British Columbia Premier John Horgan has offered the NHL a place to play if the league can find a way to resume the season.

Speaking at a COVID-19 media briefing Wednesday, Horgan said he has written a letter to both NHL Commissioner Gary Bettman and NHL Players Association head Donald Fehr to let them know B.C. is a place to potentially restart the NHL assuming the games would be played without audiences, but instead played for television.

The NHL suspended its season March 12 with 189 regular-season games left.

Dr. Bonnie Henry, British Columbias provincial health officer, was asked Monday about Vancouver hosting NHL games with no fans and said: These are the types of things that we need to think about.

Ontario Premier Doug Ford said Tuesday the Maple Leafs parent company, MLSE, has been in contact with the province about the possibility of Toronto serving as a hockey pod for teams as well. Alberta Premier Jason Kenney and Bettman spoke last month about Edmonton as another potential hub city.

Laila Anderson continues to make an impact in the lives of others one year after she became part of the Blues run to the 2019 Stanley Cup title.

On Tuesday, Anderson, who has battled HLH, a disease that causes the body to make too many immune cells, took part in an all-day livestream event called Couch2Cure to benefit Be the Match. It served as not only a fundraiser, but also a call for people to become donors for patients seeking blood stem cell matches. She was joined by FOX play-by-play man Joe Buck and Blues PA announcer Tom Calhoun.

The event was a success, raising$1.45 million and resulting in 36,000 registries to the Be the Match program.

Laila spoke with NBC Sports Kathryn Tappen on Wednesday to talk about the Couch2Cure event, the importance of the Be the Match registry, and how shes doing one year after the Blues triumph.

MORE LAILA ANDERSON: Laila introduces Blues All-Stars with gusto Blues superfan enjoying life one year after bone marrow transplant Laila meets bone marrow donor Laila gets moment with Stanley Cup

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Welcome to the PHT Morning Skate, a collection of links from around the hockey world. Have a link you want to submit? Email us atphtblog@nbcsports.com.

A really nice read about ex-Blackhawks president John McDonoughs friendship with 11-year-old Cammy Babiarz, who is unable to walk or talk because of Rett Syndrome a rare developmental disorder. [Midway Minute]

It didnt last very long, but at one point in time Michael Jordan was a minority owner of the Capitals. [ESPN]

Economies are beginning to open up again, so too are hockey rinks in the U.S. [The Hockey News]

Georges Laraque opens about his up and down relationship with his father. [Vice]

How the 2011-12 Kings became unlikely Stanley Cup champions. [The Score]

Comparing Brady Tkachuks early days in the NHL to that of Mark Stones. [Silver Seven Sens]

What if some of NHLs all-time best hadnt run into historic dynasties? [Sportsnet]

Looking ahead to whats expected to be an intriguing 2022 NHL draft class. [Stephen Ellis]

Finally, the Wild music video you were looking for:

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

After pulling the plug on the 2019-20 season in March, the KHL has decided there will be no champion and the Gagarin Cup will not be awarded for the first time in league history.

Due to this decision, the league has equally ranked the eight teams that advanced to the second round of the playoffs:Ak Bars Kazan,Barys Nur-Sultan,CSKA Moscow,Dynamo Moscow,Jokerit Helsinki,Salavat Yulaev Ufa,Sibir Novosibirsk, and SKA St. Petersburg.

From the KHL:

With the season incomplete, there is no way that a Gagarin Cup winner and other prize winners can be fairly chosen based on the results of the regular season. To announce a champion based on the regular season and one round of the playoffs would violate the sporting integrity of the competition.

The Russian Hockey Federation has drawn up a separate procedure to determine the Russian Champion for the 2019-20 season, and to award silver and bronze medals to the second and third-placed teams. This proposal will be submitted to the KHL Board of Directors for approval.

The Gagarin Cup playoffs were halted in the conference semifinals after Jokerit and Barys pulled out due to the coronavirus pandemic. Originally, the KHL was planning for a one-week break to come up with a new format for the four remaining teams. They later chose to end the season completely.

Im sure that the league has taken a fair and balanced decision in this difficult situation, said KHL president Alexei Morozov. This was the only choice that respects our sporting principles. For the first time in history, the KHL season had to be interrupted and ultimately curtailed. That was a tough, but essential decision, dictated by the need to protect the health of the nation.

MORE: Bill Peters signs two-year deal to coach KHLs Avtomobilist

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

Brendan Leipsic of the Capitals has apologized for comments made on social media that were leaked online Wednesday. Panthers prospect Jack Rodewald was also in the Instagram group chat where remarks were made about the appearances of Meaghan Pearson, whose husband, Tanner, plays for the Canucks, Lauren Kyle, the girlfriend of Oilers forward Connor McDavid, and other women.

Yesterday my friends Instagram account was hacked and an individual circulated images that are representative of private conversations I was a part of, Leipsic wrote in an apology note posted on Twitter. I fully recognize how inappropriate and offensive these comments are and sincerely apologize to everyone for my actions. I am committed to learning from this and becoming a better person by taking time to determine how to move forward in an accountable, meaningful way. I am truly sorry.

The NHL released a statement of their own stating they will address this with the players involved.

The National Hockey League strongly condemns the misogynistic and reprehensible remarks made by players Brendan Leipsic and Jack Rodewald in a private group chat that has surfaced on social media. There is no place in our League for such statements, attitudes and behavior, no matter the forum. We will address this inexcusable conduct with the clubs and players involved.

Leipsic, who has played 61 games with the Capitals this season, is on his fifth team in five years since entering the NHL. His current team wrote in a statement,We are aware of the unacceptable and offensive comments made by Brendan Leipsic in a private conversation on social media. We will handle this matter internally.

Sean Leahy is a writer for Pro Hockey Talk on NBC Sports. Drop him a line at phtblog@nbcsports.com or follow him on Twitter @Sean_Leahy.

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NHL offered place to play in British Columbia - NBCSports.com

Bone Marrow Stem Cells Comments Off on NHL offered place to play in British Columbia – NBCSports.com | May 7th, 2020

A recent market study on the global Hematopoietic Stem Cell Transplantation (HSCT) market reveals that the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to reach a value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% during the forecast period (2019-2029). The impact of the COVID-19 pandemic on the global Hematopoietic Stem Cell Transplantation (HSCT) market is discussed in the presented study.

The Hematopoietic Stem Cell Transplantation (HSCT) market study encloses a thorough analysis of the overall competitive landscape and the company profiles of leading market players involved in the global Hematopoietic Stem Cell Transplantation (HSCT) market. Further, the presented study offers accurate insights pertaining to the different segments of the global Hematopoietic Stem Cell Transplantation (HSCT) market such as the market share, value, revenue, and more.

Request Sample Report @https://www.mrrse.com/sample/9594?source=atm

The following doubts are addressed in the market report:

Key Highlights of the Hematopoietic Stem Cell Transplantation (HSCT) Market Report

The presented report segregates the Hematopoietic Stem Cell Transplantation (HSCT) market into different segments to ensure the readers gain a complete understanding of the different aspects of the Hematopoietic Stem Cell Transplantation (HSCT) market.

Request For Discount On This Report @ https://www.mrrse.com/checkdiscount/9594?source=atm

Segmentation of the Hematopoietic Stem Cell Transplantation (HSCT) market

Competitive Outlook

This section of the report throws light on the recent mergers, collaborations, partnerships, and research and development activities within the Hematopoietic Stem Cell Transplantation (HSCT) market on a global scale. Further, a detailed assessment of the pricing, marketing, and product development strategies adopted by leading market players is included in the Hematopoietic Stem Cell Transplantation (HSCT) market report.

Companies Mentioned in the Report

The report profiles key manufacturers in the hematopoietic stem cell transplantation (HSCT) Market based on various attributes such as company details, SWOT analysis, strategic overview, financials, and business overview. Major players profiled in this report include Regen Biopharma, Inc., Escape Therapeutics, Inc., Lonza Group Ltd., and Pluristem Therapeutics Inc.

The global hematopoietic stem cell transplantation (HSCT) Market has been segmented as follows:

By Transplant Type

By Disease Indication

By Application

By Region

Buy This Report @ https://www.mrrse.com/checkout/9594?source=atm

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Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -...

Bone Marrow Stem Cells Comments Off on Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -… | May 6th, 2020

As the world continues to battle the coronavirus pandemic, scientists are looking towards gene therapy to find ways to develop vaccines for the Covid-19 virus. Gene therapy itself was developed based on how viruses work.

When a virus attacks a host, it introduces its genetic material into the host cell as part of its replication cycle. The genetic material serves as an instruction manual on how to produce more copies of the virus, hijacking the host body's normal production machinery to serve the needs of the virus. The host cells then produce additional copies of the virus, leading to more host cells being infected.

Like animals, humans have found a way to domesticate viruses as well, i.e., direct the virus's function to achieve favorable results, which is prominent in gene therapy. Such viruses which physically insert their genes into the host's genome could instead be used to carry "good" genes into a human cell. Scientists would first remove the genes in the virus that cause diseases, and replace those genes with genes encoding the desired effect.

All of this sounds quite sci-fi but it has been done numerous times in the past. Peter Kolchinsky, a virologist and a biotechnology investor, compiled how different viruses have been used for gene therapy in the past.

Kolchinsky tweeted, "SARS2 is a scary menace, but did you know that we've domesticated viruses? Like wolves vs dogs, we've tamed them, including some deadly ones, to perform many useful functions (and may help us stop SARS2)."

The human immunodeficiency virus (HIV) has killed millions of people. It works by disabling the host body's immune system until it can't defend the person against common, normally mild pathogens. Kolchinsky explained that HIV's special trick is to integrate its genome into that of the host body's cells.

This feature of HIV is used for gene therapy, as explained before, by replacing a chunk of the virus's genome with the hemoglobin gene to insert it into bone marrow stem cells of patients with sickle cell anemia, whose hemoglobin genes are malfunctioning.

Kolchinsky also tweeted, "Adenoviruses typically cause mild infections, including common colds. These, too, we are trying to use for gene therapies, particularly when we just want to temporarily make a protein in cells. One company is developing such an adenovirus gene therapy for heart disease to induce growth of new blood vessels when old ones are clogged. Another is using this virus to make oral vaccines that would otherwise require injection (eg flu vaccine pill). When we use a virus to deliver code for making something in cells, we call that a virus vector."

