Third case in the world of cure of HIV after stem cell transplant – Atalayar

The IciStem consortium, coordinated by IrsiCaixa, presents the third case of cure of HIV infection in the world. This is a man who was withdrawn from supervised antiretroviral treatment for HIV after undergoing a stem cell transplant to treat myeloid leukaemia. Four years later, the virus has not reappeared. The study is published in the journal Nature Medicine, in an article demonstrating the absence of viral particles and immune response against the virus in the patient's body despite not receiving treatment for 4 years, evidence that allows the scientific team to consider the case of the patient from Dsseldorf as a new case of cure.

The study was carried out by the international consortium IciStem, coordinated by the IrsiCaixa AIDS Research Institute - a centre jointly promoted by the "la Caixa" Foundation and the Catalan Government's Department of Health - and the University Medical Center in Utrecht (The Netherlands). "Together with an excellent team of professionals from all over the world, we have been studying these exceptional cases for nine years in which, thanks to a therapeutic strategy, the virus is completely eliminated from the body. We want to understand each step of the cure process in detail in order to design strategies that can be replicated in the entire population," explains Javier Martnez-Picado, ICREA researcher at IrsiCaixa, co-director of IciStem, and co-author of the article.

The Dsseldorf patient, a story of overcoming the disease

In 2008, a medical team in Dsseldorf (Germany) diagnosed HIV infection in a person who would later be known as the Dsseldorf patient because of his uniqueness. Following the diagnosis, the patient was started on antiretroviral treatment, which brought his infection under control and reduced the amount of virus to undetectable levels in his blood. Four years later, in 2012, he developed leukaemia, a cancer of the immune system cells, and had to undergo a stem cell transplant. In such unique cases, a stem cell donor is sought who has the CCR532 mutation. This genetic alteration means that you do not produce one of the gateways for HIV to enter the cells and therefore makes infection more difficult. "It is very complicated for all these factors to coincide, only 1% of the population has this mutation and, in addition, it is necessary for the donor to be a compatible blood donor to avoid transplant rejection," says Maria Salgado, IGTP researcher at IrsiCaixa and co-author of the study. In the case of the Dsseldorf patient, a woman made it possible to fit all the pieces together.

More than 5 years after the transplant, and having gone through two relapses of leukaemia and several complications, the patient stabilised. From there, the research team agreed to take him off antiretroviral treatment for HIV. Today, the patient from Dsseldorf is 53 years old and in good health. "When he stopped treatment, we followed him for 44 months and did not detect any traces of virus in his blood or tissues," says Salgado. "Nor have we seen any immune response characteristic of a viral flare-up. Their defences are not activated against HIV because they don't have to defend themselves against the virus". All these data allow the scientific team to affirm that the person has been cured of HIV infection.

The HIV cure map of the world

The confirmation of the cure for the Berlin and London patients precedes that of the Dsseldorf patient. Although these are the only three cases where it is possible to speak of a cure, the HIV remission of two other patients, the one in New York and the one at the City of Hope Hospital in Duarte, has already been presented at scientific conferences. "Neither of them have special immune characteristics that allow them to control HIV infection spontaneously, but the virus has been eliminated from the body as a result of medical intervention. This differentiates these cases of eradication from the cases of functional cure in elite or post-treatment controllers achieved so far, where people's own bodies had special factors that allowed them to control the virus," says Salgado. The Dsseldorf patient is thus a third proof of concept that demonstrates the possibility of curing HIV and rekindles hope in the scientific world dedicated to fighting the virus.

However, this strategy is very aggressive and not scalable to the rest of the population. Stem cell transplantation is only applied to people who suffer from a haematological disease and have no therapeutic alternative. In the case of people with HIV, there is an alternative, and that is antiretroviral treatment. "One possible strategy that is already being worked on is to introduce the CCR532 mutation through gene therapy to achieve a cure for HIV without having to undergo a transplant," says Martnez-Picado.

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Third case in the world of cure of HIV after stem cell transplant - Atalayar

UC San Diego’s Astrobiotechnology Hub to Drive Drug Discovery in … – today.ucsd.edu

ISSCOR Center manager Jessica Pham (left) and Jamieson Lab manager Jane Isquith (right) hold a Space Tango CubeLab, an automated platform for performing cell culture in space.

Another line of research will investigate the effects of stress and aging on liver progenitor cells. This work is led by Tatiana Kisseleva, MD, PhD, professor of surgery at UC San Diego School of Medicine, and David A. Brenner, MD, president and chief executive officer of Sanford Burnham Prebys and former vice chancellor for Health Sciences at UC San Diego.

Kisseleva and Brenner study ailments of the liver, such as fibrosis and steatohepatitis, a type of fatty liver disease. They are interested in determining the impact of microgravity on liver function, which could provide insights into diseases on Earth, and the potential effects of space travel.

A final major research focus uses blood stem cells to study the molecular mechanisms of cancer. When stem cells in our bone marrow become mutated, they give rise to precancerous cells that can lead to leukemia. This process typically occurs over several decades on Earth, but happens much faster in space where cells are more exposed to the suns ionizing radiation. This offers Jamieson and colleagues the opportunity to look for biomarkers of cancer and immune cell malfunction in a compressed time frame.

If we can find early predictors of cancer progression on the ISS, we are ideally positioned to rapidly translate them into clinical trials back on Earth at the Sanford Stem Cell Institute, said Jamieson.

And theyre well on their way there. Jamiesons team, in partnership with Space Tango, has now completed three NASA-funded launches of blood stem cells into space, with a fourth scheduled in March. The data theyve collected, in conjunction with experiments done on Earth, has already revealed a particular protein, ADAR1, as a main driver of cancer proliferation in space.

