Category Archives: Stem Cell Clinic

‘Loving life’: 4-year-old not letting heart condition slow him down – Port Clinton News Herald

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Lucas Gutman, 4, listens along as Port Clinton City Council President Lisa Sarty reads an official proclamation from the city recognizing Conquering Congenital Heart Defect Week.(Photo: Jon Stinchcomb/News Herald)

PORT CLINTON - Though four years ago he was born with a condition making his heart critically underdeveloped, Lucas Gutman does not let it slow him down one bit.

In fact, Lucas is every bit as active as you would expect of a typical toddler and you would never know he was a child living with a congenital heart defect.

Hes playing soccer. He has tons of friends, said Jennifer Gutman, Lucas mother. Hes living life. Hes loving life.

His grandfather said Lucas has no shortage of energy. That was readily apparent when Lucas came to Port Clinton City Hall this week to be honored with an official proclamation.

The proclamation declared this week Conquering Congenital Heart Defect Week in the City of Port Clinton.

Lucas Gutman, 4, holds an official proclamation in honor of him from the City of Port Clinton. Gutman was diagnosed with hypoplastic left heart syndrome as an infant, but he does not let that slow him down.(Photo: Jon Stinchcomb/News Herald)

It coincides with a national week-long movement to raise awareness of what is actually a common but not widely known medical condition, congenital heart defects, or CHD.

Each year, the American Heart Association sponsors the Congenital Heart Defect Awareness Week from Feb. 7 to 14, an effort to encourage increased education about CHDs.

According to the federal Centers for Disease Control and Prevention, one in 100 babies are born with a heart defect, the most common type of birth defect. Annually, twice as many children die from congenital heart defects than all forms of childhood cancer combined.

Jennifer Gutman said she is so thankful to the city for the proclamation and efforts to raise awareness of CHD.

With awareness comes funding that we desperately need, she said. This is the Port Clinton that I know and love. The community that comes together for each other and Im very proud to be a citizen of Port Clinton.

The Gutman family has experienced the love of the close-knit local community before.

Lucas Gutman, of Port Clinton, was born in 2015 with hypoplastic left heart syndrome, meaning the left side of his heart was underdeveloped.(Photo: Submitted)

After Lucas was born and diagnosed, thanks to a fundraiser organized by the Port Clinton Fire Department, their very first Firelight Fund 5K glow run, the family was able to travel to Minnesota to participate in a clinical research trial at the Mayo Clinic in Rochester.

More: Firelight Fund to help local family as child battles rare disease

The form of CHD that Lucas was diagnosed with is hypoplastic left heart syndrome, or HLHS, which means the left side of the heart is critically underdeveloped.

According to the Mayo Clinic, typical treatment for HLHS usually involves medication, multiple surgeries, and can, in some cases, include a heart transplant.

However, the Mayo Clinics lead researcher on their Hypoplastic Left Heart Syndrome Program, Dr. Timothy Nelson, has been studying regenerative therapies like stem cell injections for treating HLHS.

The Gutman family decided to participate in the clinical trial and in December 2015, Lucas became the second child in the country to receive a stem cell injection from his own umbilical cord blood.

Since then, the results have been incredibly promising for Lucas.

His cardiologist is now saying that she wishes she could prescribe stem cells to all of her HLHS patients, Jennifer said. Hes doing great.

The Gutman family is taking it one step at a time as Lucas continues to live life like any other fun-loving toddler.

What the next 10-to-20 years will bring, we dont know yet, Jennifer said. But were faithful and we trust that hes going to show everybody what you can make happen with CHD. Were very proud of him.

Lucas Gutman, 4, listens along as Port Clinton City Council President Lisa Sarty reads an official proclamation from the city recognizing Conquering Congenital Heart Defect Week.(Photo: Jon Stinchcomb/News Herald)


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'Loving life': 4-year-old not letting heart condition slow him down - Port Clinton News Herald

Cancer Stem Cell Therapy Market Trends, Key Players, Overview, Competitive Breakdown and Regional Forecast by 2025 Mathematics Market Methods – Keep…

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The report segments the Global Cancer Stem Cell Therapy Market as In market segmentation by types of Cancer Stem Cell Therapy, the report covers Autologous Stem Cell TransplantsAllogeneic Stem Cell TransplantsSyngeneic Stem Cell TransplantsOther

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Major Topics Covered in this Report Chapter 1 Study Coverage Chapter 2 Executive Summary Chapter 3 Market Size by Manufacturers Chapter 4 Production by Regions Chapter 5 Consumption by Regions Chapter 6 Market Size by Type Chapter 7 Market Size by Application Chapter 8 Manufacturers Profiles Chapter 9 Production Forecasts Chapter 10 Consumption Forecast Chapter 11 Upstream, Industry Chain and Downstream Customers Analysis Chapter 12 Opportunities & Challenges, Threat and Affecting Factors Chapter 13 Key Findings Chapter 14 Appendix

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Regenexx, First to Use Stem Cells in Orthopedic Therapy, Marks 15th Anniversary – Yahoo Finance

More than 90,000 procedures performed

BROOMFIELD, Colo., Feb. 12, 2020 /PRNewswire/ --Regenexx, the world's largest, cohesive physician group dedicated to practicing advanced orthobiologics and the first to use stem cells in the treatment of many orthopedic injuries, is celebrating its 15th year since inception.To date, more than 40,000 patients have been treated and the organization has performed more than 90,000 procedures.

