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Update January 2018


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Update January 20, 2018 - January 26, 2018

In a milestone year, gene therapy finds a place in medicine

FILE - In this Monday, Nov. 6, 2017 file photo, Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children’s Hospital in Oakland, Calif. Madeux, who has Hunter syndrome, received the treatment on Monday, Nov. 13. (AP Photo/Eric Risberg)

Marilynn Marchione

After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.

Almost 20 years ago, a teen’s death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures.

“I am totally willing to use the ‘C’ word,” said the National Institutes of Health’s director, Dr. Francis Collins.

Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw.

The advent of gene editing - a more precise and long-lasting way to do gene therapy - may expand the number and types of diseases that can be treated. In November, California scientists tried editing a gene inside someone’s body for the first time, using a tool called zinc finger nucleases for a man with a metabolic disease. It’s like a cut-and-paste operation to place a new gene in a specific spot. Tests of another editing tool called CRISPR, to genetically alter human cells in the lab, may start next year.

“There are a few times in our lives when science astonishes us. This is one of those times,” Dr. Matthew Porteus, a Stanford University gene editing expert, told a Senate panel discussing this technology last month.

It’s a common path for trail-blazing science - success initially seems within reach, setbacks send researchers back to the lab, new understandings emerge over years, and studies ultimately reveal what is safe and effective.

Here is a look at what’s been achieved and what lies ahead.

A string of firsts

The year started with no gene therapies sold in the U.S. and only a couple elsewhere. Then the Food and Drug Administration approved the first CAR-T cell therapies, which alter a patient’s own blood cells to turn them into specialized cancer killers. They’re only for certain types of leukemia and lymphoma now, but more are in the works for other blood cancers.

Last week, the FDA approved Luxturna, the first gene therapy for an inherited disease, a form of blindness. People with it can’t make a protein needed by the retina, tissue at the back of the eye that converts light into signals to the brain, enabling sight. The therapy injects a modified virus containing a corrective gene into the retina so the cells can make the protein.

Children who received the treatment told what it was like to gain vision.

“Oh yikes, colors. Colors are super fun,” said 13-year-old Caroline Carper of Little Rock, Arkansas. “And the sunshine is blinding.”

Gene therapies also showed some promise against a variety of diseases including hemophilia, a blood clotting problem; “bubble boy” disease, where a flawed immune system leaves patients vulnerable to fatal infections, and sickle cell disease, a serious and painful blood disorder common among black people.

It’s not all good news, though. The therapies don’t work for everyone. They’re shockingly expensive. And no one knows how long some results will last, though scientists say the aim is a one-time repair that gets at the root cause.

“The whole promise ... is to cure diseases. It’s based on the rationale of fixing the problem,” not just improving treatment, said Dr. Carl June, a University of Pennsylvania scientist who pioneered CAR-T therapy.

A new frontier:
gene editing

In mid-November, Brian Madeux, a 44-year-old Phoenix man with a metabolic disease called Hunter syndrome, had just become the first person to try an experimental gene editing treatment.

“I believe in science,” he texted The Associated Press after doctors sent viruses containing a corrective gene and an editing tool through an IV into his body. The hope is that the gene and the editing tool would enter some of his liver cells and insert the instructions needed to start making an enzyme he lacks.

It’s not known yet if it worked. Sangamo Therapeutics is testing its therapy in several studies, and independent monitors will help decide when results are released.

“It’s a pretty exciting milestone,” Collins said, because it shows a way to treat more diseases than ones that can be addressed now by altering blood cells in the lab or injecting genes into the eye.

“You can imagine having a scalable approach to thousands of genetic diseases,” he said.

What’s next?

Top of Collins’ list: muscular dystrophy and sickle cell.

There’s been so much progress that the NIH has modified an oversight panel that just a few years ago reviewed every gene therapy experiment in the U.S. Most are considered safe enough to go ahead without the Recombinant DNA Advisory Committee’s review. The panel hasn’t even met for a year.

When the panel was formed decades ago, “there was a lot of concern that a graduate student could take some of this home and create a monster in his basement,” said one panel member, Boston scientist Dr. Howard Kaufman.

Those fears have eased, he said.

“There’s no monsters that have materialized from this.”


Update January 13, 2018 - January 19, 2018

Veterinarians seek permission to research pot meds for pets

In this Monday, Oct. 30, 2017, photo, Luke Byerly tends to his 14-year-old beagle, Robbie, during a break at Byerly’s job as a technician at a veterinary clinic in east Denver. Byerly is using CBD, a non-psychoactive component of marijuana, oil to treat the dog’s arthritis. (AP Photo/David Zalubowski)

Andrew Selsky

Bend, Ore. (AP) - Dr. Byron Maas surveys a supply of marijuana products for dogs that lines a shelf in his veterinary clinic. They’re selling well.

“The ‘Up and Moving’ is for joints and for pain,” he explains. “The ‘Calm and Quiet’ is for real anxious dogs, to take away that anxiety.”

People anxious to relieve suffering in their pets are increasingly turning to oils and powders that contain CBDs, a non-psychoactive component of marijuana. But there’s little data on whether they work, or if they have harmful side effects.

