The Forever Fix gang: Corey Haas with book, surrounded by mom Nancy and dad Ethan Haas, Ricki Lewis on left next to Lori and Hannah Sames. At book signing 3/24/12, Barnes + Noble, Albany NY.
Glenn Nichols, surrounded by his hospice team. The author is in yellow.
May 24, 2012
Hannah Sames, here with her dog Ginger, awaits gene therapy. (photo: Dr. Wendy Josephs)
The pharmaceutical industry rightly calls the stage in drug development between basic research and clinical trials the “Valley of Death.” This is when a potential treatment that’s worked in mice, monkeys, and the like catapults to a phase 1 clinical trial to assess safety. It’s rare.
Francis Collins, MD, PhD, director of the National Institutes of Health, calls this period “where projects go to die.” The reason: $.
Matthew Herper writes in Forbes
that the cost of developing a new drug is $4-11 billion, not the $1 billion that Pharma often claims. Yet even that $1 billion is unimaginable, especially when you put a face on a rare disease and witness what the family goes through to leap to phase 1.
For me, that face belongs to 8-year-old Hannah Sames, of Rexford, New York. (more…)
May 20, 2012
Clinically relevant information in DNA hides in repeats, controls, and "junk" -- not just the protein-encoding exome.
10 Things Exome Sequencing Can’t Do – But Why It’s Still Powerful
Sequencing of the exome – the protein-encoding parts of all the genes – is beginning to dominate the genetics journals as well as headlines, thanks to its ability to diagnose the formerly undiagnosable.
Pulitzer Prize in Explanatory Reporting
honored the Milwaukee-Wisconsin Journal Sentinel’s coverage of a 4-year-old whose intestinal disorder was finally diagnosed after sequencing his exome. (more…)
May 13, 2012
3D CT scan of child with ACS. Lower jaw is small and malformed (left); same aged child with normal jaw (middle); lower jaw of child with ACS inverted over upper jaw of normal skull (right). (Credit: Image courtesy of Seattle Children’s).
Body-Altering Mutations – In Children and Flies
I became a science writer, circa 1980, because I didn’t think flies with legs growing out of their heads – my PhD research – had much to do with human health. So when I spied “A Human Homeotic Transformation”
way down on the Table of Contents in the May issue of the American Journal of Human Genetics, I was as riveted as a normal person would be getting a copy of People with a celebrity on the cover. (more…)
May 5, 2012
HIV on a human lymphocyte.
Buried somewhere beneath the long-awaited announcement of the birth of Jessica Simpson’s baby on May 2 was encouraging news about HIV infection: gene therapy appears to be safe.
Anti-retroviral drugs (ARTs) have been remarkably successful, but they require daily doses and have adverse effects. Gene transfer is an alternative approach that gives selected cells the genes to manufacture proteins necessary to counter a particular disease. Gene transfer (which technically becomes gene therapy once it works) to treat an enzyme deficiency, for example, provides genetic instructions for the missing enzyme. To treat an infection or cancer, gene therapy bolsters immune system cells.
May 2, 2012
I'm reposting this 2-year-old blog about the various bacterial outposts in the human body, in celebration of today's publication of the microbiome of a very special, intimate place. (I can say no more because it is embargoed until 2PM, when I will be traveling.)
Yesterday I committed a terrible crime. I walked away from a treadmill at the Y without scrubbing the handles.
“Ricki, get back here,” admonished the attendant as I headed for the elliptical. “You forgot to wipe down!”
“But I’m not sweating, and I never get sick. I won’t pass along (more…)
Book Club Reader's Guide
Many challenging questions to stimulate thought and discussion.
38 discussion questions to get students thinking and talking about gene therapy, including the science, ethical issues, and the drug approval process.
The Forever Fix is the uplifting true story of 8-year-old Corey Haas, who was cured of hereditary blindness just 4 days after gene therapy.
A spectacularly-illustrated, clearly written human anatomy and physiology textbook, used in pre-health profession programs throughout the U.S.
A highly engaging, clearly written, beautifully illustrated introduction to the science of human genetics for the non-scientist. Now in its 10th edition.
DNA reflects who we are -- but it isn’t the whole story.
A one-hour interview, mostly about why gene therapy has been beneath the radar.
Answers for the Family (http://answers4thefamilyblog.com) on www.LATalkRadio.com
The Art of Relating BlogTalkRadio.com with Christine Kniffen
7/9/12 hourlong discussion about rare diseases + The Forever Fix
MesotheliomaHelp.net recommends The Forever Fix
Voiceamerica radio show
Kathryn Vox radio show, including "The Forever Fix"
How "The Forever Fix: Gene Therapy and the Boy Who Saved It" Wrote Itself
WOCA The Source podcast, May 21, 2012
WOCA The Source radio show podcast about The Forever Fix, May 21, 2012
The Ann Parillo Schenectady Today Show, April 17, 2012
TV segment on "The Forever Fix: Gene Therapy and the Boy Who Saved It"
Are DNA Patents Doomed? Scientific American blog 4/3/12
A brief history of patenting genes, current controversial cases, and a look ahead
"The Roundtable" WAMC Albany, NPR station, March 15, 2012
Joe Donahue interviews Ricki Lewis about "The Forever Fix: Gene Therapy and the Boy Who Saved It"
New Hope for Gene Therapy: A Young Boy's Fight Against Blindness
A short video about Corey Haas's gene therapy -- the subject of my book, "The Forever Fix: The Boy Who Saved Gene Therapy"
Publishers Weekly starred review
first advance review for The Forever Fix
Corey Plays Little League
A wonderful video of the family at the center of The Forever Fix: Gene Therapy and the Boy Who Saved It
Article on leptin and Jeffrey Friedman in RPI alumni magazine
The Forever Fix: Gene Therapy and the Boy Who Saved It
Alden March Bioethics Institute blog
Alden March Bioethics Institute blog
Advocates for published authors since 1912
Authors Guild Directory
A compendium of member websites