More than 200 people have been treated with experimental CRISPR … – MIT Technology Review

I watched scientists, ethicists, patient advocacy groups, and others wrestle with these topics at the Third International Summit on Human Genome Editing in London earlier this week.

Theres plenty to get excited about when it comes to gene editing. In the decade since scientists found they could use CRISPR to edit cell genomes, multiple clinical trials have sprung up to test the technologys use for serious diseases. CRISPR has already been used to save some lives and transform others.

But it hasnt all been smooth sailing. Not all of the trials have gone to plan, and some volunteers have died. Successful treatments are likely to be expensive, and thus limited to the wealthy few. And while these trials tend to involve changes to the genes in adult body cells, some are hoping to use CRISPR and other gene-editing tools in eggs, sperm, and embryos. The specter of designer babies continues to loom over the field.

It was at the last summit, held in Hong Kong in 2018, that He Jiankui, then based at the Southern University of Science and Technology in Shenzhen, China, announced that he had used CRISPR on human embryos. The news of the first CRISPR babies, as they became known, caused a massive ruckus, as you might imagine. Well never forget the shock, Victor Dzau, president of the US National Academy of Medicine, told us.

He Jiankui ended up in prison and was released only last year. And while heritable genome editing was already banned in China at the timeit has been outlawed since 2003the country has since enacted a series of additional laws designed to prevent anything like that from happening again. Today, heritable genome editing is prohibited under criminal law, Yaojin Peng of the Beijing Institute of Stem Cell and Regenerative Medicine told the audience.

There was much less drama at this years summit. But there was plenty of emotion. In a session about how gene editing might be used to treat sickle-cell disease, Victoria Gray, a 37-year-old survivor of the disease, took to the stage. She told the audience about how her severe symptoms had disrupted her childhood and adolescence, and scuppered her dreams of training to be a doctor. She described episodes of severe pain that left her hospitalized for months at a time. Her children were worried she might die.

But then she underwent a treatment that involved editing the genes in cells from her bone marrow. Her new super cells, as she calls them, have transformed her life. Within minutes of receiving her transfusion of edited cells, she felt reborn and shed tears of joy, she told us. It took seven to eight months for her to feel better, but after that point, I really began to enjoy the life that I once felt was just passing me by, she said. I could see the typically stoic scientists around me wiping tears from their eyes.

Victoria is one of more than 200 people who have been treated with CRISPR-based therapies in clinical trials, said David Liu of the Broad Institute of MIT and Harvard, who has led the development of new and improved forms of CRISPR. Trials are also underway for a range of other diseases, including cancers, genetic vision loss, and amyloidosis.

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More than 200 people have been treated with experimental CRISPR ... - MIT Technology Review

The 30th Biohacking Health and Wellness Biomed EXPO returning to Los Angeles on March 24-26, 2023, latest Stem Cell, Brain enhancement Technologies…

LOS ANGELES, March 7, 2023 /PRNewswire/ -- Biomed Expo ushers in new earth frequencies of biohacking and Trans-Humanism at Sonesta Los Angeles LAX Hotel, on March 24-26. The conference brings together a highly coveted group of physicians to defy the preconceived limits of the human body. With workshops and many exhibitors, this healing fest has curated the leading minds behind the latest leaps in healing modalities from scalar energy to functional homeopathy and Quantum Healing.

Attendees will receive codes on healthcare from a future timeline in which doctors harness Gaia's source codes to help patients of conditions from cancer to Alzheimer's to lupus with non-invasive modalities.

This year's programming will be the largest quantum leap to the future yet: medical researchers, doctors, and disclosure groups, EX- Military Intelligence, California DEPT of Health Whistleblower, and the New Humanity Movement leaders will speak candidly on classified topics including: Tesla's discoveries, Space Science, ET Disclosure, Origin of Man - converging the disciplines of metaphysics and medicine to enhance human cognitive functions, extend lifespan, and expand extrasensory perceptions like clairvoyance.

