Category Archives: Stem Cell Treatments

Fans of bodybuilding are familiar with the health struggles that have plagued eight-time Mr. Olympia Ronnie Coleman in recent years. Coleman has endured over a dozen surgeries to his neck and back and, as a result, walks with crutches. Through it all, Coleman maintains a positive spirit and remains optimistic thanks in part to two stem cell treatments, which are starting to pay off.

Coleman joined co-host Giles Thomas in a recent episode of the Aint Nothing but a Podcast show. In the video, released on Dec. 29, 2021, Coleman says that hes beginning to feel like his old self again, and his weight gain reflects his health improvements.

Sporting a Ronnie Coleman Signature Series shirt that showed off his noticeable arm improvement, the Texas native his home revealed that hes back up to 285 pounds. And if youre having trouble believing that Coleman weighs close to what he did in his competitive prime, youre not alone.

I weighed myself five times on the scale downstairs, and I thought maybe its because Im downstairs,' Coleman said on the podcast episode. I was freaking out, so I went upstairs, and [that scale] was the same. I was like wow.'

BarBend reached out to the 2016 International Sports Hall of Fame inductee directly to follow up on what he revealed on his podcast. Coleman was happy to share more about his progress, including what he considers the best improvement of all the return of his signature leg size.

Thats the thing Im most proud of, Coleman tells BarBend. My legs had atrophied a whole lot since 2016 when I went in and had my first surgery 2017, same thing, 2018, 2019, 2020, same thing. I was just about to give up on it, you know. Then, suddenly, about four months ago, I started feeling a pump in my legs. And then I noticed the size had come back, and the atrophy was gone. I was geeked!

The man considered the most legendary bodybuilder of all time wasnt just known for being big. His freakish feats of strength including an 800-pound back squat and deadlift only added to the mystique he brought to the stage.

While Coleman isnt going to be moving that kind of weight anytime soon, hes been more active on social media, sharing training clips, such as the one below in which he performed a set of leg extensions on Dec. 12, 2021.

Coleman isnt leg-pressing 2,300 pounds as he did in his prime, but he is throwing more 45s on the machine nowadays than during his recovery.

Im back up to doing five plates, one each side, up from three per side a few months ago, Coleman says.

The eight-time Mr. Olympias improvements arent exclusively in the lower body. Coleman shared that hes getting stronger on numerous lifts in the gym. For example, Coleman is moving weight, performing 20 reps of rear lateral raises. The new size is evident, and his trademark smile was on full display during the set (see below).

My strength has come up a whole lot. Im going to say that its up about 40 percent.

He used the flat dumbbell press as another example, saying that he is now working with 70-pound dumbbells for his sets of 20 reps, which he does for every lift. He is training six days a week, as he did during his reign as the number one bodybuilder on the planet.

Returning leg strength is undoubtedly a strong sign that Colemans physical health is improving. That said, the former police officer mentions that itll still be a while before hes able to ditch the crutches.

My feet are still numb, and my quads are still numb, but theyre not quite as numb. I can start to feel them a little bit, he says. Once Im able to relieve this numbness, I will stand a much better chance of balancing myself.

Coleman says that the stem cell specialist told him that nerve regeneration takes about two years. As Coleman explains on the podcast, the specialists claim was verified when the numbness in his neck went away after two years, almost to the day. Coleman is confident that the same will happen with his lower extremities.

I thought about what the doctor said, and he was right. Thats what Im looking at now. It will be about two years before I get my full mobility and balance back. Then I can work on walking unassisted.

With his most recent stem cell treatment having taken place on Dec. 27, 2021, Coleman is optimistic that hell keep on progressing. This positive news caps off a good year overall for the 57-year-old icon.

In September, he was honored with the Arnold Classic Lifetime Achievement award by fellow Mr. Olympia Arnold Schwarzenegger. As great as that honor was, hes even more excited to get back out to events and meet fans now that he is in better shape and spirits I cant wait!

Featured Image: @ronniecoleman8 on Instagram

Originally posted here:
Exclusive: Ronnie Coleman on Recent Weight Gain, Current Strength, and Health Progress - BarBend

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

Did you know that every three minutes someone receives a diagnosis of a blood cancer? According to the National Foundation for Cancer Research, blood cancers account for nearly 10% of new cancer diagnoses in the United States annually. That means that approximately 178,520 people will receive a diagnosis this year most commonly with leukemia, lymphoma or myeloma. And although survival rates have increased dramatically over the past 20 years, theres still a long way to go when it comes to this patient population.

