Jump to content

[Topic name broadened] Novel, potentially game-changing, advances in biomedicine


Recommended Posts

1 hour ago, Tillu said:

 

As with any industry there will be discrepancies and corrupt practices coming into light every now and then.

 

If doubting Thomases had their way we'll be still dieing and suffering from Malaria, Tuberculosis, Hepatitis, Polio etc.

 

Just within the last 100 years average life span has increased from 30 to 70 years world over. Infant mortality rates and maternal mortality rates have greatly plummeted.

 

The benefits of the medical research far outweigh whatever minor disadvantages we see. Yes we should have a sceptical mind with everything. But instead of ridiculing the poster be a little respectful.

I am not questioning the value of science but the downsides of changing building blocks of human and the effects on the human and next generation because of edited genes. Unless they do years of studies they shouldn’t rush like Covid 19 vaccine.

Edited by gattaca
Link to comment
Share on other sites

 

This guy pushed the clot shot. How is it working for you :winky: How many of you suffered debilitating diseases like long covid, loss smell taste, if you were lucky enough to not get blood clots. If you fell for it, you'll fall for this also.

This guy can't even get the windows to work on a machine properly but is talking about tampering with DNA to remove some disease that rarely affects anyone that you know. ok.

Unlike the usual, gates issues a disclaimer at the end. There may be "issues" with CRISPR :laugh: This is funny if it weren't sad. Don't try it folks. You may call me a luddite but I'm right on this

Link to comment
Share on other sites

3 hours ago, gattaca said:

I am not questioning the value of science but the downsides of changing building blocks of human and the effects on the human and next generation because of edited genes. Unless they do years of studies they shouldn’t rush like Covid 19 vaccine.

 

Yeah Science and Technology can be a double edged sword. Technology in the wrong hands can be very dangerous. The concerned authorities should enforce stringent regulations on who and how to use them. They should enforce rigorous trials and testing before they bring it to the masses.

Link to comment
Share on other sites

Meanwhile, gene editing technology continues to find cures for previously incurable diseases. The article linked below describes a cure using gene-editing technology for a disease that is described thus:

 

In a medical triumph, the US Food and Drug Administration on Monday approved a gene therapy that appears to trounce a rare, tragic disease that progressively steals children's ability to talk, move, and think, leading to a vegetative state and death. For those who begin to slip away in infancy, many die by age 5. But, with the new therapy, 37 children in an initial trial were all still alive at age 6. Most could still talk, walk on their own, and perform normally on IQ tests, which was unseen in untreated children. Some of the earliest children treated have now been followed for up to 12 years—and they continue to do well.

 

One can only hope that, after reading that paragraph, there is no one - regardless of political bent or religion or nationality - that will be against these technologies.

 

The big elephant in the room (as I have mentioned before in this thread) is cost. These therapies, while mind-blowingly successful, are also ridiculously expensive at this point in time.

 

https://arstechnica.com/science/2024/03/lifesaving-gene-therapy-for-kids-is-worlds-priciest-drug-at-4-25m/

 

Full article:

 

Quote

In a medical triumph, the US Food and Drug Administration on Monday approved a gene therapy that appears to trounce a rare, tragic disease that progressively steals children's ability to talk, move, and think, leading to a vegetative state and death. For those who begin to slip away in infancy, many die by age 5. But, with the new therapy, 37 children in an initial trial were all still alive at age 6. Most could still talk, walk on their own, and perform normally on IQ tests, which was unseen in untreated children. Some of the earliest children treated have now been followed for up to 12 years—and they continue to do well.

But, the triumph turned bittersweet today, Wednesday, as the company behind the therapy, Lenmeldy, set the price for the US market at $4.25 million, making it the most expensive drug in the world. The price is $310,000 higher than what experts calculated to be the maximum fair price for the lifesaving drug; the nonprofit Institute for Clinical and Economic Review, or ICER, gave a range last October of between $2.29 million to $3.94 million.

The price raises questions about whether state, federal, and private health insurance plans will be able to shoulder the costs. "Unless states have allocated appropriately for it, and looked at the drug pipeline, they may not be prepared for what could be significant cost spikes," Edwin Park, a research professor at the McCourt School of Public Health at Georgetown University, told CNN.

