- U.S. FDA Approves KALYDECO™ for Use in Eight Additional CF Mutations
- Congratulations to our 2014 Sacks for CF Scholarship Recipients!
- Sarah Rini, a BEF Exercise For Life Scholarship Recipient, Featured in Local Paper
- Cegedim Relationship Management Joins Boomer Esiason Foundation in Fight against Cystic Fibrosis
- AbbVie and Galapagos to Co-Develop CF Therapies
- Congratulations to our 2013 Scholarship Recipients!
- Howard Lutnick Talks 9/11 and BGC Charity Day with Boomer and Carton
- Scientists Pinpoint 105 Additional Genetic Errors that Cause CF
- Voting is Open for the AbbVie CF Scholarship
- CFurther: Planning for College and Beyond Webcast
- Cornerstone Therapeutics Inc. announced it is now actively marketing PERTZYE® in the US
- Bridgeport, WV Student Raises Funds for BEF
- Boomer and Mike Seidel of the Weather Channel talked NYC Heat and Run to Breathe
- President of Team Boomer, Michaela Seccareccia, discusses NYC marathon with the Wall Street Journal
- “Gunnar Esiason is Winning at the Game of Life”
- Boomer Esiason Continues his Fight Against CF
- Updates on Sarah Murnaghan and Pediatric Lung Transplant
- Jerry Cahill presented with the Jacoby Angel Award
- Boomer: Sarah’s Transplant is a miracle
- May is CF Awareness Month!
- Mamaroneck Flowers and BEF Team Up
- Gunnar Esiason: The Face Behind BEF
- Phase 2 Study of Kalydeco and VX-661 Shows Promising Increases in Lung Function
- One Year Post Transplant: Where is Jerry today?
- New Infection Prevention and Control Policy from the CF Foundation
- The CF Foundation Expresses Concern About Proposed Disability Benefit Changes
- 2012 Champions for Charity
- Jerry Cahill, 56-Year Old CF Survivor and Athlete, Featured on CBS’s Courage in Sports
- NHLBI has Launched a Program on Early Cystic Fibrosis Lung Disease
- Team Boomer’s First Year in the Bank of America Chicago Marathon was a Success
- CFF and Walgreens Team Up
- A Heroic Group of Women Triumphing over Cystic Fibrosis
- Congratulations to the 2012 2nd Quarter Recipients of the BEF Scholarship
- Archbishop John Hughes Council #481 to host a Disco Night BEF Fundraiser
- Boomer’s Still Battling Cystic Fibrosis
- Congratulations to the 2012 Recipients of the Scholarship of the Arts
- FDA Advisory Committee supports the use of Novartis inhalation powder, Tobramycin
- Congratulations to Courtney Robinson, 2012 Rimington Trophy Scholarship Winner
- Sabrina Smith Walker, a Club CF Participant, is In the News
- Congratulations Emily Nickels and Clayton Roll, 2012 Exercise for Life Scholarship Winners
- Congratulations to the 2012 Rosemary Quigley Scholarship Recipients!
- Gene Network Restores Cystic Fibrosis Protein Function
- Congratulations to Olivia Burke, the most recent recipient of the Bonnie Strangio Scholarship
- Additions have been made to the You Cannot Fail store!