In this special issue of CURE, youll read about some of the research going on in blood cancer right now, including new treatment options for acute myeloid leukemia and a novel therapy using BK virus-specific T cells to treat BK virus-associated hemorrhagic cystitis (a painful side effect of stem cell transplants).

Dive into the issue for two patient stories, one about a three-time acute lymphoblastic leukemia survivor who now works as a nurse on the bone marrow transplant floor at Barnes-Jewish Hospital in St. Louis. This year also marks the 20th anniversary of the Food and Drug Administrations approval of Gleevec (imatinib mesylate), the tyrosine kinase inhibitor that saved Mel Mann. Who is Mel Mann? He is the longest-living survivor who was treated with the drug as a part of a clinical trial for chronic myeloid leukemia.

And because cancer is not a disease one should have to face alone, our cover story looks at the responsibilities and experiences of caregivers to patients with blood cancer. Not only will you read about what theyve gone through, but youll also find extra resources and tips for navigating the patient-caregiver relationship.

As always, thank you for reading.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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New Treatments and Tips for Dealing With Blood Cancer - Curetoday.com

Phovia is highly effective for treating superficial and deep skin infections.

This article is sponsored by Vetoquinol.

Superficial bacterial folliculitis, also called superficial pyoderma, is a commonly diagnosed dermatological condition in dogs.1,2 These infections are secondary to primary conditions affecting normal skin barrier function (eg, allergic skin disease, trauma, burns), keratinization (eg, nutritional deficiency, liver disease), and immune regulation (eg, neoplasia, autoimmunity, endocrinopathy).2 Cats less commonly develop superficial pyoderma perhaps because of decreased adhesion of staphylococci to feline corneocytes, but the primary issues causing infection are similar to those seen in dogs.3-8

The primary pathogen associated with superficial pyoderma in dogs and cats is a normal resident of the skin, Staphylococcus pseudintermedius, but other flora may be involved.2,8-12 As the normal homeostasis of this organism is disrupted from a primary disease, these gram-positive cocci invade deeper regions of the epidermis and hair follicle epithelium, increase in number, and enhance inflammation.

Classical clinical lesions of superficial pyoderma include papules and pustules that may eventually progress to alopecia, epidermal collarettes, scales, and crusts. Often the skin is erythematous and pruritic. Chronic cases may demonstrate lichenification, hyperpigmentation, and scarring alopecia from long-standing inflammation and infection.2 Cats may develop even more unique cutaneous reaction patterns and skin lesionsespecially when allergic skin disease is presentincluding miliary dermatitis, eosinophilic plaques, rodent ulcers, and eosinophilic granulomas.5

Identifying and addressing the primary disease is paramount in achieving complete, permanent resolution of the superficial pyoderma. Therefore, treatment is multifactorial and aimed at addressing the primary disease, reducing skin inflammation, and treating the infection directly. Current guidelines for the treatment of superficial pyoderma in dogs recommend the use of topical antimicrobials as sole therapy whenever possible; however, overuse of systemic antibiotics remains common.2,13-16

Topical therapy has many benefits including direct antimicrobial effects without use of an antibiotic, reduction in antibiotic-resistant bacterial populations, restoration of the normal skin barrier, enhancement of skin hydration, physical removal of keratinous debris, and removal of offending allergens from the haircoat.2,14 However, topical therapy is met with challenges that impede clinical application. Adherence is the biggest concern when recommending topical therapy to pet owners. Frequent bathing or application of medicated solutions to the skin can be difficult when busy owner lifestyles combine with a nonadherent patient. Skin inflammation can be painful and animals may be resistant to topical therapy. Cats are fastidious groomers and may lick away a medicated topical therapy before it can achieve appropriate contact time. Additionally, some topical agents can cause oral erosions and ulcerations or even gastrointestinal disturbance when groomed off. For these reasons, systemic antibiotics continue to be a common prescribing practice for superficial pyoderma.