ADAR1 helps control the bodys innate immune response, editing RNA molecules so they wont be attacked by the immune system. This is useful in some contexts, but in disease states and the space environment, ADAR1 becomes overexpressed. This overactivity can then drive cancer cells to proliferate and develop a resistance to chemotherapeutic drugs. Once the researchers discovered this, they accelerated the development of a small molecule inhibitor of ADAR1, called Rebecsinib, which they recently showed can reverse the effects of the overactive protein.

Space research was critical in helping us scale and refine this novel drug target, said Jamieson. As part of Axiom Spaces AX-2 launch in May, Jamiesons team will start collecting blood samples from astronauts to see if there are any changes in the immune regulation of their stem cells, particularly in the activity of ADAR1. The samples will be collected longitudinally to study the short and long-term dynamics of immune dysregulation in spaceflight.

These types of experiments are just the start of a new push toward drug discovery and manufacturing in space. The burgeoning field, fueled by cross-sector collaborations, seems fit to transform the medical and biotech industries.

Together, we are creating something that not only provides an engine for economic growth but drives innovation to achieve the most important goal of all: benefiting patients, said Jamieson. The time to invest in space science is now.

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UC San Diego's Astrobiotechnology Hub to Drive Drug Discovery in ... - today.ucsd.edu

State-of-the-art Advanced Aesthetics MedSpa & Wellness Center to Open in Palm Beach – EIN News

Palm Beach Advanced Aesthetics

Advanced Aesthetics MedSpa Palm Beach (Photo Credit: AAMS)

Chase Backer, Founder (Photo Credit: AAMS)

The Advanced Aesthetics Wellness Center provides luxurious, leading edge and latest innovations in catering to individuals looking to get the most out of life

Chase Backer, Founder

Experts in the field have commented as to how Wellness facilities are in-demand and on-trend. "Plastic Surgery has gotten better over the years because you have more people who are better trained." According to Dr. Sherrell Aston, MD. Corroborating these comments, Dr. Jennifer Walden, MD recognized as one of America's top plastic surgeons adds that," the Wellness industry is in a constant state of innovation."

Advanced Aesthetics medical staff boasts a team of top doctors including Dr. Charles Pereyra, MD a leading clinical physician who has conducted extensive research in the use of regenerative medicine, anti-aging, and stem cell therapy. Dr. Pereyra takes a regenerative medicine approach to healing injuries, wellness therapies and more.

"More recently we have been developing products to help aesthetic needs. I feel like having confidence and looking good is a huge part of just being human," says Dr. Charles Pereyra, MD, "we have developed a topical stem-cell product to use on the face, a mask, as well as a micro-needling so it is like the new "vampire facial." The vampire facial used to just the Clients blood and micro-needling but now we use the micro-needling from blood and real-live stem cells and the facial is incredible. It is the future of regenerative medicine for aesthetics. So we intend to bring that treatment to Advanced Aesthetics."

in charge of the anti-aging and weight loss peptides and hormone replacement therapy is Kalev Kongro, He will make Clients look good and feel good the way they should, says Backer. Kalev has been with me from the beginning helping with research and design of the facility, he has natural born intelligence that helps with decision making and through that bond of trust we have also become the best of friends

The new Advanced Aesthetics Wellness Center will provide luxurious, leading edge and latest innovations in catering to successful individuals looking to get the most out of a happy, healthy lifestyle. Facilities include a gorgeous luxury lounge with a relaxing waterfall and juice bar that will offer everything from espresso to champagne. Private clients will also be able to access the very latest in body sculpting, oxygen therapy, IV-vitamin therapy, hormone replacement therapy, lasers, laser hair removal, injectables, fillers, Botox, body toning and massage. In turn, there will be regenerative stem-cell topical creams available to address scars, wrinkles and hair loss.

Simply put, the intention for Advanced Aesthetics Wellness Center is to be available to anyone looking for the best of the best. Entrepreneur Backer says that the ambition is for Advanced Aesthetics to provide the best anti-aging and healing facility in the Palm Beach area, "Our staff will provide the very latest technology, products and services to help everybody look and feel their very best. What drove him was his very personal experience with his Mothers Dementia. That experience has made him place education on regenerative techniques and aesthetics at the core of what makes Advanced Aesthetics Wellness Center different.

From body-sculpting to stem-cell therapy and non-invasive injectibles, Advanced Aesthetics will be the gold-standard in the health and beauty industry."

For High-Resolution Images please follow this link: https://bit.ly/3EkbDMn

Advanced Aesthetics Med-Spa & Wellness Center is located in the heart of Palm Beach. Address: 2528 Okeechobee Boulevard, West Palm Beach, FL 33409 T: (561) 360-2446

Norah LawlorLawlor Media Group, Inc.+1 212-967-6900email us hereVisit us on social media:FacebookTwitter

Advanced Aesthetics MedSpa Palm Beach (Photo Credit: AAMS)

Chase Backer, Founder (Photo Credit: AAMS)

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State-of-the-art Advanced Aesthetics MedSpa & Wellness Center to Open in Palm Beach - EIN News

