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.

Logo - https://mma.prnewswire.com/media/1846494/Lucius_Partners_Logo.jpg

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

Contract Manufacturing Market to Witness Substantial Growth of … – InvestorsObserver

Contract Manufacturing Market to Witness Substantial Growth of USD 362.72 Million with Healthy CAGR of 6.95% by 2029, Size, Share, Trends, Growth and Competitors Insight

BOSTON, March 02, 2023 (GLOBE NEWSWIRE) -- Data Bridge Market research has recently released expansive research titled Contract Manufacturing Market which guarantees you will remain better informed than your competition. This study provides a broader perspective of the marketplace with its comprehensive market insights and analysis, which eases surviving and success in the market. A steadfast Contract Manufacturing market research report serves to be a very momentous component of business strategy. This report provides important information, which assists to identify and analyze the needs of the market, the market size, and the competition with respect to the Contract Manufacturing industry. When the market report is accompanied by precise tools and technology, it helps tackle a number of uncertain challenges for the business. This market research report is one of the key factors used in maintaining competitiveness over competitors. Contract manufacturing market report supports the business to take better decisions for the successful future planning in terms of current and future trends in a particular product or industry.

Data Bridge Market Research analyses a growth rate in the contract manufacturing market in the forecast period 2022-2029. The expected CAGR of the contract manufacturing market tends to be around 6.95% in the mentioned forecast period. The market was valued at USD 211.9 million in 2021, and it would grow upto USD 362.72 million by 2029 . In addition to the market insights such as market value, growth rate, market segments, geographical coverage, market players, and market scenario, the market report curated by the Data Bridge Market Research team also includes in-depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.

Download Sample PDF of the Contract Manufacturing Market @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-contract-manufacturing-market

The contract manufacturing market is expected to witness significant growth during the forecast period. An increase in offshoring, especially in emerging countries, and the changing regulatory landscape, are the prominent trends currently present in the market. Considering the COVID-19 era, it brought in unparalleled demand for diagnostic tests, Personal Protective Equipment (PPE), ventilators, and other critical medical supplies.

Under contract manufacturing , the firms provide services such as production of goods by firm, under the label or brand of another firm. In other words, contract manufacturing is managing of one companys resources by the other company to manufacture its products. A pharma contract manufacturing company provides an array of services to drug companies, including drug development, drug manufacturing and commercial production, and documentation of compliance with FDA regulatory requirements.

The Global Market Is Analyzed in Depth in the Latest Study. Taking into account the current level of competition and its projected evolution over the next few years.

Rapidly increasing demands, a rise in industrialization, consumer awareness, growing sectors, and technical improvements are fueling the expansion of the global Contract Manufacturing market. Sales and revenue in this sector have increased at an exponential rate. The market's size and growth are both expected to increase thanks to the factors driving the market's expansion over the projected period.

Leading businesses in the worldwide Contract Manufacturing market are investing heavily in R&D in order to build a larger client base and expand their share of the market by reintroducing improved products to consumers. All of the companies' strategy, as well as their financial health, revenue, gross margin, and growth rate, are detailed in the study.

The Contract Manufacturing Market is Dominated by Firms Such as

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Opportunities

The rising demand for cardiovascular devices as a result of increasing cases of associated conditions is attributed to the growth of outsourcing of these devices. Moreover, the high complexity of cardiovascular devices and the need for technical expertise result in higher outsourcing of these devices.

The rising mergers and acquisitions are creating much opportunities in the global contract manufacturing market. For instance, in October 2018, Nordson Corporation acquired Clada Medical Devices, a design & development firm primarily focused on balloon catheters. This enhanced the design & development capabilities of the company, which further supports the companys position as a prominent contract manufacturer for OEMs across the interventional & surgical device landscape.

Key Market Segments Covered in Contract Manufacturing Industry Research

Service

Type

End User

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Key Industry Drivers:

In addition, the demand for medicines and medical devices is increasing due to factors such as the widespread use of non-invasive surgery. To meet this growing demand, OEMs outsource manufacturing of non-core manufacturing activities to help reduce labor costs, free capital, increase employee productivity, and improve manufacturing lead times

Manufacturing biologics using multiple products of plants has proven to be economically efficient and safe, as the risks associated with product carry-over are negligible or non-negligible. Supports the growth of. Rapid changes in the supply chain may be unmanageable due to the tightly regulated medical device industry. Medical device manufacturers selling products in the United States do not need to report actual or potential supply chain bottlenecks to the FDA

Contract Manufacturing Market Regional Analysis/Insights:

The major countries covered in the contract manufacturing marketreport are the U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America

America is dominating the market in the forecast period due to the large number of healthcare services and demand of products for huge storage and repository

Asia-Pacific is considered the most lucrative period due to the increasing number of biobanks and hospitals in the region. Table of Contents:

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Contract Manufacturing Market to Witness Substantial Growth of ... - InvestorsObserver

5 Takeaways From House GOP’s First Hearing on COVID-19 – Heritage.org

Long-awaited congressional investigations into COVID-19 are underway, with results so far that are wide ranging and informative.

Among those testifying during the initial inquiry under the Houses new Republican leadership were Dr. Lawrence Tabak, acting director of the National Institutes of Health; Dr. Rochelle Walensky, director of the Centers for Disease Control and Prevention; and Dr. Robert Califf, administrator of the U.S. Food and Drug Administration.

Here are five particularly noteworthy revelations, based on the transcript of sworn testimony in the hearing held Feb. 8 by two House subcommittees.

1. COVID-19 Vaccines Dont Prevent Transmission of Virus

Walensky, director of the CDC, confirmed that the vaccines largely were ineffective in preventing viral transmission from person to person.