It's also unclear whether the drug can reach the children who need it in time. Lenmeldy must be given before symptoms develop or early on in symptom development in children. However, diagnosis of the rare genetic condition can be slow, and many children treated so far were identified because older siblings, now too old for treatment, developed the condition first.

Devastating disease

Stat, for instance, spoke with a mother whose 8-year-old daughter with the condition can no longer talk or move, has frequent seizures, and requires a feeding tube and 28 different medications. Meanwhile, the child's 3-year-old brother, who has the same genetic mutation, is a typical toddler—he was able to get the new treatment when he was six months old. To get it, the family flew him to Milan, Italy, where Lenmeldy was first developed. It was approved for use in Europe in 2021.

The condition Lenmeldy treats is called metachromatic leukodystrophy (MLD), which occurs in about 40 children in the US each year. MLD is caused by a mutation in the gene that codes for the enzyme arylsulfatase A (ARSA). Without this enzyme, the body can't break down sulfatides, a fatty substance that then builds up to toxic levels in the brain and peripheral nervous system. Sulfatides are essential components of myelin, the fatty insulation on nerve cells critical for quick transmission of electrical impulses. But, too many sulfatides lead to a loss of myelin, which gradually destroys myelin-producing cells and leads to nervous system damage.

Lenmeldy prevents that damage by giving the body a working copy of the ARSA gene. In a one-time infusion, patients are given a dose of their own blood stem cells that have been genetically engineered to contain a functional ARSA gene. Patients undergo chemotherapy to clear out their own stem cells from bone marrow so the genetically modified cells can replace them. The engineered stem cells then produce myeloid cells that travel around the body in the blood, producing ARSA enzymes that can halt progression of MLD.

It's unknown how long the therapy lasts, but it's clearly buying children time and giving them hope for a full, normal life.

“MLD is a devastating disease that profoundly affects the quality of life of patients and their families," Nicole Verdun, director of the FDA's Office of Therapeutic Products, said in a statement. "Advancements in treatment options offer hope for improved outcomes and the potential to positively influence the trajectory of disease progression."

It "has the potential to stop or slow the progression of this devastating childhood disease with a single treatment, particularly when administered prior to the onset of symptoms,” Bobby Gaspar, CEO of Lenmeldy's maker, Orchard Therapeutics, said in a statement Wednesday. "We are committed to enabling broad, expedient, and sustainable access to this important therapy for eligible patients with early-onset MLD in the US."

The company is working on expanding newborn screening to include tests for MLD to try to find children early, Orchard reported. Still, with such a rare condition, it's unclear if the pricey drug will be a moneymaker for the company. Stat notes that Orchard has previously abandoned four therapies for other rare genetic conditions because of the difficulty in meeting regulatory standards for essentially custom therapies and questions about whether health plans will pay the steep, multimillion-dollar prices. In April of last year, Belgium, Ireland, and the Netherlands walked away from price negotiations with the company, saying they couldn't come to an agreement on this "extremely expensive therapy."

 

 

 

 

Link to comment
Share on other sites

Posted (edited)
On 3/20/2024 at 11:11 PM, gattaca said:

I am not questioning the value of science but the downsides of changing building blocks of human and the effects on the human and next generation because of edited genes. Unless they do years of studies they shouldn’t rush like Covid 19 vaccine.

 

Our body has 30-40 trillion cells. Each one of them has 3.2 billion deoxyribonucleotide base pairs. Every time a cell divides to form new cells, there are an average of 3200 "errors" or mutations (1 in 1 million error rate). In other words, our DNA blueprint is constantly changing.

 

In the case of gene-editing techniques (and I have tried to explain it above for SCA), a single gene (out of 30,000 genes) is corrected in specific cells that give rise to red blood cells. None of the other cells of the body are modified. And this single change gets red blood cells to make normal hemoglobin and alleviates the symptoms of SCA. And they are not inherited because egg and sperm cells are not edited.