Retired K-9 creates awareness about his terminal nerve disease – WISN Milwaukee

Retired St. Francis K-9 Bane has been out of the force for a little over two years now. He's been diagnosed with a terminal nerve disease Now he's paving the way for awareness of his disease and a new type of treatment.For Bane, Thursday's vet visit started off the same. He hopped out of the car's trunk, shook off the ride and got help from his handler Detective Holly McManus with the St. Francis Police Department. But inside the West Allis Veterinary Clinic on Greenfield Avenue, Thursday's treatment is a little different.McManus started noticing stumbling and weakness in Bane about a year ago.McManus thought it had something to do with his hip, a common issue in German shepherds, but all of Bane's tests came back normal.It wasn't until he got a DNA test in June of last year that McManus started getting some answers.Bane tested positive for degenerative myelopathy, DM for short. "Dogs will start to show signs that they're slowing down, that they have some neurologic deficits in the hind end particularly," said Dr. Harpreet Singh, a surgery specialist.On a scale of 1 to 5, five being the worst in terms of severity of DM, Singh said he would rate Bane at a four. "Then the disease will and in some cases rapidly within six months progress to where it can affect the front legs," Singh said.Singh said DM is the K-9 version of ALS in adults.After the diagnosis, Brandon Ames, owner of AniCell Biotech, a company based out of Arizona, reached out to McManus to offer Bane an extension of active life with a natural stem cell treatment."Bane invested in us," Ames told WISN 12 News. "He put his life out there and the best thing we can do is give back and do what we can for him.""The cells that we're injecting come from the innermost layer of the placenta," Singh said. "It's unlikely that the body is going to try to attack them."Ames said this is the first time this stem cell treatment is used to treat DM in Wisconsin; usually it's used by vets to treat wounds."Trying something that really isn't heard of in in Wisconsin, I think that makes a big difference," Singh said. "We are always trying to improve our outlook on what is possible.""He's terminal," McManus said. "So to me, I looked at it as if we're doing minimally invasive trial products, what do we have to lose?"McManus told WISN 12 News if you can get past the physical appearance of the disease. The silver lining here is that Bane is quite comfortable."We're pretty sure it's mostly pain-free. I think the pain he has, or the discomfort is just from being an 11-and-a-half-year-old German shepherd as opposed to the degenerative myelopathy," McManus. "I don't see it in his face that he's sad he can't run after something, or he can't go do something because he was always the work smarter, not harder type of dog anyway.""The spirit is still there," McManus said. "The will to survive is still within these animals. It really is a disability, you know?"This is Bane's second stem cell treatment.McManus said after the first one in January, she noticed a change is his movement within 48 hours. While Bane's medical team monitors his progress with this treatment, it's not necessarily a cure for his disease. "The disease doesn't scare me as much as the impending loss that I'm going to have," McManus said. "There is a line that I won't be able to cross with him, and that I know that at that point that'll be the time when he tells me it's time I need to respect that."McManus said this Bane's way of still serving his community even after being retired. She says the goal is to raise awareness about DM and help other dog owners who might be going through the same thing.Singh said a dog diagnosed with DM usually lives anywhere from six months to two years.McManus adds a life-size bronze statue of Bane will be built outside of the St. Francis Police Department by this summer. There are ways to help with Bane's medical and treatment expenses. He has a fund set up here.

Retired St. Francis K-9 Bane has been out of the force for a little over two years now. He's been diagnosed with a terminal nerve disease

Now he's paving the way for awareness of his disease and a new type of treatment.

For Bane, Thursday's vet visit started off the same.

He hopped out of the car's trunk, shook off the ride and got help from his handler Detective Holly McManus with the St. Francis Police Department.

But inside the West Allis Veterinary Clinic on Greenfield Avenue, Thursday's treatment is a little different.

McManus started noticing stumbling and weakness in Bane about a year ago.

McManus thought it had something to do with his hip, a common issue in German shepherds, but all of Bane's tests came back normal.

It wasn't until he got a DNA test in June of last year that McManus started getting some answers.

Bane tested positive for degenerative myelopathy, DM for short.

"Dogs will start to show signs that they're slowing down, that they have some neurologic deficits in the hind end particularly," said Dr. Harpreet Singh, a surgery specialist.

On a scale of 1 to 5, five being the worst in terms of severity of DM, Singh said he would rate Bane at a four.

"Then the disease will and in some cases rapidly within six months progress to where it can affect the front legs," Singh said.

Singh said DM is the K-9 version of ALS in adults.

After the diagnosis, Brandon Ames, owner of AniCell Biotech, a company based out of Arizona, reached out to McManus to offer Bane an extension of active life with a natural stem cell treatment.

"Bane invested in us," Ames told WISN 12 News. "He put his life out there and the best thing we can do is give back and do what we can for him."

"The cells that we're injecting come from the innermost layer of the placenta," Singh said. "It's unlikely that the body is going to try to attack them."

Ames said this is the first time this stem cell treatment is used to treat DM in Wisconsin; usually it's used by vets to treat wounds.

"Trying something that really isn't heard of in in Wisconsin, I think that makes a big difference," Singh said. "We are always trying to improve our outlook on what is possible."

"He's terminal," McManus said. "So to me, I looked at it as if we're doing minimally invasive trial products, what do we have to lose?"

McManus told WISN 12 News if you can get past the physical appearance of the disease. The silver lining here is that Bane is quite comfortable.

"We're pretty sure it's mostly pain-free. I think the pain he has, or the discomfort is just from being an 11-and-a-half-year-old German shepherd as opposed to the degenerative myelopathy," McManus. "I don't see it in his face that he's sad he can't run after something, or he can't go do something because he was always the work smarter, not harder type of dog anyway."

"The spirit is still there," McManus said. "The will to survive is still within these animals. It really is a disability, you know?"

This is Bane's second stem cell treatment.

McManus said after the first one in January, she noticed a change is his movement within 48 hours.

While Bane's medical team monitors his progress with this treatment, it's not necessarily a cure for his disease.

"The disease doesn't scare me as much as the impending loss that I'm going to have," McManus said. "There is a line that I won't be able to cross with him, and that I know that at that point that'll be the time when he tells me it's time I need to respect that."