Rep. Larry Bucshon, R-Ind., a physician, drilled down onthe rationalefor the Biden administrations vaccine mandates, including the mandate for foreign travelers. In his exchange with Walensky, Bucshon observed that we have just now discussed the fact that we know that [vaccination] doesnt prevent transmission.

It will prevent the individual from getting really sick, Bucshon said of vaccination against COVID-19, but it doesnt prevent the risk of someone coming into the country and spreading it to other people.

So, it does prevent severe disease and death. It doesnt prevent transmission as well as it did for prior variants [of COVID-19], but it does still prevent some, Walensky responded.

For the record: The Houserecently repealedthe Biden administrations vaccine mandate for foreign travelers as well as its mandate for members of the military.

2. CDC Stands by Masking Students at School

Walensky continued to recommend the masking of schoolchildren.

Rep. Cathy McMorris Rodgers, R-Wash., noted a dearth of reliable studies in the professional literature that demonstrate the efficacy of face masks against COVID-19. Rodgers cited Cochrane Reviews recent comprehensive examination of professional studies on the use of face masks and other nonpharmaceutical interventions to control the spread of disease.

Concerning medical or surgical masks and COVID-19, the authors of the Cochrane Review studyconcluded:

Compared with wearing no mask in the community studies only, wearing a mask may make little or no difference in how many people caught a flu-like illness/Covid-like illness (9 studies; 276,917 people); and probably makes little or no difference in how many people have flu/Covid confirmed by a laboratory test (6 studies; 13,919 people).

Conceding that this was an important study, Walensky went on to say: But the Cochrane Review only includes randomized clinical trials. And as you can imagine, many of the randomized clinical trials that were included in that were for other respiratory viruses, not COVID-19. Some of them were for COVID, just to be clear.

But it is very different for COVID-19, she said, because you have a virus that is different from the flu, potentially different from SARS or MERS, [and it] transmits before you actually have symptoms.

For the record: A randomized, controlled study, such as those included in the Cochrane Review, is the gold standard for scientific research.

While questioning Walensky, Rodgers noted that the school masking policy exacted a serious emotional, mental, and physical toll on schoolchildren. The Washington Republican observed that the CDC is currently the only national or international public health agency that recommends masking 2-year-old children.

Rodgers asked for a timeline for updating CDC guidance during the pandemic.

Walensky replied: You know, our masking guidance doesnt really change with time. What it changes with is disease. So, when there is a lot of disease in a community, we recommend that those communities and those schools mask. When there is less disease in the community, we recommend that those masks come off.

For the record: During this exchange, Walensky didnt present or refer to any scientific study that supports the efficacy of masking schoolchildren.

3. Did America Fund Disputed Coronavirus Research in Wuhan?

Whether the U.S. funded gain of function research at Chinas Wuhan Institute of Virology remains a mystery.

Rep. Buddy Carter, R-Ga., noted that Section 2315 of Congress omnibus spending bill for fiscal year 2023 bans government funding of certain types of research involving pathogens of pandemic potential. The purpose is to outlaw federal funding for gain of function research that strengthens viruses, particularly in China, North Korea, Russia, and Iran.

Tabak, acting director of the National Institutes of Health, noted in his testimony that in gain-of-function research, scientists engineer a virus and try to make it more transmissible and more pathogenic.

Tabak told lawmakers that NIH isnt conducting that type of research and hasnt funded it in the past, except for research into influenza in the Netherlands many years ago.

But in a series of follow-up questions, Rep. Debbie Lesko, R-Ariz., reopened the issue of gain-of-function research into coronaviruses at the Wuhan lab in China andthe roleof EcoHealth Alliance, a nonprofit that got substantial funding from NIH.

Lesko noted that NIH was unable to secure EcoHealths records of mice experiments at the Wuhan lab to determine the potential of infection for humans. Tabak responded that NIH terminated EcoHealths funding because the nonprofit had failed to provide adequate documentation of the experiments.

They are no longer funded by NIH to do anything, Tabak said of EcoHealth Alliance.

Lesko pressed the key point: Yes, I guess what I am saying is that if youif wecouldnt get the reports accurately, how can you definitively say that there was no [U.S.] funding of this?

Lesko pointed to EcoHealth Alliances continued failure to submit the proper reports for over two years and NIHs failure to conduct proper oversight over EcoHealths work in Wuhan.

Tabak responded that the administrative problems were genuine and were being corrected, but he emphasized that the viruses under study in EcoHealths subproject were genetically distinct from SARS-CoV-2, the scientific name for the novel coronavirus that caused COVID-19.

4. Inadequate Testing for Natural Immunity

Rep. Neal Patrick Dunn, R-Fla., also a physician, emphasized that diagnostic testing was another key failure in the federal governments response to COVID-19.

T cells, or T lymphocytes, developed from stem cells in the bone marrow, fight pathogens, and are a critical component of the human immune system.

We have known for over 10 years that the principal source of immunitythe principal mediator of immunity to coronavirusesare in T cells, not B cells, Dunn said, adding:

However, to this day, we lack coverage for any cellular immunity testing in this countrythat is, the T-cell testing that you see. NIH and CDC have ignored this kind of testing, despite the fact that we know this. This is the way the coronaviruses are principallyto the degree that we have long-lasting immunity from any coronavirus, it is mediated in T cells. Still, no coverage.

Tabak acknowledged Dunns point and responded that NIH is looking into T-cell testing.

5. Failure to Take Full Advantage of Therapeutics

Dunn also cited the initial shortage of therapeutics recommended by federal officials for early outpatient treatment of COVID-19.