 

And one only has to hear the podcast featuring the lady whose SCA was cured to get behind this technology and its potential to cure many devastating diseases.

 

 

Edited by BacktoCricaddict
Link to comment
Share on other sites

On 3/15/2024 at 9:44 PM, jf1gp_1 said:

what we need is cure for diabetes. 

 

Great point. But one must be careful to distinguish the types of diabetes.

 

What you are talking about is Type 1 diabetes (T1D), which is an in-born, genetic, auto-immune disease in which insulin producing cells are attacked and killed by the patient's own immune cells. There is no cure so far - the patient has to get insulin injections lifelong. There are some cell-based therapies (like transplants) that have seen some success. But, some companies are looking at gene-editing technologies to cure this. But, in this case, it is very complex because you are trying to disarm immune cells that are trying to kill your own insulin-producing cells and that is not an easy task. It is not like single-mutation inborn errors like sickle-cell, beta-thalassemia or even cystic fibrosis where you  can repair a single gene in specific cell types.

 

What you and @gattaca alluded to as being managed by lifestyle changes is Type 2 diabetes (T2D). In most cases, this hits at advanced ages and can be managed (even reversed) by cutting refined grains, increasing fibre and protein intake, regular exercise to increase lean mass, and keeping weight down. I don't think this is a good target for gene editing. There are some other drugs available now (like Wegovy) that control appetite and thus help with weight-loss, but I feel they have more common side-effects than they are worth.

Link to comment
Share on other sites

5 hours ago, BacktoCricaddict said:

 

Great point. But one must be careful to distinguish the types of diabetes.

 

What you are talking about is Type 1 diabetes (T1D), which is an in-born, genetic, auto-immune disease in which insulin producing cells are attacked and killed by the patient's own immune cells. There is no cure so far - the patient has to get insulin injections lifelong. There are some cell-based therapies (like transplants) that have seen some success. But, some companies are looking at gene-editing technologies to cure this. But, in this case, it is very complex because you are trying to disarm immune cells that are trying to kill your own insulin-producing cells and that is not an easy task. It is not like single-mutation inborn errors like sickle-cell, beta-thalassemia or even cystic fibrosis where you  can repair a single gene in specific cell types.

 

What you and @gattaca alluded to as being managed by lifestyle changes is Type 2 diabetes (T2D). In most cases, this hits at advanced ages and can be managed (even reversed) by cutting refined grains, increasing fibre and protein intake, regular exercise to increase lean mass, and keeping weight down. I don't think this is a good target for gene editing. There are some other drugs available now (like Wegovy) that control appetite and thus help with weight-loss, but I feel they have more common side-effects than they are worth.

 

Lately we are hearing a lot about weight loss drugs like Ozempic. How safe are these drugs? Do they really work.

Link to comment
Share on other sites

17 hours ago, Tillu said:

 

Lately we are hearing a lot about weight loss drugs like Ozempic. How safe are these drugs? Do they really work.

Ozempic and Wegovy (and others like them) are basically injectable appetite suppressants. They were released as medications to control Type 2 diabetes by decreasing caloric intake and can be useful for people who cannot exercise for whatever reason (bad knees, busy lifestyle trying to make ends meet ...). In clinical trials and post-market analyses, they work really well for these purposes.

 

Two problems:

 

(1) They say it's a long-term/lifetime thing.  Injection every few months for the rest of your life?  Is it worth it? Cost??  Are there easier ways to control appetite? I don't know.

(2) They have become all the rage for cosmetic weight-loss - like actors and celebrities and even rich college students getting their doctors to prescribe it because they want to drop a few pounds. This has made it less accessible to people with diabetes who may really need it.

 

Anecdotally, I have a friend who had to have knee replacement surgery, had to lose weight quickly, and took Wegovy.  It made him miserable - vomiting etc. He stopped and said effit.