McManus said this Bane's way of still serving his community even after being retired. She says the goal is to raise awareness about DM and help other dog owners who might be going through the same thing.

Singh said a dog diagnosed with DM usually lives anywhere from six months to two years.

McManus adds a life-size bronze statue of Bane will be built outside of the St. Francis Police Department by this summer.

There are ways to help with Bane's medical and treatment expenses. He has a fund set up here.

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Retired K-9 creates awareness about his terminal nerve disease - WISN Milwaukee

Can We Rebuild the Spinal Cord? These Scientists Are Redefining … – Inverse

After someone experiences a spinal cord injury, doctors set off on a race against the clock. Within a few hours, they rush patients into surgery and administer anti-inflammatory drugs, ranging from over-the-counter medications like Advil to the steroid methylprednisolone, to avoid as much damage as possible keeping in mind that post-injury swelling and insufficient blood supply can wreak further damage on neurons. After intervening during this narrow window of time, scientists have long thought that the chances of additional recovery grow slim.

The dominant thinking was that you should focus on acute injuries, Aileen Anderson, a stem cell researcher at the University of California, Irvine, tells Inverse. If you could just hit a magic bullet at that stage and minimize the amount of damage thats happening because it kind of rolls out over days and a couple of weeks this was the place to target.

People with spinal cord injuries can receive electrical stimulation via electrodes surgically placed near the spinal cord or stuck on the skin.

But in recent years, labs have made major strides in innovative techniques that can be applied long after the initial damage to the spinal cord, including using electrical currents to re-awaken key pathways in the nervous system and surgeries that could coax injuries to repair themselves.

These methods expand the possibilities of recovery for people who have lived with severe spinal cord injuries for years even several decades and may spend up to millions of dollars over their lifetimes on medical care and living expenses, according to the National Spinal Cord Injury Statistical Center.

The leading causes of spinal cord injuries in the U.S. include traffic accidents, falls, and sports-related injuries, a 2016 study found. Each year, they occur among a relatively small number of people around 17,000. But a large population lives with the residual damage from chronic injuries (estimates vary widely between 250,000 and 1 million people, Anderson says.)

Ultimately, even minor progress for those with chronic injuries could have significant benefits, Michael Fehlings, a neurosurgeon at Toronto Western Hospital in Canada, tells Inverse. The type of full-body paralysis experienced by the late Superman actor Christopher Reeve, for example, can run someone between $10 and $20 million when you factor in costs like multiple caregivers, an electric wheelchair, and home upgrades.

If one had a treatment that could even partially restore hand and upper extremity function and partially restore independence of a person, the economic impact and the human impact is enormous, Fehlings says.

Spinal cord damage can hinder the crucial nervous system circuitry that allows us to move and feel pain, among other important functions.

The spinal cord is a long, fragile column that contains nerve cells and skinny fibers called axons, which deliver messages back and forth between the brain and nerves located throughout the body. This constant communication tells muscles to move, helps us feel pain, and regulates heart rate, among other crucial functions.

Injuries can impair connections between nerves and hinder the nervous systems circuitry. For example, these disruptions may cause uncontrolled movements or loss of movement in certain body parts.

An individuals specific symptoms depend on the location of the injury; for example, impacts higher up in the spinal cord may cause paralysis in most of the body, referred to as quadriplegia or tetraplegia, according to the National Institutes of Health. Damage that occurs lower on the spinal cord can cause paralysis of the legs and lower body, or paraplegia.

Early interventions including surgery to decompress the spinal cord and drugs that reduce inflammation have long been considered key to recovery, Fehlings says. Researchers have also looked into techniques like inducing hypothermia in spinal cord injury patients. But ultimately, many efforts beyond surgery have produced only modest results in studies, according to Anderson.

There were a ton of strategies that people worked on in the lab to just minimize the initial damage, but there were also any number of failed clinical trials that came out of that, she says.

People with spinal cord injuries can receive rehab guided by robots or physical therapists to regain walking skills.

At the moment, patients can find some relief from side effects like muscle spasms and impaired bladder control. But most of whats currently offered in clinics cant actually fix the damage underlying these symptoms.

There are no therapies that recover people with chronic spinal cord injury right now, Susan Harkema, associate director of the Kentucky Spinal Cord Injury Research Center, tells Inverse. Most therapies that are approved with a clinical indication are to treat symptoms.

These therapies include a type of rehab called locomotor training that was pioneered by Harkema and her colleagues at the University of Louisville. During a rehab session, patients can wear a harness for support while a robot or staff member moves their legs on a treadmill. But a small number of centers offer this type of rehab, Anderson adds.

(In 2016, Harkemas team lost federal funding for a study on locomotor training due to concerns from the National Institute on Disability, Independent Living and Rehabilitation Research that the team strayed from research protocols an unusual move from a government agency. Later, an internal audit from the University of Louisville failed to find major issues with the study but noted that some aspects of the research could be improved.)

Patients can also receive a technique first developed in the 1960s called electrical stimulation. This method sends low levels of electrical current to the spinal cord through electrodes placed on the skin or implanted near the spinal cord. These devices aim to replicate how the brain typically sends signals to various parts of the body, potentially reviving movement in areas affected by the injury.

Even when people have a very severe injury, there are some circuits that remain in the nervous system, Fehlings says. So the rationale for using the electrical stimulation is to try to, if you will, trick the nervous system to try to activate some of these circuits.

Electrical stimulation has shown benefits, like restoring a degree of arm and leg movement, aiding in the functioning of the lower urinary tract, and improving the effectiveness of rehab. In fact, when combined with rigorous physical training, it has even helped people walk again by engaging nerves that control lower-body movement.