That’s because Washington has been standing in the way of clinical trials, veterinarians and researchers say. Now, a push is underway to have barriers removed, so both pets and people can benefit.

Those barriers have had more than just a chilling effect.

When the federal Drug Enforcement Administration announced last year that even marijuana extracts with CBD and little or no THC - marijuana’s intoxicating component - are an illegal Schedule 1 drug, the University of Pennsylvania halted its clinical trials. Colorado State University is pushing ahead.

The U.S. Food and Drug Administration has warned companies that sell marijuana products online and via pet shops and animal hospitals that they’re violating laws by offering “unapproved new animal drugs.” The FDA threatened legal action.

But, seeing potential benefits of CBDs, the American Veterinary Medical Association’s policy-making body said last summer it wants the DEA to declassify marijuana as a Schedule 1 drug “to facilitate research opportunities for veterinary and human medical uses.” It asked the board of the national veterinarians’ organization to investigate working with other stakeholders toward that goal. The board is awaiting a recommendation from two group councils.

“The concern our membership has is worry about people extrapolating their own dosages, looking to medicate their pets outside the realm of the medical professional,” Board Chairman Michael Whitehair said in a telephone interview. “This is an important reason for us to continue the research.”

Utah Sen. Orrin Hatch, a conservative Republican, became an unlikely champion of this push when he introduced a bill in September that would open the path for more clinical research. While Hatch said he opposes recreational marijuana use, he wants marijuana-based drugs, regulated by the FDA, produced for people with disorders.

“We lack the science to support use of medical marijuana products like CBD oils, not because researchers are unwilling to do the work, but because of bureaucratic red tape and over-regulation,” Hatch said.

Dawn Boothe, of Auburn University’s College of Veterinary Medicine, is waiting for federal approval to begin a study of marijuana’s effects on dogs with epilepsy. The classification of marijuana products containing CBD as a Schedule 1 drug, the same category as heroin and LSD, creates a “major, major, major, terrible roadblock” for researchers, Boothe said in a phone interview.

Researchers at the University of Pennsylvania School of Veterinary Medicine were studying CBDs’ effects on dogs with osteoarthritis and pruritis, or itchiness, until the DEA released its policy statement.

“The ambiguity in this process has really brought us to a screeching halt,” said Michael DiGregorio, director of the university’s clinical trials center. “It is research that needs to be done, because there are a lot of CBD products out there.”

When it clarified that marijuana CBD extracts are Schedule 1 drugs, the DEA said it was assigning a code number to those substances to better track them and to comply with international drug control treaties.

DiGregorio complained that researchers seeking federal approval to study CBD products are told to provide certain data, but that data isn’t normally available until the study is done.

“If you don’t have the data, you can’t get the registration to do the work,” he said.

On a recent morning, Maas took a break from seeing four-legged patients in the Bend Veterinary Clinic. A stethoscope dangling from his neck over green scrubs, Maas said his clients have reported CBDs help relieve pain, arthritis, anxiety, loss of appetite, epilepsy and inflammation in their pets.

“Unfortunately there’s not a lot of research out there, especially on animals, on CBD compounds,” Maas said. “The research is really necessary to help us understand how to actually use these compounds on our pets.”

Veterinarian Janet Ladyga of the Blue Sky Veterinary Clinic, also in Bend, said she doesn’t recommend marijuana products because of the unknowns.

“We don’t have a lot of evidence right now, so we don’t know the toxicity or the safety profile ... and we don’t have any good evidence to show either if it’s safe or efficacious,” she said.

The study at Colorado State University aims to provide some data. The roughly two dozen dogs in the arthritis study and the 30 in the epilepsy tests are given either CBD oil or a placebo. For the arthritis study, activity monitors are attached to the animals’ collars, to determine if they’re more mobile when they’re taking CBD.

Principal investigator Stephanie McGrath said she hopes the results will be a stepping stone for longer and more diverse studies, and that they provide useful information for human medicine.

“Every medication we’re taking has been given to a dog first,” the University of Pennsylvania’s DiGregorio noted.

Meanwhile, Boothe said she had everything ready to start her study in January, and was waiting for a green light from federal officials.

“I don’t know what’s taking so long,” she said.


Update Saturday, Jan. 6 - Jan. 12, 2018

Deaths from window blinds show need for cord ban, study says

This file photo shows a warning label of strangulation risks from mini blind cords in Washington. According to a study released this month, children’s injuries and deaths from window blinds have not stalled despite decades of safety concerns. (AP Photo/Jacquelyn Martin)

Lindsey Tanner

Chicago (AP) - Children’s injuries and deaths from window blinds have not stalled despite decades of safety concerns, according to a new U.S. study that recommends a complete ban on blinds with cords.

Nearly 17,000 young children were hurt by window blinds between 1990 and 2015, and though most injuries were minor, almost 300 died, the study shows. Most deaths occurred when children became entangled or strangled by the cords.