I mean, we had guidance nationally [from CDC] that basically said, If you test positive, go home, quarantine, wait until your lips turn blue, and then go to the hospital and maybe we can save you. That was it, the Florida Republican said.

Dunn noted that broad spectrum antivirals to combat COVID-19 were in wide use in other countries. In Japan, he said, patients had access to favipiravir, an antiviral drug that had undergone 96 clinical trials.

Califf, the FDA administrator, responded that he would look into the quality of the trials for favipiravir and follow up.

Dunn reminded Califf: Japanese trials are pretty high-quality medicine.

Next Up: The Wuhan Lab Leak

Given the Department of Energys recent conclusion that the deadly pandemiclikely originatedin a leak from the research lab in Wuhan, congressional investigators will dig deeper. How cooperative government witnesses will be is an open question.

Beyond the origins of COVID-19, abroad rangeof related issues merits Congress attention, including theattempted suppressionof legitimate scientific dissent.

At the outset of this first House hearing, Rep. Morgan Griffith, R-Va., noted that between March 18, 2021, and Nov. 30, 2022, he and his colleagues sent 14 letters to NIH requesting information, and most have gone completely unanswered.

The most recent letter requested that NIH not destroy any evidence related to the pandemic.

A commonand bipartisantheme of the hearing was the need to restore public trust in public health. Given the experience of the past three years, only an aggressivecongressional oversight agendacan begin to rebuild that trust.

Its a monumental task.

This piece originally appeared in The Daily Signal

See the rest here:
5 Takeaways From House GOP's First Hearing on COVID-19 - Heritage.org

FDA Approvals, Highlights, and Summaries: Internal Medicine – Medscape Reference

Neurology Relyvrio (sodium phenylbutyrate/taurursodiol)

Sodium phenylbutyrate/taurursodiol is indicated for treatment of amyotrophic lateral sclerosis (ALS) in adults. The precise mechanism is unknown. Sodium phenylbutyrate is a histone deacetylase inhibitor shown to upregulate heat-shock proteins and act as a small molecular chaperone, thereby ameliorating toxicity from endoplasmic reticulum stress. Taurursodiol recovers mitochondrial bioenergetics deficits through several mechanisms, including by preventing translocation of the Bax protein into the mitochondrial membrane, thus reducing mitochondrial permeability and increasing the cells apoptotic threshold. The precise mechanism of action in patients with ALS is unknown.

Approval was based on the CENTAUR trial. The phase 2 CENTAUR trial (n = 89 treatment; n = 48 placebo) showed patients treated with sodium phenylbutyrate/taurursodiol had slower progression of disease compared to those randomized to placebo. Also, the ALS functional rating scale revised (ALSFRS-R) score showed the highest score preservation in fine motor skill subscales. N Engl J Med. 202 Sep 3:383(10):919-930

The open-label extension (OLE) of the CENTAUR trial included 56 participants from the treatment group (ie, early start group) and 34 from the placebo group. In the early start group subjects, the risk of any key event was 47% lower (p = 0.003), and the risk of death or tracheostomy or permanent assisted ventilation was 49% lower (p = 0.007). Also, first hospitalization was 44% lower in this group (p = 0.03). J Neurol Neruosurg Psychiatry. 2022 May 16;93(8):871-875

The CENTAUR OLE showed the median survival in the early start group was 25 months compared to 18.5 months for the group starting treatment during the extension. Muscle Nerve. 2021 Jan;63(1):31-39

Ublituximab is a CD20-directed monoclonal antibody indicated for relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.

Approval was supported by results from the ULTIMATE I (n = 549) and ULTIMATE II (n = 545) trials. Compared with teriflunomide, ublituximab resulted in lower annualized relapse rates and fewer brain lesions on MRI than teriflunomide over a period of 96 weeks but did not result in a significantly lower risk of worsening of disability. In the ULTIMATE I trial, the annualized relapse rate was 0.08 with ublituximab and 0.19 with teriflunomide (p < 0.001); in the ULTIMATE II trial, the annualized relapse rates were 0.09 and 0.18, respectively (p = 0.002). N Engl J Med. 2022 Aug 25;387(8):704-714

Elivaldogene autotemcel is a one-time gene therapy designed to add functional copies of the ABCD1 gene into a patients own hematopoietic stem cells, resulting in the production of the adrenoleukodystrophy protein (ALDP). It is indicated to slow the progression of neurologic dysfunction in boys aged 4 to 17 years with early, active cerebral adrenoleukodystrophy (CALD), a rare, X-linked, peroxisomal disorder that affects production of ALDP.

Accelerated approval from the FDA was supported by interim results from the phase 2/3 STARBEAM study, which concluded that elivaldogene autotemcel may be an effective alternative to allogeneic stem-cell transplantation in boys with early stage CALD. N Engl J Med. 2017 Oct 26;377(17):1630-1638

Ganaxolone is a GABAA receptorpositive modulator. It binds specifically to GABAA receptors to enhance their inhibitory effects. It is indicated for seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2 years and older. CDD, a rare developmental epileptic encephalopathy, is largely a disease of pediatric and young adult patients.