In 2005, Dr. Chris Centeno and Dr. John Schultz, physicians in a small two-man pain management clinic in Colorado, were the first in the world to apply stem cells to treat many orthopedic injuries. Two years later they completed a 24 months, IRB-approved research study of the use of both culture-expanded and same-day stem cells to treat knee and hip arthritis as well as low-back degenerative disc disease. Today, there are more than 60 Regenexx affiliates worldwide, including India, Australia, the UK, China, Taiwan and the Cayman Islands.

"Today, Regenexx physicians, specializing in the use of orthobiologics for treating orthopedic injuries, are achieving results thought unimaginable 15 years ago," says Chris Centeno, MD, founder and Chief Medical Officer of Regenexx. "Fifteen years ago, this new specialty focused on using the most advanced regenerative protocols available as an alternative to many orthopedic surgeries and today our patented lab-processing and treatment protocols have allowed us to achieve unmatched results."

Regenexx Corporate is the only program where orthobiologics can get coverage through private health insurance plans. As of Jan 1, 2020, Regenexx Corporate added 50 new self-funded companies that pay for orthobiologic care delivered by Regenexx providers as a way to reduce their orthopedic costs. Regenexx also received the EHIR traction award at Cohort 3 for record number of matches in October of 2019.

Regenexx physiciansmust have specific qualifications around musculoskeletal careand only the most qualified physicians are accepted into the Regenexx network. Once accepted, Regenexx then provides the doctor with hundreds of hours of specialized, hands-on training in the Regenexx interventional orthopedics approach. Regenexx has more than 60 clinic locations worldwide with highly specialized musculoskeletal physicians trained in more than 90 different Regenexx procedures.

"Making the decision between the interventional orthobiologics route and surgery is a real choice for most patients, not something driven by how much they can afford out of pocket," says Dr. Centeno. "We will continue to support and perform the research to make that happen. In the meantime, we expect to save hundreds of millions for our self-funded health plans and disrupt healthcare delivery in the process."

Regenexx Milestones

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About RegenexxRegenexx is the leader in advanced interventional orthobiologics through R&D, treatments, techniques, and training that reduce the reliance on surgical orthopedics. We strive to continuously innovate in regenerative advancements to get people better. Our commitment extends to lowering medical spending through our Regenexx Corporate Program, which provides less costly, less invasive, and less risky treatments than traditional orthopedic surgery.

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Regenexx, First to Use Stem Cells in Orthopedic Therapy, Marks 15th Anniversary - Yahoo Finance

What Drives the Domino Effect in Cancer Drug Resistance? – Technology Networks

KAIST researchers have identified mechanisms that relay prior acquired resistance to the first-line chemotherapy to the second-line targeted therapy, fueling a domino effect in cancer drug resistance. Their study featured in the February 7 edition of Science Advances suggests a new strategy for improving the second-line setting of cancer treatment for patients who showed resistance to anti-cancer drugs.

Resistance to cancer drugs is often managed in the clinic by chemotherapy and targeted therapy. Unlike chemotherapy that works by repressing fast-proliferating cells, targeted therapy blocks a single oncogenic pathway to halt tumor growth. In many cases, targeted therapy is engaged as a maintenance therapy or employed in the second-line after front-line chemotherapy.

A team of researchers led by Professor Yoosik Kim from the Department of Chemical and Biomolecular Engineering and the KAIST Institute for Health Science and Technology (KIHST) has discovered an unexpected resistance signature that occurs between chemotherapy and targeted therapy. The team further identified a set of integrated mechanisms that promotes this kind of sequential therapy resistance.

There have been multiple clinical accounts reflecting that targeted therapies tend to be least successful in patients who have exhausted all standard treatments, said the first author of the paper Mark Borris D. Aldonza. He continued, These accounts ignited our hypothesis that failed responses to some chemotherapies might speed up the evolution of resistance to other drugs, particularly those with specific targets.

Aldonza and his colleagues extracted large amounts of drug-resistance information from the open-source database the Genomics of Drug Sensitivity in Cancer (GDSC), which contains thousands of drug response data entries from various human cancer cell lines. Their big data analysis revealed that cancer cell lines resistant to chemotherapies classified as anti-mitotic drugs (AMDs), toxins that inhibit overacting cell division, are also resistant to a class of targeted therapies called epidermal growth factor receptor-tyrosine kinase inhibitors (EGFR-TKIs).

In all of the cancer types analyzed, more than 84 percent of those resistant to AMDs, representatively paclitaxel, were also resistant to at least nine EGFR-TKIs. In lung, pancreatic, and breast cancers where paclitaxel is often used as a first-line, standard-of-care regimen, greater than 92 percent showed resistance to EGFR-TKIs. Professor Kim said, It is surprising to see that such collateral resistance can occur specifically between two chemically different classes of drugs.

To figure out how failed responses to paclitaxel leads to resistance to EGFR-TKIs, the team validated co-resistance signatures that they found in the database by generating and analyzing a subset of slow-doubling, paclitaxel-resistant cancer models called persisters.

The results demonstrated that paclitaxel-resistant cancers remodel their stress response by first becoming more stem cell-like, evolving the ability to self-renew to adapt to more stressful conditions like drug exposures. More surprisingly, when the researchers characterized the metabolic state of the cells, EGFR-TKI persisters derived from paclitaxel-resistant cancer cells showed high dependencies to energy-producing processes such as glycolysis and glutaminolysis.

