Approval of first gene editing to treat Sickle Cell

[Aseahawkfan]

A pretty amazing medical technology was approved on 12-8-2023. The first of the CRSPR Tech cures is hitting the market. These are gene editing therapies. These are possibly a full cure for sickle cell, not just continuous treatment.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease#:~:text=Today%2C%20the%20U.S.%20Food%20and,patients%2012%20years%20and%20older.

This is a truly incredible advancement in medtech. The dawn of the ability to rewrite genes to cure disease.

This type of advancement always amazes me. This technology is going to shift the world like AI or electricity or any of the other great inventions that changed the world.

[RiverDog]

Incredible breakthrough. Now, their next task will be gaining the confidence and trust of the African American and Hispanic communities. There's so much disinformation whacky conspiracy theories out there, and I suspect that they'll be more rumors and speculation that will start circulating about this one. The covid vaccines showed us how much mistrust and lack of understanding exists, particularly in the minority communities.

This could be a revolutionary break through as it could lead to cures or treatment for other genetic diseases that affect more people than sickle cell, like Alzheimer's, cystic fibrosus, etc.

This is a good example why I do not want to see drug prices regulated and profits of the evil "Big Pharma" restricted as there has to be a healthy profit motive in order to encourage companies to plop the millions of dollars of research and development into new medicines like this one. They have to make plenty of money on the drugs that successfully make it to the market to cover the costs of those that don't. These drugs are being developed by companies based in this country:

Over the past half century, the United States has been the birthplace of the majority of the world’s biomedical innovations.[1] Despite a global slowdown in the development of new medical interventions, due to the scientific shift towards more complex biologic treatments, innovation in the U.S. has remained relatively steady thanks to strong financial incentives to invest in research and development (R&D).

https://www.americanactionforum.org/wee ... s-country/

[c_hawkbob]

It's not science's job to get people to trust the science. I can understand the hesitancy but all medical science can do is the actual science. I can't think of anyone whose kid has sickle cell that isn't willing to try anything to help them though.

[RiverDog]

It's not science's job to get people to trust the science. I can understand the hesitancy but all medical science can do is the actual science. I can't think of anyone whose kid has sickle cell that isn't willing to try anything to help them though.

I agree with your first two sentences, but not sure about the 3rd. I don't know anyone with a kid who has sickle cell so I really can't comment. But the hesitation and mistrust of the government by blacks and other minorities is real and must be overcome if this and other treatments are to be successful.

Yet I find myself teetering between excitement and skepticism (about the new drug). I am thrilled that research for sickle cell disease treatments and possible cures has blossomed. However, I worry that the celebration around this approval is ignoring a significant barrier: a historic legacy of medical mistrust.

The Tuskegee Syphilis Study and the story of Henrietta Lacks are haunting chapters in the history of medical research. But as Harriet Washington’s excellent book “Medical Apartheid” describes, these are not the only historical medical traumas affecting the Black community’s relationship with health care, the drug industry, and providers alike. The unethical and inhumane treatment of our ancestors has sown seeds of doubt and mistrust that persist to this day, with three-quarters of Black Americans saying they are familiar with the misconduct in the Tuskegee Study and that it contributes to a collective hesitancy when it comes to embracing medical advancements. This backdrop cannot be ignored as we witness the rapid development and commercialization of gene therapies for sickle cell, a disease that, in the U.S., primarily affects Black people. One study found that in 2016-2018, more than 93% of those with sickle cell who were hospitalized were Black.

https://www.statnews.com/2023/12/08/cas ... -patients/

One of the things we learned from the covid vaccines is that there were a number of people were willing to take the risk for themselves but unwilling to expose their kids to that same risk, so I'm not sure if a parent with a kid with sickle cell will consent to giving their kid this drug.

[Aseahawkfan]

Incredible breakthrough. Now, their next task will be gaining the confidence and trust of the African American and Hispanic communities. There's so much disinformation whacky conspiracy theories out there, and I suspect that they'll be more rumors and speculation that will start circulating about this one. The covid vaccines showed us how much mistrust and lack of understanding exists, particularly in the minority communities.

This could be a revolutionary break through as it could lead to cures or treatment for other genetic diseases that affect more people than sickle cell, like Alzheimer's, cystic fibrosus, etc.

This is a good example why I do not want to see drug prices regulated and profits of the evil "Big Pharma" restricted as there has to be a healthy profit motive in order to encourage companies to plop the millions of dollars of research and development into new medicines like this one. They have to make plenty of money on the drugs that successfully make it to the market to cover the costs of those that don't. These drugs are being developed by companies based in this country:

Over the past half century, the United States has been the birthplace of the majority of the world’s biomedical innovations.[1] Despite a global slowdown in the development of new medical interventions, due to the scientific shift towards more complex biologic treatments, innovation in the U.S. has remained relatively steady thanks to strong financial incentives to invest in research and development (R&D).

https://www.americanactionforum.org/wee ... s-country/

American medical system is already regulated, badly. It's terrible right now. Sorry, I don't agree. Companies would still produce cures. Rich people and their kids get sick too. Money won't stop.

The reason America has been the big cure place is we may have a s*** lower education system, but we have an amazing university system. The CRSPR tech was developed in universities around the world. Many of these cures you tout as American made were developed around the world. They were commercialized in America because the American system pays the most money. The cures still make it around the world.

The two women that received the Nobel Price for gene editing. Both worked in many public universities and laboratories around the world.

https://en.wikipedia.org/wiki/Emmanuelle_Charpentier

https://en.wikipedia.org/wiki/Jennifer_Doudna

You are basically advocating that people die if they can't afford these cures. That some person with a child with sickle cell should watch their child die or suffer if they can't afford this cure because you want an unregulated system, when you have little proof that the unregulated system we have now is what brings about these cures. Really, it just brings about immense profits for a handful of companies.

The cures are developed by scientists and organizations around the world. Europe is a huge place for cures to be developed as well. Cures depend less on how much money is circulating and more on how good your upper level university system is.

Even when one studies that chart, it doesn't take into account that the contributions come from a worldwide collective and that the United States is often taking credit for developments from a worldwide investment in research that gets monetized in America, often to the detriment of Americans who have trouble affording advanced therapies and medical care due to their terrible insurance system and inefficient, problematic regulation.

Sorry man, Sweden or German-style medicine is a far better way to go. Their people get better or at least equivalent care without the insane insurance system we use in America. I hope for regulation. I'm not happy that the Democrats have positioned themselves to be so crazy that they can't get anything done because they're busy worrying about people being misgendered, overselling environmental Armageddon, and defunding the police instead of coming up with a well-designed personal, portable insurance system that would help working Americans better afford medical care. And the Republicans have zero interest in better medical care for Americans. They would rather let the wealthy make their dough, get their kids fixed, and then let the working class go bankrupt getting sick.

I know this is another one of those subjects we'll never agree on. I would vote for a candidate that pushed through a Swedish-style medical system. Swedish has tons of billionaires and wealthy people. They are a well run, efficient country supporting their working people far better than America.

I invest in biotech quite a bit. Biotech companies from all over the world and often multinationals employing scientists from around the world. Medicine is a global business and Americans are getting the short end of the stick when it comes to health insurance.

[RiverDog]

The medicines were developed around the world, but they're being marketed in this country. If not for the financial incentive from the sales of the drugs in the United States, there's no way a company, almost all of which are publicly traded, is going to plop down the hundreds of millions of dollars required to finance their R&D departments.