All antibiotic use, despite duration or frequency, contributes to the development of antibiotic-resistant bacterial populations on the animal and in the environment.17-19 From that very first dose, bacteria are constantly evolving to implement inherent and acquired resistance mechanisms necessary for survival. One well-recognized mechanism is oxacillin resistance through the mecA gene, which produces a penicillin-binding protein receptor with poor affinity for -lactam antibiotics.2,14,15,20-23 Even more concerning than these oxacillin-resistant strains are those that develop multidrug resistance, which is defined as resistance to 3 or more antibiotic drug classes. This may happen over time with repeated antibiotic exposure or after a single dose of certain antibiotics such as fluorinated quinolones.2,20,23-25 The continued emergence of antibiotic-resistant bacteria inhibits the successful treatment of bacterial infections in pets and humans. As veterinarians consider how their antibiotic use contributes to this growing pandemic, they must look for alternative, safe, effective, affordable, and convenient antibacterial treatment modalities.

Phovia as a solution

Investigation into the photobiological effects of light therapy has been ongoing for the past 50 years. Photobiomodulation (PBM) therapy is a type of light treatment that uses visible or near infrared light to promote therapeutic benefits including induction of tissue healing and regeneration and inhibition of biological responses that induce pain or inflammation. The treatment distance, wavelength, fluence, pulse parameters, spot size, and irradiation time influence the effects of light energy on tissue. Visible light with wavelengths ranging from 400 to 700 nm can stimulate positive photobiomodulatory effects that promote wound healing, reduce inflammation and pain, modulate stem cell populations, and reduce bacterial contamination of wounds.26,27

Once visible light enters the skin, it is absorbed by the cells and initiates chemical changes dependent on the wavelength (or color) of light and the chromophore within the skin.27 Within each cell, membrane-bound organelles called mitochondria contain chromophores that absorb the light energy and begin making energy (adenosine triphosphate; ATP) via activation of cytochrome c oxidase. Outcomes of the mitochondrial respiratory pathway activation include stimulation of secondary messenger pathways, production of transcription factors and growth factors, and increased ATP production. However, excessive light energy exposure will overstimulate mitochondrial respiration and cause expenditure of all ATP reserves, which creates oxidative stress resulting in damaging elevations of nitric oxide, production of harmful free radicals, and activation of cytotoxic mitochondrial-signaling pathways leading to apoptosis.27,28 This is why creating PBM therapy protocols is important for targeting the beneficial effects while avoiding unintended harm.

Specific benefits of light energy within the visible light spectrum can be broken down into each color of light. Blue light (400-500 nm) has a lower penetration depth and primarily interacts with keratinocytes, reduces bacterial adhesion and growth, and increases intracellular calcium and osteoblast differentiation.29-31 Green light (495-570 nm) affects the superficial tissue and alters melanogenesis, reduces hyperpigmentation of the skin, and reduces tissue swelling.29,30 Red light (600-750 nm) penetrates deeper into the dermis and subcutis where it acts on cellular mitochondria to reduce inflammation and promote collagen synthesis through fibroblast proliferation and production of transforming growth factor-, fibroblast growth factor, platelet derived growth factor, and others.26-28,32,33 Red light has proliferative effects on mesenchymal stem cells and induces proliferation of epithelial colony forming units important for tissue repair and regeneration.34,35

Phovia, sold by Vetoquinol, is a form of fluorescent PBM therapy utilizing a blue light emitting diode (LED lamp, 400-460 nm) and topical photoconverter gel that emits low-energy fluorescent light (510-600 nm) when illuminated by the LED lamp.36,37 This interaction results in the formation of multiple wavelengths of visible light, each with a unique depth of penetration and effect on the tissue as described above. Application is fast and simple. The affected skin may be clipped free of hair and cellular debris removed with gentle cleaning. The skin is allowed to dry before application of the photoconverter gel. Just prior to application, 1 ampule of fluorescence chromophore gel is added to 1 container of photoconverter carrier gel and mixed thoroughly. The mixture is applied in a 2-mm layer to the affected skin, and the LED lamp is held 5 cm above the lesion and used to illuminate the area for 2 minutes. The gel is wiped away using saline-soaked gauze. The application can be repeated immediately after 5 to 10 minutes of rest or a second application can occur a few days later. Twice-weekly applications are continued until the wound is healed. Appropriate eyewear is required to protect the operator from the intensely bright light. Application is pain free and stress free for the patient, so sedation is not typically required.