Keynote speakers:

Dr. Barry Morguelan, M.D., the only western Grand Master of a 5,000-year-old Chinese Source Energy discipline and Founder of Upgrade Labs

Dr. Todd Ovokaitys, M.D. Pioneering Qi-laser resonance technology inventor

Dr. Michael Grossman, M.D. Stem Cell researcher

Dr. Patrick Porter, CEO of BrainTap, Brain Advancement Technologies

Dr. Beverly Rubik, International Biophysicist & EMF Researcher

more amazing speakers:

Dr. Robert Young, The Worldwide Renowned Microbiologist and Virologist.

Dr. Scott Werner, Dr. Jeffrey Benton, Dr. Doug Lehrer, Dr. Vanessa Pavey, Dr. Rollin McCraty, SIR Kaya Redford, Brooks Agnew, Michael Cremo, Dr. Lori Smiskol, Saeed David Farman, Ryan Veli, Ismael Perez, Perry Kamel, Debbie Hawkins, Nichola Burnett, Shehnaz Soni, Veronica Bucheli, Michael Schratt, Azucena Avila, Eric Dadmehr, Robert Potter, Alan Bedian, Michelle Jewsbury, Amelia Brummel, Phillip Wilson, Sandra Biskind, Geraldine Orozco & Julia Galasso.

Story continues

Registration for this three-day extravaganza of meet-and-greet banquet dinners with daily Functional wellness panels and over 100 lectures, workshops on biohacking is now open at https://biomedexpo.com

Cision

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The 30th Biohacking Health and Wellness Biomed EXPO returning to Los Angeles on March 24-26, 2023, latest Stem Cell, Brain enhancement Technologies...

New gene therapies are a near-cure for Louisiana’s sickle cell patients. But at $1-2M, who will pay for it? – NOLA.com

When Leola Conleys daughter was born, everybody said she looked like she was already three months old.

The girl was 8 pounds, 13 ounces, cute, with a full head of hair, said Conley, a counselor in Lafayette.

About a week after her birth, a letter arrived from the state Department of Health. A standard pinprick on the babys heel at birth showed a mutation in a gene that makes red blood cells. Usually, red blood cells are round, like a doughnut. This babys cells were shaped like a crescent moon. She had sickle cell disease, a genetic disorder that affects about one in every 365 Black babies. Those with severe cases have a life expectancy of about 45 years.

It put me in a depression like nobody's business, said Conley. It broke me, because she looked so healthy.

Now 23, Conleys daughter, Adria, guesses shes been hospitalized about 30 times. She graduated with her MBA in December but is limiting her job search to positions with good insurance close to her doctors in south Louisiana. She doesnt look sick, but unpredictable pain episodes can throw her life off track, requiring weeks of recovery.

But new gene therapies two of which will submit for FDA approval by the end of March offer what could be a life-changing alternative for sickle cell patients in Louisiana,which has the second-highest prevalence of sickle cell disease among Medicaid patients. The problem? At a predicted $1 million to $2 million for the therapy, its unclear how states will pay for it.

If just 7% of the eligible sickle cell population of Louisiana wanted the treatment, it would cost the state a predicted $31.6 million in the first year, according to an analysis published in JAMA Pediatrics by Dr. Patrick DeMartino, a physician specializing in blood diseases among children. Thats for just 17 patients.

Nobody really knows whether it's going to be three people getting the gene therapy in the first year or 15, said DeMartino. And that upper end of 15 to 20 people a year would almost certainly be very consequential for the Medicaid budget.

No one tracks how many Louisiana residents have sickle cell disease, though efforts to create a registry are underway. There are at least 3,000 people on Medicaid with sickle cell disease, according to the state. The 1,400 who were hospitalized in Louisiana in 2020 spent an average of 30 days in the hospital. About one in 10 children with sickle cell anemia will have a symptomatic stroke by age 20, and more have strokes that go undetected.

These people do not live normal lives at all, said DeMartino. The existing therapies are supportive. They don't change the underlying disease, and people still have massive amounts of disability.

