UC San Diego Researchers Receive Close to $10M from California … – University of California San Diego

Two researchers at the University of California San Diego received close to $10 million in grants from the California Institute of Regenerative Medicine, the agency announced.

Eric Adler, MD, cardiologist and director of the Strauss-Wilson Center for Cardiomyopathy at UC San Diego Health, received $5.2 million to advance his research in modified stem cells to help treat Danon Disease. Danon is a rare condition, which, when left untreated, results in death as early as age 20.

Karen Christman, a professor in the Shu Chien-Gene Lay Department of Bioengineering at UC San Diego, received $4.6 million to advance her work on biomaterials that can repair damage to muscle after a heart attack. The biomaterials her research team developed can be injected into the bloodstream when a patient is in the catheterization lab undergoing an angioplasty and stent placement.

The goal of CIRMs translational program is to support promising stem cell-based or gene projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Those can include therapeutic candidates, diagnostic methods or devices and novel tools that address critical bottlenecks in research.

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UC San Diego Researchers Receive Close to $10M from California ... - University of California San Diego

60 years after its first organ transplant, Mayo Clinic looks to the future – Star Tribune

ROCHESTER - Sheila Maines didn't know if she'd live long enough to get a new kidney.

The 58-year-old Oklahoma woman felt crushed after doctors in that state refused her for a transplant, citing her medical history and a number of afflictions including lupus. They recommended the Mayo Clinic as her last hope.

She entered treatment at Mayo in March, where doctors told her with certainty she qualified for a transplant operation. Maines knew she could wait up to six years for a donor, but a 2 a.m. call on Nov. 18 and a hurried flight to Rochester led to surgery at noon that day.

Maines spent the last week recovering at Gift of Life Transplant House in Rochester. "It's a godsend," she said, choking up.

Saturday marks the 60th anniversary of the first kidney transplant at Mayo Clinic, and almost 70 years since the first long-term successful kidney transplant. Medical technology has advanced greatly since Mayo's first transplant, but researchers there say U.S. patients still face too many hurdles to receiving an organ transplant.

Wait lists are too long. Operations aren't successful enough. And too many new organs fail people in need. Mayo researchers hope several key projects will contribute to ongoing research around the globe to improve organ transplantation.

"Our primary goal is making sure more patients can access transplants ... and developing new treatments to address all these problems," said Dr. Julie Heimbach, head of Mayo's transplant center in Rochester.

More than 150,000 people received an organ transplant worldwide last year, according to data from the World Health Organization. In the U.S., the nonprofit United Network for Organ Sharing tracked almost 43,000 organ transplants in 2022. The majority of transplant organs come from deceased donors.

Despite record-setting numbers of organ transplants across the board, there aren't enough supplies to go around. About 3 in 10 donated kidneys go unused each year, according to the National Kidney Foundation, either from miscommunication between medical systems or long transportation times.

Patients often can't wait. About 1 in 4 or 5 patients are removed from organ transplant wait lists across the U.S. either because they've died or become too sick for successful surgery, Heimbach said.

Medical researchers across the globe are working on potential solutions, from using pig organs for transplants to 3-D printing makeshift organs and, maybe one day, growing human organs from scratch.

Mayo researchers say they're focused on several new projects that could bear results sooner.

Mayo performed its first robot-operated kidney transplant last month, following similar types of machine-assisted surgeries across the country. Mayo is also exploring how to use a type of double surgery for weight loss and liver transplants for kidney surgeries by far the most prevalent organ in need among ailing patients. And a study started at the beginning of the year could help patients better accept new organs and cut down on chronic organ failure.

That study involves using a type of stem cell to mimic the body's natural immunity responses, quelling rejection from a person's immune system. It's still in the early stages and isn't ready for human trials, but Mayo officials say the idea is promising based on previous research efforts.

"We know there is a good safety profile," said Dr. Timucin Taner, the study's lead researcher. "We're very hopeful that this will be a better option for a lot of patients."

Human trials could begin in 2024.

These kinds of studies are a far cry from the first long-term successful kidney transplant, done in Boston in 1954. Doctors there used identical twins in the surgery sterilization and anti-rejection drugs hadn't yet advanced to the point where patients could accept new organs, so the doctors thought a kidney donated from a twin would work better.

