that they utilised data from the first 35,000 volunteers to confirm the promise of 250 genes on the target list for drugs focused at common medication conditions which comprise of high level of cholesterol and triglycerides.

Regeneron Recognized New Gene Targets – Obesity

Regeneron informs that it has also recognized several dozen new gene targets which include a novel gene that plays a role in obesity. Biogen Inc., Pfizer Inc. and Roche Holding AG are working on identical projects which use DNA as well as patient health data to discover new drug targets or predict the effects of drugs.

These have been inspired by earlier successes in cancer with drugs like Pfizer’s lung cancer treatment Xalkori that gained approval in 2011 and targets mutation in tumours eliminating the disease. Recently, Vertex Pharmaceutical changed the treatment of cystic fibrosis with Kalydeco that targeted the diseases’ primary genetic cause.

 Earlier, the discovery of such genes was a difficult procedure which involved several years of research into isolated population. For instance, in 1991, researchers discovered a rare mutation in a gene known as Angpt13 which instigated low levels of artery-clogging cholesterol and triglycerides amid families in the remote Italian village of Campodimele. This took additional two decades together with several groups of scientist to thoroughly comprehend the potential cardiovascular benefits connected to mutations in that gene.

First Step – Recognizing Target Genes

The Regeneron Genetics Centre, since last autumn has sequenced the DNA of over 35,000 Geisinger patients and is now on the track in sequencing 100.000 by the end of this year. The company has already identified 100 people with similar cholesterol affecting mutations to the ones first observed in Compodimele and other areas.

Dr George Yancopoulos, chief scientific officer of Regeneron comments that `one would no longer have to find that one rare family in Italy because it is in the database’.Recognizing target genes is a first step but does not guarantee that a drug could be developed on a genetic lead or if it could be effective and safe enough to be used. Regeneron is sequencing exomes which is a protein making genes comprising of 1 to 2 percent of the genome, a search which costs around $700 for each person.

Others prefer looking at the whole genome costing around $1,500 per person. One of the first scientists, Craig Venter, to sequence the human genome is of the belief that the whole genome approach would be meaningful over a period of time.

He has stated that he would rather have a gold mine with a deep vein of gold and modern industrial equipment to mind it rather than sit there with a pan in a stream looking for gold. Both would find gold and it’s a question of how much you find.

Anticipating Winning Regulatory Approval for Cholesterol Lowering Drug

Regeneron is pushing for the Geisinger database in becoming a cornerstone of that attempt and intends creating a consortium of drug-makers to finance it. In response, Yancopoulos stated that the company hopes to regainsome of its investment. Regeneron has been identified by Dr Francis Collins, director of the National Institutes of Health, in charge of the precision medicine project, among a short list of potential contributors to the one million strong DNA study.

Others include Kaiser Permanente, Mayo Clinic and the Marshfield Clinic in Wisconsin and a decision is anticipated by early fall. Attraction of big payoff is strong for drug companies. Regeneron together with its partner Sanofi are looking forward in winning regulatory approval for a cholesterol lowering drug which works in blocking the PCSK9 gene. Pfizer and Amgen have also developed identical treatments.

Those born with non-functioning forms of PCSK9 tend to have very low cholesterol and the new drugs have the same effect and are considered poster child for treatment. This tends to take advantage of faults in the genome that could prove beneficial to the rare individuals carrying them. Wall Street analysts project that by 2019, Regeneron/Sanofi’s PCSK9 drug would be generating revenues of around $4.4 billion.

Regeneron has also partnered with academic centres in studying families having extreme genetic disorders that have led to the discovery of various new candidate genes according to Dr Aris Baras running the Regeneron’s genetic centre.