Instead of easily transporting oxygen through their bodies, the warped cells become wedged in blood vessels, causing blockages and injuring the vessels, resulting in immense pain.

Have you ever felt your heart beating in your ear? It feels like that but all over your body, said Whitney Carter, a 37-year-old who works from home for an insurance company in Baton Rouge.

The sickled cells getting jammed in her blood vessels feels "like a walnut trying to fit through a straw.

Without a potential pain episode looming over Rhonda Chubes 9-year-old son, he would be able to keep up with his older brothers, play football, swim and collect rocks to his hearts content. With treatment, hed be a regular kid, said Chube, a child care assistant at a Baton Rouge hospital.

His little spirit, he wants to do more, keep going, said Chube. But his little body won't let him.

Prior to gene therapy, a bone-marrow transplant was the only thing close to a cure, but required a close genetic match, usually a sibling, resulting in a match for about 25% of patients. There is also risk that the body will reject the transplant, causing serious complications or death.

Researchers shy away from using the word cure to describe gene therapy, because its not yet clear how long its effects last since the first was given in 2017. But it has been shown to deliver a pain-free existence for trial participants for the first time in their lives.

There are two different gene therapy drugs to treat sickle cell expected to be approved soon one from Bluebird Bio and the other from Vertex and CRISPR Therapeutics.

Its highly transformative, said Dr. Julie Kanter, a hematologist at the University of Alabama at Birmingham who oversees a Bluebird trial that includes patients from Louisiana.

Whats most notable when I look at my patients is how many are now working that couldnt work before, said Kanter. I have one in nursing school, one in a full-time construction job They dont have to worry about being too far from a hospital or too far from pain medicines.

But how to deliver the medications, which will require an expensive and lengthy hospital stay not included in the drugs price tag, is something the world is trying to figure out right now, said Kanter, a New Orleans native who was previously head of the Sickle Cell Center of Southern Louisiana.

Figuring out a way to fund sickle cell treatment is a priority of the Biden administration. And Louisiana has gotten creative with paying for expensive therapies before, Dr. Joe Kanter, state health officer, pointed out. The state got unlimited access to pricey hepatitis C medication for five years for a lump sum instead of paying per dose.

I think the hepatitis C work laid a good groundwork for the feds and other states to follow if they choose to, said Kanter, who is not related to Julie Kanter.

The U.S. Department of Health and Human Services released a plan in mid-February that aims to address the sky-high cost of gene therapy. A pilot program would allow the federal government to coordinate a way to pay on behalf of states. For example, Medicaid might negotiate to pay for treatment in installments dependent on the therapy continuing to prevent pain, which has not yet been proven.

The plan will likely take years to implement.

Still, not everyone will want the therapy, which requires a long hospital stay and chemotherapy to make room for new stem cells.

For many patients, even words like transformative and life-changing can seem empty after years of mistreatment.

Every time I go to the hospital, I feel like I have to put my shield on, because Im going to battle, said Leola Conley. Who will I fight today?

Conley and her daughter have endured 11-hour waits in emergency rooms to get admitted and health care workers who dont believe her pain.

A lack of investment in treatment for sickle cell patients means that the only option for some is often opioids in an emergency room, said Dr. Jennifer Avegno, an emergency medicine physician and director of the New Orleans Health Department.

"Being on chronic opiates is really a terrible way to live," said Avegno. "We've really taken a terrible disease and made it worse in America."

In comparison to sickle cell, genetic diseases that mainly affect White people, such as cystic fibrosis, have much more investment, Avegno pointed out. A federal study found funding for cystic fibrosis was 11 times that of per-person funding for sickle cell disease, even though it impacts about one-third of the number of people.

In a world without sickle cell,Adria Conleyimagines shed move to the Netherlands, where shes read people are happy.Maybe one day, with gene therapy, that will be a reality. For now, sickle cell is calling the shots.

If gene therapy was offered as a safe and affordable option, we would run to it, said Leola Conley. Her fear is that one day, her daughter will be in pain and she wont be around to make sure she gets the right treatment.