There is now a wealth of clinical experience with numerous vector types that include primarily vaccinia, measles, vesicular stomatitis virus (VSV), polio, reovirus, adenovirus, lentivirus, -retrovirus, adeno-associated virus (AAV) and herpes simplex virus (HSV).

However, as with all other procedures, viral vector-gene therapy has associated risks. Viruses can usually infect more than one type of cell, so, when viralvectorsare used to carrygenesinto the body, they might infect healthy cells as well as cancer cells.

Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. Moreover, when viruses are used to deliver DNA to cells inside the patient's body, there is a slight chance that this DNA could unintentionally be introduced into the patients reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.

One study to help find a vaccine for Covid-19 aims to use the principles behind gene therapy to get the vaccine ready. The researchers' method uses a harmless virus as a vector to bring DNA into the patient's cells. The DNA should then instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.

While a mass-produced vaccine may still take a while, this study is one of at least 90 vaccine projects around the world trying to find a cure for Covid-19. However, some experts are worried that a vaccine may never be available. According to our previous report, Dr David Nabarro, a professor of global health at Imperial College London, who also serves as a special envoy to the WHO on Covid-19, said, "There are some viruses that we still do not have vaccines against."

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough - MEAWW

Bone Marrow Stem Cells Comments Off on Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough – MEAWW | May 6th, 2020

MIKE TYSON revealed how he has started training again having not thrown a punch for a staggering 15 years - and is being aided by stem-cell research therapy.

The Baddest Man on the Planet, who hung up his gloves in 2005 following defeat by Kevin McBride, wouldn't elaborate on why he was having the treatment but added that it was 'really wild what scientists can do'.

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Tyson was having an Instagram chat with basketball legend Shaquille O'Neal when he revealed that he had been training for the previous three days after 15 years away - and his new health regime.

Iron Mike said: "You know what I had done? I had stem-cell research therapy.

"I feel like a different person but I can't comprehend why I feel this way. It's really wild what scientists can do."

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.

He did not reveal what the exact condition was that was being treated.

Despite letting the gloves gather dust in the corner, it didn't take long for Iron Mike to show off that lethal punching power.

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The 53-year-old delighted fans in lockdown by uploading a viral video of him laying into a punchbag with his trademark speed and power.

Tyson's weight ballooned after retiring following battles with drug addiction and depression.

But he has since partnered with a new trainer, MMA coach Rafael Cordeiro, to kick-start his training after sensationally revealing his 15-year break.

During their chat, basketball legend O'Neal revealed how he ached for three days after playing with his sons.

Tyson responded: "That's just because you haven't done it for a while.

WHAT IS STEM CELL TREATMENT USED FOR?

Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.

It can also be used to replace damaged blood cells as the result of intensive cancer treatment.

Here are conditions that stem cell transplants can be used to treat:

"If you continue to do it consistently you'll be back to normal.

"It's just like me, I haven't boxed or hit the bag for 15 years - it has been three days so far and I feel incredible."

Tyson, who has a 50-6 record, is reportedly gearing up for a sensational return amid plans to compete in exhibition bouts for charity later this year.

He told rapper T.I. last month: "I've been hitting the mitts for the last week.

"That's been tough, my body is really jacked up and really sore from hitting the mitts.

MIKE DROPTyson shows off body transformation with 53-year-old preparing for boxing return

NO GLOVE LOSTJoshua tells Froch to 'shut up, blud' as war of words continues

HOLY MOLYHolyfield hints at comeback aged 57 after Tyson announces plans to return to ring

WHATSAPP-ENING?Joshua sent Froch four now-deleted messages after his P4P rant

IT WILL ALL END IN PIERSPiers Morgan and Eddie Hearn in Twitter spat over coronavirus

SHOWDOWNHall vs Bjornsson: How giants compare as Beast threatens deadlift record holder

HOME FIGHTERDubois enjoys lockdown sparring with siblings after buying family pad

NEED FOR SPEEDMike Tyson, 53, shows off speed as heavyweight legend gears up for return

MIKED UPTrump, The Rock and Khabib react to Mike Tysons stunning boxing training footage

"I've been working out, I've been trying to get in the ring, I think I'm going to box some exhibitions and get in shape.

"I want to go to the gym and get in shape to be able to box three or four-round exhibitions for some charities and stuff.

"Some charity exhibitions, make some money, help some homeless and drug-affected motherf****er like me."

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Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t - The Sun

Bone Marrow Stem Cells Comments Off on Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t – The Sun | May 5th, 2020

Veteran Bollywood actor Rishi Kapoor, who was battling leukemia for the last two years, died at Mumbais Sir HN Reliance Foundation Hospital on Thursday (April 30) morning. He was 67. Rishi Kapoor got treated for leukemia in the US for a year and had returned to India in September 2019.

Leukemia is a blood cancer caused by a rise in the number of white blood cells in your body. Those white blood cells crowd out the red blood cells and platelets that your body needs to be healthy. The extra white blood cells dont work right. A cancer of blood-forming tissues, hindering the body's ability to fight infection.

Dr Rahul Bhargava, Director, Haematology, Haemato Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram says:

Leukemia, often fatal, is a blood cancer. As with cancer, it is associated with an exponential rise in the number of white blood cells. Symptoms begin to manifest slowly and can often be mistaken for something else sweating uncontrollably, shaking, fever, vomiting. It is caused by huge was is reduced focus on ones own health.

Intake of processed food, soda, artificial and synthetic sweets and preservatives, pesticide use in farms, lack of physical exercise, excessive tobacco and drug consumption, excessive alcohol intake, caffeine consumption are the main culprits causing cancer. Family history also plays an important role in a leukemia diagnosis. Only through screening can it be identified and if caught early managed. One needs to be mindful and ensure that they are employing good lifestyle habits.

This means eating green leafy vegetables and fruits, going for a 30 minutes walk every day or engaging in any other form of exercise for 30 minutes a day, drinking adequate amount of water to keep flushing out the toxins, controlling or completely eliminating our use of tobacco and other unnatural substances. Processed food and sugars should be consumed in control, they are the single largest cause of mutation to cells.

Leukemia symptoms vary, depending on the type of leukemia. Common leukemia signs and symptoms include:

Leukemia can also cause symptoms in organs that have been infiltrated or affected by the cancer cells. For example, if the cancer spreads to the central nervous system, it can cause headaches, nausea and vomiting, confusion, loss of muscle control, and seizures.

Leukemia can also spread to other parts of your body, including:

The major types of leukemia are:

Blood has three types of cells: white blood cells that fight infection, red blood cells that carry oxygen, and platelets that help blood clot. Every day, your bone marrow makes billions of new blood cells, and most of them are red cells. When you have leukemia, your body makes more white cells than it needs. These leukemia cells cant fight infection the way normal white blood cells do. And because there are so many of them, they start to affect the way your organs work. Over time, you may not have enough red blood cells to supply oxygen, enough platelets to clot your blood, or enough normal white blood cells to fight infection.

Leukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer. The treatment depends on the type and stage of the cancer. Some forms of leukemia grow slowly and don't need immediate treatment. However, treatment for leukemia usually involves one or more of the following:

Source: Information has been taken from various health website

Read more| National Honesty Day 2020: Date, history, significance and quotes on the value of honesty

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What Is Leukemia? Here's all you need to know - India Today

Bone Marrow Stem Cells Comments Off on What Is Leukemia? Here’s all you need to know – India Today | May 5th, 2020

The first-in-human, universal chimeric receptor antigen (CAR) T-cell (CAR-T) therapy GC027 was tolerable and resulted in antileukemic responses among patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.1

Theuniversal CAR T cells target CD7, which, according to Xinxin Wang, PhD, ofGracell Biotechnologies Co, Ltd, in China, and lead author and presenter of thestudy, is a good target for T-ALL because it is expressed by more than 95% ofT-ALL patients.

GC027 isallogeneic, which may prevent the development of graft-versus-host disease. Theproduct is introduced using lentivirus for rapid elimination of T-ALL cells. Preclinicalstudies showed efficacy in a T-ALL xenograft model, and this prospective studyevaluated the safety and efficacy in humans.

Thesingle-arm, open-label study treated 5 adult patients with relapsed/refractoryCD7-positive T-ALL with a single infusion of 1 of 3 different dose levels ofG027: 0.6 x 107/kg, 3 x 107/kg, and 1.5 x 107/kg.Lymphodepletion therapy was administered prior to the G027 infusion. Theprimary endpoint was safety and the secondary endpoints included objectiveresponse rate (ORR) within 3 months after G027 infusion.

Patientswith extramedullary or central nervous system disease were excluded. Atbaseline, the median age was 24 (range, 19-38). Patients were heavilypretreated, with 5 median number of prior therapies (range, 1-9). Two patientshad high-risk disease and the median bone marrow tumor burden was a median of38.2% of blasts. None of the patients had undergone a prior allogeneic hematopoieticstem cell transplant.

Allpatients developed cytokine release syndrome (CRS), 4 of which were grade 3 and1 was grade 4. All cases were manageable and resolved with treatment andsupportive care. None of the patients developed neurotoxicity.

The completeremission (CR)/CR with incomplete hematologic recovery was 100%. By day 28, 4patients achieved a CR with negative for minimal residual disease (MRD) and 3of these patients remained MRD negative up to day 161. One patient achieved CRbut was MRD positive, and relapsed by day 29.

Peak CART-cell expansion in peripheral blood occurred between week 1 and 2.

As the first-in-human, universal CAR T-cell therapy for adult relapsed/refractory T-ALL, Dr Wang said, GC027 has demonstrated superior clinical efficacy and induced deep response in patients with acceptable safety profile. She added that trial enrollment is ongoing.

Reference

Wang X, Li S, Gao L, et al. Clinical safety and efficacy study of TruUCAR GC027: The first-in-human, universal CAR-T therapy for adult relapsed/refractory T-cell acute lymphoblastic leukemia (r/r T-ALL). Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I; April 27-28, 2020. Abstract CT052.

This article originally appeared on Cancer Therapy Advisor

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First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL - Oncology Nurse Advisor

Bone Marrow Stem Cells Comments Off on First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL – Oncology Nurse Advisor | May 5th, 2020

Most doctors are prisoners of their education and shackled by their profession.

