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The ‘Cruel Joke’ of Compassionate Use and Right to Try: Pharma Companies Don’t Have to Comply

2 weeks 4 days

(STAT News) – That changed a few weeks ago when my 28-month-old daughter, Radha, was diagnosed with a lysosomal storage disorder. Now I know far more about these diseases than I did in medical school. I’ve also learned a frustrating fact that no medical school teaches its students: While the FDA has a compassionate use program to allow people access to experimental drugs, it can’t compel a company to provide those drugs. The newly signed “right-to-try” law doesn’t either.

‘Remarkable’ Therapy Beats Terminal Breast Cancer

2 weeks 4 days

(BBC) – The technology is a “living drug” made from a patient’s own cells at one of the world’s leading centres of cancer research. Dr Steven Rosenberg, chief of surgery at the National Cancer Institute, told the BBC: “We’re talking about the most highly personalised treatment imaginable.”  It remains experimental and still requires considerably more testing before it can be used more widely, but this is how it works: it starts by getting to know the enemy.

U.S. High Court Throws Out Immigrant Teen Abortion Ruling

2 weeks 4 days

(Reuters) – The U.S. Supreme Court on Monday threw out a lower court ruling that let a pregnant illegal immigrant minor held in federal immigration custody obtain an abortion last year at age 17 over the objections of President Donald Trump’s administration. The justices provided a legal victory to Trump’s administration even though the teenager already has had the abortion because it eliminated a precedent at the federal appeals court level that could have applied in similar circumstances in which other detained minors sought abortions.

As Aid Workers Move to the Heart of Congo’s Ebola Outbreak, ‘Everything Gets More Complicated’

3 weeks 21 hours

(New York Times) – Aiming to squelch an Ebola outbreak that has infected 54 people, killing almost half of them, aid workers in the Democratic Republic of the Congo have begun giving an experimental vaccine to people in the rural region at the epicenter of the outbreak. Epidemiologists working in the remote forests have not yet identified the first case, nor many of the villagers who may have been exposed. Investigators will need to overcome extreme logistical hurdles to reconstruct how the virus was transmitted, vaccinate contacts and halt the spread.

Scientists Have 3D Printed the Most Advanced Artificial Cornea Ever Using Human Cells

3 weeks 21 hours

(The Verge) – Scientists have 3D printed the thin protective film over the eye, called the cornea, using human cells — and it’s the most advanced version of an artificial cornea yet. Should the technology improve, it could help millions of people see again. It was tricky to find the right recipe for an ink that’s thin enough to squirt through a 3D printer’s nozzle, says Che Connon, a tissue engineer at Newcastle University who was one of the creators of the artificial cornea. This bio-ink didn’t just have to be thin — it also had to be stiff enough that it could hold its shape as a 3D structure.

Gallup: Most Americans Say Euthanasia Should Be Legal

3 weeks 21 hours

(UPI) – Nearly three-quarters of Americans support the practice of patient euthanasia, results of a Gallup poll published Thursday indicate. According to Gallup’s results, 72 percent of Americans believe doctors should be legally allowed, at a patient’s and a family’s request, to end a terminally ill patient’s life using painless means. Young adults, men, Democrats and liberals are especially likely to favor euthanasia, Gallup said. Only among weekly churchgoers does support for euthanasia drop below a majority.

FDA Halts One of the First Human CRISPR Studies Before It Begins

3 weeks 1 day

(MIT Technology Review) – A trial planning to use the gene-editing tool CRISPR on sickle-cell patients has been put on hold because of unspecified questions from US regulators. CRISPR Therapeutics, which is developing the therapy, sought approval from the US Food and Drug Administration in April to begin the study. The therapy involves extracting stem cells from a patient’s bone marrow and editing them with CRISPR in the lab. The idea is that the edited cells, once infused back into the patient, would give rise to healthy red blood cells.

Another Antibiotic Crisis: Fragile Supply Leads to Shortages

3 weeks 1 day

(Reuters) – Shortages of some life-saving antibiotics are putting growing numbers of patients at risk and fuelling the evolution of “superbugs” that do not respond to modern medicines, according to a new report on Thursday.  The non-profit Access to Medicine Foundation (AMF) said there was an emerging crisis in the global anti-infectives market as fragile drug supply chains – reliant on just a few big suppliers – come close to collapse.