Mayo made its own history on Nov. 25, 1963, where doctors performed a transplant using a solid kidney from a live donor. Mayo's achievement was overshadowed by the news of the day: the aftermath of President John F. Kennedy's assassination.

Retired Mayo surgeon Dr. Sylvester Sterioff noted that transplantation had a 35% to 50% success rate until anti-rejection drugs got much better in the 1980s. For Sterioff, progress can't come fast enough.

"There are 100,000 people in the U.S. waiting for organs," Sterioff said. "Some of them will die while waiting."

Most patients wait three to five years for an organ. Others, like Maines, are lucky. She's had kidney issues for about 25 years because of an autoimmune disorder. The pain, combined with other issues, led her to quit her job as a medical assistant about eight years ago.

There were days she was so tired, she simply couldn't get out of bed or off the couch.

"I couldn't do anything," she said. "I couldn't wash a dish."

Before her surgery, her kidneys were operating at only about 20% of capacity. Maines faced dialysis or worse problems until last week, when a deceased donor's kidney proved a "perfect match," according to her doctors.

Now she can't praise her doctors enough.

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60 years after its first organ transplant, Mayo Clinic looks to the future - Star Tribune

Lab-Grown Brain Blood Vessels Show New Ways to Prevent Stroke … – HealthDay

MONDAY, Nov. 20, 2023 (HealthDay News) -- Lab-grown blood vessels are providing new insight into how damage to the tiny vessels in the brain can cause them to leak, contributing to dementia and stroke.

Even better, this research has identified a drug target that could plug these leaks and potentially reduce a persons risk of brain-damaging blood vessel leaks.

Antibiotic and anti-cancer drugs that inhibit a class of biochemical called metalloproteinases (MMPs) reversed damage occurring in the lab-grown blood vessels and stopped leakages.

These particular drugs come with potentially significant side effects, so wouldnt in themselves be viable to treat small vessel disease, said study author Dr. Alessandra Granata, of the department of clinical neurosciences at the University of Cambridge in England.

But they show that in theory, targeting MMPs could stop the disease, Granata added in a university news release. Our model could be scaled up relatively easily to test the viability of future potential drugs.

Cerebral small vessel disease (SVD) contributes to almost half (45%) of dementia cases worldwide, researchers said in background notes.

It is also responsible for about one in five (20%) ischemic strokes, which occur when a blood clot blocks blood flow to the brain. Most cases are associated with chronic illnesses like high blood pressure and type 2 diabetes, and they typically affect people in middle age.

For this study, Cambridge researchers gathered cells from skin biopsies of patients with a rare genetic form of small vessel disease, which is caused by a mutation in a gene called COL4.

The research team reprogrammed the skin cells into stem cells, which have the capacity to develop into nearly any type of cell within the body.

They then used these stem cells to generate brain blood vessels, creating a model that mimics the defects seen in patients with small vessel disease.

Despite the number of people affected worldwide by small vessel disease, we have little in the way of treatments because we dont fully understand what damages the blood vessels and causes the disease, Granata explained.

Most of what we know about the underlying causes tends to come from animal studies, but they are limited in what they can tell us, she noted. Thats why we turned to stem cells to generate cells of the brain blood vessels and create a disease model in a dish that mimics what we see in patients.

Blood vessels are built around a scaffolding called an extracellular matrix, which lines and supports the tiny vessels in the brain. The COL4 gene is important for the health of this matrix.

Researchers found that disruption of this matrix leads to small blood vessels becoming leaky.

Further, researchers identified MMPs as playing a key role in this damage. MMPs typically are important for maintaining the matrix, but if too many are produced they can damage the structure.

The new study was published Nov. 16 in the journal Stem Cell Reports.

More information

The Cleveland Clinic has more about cerebral small vessel disease.

SOURCE: University of Cambridge, news release, Nov. 16, 2023

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Lab-Grown Brain Blood Vessels Show New Ways to Prevent Stroke ... - HealthDay

Getting a Transformative, Low-Priced Hair Transplant in Turkey – OK!

Nov. 24 2023, Published 2:20 a.m. ET

Getting a hair transplant in Turkey has become so popular that the buzzword has reached nearly every household worldwide. Everyone knows hair transplants are a bargain in Turkey, helping people restore their youthful appearance and confidence.