Benefits of Phovia

Phovia shows great promise as a safe, effective therapy for treatment of numerous inflammatory dermatoses in dogs including superficial pyoderma,38 deep pyoderma,39 perianal fistula,40 interdigital dermatitis,41 calcinosis cutis,42 acute traumatic wounds,43 chronic wounds,37 surgical wounds,44 and otitis externa.45 Phovia as a sole therapy speeds time to healing by 36% in canine superficial pyoderma as compared with dogs receiving oral antibiotics alone.38 In one study, dogs with superficial pyoderma were treated with Phovia alone or with an oral antibiotic alone. Dogs treated twice weekly with Phovia demonstrated complete clinical healing in about 2.3 weeks (P < .05)whereas dogs receiving oral antibiotic healed in about 3.75 weeks.38 Additionally, Phovia speeds time to healing by nearly 50% in deep pyoderma when used with an oral antibiotic (5.7 weeks of treatment) compared with dogs receiving only oral antibiotic (11.7 weeks of treatment).39 The ability of this fluorescent PBM therapy to eliminate or significantly reduce duration of exposure to antibiotics will decrease the spread of antibiotic-resistant bacterial strains within pets and humans.

Phovias high safety profile makes it a beneficial tool to implement in everyday practice. Training the veterinary team to communicate therapy benefits with clients as well as to perform treatments is fast and easy. Training the veterinary technicians to perform treatments will give the veterinarian time to examine other patients. A single back-to-back application takes about 15 minutes, so pet owners can be in and out of the clinic quickly; however, the 2 weekly treatments can be separated by a few days if the veterinarian prefers to evaluate the patient more frequently. Additionally, when used as a sole therapy, clients are not required to administer oral or topical medications at home. This greatly improves treatment adherence and success. Instruct clients to use once-daily smartphone photos to document improvement at home. This can be useful when deciding how many treatments to perform. Most cases of superficial pyoderma will resolve completely by the third treatment.38 It is a good idea to communicate to clients that 3 to 4 weekly treatments may be required.

Conclusion

Phovia is a versatile, innovative therapeutic approach to numerous types of dermatitis.36 It is easy to implement in general practice, and is safe, pain free, and affordable. Phovia is highly effective for superficial and deep skin infections and eliminates the need for clients to administer numerous at-home treatments. This greatly improves the pet-owner bond and treatment outcomes by promoting adherence. Phovia accelerates time to wound healing, which decreases duration of antibiotic exposure and may reduce risk of antibiotic resistance development in these cases.2,13,36-39 Phovias efficacy against antibiotic-susceptible and antibiotic-resistant bacteria shows promise as an alternative therapeutic approach that promotes the principles of antimicrobial stewardship.36 If you are interested in purchasing this medical device for your practice, contact your Vetoquinol service representative.

Amelia G. White, DVM, MS, DACVD is an associate clinical professor of dermatology at Auburn University College of Veterinary Medicine.

REFERENCES

Original post:
Managing superficial pyoderma with light therapy - DVM 360

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapyPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR049Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-SequencingPresenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR058Date/Time: Friday, October 22, 2021 at 12:46 CEST

Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD)Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, ItalyAbstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product ManufacturePresenting Author: Saranya Elavazhagan, Orchard TherapeuticsAbstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIAPresenting Author: Brian Bigger, Ph.D., University of ManchesterAbstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsPresenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease backgroundPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapyPresenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapyPresenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P186

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Outlines Comprehensive Presence at the - GlobeNewswire

The first cell therapies made from a patients own cells were cancer treatments T cells engineered to hit tumors. Immusoft is taking a similar approach with a different type of immune cell, aiming to deliver these cell therapies into the brain. The biotech startup is still preclinical, but Takeda Pharmaceutical sees enough promise to begin a research and development alliance that could yield new treatments for rare neurometabolic diseases.

Seattle-based Immusoft develops its treatments by reprogramming B cells, a type of white blood cell. The process for making this treatment is nearly identical to that of CAR T, the first autologous cancer cell therapies. Blood is collected from the patient and the desired immune cells are selected. Those cells are engineered, then multiplied in a lab. After the process produces enough cells, that treatment is infused into the patient.

Besides the type of cell that is used, the key difference between CAR T and Immusofts approach is the engineering step. In CAR T, this stage involves engineering T cells to recognize a particular antigen on tumors. For Immusoft, this step means programing B cells with DNA that enables them to produce large amounts of therapeutic protein. Immusoft calls its technology Immune System Programing, or ISP.

Takeda and Immusoft did not specify the diseases they aim to address under their new alliance, though the companies said the collaboration will focus on delivering therapies across the blood-brain barrier, the protective layer that keeps certain substances, including some drugs, from reaching the organ. By reaching the brain, these B cell therapies have the potential to address a range of neurological disorders.