We found that, without an energy stimulus like glucose, these cells transform to becoming more senescent, a characteristic of cells that have arrested cell division. However, this senescence is controlled by stem cell factors, which the paclitaxel-resistant cancers use to escape from this arrested state given a favorable condition to re-grow, said Aldonza.

Professor Kim explained, Before this research, there was no reason to expect that acquiring the cancer stem cell phenotype that dramatically leads to a cascade of changes in cellular states affecting metabolism and cell death is linked with drug-specific sequential resistance between two classes of therapies.

He added, The expansion of our work to other working models of drug resistance in a much more clinically-relevant setting, perhaps in clinical trials, will take on increasing importance, as sequential treatment strategies will continue to be adapted to various forms of anti-cancer therapy regimens.

This study was supported by the Basic Science Research Program of the National Research Foundation of Korea (NRF-2016R1C1B2009886), and the KAIST Future Systems Healthcare Project (KAISTHEALTHCARE42) funded by the Korean Ministry of Science and ICT (MSIT). Undergraduate student Aldonza participated in this research project and presented the findings as the lead author as part of the Undergraduate Research Participation (URP) Program at KAIST.

Reference:Aldonza, et al. (2020) Prior acquired resistance to paclitaxel relays diverse EGFR-targeted therapy persistence mechanisms. Science Advances DOI: 10.1126/sciadv.aav7416

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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What Drives the Domino Effect in Cancer Drug Resistance? - Technology Networks

CRISPR, CAR-T, Consolidation: Top Advanced Therapy Milestones of 2019 – Xconomy


CRISPR, capacity, and consolidation powered the cell and gene therapy space in 2019, but a proactive focus on patient access topped Falcon Therapeutics CEO Susan Nichols annual roundup.

In what has become one of the most anticipated presentations at the Phacilitate Leaders World Conference, Susan Nichols, CEO of private North Carolina-based cell therapy firm Falcon Therapeutics, laid out the top 10 events of the previous year that shaped the regenerative medicine space, driving conversation, investment, and innovation.

The top spot in 2019 focused on efforts to increase patient access to the life-changing therapies entering the market, withMays approval of Novartis/AveXis Zolgensma(onasemnogene abeparvovec) and its unprecedented $2.1 million (1.9 million) price tag being the catalyst for change.

Nichols number one spot in 2018 centered around reimbursement conversations. Sparks approval of gene therapy Luxturna (voretigene neparvovec) shook 2017, while Europes approval ofex-vivostem cell gene therapy Strimvelis significantly advanced the sector in 2016.

For further context,check out theTop 10 cell and gene therapy milestones of 2018here, but below in reverse order is the full list of the top 10 key events of the previous 12 months, as presented at the conference in Miami, FL:

In December, a jury found Kite Pharma owned by Gilead Sciences (NASDAQ: GILD) guilty of infringing a patent exclusively licensed by Juno Therapeutics owned by Bristol-Myers Squibb (NYSE: BMS) from researchers at the Memorial Sloan Kettering Cancer Center.

The 190 patentrelates to technology used in Kite/Gileads chimeric antigen receptor (CAR) T-cell therapy Yescarta (axicabtagene ciloleucel).

The jurys decision left Gilead to pay $585 million plus 27.6% in royalties, totaling $752 million, to Bristol and Sloan Kettering, resolving a case filed a day after Yescarta won approval in October 2017.

But in a post-script that could well feature on Nichols 2020 list,it has been suggested the emboldening of Bristol has led the firm to file a motion last month to include punitive damages that would raise Gileads penalty to $1.5 billion.

Vertex (NASDAQ: VRTX) and CRISPR Therapeutics (NASDQ: CRSP) opened clinical trials in b-thalassemia and sickle cell disease to replace the defective genes that case these disorders andin November, the firms announced positive efficacy data from the first two patients treated with the investigational therapy CTX001.

Meanwhile, Editas Medicine (NASDAQ: EDIT) and Allergan initiated clinical trials for their CRISPR-based candidate AGN-151587 (EDIT-101), aimed at treating Leber congenital amaurosis 10 (LCA10), an inherited form of blindness.

The significance is CRISPR therapies have finally arrived in the clinic, Nichols said.

8) Pharma and biotech inhouse manufacturing

With a lack of third-party capacity especially for viral vector production, 2019 saw numerous investments by major cell and gene therapy players to grow their internal networks. Some of the examples Nichols pointed out include:

Susan Nichols, CEO of Falcon Therapeutics, spoke at Phacilitate in Miami, Florida in January

Positive data from Decembers American Association of Hematology (ASH) meeting in San Diego, CA was a further boon for the sector, said Nichols.

Johnson & Johnsons (NYSE: JNJ) JNJ-4528, a CAR-T Cell Therapy Directed Against B-Cell Maturation Antigen (BCMA), reported a 100% remission rate and response from its Phase Ib/II CARTITUDE-1 trial. 69% of patients showed complete remission or better.

The candidate licensed fromNanjing Legend in a $350 million deal will move into a full Phase II study this year.

ASH also brought positive news from bluebird and Bristol-Myers Squibb, which saw a 73.4% overall response rate in a Phase II KarMMa trial of its BCMA-targeted CAR-T candidate idecabtagene vicleucel.

The $950 million deal,announced in September, adds Semma Therapeutics a firm focusing on using stem-cell derived human islets as a possible cure for type 1 diabetes to Vertex growing regenerative medicine portfolio.

For Vertex, the deal represented its entry into the cell therapy space, complementing its move into gene editing just months prior with theacquisition of Exonics and a research expansion with CRISPR Therapeutics.