- Vertex Receives European Approval for KALYDECO™
- Jerry Cahill featured in Everyday Health
- Team Boomer Website
- Boomer’s Cystic Fibrosis Run to Breathe
- 3 Months Post-Transplant, Jerry Cahill will Run to Breathe
- Barron helps kids with Cystic Fibrosis Climb the “Poor Man’s” Everest
- Final Results from Phase 2 Combination Study of Kalydeco and VX-809 Show Significant Improvements
- Legislation to Expedite Approval of Rare-Disease Drugs Moving Through Congress
- BEF Releases New Wind Sprint on Working Out While Traveling
- PTC Therapeutics Announces Promising Results from Phase 3 Trial of Ataluren
- BEF Announces ‘You Cannot Fail… You are the Hero of Your Own Story’ Scholarship for CF Students
- June Deadlines Near for Five CF Scholarships
- CFChef Announces New Recipe Contests for 2012
- A Letter From Boomer: Causes for Celebration
- New Podcast: Bobby Bebber on Overcoming All Obstacles with CF
- Progress Made on Cayston® Supply Challenges, CFF Reports
- Concentrated Saline Therapy Not Effective in Young Children with CF, Study Finds
- FDA Approves New Drug Application for Digestive Care’s PERTZYE
- Double-Lung Transplant Has ‘Humbled Me Dramatically,’ Cahill Says
- Online CF Quiz from Gilead Sciences Tests Knowledge, Supports BEF
- BEF Expands Amazon Storefront to Include More ‘You Cannot Fail’ Merchandise
- Second Annual NY Yankees Celebrity Softball Event to Benefit BEF
- Application Deadline for Scholarship of the Arts is May 16
- NYRR Opens Registration for 2012 Boomer’s CF Run to Breathe
- Vertex Reports Promising Interim Results for Phase 2 Trial of Kalydeco and VX-809
- Charity Navigator Awards BEF Second Consecutive Four-Star Rating
- ‘You Cannot Fail’ Book Now Available on Amazon.com
- Tickets on Sale Now for 2012 Refi Rock Spring Fling!
- Documentary on Eva Markvoort to Air May 3 on Oprah Winfrey Network
- New Facebook Tool Allows Members to Share Organ Donor Status
- Two Weeks Post-Transplant, Jerry Cahill Speaks Out on Organ Donation, CF Awareness Month
- ‘Donate Life Month’ Takes on New Meaning with Jerry Cahill Lung Transplant
- New Website Offers Insight on Cystic Fibrosis Gene Mutations
- New Wind Sprint: Club CF Is Valuable Online Resource for CF Community
- Boston College Club Lacrosse Teams Compete to Fight CF
- Maryland Football ‘Lift for Life’ Event to Support BEF and CF Community
- Atlantic Cup Sailing Race to Benefit BEF and CF Community
- April 17 Deadline Approaches for CFCareForward Scholarship
- Celebrate ‘Donate Life Month’ By Registering to Become an Organ Donor
- BEF Launches Online Shopping Cart Featuring ‘You Cannot Fail’ Merchandise
- New Podcast: Rick Lerz One Year After a Double-Lung Transplant
- Team Boomer Hits the Streets at NYC Half-Marathon
- Hoops and Hops: Check Out the Photos
- PMD Healthcare Giving Away Spiro PDs for World Asthma Day
- iTunes Offers App for CF Wind Sprint Video Series
- New CF Wind Sprint Looks at Transitioning from College to the Career World
- FDA Approves New Enzyme Therapy for People with CF
- New CF Wind Sprint Looks at Transitioning from High School to College
- Rimington Trophy Scholarship Established to Support Students with CF
- Gilead Sciences Working to Address Supply Shortage of Cayston
- PMD Healthcare Launches Personal Spirometer To Monitor Lung Function
- New CF Wind Sprint Looks at Transitioning from Pediatric to Adult CF Care
- BEF Awards ‘Sacks for CF’ Scholarships to Outstanding Students with Cystic Fibrosis
- Sales of Team Boomer ‘Vintage’ Tees to Benefit Fight Against CF
- FDA Approves KALYDECO™; Vertex to Begin Shipments Immediately
- Rimington Trophy Presented to ‘Unquestioned Leader’ of Michigan Football Team
- New Podcast: Colleen Caul’s ‘Run for Roses’
- Vertex Planning Additional KALYDECO™ Clinical Trials in 2012
- BEF Establishes Gunnar Esiason Scholarship Fund at Boston College
- Boomer Esiason to Co-Host ‘Live! with Kelly’ Show on January 11
- Rimington Trophy to be Presented to Michigan’s Molk on January 14
- Help Moganko Meet the Muppets and Boost CF Awareness
- What’s Your New Years Resolution? Want to Exercise More? Join Team Boomer!
