Congress wants a single ALS patient to get a therapy never tested in humans. A family in Iowa believes the Food and Drug Administration will decide whether their only surviving daughter lives or dies, and they’ve been on a monthslong crusade to break through its bureaucracy. And they’re succeeding
Congress wants a single ALS patient to get a therapy never tested in humans. A family in Iowa believes the Food and Drug Administration will decide whether their only surviving daughter lives or dies, and they’ve been on a monthslong crusade to break through its bureaucracy. And they’re succeeding
WASHINGTON — A family in Iowa believes the Food and Drug Administration will decide whether their only surviving daughter lives or dies, and they’ve been on a monthslong crusade to break through its bureaucracy. And they’re succeeding.
Just last week, the FDA gave Jaci Hermstad, a 25-year old Iowan who is dying from a rare form of ALS, an early sign that she will receive the first dose of an experimental drug never before tested in humans. The FDA’s move, which was confirmed to STAT by Jaci’s family and doctor, is a breakthrough for the Hermstads.
For months, the family has circulated petitions, tweeted at President Trump, and called on some of Washington’s most controversial and powerful politicians — including the speaker of the House and a Republican under fire for racist comments — all to convince the FDA to waive even the most basic of studies before the drug is injected into Jaci’s already fragile immune system.
A panel of government-appointed experts in Germany agreed unanimously that the human germline “is not inviolable,” rejecting one objection to using genome editing technologies such as CRISPR to make heritable changes in the DNA of human embryos, sperm, or eggs
A panel of government-appointed experts in Germany agreed unanimously that the human germline “is not inviolable,” rejecting one objection to using genome editing technologies such as CRISPR to make heritable changes in the DNA of human embryos, sperm, or eggs
A panel of government-appointed experts in Germany agreed unanimously that the human germline — DNA that is inherited by children from their parents — “is not inviolable,” rejecting one objection to using genome editing technologies such as CRISPR to make heritable changes in the DNA of human embryos, sperm, or eggs.
In a 47-page report made public on Monday, the independent German Ethics Council concluded that the power of CRISPR, and the announcement last November that a scientist in China had used it to edit two IVF embryos that resulted in the birth of twin girls, means that “the possibility of intervening more easily and precisely in the human germline is drawing closer and closer.”
Although the council’s 26 ethicists, legal scholars, scientists, and other experts agreed unanimously that there are no compelling philosophical arguments against altering human germlines, they also concluded that it is ethically irresponsible to do so now.
“2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense,” says Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley
“2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense,” says Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley
But scientists have long hoped CRISPR — a technology that allows scientists to make very precise modifications to DNA — could eventually help cure many diseases. And now scientists are taking tangible first steps to make that dream a reality.
For example, NPR has learned that a U.S. CRISPR study that had been approved for cancer at the University of Pennsylvania in Philadelphia has finally started. A university spokesman on Monday confirmed for the first time that two patients had been treated using CRISPR.
Image: By OpenStax College – Anatomy & Physiology, Connexions Web site. http://cnx.org/content/col11496/1.6/, Jun 19, 2013., CC BY 3.0, https://commons.wikimedia.org/w/index.php?curid=30148180
“It’s easy to get on your high horse when you’re not in our position,” she said. “If editing an IVF embryo is the best option to mitigate the pain that a child would otherwise suffer, then give us the choice.”
“It’s easy to get on your high horse when you’re not in our position,” she said. “If editing an IVF embryo is the best option to mitigate the pain that a child would otherwise suffer, then give us the choice.”
Neena Nizar’s earliest memory is of her father tying her to an ironing board. His beloved toddler, who seemed fine when she was born, had something very, very wrong with her: Neena’s bones bent and curved and she wasn’t growing normally, so his engineer’s mind desperately seized on the ironing-board solution.
But the problem — which some doctors diagnosed as polio and others as rickets or “we have no idea” — was even worse than bones that wouldn’t stay straight. They also broke down faster than they grew, with weak cartilage where strong bone should be. By the time other little girls were skipping and running and kicking balls, she was in pain and could barely get around. “I had to be carried into school, and I had rods in my hips and metal clamps to hold my bones in place,” said Nizar, who was born in Dubai. “Growing up, that was beyond hard. It was horrible.”