- Richard Diaz

A month ago we offered up the analysis below on this 'watch item' biotech stock from across the pond exclusively to Biotech Forum members. The stock is up over 40% since then but the concern is still intriguing. Today, we update our original thesis to account for all company-specific news and analyst ratings on this name since our original research was posted.

Orchard Therapeutics (ORTX) is a London, United Kingdom-based biopharmaceutical company that IPOd in 2018. The companys mission is to help patients with rare conditions by leveraging its understanding of gene therapy. The companys approach is to take a patients own blood stem cells and insert into them a working copy of the missing or malfunctioning gene. The companys approach circumvents the need for a bone marrow transplant by taking advantage of blood stem cells' intrinsic capacity to self-renew in a patients bone marrow and produce new blood cells of all types. The ultimate goal is to permanently correct genetic disorders through the use of a single treatment. The companys pipeline is broken down into three overarching programs: neurometabolic disorders, primary immune deficiencies, and blood disorders. The companys lead product candidates are OTL-200, OTL-101 and OTL-103. The companys only commercial product is Strimvelis, which has been approved by the EMA but not by the FDA. Orchard Therapeutics has a market capitalization of just over $1 billion and trades for around $11 a share.

Pipeline:

Source: Company Presentation

OTL-200:

OTL-200 is an ex vivo autologous gene therapy in development to treat Metachromatic leukodystrophy. The drug uses a modified virus to insert an operational copy of the ARSA gene into a patients cells. OTL-200 has received rare pediatric disease designation from the FDA. MLD is a rare and deadly inherited disease. The disease is characterized by the accumulation of fats called sulfatides, which causes a breakdown in the protective fatty layer surrounding nerves in the central and peripheral nerve systems. It is estimated that 1 in 40,000 to 1 in 160,000 people have the disease worldwide. OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Source: Company Presentation

On December 2nd, 2019, under accelerated assessment status, The EMA accepted the review for a marketing application of OTL-200 for MLD. Under accelerated assessment, the review period is 150 days, compared to the normal 210 days. The action date should occur very shortly.

Source: Company Presentation

Looking ahead, the company expects to obtain approval and launch the drug in Europe in the second half of 2020. The company is currently preparing for a commercial launch. Furthermore, the company expects a U.S. regulatory filing, a BLA, in late 2020 to early 2021.

Source: Company Presentation

OTL-101:

OTL-101 is an ex vivo autologous gene therapy in development to treat adenosine deaminase severe combined immunodeficiency. The drug uses a modified virus to insert an operational copy of the ADA gene into a patients cells. OTL-101 has received both Breakthrough Therapy designation and Rare Pediatric Disease designation from the FDA. ADA-SCID is a rare, inherited, pediatric disorder that is commonly fatal when undiagnosed or left untreated. The disorder is the result of a deficiency in adenosine deaminase. The disease culminates in a complete lack of/minimal immune system development. It is estimated that the diseases annual incidence is between 1 in 200,000 and 1 in 1 million live births.

Looking ahead, Orchard Therapeutics will initiate a rolling BLA filing in the U.S. for OTL-101 in ADA-SCID in the first half of 2020 with an anticipated completion of the filing within 12 months.

Source: Company Presentation

OTL-103:

0TL-103 is an ex vivo autologous gene therapy in development to treat Wiskott Aldrich syndrome. The drug uses a modified virus to insert a working copy of the WAS gene into a patients cells. WAS is a rare, X-linked, recessive, inherited immune disorder, which is characterized by reoccurring severe infections, autoimmunity, eczema and severe bleeding episodes. The company has received Rare Pediatric Disease designation and Regenerative Medicine Advanced Therapy designation from the FDA. OTL-103 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Looking ahead, the company is preparing to file a BLA in the U.S. and an MAA in the EU for OTL-103 in WAS in 2021.

Source: Company Presentation

As of December 31st, 2019, the company had cash and cash equivalents of over $300 million including marketable securities. Research and development expenses for the fourth quarter were $30.8 million, compared to $17.4 million in Q4 of 2018. Selling, general and administrative expenses were $18.5 million in the quarter, compared to $11.9 million in the same quarter of 2018. Overall, the company had a net loss of $45.4 million in the quarter, compared to a net loss of $25 million in the same period of 2018. The company believes that its current balance sheet will allow it to fund all anticipated operations and capital expenditures into the second half of 2021.

Despite a sizable market cap, Orchard gets little in the way of attention from analysts. That is probably because it is headquartered overseas. The current median analyst price target on Wall Street is just over $25.00 a share.

Oppenheimer reissued its Buy rating and $28 price target on April 1st. In mid-March, Cowen & Co. did the same with an identical price target in Mid-March. This follows a similar call at Cowen in December with strongly bullish commentary:

Orchard Therapeutics has a differentiated and promising lentiviral, ex vivo pipeline with gene therapies for ADA-SCID, WAS and MLD likely to get approved in 2021-22. With a deep early stage pipeline focused on validated targets, we anticipate strong stock appreciation as drugs are approved and early stage programs yield positive clinical results and advance into pivotal studies in 2020/21."

On September 17th, 2019, Guggenheim initiated coverage with a buy rating and a $31 a share price target. The analyst at Guggenheim views the company's ex vivo, lentiviral gene therapy platform as differentiated and de-risked. Furthermore, three drug approvals within the next 24 months bode well for share appreciation. Finally, in early September of last year, Barclays (NYSE:BCS) initiated coverage with an overweight rating and a $21 price target. The analyst at Barclays sees a company that strategically targets genetic diseases that have validated approaches with hematopoietic stem cell transplant, a fairly de-risked pipeline and multiple catalysts over the next 24 months.

Orchard has multiple shots on goal and several definable milestones in the coming quarters. Given some of the recent turmoil in the credit markets, anything that will need to raise capital in the foreseeable future is taken out and shot when markets have deep down days.

Given that, I still have Orchard as a solid 'watch item' holding and added several hundred shares in my personal account in late March. The shares appear to have an attractive risk/reward profile on a longer term basis, but I would accumulate on dips given the shares run up in April. Once volatility ebbs and we have more confidence in the economy and markets, this is a name we will revisit and maybe upgrade to a larger recommended holding.

The securest place is a prison cell, but there is no liberty

- Ben Franklin

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

Live Chat on The Biotech Forum continues to be very active with new trade and the lucrative covered call ideas available thanks to the spike in market volatility throughout the trading day. If you join the The Biotech Forum today by clicking HERE you will automatically get access to our model portfolio, Live Chat, investment archives and our next 'option play of the week.'

Disclosure: I am/we are long ORTX. 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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Orchard Therapeutics: Updating The Investment Thesis - Seeking Alpha

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics: Updating The Investment Thesis – Seeking Alpha | May 5th, 2020

Researchers are learning more about how to diagnose and better treat blastic plasmacytoid dendritic cell neoplasm, a rare cancer that often presents with skin manifestations, according to a review published March 2020 in Current Opinion in Hematology.1

Blastic plasmacytoid dendritic neoplasm patients have suffered historically poor outcomes. Years ago, doctors were limited to treating these patients primarily with intensive chemotherapy regimens used to treat acute myeloid leukemia or acute lymphoblastic leukemia patients.

But in 2018, the U.S. Food and Drug Administration (FDA) approved tagraxofusp-erzs (Elzonris, Stemline).

Tagraxofusp-erz is the first approved drug indicated specifically for blastic plasmacytoid dendritic neoplasm, and its use is recommended in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology.Newer targeted agents to treat the hematologic malignancy are in the pipeline.

Notable changes in recent years

The World Health Organization (WHO) named blastic plasmacytoid dendritic cell neoplasm (BPDCN) and classified it under acute myeloid leukemia and related precursor neoplasms in 2008. Some eight years later, WHO established BPDCN as a distinct entity.

Just how many people have BPDCN isnt clear. But it is thought that there are about 0.04 cases of the cancer per 100,000 people. And about three in four patients are older men.

Derived from plasmacytoid dendritic cells, BPDCN generally is an aggressive disease. It presents clinically on the skin in about nine out of every 10 cases. Skin lesions tend to be asymptomatic, often appearing as bruise-like lesions, plaques or nodules, according to the paper.

While a small percentage of patients will present with skin disease only, most show signs of BPDCN in the bone marrow, lymph nodes or visceral organs. Rarely, patients will have no cutaneous evidence and instead present with the leukemic phase of the cancer. About 30% of patients also have central nervous system involvement.

Flow cytometry to determine the immunophenotype is an essential component of diagnosing [blastic plasmacytoid dendritic cell neoplasm], the author wrote.

CD123, an interleukin-3 receptor alpha, is over expressed in nearly all BPDCN cases. These cancer cells also may be positive for CD4, CD56, CD303 or TCL1, according to the paper.

Some authors have found a recurrent MYC gene rearrangement in these patients. That particular genetic aberration is associated with an older age at diagnosis and worse prognosis.

Treatment is evolving

Unfortunately, doctors have to rely largely on retrospective studies looking at BPDCN treatment options.Those studies suggest that BPDCN, generally, responds better to acute lymphoblastic leukemia regimens compared to acute myeloid leukemia treatment options. However, most responses to these regimens are transient, the author reported.

Retrospective studies suggest allogeneic stem cell transplant for eligible patients in their first remission offer the highest overall survival rates, including 3- and 4-year overall survival rates ranging from 74% to 82%.Tagraxofusp-erzs targets CD123. It consists of recombinant human interleukin-3 fused to a truncated diphtheria toxin, according to the paper.

Binding the drug to CD123 on the cell surface leads to cellular internalization of the diphtheria toxin, which ultimately leads to inhibition of protein synthesis and cell death, the author wrote.

In a phase I/II clinical trial of 44 untreated or relapsed/refractory BPDCN patients, 21 of 29 previously untreated patients achieved complete remission and 13 of those went on to have a stem cell transplant. Overall response rate of the 15 patients with relapsed/refractory BPDCN was 67% with tagraxofusp-erzs, with an average overall survival of 8.5 months.

Eighteen of the 44 patients studied developed the most critical treatment-related adverse event, capillary leak syndrome. Two patients died from capillary leak syndrome during the study.Researchers are studying investigational agents aimed at treating BPDCN. These include IMGN632, a humanized antibody-drug conjugate with an anti-CD123 monoclonal antibody conjugated to a DNA-alkylating payload, the author wrote.