We continue to build our internal capabilities as well as partner with innovative companies early on in the discovery process to advance our next-generation gene and cell therapy ambitions for rare genetic and hematologic diseases, Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan said in a prepared statement. Working together with Immusoft, we hope to validate their ISP technology for [central nervous system] delivery of innovative therapeutics for rare neurometabolic diseases.

Rare neurometabolic disease is already one of the areas of focus for Immusoft. The rare enzyme deficiency Hurler syndrome is the target for its most advanced internal program, ISP-001. That cell therapy candidate is on track for an investigational new drug application filing by the end of this year, according to the companys website.

According to deal terms announced Wednesday, Takeda will pay Seattle-based Immusoft an upfront payment as well as research funding. The specific amounts were not disclosed. When the drug candidates covered under the partnership reach preclinical development, Takeda may elect to choose an unspecified number of them to continue their development. The Japanese pharma giant would owe option fees plus milestone payments tied to the progress of those programs. The companies gave no specific breakdown of those payments, other than to say the total value could top $900 million if all options are exercised and all milestones are achieved.

Working with B cells offer several advantages over other approaches to treating disease. Gene therapies delivered via engineered viruses cant be re-dosed because the antibodies that patients develop to the virus will render subsequent doses ineffective. By using a patients own cells, Immusoft aims to produce therapies that can be dosed multiple times. Also, gene therapies made by collecting a patients stem cells require a preconditioning step that knocks out the immune system to make room for the transplanted stem cells to grow. This step opens the door to a range of potential complications. Takedas recent dealmaking shows the pharma giants interest in avoiding therapies that employ viral delivery. In unveiling a multi-program alliance with Poseida Therapeutics on Tuesday, the pharma giant cited the biotechs non-viral technologies.

There are other companies developing ways to engineer B cells into new therapies. South San Francisco-based Walking Fish Therapeutics unveiled a $50 million Series A round of funding last month to support its research, still preclinical. Be Biopharma of Boston emerged nearly a year ago with a $52 million Series A financing.

Immusofts research so far has produced a preclinical drug pipeline spanning both rare and common diseases. Besides the Hurler syndrome candidate, the other rare disease programs include potential treatments for muscle-wasting disorders amyotrophic lateral sclerosis and Duchenne muscular dystrophy; the metabolic disease Hunter syndrome; and Pompe and Gaucher diseases, both enzyme deficiency disorders. Immusofts common disease research is still in the discovery stage. For common diseases, Immusoft is developing treatments for cardiovascular disorders, rheumatoid arthritis, and Parkinsons disease.

Image by Jolygon via Getty Images

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Taking aim at the brain, Takeda strikes up cell therapy R&D alliance with Immusoft - MedCity News

By Alycea Wood and Kamran Zamanian, Ph.D., iData Research Inc.

When choosing a treatment option for orthopedic procedures, biomaterials have become widely popular. Biomaterials are biomedical materials that can be safely implanted or injected into the body and are, more often than not, a form of biologically active tissue themselves.1 Their prevalence in orthopedic procedures is largely attributed to their ability to mimic the structure or properties of osseous tissue. Many products can offer a number of beneficial properties, such as promoting bone growth within the body (osteoinduction), promoting bone growth on the biomaterials scaffold (osteoconduction), or inducing the differentiation of stem cells into osseous tissue (osteogenesis).2,3 The orthopedic biomaterials market includes bone graft substitutes, growth factors, cellular allografts, cell therapy, hyaluronic acid viscosupplementation, and even cartilage repair devices. The U.S. orthopedic biomaterials market saw a dramatic dip and subsequent rebound in market value in 2020 and 2021 as a result of the COVID-19 pandemic. After recovery, the market is projected to see a consistently steady growth in value within the next few years. This growth is expected to be seen across all market segments apart from cellular allograft devices (Figure 1).4

Figure 1: Orthopedic biomaterials market growth trends by market segment, U.S., 20192028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

Cellular allografts may consist of either allograft bone (donated bone tissue) in conjunction with adipose-derived adult stem cells or viable cells within a cortical cancellous bone matrix.4,5 In both scenarios, the devices provide osteoconduction, osteoinduction, and osteogenesis to the site of implantation. Historically, this market had seen promising growth because of the optimal environment for bone growth they can provide.6 The cellular allograft market is projected to see a much slower rate of growth in market value in the next few years despite its market potential due to increased constraints on the market itself. These include, but are not limited to, direct federal restriction on product research, cost of product development, and product recalls.4