Before I leave this earth, I hope to heck my child can be cured, said Conley. She has so much to offer the world.

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New gene therapies are a near-cure for Louisiana's sickle cell patients. But at $1-2M, who will pay for it? - NOLA.com

Medical tourism to Mexico is on the rise, but it can come with risks – WMUR Manchester

One of the four Americans who were kidnapped in Mexico last week was traveling for medical tourism, a friend said. A growing number of U.S. residents are traveling internationally to seek more affordable medical care, more timely care or access to certain treatments or procedures that are unapproved or unavailable in the United States.Latavia "Tay" Washington McGee, 33, drove to Mexico with Shaeed Woodard, Zindell Brown and Eric Williams for cosmetic surgery that was scheduled to take place Friday, according to a close friend of Washington McGee's who did not want to be identified.The four Americans were found Tuesday near the border city of Matamoros, officials said. Washington McGee and Williams were found alive, and Woodard and Brown were found dead, a U.S. official familiar with the investigation told CNN. Investigators are still piecing together what happened after they were abducted.Video below: Hear from the family of some of the Americans recently kidnapped in Mexico.Medical tourism takes people all over the world, including to Mexico, India and Eastern Europe. Violence against medical tourists is generally thought to be rare, but the U.S. Centers for Disease Control and Prevention warns about other risks such as quality of care, infection control and communication challenges with medical staff."It's on the daily, without a doubt. There are people going daily to get this kind of stuff done," said Dr. Nolan Perez, a gastroenterologist in Brownsville, Texas, which is across the border from Matamoros. "Whether it's primary care provider visits or dental procedures or something more significant, like elective or weight loss surgery, there's no doubt that people are doing that because of low cost and easier access."A growing trend One study published in the American Journal of Medicine estimated that fewer than 800,000 Americans traveled to other countries for medical care in 2007, but by 2017, more than 1 million did. More current estimates suggest that those numbers have continued to grow.Video below: Americans are saving by traveling abroad for medical care"People travel because there may be a long waiting time, wait lists or other reasons why they can't get treatment as quickly as they would like it. So they explore their options outside the United States to see what's available," said Elizabeth Ziemba, president of Medical Tourism Training, which provides training and accreditation to international health travel organizations.Also, "price is a big issue in the United States. We know that the US health care system is incredibly expensive," she added. "Even for people with insurance, there may be high deductibles or out-of-pocket costs that are not covered by insurance, so that people will look based on price for what's available in other destinations."The most common procedures that prompt medical tourism trips include dental care, surgery, cosmetic surgery, fertility treatments, organ and tissue transplants and cancer treatment, according to the CDC."With Mexico and Costa Rica, it's overwhelmingly dental and cosmetic surgery. However, certain countries are known for specialties. For example, in Singapore, stem cell and oncology is huge. In India, South India and Chennai Apollo hospitals does incredible work with hip and knee surgeries," Josef Woodman, founder of Patients Beyond Borders, an international health care consulting company, said."In Eastern Europe, a lot of people from the UK but also people from the United States travel to Hungary, Croatia and Turkey for everything from dental to light cosmetic surgery," he said.Mexico is the second most popular destination for medical tourism globally, with an estimated 1.4 million to 3 million people coming into the country to take advantage of inexpensive treatment in 2020, according to Patients Beyond Borders. Matamoros where officials said the four kidnapped Americans were found is "not considered a primary medical travel destination," Woodman said, "largely because there are no internationally accredited medical centers/specialty clinics there or in the immediate region."Mexico City, Cancun and Tijuana are more frequented and reliable destinations in the country, Woodman said.On average, Americans can save 40% to 60% across the most common major procedures received by medical tourists in Mexico, according to an analysis of 2020 health ministry data conducted by Patients Beyond Borders.Woodman said that violence against medical tourists was extremely rare, but he added that "price shopping" searching for the cheapest location for a procedure is a "blueprint for trouble," namely substandard medical care.Video below: U.S. Attorney General Merrick Garland offers remarks about the Americans who were kidnapped in Mexico.'There are the complexities' Medical tourism can be dangerous, depending on the destination and the person's condition."There are the complexities of traveling if you have a medically complex situation. There are fit-to-fly rules. And your health care providers should take into consideration the impact of traveling if you have orthopedic injuries or issues," Ziemba said."The quality of care may be an unknown," she said. "It may be that the quality of care is not up to the standards that you would like. So there's a bit of an unknown there, and then the last thing I would say is, if something goes wrong, what's going to happen?"Perez said he commonly manages complications from medical tourism in his practice."There are a lot of bad outcomes. There are a lot of infections and a lot of botched procedures gone wrong, and patients have to come back to the United States and then have a revision of the surgery," he said. "So it's really unfortunate."Yet Ziemba added that there can be benefits to medical tourism, including that someone could receive a service that they need faster overseas than locally."And price: If you simply can't afford the out-of-pocket costs of health care in the United States, and assuming the risks involved, it may make much more sense for you financially to travel outside the United States," she said.Medical tourism is not just for people traveling around the world. Many living along the U.S.-Mexico border, where access to health care can be scarce, cross into Mexico for care.The Rio Grande Valley, at the southernmost point of Texas, is considered to be a medically underserved area. The region has some of the nation's highest rates of comorbidities, including obesity and diabetes, and one of the lowest physician-to-patient ratios.There is a "dire need" for health care professionals along the border, Perez said."There are not as many doctors given our big and our growing population down here. So the demands on primary care doctors and specialists are very high because there are not enough of us for this population," he said. "So that's one reason why people end up going to Mexico to visit with physicians, because of easier access."People interested in medical tourism can take some steps to help minimize their risk, the CDC says.Those planning to travel to another country for medical care should see their health care provider or a travel medicine provider at least four to six weeks before the trip and get international travel health insurance that covers medical evacuation back to the United States.The CDC advises taking copies of your medical records with you and checking the qualifications of the providers who will be overseeing your medical care. Also, make sure you can get any follow-up care you may need.