Researchers are evaluating the safety and efficacy of treating CD123-positive malignancies including BPDCN with the monoclonal antibody targeting CD123 and CD3 XmAb14045.

Venetoclax, a BCL-2 inhibitor, is yet another agent in the pipeline for BPDCN patients.

as knowledge is gained on the molecular changes that occur in [blastic plasmacytoid dendritic cell neoplasm], this will ideally lead to more targeted and effective therapies in the years to come, the author wrote.

Disclosures:

Kendra Sweet, MD, has received honoraria from Stemline Therapeutics.

References:

1 Sweet K. Blastic plasmacytoid dendritic cell neoplasm: diagnosis, manifestations, and treatment. Curr Opin Hematol. 2020;27(2):103-107.

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Investigational agents to treat hematologic malignancy in pipeline - Dermatology Times

Bone Marrow Stem Cells Comments Off on Investigational agents to treat hematologic malignancy in pipeline – Dermatology Times | May 4th, 2020

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced a new development and commercialization agreement with Saladax Biomedical, Inc. (Saladax), a leading diagnostics provider focused on developing blood tests for personalized dosing, to develop and validate a fully automated nanoparticle immunoassay kit designed to simplify and streamline therapeutic drug monitoring (TDM) for patients treated with the conditioning agent busulfan.

At AVROBIO, we push ourselves to be at the forefront of technologies advancing lentiviral gene therapy, and its in this spirit that were funding the development of this kit, said Geoff MacKay, AVROBIOs president and CEO. Our personalized conditioning approach is already delivering results. We believe this new assay kit will, for the first time, provide convenient busulfan TDM close to the patient, potentially improving both the patient experience and long-term outcomes, as well as enabling many more hospitals and clinics to become TDM-capable sites.

AVROBIOs state-of-the-art plato gene therapy platform incorporates TDM protocols designed to optimize busulfan dosing over four days, with the goal of maximizing stem cell engraftment while minimizing side effects. TDM evaluates how quickly a patient metabolizes busulfan a rate that can vary significantly from patient to patient and even from one day to the next for the same patient. Current assays that inform that dose adjustment can take hours to return results and must be processed at specialized laboratories with trained staff that may not be geographically convenient to the gene therapy dosing site.

The technology used to deliver these rapid test results is based on an extensive intellectual property portfolio developed by Saladax in the field of TDM. The new assay kit under development by Saladax, which collects a small blood sample, is able to return results on patient metabolization of busulfan in minutes using hospitals standard analytical devices, greatly expanding access to personalized conditioning with busulfan.

Personalized Gene Therapy to Optimize Durable Protein Expression including in Brain, Muscle and Bone

AVROBIOs investigational gene therapies start with collecting the patients own hematopoietic stem cells. In the companys manufacturing process, a lentiviral vector is used to integrate a therapeutic gene designed to produce functional protein essential to cellular health into the patients chromosomes. Prior to dosing, treating clinicians use busulfan, an extensively validated conditioning agent generally considered to be the gold standard for ex vivo lentiviral gene therapy, to create space in the patients bone marrow. Finally, the patient receives the gene therapy and the therapeutic stem cells are expected to engraft in the bone marrow and produce generations of daughter cells, each containing the therapeutic gene. This approach is designed to drive durable production of the functional protein throughout the patients body, including hard-to-reach tissues such as the brain, muscle and bone. A distinguishing feature of this type of gene therapy with busulfan conditioning is that some of the corrected cells are expected to cross the blood-brain barrier and thereby potentially address central nervous system manifestations.

Earlier this year, AVROBIO reported initial clinical results for the first patient conditioned with busulfan using TDM prior to dosing in AVROBIOs Phase 2 clinical trial of its investigational gene therapy, AVR-RD-01, for Fabry disease. The early data from this patient showed increased endogenous enzyme activity at one month following dosing, as compared to other patients in the trial who received a different conditioning agent. Initial data suggest side effects, including nausea, mucositis, fever, rash and hair loss, which were consistent with those expected based on clinical experience of busulfan, developed eight to 10 days after dosing with busulfan and resolved quickly.

About AVROBIO

Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits of Saladaxs immunoassay kits, including the ability to improve, simplify and streamline therapeutics drug monitoring for patients treated with the conditioning agent busulfan and enable local commercialization of AVROBIOs proprietary platform worldwide, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that AVROBIO may not realize the intended benefit of Saladaxs immunoassay kits, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO to Collaborate with Saladax Biomedical on New High-Speed Diagnostic Assay Used with Busulfan Conditioning to Enable Widespread...

Bone Marrow Stem Cells Comments Off on AVROBIO to Collaborate with Saladax Biomedical on New High-Speed Diagnostic Assay Used with Busulfan Conditioning to Enable Widespread… | May 4th, 2020

People with cancer who contract the new coronavirus appear to have a greater risk for severe COVID-19 illness and death, but this may depend on their cancer stage and the type of treatment they are receiving, according to recent research. In fact, those with early-stage cancer may fare as well as people who have not had cancer.

Researchers from some of the earliest and hardest hit epicenters of the COVID-19 pandemic described outcomes among cancer patients with the coronavirus (officially known as SARS-CoV-2) during a special session the American Association for Cancer Research (AACR) virtual annual meeting last week. Soon after the conference, another group of researchers published an analysis of mortality among cancer patients in New York City.

Early reports from China, where the pandemic originated in late December, showed that older people, those with compromised immune systems and those with underlying health conditions are more susceptible to severe COVID-19. One study saw a death rate of 6% for people with cancermore than twice as high as the overall estimated COVID-19 mortality rate in China, but lower than the rates seen in people with diabetes (7%) or cardiovascular disease (11%).

Chemotherapy medications and some targeted therapies for cancer can cause neutropenia, a temporary depletion of immune system white blood cells that fight infection. People who receive bone marrow stem cell transplants or CAR-T therapy or for blood cancers typically receive strong chemotherapy to kill off existing blood cells and make room for the new ones. Conversely, immunotherapies such as checkpoint inhibitors and CAR-T therapy unleash natural or engineered T cells to fight cancer, which in some cases can trigger an excessive immune response that leads to harmful inflammation.

Two reports at the AACR meeting provided updates from China. Li Zhang, MD, PhD, of Tongji Medical College described outcomes among 28 cancer patients with COVID-19 in Wuhan, the initial epicenter of the pandemic.

Seven had lung cancer and the remainder had 13 other cancer types. Just over a third had Stage IV, or metastatic, cancer. Nearly 30% acquired the coronavirus at medical facilities. About half had severe disease, 10 patients required mechanical ventilators and eight diedmostly from acute respiratory distress syndromegiving a mortality rate of 29%.

Although three quarters had ever undergone surgery, radiation or chemotherapy, a majority had not received treatment recently. Only one person received radiation, three received chemotherapy, two received targeted therapy and one received immunotherapy within two weeks prior to their COVID-19 diagnosis. Recent cancer treatment was associated with a fourfold increased risk of severe outcomes. However, the single patient treated with a checkpoint inhibitor (for liver cancer) had mild COVID-19 and a short hospital stay.

Similarly, as part of his discussion of immunotherapy for cancer in the COVID-19 era, Paolo Ascierto, MD, of the National Tumor Institute in Naples, noted that just two out of 400 patients on immunotherapy at his institute tested positive for the coronavirus, they were asymptomatic and they recovered quickly, leading him to speculate that immunotherapy might somehow be protective against COVID-19.

Hongbing Cai, MD, of Zhongnan Hospital of Wuhan University, presented data on 105 cancer patients and 536 age-matched people without cancer at 14 hospitals in Hubei province who developed COVID-19. Results were also published in Cancer Discovery. Twenty-two had lung cancer, 13 had gastrointestinal cancers, 11 each had breast cancer and thyroid cancer, nine had blood cancers such as leukemia or lymphomawhich affect white blood cells that carry out immune responsesand six each had cervical and esophageal cancer.

In general, patients with cancer deteriorated more rapidly than those without cancer, Cais team reported. Cancer patients with COVID-19 were nearly three times more likely to have severe or critical illness (34%), be admitted to an intensive care unit ICU (19%) or be put on a ventilator (10%). Whats more, people with cancer were about twice as likely to die as COVID-19 patients without cancer (11% versus 5%, respectively).

People with blood cancers or lung cancer, as well as those with metastatic cancer, had a higher risk of severe events. Two thirds of the blood cancer patients and half of the lung cancer patients had such events. Among the lung cancer patients, 18% were put on ventilators and 18% died. In contrast, no one with breast, thyroid or cervical cancer required ventilators or died.

In particular, those with blood cancersmore than half of whom had severe immune suppressionhad about a 10-fold higher risk of severe events or death. Two thirds had severe symptoms, 22% were put on ventilators and 33% died. These patients all had a rapidly deteriorated clinical course once infected with COVID-19, the researchers wrote.

People with metastatic cancer had about a six-fold higher risk of severe events or death. But people whose cancer had not yet spread were not significantly more likely to have severe events or die than COVID-19 patients without cancer. People currently on cancer treatment and those with a history of cancer who had completed treatment were both at higher risk.

People who underwent surgery within the previous 40 days had higher rates of severe events, ICU admission, ventilator use and death, but this was not the case for those who received only radiation. In this study, unlike Zhangs and Asciertos, people treated with immunotherapy did not fare so well. Four of the six patients who recently received checkpoint inhibitors had critical symptoms and two died.

Based on our analysis, COVID-19 patients with cancer tend to have more severe outcomes when compared to the non-cancer population, the researchers wrote. Although COVID-19 is reported to have a relatively low death rate of 2% to 3% in the general population, patients with cancer and COVID-19 not only have a nearly three-fold increase in the death rate than that of COVID-19 patients without cancer, but also tend to have much higher severity of their illness.

In a related study, Marina Chiara Garassino, MD, of Fondazione IRCCS National Tumor Institute in Milan, presented the first data from the international TERAVOLT registry, which is collecting data about COVID-19 among people with lung cancer and other thoracic malignancies. She noted that TERAVOLT was registering around 70 new cases per week from around the world per week.

This population may be especially vulnerable to COVID-19 due to older age, lung damage, smoking and underlying health conditions, Garassino said. Whats more, the symptoms of COVID-19 overlap with lung cancer, making diagnosis very challenging.