But for the industry, the investment in a company developing a cell therapy for a large indication other than cancer is of major significance, said Nichols.

As mentioned before, Astellas acquired Audentes for $3 billion, but the Japanese pharma firm also bought South San Francisco-basedCAR technology developer Xyphos Biosciencesas part of an end-of-year buying spree.

According to Nichols, these deals by Astellas are a signifier that medium pharma may be using advanced therapies to grow and expand.

With a wealth of therapies moving through the clinic, capacity is at a premium and 2019 saw contract development and manufacturing organizations (CDMOs) scrabbling to secure capabilities.

Thermo Fisher Scientific (NYSE: TMO) acquiredBrammer Bio for $1.7 billion, then Catalent (NYSE: CTLT) paid $1.2 billion toadd Paragon Bioservicesto its CDMO offering. Both marked the first move into gene therapy services by the two large contract manufacturers. Nichols noted the size of the deals as being somewhat impressive.

In other signs of CDMO consolidation, Hitachi Chemical Advanced Therapeutics Solutions (HCATS) entered Europe byacquiring German cell therapy manufacturing firm apceth Biopharma, and Tennessee-based cell therapy firm Cognateacquired Swedish DNA and viral vector manufacturer Cobra Biologics.

2019 also saw a flood of licensing deals with large upfront payments.

Roche (OTCGX: RHHBY) is paying more than $1 billion upfrontfor the rights to Sarepta Therapeutics (NASDAQ: SRPT) Duchenne muscular dystrophy (DMD) gene therapy outside of the US.

Genentech entered a $300 million with Adaptive Bio (NASDAQ: ADPT) for access to its T-receptor discovery and immune profiling platform, though the deal could be worth up to $2 billion.

And Vertex, as previously mentioned, inked a $175 million deal with CRISPR Therapeutics for its gene therapy pipeline.

We saw medium pharma grow. We saw major licensing deals. We saw CDMO consolidation. But we also saw Big Pharmas buying power with the sector making a significant impact on the cell and gene therapy space in 2019.

The biggest deal sawBristol buy Celgenefor a whopping $74 billion, bringing with it several CAR-T programs.

But Roches $4.8 billionacquisition of Spark Therapeutics which has already seen commercial success with Luxturna was also significant, as was Biogens (NASDAQ: BIIB) $877 million purchase ofNightstar Therapeutics, Pfizers stake-in and optionto buy out Vivet, and Bayers acquisition of the remaining shares ofBlueRock Therapeutics.

These signal that Big Pharma is optimistic to M&A in the advance therapy space and the value that these therapies can bring, said Nichols.

The business model for this new breed of curative medicines is significantly different to that of traditional pharma and biologics, and patient access poses a challenge. With the arrival of Zolgensma and its $2.1 million price tag, the conversations have changed, and all elements of the industry have been forced to address how to manage patient access.

Zolgensma represents a life or death drug for 68% of pediatric patients with SMA1. The patients must be dosed before the age of two, yet only around ten states offer screening before this age.

We need to work as an industry to ensure reimbursement and access is in sync with approvals, said Nichols. However, she added, we must move the conversation to state level and bring state Medicaid and insurance companies to the core of the conversation.

The year saw positive signs that change is happening.

Nichols noted that patient advocacy voices are loud across all disease indications pushing for access to these next-generation medicines. Meanwhile Novartis suggested lottery-style free drug program despitesome criticism demonstrates industry itself is looking for innovative ways to improve access.

This article first appeared in Bioprocess Insider on January 27.

Image: iStock/PashaIgnatov

Dan Stanton is Xconomy's managing editor and is based in France. You can reach him at

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CRISPR, CAR-T, Consolidation: Top Advanced Therapy Milestones of 2019 - Xconomy

Science team designs a new CAR they say may work much better than BCMAs in fighting multiple myeloma – Endpoints News

Right now the big R&D play in the multiple myeloma field is centered around a raft of experimental BCMA approaches, including CAR-Ts. But a scientific team at the University of Utah says they may have found a better approach.

Focusing on the high rate of relapse using current therapies, as well as the waning efficacy of the clinical CAR-Ts, a team at the Huntsman Cancer Institute at the University of Utah built a new CAR focused on CD229.

That target, they say, goes after a molecule that endures through the course of the disease on the surface of cancer cells, including myeloma stem cells at the root of relapse.

We were dismayed that although some of our patients respond quite well to currently available immunotherapies, they relapsed as early as one year after treatment, says physician-scientist Djordje Atanackovic. We thought if we could target every last cancer cell in a patients body, including the cancer stem cell, this could make the critical difference and yield more durable, deeper responses to treatment.

The study was published in Nature Communications.

Working with a protein engineer, the team produced an antibody that could hook onto CD229, an essential part of their new CAR-T. And it checked out in preclinical animal and cell models leaving plenty of work ahead in the clinic if this ever gets to the marketplace.

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Science team designs a new CAR they say may work much better than BCMAs in fighting multiple myeloma - Endpoints News

Omeros: 2 Major Catalysts On The Horizon – Seeking Alpha

Back in the thirties we were told we must collectivize the nation because the people were so poor. Now we are told we must collectivize the nation because the people are so rich. William F. Buckley Jr.

Today, we revisit a 'Tier 3' biotech stock whose stock has been under some recent pressure. However, it has two potential significant catalysts on the horizon. We update our investment case on this intriguing small-cap concern in the paragraphs below.