- Sydney Esiason Discusses Being a CF Sibling
- 7th Annual Berkeley College Charity Car Show Raises More Than $25,000 for BEF
- Kalydeco (VX-770) Available to People with Critical Medical Need
- Mannitol Shown to Improve Lung Function When Added to Standard CF Therapy
- FDA Grants Priority Review for KALYDECO™ from Vertex Pharmaceuticals
- New CF Wind Sprint Demonstrates Exercises with Kettlebells
- Help Us Make a Difference in the Cystic Fibrosis Community
- YOU CANNOT FAIL Book and Merchandise Available Online, Just In Time for Holiday Gift Giving
- Michigan’s David Molk Named Recipient of 2011 Rimington Trophy
- New Podcast: Josh Mogren on Moving Forward After Losing a Sibling to CF
- New Clues for Improving Antibiotics for Tolerant Bacteria
- How CF-Related Diabetes Differs from Types 1 and 2
- Genzyme and CFFT Announce Collaboration to Discover New CF Drugs
- December 15 Deadline Approaching for BEF Academic Scholarship
- Boomer Esiason Foundation Unveils New Logo for Team Boomer Athletic Program
- Team Boomer Opens Registration for 2012 NYC Half-Marathon
- Abbott Announces Winners of ‘CFChef Challenge’ Recipe Contest
- Team Boomer Marathoners Raise More Than $500,000 to Support CF Community
- CBS New York Features Team Boomer in Marathon Coverage
- New York Times Article Features Team Boomer Marathoner
- BEF Launches YOU CANNOT FAIL Campaign with Sale of Inspirational Book by Jerry Cahill
- Boomer’s Team Triumphs in Fordham Basketball Scrimmage
- New England Journal of Medicine Features Study of Kalydeco (VX-770)
- Check Out the Photos from the 2011 Refi Rock Halloween Event
- Thirty Outstanding Students with CF to Receive ‘Sacks for CF’ Scholarship
- Vertex Applies to FDA for Priority Review, Approval of VX-770
- New Cystic Fibrosis Wind Sprint Demonstrates Reaching the Back with a Percussor
- Team Boomer Marathoner Qualifies for 2012 Olympic Trials
- Boomer Esiason to Host CF Education Event in Philadelphia
- NYRR Highlights Team Boomer Marathoner with CF
- Agile Sciences Gets NIH Grant to Study Treatment for Lung Infections
- FDA Requests Additional Study of Arikace®, Insmed Says
- New Book on Ironman Triathletes Highlights Athlete with CF
- New Podcast: Marc Smolowitz, Director of ‘The Power of Two’
- Fishing and Golf Event in Mexico to Benefit CF, MS
- Nick News Special Focuses on Organ Donation, Features Teen with CF
- Tickets on Sale for Annual Refi Rock Halloween Party
- Troupe with CF Dancer Finishes in Second Place on America’s Got Talent
- CF Chef Recipe Contest Deadline is September 28
- Boomer and Carton Host Special Guests at BGC Charity Day Broadcast
- New Podcast Features Jerry Cahill on Getting Involved in the CF Community
- Boomer Esiason Remembers 9/11 in NFL Video Interview
- Dancer With CF Part of Troupe in Finals of 'America's Got Talent'
- Sales of Vineyard Vines Tie and Tote Bag Benefit BEF
- BEF Board Member Timothy O’Brien Remembered as 9/11 Anniversary Nears
- 2011 Exercise for Life Scholarship Recipients Named by Boomer Esiason Foundation
- 'Power Of Two' Movie Hits Oscar Qualifying Circuit; Documentary Features Sisters With CF
- Team Boomer Issues Last Call for Guaranteed Spots in the 2011 ING NYC Marathon
- Save the Date: 2012 Boomer's CF Run to Breathe
- Pulmatrix Invests in Clinical Programs in Cystic Fibrosis and COPD
- BEF Releases Video Recapping Boomer's CF Run to Breathe
- 2011 CF Scholarship Recipients Named by Boomer Esiason Foundation