Garassino described results from the first 200 cancer patients with COVID-19 in more than 20 countries. Non-small-cell lung cancer was the most common type, and nearly three quarters had metastatic disease. About 20% received only targeted therapy, 33% received chemotherapy alone and 23% received immunotherapy alone.

A majority (76%) were hospitalized, but most were not offered intensive care for COVID-19; just 9% were admitted to an ICU and 3% were put on ventilators. More than a third (35%) died, mostly due to COVID-19 rather than cancer. Specific types of cancer treatment were not significantly associated with an increased risk of death.

But not all studies have seen worse COVID-19 outcomes among people with cancer. Fabrice Barlesi, MD, PhD, and colleagues looked at 137 COVID-19 patients with cancer at Gustave Roussy, a cancer center near Paris. They had a variety of cancer types, with blood cancers and breast cancer being most common. Nearly 60% had active advanced disease while 40% were in remission or being treated with potentially curative therapy.

Within this group, 25% had worsening COVID-19 after admission, 11% were admitted to the intensive care unit (ICU) and 15% died. Again, people with blood cancers were more likely to have worse outcomes. Treatment with chemotherapy within the past three monthsbut not targeted therapy or immunotherapydoubled the likelihood of worsening disease. But this only applied to people with active or metastatic cancer, not those who had localized disease or were in remission.

The 15% death rate among people with cancer at Gustave Roussy was lower than the 18% rate for all COVID-19 patients in Paris and in France, Barlesi said. His team concluded that both incidence and outcomes of COVID-19 among cancer patients seem to be comparable to the population as a whole. However, people with blood cancers, those treated with chemotherapy and frail patients are at greater risk.

Discussing how to manage cancer patients during the COVID-19 pandemic, Cai recommended self-protective isolation, strict infection control in hospitals and shifting some medical services online.

With regard to cancer treatment, she said, clinicians need to develop individualized plans based on a patients tumor type and stage of disease. She added that postponing surgery, if appropriate, should be considered in areas with current outbreaks. Radiation therapy, she said, could go ahead according to existing treatment plans with intensive protection and surveillance. Whether people with early-stage cancer need to postpone their treatment remains an unanswered question, she said.

Click hereto read the abstracts from the AACR COVID-19 and cancer session.Learn about What People With Cancer Need to Know About the New Coronavirus.

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Cancer Patients With COVID-19 May Have Higher Risk of Severe Illness and Death - Cancer Health Treatment News

Bone Marrow Stem Cells Comments Off on Cancer Patients With COVID-19 May Have Higher Risk of Severe Illness and Death – Cancer Health Treatment News | May 4th, 2020

A novel, bispecific CD19/CD22 chimeric antigen receptor T-cell (CAR-T) therapy was tolerable and resulted in responses among patients with acute lymphoblastic leukemia (ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.

Thenovel CAR-T therapy was developed with the hypothesis that dual antigen-targetingstrategies may prevent antigen negative escape, Haneen Shalabi, DO, of theNational Cancer Institute and lead author and presenter of the study, said.

Thephase 1, dose-escalation study treated 13 young patients with ALL with theCD19/CD22 CAR-T therapy at 3 different dose levels, including 3 x 105,1 x 106, and 3 x 106. The bispecific construct containedFMC63 (CD19 scFv) linked with m971 (CD22 scFv) and a 4-1 BB costimulatorydomain.

Patientsunderwent lymphodepletion with fludarabine plus cyclophosphamide prior to theirCAR-T infusion. The primary endpoints were safety and toxicity, and thesecondary endpoints were efficacy, chimeric antigen receptor (CAR) expansion,and CAR persistence.

Atbaseline, the median age was 19.6 (range, 5.4-28.5). Patients had receivedprevious treatments, including hematopoietic stem cell transplant (54%),CD19-targeted therapy (69%), prior CD19 CAR T cell therapy (38.4%),blinatumomab (61.5%), CD22-targeted therapy (38.4%), inotuzumab (30.7%), andCD22 CAR-T therapy (15.4%). Extramedullary disease was present in 46.2% ofpatients.

CAR Tcells were well tolerated and toxicities were reversible in all patients, DrShalabi said.

Cytokinerelease syndrome (CRS) developed in 46% of patients, 15.4% of which was grade 3or higher. Both patients who developed grade 3 or higher CRS had received the 1x 106 dose level of the CD19/CD22 CAR-T product and both requiredtreatment with tocilizumab. One patient developed neurotoxicity, and hadreceived the 3 x 106 dose level.

Of the12 patients evaluable for efficacy, a complete response (CR) was achieved by42% (5) of patients, including all patients who received the 1 x 106 or 3 x 106 dose levelsof the CD19/CD22 CAR-T therapy. There were 2 nonresponders.

Two patientswho received 1 x 106 CAR-T and all patients who received the 3 x 106dose level were negative for minimal residual disease (MRD), with the remainingCRs demonstrating bone marrow clearance. Four of the 5 patients who were MRDnegative were also naive to CAR-T therapy.

Of the 5patients who achieved a CR, 2 relapsed with CD19-positive/CD22-positive diseaseand 3 remained in remission at a median 7 months after CAR T cell infusion.

Severalpatients, however, who were MRD negative in the bone marrow did not achieve CRin their extramedullary disease. Dr Shalabi said that these discrepant resultsbetween marrow and extramedullary disease suggests potentially limited CAR-Ttrafficking to sites of extramedullary disease. She suggested that treatmentat higher dose levels may be needed to overcome this limitation.

CAR T-cellexpansion occurred in all patents who responded, with a median peak inperipheral blood of 7%. At day 28, there were 1.3% CAR T cells in the bonemarrow. The persistence of the CAR T cells in peripheral blood was a median of45.6 days, as measured by flow cytometry.

Dr Shalabi concluded that this early experience with bispecific CD19/CD22 CAR T cells demonstrates clinical activity with reversible CRS and limited neurotoxicity. She noted that future studies will explore a 1 x 107 dose level, intensification of lymphodepletion prior to CAR-T infusion, and consideration of the potential role of immune checkpoint inhibitors to augment CAR-T in extramedullary disease.

References

Shalabi H, Yates B, Shahani S, et al. Safety and efficacy of CD19/CD22 CAR T cells in children and young adults with relapsed/refractory ALL. Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020; April 27-28, 2020. Abstract CT051.

This article originally appeared on Cancer Therapy Advisor

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Novel Bispecific CD19/CD22 CAR-T Therapy Deemed Tolerable in Relapsed/Refractory ALL - Oncology Nurse Advisor

Bone Marrow Stem Cells Comments Off on Novel Bispecific CD19/CD22 CAR-T Therapy Deemed Tolerable in Relapsed/Refractory ALL – Oncology Nurse Advisor | May 4th, 2020

Global Cell Therapy Technologies Market was valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

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The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.

Global Cell Therapy Technologies Market: Overview

Cell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

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Global Cell Therapy Technologies Market: Drivers and Restraints

The growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation Analysis

On the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional Analysis

North America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at: https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

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Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2019-2026) - MR Invasion

Bone Marrow Stem Cells Comments Off on Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2019-2026) – MR Invasion | May 4th, 2020

Leukemia are a heterogeneous group of cancers affecting the bone marrow and White Blood Cells (WBC). Leukemia is characterized by the rapid increase of abnormal blood cells growth or blasts, resulting in a decrease in the numbers of healthy, normal fully modified blood cells, leading to the typical symptoms of bleeding, anemia, and high risk of infection. Leukemia can grow along either the myeloid or lymphoid stem cell lines, it depends on the effect of genetic and epigenetic mutations on the progression of pluripotent stem cells to the various lines of mature cells which then pass into the blood. The effected line, combined with the rate of action and growth of disease reflects the four types of leukemias- Acute Myeloid Leukemia (AML), chronic lymphoblastic leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia. AML: Acute Myeloid Leukemia, is a serious condition, its the most common leukemia suffered by adult people. According to a report from American Cancer Society, the average age for first diagnostic for AML is 64. With few days without treatment, AML develops fast, in duration of few weeks, the patient becomes severely ill. Due to its fast onset and acuteness in nature, there is no staging system for Acute Myeloid Leukemia (AML).The treatment for Acute Myeloid Leukemia (AML) has changed in last 4 decades.

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The drug approval process is difficult in AML, (many drugs have not been approved by USFDA, for instance Laromustine, Dacogen and Tipitarnib) efforts have been made to introduce new therapies in the AML market.

Primary drivers boosting the growth of acute myeloid leukemia (AML) therapeutics market are minimal but increased prevalence of acute myeloid leukemia (AML), increased drug approval rate for AML, classification of acute myeloid leukemia (AML) as an orphan disease. Over the forecast period, population of people over 65 year is anticipated to increase, which is another key driver for acute myeloid leukemia (AML) therapeutics market.

However, lack of targeted therapies in current acute myeloid leukemia (AML) therapeutics landscape, the drug difficult approval process in AML can hinder the growth of acute myeloid leukemia (AML) therapeutics market, but this restraint has opened an opportunity for key players to innovate acute myeloid leukemia (AML) therapeutics market.

How the Coronavirus Threat has Taken Global Business into Uncharted Waters

The global acute myeloid leukemia (AML) therapeutics market is segmented on the basic of disease subtype, treatment type, end user and region.

Based on the disease subtype, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on treatment type, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on end user, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

The global acute myeloid leukemia (AML) therapeutics market is anticipated to show lucrative growth owing to increased investment in innovative technologies by key players. Players in this market using various strategies to fuel their global footprint and to gain a competitive edge. Product pipelines, new product launches, agreements and collaborations, acquisitions, mergers and clinical trials are some key strategies applied from global players in recent years are anticipated to give a robust hike to the market in the forecast period.

Geographically, acute myeloid leukemia (AML) therapeutics market is segmented into regions viz. North America, Latin America, Europe, Asia Pacific and Japan, Middle East and Africa. North America is anticipated to be major contributor to this market accounting maximum percent of share in AML therapeutics market followed by Europe. Slow but constant growth in prevalence for AML in North America is anticipated to fuel the growth in acute myeloid leukemia (AML) therapeutics market. In Asia pacific region, China and India are anticipated to show high growth in acute myeloid leukemia (AML) therapeutics market due to new developments in healthcare infrastructure in the region.

