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medicine

Lucas Jemeljanova, then aged 5, with his parents and sister a year before he was diagnosed with DIPG. (via Facebook)
Lucas Jemeljanova poses with his family a year before being diagnosed with cancer (Facebook)

It's a parent's worst nightmare: Taking your child to the doctor and receiving a life-changing diagnosis. It only adds to the heartbreak when they find out there may be no effective treatment at all, and that all they can do is hope for the best.

Few diagnoses strike fear in the heart of parents and doctors more than a cancer called diffuse intrinsic pontine glioma, or DIPG. Primarily found in children, DIPG is a highly aggressive brain tumor that is uniformly fatal, with less than 10 percent of children surviving longer than two years after diagnosis. The tumors grow fast and on extremely vital areas like the spine and brain stem, making them exceptionally hard to remove. Though young patients have been treated with radiation, chemotherapy, and surgeries, no one had ever been cured of the fatal cancer.

But for the first time ever, a 13-year-old boy from Belgium named Lucas Jemeljanova has beaten the odds.


cancer, childhood cancer, pediatric cancer, kids, medicine, healthcare, research, innovation, clinical trials, DIPG Various brain scans. Photo by National Cancer Institute on Unsplash

Diagnosed with DIPG at age six, Lucas’ doctor Jacques Grill told Lucas’ parents, Cedric and Olesja, that he was unlikely to live very long. Instead of giving up hope, Cedric and Olesja flew Lucas to France to participate in a clinical trial called BIOMEDE, which tested new potential drugs against DIPG.

Lucas was randomly assigned a medication called everolimus in the clinical trial, a chemotherapy drug that works by blocking a protein called mTOR. mTOR helps cancer cells divide and grow new blood vessels, while everolimus decreases blood supply to the tumor cells and stops cancer cells from reproducing. Everolimus, a tablet that’s taken once per day, has been approved in the UK and the US to treat cancers in the breast, kidneys, stomach, pancreas, and others—but until the BIOMEDE clinical trial, it had never before been used to treat DIPG.

cancer, childhood cancer, pediatric cancer, kids, medicine, healthcare, research, innovation, clinical trials, DIPG Lucas Jemeljanova poses with his mother (lesja Jemeljanova / Facebook)

Though doctors weren’t sure how Lucas would react to the medication, it quickly became clear that the results were good.

“Over a series of MRI scans, I watched as the tumor completely disappeared,” Grill said in an interview. Even more remarkably, the tumor has not returned since. Lucas, who is now thirteen, is considered officially cured of DIPG.

Even after the tumor was gone, Grill, who is the head of the Brain Tumor Program in the Department of Child and Teenage Oncology at Gustave Roussy cancer research hospital in Paris, was reluctant to stop Lucas’ treatments. Until about a year and a half ago, Lucas was still taking everolimus once every day.

“I didn’t know when to stop, or how, because there was no other reference in the world,” Grill said.

While Lucas is the only one in the clinical trial whose tumor has completely disappeared, seven other children have been considered “long responders” to everolimus, meaning their tumors have not progressed for more than three years after starting treatment.

cancer, childhood cancer, pediatric cancer, kids, medicine, healthcare, research, innovation, clinical trials, DIPG Lucas Jemeljanova with his mother (Facebook) (lesja Jemeljanova / Facebook)

So why did everolimus work so well for Lucas? Doctors think that an extremely rare genetic mutation in Lucas’ tumor “made its cells far more sensitive to the drug,” Grill said, while the drug worked well in other children because of the “biological peculiarities” of their tumors.

While everolimus is by no means a cure, the trial has provided real hope for parents and families of children diagnosed with DIPG. Doctors must now work to better understand why Lucas’ tumor responded so well to the drug and how they can replicate those results in tumor “organoids”—artificially-grown cells that resemble an organ. After that, said Marie-Anne Debily, a researcher in the BIOMEDE trial, “the next step will be to find a drug that works as well on tumor cells.”