There is a strong interest in the scientific community in embryonic stem cell (ESC) research, which is largely due to ESCs high differentiability when compared to adult stem cell (ASC) lines.7 The development of new cellular allograft products, and the resulting growth in the market, is dependent on continued research into realizing the full medical potential of stem cell use. In 2019, the Trump administration eliminated federal funding of research relying on ESC tissue and instituted the National Institutes of Health (NIH) Human Fetal Tissue Research Ethics Advisory Board. This negatively impacted a large number of studies in progress while restricting the ability of new projects to commence.8,9,10 While the board was in effect, it rejected all but one application for funding.11 In April 2021, the Biden administration removed both the board and the restrictions on current projects, allowing federally funded research using ESC to continue.12 This was not the first instance where restrictions were placed and then removed on ESC research. In March 2009, President Obama signed an executive order to overturn the Bush administrations restriction on ESC research.13

The repeated restrictions on ESC research have a number of long-term ramifications in the development and implementation of new, effective cellular allograft treatments. Scientists may need to divert their research efforts away from stem cells and into less turbulent fields, and the progress of product development slows down as studies have funding pulled; this may contribute to increased hesitancy by end users to use stem cell products. Reduced research efforts, funding, and faith in stem cell products will continue to limit the growth of the cellular allograft market.

Cellular allografts tend to be notably more expensive than others within the broader cell-based biomaterials market. When compared to the cell therapy market, which uses either concentrated platelet-rich plasma (PRP) or bone marrow aspirate concentrate (BMAC) in its treatment, the cellular allograft average selling price (ASP) sits over three times higher (Figure 2).3

Figure 2: The average selling price (ASP) of the cellular allograft & cell therapy markets, U.S., 20182028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

The ASP of the cellular allograft market is so high because of the prohibitively expensive cost of developing new products. During the development process, reliable efficacy of a new product is uncertain, and using protein markers to help distinguish stem cell types can be very challenging.4,14 The increased cost of product development acts as a significant barrier to parties looking to enter the U.S. cellular allograft market. The result is fewer products entering and rejuvenating the market, and existing products sit at prohibitively high prices as they have low direct competition.4 The high cost of cellular allograft products hinders new entrants from introducing products and prevents end users from being able to afford existing ones. A broader consequence of this is end users turning to more affordable orthopedic biomaterial types to reduce procedural costs.

Any product recalls within the U.S. orthopedic biomaterials market, especially within cell-based therapies, will negatively impact the use of cellular allografts. This impact is amplified when a recall occurs within the market segment itself, which was seen in the cellular allograft market as recently as June 2021. On June 2, 2021, Aziyo Biologics recalled its product FiberCel following a number of patients contracting tuberculosis.15 Recalls deter the use of cell-based products through increased distrust in the safety of the products themselves, potential public backlash against the specific product itself or, in the market more broadly, reduced reimbursement from health insurance providers as well as the introduction of more restrictive FDA protocols. This is another reason why effective, safe, cell-based products are necessary for the cellular allograft market to move forward.

Conclusion

Federal research restrictions, high development costs, and product recalls all negatively impact the growth of the cellular allograft market in the United States. These constraints contribute to the projected low growth rate in market value in the coming years despite the potential uses for stem cell therapies. To shift the tide back toward growth, the cellular allograft space will need consistent research progress through large-scale studies, more affordable product development, and strict enforcement of sanitization protocols for existing products to prevent future product recalls. The large therapeutic potential of stem cell therapy has been discussed extensively in scientific and popular literature, but it may take a while to realize it.

References

About The Authors:

Alycea Wood is a research analyst at iData Research. She develops and composes syndicated research projects regarding the medical device industry, and published the U.S. Orthopedic Biomaterials report series.

Kamran Zamanian, Ph.D., is CEO and founding partner of iData Research. He has spent over 20 years working in the market research industry with a dedication to the study of medical devices used in the health of patients all over the globe.

About iData Research

For 16 years, iData Research has been a strong advocate for data-driven decision-making within the global medical device, dental, and pharmaceutical industries. By providing custom research and consulting solutions, iData empowers its clients to trust the source of data and make important strategic decisions with confidence.