 

 

 

 

Link to comment
Share on other sites

Xenotransplants (organ transplants from other species to humans) - and sometimes even human-to-human transplants - can be very risky due to a violent immune rejection response in our bodies. For this reason, compatible kidney donors are so difficult to find and 1000s of patients die each year without the opportunity to get a transplant.  The following research may provide a solution:

--------------------------------------------------------------------------------------

https://www.massgeneral.org/news/press-release/worlds-first-genetically-edited-pig-kidney-transplant-into-living-recipient

 

Today, Massachusetts General Hospital (MGH), a founding member of the Mass General Brigham health care system, announced the world’s first successful transplant of a genetically-edited pig (porcine) kidney into a 62-year-old man living with end-stage kidney disease (ESKD). The patient, Mr. Richard ‘Rick’ Slayman of Weymouth, Mass., is recovering well at MGH and is expected to be discharged soon.  “The real hero today is the patient, Mr. Slayman, as the success of this pioneering surgery, once deemed unimaginable, would not have been possible without his courage and willingness to embark on a journey into uncharted medical territory.

 

“The success of this transplant is the culmination of efforts by thousands of scientists and physicians over several decades. We are privileged to have played a significant role in this milestone. Our hope is that this transplant approach will offer a lifeline to millions of patients worldwide who are suffering from kidney failure,” Kawai said.

 

The pig kidney was provided by eGenesis of Cambridge, Mass., from a pig donor that was genetically-edited using CRISPR-Cas9 technology to remove harmful pig genes and add certain human genes to improve its compatibility with humans. Additionally, scientists inactivated porcine endogenous retroviruses in the pig donor to eliminate any risk of infection in humans. Over the past five years, MGH and eGenesis have conducted extensive collaborative research, with the findings published in Nature in 2023.

----------------------------------------------------------------------------------------

 

Ethical concerns regarding taking another mammal's life to save a human's life may be raised (and will make for interesting debate), but if the patient was oneself or one's loved one, I'd imagine there would be no dilemma.

Link to comment
Share on other sites

hows the covid vaccine going? you have been pushing them a couple of years ago. We have seen them not just as something that failed protecting from a disease but also something really bad for your health. your back into newer and newer technologies in the same gene editing field. Have some shame

 

 

Link to comment
Share on other sites

Posted (edited)

This has nothing to do with gene editing, but it is another fascinating illustration of science and technology making people's lives better. This article is about a year old and more such examples of brain implants being used to help with severe neural dysfunction are emerging. But there is still a long, long way to go. As someone who had a very, very close family member experience quadriplegia due to an accident, it is gratifying to see that such developments in science and technology will help many in future generations!

 

https://www.sciencenews.org/article/brain-implant-paralysis-walk


A brain implant helped a man with paralysis walk more naturally

 

Quote

 

A system that restores communication between the brain and spine has enabled a man paralyzed by a spinal cord injury to regain near-natural walking ability.

Once the patient’s brain activity was decoded, the brain-spine interface took mere minutes to calibrate, after which the man reported natural-feeling control over movements.

 

He still needs crutches but can easily navigate ramps and steps, surpassing gains from previous treatments, researchers report May 24 in Nature.

 

“The results are consistent with what I’d hope would happen, which is encouraging,” says V. Reggie Edgerton, a physiologist at Rancho Los Amigos National Rehabilitation Center in Downey, Calif., who was not involved in the study. Still, in terms of treating spinal cord paralysis, he says, “we’re at the stage of the Wright brothers and flight.”

 

 

Edited by BacktoCricaddict
Link to comment
Share on other sites

6 hours ago, BacktoCricaddict said:

This has nothing to do with gene editing, but it is another fascinating illustration of science and technology making people's lives better. This article is about a year old and more such examples of brain implants being used to help with severe neural dysfunction are emerging. But there is still a long, long way to go. As someone who had a very, very close family member experience quadriplegia due to an accident, it is gratifying to see that such developments in science and technology will help many in future generations!

 

ScIeNCe and TeCHnOloGy making people's lives better? :lol: Why don't you say the same about those people who got their covid vaccine and boosters who are suffering now? I wonder why you keep at it when the whole thing is exposed as a fraud and you are back here with another snake oil. Are you being paid by someone in this industry?