While thats likely not possible for people with full-body paralysis due to the degree of damage, electrical stimulation may still help achieve a degree of movement that wouldnt otherwise be possible, such as improved hand grip and strength.

If we stimulate the spinal cord itself, people can move voluntarily who are fully paralyzed, even up to 40 years post-injury, Harkema says.

Over the last few years, the U.S. Food and Drug Administration has approved a handful of electrical stimulation devices, including Abbotts Proclaim Plus device and Saludas Evoke System.

Moving forward, scientists want to pinpoint the precise group of neurons behind stimulations success so that they can be more effectively targeted during the process.

Some labs are even working on high-tech clothing that includes electrodes to help people on the go without the need for surgical implants a potentially crucial breakthrough since real-time stimulation gives people a higher degree of mobility. Eventually, the goal is for people to move without requiring stimulation.

This is an exciting approach, Fehlings says. Its not a cure for spinal cord injury it needs to be validated in larger clinical trials but its something that does have potential hope for individuals who have a chronic spinal cord injury.

Scientists think transplanting stem cells into the spinal cord may help repair the nervous system.

In what is perhaps the most revolutionary proposal, some scientists want to transplant stem cells into the spinal cord to restore sensory and motor function. This method has shown promise in animal studies, and several phase 2 human clinical trials are in the works.

The hope is that the new cells can replace the ones lost to injury and repair the spinal cords signaling highway. Plus, future transplants could use stem cells derived from the patients own body, potentially avoiding the negative health impacts of transplant rejection, Anderson explains.

Above all, this option could help patients who lack enough viable neural cells to benefit from other therapies that are currently in use.

The cellular transplantation approach seeks to address the individuals who have such a devastating spinal cord injury that even electrical stimulation is just not going to work, Fehlings says.

Some teams, including Anderson and her colleagues, are also trying to put specialized materials into peoples spinal cords, such as scaffolds made of hydrogels, as another method to help the spinal cord reconnect itself. It could also help to combine scaffolding and stem cells, Anderson says, an idea currently in the early stages of development.

Even if some of these approaches prove to be effective in trials, a lack of funding could prevent them from reaching wide swaths of patients. Since the number of spinal cord injuries that occur every year is relatively tiny, pharmaceutical companies may not see these concepts as worthwhile investments.

But these injuries share many features with conditions that also affect the central nervous system, such as multiple sclerosis, strokes, and traumatic brain injuries.

We hope that [with] what we develop for spinal cord injury, we can make the case that it might be able to impact this broader set of diseases and therefore its worth investing in, she says.

Despite looming challenges, Fehling says hes feeling optimistic that regenerative medicine approaches like stem cell transplants could arrive in clinics within the next five to 10 years. If so, it could transform the lives of patients who may not benefit from todays options.

Were at an inflection point in the regenerative medicine era, he says. Im extremely hopeful.

These are the innovations of today that will shape the world of tomorrow. Subscribe for free to Inverses weekly newsletter that explores our future.

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Can We Rebuild the Spinal Cord? These Scientists Are Redefining ... - Inverse

Lucius Partners Portfolio Company Voltron Therapeutics, Inc … – PR Newswire

Self Assembling Vaccine will target Prostate Stem Cell Antigen (PSCA)

Key target in Prostate, Renal and Urothelial Cancers

NEW YORK, Feb. 28, 2023 /PRNewswire/ -- Voltron Therapeutics, Inc, a Lucius Partners portfolio company, announced today that it has signed a new Sponsored Research Agreement (SRA) with the Vaccine and Immunotherapy Center(VIC) at Massachusetts General Hospital (MGH), Harvard Medical School to initiate a pre-clinical immuno-oncology trial targeting prostate stem cell antigen (PSCA) in prostate, renal cell and urothelial cancers, adding important potential indications to its growing pipeline.

James Ahern, Director at Voltron Therapeutics, has announced that the biotechnology company recently generated robust and compelling data in two pre-clinical trials for human papillomavirus (HPV)-related cancers sponsored at MGH. The Self-Assembling Vaccine (SAV) targeting HPV demonstrated a positive and highly statistically significant survivalimprovement in a dose-related manner. This new pre-clinical program will leverage that important work and expand the Company's potential pipeline into additional types of antigens and cancers.

Pat Gallagher, Voltron's Chief Executive Officer, commented, "In our HPV-related cancer proof of concept trial, Voltron's SAV demonstrated highly statistically significant increases in survival driven by reduced tumor growth as well as increased tumor infiltration by beneficial immune cells (e.g., CD8+ T cells) using two HPV targeting immunogenic peptides. By using a full protein in our PSCA trials to target cancers of interest instead of specific peptides, we hope to demonstrate unparalleled platform flexibility that would allow us to use full protein sequences to target tumors or viruses of interest. This affords broader targeting in heterogeneous immune systems and could, in theory, allow our vaccine to induce an immune response to any specific tumor antigen of interest."

Relative to other vaccine approaches, the SAV shows key advantages. It only requires two elements for all vaccines; it has not shown potential to date for off-target immune activation; it has no risks inherent with viral vectors; it does not require special synthetic processes, storage or a complex cold chain like mRNA vaccines.

Dr.Mark Poznansky, Director, Vaccine and Immunotherapy Center, MGH, commented: "This new and important industry sponsored study explores a broader acting therapeutic approach by exploiting the diverse immunologic functions of the SAV platform to enhance the targeting capabilities of immunogenic epitopes from full proteins. We will explore whether Voltron's SAV platform is capable of presenting the tumor antigen, in this case PSCA, to multiple types of antigen-presenting cells and thereby facilitate epitope presentation to both MHC class I and class II in the context of immune stimulation."