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The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market - Med Device Online

15 October 2021

Perkins Coie LLP

To print this article, all you need is to be registered or login on Mondaq.com.

Key Takeaways:

On July 1, 2021, the FTC adopted aseries ofresolutionsauthorizing investigations into several areasdesignated as key law enforcement priorities during the next tenyears.One of thoseresolutionsdesignates "acts or practices affectinghealthcare markets" as an enforcement priority.

In the months after adopting this broad mandate, the FTC hasactively pursued false and deceptive advertising in healthcaremarkets.

For example, on August 16, 2021, the FTC and the GeorgiaAttorney Generalsuedthe co-founders ofthe Stem Cell Institute of America for false marketing inadvertising stem cell therapy to seniors. Specifically, thedefendants advertised their stem cell therapies as effectivelytreating orthopedic conditions and diseases. The FTC and theGeorgia Attorney General alleged that the defendants neverconducted any randomized clinical testing of the treatments tosupport these claims and that no other studies exist demonstratingthat stem cell treatments are effective in treating any orthopedicconditions or diseases.

Similarly, in early September 2021, theFTC sent cease-and-desistlettersto ten different companies demanding that they,among other things, stop selling unapproved diabetes products andmaking efficacy claims that are not supported by sufficientevidence.

These two actions, coming on the heels of the resolutionprioritizing enforcement in the healthcare industry, may portendaggressive FTC policing of healthcare-related advertising.Accordingly, companies offering healthcare services orproductsespecially those offering treatmentsshouldensure that their advertisements are backed up by competent andreliable scientific evidence.

The content of this article is intended to provide a generalguide to the subject matter. Specialist advice should be soughtabout your specific circumstances.

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Bryan Cave Leighton Paisner LLP

With government support instigated by the Covid-19 pandemic coming to an end, there is an inevitability that some hotel owners will sadly not have the liquidity to continue to operate in the medium term.

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FTC Follows Through On Prioritizing Investigations Into False Advertising In Healthcare Markets - Media, Telecoms, IT, Entertainment - United States -...

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Researchers hope stem cells will one day be effective in the treatment of many medical conditions and diseases. But unproven stem cell treatments can be unsafeso get all of the facts if youre considering any treatment.

Stem cells have been called everything from cure-alls to miracle treatments. But dont believe the hype. Some unscrupulous providers offer stem cell products that are both unapproved and unproven. So beware of potentially dangerous proceduresand confirm whats really being offered before you consider any treatment.

The facts: Stem cell therapies may offer the potential to treat diseases or conditions for which few treatments exist. Sometimes called the bodys master cells, stem cells are the cells that develop into blood, brain, bones, and all of the bodys organs. They have the potential to repair, restore, replace, and regenerate cells, and could possibly be used to treat many medical conditions and diseases.

But the U.S. Food and Drug Administration is concerned that some patients seeking cures and remedies are vulnerable to stem cell treatments that are illegal and potentially harmful. And the FDA is increasing its oversight and enforcement to protect people from dishonest and unscrupulous stem cell clinics, while continuing to encourage innovation so that the medical industry can properly harness the potential of stem cell products.

To do your part to stay safe, make sure that any stem cell treatment you are considering is either:

And see the boxed section below for more advice.

The FDA has the authority to regulate stem cell products in the United States.

Today, doctors routinely use stem cells that come from bone marrow or blood in transplant procedures to treat patients with cancer and disorders of the blood and immune system.

With limited exceptions, investigational products must also go through a thorough FDA review process as investigators prepare to determine the safety and effectiveness of products in well-controlled human studies, called clinical trials. The FDA has reviewed many stem cell products for use in these studies.

As part of the FDAs review, investigators must show how each product will be manufactured so the FDA can make sure appropriate steps are being taken to help assure the products safety, purity, and strength (potency). The FDA also requires sufficient data from animal studies to help evaluate any potential risks associated with product use. (You can learn more about clinical trials on the FDAs website.)

That said, some clinics may inappropriately advertise stem cell clinical trials without submitting an IND. Some clinics also may falsely advertise that FDA review and approval of the stem cell therapy is unnecessary. But when clinical trials are not conducted under an IND, it means that the FDA has not reviewed the experimental therapy to help make sure it is reasonably safe. So be cautious about these treatments.

About FDA-approved Products Derived from Stem Cells

The only stem cell-based products that are FDA-approved for use in the United States consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood.