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The players in acute myeloid leukemia (AML) therapeutics market include,

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Acute Myeloid Leukemia Therapeutics Market Latest Innovations, Drivers and Industry Key Events During Forecast 2017 2025 - Jewish Life News

Bone Marrow Stem Cells Comments Off on Acute Myeloid Leukemia Therapeutics Market Latest Innovations, Drivers and Industry Key Events During Forecast 2017 2025 – Jewish Life News | May 4th, 2020

Earlier, treating orthopedic problems just led to temporary cure but not the root cause of the problem. However, the case is not the same now. With emerging research and experience, Dr. David C. Karli says that regenerative medicine can completely repair the tissues using stem cell therapies. From professional athletes to ordinary people who strive to live a healthy and active life, regenerative medicine is a new ray of hope providing a healthy alternative for sports and musculoskeletal conditions and injuries.

Dr. David C. Karli is an Ivy-trained physician, a pioneer in orthopedic regenerative medicine and sports medicine, and the founder of Greyledge Technologies, one of the first FDA-audited biotech companies that prepare biologic implants to repair humans diseased or damaged tissues. Talking about the advancements in regen medicine, he says, Regenerative medicines multidisciplinary approach can cater to solutions for several untreatable orthopedic problems. With experts around the world pooling their knowledge, skills, and resources, a breakthrough in orthopedic treatment is leading to a miracle cure.

What is Regenerative Medicine?

Regenerative medicine is an interdisciplinary field that helps repair or replaces damaged or diseased human cells or tissues to restore normal function. The relatively new field of study comprises a broad range of scientific disciplines like molecular biology and genetics to immunology and biochemistry. Dr. Karli specializes in orthopedic applications where a patients diseased cells are replaced and re-implanted by the autologously collected healthy cells from the same patient.

About Dr. David C. Karli

Dr. David Karli graduated from Elizabethtown College, Pennsylvania, in 1993. Followed by receiving his MD degree from the University of Maryland, he pursued his residency in physical medicine and rehabilitation at Harvard Medical School, where he served as a chief resident in his final year. While serving as an attending physician at Harvard Medical School, Dr. Karli collaborated with his orthopedic surgeon colleagues and participated in the development of rehabilitation protocols for spinal disorders. He joined the Steadman Clinic in Vail, Colorado, where his research interest led him to take up Regenerative Medicine and successfully launch and develop Greyledge Technologies. This company focuses on autologous blood-based biotherapies for orthopedic injuries.

In his 23 years of experience, Dr. Karli has authored several research papers and publications on stem cell therapies and rehabilitation and lectured on spinal and musculoskeletal topics. He is a Diplomate of the American Board of Physical Medicine and Rehabilitation. Also, Dr. Karli is an active member of several societies, including the Regenerative Medicine Organization., the American Academy of PM&R, and the International Spinal Injection Society.

Greyledge Technologies

He founded the company Greyledge Technologies in 2010 with the mission to redefine orthopedic treatments and enhance the bodys response to injury. Greyledge Technologies develops biologic products by processing materials like human blood and bone marrow into implantable preparations. These biologic preparations are further developed and inserted into the human body replacing the diseased cells hence, stimulating the healing process and self-repair.

At Greyledge Technologies, every patient is completely assessed and analyzed not only to guarantee that theyre fit to undergo the procedure yet additionally to create a personalized dynamic strategy that each patient can follow. The whole treatment takes a certain number of days wherein the family members and caretakers are informed about the medicinal dosages to follow after the procedure. After surgery, every patient is regularly followed-up by the rehabilitation team.

When asked in-depth about practicing regenerative medicine at Greyledge Technologies, Dr. Karli said, The treatment is entirely safe and effective as it requires no big surgeries or complicated operations. As the cells used are autologous, they do not pose any chances of immune rejection or untoward irreversible side effects. Neither do the cells need to be preserved. This makes the procedure swift and safe for all ages.

Dr. Karli says, Regenerative Medicine is emerging as one of the trending treatment options for patients who have lost all hope. Whats brilliant about this treatment is that it has the capability to thoroughly repair the damaged tissues at the molecular, structural, and functional level.

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David C. Karli is Offering a New Ray of Hope Through Regenerative Medicine - RESPECT.

Bone Marrow Stem Cells Comments Off on David C. Karli is Offering a New Ray of Hope Through Regenerative Medicine – RESPECT. | May 3rd, 2020

DetailsCategory: AntibodiesPublished on Saturday, 02 May 2020 12:42Hits: 149

- Innovative, fixed-dose formulation significantly reduces treatment time from hours to minutes and demonstrates consistent efficacy with a reduction in administration-related reactions compared to DARZALEX (daratumumab) for approved indications

- DARZALEX FASPRO is the only subcutaneous CD38-directed antibody approved in the treatment of multiple myeloma

HORSHAM, PA, USA I May 1, 2020 I The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) approved DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a new subcutaneous formulation of daratumumab. DARZALEX FASPRO is approved in four regimens across five indications in multiple myeloma patients, including newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients.As a fixed-dose formulation, DARZALEX FASPRO can be administered over approximately three to five minutes, significantly less time than DARZALEX,which is given intravenously over hours. In the Phase 3 COLUMBA study supporting the approval, DARZALEX FASPRO demonstrated a consistent overall response rate (ORR) and pharmacokinetics and a similar safety profile compared with intravenous DARZALEX in patients with relapsed or refractory multiple myeloma. In addition, there was a nearly two-thirds reduction in systemic administration-related reactions (ARRs) for DARZALEX FASPRO compared to intravenous DARZALEX (13 percent vs. 34 percent, respectively).

"This approval exemplifies Janssen's mission and commitment to bringing together passion, science and ingenuity to advance novel solutions for patients," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC. "We are excited about the potential of this meaningful innovation in transforming the treatment experience for patients with multiple myeloma where DARZALEX FASPRO can be administered in approximately three to five minutes, significantly less time than intravenous DARZALEX, which is given over hours. Based on its favorable profile, we are accelerating the development of DARZALEX FASPRO and evaluating its potential in multiple ongoing studies."

Click to Tweet: #NEWS: #FDA approves subcutaneous CD38-directed antibody for the treatment of multiple #myeloma. See here for more details: https://bit.ly/2VozhzY

The approval is based on data from the Phase 3 COLUMBA (MMY3012)and Phase 2 PLEIADES (MMY2040) studies.1,2 In the COLUMBA study, the ORR was non-inferior for patients taking DARZALEX FASPROas monotherapycompared to those taking intravenous DARZALEXas monotherapy (41 percent vs. 37 percent, respectively). In addition, there were fewer systemic ARRs with DARZALEX FASPRO versus intravenous DARZALEX (13 percent vs. 34 percent, respectively). In a pooled safety population of 490 patients who received DARZALEXFASPRO as monotherapy or in combination, the ARR rate wFas 11 percent. The safety profiles of intravenous DARZALEX and DARZALEX FASPRO were otherwise similar.1 Additionally, in the Phase 2 PLEIADES study evaluating the efficacy and safety of DARZALEX FASPRO in combination therapies, objective responses were demonstrated in combination with bortezomib, melphalan and prednisone (D-VMP) in newly diagnosed transplant ineligible patients. In addition, objective responses were demonstrated in combination with lenalidomide and dexamethasone (D-Rd) in relapsed or refractory patients who received one prior line of therapy.2

"The Multiple Myeloma Research Foundation shares a common goal with Janssen in advancing treatments for multiple myeloma and addressing the unmet needs of this patient community," said Paul Giusti, President and CEO of the Multiple Myeloma Research Foundation (MMRF). "The approval of DARZALEXFASPRO marks an important milestone which will help make a positive difference in the lives of patients who depend on this effective therapy."

Click to Tweet: .@theMMRF talks about advancing treatments for multiple #myeloma and addressing patient needs with latest #FDA approval. Read more here: https://bit.ly/2VozhzY

"Since the approval of daratumumab, a robust body of evidence has established its use as a treatment for multiple myeloma in both the frontline and relapsed and refractory settings," said Saad Z. Usmani, M.D., Division Chief of Plasma Cell Disorders, Levine Cancer Institute. "With DARZALEX FASPRO there may be fewer administration-related reactions compared to intravenous DARZALEX, providing an additional treatment option that may help patients, oncologists and nursing staff."

DARZALEX FASPROis co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) [Halozyme'sENHANZEdrug delivery technology].DARZALEX FASPRO will be available to patients and physicians as soon as the week of May 11, 2020. The intravenous DARZALEX formulation will also remain available as an option for patients and their physicians.

DARZALEX FASPROis approved in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant, in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy, as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.

The U.S. FDA approval of DARZALEX FASPRO marks the first approval for this innovative subcutaneous formulation globally, and Janssen continues to work with health authorities around the world in an effort to bring this new treatment option to patients living with multiple myeloma.

Access to DARZALEX FASPRO (daratumumab and hyaluronidase-fihj)Janssen offers comprehensive access and support information, resources and services to assist U.S. patients in gaining access to DARZALEX FASPROthrough the Janssen CarePath Program. Through the program, eligible commercial patients pay no more than $5 per injection, regardless of individual income level. Information on the enrollment process is available online atwww.CarePathSavingsProgram.com/DARZALEX.

For more information, healthcare providers or patients can contact: 1-844-55DARZA (1-844-553-2792). Information will also be available atwww.DARZALEX.com. Dedicated case coordinators are available to work with both healthcare providers and patients.

About the COLUMBA Study 1The randomized, open-label, multicenter Phase 3 COLUMBA study (MMY3012) included 522 patients (median age of 67 years) with multiple myeloma who had received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD), or whose disease was refractory to both a PI and an IMiD. In the arm that received DARZALEX FASPRO(n=263), patients received a fixed dose of DARZALEX FASPRO1,800 milligrams (mg), co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) 2,000 Units per milliliter (U/mL), subcutaneously weekly for Cycles 1 2, every two weeks for Cycles 3 6 and every four weeks for Cycle 7 and thereafter. In the intravenous DARZALEXarm (n=259), patients received DARZALEXfor intravenous infusion 16 milligrams per kilogram (mg/kg) weekly for Cycles 1 2, every two weeks for Cycles 3 6 and every four weeks for Cycle 7 and thereafter. Each cycle was 28 days. In the arm that received DARZALEX FASPRO, itwas given in a fixed volume of 15 mL over three to five minutes; the median injection time was five minutes. In the arm that received theintravenous administration, the median durations of the first, second and subsequent intravenous DARZALEXinfusions were 7.0, 4.3 and 3.4 hours, respectively.Patients in both arms continued treatment until disease progression or unacceptable toxicity.