A more recent clinical trial tested a new immunotherapy treatment on young DIPG patients and showed promising results. Many of the patients' tumors shrank and several participants saw functional improvements in their symptoms and day-to-day lives. But only one of the 11 patients has seen success that rivals Lucas' — a young man identified only as Drew, who has been thriving tumor-free for over four years after receiving treatment.

Once considered a definitive death sentence, there is real hope for the first time. But there's much more research and work to be done. Until then, however, Lucas’ doctors are thrilled.

“Lucas’ case offers real hope,” said Debily.

cancer, childhood cancer, pediatric cancer, kids, medicine, healthcare, research, innovation, clinical trials, DIPG Lucas Jemeljanova with his parents and sister (lesja Jemeljanova / Facebook)

This article originally appeared last year. It has been updated.


Innovation

7 mindblowing medical breakthroughs that could occur in our lifetime

One day soon, you might be able to grow new teeth. Seriously.

Canva Photos

My family has a history of Parkinson's Disease, so I always see the headlines that promise some major scientific breakthrough in regards to the disease based on small, lab-based experiments or trials run on animals. They give us a glimmer of hope but, far too often, these treatments never see the light of day. The road from promising idea to having a real treatment available for patients is extremely long and arduous. Most potential new medications and therapies don't survive.

But... things do sometimes happen! There are massive medical breakthroughs being achieved all the time. Sometimes it's a new discovery, sometimes it's a successful clinical trial, and sometimes it's the fruit of all that labor finally becoming publicly available to people who need it the most. For example, an RSV vaccine just became available in 2023 for the first time. A powerful non-opioid pain killer was approved in 2024. A new drug came out to treat schizophrenia that was completely different than anything that had come before. Progress is being made every single year.

In our lifetime, say the next five to 20 years, we could see unbelievable advancements in treating certain medical conditions. Here are a few pretty amazing potential developments that are on the horizon.

Scientist working in a laboratory setting. Photo by Adam Bezer on Unsplash

1. Growing new teeth

Unlike sharks, humans are known to grow only two sets of teeth, our baby teeth and permanent adult teeth. If one gets knocked out or lost due to decay or infection, you're out of luck!

But researchers say growth buds exist in our gums for a third set, and scientists in Japan have had success in activating them to grow naturally. It could be a major gamechanger that could one day replace dentures and prosthetics. The team has moved onto human trials and has a target date of having a drug ready by 2030.

2. Cancer vaccines

Medical scientists have made amazing strides in cancer research, especially in the field of vaccines.

Did you know that lung cancer is considered to be the most deadly of the bunch? A lung cancer vaccine could be available very soon. If a cancer vaccine sounds odd, think of it this way: Cancer can be treated by chemotherapy and radiation, but there's always a chance of it coming back. People who have survived cancer could theoretically take the vaccine and massively improve their chances of reoccurrence.

A similar vaccine is showing promising results in clinical trials for pancreatic cancer.

3. Better treatment for Alzheimers

Alzheimer's Disease is one of the most destructive and heartbreaking conditions imaginable. It's also extremely difficult to treat, with most currently available therapies and medications focusing on slowing or temporarily relieving certain symptoms.

One new avenue scientists are exploring is a vaccine that could target a protein called tau, which clumps together and tangles up crucial neurofibers that deliver information in the brain. One such medication aims to activate a patient's immune system and produce "a strong antibody response against both pTau and its harmful aggregated form." The drug was recently fast-tracked by the FDA and has shown promising results in human clinical trials.

Another relatively new development is the availability of monoclonal antibody treatment, which helps slow the progression of the disease. Currently, the treatments are not particularly cost-effective but may become moreso in the near future.


smiling woman in gray cardigan Photo by Tatiana Zanon on Unsplash

4. Breakthrough treatment for Huntington's Disease

Huntington's is a fatal central nervous system disorder that shuts down key brain functions in patients like speech, walking, and cognitive function. Some of the symptoms can be treated but, so far, little can be done to slow or stop the progression of the disease.