- Insmed Announces Clinical Hold on ARIKACE® Phase 3 Clinical Trials
- New York Yankees Present BEF with Proceeds from Softball Event
- New CF Wind Sprint Demonstrates Simple Exercises for the Legs
- CF Foundation Kicks Off 'Make Every Breath Count' Campaign
- National Minority Donor Awareness Day Marks Fifteenth Anniversary on August 1
- Teen with CF Educates Multicultural Populations About Organ Donation and Coping with Serious Illness
- Vertex Plans to Submit New Drug Application for VX-770 to FDA in October
- Most Parents Unaware of Medical Research Opportunities for Their Kids
- Changes in Lung Stem Cells May Contribute to CF Progression
- Electromed Awards Jerry Cahill First Annual Distinguished Service Award
- New Podcast Discusses Organizing CF Medical Information and Being Your Own Patient Advocate
- Battle of the Bugs: Pseudomonas Breaches Cell Wall of Rival Bacteria Without Hurting Itself
- Bacteria Use Batman-Like Grappling Hooks to 'Slingshot' on Surfaces, Researchers Find
- CF Chef Recipe Contest and Online Nutrition Program Launched by Abbott
- New Invasive Diagnostic Procedure Seems to Be No Advantage Over Current Standard Procedure
- FDA Approves 25,000 Lipase-Unit Strength of ZENPEP®
- Results and Photos from the CF Run to Breathe
- Thousands Participate in CF Run to Breathe
- BEF President Dave Rimington Featured on ESPN.com
- New Podcast Discusses Being a CF Parent
- The Summer's Hottest Party: Boomer and Carton on Fire Island
- Civilian Military Combine to Benefit BEF
- New CF Wind Sprint Demonstrates Simple Upper Body Exercises
- NY Yankees Fundraiser: Check Out the Photos!
- Researchers Discover New Airway Stem Cell
- Esiason Hosts Charity Softball Games at Yankees Stadium
- New Report Helps Adolescents Transition to Adult Care
- Empire Challenge Photos Available Online
- Refi Rock Spring Fling ROCKS Statue of Liberty
- Support Gunnar Esiason and Jerry Cahill in the Run to Breathe
- LI Bests NYC in 16th Empire Challenge All-Star Game
- Irish Woman Receives Groundbreaking Lung Transplant
- Signaling Pathway Is 'Executive Software' of Airway Stem Cells, Researchers Find
- Changing Genetic ‘Red Light’ to Green Holds Promise for Treating Disease
- FDA Approves Infant Dosage for CREON
- Rare Diseases Often Caused by Mutations in Essential Genes, Researchers Find
- Vertex to Expand Access to VX-770 for Patients with Critical Medical Need
- VX-770 Shows Positive and Lasting Results Throughout 48-Week Phase 3 Study
- Phase 2 Study of VX-770 and VX-809 Show Promising Results in Patients with Most Common Mutation
- Aradigm Gets FDA Orphan Drug Designation for Ciprofloxacin for Inhalation in Bronchiectasis
- HHS Reduces Insurance Premiums for Pre-Existing Condition Plan
- Jerry Cahill Inducted into CHSAA Hall of Fame
- NY Yankees Celebrity Softball Event to Benefit BEF
- Exercise: A CF Photo Essay
- Drug Shortages Force Hospitals to Hunt for Substitutes
- CF Bacteria Could Help Combat Other Superbugs, Researchers Find
- Boomer and Carton to Guest Judge on 'Iron Chef America'
- UnitedHealthcare to Present Annual Empire Challenge
- New CF Wind Sprint Demonstrates How to Use the PowerLung
- WCBS-TV to Air PSA on CF Run to Breathe
- Researchers Identify Genes Linked to Worsening of CF
- U.S. News Releases 2011-12 Rankings of Best Children's Hospitals
- Abbott Test Assists in CF Screening
- New Podcast Discusses Life Before and After a Double-Lung Transplant
- Run for a Cause: Set - and Achieve - an Exercise Goal
- What's Your CF IQ?