The question is, are they worth it? Is there a catch, or do you genuinely get high-quality, natural-looking results at a fraction of the cost?

Weve dug deeper into the matter and were pleasantly surprised. Learn about hair transplant costs below and discover one reputable Turkish clinic where men and women can restore their hair without paying a small fortune.

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Medart Hair is a renowned hair clinic in Istanbul, Turkey, with a highly-skilled medical team of surgeons, doctors, anesthesiologists, and other physicians. They had earned their MDs and undergone extensive training across Turkey and beyond before assembling at the clinic to change peoples lives. Over 15,000 successful hair transplant operations highlight their achievements.

Dr Tuna Tantan Williams is the clinics lead hair transplant surgeon, holding an MD from the Faculty of Medicine at Tbilisi State University.

Besides hair transplants and dermatological applications, she has a pharmaceutical background and combines her experience and expertise to provide the best treatments. She also has certifications for regenerative stem cell surgery and mesotherapy.

Medart Hair is famous as a DHI hair transplant clinic in Turkey. Direct Hair Implantation (DHI) is the most cutting-edge hair restoration method (more on that shortly). It does offer other treatments, but it stands out for this one.

Another feature differentiating Medart Hair from others is personalised treatments. Everyone says they tailor hair transplants to patients needs, but this clinic takes it one step further.

When performing a physical exam during an initial consultation, its experts use micro cameras to uncover potential hair loss and devise a prevention plan accordingly. Therefore, they address the current problem and ensure it doesnt worsen in the future.

Hair transplant surgery costs an arm and a leg in the UK. Do you want to transplant 3,000 hair grafts? Ensure you have 7,50015,000 in your bank account. How about 4,000 grafts? Prepare to pay 10,00020,000 or up to 30,000 at some high-end clinics.

Who knew restoring your hair would set you back that much money? The price depends on many factors, including the number of grafts, the hair transplantation technique, the surgeons expertise, and the hair transplant area. Still, its too much for many, who eventually turn to Turkey to save thousands of pounds.

Turkish hair transplants are unbelievably more affordable. They cost between 1,500 and 6,700, although most treatments dont surpass 3,000. Thats only 10% of the highest price tag in the UK.

To answer your most burning question: theres no catch. Turkish hair transplant specialists are famous for exceptional skills, a thirst for innovation, and outstanding results. Thats why Turkey is a cosmetic surgery hub. However, it has a weak economy and a devalued currency, ultimately impacting the prices.

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Now that you know how much you can save with a hair transplant in Istanbul, its time to see what you can get. Spoiler alert: it isnt only the surgery.

Turkish hair clinics, including Medart Hair, offer all-inclusive deals. Astronomical hospital fees and other charges awaiting you in post-op? Forget about it. You pay for your chosen package, and thats it.

Here are hair transplant packages at Medart Hair:

Initial online consultation with hair analysis;

34 nights at a five-star hotel;

Private transfers (airport-hotel-clinic);

Language interpretation for international patients;

Physical examination with micro cameras before the surgery;

Pre-op blood work;

Hair transplant procedure under local anesthesia;

Dressing removal one day after the hair transplant;

Professional hair wash the following day;

Aftercare meds and hair care kit;

At least 12-month follow-up meetings.

These packages differ slightly among clinics, but most include hotel accommodation, transfer, the procedure, a blood test, post-op medications, hair products, and follow-ups varying in length. Check before scheduling the surgery.

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Medart Hair specializes in hair transplants, including female treatments and those without shaving the head. However, you can also get fuller eyebrows or a thicker, more defined beard with a hair transplant at this clinic.

Depending on the area and desired hair density, the clinics experts use the FUE (Follicular Unit Extraction) method or innovative FUE-based techniques. The most advanced include sapphire FUE and DHI.

A traditional FUE hair transplant involves making tiny incisions in the donor area with a steel-blade instrument, extracting follicular units, and opening channels in the recipient site to insert the harvested grafts.

A sapphire hair transplant replaces steel with sapphire for more precision. Its sharper, smoother, and more durable.