However, a new drug known as PTC518 was recently fast-tracked by the FDA due to promising results in clinical trials. It attacks mutant Huntington protein in blood cells and in cerebrospinal fluid and has been shown to improve symptoms of the disease in a 12-month trial run. What's especially exciting is that this drug attacks the disease itself rather than just treating symptoms at the surface level.

5. Targeted viruses that kill antibiotic-resistant bacteria

Bacteria is getting smarter and evolving. Antimicrobial resistance is a grave threat to the global population as well-known antibiotics become less effective against infection and new antibiotics are expensive and difficult to develop.

(If you've ever dealt with a staph infection, you know how scary it is when drugs just... don't work.)

This is where special viruses, called bacteriophages or just phages, come into play. These viruses selectively target and kill bacteria, and were actually first used about 100 years ago. Scientist are coming around to using them again. In select settings, they've proven to be incredibly effective against resistant infections, but will need more testing before they become widely available. However, it's definitely possible that this become a viable alternative to antibiotics in some cases in the next few decades!

6. Synthetic and bioprinted organs


human heart Photo by camilo jimenez on Unsplash

There are simply not enough donor organs going around for all the people who need a healthy liver, heart, or kidney.

Luckily, incredible strides have been made in creating artificial organs that can function like the real thing. A man in Australia just lived for 100 days with a titanium heart while he awaited a transplant! And bioprinting technology – quite literally 3D printing using real tissue and living cells — has the potential to create new working organs from scratch in the near future. Genetically modifying animal organs to function in humans temporarily or maybe even permanently has also come an extremely long way and is a process that will drastically improve in the coming years.

7. A one-pill cure for Hepatitis B

Hepatitis B is a potentially fatal infection of the liver that affects hundreds of millions of people worldwide. There is a safe and effective vaccine available to some people, but it's only preventative and won't treat active infections.

A research team in Israel recently discovered that a low dose of well-known chemotherapy drug, Curaxin-137, completely destroyed a crucial and mysterious protein that the Hepatitis virus depends on. The testing was done in a lab setting, so the next step is clinical trials. The team hopes that one day in the not-so-distant future, patients could take a single dose and completely eradicate the virus in their system. That's about as close to a miracle-cure as you can get!

Not all of these potential breakthroughs will come to fruition, but it's pretty amazing to dig into the advancements and discoveries that are being made every day. This list is just scratching the surface, to say nothing of robotic surgeons that will make major surgeries less invasive, new treatments for baldness, nanotechnologies that can deliver medicine to specific parts of the body, and more. Ultimately, good news is coming relatively soon for people who need it.


We know a lot, but we clearly don't know everything.

Ever watch a period piece movie or TV show where the well intentioned doctors used maggots in hopes of cleaning a wound? Or when lobotomies were the tried-and-true method of relieving mental disorders? We’ve come quite a long way in terms of medical progress since those days, but in other ways, we are just as much making our best guess about how the human body actually works as we were in the maggot days.

Even doctors can admit that many biological ins-and-outs remain a mystery, which can be frustrating as a patient when you’re told to take certain medications "because it helps” this or that ailment, without actually knowing why. But at the same time, you have to kind of marvel at how much there is yet to discover, even within us. It only further shows the need to continue investing in medical research, but that’s a different conversation.

Recently, several doctors online shared many things that we still don’t really know about the human body, and some of the answers were pretty surprising:

“I’m an anesthesiologist. We still don’t really know why inhaled volatile anesthetics like sevoflurane, the principal anesthetic agent used to maintain general anesthesia, work. We kind of have an idea of maybe how it happens, but really we don’t know. It’s commonly said in my field that whoever figures this out will win the next Nobel prize in medicine.”

doctor providing anesthesia to a patientWeird...but effective. Photo credit: Canva

That’s interesting. And now for something a tad grosser…

“We apparently don't know precisely how our bodies can distinguish gas from poop. We have some ideas, we know there are a ton of nerve endings in the area, but the precise mechanism of our bodies telling our brains ‘this is a fart, let loose’ isn't really understood. What blows my mind is, it's distinct enough that we even pass gas while asleep. That difference must be wired DEEP!”

a person holding their behind that's glowing red to signify gasWe don't know how our bodies know, but thank goodness they do. Photo credit: Canva

Quite a few answers pertained to some topics we might never actually solve.