Approval was based on the phase 3 MARIGOLD trial. Patients treated with ganaxolone (n = 49) showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a 6.9% reduction for those receiving placebo (n = 51) (p=0.0036). In the open-label extension study, patients treated with ganaxolone for at least 12 months (n = 48) experienced a median 49.6% reduction in major motor seizure frequency. Prescribing Information

Other neurology approvals

DaTscan (ioflupane I 123) New indication as an adjunct to other diagnostic evaluations for striatal dopamine transporter visualization using single photon emission computed tomography (SPECT) brain imaging in adult patients with suspected Lewy body dementia

Adlarity (donepezil transdermal) New transdermal patch indicated for treatment of mild, moderate, and severe Alzheimer dementia; originally approved as tablet and oral disintegrating tablet

Hyftor (sirolimus topical) New dosage form indicated for treatment of facial angiofibroma associated with tuberous sclerosis in patients aged 6 years and older

Fintepla (fenfluramine) New indication for seizures associated with Lennox-Gastaut syndrome (previously approved for seizures associated with Dravet syndrome) in patients aged 2 years and older

Ultomiris (ravulizumab) C5 complement inhibitor, new indication for generalized myasthenia gravis

Radicava ORS (edaravone) New oral suspension dosage form for adults with ALS

Dextromethorphan/bupropion is a new fixed-dose combination product indicated for treatment of adults with major depressive disorder (MDD). Dextromethorphan is an antagonist of the N-methyl-D-aspartate (NMDA) receptor. The tablet is formulated to increase the bioavailability and half-life of dextromethorphan by utilizing the bupropion component to increase plasma dextromethorphan concentrations by inhibiting its metabolism.

In the phase 3 GEMINI (glutamatergic and monoaminergic modulation in depression) study, there were significant improvements in depressive symptoms compared to placebo starting 1 week after treatment initiation. J Clin Psychiatry 2022 May 30;83(4)

Daridorexant is a dual orexin receptor antagonist. The orexin neuropeptide signaling system plays a role in wakefulness. Blocking the binding of wake-promoting neuropeptides orexin A and orexin B to receptors OX1R and OX2R is thought to suppress wake drive. Daridorexant is indicated for adults with insomnia characterized by difficulties with sleep onset and/or sleep maintenance.

Approval was based on two phase 3 multicenter, randomized, double-blind, placebo-controlled trials. Participants were randomized 1:1:1 to receive daridorexant 50 mg, daridorexant 25 mg, or placebo (study 1; n = 930); or daridorexant 25 mg, daridorexant 10 mg, or placebo (study 2; n = 307) every evening for 3 months. The primary endpoints were change from baseline in wake time after sleep onset (WASO) and latency to persistent sleep (LPS), measured by polysomnography, at months 1 and 3. In study 1, the daridorexant 25-mg and 50-mg doses showed a statistically significant improvement in all endpoints, compared to placebo, at both month 1 and month 3 (p<0.0001). In study 2, the daridorexant 25-mg dose showed statistically significant improvement in WASO at month 1 (p=0.0001) and month 3 (p=0.0028) compared to placebo. Lancet Neurol. 2022 Feb;21(2):125-139

Other psychiatry approvals

Vraylar (cariprazine) New indication for adjunctive therapy for major depressive disorder

Xelstrym (dextroamphetamine transdermal) New transdermal patch indicated for treatment of attention deficit hyperactivity disorder (ADHD) in adults and children aged 6 years and older

Nalmefene Opioid antagonist reintroduced to the US market for management of known or suspected opioid overdose

Qelbree (viloxazine) Indication for ADHD expanded to include adults

Igalmi (dexmedetomidine) New buccal dosage form and indication for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder

Zulresso (brexanolone) Indication for postpartum depression expanded to include adolescents aged 15 years and older

Olipudase alfa is indicated for noncentral nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adults and children. It is a recombinant enzyme for replacement therapy. ASMD is a lysosomal storage disease that results from reduced activity of the enzyme acid sphingomyelinase (ASM), caused by pathogenic variants in the sphingomyelin phosphodiesterase 1 (SMPD1) gene. ASM degrades sphingomyelin to ceramide and phosphocholine. ASM deficiency causes intra-lysosomal accumulation of sphingomyelin (and cholesterol and other cell membrane lipids) in various tissues.

Approval of olipudase alfa was established by the ASCEND and ASCEND-Peds trials. The ASCEND trial assessed 31 adults with ASMD type A/B or type B and their percent predicted diffusing capacity of the lung for carbon monoxide and spleen volume. The olipudase alfa group demonstrated improved lung function and reduced splenomegaly compared to the placebo group from baseline to week 52 (increased predicted diffusing capacity for carbon monoxide, 22% vs 3%; spleen volume, 39% decrease vs 0.5% increase). In the ASCEND-Peds trial, 9 patients treated with olipudase alfa who were able to perform test at baseline saw improvement in lung performance from baseline to week 52 (mean increase of 33% predicted diffusing capacity for carbon monoxide). Genet Med. 2022 Jul;24(7):1425-1436 and Genet Med. 2021 Aug;23(8):1543-1550

Teplizumab is a humanized monoclonal antibody that targets the cluster of differentiation 3 (CD3) antigen, which is coexpressed with the T-cell receptor (TCR) on the surface of T-lymphocytes. It is indicated to delay the onset of stage 3 type 1 diabetes mellitus (T1DM) in adults and children aged 8 years and older who currently have stage 2 T1DM.