Injuries continued even after manufacturers adopted voluntary safety standards including warning labels. The industry now has a plan in the works to make cordless blinds the only option at retail stores and online.

The study “should be a huge wake-up call to the public, to the retailers, to the manufacturers and to parents all over the nation to really see how hazardous the cords on the blinds are,” said Linda Kaiser of St Louis. Her 1-year-old daughter died in 2002 from strangulation when she pulled a looped hidden cord from a blind and put it around her neck. Kaiser later formed the advocacy group Parents for Window Blind Safety.

While study’s data analysis doesn’t show an up or down trend in injuries and deaths, the fact that they’re still occurring shows that safety standards have been inadequate, said lead author, Dr. Gary Smith, who directs injuries research at Nationwide Children’s Hospital in Columbus, Ohio.

Paul Nathanson, spokesman for the Window Covering Manufacturers Association, said a soon-to-be adopted industry standard drafted with input from the Consumer Product Safety Commission will make corded blinds unavailable in stores and online, although consumers could buy them through custom orders.

The safety commission says windows and window blinds are among the top five hidden hazards in U.S. homes and in a statement, it called the draft standard “a major step forward in protecting children.”

That standard is awaiting approval by the American National Standards Institute and is expected to take effect by late 2018, Nathanson said.

Smith said 20 percent are custom blinds and a total ban on corded blinds is needed.

The study was published Monday in the journal Pediatrics.

His research team analyzed 26 years of U.S. government data on emergency room treatment and fatal injuries. The study notes that the dangers have been addressed in medical journal articles as far back as a 1945 report on two accidental hangings in children who survived.

“Seventy years ago we recognized that this was a product that was killing kids,” Smith said. “We should put child safety first.”


Baby gene therapy study offers hope for fatal muscle disease

Lauran Neergaard

Washington (AP) - A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots’ lives, and some began to roll over, sit and stand on their own, researchers reported.

Only 15 babies with spinal muscular atrophy received the experimental gene therapy, but researchers in Ohio credited the preliminary and promising results to replacing the infants’ defective gene early - in the first few months of life, before the neuromuscular disease destroyed too many key nerve cells.

“They all should have died by now,” said Dr. Jerry Mendell of Nationwide Children’s Hospital, who led the work published by The New England Journal of Medicine. Yet, “those babies are still improving.”

Mendell cautioned that much more study is needed to prove the gene therapy works and is safe. Nor is it clear whether the replacement gene’s effects would wane over time.

Spinal muscular atrophy occurs in about 1 in 10,000 births, and those with the most severe form, called SMA Type 1, rarely reach their second birthday. They can be born looking healthy but rapidly decline. One study found just 8 percent of the most severely affected survived to age 20 months without needing permanent mechanical ventilation to breathe.

There is no cure. The first treatment wasn’t approved until last December - a drug named Spinraza that requires spinal injections every few months.

The experimental gene therapy approach aims for a one-time fix.

What goes wrong

Spinal muscular atrophy is caused when a mutated gene can’t produce a protein crucial for survival of motor neurons, nerve cells in the spinal cord that control muscles.

Some children carry extra copies of a backup gene that produces small amounts of the vital protein, and thus have much milder forms of the disease.

Gene replacement

Scientists loaded a healthy version of the gene into a virus modified so it couldn’t cause illness. Then 15 babies got a one-time intravenous injection. The virus carried the healthy gene into motor neurons, where it got to work producing the protein those nerve cells require to live.

Three babies received a low dose of the gene therapy, as a first-step safety precaution. The remaining 12 got a high dose.

Results

All of the children are alive, Mendell said, about two years and counting after treatment. All beat the odds of needing permanent machine help to breathe by age 20 months.

But only the high-dose recipients saw better motor control, reaching some developmental milestones usually unthinkable for these patients. Eleven could sit unassisted at least briefly; nine could roll over. Eleven are speaking and able to swallow. Two were able to crawl, stand and then walk, Mendell’s team reported.

Those results are “very striking,” said Dr. Basil Darras, who directs Boston Children’s Hospital’s neuromuscular center and wasn’t involved in the new research.

While the treatment needs testing on far more babies, usually “there are no further developmental gains” after diagnosis, Darras explained. “They stagnate for a while and they go downhill very fast and die.”

The only serious side effect attributed to the gene therapy so far involved possible signs of a liver problem that eased with treatment.

Next steps

AveXis Inc., which is developing the gene therapy and helped fund Wednesday’s study, has opened a second small trial at seven hospitals.

Meanwhile, doctors are prescribing SMA patients the new medication Spinraza, which works by increasing that backup gene’s protein production and, according to a separate New England Journal study, had some benefit in about half of patients. The first year of treatment costs about $750,000, an accompanying editorial noted.

With the drug’s availability, some health groups are urging that SMA be added to the list of diseases that all newborns are screened for, so parents can seek early treatment.
 


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HEADLINES [click on headline to view story]

In a milestone year, gene therapy finds a place in medicine


Veterinarians seek permission to research pot meds for pets


Deaths from window blinds show need for cord ban, study says

Baby gene therapy study offers hope for fatal muscle disease


 



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