“The Role of Our DNA: We’ve sequenced the human genome, but a large part of it remains unexplained.”

a hand holding a strand of DNASeems pretty important. Photo credit: Canva

“I’m a derm. We don’t know what exactly causes itching, like the molecular pathways for it. That’s why it can be so hard to find a good treatment when a patient comes in for itchy skin.”

a woman scratching her armTrial and error.Photo credit: Canva

“I'm a sleep specialist. While we do have some good theories about some of the functions of REM as far as how it affects the brain and health, we still don't fully understand the purpose of dreaming. Like, why do we dream at all and why do dreams have a narrative instead of random incomprehensible imagery? Unfortunately this is unlikely to even be solved..”

a woman sleepingMaybe our brains get bored while we're out and want to watch a self-made movie? Photo credit: Canva

There were a few mysteries that pertain specifically to women’s health. Historically, women's health research has been significantly underfunded and understudied, so this is fairly unsurprising.

“OBGYN here: we still don’t know exactly what makes labor start. We know all about the mechanics and physiology, but we don’t know what makes the average uterus say it’s ‘go time.’”

pregnant woman holding her stomach in pain When the body knows, it knows. Photo credit: Canva

“Apparently we know next to nothing about fibroids, which like 75% of women have at some point in their lives. That's great, considering that the largest one removed was 100 lbs- so not exactly a minor issue. There are theories about different hormones and what things put you at higher risk, but aside from having surgery to have your existing ones removed, there is basically no information on what you can to do prevent them from coming back.”

doctor presenting a model of a uterus while a woman sits on a medical chair in the backgroundIt's a big deal and we need to understand it. Photo credit: Canva

There was also talk of how our gut—how we process nutrition, and how that affects our mental health—is somewhat of a final frontier in medicine.

“We don't know the precise mechanism by which B12 deficiency causes nerve damage. We know that it happens, but not why.”

collection of foods around a sign that says, "Vitamin B12"B12, the final frontier.Photo credit: Canva

“Not MD but PhD, right now we are working on the connection between our intestinal microbiome and neuropsychiatric disease and brain aging. For instance, people with inflammatory bowel disease are more likely to develop dementia and experience co-morbid anxiety and depression, but we don't know why.”

“The Gut Microbiome: While it's well-known that the gut plays a huge role in digestion, researchers are discovering just how much our gut bacteria affect other parts of our health, like mood, immunity, and even brain function.”

diagram of the digestive system with a close up of gut bacteriaSome say we have two brains: in the head and in the gut.Photo credit: Canva

For those who have ever taken anti-anxiety medication…

“Benzodiazepines, BZD, are medications like Xanax and Valium. They produce anti-anxiety effects. And they have a very distinct chemical shape to fit into the BZD site in a group of five proteins. But we don’t know what is supposed to go there. Many medications are analogs of naturally binding molecules that we copy and then use to create an effect. The BZD site is for something, we just don’t know what.”

close up of a bunch of pillsPhoto credit: Canva

By and large, mental health is still the Wild Wild West for medical professionals, it seems.

“Doctor here. Off the top of my head, here's a few deceptively big ones:

  1. Psychiatry is still shockingly infantile in our understanding of human disorders. It's constantly in a state of flux, we don't understand a lot about the meds we currently use, and the diagnostic criteria for disorders still changes as we realize "hey maybe all these behaviors aren't the same source disorder". It's incredibly hard to diagnose when the criteria is largely based on self report and subjective observations.
  2. To a lesser degree than #1, neurology is still learning a lot. It's further because you can observe more objective findings in neuro than psych, but we still struggle a lot with how brains function."

a red cut out of a head with a tangle of string where the brain would be; a blue cut out of a head with a spiral of string where the brain should beAren't we all just brains trying to understand ourselves? Photo credit: Canva

And yet, another doctor writes:

“One of the few ABSOLUTES in medical science is that nobody born blind has ever developed schizophrenia.”