DHI removes incisions from the process, reducing trauma and hair damage. It requires multiple single-use Choi pensimplanters featuring a hollow needle, a cylindrical tube for storing hair, forceps for individual hair follicle extraction, and a plunger for direct implantation.

After sifting through patient reviews, we can confidently say you can get the best DHI hair transplant Turkey offers at Medart Hair.

A botched hair transplant procedure can cause more hair loss than a patient initially dealt with, among other more severe complications. Weve encountered many such instances.

However, you dont have to worry about complications with esteemed clinics like Medart Hair. Its professionals provide natural-looking results. Their patients reviews and before and after photos stand as an irrefutable testament to their medical prowess.

You can see hair growth within 12 months because it takes time for transplanted hair to stimulate natural hair. Some of it might shed initially, but thats normal.

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Hair transplantation doesnt need to break the bank. Undergoing the procedure in the UK might, but youll save thousands of pounds if you opt for Turkey.

Medart Hair is a brilliant choice if youre looking for a quality hair transplant clinic in Turkey. We recommend scheduling a free consultation to see if it suits your needs.

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Getting a Transformative, Low-Priced Hair Transplant in Turkey - OK!

Health Beat: A possible solution for sickle cell disease – 69News WFMZ-TV

CLEVELAND, Oh. - Danielle Lees adventurous spirt shines through, but behind the smiles of this aspiring actress is an unimaginable pain.

I went from like screaming, crying, please don't let me die to I just wanted the pain to be over. It is very sharp pain most of the time, to the point where holding my cell phone can hurt, Danielle painfully recalled.

Danielle was diagnosed before birth with sickle cell disease, where abnormal hemoglobin makes red blood cells rigid and shaped like sickles. These cells die early, leading to a chronic shortage of healthy red blood cells which are essential for carrying oxygen throughout the body.

The blood, basically, doesnt go to where it should be. So, the oxygen doesn't go to potential parts of the body, explained Dr. Rabi Hanna, pediatric oncologist at the Cleveland Clinic.

Sickle cell causes severe pain in the bones and can impact a patients heart, lungs, eyes and even cause strokes.

Dr. Hanna says medications can relieve some of the symptoms. Chemo also helps. Bone marrow transplants work when theyre not rejected.

But now, a one-time gene editing cell therapy is able to modify a patients own blood-forming stem cells to correct the mutation responsible for sickle cell disease.

We attach the patient to apheresis machine that's able to separate the stem cell from the red blood cells from the plasma, Dr. Hanna said.

Once the stem cells are collected and sent to the lab for gene editing, patients undergo chemo to destroy their remaining bone marrow, then the edited stem cells are infused back into their body.

The cells, they can go to their bone marrow and they start to build their house that will produce new white blood cells, new red blood cells, Dr. Hanna further explained.

Results have shown new white blood cells in patients at four weeks with no severe adverse effects.

The patients have also been free of sickle cell diseases pain attacks for an entire year. Danielle was one of the first in the CRISPR gene editing clinical trial.

I have so much more energy, thank God, Danielle said with relief.

The average life of a sickle cell patient is mid-forties. Doctors hope the CRISPR gene editing technology will change that and allow patients to live a long, pain-free life.

The Ruby clinical trial aims to enroll 40 more patients ages 18 to 50 with severe sickle cell disease.

More information is available at clinicaltrials.gov

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Health Beat: A possible solution for sickle cell disease - 69News WFMZ-TV

Music To Sit on The Sidelines To – Ransom Note

Drama has been curating the Music To Cut Hair To column on Ransom Note for some time now, and just two short years ago, this shimmering star was diagnosed with Multiple Sclerosis. Since then, her life has changed dramatically with a capital D.

On the 3rd of December Londons club scene comes together for Dance For Drama, an event to drive the fundraiser to raise 60K for Victoria to get some pioneering, life-changing, stem cell treatment in a state of the art clinic in Mexico.

Where are you this moment Drama and how are you spending the day?

Im at home in Earls Court. I spend pretty much every day managing this condition and its restrictions on my life.

How much has life changed?

Everything has changed. I still cant get my head round it. I never thought about my ability before.

What has been the hardest part for you?

Not being able to walk has been the hardest part.

What is MS exactly?

Multiple Sclerosis essentially, is your body attacking itself. It affects the brain and nerves.