- Toronto Student Finds CF Treatment by 'Hacking' Supercomputer
- Jerry Cahill Awarded Ellis Island Medal of Honor
- New Podcast Focuses on Adulthood With CF
- WBUR Special Report on VX-770
- New CF Wind Sprint Demonstrates Cardio Exercises
- Simple Exercise Improves Lung Function in Children with Cystic Fibrosis
- NYRR Opens Registration for Boomer's Cystic Fibrosis Run to Breathe
- CF Awareness Month 2011: The Power of Exercise
- New CF Wind Sprint on Stretching
- Drug Combo for CF Lung Infections Could Address Antibiotic Resistance, Researchers Say
- FDA Rejects Liprotamase, Calls for More Studies
- Boomer Urges Donor Registrations in New York State Campaign
- New Podcast on Getting Motivated Before and After a Lung Transplant
- CF Patients Fight Grim Prospect with Bright Outlook
- CFF Expands Vertex Partnership
- New Molecular Structures Could Fight Infectious Diseases Better than Conventional Antibiotics
- Clinical Trials Legislation Takes Effect
- Pittsburgh Hospital Leads in Lung Transplants
- Vertex Press Release on VX-770 Phase 3 Clinical Trial
- VX-770 Trial: 'Profound Improvement' in Kids
- New Wind Sprint on Core Exercises
- Pig Model Of CF Improves Understanding Of Disease
- Researchers Explore 4-D Imaging of CF Lungs
- BEF Scholarship Apps Due 3/15
- FDA Panel Not Sold on Personal Genetic Testing
- New Team Boomer Video: Why Do You Run?
- VX-770 Achieves Positive Results in Phase 3 Clinical Trial, Vertex Announces
- Axcan Pharma CEO Explains Impact of Axcan-Eurand Merger
- Phase 3 Trial of Ataluren Expected to be Completed in 2011
- Denufosol Phase 3 Trial Published in Leading Medical Journal
- TCU Senior Wins 2010 Rimington Trophy
- Samsung, Sears and BEF Team Up to Tackle Cystic Fibrosis
- New England Journal of Medicine Publishes Phase 2 Study of VX-770 for Treating Cause of CF
- Phase 2 Results Show Ataluren Restores Production of Functional CFTR Protein
- Cystic Fibrosis Gene Typo is a Double Whammy
- Growth Defects in CF May Start Before Birth, Study Finds
- Discovery Labs KL4 Surfactant Granted Orphan Drug Designation for the Treatment of CF
- Pharmaxis Announces Positive Combined Phase 3 CF Trial Results
- Gilead's Cayston Beats Tobramycin Inhalation Solution in Six-Month Head-to-Head Study
- Analysis Shows TOBI Associated With Reduced Mortality in CF Patients With Common Lung Infection
- Vertex Pharmaceuticals Clinical Trial to Evaluate Combo of VX-770 and VX-809 Targeting CFTR Protein
- Impaired Metabolism in CF Lung Epithelial Cells Revealed by Metabolic Analysis
- Bacteria Identified as New Foe for CF Patients
- Human Growth Hormone Shows Promise in Treating Cystic Fibrosis Symptoms
- University of Texas Researcher Receives $2.1 Million Grant to Focus on Drug Delivery to Lungs
- 'Improving Access to Clinical Trials Act' Passes U.S. House; Heads to President for Signatur
- PTC Therapeutics Awarded $1.6 Million FDA Grant to Support Ongoing Phase 3 Study of Ataluren
- iTunes Offers New App for Jerry Cahill's CF Podcasts
- Researchers: Vitamin D May Treat, Prevent Allergic Reaction to Mold in CF Patients
- Luminex Announces Commercial Launch of New CF Genetic Screening Test
- BEF Awards $10,000 Scholarships to Two Student-Athletes
- Out of Grief Sprouts a Life-Saving Legacy
- 'Improving Access to Clinical Trials Act' Passes U.S. Senate in Victory for CF Advocates
- New CF Drugs Bring Hope; More Survive Into Adulthood, But They Face New Challenges
- Eurand Reports Further Positive Data from Pancreatic Insufficiency Drug Study
- Cincinnati Children's Cited in US News Report As a 'Best' Hospital for CF Kids
- Limit Antibiotics for CF Exacerbations to 10 Days, Registry Study Says
- Lilly to Acquire Alnara; Company's Liprotamase Currently Under FDA Review
- New Study Examines Impact of MRSA on CF
- Researchers Still Expect Genetic Mapping Project Will Lead to New Drugs