FDA approval was based on a phase 2, randomized, placebo-controlled trial involving 76 at -risk children and adults. The study demonstrated that a single 14-day regimen of daily IV infusions of teplizumab in 44 patients delayed clinical T1DM by a median of 2 years compared to 32 participants who received placebo (p = 0.006). N Engl J Med. 2019 Aug 15;381(7):603-613

Data from 3 years of follow-up (median 923 days) showed 50% of the teplizumab group remained diabetes free, compared to 22% of the placebo group (p = 0.01). Those who received teplizumab had a greater average C-peptide AUC compared to those given placebo, reflecting improved beta-cell function (1.96 vs 1.68 pmol/mL; p = 0.006). C-peptide levels declined over time in the placebo group, but they stabilized in patients who received teplizumab (p = 0.0015). Sci Transl Med. 2021 Mar 3;13(583)

Tirzepatide is indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus. It acts by dually targeting glucagonlike peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP). GIP is an incretin hormone that induces insulin secretion in response to a meal (primarily by hyperosmolarity of glucose in the duodenum) to facilitate the metabolism of carbohydrates, fats, and proteins. GLP-1 receptor agonists increase insulin secretion in the presence of elevated blood glucose, suppress glucagon postprandially, delay gastric emptying to decrease postprandial glucose, and decrease glucagon secretion.

The SURPASS clinical trials investigated the use of tirzepatide. SURPASS-2 was an open-label, 40-week, phase 3 trial. The comparison by Frias et al of tirzepatide versus semaglutide in patients with type 2 diabetes mellitus (SURPASS-2) found that tirzepatide at all doses was noninferior and superior to semaglutide in lowering HbA1c. Tirzepatide was also superior to semaglutide in body-weight reductions. N Engl J Med 2021 Aug 5;385(6):503-515

Other endocrinology approvals

Tymlos (abaloparatide) New indication to increase bone density in men with osteoporosis at high risk for fracture or patients who have failed on or are intolerant to other available osteoporosis therapy

Olpruva (sodium phenylbutyrate) New oral pellets for suspension indicated as an adjunct to dietary protein restriction and essential amino acid supplementation for the chronic management of patients who weigh at least 20 kg with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS)

Imcivree (setmelanotide) Indication for chronic weight management in patients aged 6 years and older with obesity caused by certain genetic disorders expanded to include Bardet-Biedl syndrome

Qsymia (phentermine/topiramate) Indication for chronic weight management expanded to include adolescents aged 12 years and older with an initial BMI in the 95th percentile or greater for age and sex

Mavacamten is a first-in-class cardiac myosin inhibitor indicated for symptomatic New York Heart Association class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve exercise capacity and symptoms in adults. Mavacamten modulates the number of myosin heads that can enter on actin (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM.

Approval of mavacamten was based on results from the multicenter, phase 3 EXPLORER-HCM trial (n = 251). Of 123 patients randomly assigned to mavacamten, 92 (75%) completed the Kansas City Cardiomyopathy Questionnaire (KCCQ) at baseline and week 30; and of the 128 patients randomly assigned to placebo, 88 (69%) completed the KCCQ at baseline and week 30. At 30 weeks, the change in KCCQ-OS (overall summary) score was greater with mavacamten than with placebo (mean score, 14.9 vs 5.4; difference +9.1; p<0.0001), with similar benefits across all KCCQ subscales. The proportion of patients with a very large change (KCCQ-OS 20 points or more) was 36% in the mavacamten group versus 15% in the placebo group, with an estimated absolute difference of 21%. These gains returned to baseline after treatment was stopped. Lancet. 2021 Jun 26

Other cardiology approvals

Furoscix (furosemide) New formulation and administration method: SC delivery by on-body infusor for treatment of congestion due to fluid overload in adults with NYHA class II/III CHF

Jardiance (empagliflozin) Indication to reduce the risk of cardiovascular death plus hospitalization in adults with heart failure (HF) broadened to include HF with either reduced or preserved ejection fraction.

Xigduo XR (dapagliflozin/metformin) New indication approved to reduce risk of CV death and hospitalization for HF in adults with T2DM who have HF (NYHA class II-IV) with reduced ejection fraction.

Deucravacitinib is an oral tyrosine kinase 2 (TYK2) inhibitor. TYK2 pairs with JAK1 to mediate multiple cytokine pathways and pairs with JAK2 to transmit signals shown in cell-based assays. The precise mechanism linking inhibition of TYK2 enzyme to therapeutic effectiveness is unknown. It is indicated for moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.

Approval was supported with results from the POETYK PSO-1 trial. At week 16, the PASI 75 (Psoriasis Area and Severity Index showing at least 75% reduction of symptoms from baseline) response rates were significantly higher with deucravacitinib than with placebo or apremilast (58.4% vs 12.7% vs 35.1%; p<0.0001). The static physicians global assessment score 0 / 1 (sPGA 0/1) also favored deucravacitinib (53.6% vs 7.2% vs 32.1%; p<0.0001). Efficacy improved beyond week 16 and was maintained through week 52. J Am Acad Dermatol. 2022 Jul 9

Spesolimab is an anti-interleukin-36 (IL-36) monoclonal antibody. Binding to the IL-36 receptor decreases release of proinflammatory and profibrotic pathways in patients with inflammatory dermatoses. It is indicated for treatment of generalized pustular psoriasis (GPP) flares.

Evidence from the Effisayil 1 trial supported the approval. The Effisayil 1 trial assessed 53 adults with GPP based on the Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation score at 1 week after receiving spesolimab or placebo. At the end of 1 week, the spesolimab treatment group saw improved pustulation. In patients treated with spesolimab, GPPGA pustulation subscore of 0 was observed in 54% compared to 6% in the placebo group. GPPGA total score of 0 or 1 was 43% with spesolimab compared to 11% for placebo. N Engl J Med. 2021;385(26):2431-2440

DaxibotulinumtoxinA is the first peptide-formulated, long-acting neuromodulator approved for temporary improvement of moderate-to-severe glabellar lines in adults. As a botulinum toxin, it blocks cholinergic transmission at neuromuscular junctions by inhibiting the release of acetylcholine.