About the PLEIADES Study 2The non-randomized, open-label, parallel assignment Phase 2 PLEIADES study (MMY2040) included more than 240 adults with multiple myeloma, including 67 patients with newly diagnosed multiple myeloma who were treated with 1,800 mg of DARZALEX FASPROin combination with bortezomib, melphalan, and prednisone (D-VMP) and 65 patients with relapsed or refractory disease who were treated with 1,800 mg of DARZALEX FASPROplus lenalidomide and dexamethasone (D-Rd). The primary endpoint for the D-VMP and D-Rd cohorts was overall response rate.

About DARZALEXand DARZALEX FASPROJanssen is committed to exploring the potential of DARZALEX (daratumumab) for patients with multiple myeloma across the spectrum of the disease. DARZALEX has been approved in seven indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.

DARZALEX has become a backbone therapy in the treatment of multiple myeloma, having been used in the treatment of more than 58,000 patients in the U.S. alone since its U.S. FDA approval in 2015. DARZALEX is the first CD38-directed antibody approved globally to treat multiple myeloma and in 2020, DARZALEX FASPRO(daratumumab and hyaluronidase human-fihj) follows as the only subcutaneous CD38-directed antibody approved to treat patients with multiple myeloma.2

CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.4 DARZALEX binds to CD38 and inhibits tumor cell growth causing myeloma cell death.5 DARZALEX may also have an effect on normal cells.3 Data across seven Phase 3 clinical trials, in both the frontline and relapsed settings, have shown that DARZALEX-based regimens resulted in significant improvement in progression-free survival and/or overall survival. 4,5,6,7,8,9,10,11 Additional studies are underway to assess the efficacy and safety of DARZALEXFASPRO in the treatment of other malignant and pre-malignant hematologic diseases in which CD38 is expressed, including smoldering myeloma and in amyloidosis.12,13

Key DARZALEX Milestones:

Please see full Prescribing Information at http://www.DARZALEX.com.

About Multiple MyelomaMultiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.21,22When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow. In 2020, it is estimated that 32,270 people will be diagnosed and 12,830 will die from the disease in the U.S.24 While some patients with multiple myeloma have no symptoms, most patients are diagnosed due to symptoms, which can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.23

Please see full Prescribing Information at http://www.DARZALEX.com.

About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at http://www.janssen.com. Follow us at http://www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

ENHANZEis a registered trademark of Halozyme.

1Mateos M-V et al. Efficacy and Safety of the Randomized, Open-Label, Non-inferiority, Phase 3 Study of Subcutaneous (SC) Versus Intravenous (IV) Daratumumab (DARA) Administration in Patients (pts) With Relapsed or Refractory Multiple Myeloma (RRMM): COLUMBA. 2019 American Society of Clinical Oncology Annual Meeting. June 2019.

2Janssen Research & Development, LLC. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 [cited July 5, 2019]. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565. Identifier: NCT03412565.

32020Fedele G et al. CD38 Ligation in Peripheral Blood Mononuclear Cells of Myeloma Patients Induces Release of Protumorigenic IL-6 and Impaired Secretion of IFN Cytokines and Proliferation. Mediators Inflamm. 2013;564687.

4Janssen Research & Development, LLC. A Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02076009?term=mmy3003&rank=1 Identifier: NCT02136134 .

5Janssen Research & Development, LLC. Addition of Daratumumab to Combination of Bortezomib and Dexamethasone in Participants With Relapsed or Refractory Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02136134?term=mmy3004&rank=1 Identifier: NCT02076009.

6Janssen Research & Development, LLC. A Study to Evaluate Daratumumab in Transplant Eligible Participants With Previously Untreated Multiple Myeloma (Cassiopeia). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02541383?term=mmy3006 Identifier: NCT02541383.

7Janssen Research & Development, LLC. A Study of Combination of Daratumumab and Velcade (Bortezomib) Melphalan-Prednisone (DVMP) Compared to Velcade Melphalan-Prednisone (VMP) in Participants With Previously Untreated Multiple Myeloma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02195479?term=mmy3007&rank=1 Identifier: NCT02195479.

8Janssen Research & Development, LLC. Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Participants With Previously Untreated Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02252172?term=mmy3008&rank=1 Identifier: NCT02252172.

9Janssen Research & Development, LLC. A Study of VELCADE (Bortezomib) Melphalan-Prednisone (VMP) Compared to Daratumumab in Combination With VMP (D-VMP), in Participants With Previously Untreated Multiple Myeloma Who Are Ineligible for High-Dose Therapy (Asia Pacific Region). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT03217812?term=MMY3011&rank=1 Identifier: NCT03217812.

10European Myeloma Network. Compare Progression Free Survival Btw Daratumumab/Pomalidomide/Dexamethasone vs Pomalidomide/Dexamethasone (EMN14). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24] Available at: https://clinicaltrials.gov/ct2/show/NCT03180736?term=MMY3013&rank=2 Identifier: NCT03180736

11Amgen. Study of Carfilzomib, Daratumumab and Dexamethasone for Patients With Relapsed and/or Refractory Multiple Myeloma. (CANDOR). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24] Available at: https://clinicaltrials.gov/ct2/show/NCT03158688?term=NCT03158688&rank=1 Identifier: NCT03158688.

12Janssen Research & Development, LLC. A Study to Evaluate 3 Dose Schedules of Daratumumab in Participants With Smoldering Multiple Myeloma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 March 19]. Available at: https://clinicaltrials.gov/ct2/show/NCT02316106?term=smm2001&rank=1 Identifier: NCT02316106.

13Janssen Research & Development, LLC. An Efficacy and Safety Proof of Concept Study of Daratumumab in Relapsed/Refractory Mantle Cell Lymphoma, Diffuse Large B-Cell Lymphoma, and Follicular Lymphoma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 March 19]. Available at: https://clinicaltrials.gov/ct2/show/NCT02413489?term=lym2001&rank=1 Identifier: NCT02413489

14Janssen Biotech, Inc. "Janssen Biotech Announces Global License and Development Agreement for Investigational Anti-Cancer Agent Daratumumab." Issued August 30, 2012.

15Janssen Biotech, Inc. "DARZALEX (daratumumab) Approved by U.S. FDA: First Human Anti-CD38 Monoclonal Antibody Available for the Treatment of Multiple Myeloma." Issued November 16, 2015.

16Janssen Biotech, Inc. "DARZALEX (daratumumab) Approved by U.S. FDA in Combination with Two Standard of Care Regimens for the Treatment of Patients with Multiple Myeloma Who Have Received At Least One Prior Therapy." Issued November 21, 2016.

17Janssen Biotech, Inc. "DARZALEX (daratumumab) Approved by the U.S. FDA in Combination with Pomalidomide and Dexamethasone for Patients with Multiple Myeloma Who Have Received At Least Two Prior Therapies." Issued June 16, 2017.

18Janssen Biotech, Inc. "Janssen Announces DARZALEX (daratumumab) U.S. FDA Approval for Newly Diagnosed Patients with Multiple Myeloma who are Transplant Ineligible." Issued May 7, 2018.

19Janssen Biotech, Inc. "Janssen Announces U.S. FDA Approval of DARZALEX (daratumumab) in Combination with Lenalidomide and Dexamethasone for Newly Diagnosed Patients with Multiple Myeloma Who Are Transplant Ineligible." Issued June 27, 2019.

20Janssen Biotech, Inc. "Janssen Announces U.S. FDA Approval of DARZALEX (daratumumab) Combination Regimen for Newly Diagnosed, Transplant-Eligible Patients with Multiple Myeloma." Issued September 26, 2019.

21Kumar, SK et al. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012 Jan; 26(1):149-57.

22American Cancer Society. "What Is Multiple Myeloma?" Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed June 2019.

23American Cancer Society. "Key Statistics About Multiple Myeloma." Available at: https://www.cancer.org/cancer/multiple-myeloma/about/key-statistics.html. Accessed January 2020.

SOURCE: Janssen

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US Food and Drug Administration Approves DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a New Subcutaneous Formulation of Daratumumab in the...

Bone Marrow Stem Cells Comments Off on US Food and Drug Administration Approves DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a New Subcutaneous Formulation of Daratumumab in the… | May 2nd, 2020

Aging is an inevitable process. We cannot escape or prevent getting older but what if theres a fascinating field of medicine that can manage the aging process and prolong our health and vitality and longevity as we age?

Aging is an inevitable process. We cannot escape or prevent getting older but what if theres a fascinating field of medicine that can manage the aging process and prolong our health and vitality and longevity as we age?

Keeping in mind that there may never be an approach to totally stop or reverse aging, there have been some surprising disclosures to how Regenerative Medicine can naturally heal our body without the use of any surgical procedure.

Rejuvenating Old Cells to Healthy ones

The paces, stresses, and complexities in life drive us to age prematurely thereby breaking down our cells. Cell breakdown may lead to several health conditions like cancer, heart disease, Alzheimers and others.

Driving our bodies to age quickly, cell-breakdown is host to many age-related diseases, causing more than 100,000 deaths per day.

Dr David C Karli is an Ivy-trained physician, specialized in treating athletic injuries by inducing regenerative medicine and stem cell therapy in treatments.

He accepts the fact that patients can increase an additional 30 years of life by using Regenerative Medicine. One such innovation uses stem cells, however, there are issues with these cells.

They may not replace the original, diseased cells rapidly enough, or they may start to replicate uncontrollably, bringing about malignant growth.

Yet, Regenerative Medicine definitely guarantees the complete curing of a wide range of diseases, and ideally, slowing down the aging process too.

Stem Cell Therapy Programs with Promising Results

With solid funding and rapid advancements, one stem cell therapy that promises great outcomes is transfusions. In this therapy, stem cells are extracted from the patient and grown in cell culture to increase the number of cells. Following this, those cells are injected back into the patients body.

Dr. Karlis keen interest in Transfusions led him to create biologic products that can cause an age-related decline in a persons strength, endurance, and various other physical abilities.

At his biotech firm, Greyledge Technologies, biologic products are prepared by processing materials (blood or bone marrow) and implanting them into the human body to replicate the diseased tissues.