Omeros Corporation (OMER) is a Seattle based commercial-stage biopharmaceutical concern focused on the development of small molecule and protein therapeutics for the treatment of inflammation, complement-mediated diseases, central nervous system disorders, and immune-related diseases, including cancer. The company has one commercial asset, one late-stage candidate being evaluated for three indications, and several early and preclinical compounds. Omeros was formed in 1994 and went public in 2009, raising net proceeds of $61.8 million at $10 a share. The company completed a secondary offering at $13.10 in December 2019 following the release of positive data on its late-stage candidate, OMS721 (narsoplimab). The current market capitalization of OMER is just under $700 million.


Omidria. Omeros revenue is generated from Omidria, a phenylephrine and ketorolac intraocular solution that is approved for use during cataract surgery or intraocular lens replacement to maintain pupil size by preventing intraoperative miosis (pupil constriction) and reducing postoperative pain. Omidria was launched in 2015 and in 2017 generated net revenue of $64.8 million. However, the Centers for Medicare and Medicaid Services (CMS) determined to let its separate reimbursement under Medicare Part B expire on January 1, 2018, causing sales to plummet ~90%. Fortunately for Omeros, an act of Congress circumvented the CMS and reinstated its pass-through status for two years starting October 1, 2018. Omidria sales rebounded, likely eclipsing $110 million in 2019.

The reason for the pushback from the CMS regarding Omidria has to do with the fact that its active ingredients (phenylephrine and ketorolac) have been around for decades and a similar solution can be prepared by surgeons at a fraction of Omidrias cost. Omeros continues to pursue permanent separate reimbursement for Omidria and the CMS left the door open, indicating a need to find non-opioid alternatives. However, despite the company providing evidence demonstrating Omidria use reduced the need for fentanyl by nearly 80%, the CMS own study suggested otherwise, and it declined to grant Omidria separate payment status. News of this rejection sent shares 16% lower on November 4, 2019.

The CMS continues to analyze and monitor Omidria, and Omeros will exhaust all legislative and administrative avenues to secure permanent or similar status before the September 2020 expiration, including bipartisan anti-opioid legislation that could grant Omidria separate payment status for up to an additional five years. Management remains confident in its ability to gain permanent or similar status beyond September 2020. If it does not prevail, the blow to its top line will be harsh but not likely as severe as in 2018, owing to Omidria receiving its own J-Code in October 2019, which expands separate payment across commercial Med Advantage and Medicaid insurers, as well as in the office setting.

It goes without saying that Omidrias status will alter by a number of years how fast the company will achieve cash-flow positive levels.


OMS721. In the meantime, Omeros has initiated a rolling BLA for OMS721, its monoclonal antibody (MAB) targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a protein involved in the activation of the complement system, a branch of the bodys immune system that destroys and removes foreign particles and is engaged in the bodys inflammatory response. OMS721 is currently being evaluated in the treatment of three diseases that are all the result of complement system dysfunction.

The indication for which Omeros is filing a BLA is hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a multifactorial disorder induced by systemic vascular endothelial injury that can be triggered by several mechanisms during the transplant process. It occurs in ~40% of the ~60,000 patients undergoing allogenic HSCT in the U.S. and EU annually and is characterized by aggressive blood clotting usually resulting in acute renal failure. Severe cases have a mortality rate north of 90%. There are currently no approved therapies for HSCT-TMA.

That may change as the FDA was impressed enough with February 2018 interim data from OMS721s Phase 2 HSCT-TMA trial, in which median overall survival in 19 patients improved to 347 days versus the historical norm of 21 days (p<0.0001), to treat the small proof-of-concept study as registrational. Omeros released additional data on December 4, 2019, showing OMS721 demonstrated a 68% complete responder rate and a 100-day mortality rate of 19% versus the historical norm of 53% in HSCT-TMA patients who received at least four weeks of dosing. This prompted a 6% rally in shares of OMER, the trading session before the secondary offering was announced.

It should be noted that there are other candidates in the clinic for the HSCT-TMA indication, including Alexions (ALXN) already approved (for other indications) C5 inhibitor Ultomiris. However, Alexion is well behind, planning to initiate a Phase 3 trial (pending FDA feedback) in 1H20. The same can be said regarding Akari Therapeutics (AKTX) nomacopan, which plans to initiate a Phase 3 pediatric study in 1Q20. These schedules should give OMS721 a significant jump on any competition, which should have its BLA completed in 1H20. In addition to Breakthrough Therapy designation from FDA, OMS721 has Orphan drug status in both the U.S. and Europe and will likely receive a priority review from the FDA for HSCT-TMA.

OMS721's second most advanced indication is Immunoglobulin A (IGA) nephropathy, an ailment characterized by inflammation and kidney damage due to a buildup of the IgA antigen that affects 130,000150,000 people in the US and ~200,000 people in Europe with no approved remedies. After positive data from a very small Phase 2 study in which OMS721 reduced proteinuria in IgA nephropathy patients by 50-90%, Omeros finalized the particulars of a Phase 3 trial with the FDA in January 2019. The trials primary endpoint is the same: the relatively novel reduction in proteinuria levels at week 36. By obtaining approval on this endpoint (versus say renal function as measured by estimated glomerular filtration rate), it could potentially shorten the approval process by several years. Enrollment in the ~280-patient study is ongoing and accelerating. For this indication, OMS721 has received Breakthrough Therapy designation from the FDA and Orphan status in both the U.S. and EU.