What are the daily challenges you face?

Seeing, walking, no driving, slurred voice, standing, falling over, prepping for the smallest tasks as my fatigue is off the charts.

What is the prognosis of this disease without treatment?

Having HSCT should put the disease into remission, without it, Im facing a future that is hard work, probably a wheelchair, loss of my independence.

How have you been passing the days?

I read books and listen to podcasts. I havent been able to listen to music, not sure if it is the wrong PH balance or a trigger of a much- missed previous life.

What do you miss most about your life?

EVERYTHING.

What is your next step in your war on MS?

Ive spent the last two years surrounding myself with other MS sufferers. It has changed my view on life with a chronic illness. Its hard work making yourself heard and its very expensive. Somethings can help you to manage but there is no cure.MS is totally individual to each person.

When are you going to Mexico and what will happen while youre there?

I leave on the 6th of January. I spend a month in hospital receiving the stem cell treatment. I will return to the UK one month later to isolate for 2-3 months to give my immunity time to grow again.

Haematopoietic Stem cell transplantation is a chemotherapy treatment for MS that aims to reset the immune system by wiping it out and regrowing it, using the patients own stem cells. The idea is that it stops the immune system from attacking the protective layer around the nerves.

What do you hope to happen after the treatment?

After seeing the fantastic results, I hope to walk again unaided. Lets start small.

Tell us a bit about the funds you need to raise?

I need to raise 60k. Ive had to borrow the money. Not something I am comfortable with, but this will be my last stab at life.

The surgery is quite major. If you want to understand more watch Discovering Selma documentary.

Tell us about the fundraiser?

3rd December is the day for Dance for Drama at the Star of Liverpool St. My Fundraising Team have held my hand as being centre of attention is the furthest thing for my mind at the moment.

Who is on the line up?

Justin Robertson, Sophie Lloyd, Terry Farley, Dani Moore (Crazy P) Heidi, PBR Streetgang, Nancy Noise, Steve Lee, Lisa Loud, Stu Patterson, Tayo, Simon & Antony Phonica. Dave Jarvis, Orin Afronaut, Andy Taylor and the Size Doesnt Matter Crew, the list goes on and is epic. Not forgetting one of my chief Fundraisers Dave Harvey who has a special place in my heart.

What can we do to help?

Dig deep if you can. Every little counts. We have some really great things coming, a raffle, an art auction, the disco. Debbie Smith has offered to take over a karaoke room and you can pay her to get off. Ha!

There is also a raffle so if anyone has anything to throw into the tombola, please contact Rob Star rob@electricstarpubs.co.uk

Whats your message others with MS?

Thank you for welcoming me with open arms. I was told I am part of a gang that I didnt want to be part of. I am never going to offer any advice, as I know too well how awkward that be sometimes. But know I will never let go of your hand, and I will always have time for you. I have met some truly inspirational people.

What keeps you fighting?

The strength that I already have. I never fully acknowledged it before. My closest friends for picking me up when I am down. Family. And Benny.

Please visit the Crowdfunder HERE.

Below is a collection of music curated by Drama:

Excerpt from:
Music To Sit on The Sidelines To - Ransom Note

KYV-101 found safe, effective for woman with severe gMG – Myasthenia Gravis News

Kyverna Therapeutics investigational cell therapy KYV-101 safely and effectively improved muscle strength and reduced fatigue in a woman with severe, hard-to-treat generalized myasthenia gravis (gMG), according to a case report.

The patient was treated based on an individual case evaluation and outside of a clinical trial.

This groundbreaking case report rewards and reinforces our commitment to provide potentially paradigm-shifting therapeutic options to patients suffering from autoimmune diseases, Peter Maag, PhD, Kyvernas CEO, said in a company press release. We want to commend patients and their medical care teams that are helping advance the field of treatment options for B cell-driven autoimmune diseases.

After receiving the green light from the U.S. Food and Drug Administration last week, the company will launch a multicenter Phase 2 clinical trial called KYSA-6 to test the therapy in MG patients.

The case study, Anti-CD19 CAR T cells for refractory myasthenia gravis, was published in the journal The Lancet Neurology.