- FDA Extends Availability of Pancreatic Enzyme Therapy Pancrease Until Mid-July
- Pancreatic Enzyme Therapy Pancrease to Have Limited Availability, CFF Says
- Data Suggest Denufosol May Hold Promise as an Early Intervention Treatment for CF
- Mpex Presents Positive Phase 2 Clinical Trial Results of Aeroquin Treatment in CF
- Aradigm Receives FDA Clearance for Clinical Trial of Inhaled Liposomal Ciprofloxacin
- Azithromycin Doesn't Always Help Lungs in CF Patients Without P. Aeruginosa, Study Finds
Researchers Still Expect Genetic Mapping Project Will Lead to New Drugs
NORTH WALES, PENN. -- At Merck's ''automated biotechnology facility'' here, robot arms adapted from automobile factories deftly shuttle plates containing human cells.
Assisted by the robots and other complex machinery, scientists are studying what happens to the cells as each of the roughly 22,000 human genes is turned off. They hope to find the genes involved in different diseases, the starting point for creating a drug.
It is a merger of sophisticated biology and brute force made possible only because the Human Genome Project provided the identity of all the human genes. But as with so much else that has spun off from the genome project, it is also an expensive gamble, with success far from assured.
''Can I point to a single drug right now that this has facilitated?'' said Michele Cleary, Merck's senior director for automated biotechnology. ''No, because we are in the early stages of this. There's information feeding into the early stages of the pipeline that we'll see the fruits of in years to come.''
Ten years after President Bill Clinton announced the completion of the first draft of the Human Genome Project, in June 2000, its application to drug development is still, at best, a work in progress. But while many genetics scientists outside the drug industry say the project has had few medical benefits, industry researchers urge a wait-and-see patience.
Some of the first drugs based on genomic studies are starting to reach the market. Several new ''targeted'' cancer drugs, for instance, block the effects of genetic abnormalities that spur tumor growth. A drug from the biotech company Amgen, an osteoporosis treatment called Prolia, was approved two weeks ago. The company got its initial clue for the drug by making different genes in mice overactive. Mice with one particular overactive gene had unusually thick bones.
And Human Genome Sciences, a drug company founded in 1992 as the genome project was just getting under way, applied last week for approval of Benlysta, which could be the first new drug in decades for treating lupus.
That is nothing like the cornucopia of new drugs that some experts predicted the genome project would yield. A decade ago, drug companies spent billions of dollars equipping themselves to harness the newly revealed secrets of human biology. Investors bid the stocks of tiny genomic-companies to stratospheric heights.
That ''genome bubble'' has long since popped. And not only has there been no pharmacopeia, but some experts say the Human Genome Project might have at least temporarily bogged down the drug industry with information overload.
As the head of Novartis's pharmaceutical business lamented in 2000, ''Data, data everywhere, and not a drug, I think.''
Indeed, even though research and development spending by major pharmaceutical companies has roughly doubled in the decade since the genome project was largely completed, reaching $46 billion last year, the number of new drugs approved in the United States each year has stayed about the same. There were 25 in 2009.
Genomics is not the only reason for the decline in the bang from the research buck. A big factor has been stiffer testing requirements by the U.S. Food and Drug Administration.