To date, OMS721 has not been menaced by any significant safety or tolerability issues, which will help it in its pursuit of approval in the treatment of atypical hemolytic uremic syndrome (aHUS), a very rare disorder characterized by uncontrolled activation of the bodys complement system, manifesting itself in strokes, heart attacks, and kidney failures. Approximately 65% of patients diagnosed with aHUS die, require dialysis, or incur permanent renal damage within one year after diagnosis. The only approved treatment on the market is Alexions mAb Soliris, which has a Black Box warning due to risk of fatal infections as a result of suppression of the immune system. In most instances, patients must be immunized with a meningococcal vaccine at least two weeks prior to first administration of Soliris.

Armed with Fast Track and Orphan designations, Omeros only needs to conduct a 40-patient, single-arm (i.e., no control group), open-label Phase 3 trial to satisfy both the FDA and EMA for accelerated and full approvals, respectively. To achieve full approval in the U.S., OMS721 will need to add ~40 patients to the study. The issue confronting Omeros is that the trial began enrollment in 4Q16 and three years later management has not provided any definitive timetable regarding the trials progress, providing a frustrating connotation of accelerated approval for investors.

OMS527. Omeros other clinical asset is OMS527, which is being investigated in patients with addictions and compulsive disorders. After a successful Phase 1 study readout in 3Q19, OMS527 is expected to enter a Phase 2a trial in 2020 with a focus on nicotine addiction.

OMS906 and GPR174. The company also has assets that have demonstrated promise in the pre-clinic. OMS906 is a MASP-3 inhibitor for paroxysmal nocturnal hemoglobinuria and other alternative pathway disorders. Pre-clinical research on GPR174 inhibition has displayed promise in immuno-oncology. OMS906 is expected to enter the clinic in 1H20; GPR174 inhibitors will see the clinic when the company has more resources.

On that front, Omeros raised net proceeds of $54.5 million in a December 2019 secondary, which should leave it with ~$70 million at YE19. It has convertible debt with a face value of $210 million ($155 million carrying value) due 2023. The company also has an untapped vehicle through which it can borrow 85% of its receivables up to $50 million. Its cash runway will be contingent on securing separate payment status for Omidria post-September 2020 and the cadence of its development programs.

Like the investment community, Street analysts are somewhat split on Omeros prospects with one outperform rating sandwiched in between two buys and two holds. Their median twelve-month price target, however, is around $25 a share.

There are some unknowns regarding Omeros. Besides Omidrias status, the timing surrounding the completion of two of its pivotal OMS721 trials is still unclear in one instance, after three years. What does seem clear is that the FDA wants to approve OMS721. Given the lack of approved remedies for these complement systems diseases, OMS721 has relatively low hurdles to jump. If eventually approved for all three indications, OMS721 has blockbuster potential. If Omidria obtains five years of separate payment status, it will pave the way for Omeros to finance its own R&D without any more trips to the capital markets. With many shots on goal and what appears to be a helping hand from the FDA, continued investment in the shares of OMER is merited.

Idealism is fine, but as it approaches reality, the costs become prohibitive. William F. Buckley

Bret Jensen is the Founder of and authors articles for the Biotech Forum, Busted IPO Forum, and Insiders Forum

The Biotech Forum sports one of the liveliest collections of seasoned biotech investors on Seeking Alpha along with a 20-stock model portfolio. Join us during every trading day on Live Chat where the community swaps trading ideas, breaking news and opinions around all things Biotech. Initiate your risk-free, two week trial into The Biotech Forum by clicking HERE.

Disclosure: I am/we are long ALXN,OMER. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Omeros: 2 Major Catalysts On The Horizon - Seeking Alpha

Blood In The Joint – Economic Times

When a major injury occurs, the joint swells. Part of the swelling is good, and part is bad. Lets look at what can be done to optimize the outcome.

Joint swelling after an injury is usually due to blood vessels being broken as tissue tears. These tears leak blood into the joint, which already holds about a milliliter of synovial (joint) fluid. Blood contains both healing factors and inflammatory agents, the latter mainly white blood cells. The swollen joint becomes inflamed, heated by the rush of blood into the joint spaces, and additionally swollen with the extracellular fluid that surrounds cells in normal tissue. This chicken soup of bioactive factors sends siren calls to the stem cells located throughout the body. These stem cells mobilize to the joint, fulfilling a range of key functions to stimulate the healing response.

So: What are the fluids that we want to have in the joint and which ones do we not want there?

As with most of biology, there are positive and negative aspects to each response. The white blood cells are primarily responsible for the inflammatory response and produce pain and more swelling. On the positive side, however, white cells are scavengers. They consume any foreign body or bacteria, clearing our injured tissues of some of the detritus produced when collagen tissue is ruptured.

The red blood cells carry oxygen to the damaged and healing tissue. Oxygen is required for most tissues to repair themselves. Sometimes, as with hyperbaric chambers, additional oxygen is provided to accelerate the healing. Red blood cells, however, have a toxic effect on articular cartilage, which is normally avascular (i.e., lacking blood vessels). Repeated exposure to these cells (hemarthrosis) becomes toxic to articular cartilage, leading to arthritic joints. People suffering from hemophilia, for example, have frequent episodes of bleeding into their joints, due to their deficient clotting mechanism, and unfortunately, frequently get joint arthritis.