In MG, self-reactive antibodies disrupt the communication between nerves and muscles, leading to muscle weakness and other MG symptoms. Antibodies are proteins produced by immune B-cells to help fight infections, but they can also mistakenly target healthy tissues and drive autoimmune disorders like MG.

KYV-101 is a CAR T-cell therapy designed to destroy such disease-causing B-cells by targeting CD19, a cell surface protein found at high levels in plasma cells, the matured form of B-cells that produce high amounts of antibodies.

It involves collecting a patients immune T-cells and modifying them in the lab to produce a chimeric antigen receptor, or CAR, that selectively binds to CD19. Modified T-cells are then infused back into the patient, where they are expected to eliminate CD19-positive B-cells.

In this report, researchers in Germany and at Kyverna described the first successful use of anti-CD19 CAR T-cell therapy in a person with severe and refractory, or treatment-resistant, gMG the most common type of MG which is characterized by widespread muscle weakness and fatigue.

We believe this case report provides compelling evidence for the potential of anti-CD19 CAR T-cell-mediated deep B cell depletion in inducing remission and improving symptoms in severe, treatment-refractory MG, said Aiden Haghikia, MD, the studys first author and director of the department of neurology at Otto-von-Guericke University Magdeburg, in Germany.

A 33-year-old woman was diagnosed with gMG in 2012 and tested positive for self-reactive antibodies against the acetylcholine receptor (AChR) protein, the most common target of MG-driving antibodies.

From 2021 to 2023, she experienced difficulties in breathing, swallowing, and walking without mobility aids. She also had several myasthenic crises, or sudden worsening of symptoms, requiring invasive breathing support during five hospitalizations at the researchers institution.

She tried several approved MG treatments, including the B-cell-depleting therapy rituximab, but none stabilized her disease, which was classified as severe. Between March and May 2023, her disease progressed despite treatment with standard immunosuppressive drugs and corticosteroids.

Given the refractory nature of the disorder, and following successful use of anti-CD19 CAR T cells in autoimmune rheumatic diseases, we decided to treat her with a rationally designed CAR T approach, the researchers wrote.

She was given a single infusion of KYV-101 using her own T-cells. Consistent with previous results in treated people with other conditions, CAR T-cells in her bloodstream reached their peak growth 16 days after infusion, and were still detectable after about two months.

CD19-positive B-cells, which were already reduced due to prior treatments, were eliminated from her bloodstream after eight days and remained undetectable after about two months. At the same time, the levels of anti-AChR antibodies fell by 70%, while those of protective antibodies associated with vaccinations remained unchanged.

These findings indicate most disease-causing antibodies were produced by short-lived plasma cells positive for CD19, which are targeted by KYV-101, the researchers noted.

By contrast, protective antibodies produced by long-lived plasma cells in the bone marrow that do not have CD19 are shielded from the effects of CD19 CAR T cells, the team wrote.

In addition, the patients muscle strength and fatigue improved during the first two months after treatment. She was able to hold out her arm horizontally for longer, walk without supportive devices, and had lower scores for disease activity and severity, as assessed by validated measures.

Notably, rituximab targets CD20, a protein found at high levels on the surface of B-cells across their several stages of maturation, but at lower levels on plasma cells. This may explain why KYV-101 was successful in this patient when rituximab was not.

Additionally, the observed clinical improvements occurred despite very reduced exposure to corticosteroids and the MG therapy Mestinon (pyridostigmine bromide), both of which were to be stopped in the coming months.

The woman experienced no adverse events associated with CAR T-cell therapies, such as excessive immune responses, immune cell-related neurological damage, or deficient levels of overall antibodies.

However, she had a mild increase in liver enzymes, suggesting liver injury, that resolved without treatment.

We are extremely happy with the outcome so far, which suggests that a different CAR T-cell approach targeting CD19 with a stably expressed CAR has the potential to be safe and effective in severe and refractory MG, said Dimitrios Mougiakakos, MD, the studys senior author and director of the clinic of hematology, oncology, and stem cell transplantation at the university.

Kyverna is developing KYV-101 as a potential treatment for various other autoimmune diseases driven by B-cells, including lupus, scleroderma, and multiple sclerosis.

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KYV-101 found safe, effective for woman with severe gMG - Myasthenia Gravis News