Approval was supported by the SAKURA phase 3 trial, which included more than 2700 adults who received approximately 4200 treatments. The median duration of effect was 6 months, with some patients maintaining results at 9 months, compared to 3-4 months with other botulinum toxins. Dermatol Surg. 2021 Jan 1;47(1):48-54

Tapinarof is a first-in-class aryl hydrocarbon receptor (AhR) agonist for treatment of plaque psoriasis in adults. Efficacy of tapinarof in psoriasis is attributed to its binding and activation of AhR, a ligand-dependent transcription factor, leading to the downregulation of proinflammatory cytokines, including interleukin-17.

Approval was based on the PSOARING clinical trials, which compared use versus topical placebo. Approximately 35-40% of patients who received active drug had clear or almost clear scores after 12 weeks, as compared to 6% of patients on placebo. N Engl J Med 2021;385:2219-2229

Abrocitinib is an oral Janus kinase (JAK)-1 inhibitor indicated for refractory moderate-to-severe atopic dermatitis in adults whose disease is not adequately controlled with other systemic therapies or for whom those therapies are inadvisable. JAK1 inhibitors reduce interleukin-4 (IL-4) and IL-13 signaling.

Approval was based on the JADE COMPARE trial, which compared abrocitinib 200 mg or 100 mg once daily, dupilumab 300 mg SC every other week (after a 600-mg loading dose), and placebo. Additionally, all patients received topical therapy. An IGA response at week 12 was observed in 48.4% of patients in the 200-mg abrocitinib group, 36.6% in the 100-mg abrocitinib group, 36.5% in the dupilumab group, and 14% in the placebo group (p<0.001 for both abrocitinib doses vs placebo). An eczema area and severity index-75 (EASI-75) response at week 12 was observed in 70.3%, 58.7%, 58.1%, and 27.1%, respectively (p<0.001 for both abrocitinib doses vs placebo). The 200-mg dose, but not the 100-mg dose, of abrocitinib was superior to dupilumab with respect to itch response at week 2. N Engl J Med. 2021 Mar 25;384(12):1101-1112

Alpelisib is the first drug approved for patients aged 2 years and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. Klippel-Trenaunay-Weber syndrome is part of this spectrum of diseases. FDA approval of alpelisib was supported by real-world evidence from the open-label EPIK-P1 trial. A retrospective chart review showed patients treated with alpelisib had reduced target lesion volume and improvement in PROS-related symptoms and manifestations. After 24 weeks, 27% of patients (10/37) achieved a confirmed response to treatment, defined as 20% or greater reduction in the sum of PROS target lesion volume. Also, 23 of 31 patients (74%) showed some reduction in target lesion. Prescribing Information

Baricitinib is the first systemic treatment to gain FDA approval for adults with severe alopecia areata. Baricitinib is a Janus kinase (JAK) inhibitor that blocks phosphorylation and activation of signal transducers and activators of transcription (STATs), which modulate intracellular activity, including gene expression involved in the inflammatory pathway.

Approval was based on two phase 3 trials, BRAVE-AA1 and BRAVE-AA2. In BRAVE-AA1, 22% of the 184 patients who received baricitinib 2 mg and 35% of the 281 patients who received 4 mg achieved adequate scalp hair coverage, compared to 5% of the 189 patients who received placebo. In BRAVE-AA2, 17% of the 156 patients who received baricitinib 2 mg and 32% of the 234 patients who received 4 mg achieved adequate scalp hair coverage, compared to 3% of the 156 patients who received a placebo. Results were statistically significant for each treatment group compared to placebo (p<0.001). N Engl J Med 2022 May 5;386(18):1687-1699

Ruxolitinib topical cream gained approval in July 2022 for treatment of adults and adolescents aged 12 years and older with nonsegmental vitiligo. Approval was based on data from two phase 3 trials (TRuE-V1 and TRuE-V2) that evaluated the safety and efficacy of ruxolitinib cream compared to vehicle in more than 600 people.

Topical ruxolitinib resulted in significant improvements in vitiligo area scoring index (VASI), which represent improvements in facial and total body repigmentation, at week 24 (primary analysis) compared to vehicle and in an open-label extension at week 52.

Results at week 24, which were consistent across both studies, showed approximately 30% of patients treated with topical ruxolitinib achieved at least 75% improvement from baseline in facial repigmentation (F-VASI75), the primary endpoint, compared to approximately 8% and 13% of patients treated with vehicle in TRuE-V1 and TRuE-V2, respectively. At week 52, approximately 50% of ruxolitinib-treated patients achieved F-VASI75.

Additionally, at week 24, more than 15% of patients treated with topical ruxolitinib achieved at least 90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients treated with vehicle. At week 52, the percentage of ruxolitinib-treated patients who achieved F-VASI90 doubled to approximately 30%. Medscape Medical News

Other dermatology approvals

Dupixent (dupilumab) New indication approved for adults with prurigo nodularis

Rinvoq (upadacitinib) JAK inhibitor that gained approval by the FDA for refractory moderate-to-severe atopic dermatitis, including adults and adolescents aged 12 years and older

Skyrizi (risankizumab) New indication approved for adults with active psoriatic arthritis

Zoryve (roflumilast topical) New dosage form approved for adults and adolescents with plaque psoriasis

Juvederm Volbella XC (hyaluronic acid, non-animal stabilized) New indication approved for dermal filler for improvement of infraorbital hollowing

NexoBrid (anacaulase) Proteolytic enzymes indicated for eschar removal (debridement) in adults with deep partial-thickness and/or full-thickness thermal burns

Omidenepag isopropyl is a prodrug of omidenepag and a selective prostaglandin E2 (EP2) receptor agonist, which increases aqueous humor drainage. Omidenepag isopropyl is indicated for primary open-angle glaucoma (POAG) or ocular hypertension.