Still smaller particles, called platelets, are packed with granules that contain potent growth factors. The platelets release their growth factor packets at the site of collagen injury. These factors stimulate the healing response, much as anabolic steroids stimulate muscle building. Other bioactive factors in the platelet granules are chemotacticmeaning they send out signals that recruit other cell types, such as macrophages, which eat the torn tissues and damaged cells. They induce the first type of macrophage to become Type II macrophages, which are directly involved in tissue repair. Several of these bioactive factors are the most potent stem cell recruitment compounds ever discovered. Since everyone has billions of stem cells living on their blood vessels, these factors do a great job of ramping up the healing response.

Now the question becomes: When you injure your joint and the joint swells, should the doctors draw off the blood and fluid? The answer is sometimes yesif it makes you feel better and lessens the pain from the swelling. But your doctor should then spin the aspirate down in a centrifuge, removing the white and red blood cells and concentrating the platelets. These platelets, with their multiple growth factor packets, can then be reinjected into the joint.

The next question is: Should all injured joints be injected with bioactive factors to accelerate healing? The answer is coming, as our research in this field continues. My intuition is yes: We will not let injured joints become arthritic if we can change the course of the injury from devastating to just inconvenient with a rapid return to normal. If we aim to reach age 100 playing the sports we love, we cant afford to let sports injuries become fatal for the joints.

DISCLAIMER : Views expressed above are the author's own.

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Blood In The Joint - Economic Times

PRACTICALLY ACTIVE: Screening is a smart way to start a healthier life – NWAOnline

I've been endeavoring to eat healthfully these days, but sometimes it seems like an uphill battle. I'm eating fewer carbohydrates and have noticed an improvement in my blood glucose levels. But there are those times i check "my sugars," and when they are too high, I can usually trace it back to something i ate.

I try not to eat too late at night, and if I do, I try to go for something that offers a good amount of protein. But there are other times that I get into something I shouldn't. Thankfully, after all these years, I've finally let it sink in that beating myself up for that does not help. I'm too old to deprive myself and old enough to know I need to do better for my health, my family and my future.

I had a physical a few years ago from stem to stern. I had a mammogram as well and got a pretty good report, considering I'm diabetic. I've worked to clean up my cholesterol. I do have blood pressure concerns, but at least I know about it and can work to improve my situation.

Insurance is expensive and out-of-pocket costs can stop us cold. How many people just endure health problems because they don't have the funds or insurance coverage? How many people have high insurance premiums and high deductibles to the point that their policy is basically something to have in case of a catastrophic event? Millions.

If you can't afford a full physical, there are options. It takes some investigative work and diligence, but there are clinics and nonprofit hospitals that could help.

While watching TV the other day, I saw a commercial for free health screenings courtesy of the Arkansas Minority Health Commission ( It's part of the Arkansas Department of Health. They screen for blood pressure, body mass index and blood glucose. Screening is a good place to start to get a grip on your health.

The screenings, which are open to everyone, are offered on the first Wednesday of every month from 10 a.m. to 2 p.m. at 1501 S. Main St., Suite A, Little Rock. For information, call (501) 686-2720.

I always wished I was a person who loved to exercise. As children, my sisters and I played outside for hours on end. But that was playing, I wanted to do it. These days I don't want to exercise, and I don't enjoy it. Some days I'm worn out by the time I get home. But I'm trying to let it sink in that for my health and well-being, I need to stop being so sedentary.

I know how important health care, exercise and healthful eating are to longevity. But it takes effort, and I have to really work on my motivation.

One aspect of health that I often don't consider is mental, especially in regard to aging. While diseases like Parkinson's or Alzheimer's might affect us regardless of our physical health, there are other problems that could beset us because we get stagnant and, dare I say it, lazy.

I ran across an article on the Mayo Clinic website about the association between cardiorespiratory fitness and brain health, particularly in gray matter and total brain volume, loss of which is involved in cognitive decline and aging.

Brain tissue is made up of gray matter, or nerve cell bodies, and nerve filaments, called white matter, that extend from the cells. It was found that increases in peak oxygen uptake were strongly associated with more gray matter volume.

According to Dr. Ronald Petersen, a Mayo Clinic neurologist, there is indirect evidence that regular aerobic exercise can have a positive impact on cognitive function, in addition to physical conditioning.

Cardiorespiratory fitness is a measure of how well your body is able to transport oxygen to your muscles during prolonged exercise, and how your muscles are able to absorb and use oxygen.

The experts recommend moderate and regular exercise about 150 minutes a week. The recommended exercises include things like running/jogging, swimming, cycling, aerobics and jumping rope. Choose an exercise that involves fast movements for an extended period of time. You can choose a variety of types to keep your training routine from growing stale.

Now, I'm on to finding workouts to suit my mobility limits. I've had results from chair workouts that include weight-bearing exercises too. I'll share what I find with you.

Email me at:

Style on 02/10/2020

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PRACTICALLY ACTIVE: Screening is a smart way to start a healthier life - NWAOnline

Anemia: Causes, symptoms and treatment –

Anemia also known as iron-poor blood is a condition that develops when either the blood doesn't have enough red blood cells or the concentration of hemoglobin in red blood cells is very low. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen from the lungs to the rest of the body. When there are fewer red blood cells than normal or low levels of hemoglobin, the body doesn't get enough oxygen-rich blood for healthy functioning, which is what causes the symptoms of anemia.

Anemia is the most common blood disorder in the United States, affecting nearly 3 million Americans, according to the Centers for Disease Control and Prevention (CDC).