James Ahern, who is also Managing Partner of Laidlaw & Company (UK) and Founder of Lucius Partners, stated, "The team continues to identify additional programs for Voltron, driven by their expertise and disciplined process. We will pursue indications in both oncology and infectious disease that leverage our highly flexible vaccine platform to create new solutions for clinicians and patients. We remain focused on de-risking our programs and to provide value to our shareholders."

About Voltron Therapeutics, Inc.

Voltron Therapeutics, Inc., a Delaware corporation, was founded in 2017 to lead and accelerate the development of the Vaccine and Immunotherapy Center (VIC), and the Massachusetts General Hospital's novel Self Assembling Vaccine technology in a variety of indications, including in Oncology and Emerging Infectious Diseases. Voltron holds an exclusive worldwide license to this technology. With the work of our world class team of researchers and development team, this technology has shown in certain pre-clinical studies initial proof of concept in two infectious diseases (including Lassa Fever) as well as three oncology indications (HPV Related Cancers). For more information, please visit http://www.voltrontx.com.

About Lucius Partners, LLCFounded by James Ahern, Lucius Partners is a consultancy that provides a broad suite of services to help healthcare companies grow, achieve milestones and generate value for their shareholders.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the VaxCelerate Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Voltron's current expectations and various assumptions. Voltron believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Voltron may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and any Voltron filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Voltron's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Voltron cannot guarantee future results, events, levels of activity, performance or achievements. Voltron does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

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Contact:

Patrick Gallagher, CEOVoltron Therapeutics, Inc.

Managing PartnerLucius Partners, LLC[emailprotected]

Matthew Duffy, PresidentVoltron Therapeutics, Inc.

Managing PartnerLucius Partners, LLC[emailprotected]

SOURCE Lucius Partners

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Lucius Partners Portfolio Company Voltron Therapeutics, Inc ... - PR Newswire

Little boy with brain cancer is one recipient of South High Marathon … – WNYT NewsChannel 13

Students at South Glens Falls High School will be busting a move Friday and Saturday for the South High Marathon Dance.

The 20 recipients who will benefit from the dance include Aiden Rodriguez, 7, who is battling brain cancer and lives in Washington County.

After years of noticing irregularities with his speech and walking, Aiden was finally diagnosed with brain cancer in September. He spent his 7th birthday in the hospital, recovering from brain surgery.

Aiden had surgery to remove part of the tumor, then another surgery, and four more. Theres also been stem cell treatments in Rochester.

Aiden came home on Wednesday after more than a month in Albany Medical Center. Though he still has a long road ahead of him, he will be able to attend the dance with his family.Its been an expensive journey for the Rodriguez Family. Theyre headed back to Rochester soon for high dose chemotherapy.Meantime, they hope to soak in the love of a caring community.

Hear the family express their gratitude for the communitys help by watching the video of Mark Mulhollands story.

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Little boy with brain cancer is one recipient of South High Marathon ... - WNYT NewsChannel 13

Mining cell therapy to repair injured bones and tendons – EurekAlert

Two new discoveries led by Cedars-Sinai investigators show stem cell therapies can help accelerate and promote the healing of bone and tendon injuries.

Were using developmental biology and stem cell technology to repair and regenerate damaged bones and tendon tissues, which has the potential to dramatically alter patient outcomes, saidDmitriy Sheyn, PhD, assistant professor in the departments ofOrthopaedics,SurgeryandBiomedical Sciencesat Cedars-Sinai.

Using iMSCs to Repair Tendon and Ligaments

In the first study, published in theJournal of Orthopaedic Research, Cedars-Sinai scientists showed that specialized engineered stem cells called induced pluripotent stem cell-derived mesenchymal stromal cells (iMSCs) can regenerate and repair the tendon more effectively than other types of cells.

The finding is a step forward in developing an efficient cell-therapy approach for repairing injured tendons and ligaments, which is a significant concern in sports medicine. Tendon injuries are often associated with high morbidity, prolonged disabilities and painful rehabilitation periods, during which secondary tendon ruptures can often occur.

One of the main advantages of using pluripotent stem cells is that they potentially represent an unlimited source of tenocytes, the cells in tendons and ligaments, said Sheyn, who is also an investigator in theCedars-Sinai Board of Governors Regenerative Medicine Institute. These cells could also one day offer an off-the-shelf allogeneic source for tendon cell-therapy applications.

The tendonwhich is a soft tissue that connects muscles to bones or, in case of ligaments, bones to boneshas very low capacity to heal, and tendon tears or ruptures can cause extreme pain. Even after the tendon heals, it never goes back to the same function as before the injury and has high rates of re-rupture.

To find a way to better repair these injuries, investigators looked to iMSCs.

Sheyn, who is the senior and corresponding author of the study; Angela Papalamprou, PhD, who is a postdoc in theSheyn Laboratoryand first author of the study; and the study team engineered the cells to produce a transcription factor called scleraxis, which is important in tendon development.

They found iMSCs were able to successfully increase the amount of scleraxis that was produced, which helped the iMSCs form tendon-like tissue and become more like tendon cells.

Then they exposed the cells to a type of mechanical stress called cyclic loading, which is a process that helps guide the iMSCs to develop into tenocytes.

While further studies are needed to determine the full potential of these modified iMSCs for tendon repair, the results showed that the iMSCs can effectively change into tendon cells and show promise as a potential cell therapy for tendon repair, said Sheyn.