Goes to show, we might be in the age of information, but there is still so much of the unknown to marvel at. Hopefully it keeps us humble and curious.

Health

Impossible-sounding surgery cures blindness by implanting a patient's own tooth in their eye

"Tooth-in-eye" surgery sounds like something out of science fiction, but it permanently restores vision in 94% of patients.

Canva Photos

You have to see it to believe it.

About 12 million people in the United States live with significant visual impairment. About a million of those people live with blindness. Unfortunately, there is no known cure for blindness. Therapies exist to slow vision loss, and some vision loss can be improved with treatment, but complete vision loss has proved to be a very tough nut for scientists to crack, in part because there are so many different potential causes of blindness.

Medical researchers have and continue to look into lots of diverse options, including stem cell therapies, gene therapies, bionic eyes, and now...teeth. Yes, you read that right. Teeth.

A 33-year-old man recently became the first person in Canada to have their sight restored by a rare and outrageous-sounding procedure: Osteo-odonto-keratoprosthesis, or OOKP.

OOKP has been around since the 1960s and has been performed around the globe, but is still relatively rare overall. Invented by Professor Benedetto Strampelli, the procedure is better known by its easy-to-remember, and fairly gross, nickname: "Tooth-in-eye surgery." Why in God's name is it called that? Buckle up if you're squeamish. Here's how it works:

gif of man buckling seatbeltBuckle up; this is going to be wild. Giphy

First, a tooth is removed from the patient. A small rectangular section of the tooth is shaved off and a hole is drilled in the middle of it. Imagine cutting a small circle out of the middle of a sheet of paper.

Then, a small plastic lens is inserted into the hole, almost like a window covering or a makeshift camera lens. The section of tooth acts like a frame to hold the new lens.

OK, now for the really wild part: The rectangular shaving of tooth, complete with plastic lens, is then embedded into the patient's cheek in order to grow new tissue and blood vessels. It will stay there for around two months while the new tissue develops.

The tooth is then removed from the cheek and surgically embedded into the patient's eyeball, effectively replacing the damaged cornea. The new eye is pink and bloodshot in appearance, and a little bulbous, with only a small block hole as an iris. But, miraculously, it works!

The patient's own teeth are used to prevent the body rejecting foreign tissue or materials. Tooth-in-eye surgery only works for vision loss caused by corneal damage, or damage to the surface of the eyes. It won't heal or replace the optic nerve or retina, so it's not a miracle cure for all forms of blindness. It also comes with some risks, but when it works, it works. One study showed that 94% of successful cases still had good eyesight almost 30 years later, with some formerly blind patients seeing well enough to drive cars.

The Canadian man became blind at the age of 13 after a terrible autoimmune reaction to ibuprofen. After dozens of surgeries and therapies, OOKP was his last-ditch effort to permanently restore his sight. Other treatments had helped but his sight would fade away over time. For now, his story is a massive success that doctors are hoping will inspire others to give the controversial procedure a try.

The video below shows the procedure and its aftermath:


@garcidltth1

#popularscience #knowledge #fyp #tiktok

We have to keep funding bizarre, off-the-beaten-path scientific research.

This sounds like one of those wacky things you'd see in the news and say, "Why are we spending millions of dollars trying to see if we can grow new eyeballs from teeth?!" But without critical and creative research like this, we'd be no further in treating conditions like blindness. A version of OOKP was performed successfully in the United States at The University of Miami in 2009.

Amazing medical breakthroughs can come from the strangest places. Fascinating discoveries have been made by making people drink their own blood and monitoring the effects, studying the chemical makeup of goose poop, and looking for answers in tree bark, snake venom, and rat poison.

Cheers to Professor Benedetto Strampelli, and his weird but effective stroke of inspiration.