With an FDA-registered laboratory environment, the outcomes are promising and are an anti-aging protocol.

Telomeres may be the next-gen solution for Anti Aging

Telomeres are essential parts of our DNA that are connected to the premature aging cells. Situated at the end caps of our DNA strands, the information within Telomeres is lost while DNA replicates to the extent that they stop replicating.

If DNA replicates without losing information, scientists believe that Telomeres can significantly help to slow down the aging process.

Similar is the case with Metformin, a pharmaceutical reagent that improves wound healing. Proven to counteract aging, Metformin is now being tested for its unique ability to mimic calorie restriction.

Anti-Aging Through Regeneration

Utilizing induced tissue regeneration, this technology is a new approach to anti-aging treatment. Combining telomerase therapy and induced tissue regeneration, anti-aging through regeneration includes the study of the impact on age-related diseases like diabetes, metabolic disorders, cardiovascular disease, and others.

This technique focuses on the cells that are generated in our body during youth. As we age, these cells are lost and lead to a metabolic imbalance.

Scientists and Researchers are trying to find a way in which these cells can be restored to reverse the signs of aging and create a balance.

Humans have the ability to regenerate damaged and diseased tissues. However, this only happens during the first few weeks of development. With the help of Artificial Intelligence, scientists are trying to unlock this potential ability in humans.

The Future of Anti-Aging

With several breakthroughs on the horizon, cure-all promises and best outcomes, these anti-aging protocols have a long way to go.

While the introduction of regenerative medicine and stem cell therapies to redefine orthopedic treatment sounds like a miracle, there are still unexplored paths that need to be taken.

With all the benefits regenerative medicine has to offer, there will always be an eye on the never-ending search for the fountain of youth.

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Dr. David C. Karli's Opinion on Regenerative Medicine and Age Prevention | - SpaceCoastDaily.com

Bone Marrow Stem Cells Comments Off on Dr. David C. Karli’s Opinion on Regenerative Medicine and Age Prevention | – SpaceCoastDaily.com | May 2nd, 2020

Thursday, May 7, 2020, 8:30 a.m. EDT

NEW YORK, April 29, 2020 (GLOBE NEWSWIRE) -- BrainStorm-Cell Therapeutics Inc.(NASDAQ: BCLI), a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today, that the Company will hold a conference call to update shareholders on financial results for the first quarter endedMarch 31, 2020, and provide a corporate update, at 8:30 a.m, Eastern Daylight Time, onThursday, May 7, 2020.

BrainStorms CEO,Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will beRalph Kern, MD, MHSc, President and Chief Medical Officer, David Setboun, PhD, MBA, Executive Vice President and Chief Operating Officer andPreetam Shah, PhD, MBA, Executive Vice President and Chief Financial Officer.

Participants are encouraged to submit their questions prior to the call by sending them to:q@brainstorm-cell.com. Questions should be submitted by5:00 p.m. EDT, Tuesday, May 5, 2020.

Teleconference Details BRAINSTORM CELL THERAPEUTICS 1Q 2020

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the "Investors & Media" page of BrainStorm's website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available for 14 days.

ABOUT NUROWNNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

ABOUT BRAINSTORM CELL THERAPEUTICS INC.:BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in theU.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. BrainStorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

SAFE HARBOR STATEMENT:Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm-Cell Therapeutics to Announce First Quarter Financial Results and Provide a Corporate Update - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm-Cell Therapeutics to Announce First Quarter Financial Results and Provide a Corporate Update – Yahoo Finance | May 2nd, 2020

Updated: May 25, 2018:

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Biopharma Develops Antibody and Stem Cell Therapies in the Fight Against COVID-19 - JD Supra

Bone Marrow Stem Cells Comments Off on Biopharma Develops Antibody and Stem Cell Therapies in the Fight Against COVID-19 – JD Supra | May 1st, 2020

Cryopreservation has become an indispensable step in the daily routine of scientific research as well as in a number of medical applications, ranging from assisted reproduction and transplantations to cell-based therapies and biomarker identification. It is hardly possible to picture todays scientific and medical advancements without this technique.The successful development and implementation of all the therapeutic and scientific discoveries involving cryopreservation relies on the correct and safe translation of the method from the laboratory to the clinical and manufacturing scale.

With the need to correctly use this technique, more research is focusing on optimizing cryopreservation methods and investigating what the long-term effects and consequences are on the physiology of the cryopreserved material.

An important part of cell therapy research is focused on adult stem cells (ASCs). ASCs can be derived from different sources such as peripheral blood, bone marrow or adipose tissue and display strong promises because of their capacity to differentiate into any cell type of the human body.In recent work3, the team of Michael Pepper at the Institute for Cellular and Molecular Medicine in Pretoria, South Africa, explored the effects of cryopreservation on the differentiation ability of adipose tissue-derived stem cells (ADSCs). After analyzing gene expression of key adipogenic genes and the degree of differentiating cells, characterized with high levels of CD36 and intracellular lipid droplets, the scientists reported that slow freeze cryopreservation of cells shortly after their isolation causes no alterations on their ability to differentiate. Pepper is convinced of the necessity to perform such analysis when cryopreserving important cell pools: It is critical to do a post-thaw analysis of cell function to determine how the cryopreservation may have affected the cells.His team is analyzing the effects of cryopreservation on other cell types largely used in cell-based therapies such as hematological stem cells and peripheral blood mononuclear cells (PBMCs). Although they didnt observe major alterations in terms of immunophenotyping or the post-thaw proliferation of the cells, Pepper expresses his concern that more subtle characteristics might be affected.

Correct cryopreservation of cells intended for therapeutic use is crucial. This is very important particularly as cells may persist for a long time in the recipient. This area of cell therapy research definitely requires more attention, Pepper says. Moreover, his words reflect on the need to evaluate not only the direct post-thaw recovery, but to look deeper into the late-onset effects cryopreservation might have and ensure that transplanted cells have preserved their therapeutic properties.

In contrast to slow freezing, vitrification relies on the fast freezing of the material by putting it in high concentration of cryoprotectant and in contact with liquid nitrogen. This method allows the direct transition of water from liquid to solid state without crystal formation. The highly concentrated cryoprotectant prevents ice formation and therefore there is no need for slow cooling.

Although vitrification has a great potential, there are a couple of parameters that are a point of concern. The quick and drastic freeze is possible thanks to the high concentration of cryoprotectant, but the latter is also associated with higher toxicity. In some cases, an additional limitation is the direct contact of the sample with liquid nitrogen which is a predisposition for viral or bacterial contamination.The team of Christiani Amorim at the Institute for Experimental and Clinical Research in Louvain, Belgium, is approaching the challenges of vitrification in the context of ovarian auto-transplantation. Ovarian auto-transplantation consists of preserving a piece of ovarian tissue with active follicles from the pre-therapeutic ovary of a cancer patient, as chemotherapy often has damaging effects on the reproductive organs. This tissue sample will be conserved and auto-transplanted onto the patients ovary when she has recovered and wishes to become pregnant.In their recent research4, the authors used stepped vitrification, in which the concentration of the cryoprotectant is gradually increased while simultaneously temperature decreases. This avoids ice crystal formation and also prevents cryoprotectant toxicity.Although stepped vitrification has previously given good results in bovine ovarian tissue5, this was not the case for human ovarian tissue. The scientists didnt detect normal follicles following thawing and linked this to high cryoprotectant toxicity. Indeed, they observed all signs of dimethyl sulfoxide (DMSO)-related cell membrane damage: significant organelle damage, cell membrane disintegration and apoptosis. These observations imply on the variability of outcomes that the method could give when applied to the same type of tissue but from a different organism.Amorim is positive about the future of their method and recognizes the need for further research on the topic: I can see a great potential in the stepped vitrification approach, but I also believe that there is a lot we still need to learn before thinking about using it as method of choice for human ovarian tissue cryopreservation. The high cryoprotectant concentration that should be applied in this approach is my first concern. () Our study clearly showed that 50% DMSO is too high, so we need to try lower concentrations or combine it with other cryoprotectants.

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Freezing Life: The Current Trends in Cryopreservation - Technology Networks

Bone Marrow Stem Cells Comments Off on Freezing Life: The Current Trends in Cryopreservation – Technology Networks | May 1st, 2020

What is bone marrow cancer? Types, symptoms and treatment methods you must know  |  Photo Credit: Getty Images

New Delhi: Cancer is becoming a relatively very common disease, among people all around the world. It is a condition where abnormal cells divide uncontrollably in the body and destroy healthy body tissues as well. Cancer usually requires extensive treatment, and can also be life-threatening. Cancer accounted for 9.6 million deaths in 2018, according to WHO. Various popular personalities like Indian actors Irrfan Khan and Rishi Kapoor recently lost their lives to different types of cancer.

Rishi Kapoor was suffering from a bone marrow cancer, and breathed his last on Thursday morning, 30th of April. Here is what you need to know about bone marrow cancer, the cancer veteran actor was suffering from.

The bone marrow is a spongy tissue inside some of our bones. In humans, thesebones include the hips, thighs, etc. The bone marrow is the tissue where stem cells can develop into red blood cells, white blood cells, and platelets, all of which perform specific functions in the body.

Bone marrow cancer is a rare type of cancer. Bone marrow cancer happens when cells in the marrow start growing abnormally. It is a type of blood cancer and not bone cancer. Bone marrow cancer is the type that originates in the marrow and not the type that spreads to the bone or the marrow, after originating in some other part of the body.

Bone marrow cancer can be of various types, depending on the type of cells it affects -

Symptoms of bone marrow cancer vary, according to the type of cells that cancer affects. These include -

Multiple Myeloma Symptoms

Leukaemia symptoms

Lymphoma symptoms

Bone Marrow cancer can be treated by a medical professional. Chances of survival of the patient depend on the stage of cancer, how early the diagnosis is made, and other factors. Treatment methods include -

Disclaimer: Tips and suggestions mentioned in the article are for general information purposes only and should not be construed as professional medical advice. Always consult your doctor or a professional healthcare provider if you have any specific questions about any medical matter.

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What is bone marrow cancer? Leukaemia and other types, symptoms and treatment methods you must know - Times Now

Bone Marrow Stem Cells Comments Off on What is bone marrow cancer? Leukaemia and other types, symptoms and treatment methods you must know – Times Now | May 1st, 2020