Repairing the Skull With Cranial-Specific Stem Cells and 3D Printing

In the second study, published in the peer-reviewed journalScientific Reports, scientists found using iPSC-derived cranial-specific stem cells and embedding them in a special 3D-printable material can help heal injured bones in the skull.

Investigators found the material, called bio-ink, helped the cells survive and form new bone cells more effectively than other types of cells, like iMSCs. When tested in mice, the combination of using the bio-ink with the cranial-specific stem cells helped repair the skull better than other types of cells that were tested.

Treating cranial bone injuries is a major clinical challenge, said Sheyn, the senior and corresponding author of the study. New regenerative approaches like this one have the potential to be a powerful new option that may help address craniofacial reconstruction needs.

Currently, when someone has a traumatic skull bone injury, they usually need a graft using bones or synthetic material to repair the wound. While using a persons own bone is usually the best option, donor-site morbidity is a limiting factor, and using synthetic materials like titanium or polymers is often associated with high rates of infections and complications, especially for children.

Investigators have found 3D bioprinting to be a potential solution. Its a developing technology that can create scaffolds from different biomaterials that can mimic the shape, size and dimensions of the injury.

However, investigators have found using the 3D bioprinted materials alone is not enough.

While 3D printing can give us the shape that we want, the problem that we have in the 3D printing field is that we cannot print with cells because the printing technology is not cell compatible, said Sheyn. And its the cells that will really help with healing and repairing of the injury.

To see if they can improve the new technology, Sheyn; Giselle Kaneda, a research assistant in the Sheyn Laboratory and study author; and other members of Sheyns laboratory tested two bio-inks to see which one provided better viability and better use for cells to differentiate and regrow new cells in the skull that could potentially repair the defect.

They specifically looked at different types of iPSC-derived cells and added a special protein called BMP6 to see if it could help generate viable bone graft alternatives for cranial reconstruction.

They found the combination of the cranial-specific stem cells with BMP6 and the bio-ink stimulated injury healing in the bone more efficiently than when they used iMSCs.

These results highlight the printability of bio-ink scaffolds and their ability to support stem cell survival and differentiation, making them attractive for craniofacial reconstruction, said Sheyn.

Funding: The first study was funded in part by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (award K01AR071512). The second study was funded in part by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (award K01AR071512), the Kosciuszko Foundation, and the American Centre of Polish Culture.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Mining cell therapy to repair injured bones and tendons - EurekAlert

U.S. Department of Defense Awards Theradaptive $4 Million … – OrthoSpineNews

FREDERICK, Md.,March 1, 2023/PRNewswire/ Today,Theradaptive,a leading biotechnology company specializing in therapeutic delivery platforms, announces a Technology & Therapeutic Development Award (TTDA) of$4 millionfrom the U.S. Department of Defense (DOD) awarded through the Peer Reviewed Medical Research Program (PRMRP) of the Congressionally Directed Medical Research Programs (CDMRP). The contract will fund its OsteoAdapt regenerative therapeutic product for spine and trauma repair to first in human clinical studies.

Theradaptives OsteoAdapt product was granted three breakthrough medical device designations by the U.S. Food and Drug Administration (FDA) in 2021 and 2022 for various spinal indications. OsteoAdapt is created by combining AMP2 protein, a novel bone regenerative biologic, with ReBOSSIS, a510K-approved implant material. OsteoAdapt has the capability to precisely direct bone regrowth where it is needed in the body.

The funds from the DOD contract will enable Theradaptive to continue its work to meet regulatory requirements and scale up Good Manufacturing Practices-compliant manufacturing of the OsteoAdapt product in preparation for clinical studies. After a request for an Investigational Device Exemption (IDE) is submitted to the FDA for approval, Theradaptive will initiate human clinical trials.

Theradaptives OsteoAdapt, powered by their proprietary AMP2 biologic, has beaten the standard of care in all preclinical studies to date. The DOD funding will enable the development of OsteoAdapt and the promise of life-changing surgery for Service Members and civilians with degenerative or traumatic spinal, extremity, craniomaxillofacial, and dental injuries. 37% of adults over the age of 30 and over 80% of adults over the age of 50 have at least one degenerated vertebral disc.

CEO and founder of Theradaptive, Dr.Luis Alvarez, Ph.D., believes the funding is a major step towards clinical trials: This award affirms Theradaptives rapid and successful execution of earlier stage programs funded by Department of Defense, and provides funding crucial to advance a new kind of therapeutic that upon FDA approval will address massive unmet needs among Service Members, Veterans, and in the general population.

We understand the challenges that many service members face, particularly when traumatic extremity injuries progress to limb amputation or when years of physical activity lead to spinal degeneration, disc injury, and pain. The support provided by this contract will accelerate the completion of product development milestones that will allow Theradaptive to reach clinical trials faster and get us one step closer to providing this game-changing therapy to patients who need it most.

Dr.Leon J. Nesti, a surgeon at the Walter Reed National Military Medical Center offers additional perspective: Starting from early inspiration from Luiss military career, Theradaptive has made significant progress in developing a regenerative technology that has now been demonstrated for orthopedic repair. Their technology offers a transformative approach to address medical challenges through regenerative mechanisms that stimulate stem cells, including the potential to restore patients quality of life by accelerating the process of bone regeneration. This technology is one of the few that addresses the stem cell niche directly by delivering growth factors to those stem cells. This work will have a large scientific impact and will benefit Service Members and Veterans.

About Theradaptive

Founded in 2016 and headquartered inMaryland, U.S.,Theradaptiveis a venture-backed biopharmaceutical company with the goal of leveraging their therapeutic delivery platform that can deliver biologics where they are needed in the body with high precision and persistence to address unmet medical needs. Theradaptive is led by CEOLuis Alvarez, Ph.D., and its innovative platform has started to enable new therapeutics in spine, orthopedic and soft tissue repair as well as targeted immuno-oncology.