For all that, drug industry executives say it is simply a matter of time before the Human Genome Project pays off. They note that because it can take 15 years or more to go from a basic discovery to a marketed drug, it is too early to expect many drug approvals yet.
Moreover, pharmaceutical executives say that even if there are not many drugs yet, the genome project has transformed the way research is done.
''It's become a very standard part of what we do,'' said Peter S. Kim, the executive vice president in charge of research at Merck.
The company spent $1.1 billion in 2006 to acquire Sirna Therapeutics, which is developing the RNA interference technology that Merck uses to turn off genes. Dr. Kim said that having the genome sequence gave scientists ''the ability to do science at a different level.''
At Bristol-Myers Squibb, two-thirds of the drugs being developed have been ''touched'' in some way by genomics, said Elliott Sigal, president of research and development.
At Genentech, a third of the drugs in clinical trials and two-thirds of the newer compounds earlier in development ''have been enabled in a significant way'' by the genome project, said Marc Tessier-Lavigne, executive vice president for research at Genentech, a unit of Roche.
One of those ways, he and others said, is allowing the potential side effects of a drug to be detected earlier. Often, drugs meant to inhibit one protein in the body also inhibit other fairly similar proteins, causing unintended effects. Knowing the genes can enable companies to find these similar proteins in advance and make sure their drug interacts with only the intended target.
''By having the genome sequence you can make better drugs, more specific drugs,'' Dr. Tessier-Lavigne said. ''In the old days you would only discover the side effects much later in the process and the drug would die.''
Many drug companies collect and analyze the DNA of patients in clinical trials, hoping to find genetic signatures that will allow drugs to be better tailored to specific patients. It was recently discovered, for example, that patients with a certain variation in a gene called CYP-2C19 did not respond well to Plavix, a widely used anti-clotting drug, leaving them at a higher risk of having a heart attack.
Still, some executives concede that the genome project has not lived up to expectations, and in some ways might have even made life more difficult for drug companies.
''I don't think any of us in the business believed it would be a cornucopia,'' said Frank L. Douglas, the former head of research and development at the drug company Aventis. ''What we did believe, however, was that it would get easier. We forgot our history.''
The history is that identifying a gene involved in a disease is a long way from having a drug. The gene for <strong>cystic fibrosis</strong>, for instance, was discovered in the pregenomics age, 1989, by studying families with that condition. Today, 21 years later, there is still no drug on the market resulting from that discovery, although two drugs from Vertex Pharmaceuticals and one from PTC Therapeutics are in clinical trials.
Finding a gene is merely the first step in a long process to develop a drug. Genes are the recipes the body uses to make proteins, and most drugs work by inhibiting or promoting the activity of a particular protein, which is known as the drug's target.
The initial attraction of genomics was the assumption that knowing all the genes would lead to the discovery of thousands of new targets. And to some extent that has happened.
But compared with the past, when targets tended to be discovered by academic scientists already studying a disease and its genetic context, the genome project provided companies with thousands of potential new targets all at once. Targets discovered this way, without years of academic research behind them, can require companies to spend years to understand the targets' role in disease.
''Putting the players on the stage does not tell you what they do,'' said Stephen H. Friend, president of Sage Bionetworks, a nonprofit organization studying the genetic mechanisms of disease.
Even after a target is understood, companies must then create a drug to interact with it. Some targets, because of their structure and location in the body, do not lend themselves to this. In industry parlance, the targets are ''undruggable.''
And when a drug is created, it must be tested for safety and efficacy, first in animals and then in lengthy clinical trials with humans. These elements of drug creation and testing have not been greatly accelerated by genomics.
''If on the first day we had discovered a new molecular target, it's still going to take 15 to 20 years to make the drug,'' said Robert R. Ruffolo Jr., who ran research and development at Wyeth until 2008. ''Genomics did not speed up drug development. It gave us more rapid access to new molecular targets.''
Source: International Herald Tribune