Approval was supported by data from a phase 3 open-label trial, RENGE, which evaluated the safety and efficacy of omidenepag isopropyl ophthalmic solution 0.002% in 125 patients with primary open-angle glaucoma or ocular hypertension. Omidenepag isopropyl ophthalmic solution demonstrated noninferiority to timolol ophthalmic solution 0.5% in the duration of the 52-week study. Japanese Journal of Ophthalmology. 2021 65:810819

Additionally, prostaglandin-associated periorbitopathy in patients with glaucoma has been reported not to be caused by EP2 receptor agonists but, rather, to be a cosmetic problem with prostaglandin F receptor (FP) agonists. Alleviation of periorbitopathy symptoms were reported after switching from an FP agonist to omidenepag isopropyl. Objective deepening of the upper eyelid sulcus improved by 76% at 7 months, and subjective questionnaires reported improvement in 95%. J Ocul Pharamcol Ther. 2022 Oct 31

Vabysmo (faricimab) First bispecific antibody for treatment of adults with neovascular (wet) age-related macular degeneration (AMD) and diabetic macular edema (DME). Faricimab targets 2 distinct pathways angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). By inhibiting VEGF-A, faricimab suppresses endothelial cell proliferation, neovascularization, and vascular permeability. By inhibiting Ang-2, faricimab promotes vascular stability and desensitizes blood vessels to the effects of VEGF-A. Ang-2 levels are increased in some patients with nAMD and DME.

Approval was based on the phase 3 TENAYA and LUCERNE studies for wet AMD and the YOSEMITE and RHINE studies for DME. The multicenter trial sites (n = 271) randomly assigned patients with wet AMD (TENAYA: n = 334 faricimab; n = 337 aflibercept) (LUCERNE: n = 331 faricimab; n = 327 aflibercept) to determine noninferiority. The primary endpoint was mean change in best-corrected visual acuity (BCVA) from baseline. BCVA with faricimab was noninferior to aflibercept in both studies, and ocular adverse events were comparable between faricimab and aflibercept. Lancet 2022 Jan 21

The YOSEMITE and RHINE studies (353 worldwide sites) randomized adults with vision loss due to center-involving diabetic macular edema to receive intravitreal faricimab 6 mg every 8 weeks, faricimab 6 mg per personalized treatment interval (PTI), or aflibercept 2 mg every 8 weeks up to week 100. The primary endpoint was mean change in best-corrected visual acuity at 1 year. Patients were randomly assigned to faricimab every 8 weeks (YOSEMITE, n = 315; RHINE, n = 317), faricimab PTI (n = 313; n = 319), or aflibercept every 8 weeks (n = 312; n = 315). Vision gains and anatomic improvements with faricimab were achieved with adjustable dosing up to every 16 weeks. Lancet 2022 Jan 21

Tebentafusp is the first drug approved for treatment of HLA-A*02:01-positive adults with unresectable or metastatic uveal melanoma (mUM). It is a first-in-class bispecific protein comprising a soluble T-cell receptor (TCR) fused to an anti-CD3 immune-effector function that specifically targets gp100, a lineage antigen expressed in melanocytes and melanoma. Immune-mobilizing monoclonal TCRs against cancer (ImmTAC) molecules bind cells that present a peptide derived from an antigen of interest, and they recruit T-cells to lyse the target cells.

Approval of tebentafusp was based on the results of the phase 3 IMCgp100-202 clinical trial, which evaluated overall survival (OS) of tebentafusp compared to investigators choice (either pembrolizumab, ipilimumab, or dacarbazine) in patients with previously untreated mUM. The trial randomized 378 patients in a 2:1 ratio to either tebentafusp or investigators choice. Results demonstrated OS was 73% in the tebentafusp group compared to 59% in the investigators-choice group (82% pembrolizumab; 13% ipilimumab; 6% dacarbazine) at 1 year (p<0.001). Progression-free survival was also significantly higher in the tebentafusp group than in the control group (31% vs. 19% at 6 months; p=0.01). N Engl J Med. 2021 Sep 23;385(13):1196-1206

Other ophthalmology approvals

Iheezo (chloroprocaine ophthalmic) New topical ophthalmic gel indicated for ocular surface anesthesia

Acuvue Theravision with Ketotifen (ketotifen, drug-eluting contact lens) Daily disposable contact lenses indicated for the prevention of ocular itch caused by allergic conjunctivitis and to provide vision correction in patients aged 11 years and older who do not have red eyes, who are suitable for contact lens wear, and who do not have more than 1.00 D of astigmatism

Beovu (brolucizumab intravitreal) New indication approved for diabetic macular edema

Iyuzeh (latanoprost) Preservative-free ophthalmic solution approved for elevated intraocular pressure

Terlipressin is a synthetic vasopressin with twice the selectivity for V1 receptors compared to V2 receptors. V1 vasopressin receptors are abundantly expressed in the mesenteric arteries as compared to other vascular areas, whereas V2 receptors are expressed in the renal tubules. The primary actions of V1 and V2 are stimulation of vasoconstriction and water resorption, respectively, resulting in decreased portal blood inflow and reduced portal hypertension. It is indicated to improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function.