The term anemia is a broad one that represents several hundred different conditions some of them mild and treatable, others that are quite serious, said Dr. Nancy Berliner, chief of hematology at Brigham and Women's Hospital in Boston. There are three reasons that people are anemic, Berliner said: Either their body can't make enough red blood cells, something is destroying the red blood cells faster than their body can make news ones or blood loss (from menstrual periods, colon polyps or a stomach ulcer, for example) is greater than blood cell production.

There are more than 400 different types of anemia, according to the Pacific Heart, Lung & Blood Institute. Here are a few of the more common and better understood types:

Iron-deficiency anemia: The most common form of anemia is caused by low-iron levels in the body. Humans need iron to make hemoglobin, and most of that iron comes from dietary sources. Iron-deficiency anemia can result from a poor diet or from blood loss through menstruation, surgery or internal bleeding.

Pregnancy also increases the body's need for iron because more blood is needed to supply oxygen to the developing fetus, which may quickly drain the body's available iron stores, leading to a deficit. Problems absorbing iron from food because of Crohn's disease or celiac disease can also result in anemia.

Vitamin deficiency anemia: Besides iron, the body also needs two different B-vitamins folate and B12 to make enough red blood cells. Not consuming enough B12 or folate in the diet or an inability to absorb enough of these vitamins can lead to deficient red blood cell production.

Sickle cell anemia or sickle cell disease (SDC): This inherited disease causes red blood cells to become crescent-shaped rather than round. Abnormally shaped red cells can break apart easily and clog small blood vessels, resulting in a shortage of red blood cells and episodes of pain, according to the Mayo Clinic. People become chronically anemic because the sickle-shaped red cells are not pliable and can't get through blood vessels to deliver oxygen, Berliner said.

SDC occurs most often in people from parts of the world where malaria is or was common, according to the CDC; the sickle cell trait may provide protection against severe forms of malaria. In the U.S., SDC affects an estimated 100,000 Americans.

Thalassemia: Thalassemia is an inherited blood disorder that results in lower-than-normal levels of hemoglobin. This type of anemia is caused by genetic mutations in one or more of the genes that control the production of hemoglobin, according to the National Heart, Lung & Blood Institute (NHLBI).

Aplastic anemia: Aplastic anemia is a rare, life-threatening condition that develops when bone marrow stops making enough new blood cells, including red cells, white cells and platelets.

Aplastic anemia may be caused by radiation and chemotherapy treatments, which can damage stem cells in bone marrow that produce blood cells. Some medications, exposure to toxic chemicals like pesticides, viral infections and autoimmune disorders can also affect bone marrow and slow blood cell production.

Hemolytic anemias: This disorder causes red blood cells to be destroyed faster than bone marrow can replace them. Hemolytic anemias may be caused by infections, leaky heart valves, autoimmune disorders or inherited abnormalities in red blood cells, according to the American Society of Hematology.

Anemia of inflammation: Also called anemia of chronic disease, anemia of inflammation commonly occurs in people with chronic conditions that cause inflammation. This includes people with infections, rheumatoid arthritis, inflammatory bowel disease, chronic kidney disease, HIV/AIDS and certain cancers, according to the National Institute of Diabetes and Digestive and Kidney Diseases.

When a person has a disease or infection that causes inflammation, the immune system responds in a way that changes how the body works, resulting in anemia. For example, inflammation suppresses the availability of iron, so the body may not use and store the mineral normally for healthy red blood cell production, Berliner said. Inflammation may also stop the kidneys from producing a hormone that promotes red blood cell production.

The risk for anemia is higher in people with a poor diet, intestinal disorders, chronic diseases and infections. Women who are menstruating or pregnant are also prone to the disorder.

The risk of anemia increases with age, and about 10% to 12% of people over 65 are anemic, Berliner said. But the condition is not a normal part of aging, so the cause should be investigated when it's diagnosed, she said. Older adults may develop anemia from chronic diseases, such as cancer, or iron-deficiency anemia from abnormal bleeding.

According to NHLBI, the following types of people have an increased risk of developing anemia:

Mild forms of anemia may not cause any symptoms. When signs and symptoms of anemia do occur, they may include the following, according to the NHLBI:

The first test used to diagnose anemia is a complete blood count, which measures different parts and features of the blood: It shows the number and average size of red blood cells, as well as the amount of hemoglobin. A lower-than-normal red blood cell count or low levels of hemoglobin indicate anemia is present.

If more testing is needed to determine the type of anemia, a blood sample can be examined under a microscope to check for abnormalities in the size and shape of the red cells, white cells and platelets.

Related: This man's taste buds disappeared because of a blood condition

The treatment of anemia depends on the specific type of anemia, Berliner said, and anemias caused by nutritional deficiencies respond well to changes in diet. People with iron-deficiency anemia may need to take supplemental iron for several months or longer to replenish blood levels of the mineral. Some people, especially pregnant women, may find it hard to take iron because it causes side effects, such as an upset stomach or constipation, Berliner said.

For vitamin-deficiency anemias, treatment with B12 or folate from supplements (or a B12 shot) and foods, can improve levels of these nutrients in the blood, Berliner said.

Serious problems, such as aplastic anemia, which involves bone marrow failure, may be treated with medications and blood transfusions. Severe forms of thalassemia might need frequent blood transfusions.

Treatment for sickle cell anemia may include pain medications, blood transfusions or a bone marrow transplant.

Additional resources:

This article is for informational purposes only, and is not meant to offer medical advice.

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Anemia: Causes, symptoms and treatment -