 

The last line is anecdotal. Same thing was said when I tried to bring up someone who was getting affected by the covid shots. So same applies to you. As a research oriented person, stick to scientific journals and stop bringing your ancedotes

Link to comment
Share on other sites

Posted (edited)

https://health.economictimes.indiatimes.com/news/industry/prez-launches-indias-first-homegrown-car-t-cell-therapy-for-cancer-treatment-calls-it-new-hope/109036169

President Droupadi Murmu launches India's first fully indigenous CAR T-cell cancer immunotherapy! 

 

This is a heartwarming development. CAR T-cell cancer immunotherapy is the cutting edge in cancer treatment. Scientists precisely genetically engineer the patient's own immune cells to specifically recognize and destroy cancer cells. It has been shown to be successful in treating many different cancers, including extremely dangerous ones like multiple myeloma.

 

A common theme in these cutting-edge gene-based treatments is that one's own cells are altered and used. This minimizes (possibly eliminates) adverse immune responses, making them highly effective. Cost is a great barrier, but I am betting Indian companies and scientists can show the world how to make these work at lower costs thus improving accessibility.

 

 

Edited by BacktoCricaddict
Link to comment
Share on other sites

  • The title was changed to [Topic name broadened] Novel, potentially game-changing, advances in biomedicine

Still very early days, but if this holds up, it could be a game-changer in treating hard-to-treat cancers like pancreatic cancer (which killed Steve Jobs).

 

https://www.aacr.org/about-the-aacr/newsroom/news-releases/immune-response-to-investigational-rna-vaccine-for-pancreatic-cancer-continues-to-correlate-with-clinical-benefit/


 

Quote

 

An adjuvant treatment regimen that included autogene cevumeran, an investigational individualized neoantigen-specific mRNA vaccine, induced durable and functional T-cell responses that were associated with a reduced risk of disease recurrence in certain patients with resectable pancreatic cancer, according to three-year follow-up results reported at the American Association for Cancer Research (AACR) Annual Meeting 2024, held April 5-10.

Pancreatic cancer is a highly fatal disease, and even with surgery, only about 13% of patients are alive five years after diagnosis, explained Vinod Balachandran, MD, a surgical oncologist and member of the David M. Rubenstein Center for Pancreatic Cancer Research at Memorial Sloan Kettering Cancer Center. “Chemotherapy, radiation, targeted therapy, and current immunotherapies are also largely ineffective in pancreatic cancer, so new therapies are urgently needed for patients who face this deadly disease.”

 

“Our data indicate that autogene cevumeran can induce CD8+ T cells with significant longevity, substantial magnitude, and durable function,” summarized Balachandran. “The findings that individualized neoantigen-specific cancer vaccines can induce a robust immune response that correlates with delayed disease recurrence continues to support these vaccines as an encouraging therapeutic approach for pancreatic cancer.”

He added that an ongoing randomized phase II clinical trial will compare the efficacy and safety of autogene cevumeran in combination with atezolizumab and FOLFIRINOX with standard-of-care FOLFIRINOX in patients with resectable pancreatic cancer.

 

A limitation of the study was the small sample size and its single-arm design.

 

 

 

 

Link to comment
Share on other sites

39 minutes ago, BacktoCricaddict said:

Still very early days, but if this holds up, it could be a game-changer in treating hard-to-treat cancers like pancreatic cancer (which killed Steve Jobs).

 

https://www.aacr.org/about-the-aacr/newsroom/news-releases/immune-response-to-investigational-rna-vaccine-for-pancreatic-cancer-continues-to-correlate-with-clinical-benefit/


 

 

 

 

 

It still uses mRNA vaccine. How did the recent covid mRNA vaccine work out? You can change posts as many times as you like. But the situation still remains and you have to answer for it. Nobody would want to be guinea pigs to gene therapy. Everyone knows about sCiEnCe and their connections to big pharma

 

 

In the OP, you say you can watch npr because it is sCiEnCe not politics right. sCiEnCe as shown in mainstream media IS political chicanery as is shown in the coviid vaccine effects. Also I suggest those who watch this thread to also find out who runs npr and are their any political interests behind it.

Link to comment
Share on other sites

×
×
  • Create New...