One of the four Americans who were kidnapped in Mexico last week was traveling for medical tourism, a friend said.

A growing number of U.S. residents are traveling internationally to seek more affordable medical care, more timely care or access to certain treatments or procedures that are unapproved or unavailable in the United States.

Latavia "Tay" Washington McGee, 33, drove to Mexico with Shaeed Woodard, Zindell Brown and Eric Williams for cosmetic surgery that was scheduled to take place Friday, according to a close friend of Washington McGee's who did not want to be identified.

The four Americans were found Tuesday near the border city of Matamoros, officials said. Washington McGee and Williams were found alive, and Woodard and Brown were found dead, a U.S. official familiar with the investigation told CNN.

Investigators are still piecing together what happened after they were abducted.

Video below: Hear from the family of some of the Americans recently kidnapped in Mexico.

Medical tourism takes people all over the world, including to Mexico, India and Eastern Europe. Violence against medical tourists is generally thought to be rare, but the U.S. Centers for Disease Control and Prevention warns about other risks such as quality of care, infection control and communication challenges with medical staff.

"It's on the daily, without a doubt. There are people going daily to get this kind of stuff done," said Dr. Nolan Perez, a gastroenterologist in Brownsville, Texas, which is across the border from Matamoros. "Whether it's primary care provider visits or dental procedures or something more significant, like elective or weight loss surgery, there's no doubt that people are doing that because of low cost and easier access."

One study published in the American Journal of Medicine estimated that fewer than 800,000 Americans traveled to other countries for medical care in 2007, but by 2017, more than 1 million did. More current estimates suggest that those numbers have continued to grow.

Video below: Americans are saving by traveling abroad for medical care

"People travel because there may be a long waiting time, wait lists or other reasons why they can't get treatment as quickly as they would like it. So they explore their options outside the United States to see what's available," said Elizabeth Ziemba, president of Medical Tourism Training, which provides training and accreditation to international health travel organizations.