Who Gets Credit for CRISPR? Prestigious Award Singles Out Three, and Leaves Out a Notable Scientist

3 weeks 1 day

(STAT News) – One of the world’s richest science awards, given only in alternate years, will go to three discoverers of the CRISPR-Cas9 genome-editing tool, the Norwegian Academy of Science and Letters announced on Thursday. Emmanuelle Charpentier of the Max Planck Institute for Infection Biology, Jennifer Doudna of the University of California, Berkeley, and Virginijus Šikšnys of Vilnius University will each receive a gold medal and share the $1 million that comes with the Kavli Prize in nanoscience (there are also Kavli prizes for astrophysics and neuroscience).

Gene Therapy Is Saving Children’s Lives–But Screening to Discover Who Needs It Is Lagging Behind

3 weeks 1 day

(MIT Technology Review) – Newborn screening tests in the US cover a minimum of 34 disorders, and many states have opted to add more. Yet most don’t look for spinal muscular atrophy (commonly abbreviated as SMA), even though it’s the leading genetic cause of infant death. About 400 babies in the US are born with it every year. AveXis’s therapy is for the most common form, type 1. For parents of children with SMA, like Krystal Davis, newborn screening means giving those with the condition as close to a normal life as possible.

In Utero Transplant in First Clinical Trial Successful

3 weeks 1 day

(The Scientist) – Pediatric surgeons at the University of California, San Francisco, have treated a second-trimester fetus with stem cells taken from her mother’s bone marrow. The baby, born in February, was the first patient enrolled in the world’s first clinical trial using stem cells transplanted prior to birth. She is “apparently healthy,” despite living with a deadly genetic disease called alpha thalassemia, according to a statement from the university.

Pioneering Surgery Makes a Prosthetic Foot Feel Like the Real Thing

3 weeks 2 days

(STAT News) – The key to making the bionic like the biological was combining a surgical advance with a technological one, the researchers say. Ewing — who mangled his left foot when he fell about 50 feet from a cliff he was scaling in the Cayman Islands — was the first person to undergo an entirely new kind of amputation, pioneered by Carty and MIT professor Hugh Herr. The engineers, meanwhile, developed a prosthetic foot that would enable two-way communication, with signals traveling from Ewing’s brain to his residual lower leg and into the bionic limb, and then back again.

Study Puts Puerto Rico Death Toll from Hurricane Maria Near 5,000

3 weeks 2 days

(NPR) – Perhaps 5,000 people died in Puerto Rico in 2017 for reasons related to September’s Hurricane Maria, according to a study that dismisses the official death toll of 64 as “a substantial underestimate.” A research team led by scientists at the Harvard T.H. Chan School of Public Health didn’t simply attempt to count dead bodies in the wake of the powerful storm. Instead, they surveyed randomly chosen households and asked the occupants about their experiences.

Supreme Court Leaves in Place Law that Effectively Bans Abortion By Pill–For Now

3 weeks 2 days

(NPR) – The U.S. Supreme Court refused on Tuesday to hear a challenge to an Arkansas abortion law that, in practice, bars abortions by pill instead of by surgical procedure. The result is that Arkansas is now the only state in the country that essentially bans abortion by pill, a method certified by the federal Food and Drug Administration as at least as safe as surgical abortions. The Supreme Court’s decision not to intervene in the case at this point, however, is not final.

The Latest: Portugal’s Parliament Rejects Legal Euthanasia

3 weeks 2 days

(ABC News) – Portuguese lawmakers have rejected proposals to make Portugal one of only a handful of countries in the world allowing euthanasia and doctor-assisted suicide. After a landmark debate Tuesday, lawmakers voted to reject four broadly similar bills introduced by left-leaning parties. The bill that came closest to succeeding was the governing center-left Socialist Party’s proposal, which lost on a 115-110 vote.

‘Reprogrammed’ Stem Cells Approved to Mend Human Hearts for the First Time

3 weeks 2 days

(Nature) – Scientists in Japan now have permission to treat people who have heart disease with cells produced by a revolutionary reprogramming technique. The study is only the second clinical application of induced pluripotent stem (iPS) cells. These are created by inducing the cells of body tissues such as skin and blood to revert to an embryonic-like state, from which they can develop into other cell types. On 16 May, Japan’s health ministry gave doctors the green light to take wafer-thin sheets of tissue derived from iPS cells and graft them onto diseased human hearts.