Approval by the FDA in September 2022 was established by the CONFIRM trial, a phase 3, randomized, controlled trial that included patients with type 1 hepatorenal syndrome (HRS-1) and rapidly worsening renal function. This trial accessed percentage of patients with improved renal function via verified HRS reversal, without need for renal replacement therapy for 10 days after treatment. Verified HRS reversal was observed in 32% of those treated with terlipressin compared to 17% of those in the placebo group (p = 0.0006). Deaths due to respiratory disorders were higher in the terlipressin group compared to the placebo group after 90 days (11% vs 2%). Use of terlipressin is not recommended in patients with hypoxia (SpO2 <90%), and oxygenation levels should be monitored during treatment. N Engl J Med. 2021 Mar 4;384(9):818-828

Microbiota rectal is a live biotherapeutic delivered rectally to provide a broad consortium of diverse microbes to the gut to reduce recurrent Clostridioides difficile infection (CDI) after antibiotic treatment.

Approval was based on the PUNCH CD3 phase 3 clinical trial. Adults who had at least 1 CDI recurrence with a positive stool assay for C difficile and who were previously treated with standard-of-care antibiotics were randomly assigned 2:1 to receive a subsequent blinded, single-dose enema of microbiota or placebo. The primary endpoint was treatment success, defined as the absence of CDI diarrhea within 8 weeks of study treatment. In 267 patients who received blinded treatment (n = 180, microbiota; n = 87, placebo), successful treatment was 70.6% versus 57.5% with microbiota and placebo, respectively. More than 90% of the participants who achieved treatment success at 8 weeks had sustained response through 6 months in both the microbiota and the placebo groups. Drugs. 2022 Oct;82(15):1527-1538

Vonoprazan is a first-in-class potassium-competitive acid blocker (PCAB) for treatment of H pylori in combination with clarithromycin and/or amoxicillin. It suppresses basal and stimulated gastric acid secretion at the secretory surface of the gastric parietal cell through inhibition of the H+, K+-ATPase enzyme system in a potassium-competitive manner. The combinations are supplied as prepackaged 15-day dosage regimens.

Approval of vonoprazan double (DT) and triple (TT) therapies was based on the phase 3 PHALCON-HP trial. Each proved noninferior to lansoprazole triple therapy in patients with H pylori not resistant to ampicillin or clarithromycin: vonoprazan TT (84.7%; p<0.0001) and vonoprazan DT (78.5%; p=0.0037) vs lansoprazole TT (78.8%). Am J Gastroenterol 2021 Oct;116(S634)

Other gastroenterology approvals

Rinvoq (upadacitinib) New indication for moderate-to-severe active ulcerative colitis (UC) in adults who had inadequate response or intolerance to 1 or more TNF blockers

Dupixent (dupilumab) New indication approved for adults and adolescents for eosinophilic esophagitis

Skyrizi (risankizumab) New indication approved for adults with Crohn disease

Lenacapavir is a first-in-class, twice-yearly capsid inhibitor indicated for treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug-resistant infection. Lenacapavir directly binds to the interface between capsid protein (p24) subunits in hexamers, thereby interfering with multiple essential steps of the viral lifecycle, including capsid-mediated nuclear uptake of HIV-1 proviral DNA, virus assembly and release, and capsid core formation.

Approval was supported by data from the phase 2/3 CAPELLA trial, which evaluated lenacapavir in combination with an optimized background regimen in people with multidrug-resistant HIV-1. At week 26, a viral load of less than 50 copies/mL was reported in 81% of the patients in cohort 1 (blinded lenacapavir) and in 83% in cohort 2 (open-labeled lenacapavir. N Engl J Med. 2022 May 12;386;(19):1793-1803

Other infectious disease approvals

Jynneos (smallpox and monkeypox vaccine) is approved by the FDA as a 2-dose subcutaneous injection for prevention of smallpox and monkeypox disease in adults aged 18 years and older who are at high risk for smallpox or monkeypox infection. Because of limited global supply of the vaccine during the 2022 outbreak, emergency use authorization (EUA) was granted for adults to receive a lower 2-dose intradermal dosage. Additionally, the EUA allows children at risk to receive a 2-dose subcutaneous regimen.

Xofluza (baloxavir marboxil) Indication expanded for acute uncomplicated influenza in otherwise healthy patients to include children as young as 5 years

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FDA Approvals, Highlights, and Summaries: Internal Medicine - Medscape Reference

Tech Inventions That Changed The Health Industry Forever – SlashGear

Medical imaging is not, of course, a single technology, but an umbrella term for a bunch of different methods of getting a handle on what's going on within our bodies. One example might be a combination of these methods, like the Explorer total-body scanner, which performs both PET and CT scans. Without these advancements, we'd be devoting a lot more resources to palliative care. The tech in question includes x-rays, CT scans, MRIs, and ultrasounds, and each has changed the diagnostic landscape in its own way.

But x-rays themselves aren't just a diagnostic tool. They are used to guide surgeons, monitor the progress of therapies, and inform treatment strategies for the use of medical devices, cancer treatments, and blockages of various sorts. In 1896, the then-hyphenated New-York Times mocked Wilhelm Conrad Rntgen's medical application of X-ray imaging as an "alleged discovery of how to photograph the invisible." Five years later, Rntgen won the Nobel Prize in Physics. A century later, X-rays have replaced invasive surgeries and guesswork as a core diagnostic tool for doctors at every level.

Less a new imaging technology than a brilliant implementation of existing methods, computed tomography (CT) uses cross-sectional X-ray images acquired from various angles and computer algorithms to rapidly create a navigable, three-dimensional image of small or large parts of the body. Because it's based on X-rays, CT scans are better at imaging bones than soft tissues. CT scans provide many of the same benefits as other medical imaging methods, enhanced for many purposes by their speed and superior imaging of bones.

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Tech Inventions That Changed The Health Industry Forever - SlashGear