Also, "price is a big issue in the United States. We know that the US health care system is incredibly expensive," she added. "Even for people with insurance, there may be high deductibles or out-of-pocket costs that are not covered by insurance, so that people will look based on price for what's available in other destinations."

The most common procedures that prompt medical tourism trips include dental care, surgery, cosmetic surgery, fertility treatments, organ and tissue transplants and cancer treatment, according to the CDC.

"With Mexico and Costa Rica, it's overwhelmingly dental and cosmetic surgery. However, certain countries are known for specialties. For example, in Singapore, stem cell and oncology is huge. In India, South India and Chennai Apollo hospitals does incredible work with hip and knee surgeries," Josef Woodman, founder of Patients Beyond Borders, an international health care consulting company, said.

"In Eastern Europe, a lot of people from the UK but also people from the United States travel to Hungary, Croatia and Turkey for everything from dental to light cosmetic surgery," he said.

Mexico is the second most popular destination for medical tourism globally, with an estimated 1.4 million to 3 million people coming into the country to take advantage of inexpensive treatment in 2020, according to Patients Beyond Borders.

Matamoros where officials said the four kidnapped Americans were found is "not considered a primary medical travel destination," Woodman said, "largely because there are no internationally accredited medical centers/specialty clinics there or in the immediate region."

Mexico City, Cancun and Tijuana are more frequented and reliable destinations in the country, Woodman said.

On average, Americans can save 40% to 60% across the most common major procedures received by medical tourists in Mexico, according to an analysis of 2020 health ministry data conducted by Patients Beyond Borders.

Woodman said that violence against medical tourists was extremely rare, but he added that "price shopping" searching for the cheapest location for a procedure is a "blueprint for trouble," namely substandard medical care.

Video below: U.S. Attorney General Merrick Garland offers remarks about the Americans who were kidnapped in Mexico.

Medical tourism can be dangerous, depending on the destination and the person's condition.

"There are the complexities of traveling if you have a medically complex situation. There are fit-to-fly rules. And your health care providers should take into consideration the impact of traveling if you have orthopedic injuries or issues," Ziemba said.

"The quality of care may be an unknown," she said. "It may be that the quality of care is not up to the standards that you would like. So there's a bit of an unknown there, and then the last thing I would say is, if something goes wrong, what's going to happen?"

Perez said he commonly manages complications from medical tourism in his practice.

"There are a lot of bad outcomes. There are a lot of infections and a lot of botched procedures gone wrong, and patients have to come back to the United States and then have a revision of the surgery," he said. "So it's really unfortunate."

Yet Ziemba added that there can be benefits to medical tourism, including that someone could receive a service that they need faster overseas than locally.

"And price: If you simply can't afford the out-of-pocket costs of health care in the United States, and assuming the risks involved, it may make much more sense for you financially to travel outside the United States," she said.

Medical tourism is not just for people traveling around the world. Many living along the U.S.-Mexico border, where access to health care can be scarce, cross into Mexico for care.

The Rio Grande Valley, at the southernmost point of Texas, is considered to be a medically underserved area. The region has some of the nation's highest rates of comorbidities, including obesity and diabetes, and one of the lowest physician-to-patient ratios.

There is a "dire need" for health care professionals along the border, Perez said.

"There are not as many doctors given our big and our growing population down here. So the demands on primary care doctors and specialists are very high because there are not enough of us for this population," he said. "So that's one reason why people end up going to Mexico to visit with physicians, because of easier access."

People interested in medical tourism can take some steps to help minimize their risk, the CDC says.

Those planning to travel to another country for medical care should see their health care provider or a travel medicine provider at least four to six weeks before the trip and get international travel health insurance that covers medical evacuation back to the United States.

The CDC advises taking copies of your medical records with you and checking the qualifications of the providers who will be overseeing your medical care. Also, make sure you can get any follow-up care you may need.

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Medical tourism to Mexico is on the rise, but it can come with risks - WMUR Manchester