‘Right-to-Try’ Bill Passes Congress

3 weeks 3 days

(CNN) – With a House of Representatives vote Tuesday, Congress passed legislation that could give terminally ill patients a way to independently seek drugs that are still experimental and not fully approved by the US Food and Drug Administration. The House voted 250-169 in favor of the bill, which the Senate passed in August. The bill will now be sent to President Trump, who is expected to sign it.

Irish Abortion Referendum: Ireland Overturns Abortion Ban

3 weeks 3 days

(BBC) – The Republic of Ireland has voted overwhelmingly to overturn the abortion ban by 66.4% to 33.6%. A referendum held on Friday resulted in a landslide win for the repeal side. Currently, abortion is only allowed when a woman’s life is at risk, but not in cases of rape, incest or fatal foetal abnormality. The Eighth Amendment, which grants an equal right to life to the mother and unborn, will be replaced. The declaration was made at Dublin Castle at 18:13 local time.

Polio Eradication Initiative Meets Resistance to Appeals to Destroy Samples

4 weeks 1 day

(STAT News) – For more than two years, the global initiative to eradicate polio has been trying to convince companies and laboratories around the world to get rid of stored samples of an extinct poliovirus, with little success. Thirty countries have indicated they plan to hold on to type 2 polioviruses, in a total of 99 facilities. That, health officials warn, could endanger years of efforts to stop the virus from crippling children.

Children, Fatal Illness and the Nature of Suffering

4 weeks 1 day

(Scientific American) – But what about a nonverbal child or infant? How do we determine if they are suffering? This is what is at the heart of the public debates about how to care for children with incurable illness, and much of this involves our own perception, and even projection, while observing a child. But is a child necessarily suffering just because for us, as observers, it feels like suffering to watch? In the absence of hard evidence to the contrary, we need to rely on parents to guide us about how they identify and perceive their child’s suffering.

Court Upholds Ruling Against California’s Assisted Suicide Law

4 weeks 1 day

(NPR) – An appeals court has let stand a lower court ruling overturning a California law that allows physicians to prescribe life-ending drugs to the terminally ill. California’s Fourth District Court of Appeals on Wednesday refused to stay last week’s decision by the Riverside County Superior Court, which ruled that state lawmakers should not have passed the law during a special session on health care funding. However, the constitutionality of the law itself — passed nearly three years ago — was not challenged.

Kosovo Court Imprisons 2 over Illegal Organ Harvesting

4 weeks 1 day

(ABC News) – A Kosovo court on Thursday imprisoned two ethnic Albanians for involvement in organ harvesting. The Pristina court international judge Francesca Fischer sentenced former urologist Lutfi Dervishi to 7 years and a fine of 8,000 euros ($9,400). He cannot exercise his profession for two years after his prison term ends. Sokol Hajdini, an anesthetist, was sentenced to one year in prison after it was proven that he was aware of the work and willingly took part.

No Country Resorts to IVF More Than Japan–Or Has Less Success

1 month 3 min

(The Economist) – Japan has come a long way since journalists were warned off the taboo story of Princess Masako’s visits to fertility clinics 20 years ago. The wife of the crown prince, then in her late thirties, was being nudged to produce an heir to the throne (in the end, she disappointed traditionalists by having a girl). Today Japan has less than half America’s population, but more than a third more hospitals and clinics that offer fertility treatment. Over 50,000 babies were born last year with the help of in vitro fertilisation (IVF)—5% of all births.

The Success of the New Ebola Vaccine Will Hinge on Trust

1 month 6 min

(Vox) – The quest to convince people not to fear a lifesaving vaccine is underway in the Democratic Republic of Congo, where an ongoing outbreak of the Ebola virus has transfixed the global health community. For the first time ever in an Ebola outbreak, health care workers have a vaccine to offer people at risk of infection. The experimental shot has had a remarkable rate of success in clinical trials, and it could be a game changer in the DRC outbreak, which has already infected 58 people, including 27 deaths. But success hinges on one major obstacle: People in the country need to trust and accept the new vaccine, and it’s not a given they will.

Hybrid Human-Chicken Embryos Illuminate Key Developmental Milestone

1 month 34 min

(Nature) – Before a cluster of cells can develop into an embryo, it must first decide which end is up. But that process had never been observed in humans — until now. For the first time, researchers have watched human ‘organizer’ cells direct the formation of an embryo’s top, bottom, front and back. They did so by developing a technique that sidesteps restrictions on research with human embryos by grafting human cells onto chicken embryos. The method, published on 23 May in Nature, could supplant the use of human embryos in some laboratory experiments.


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