These products are approved for limited use in patients with disorders that affect the body system that is involved in the production of blood (called the hematopoietic system). These FDA-approved stem cell products are listed on the FDA website. Bone marrow also is used for these treatments but is generally not regulated by the FDA for this use.

All medical treatments have benefits and risks. But unproven stem cell therapies can be particularly unsafe.

For instance, attendees at a 2016 FDA public workshop discussed several cases of severe adverse events. One patient became blind due to an injection of stem cells into the eye. Another patient received a spinal cord injection that caused the growth of a spinal tumor.

Other potential safety concerns for unproven treatments include:

Note: Even if stem cells are your own cells, there are still safety risks such as those noted above. In addition, if cells are manipulated after removal, there is a risk of contamination of the cells.

When stem cell products are used in unapproved waysor when they are processed in ways that are more than minimally manipulated, which relates to the nature and degree of processingthe FDA may take (and has already taken) a variety of administrative and judicial actions, including criminal enforcement, depending on the violations involved.

In August 2017, the FDA announced increased enforcement of regulations and oversight of stem cell clinics. To learn more, see the statement from FDA Commissioner Scott Gottlieb, M.D., on the FDA website.

And in March 2017, to further clarify the benefits and risks of stem cell therapy, the FDA published a perspective article in the New England Journal of Medicine.

The FDA will continue to help with the development and licensing of new stem cell therapies where the scientific evidence supports the products safety and effectiveness.

Know that the FDA plays a role in stem cell treatment oversight. You may be told that because these are your cells, the FDA does not need to review or approve the treatment. That is not true.

Stem cell products have the potential to treat many medical conditions and diseases. But for almost all of these products, it is not yet known whether the product has any benefitor if the product is safe to use.

If you're considering treatment in the United States:

If you're considering treatment in another country:

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FDA Warns About Stem Cell Therapies | FDA

This umbilical cord blood bank encourages parents to get the best healthcare option for their children.

When Anja Health founder Kathryn Cross was 3 and her brother was 1, he was in a near-drowning accident. His physicians quickly diagnosed him with cerebral palsy, and her parents began searching for a treatment. When they discovered umbilical cord blood, the possibilities that the future held suddenly widened. Umbilical cord blood was increasingly being studied as a treatment for cerebral palsy via clinical trials. Its versatile stem cells were already FDA-approved to treat 85+ diseases, and approximately 1,300 ongoing clinical trials were examining other use cases. Research for cerebral palsy in particular was promising, but the Cross family was ultimately unable to find an umbilical cord blood stem cell match that could have acted as a treatment.

When the founder later lost her brother to cerebral palsy and pneumonia complications, she realized that there was not only a limited supply of life-changing stem cells, but there was also limited awareness. In that moment, Anja Health was born.

Anja Health is a preventative care brand that offers Anja Health Family Defense, Anja Health Family Defense Plus, and Anja Health Family Defense Bundle to collect stem cells from the umbilical cord blood, cord tissue, and placenta.

With stem cell treatments unique ability to grow and develop into almost any cell type in the body, it has earned a reputation for being one of the most sought-after medical solutions and is now at the forefront of a brighter future for medicine. With its proven track record of saving lives, many parents now consider collecting their babys umbilical cord blood to preserve it for future use.

Anja Health collects, cryopreserves, and safe-keeps samples in its New-Jersey-based AABB-accredited, FDA-approved partner facility. It also updates its clients (even after birth and collection) of the newest stem cell uses to illustrate how clients can use samples when a need arises.

Stem cell treatments from banked cord blood, tissue, and placenta stem cells can be wonderfully life-altering for those who suffer from different diseases, said Anja Health founder Kathryn Cross. Today, banking is not simply an insurance policy in the low-probability case that your child gets a disease. Rather, if someone has access to stem cells and they stay up to date with its use cases as they develop, they will use stem cells at some point in their life.

Visithttps://anjahealth.com/for more information about the company and its services.

About Anja Health

Anja Health is a preventative care brand that provides services for collecting and preserving stem cells. Anja aims to bring the revolutionary benefits of stem cell treatments to individuals, especially those who suffer from various diseases.

Media ContactCompany Name: AnjaContact Person: Kathryn CrossEmail: Send EmailCountry: United StatesWebsite: https://anjahealth.com/

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Anja Health Disrupts Cord Blood Banking with Heartfelt Founding Story and Ground-Breaking Mission - Digital Journal