So when Nizar, now 41 and living in Nebraska, hears scientists’ emphatic calls to prohibit “embryo editing” of disease-causing genes, her reaction is shaped by decades of her own suffering — compounded by that of the two sons who inherited her devastating mutation.
Watch now: The NAM & CSIS Commission on Strengthening America’s Health Security hosted a conversation on whether human germline genome editing should be permitted, the types of applications which might be appropriate, the standards and criteria that should be followed, and what regulatory or governance framework is needed. Panel includes our Jeffrey Kahn
Watch now: The NAM & CSIS Commission on Strengthening America’s Health Security hosted a conversation on whether human germline genome editing should be permitted, the types of applications which might be appropriate, the standards and criteria that should be followed, and what regulatory or governance framework is needed. Panel includes our Jeffrey Kahn
On Wednesday, March 27, 2:00-3:30 pm, the National Academy of Medicine and the CSIS Commission on Strengthening America’s Health Security hosted a conversation on the unfolding debate as to whether human germline genome editing should be permitted, the types of applications which might be appropriate, the standards and criteria that should be followed, and what regulatory or governance framework is needed.
Featuring:
Dr. Victor Dzau
President, National Academy of Medicine
Tim Hunt
Senior Vice President, Corporate Affairs, Editas Medicine
Jeffrey Kahn
Andreas C. Dracopoulos Director, Johns Hopkins Berman Institute of Bioethics
Anne-Marie Mazza
Senior Director, Committee on Science, Technology, and Law, The National Academies of Sciences, Engineering, and Medicine
Moderated by
J. Stephen Morrison
Senior Vice President and Director, CSIS Global Health Policy Center
The 18-member committee “will examine the scientific, ethical, social and legal challenges associated with human genome editing,” according to the WHO announcement
The 18-member committee “will examine the scientific, ethical, social and legal challenges associated with human genome editing,” according to the WHO announcement
The World Health Organization Thursday announced the formation of an international committee aimed at establishing uniform guidelines for editing human DNA in ways that can be passed down to future generations.
The 18-member committee “will examine the scientific, ethical, social and legal challenges associated with human genome editing,” according to the WHO announcement.
“The aim will be to advise and make recommendations on appropriate governance mechanisms for human genome editing,” the WHO says.
Real progress is being made using CRISPR and related gene-editing techniques to alter cells taken from people with diseases such as sickle cell and genetic forms of blindness. These approaches are likely to offer safer and more reliable ways of making changes to a person at the genetic level. What makes these uses much less ethically challenging [than germline editing] is that they only affect the person being treated; the genetic modifications can’t be passed on to future generations.
In last year’s action film “Rampage,” which featured Dwayne “The Rock” Johnson, CRISPR gene-editing technology turns a gorilla, a wolf, and a crocodile into monsters the size of skyscrapers that destroy Chicago.
In last year’s action film “Rampage,” which featured Dwayne “The Rock” Johnson, CRISPR gene-editing technology turns a gorilla, a wolf, and a crocodile into monsters the size of skyscrapers that destroy Chicago.
In last year’s action film “Rampage,” which featured Dwayne “The Rock” Johnson, CRISPR gene-editing technology turns a gorilla, a wolf, and a crocodile into monsters the size of skyscrapers that destroy Chicago.
If you missed the movie, you may have caught wind of He Jiankui. Near the end of 2018, the Chinese scientist reported having used CRISPR to produce the first-ever gene-edited babies. (He currently faces criminal charges in his home country for violating national guidelines, not to mention global scientific ethical standards.)
As the mother of a son with Duchenne muscular dystrophy, a cruel genetic disease that breaks down muscle until the body can no longer function, I don’t see CRISPR as scary or dangerous. To me, it represents the best hope we have for curing my son’s disease and possibly many others. I believe CRISPR is a weapon — not a giant gorilla type of weapon — but a quiet, surgically precise weapon against diseases that have long confounded the scientific community.
Real progress is being made using CRISPR and related gene-editing techniques to alter cells taken from people with diseases such as sickle cell and genetic forms of blindness. These approaches are likely to offer safer and more reliable ways of making changes to a person at the genetic level. What makes these uses much less ethically challenging [than germline editing] is that they only affect the person being treated; the genetic modifications can’t be passed on to future generations.