This work is supported by the Office of the Assistant Secretary of Defense for Health Affairs through the Congressionally Directed Medical Research Programs under Award No.(W81XWH-22-1-0875) for an amount of$3,979,465.00. Opinions, interpretations, conclusions, and recommendations are those of the author and are not necessarily endorsed by the Department of Defense.

Contact:Serena Lertoraserena.lertora@theradaptive.com

SOURCE Theradaptive

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U.S. Department of Defense Awards Theradaptive $4 Million ... - OrthoSpineNews

Tisa-cel CAR-T reinfusion lacks durability for younger patients with B … – Healio

February 28, 2023

3 min read

Krupski C, et al. Abstract LBA4. Presented at: Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, Feb. 15-19, 2023; Orlando.

Disclosures: Krupski reports no relevant financial disclosures. Please see the abstract for all other researchers relevant financial disclosures.

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A small percentage of children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia who received a second infusion of tisagenlecleucel had clinically meaningful responses, data from a retrospective study showed.

Despite the drug appearing to be safe and well-tolerated, findings presented at Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR revealed only transient responses to repeat therapy with the CD19-directed chimeric antigen receptor T-cell therapy, including mostly nondurable remissions.

We have shown that reinfusion with [tisagenlecleucel] is not a definitive therapy, Christa Krupski, DO, MPH, professor in the department of pediatrics at University of Cincinnati and pediatric bone marrow transplant physician at Cincinnati Children's Hospital Medical Center, told Healio. The remissions it produces are unlikely to be long lasting, so physicians who treat these patients will need to have something else in mind before moving forward.

Tisagenlecleucel (Kymriah, Novartis) also known as tisa-cel has historically produced initial complete responses among approximately 80% of younger patients with B-cell ALL, Krupski said.

Those who experience disease progression after CAR-T have few treatment options, one of which is a second tisa-cel infusion. Prognosis is poor after this treatment, with the limited data available showing response rates to reinfusion ranging from 28% to 52%, Krupski said during a presentation.

Additionally, previous research suggested that patients with a short duration of B-cell aplasia after CAR-T infusion are susceptible to disease relapse.

Typically, the manufacturer has enough cells and produces a second dose of the agent that is kept in reserve, but little data exist regarding the effectiveness of reinfusion for patients who relapse after CAR-T, Krupski said.

A few patients at Krupskis center experienced loss of B-cell aplasia early after CAR-T infusion and received reinfusions with a second dose of tisa-cel.

We were unable ... to get them back into remission, or even get them to a point of B-cell aplasia again, she said, adding that her group was unsure if the poor results were attributable to the patients they treated or a lack of robustness with the overall approach of tisa-cel reinfusion.

This is what sparked our interest, and we have used our membership in the Pediatric Real World CAR Consortium to harness data from many centers and get some meaningful results, Krupski said.

Krupski used the Pediatric Real World CAR Consortium a group of 15 institutions that perform CAR-T for younger patients and collect data on patient outcomes to collect data on 42 younger patients (median age at first CAR-T infusion, 12.5 years; range, 0-26) with B-cell ALL who received reinfusion of tisa-cel at one of 13 participating sites.

Twenty-four patients (57%) received tisa-cel infusion for B-cell aplasia loss while having an ongoing complete response during their first CAR-T infusion. Seventeen patients (41%) received reinfusion for having detectable disease at day 28 after infusion. The remaining patient received reinfusion for having no response to the first infusion.

Median time between tisa-cel infusions was 173 days (range, 52-521).

The complete response rate 28 days after tisa-cel reinfusion served as the studys primary outcome measurement. Secondary outcome measurements included rates of reestablishing B-cell aplasia, OS and EFS after tisa-cel reinfusion.

Median follow-up was 496 days (range, 150-1,335).

Researchers reported a 1-year OS rate of 84% after reinfusion with tisa-cel for the entire study group.

Reinfusion with tisa-cel conferred a 41% EFS rate at 1 year.

Treatment-related toxicities appeared manageable overall, according to investigators. Twenty-four percent of patients developed cytokine release syndrome, with only 2% of cases being grade 3 or higher.

Investigators reported limited neurotoxicity after tisa-cel reinfusion (overall incidence, 7%; grade 3 or higher, 2%).

Reinfusion served as definitive therapy requiring no further treatment for five of the 24 patients who received tisa-cel reinfusion for B-cell aplasia loss while in complete remission.

Eleven of 17 patients who received a second infusion for treatment of disease progression had detectable disease 28 days after treatment, compared with six patients reported as minimal residual disease (MRD)-negative 28 days after infusion. Four of the patients with MRD-negative disease eventually experienced disease relapse.

The results confirm what Krupski and colleagues expected based on limited available data and their own experience: The use of tisa-cel reinfusion has limited effectiveness and should be given as a bridge toward another definitive therapy or with a strategy regarding next steps in mind.

This is especially true for patients who can tolerate hematopoietic stem cell transplantation, because delaying providing a second infusion of tisa-cel instead could miss a window of opportunity to provide a curative option, Krupski said.

[Physicians] dont want to falsely put our hope in something that is ultimately not going to be of benefit to the patient, she told Healio.

Krupski said the studys findings will change the way she approaches management of patients who have early relapse or loss of B-cell aplasia after tisa-cel infusion.

The main takeaway is that if you're going to consider a second infusion ... then it should not be looked at as definitive therapy, Krupski said. It needs to be a bridge to something, and that something, in my opinion, is a transplant for definitive therapy.

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