The ability to turn human cells into anything we want sounds like the stuff of science fiction. But one Cambridge biotech says it's cracked the code

A sustainable source of human stem cells is one of the holy grails of modern medicine.

With applications as broad as re-growing failed organs, fighting cancer, and stopping animal testing, stem cell therapy is predicted to be worth US$35bn by 2023.

Now, Cambridge startup bit bio, has a new approach to re-coding skin cells from adult humans, and rewinding the clock to give them the power of stem cells, and then turn them into whatever we want them to be all without the controversial involvement of human embryos.

This, says neurosurgeon and founder Dr Mark Kotter, will democratise stem cells, so that anyone can use them, at any time.

The private sector is already placing big bets on the technology, with start-ups in the space raising as much as US$16mln in recent funding rounds.

Kotter says that our inability to produce enough human stem cells to match our need puts troubling limits on research and drug development.

In drug discovery, the biggest bottleneck is the mismatch between animal models and animal cell lines used for drug discovery, and then human setting used in the clinical trial, he explains.

Around 3% of new drugs make it all the way through trials and to market, he says, and the biggest reasons treatments tend to fail in clinical study is that they are either toxic to humans, or they dont work.

The only solution is to bring the human element back to the early stages, says Kotter.

If new therapies were tested on human tissue first, it would reduce or even bypass the need to test on animals, as well as speeding up development.

Kotter founded bit bio, formerly known as Elpis BioMed, in 2016, in addition to startup Meatable, which produces meat by growing cultures in the lab, rather than rearing animals for the table.

The time is now for bit bio, because what it is doing has only been possible since a Nobel Prize-winning discovery twelve years ago, which turned the world of stem cell research upside down.

Kyoto University researcher Shinya Yamanaka proved that it was possible to take a mature human skin cell and reprogram it to be like the stem cell of an embryo.

Until this revelation, stem cell research had been dogged by controversy and expense, as scientists had to use human embryos and umbilical cords as a source of stem cells, and then simulate complex conditions inside the womb in order to make them develop into the cells they desired.

One big problem in early cell reprogramming was that stem cells are incredibly alert to invading DNA and silences any foreign material it detects.

This meant that past attempts run a different program inside a cell often failed, because the cell destroyed it.

What happened next was a moment of "serendipity" in the lab, says Kotter.

Through trial and error, bit bio found they could use certain safe harbours where information is protected within cells, to stop theinterference.

By taking the genetic switch for gene silencing and placing it inside a safe harbour, and then separately running the new cell program inside another safe harbour, scientists found they could override gene silencing in order to change the cell type.

This approach is what Kotter says makes bit bio unique.

The lab can produce up to a kilogram of human cells now, and its tech platform OptiOx has also proved that it can generate two human cell types with 100% accuracy.

Kotter says that now the range of cells able to be produced matters more than the quantity.

The company is now focused on discovering what separates one type of cell from another, which Kotter says will allow the firm to decode the building blocks of life.

To this end, bit bio is using machine learning to analyse the differences between every type of human cell, from bone marrow cells to liver cells, and create a reference map for all the different types.

Once the research is complete, the company hopes it willbe able to generate any type of human cell, at scale, and with ultimate precision.

Preparations are underway for a Series A funding round, and Kotter says that he is determined not to sell the business, having already rejected offers from would-be buyers.

Bit bio though is in an area hot with competition, which moves quickly.

A US$16mln Series A mega funding round was recently announced in October by another Cambridge start-up, Mogrify, which is hoping to master direct cell reprogramming and turn blood cells straight into brain cells, or any other type.

Mogrify uses big data to identify the small molecules needed to convert, maintain and culture a target cell type.

While both companies were finalists in the 2018 Cambridge Startup of the Year award, bit bio was the one to scoop the prize.

One aspect that separates the two companies is that Mogrify uses its technology to turn cells directly into other cell types, rather than using it to rewindto the stem cell phase, which is when cells can reproduce very quickly,

Kotter says that this stem cell phase focusis whatallows bit bio to havea stable supply of human cells.

If bit bio completes a similar, or even bigger, fundraise, it could advance the fledgling firm from seed to stem, in its attempt to stabilise a production line for essential cell technology.

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Decoding the building blocks of life: bit bio races toward a sustainable source of human cells - Proactive Investors UK

Bone Marrow Stem Cells Comments Off on Decoding the building blocks of life: bit bio races toward a sustainable source of human cells – Proactive Investors UK | November 20th, 2019

Simple arthritis drugs used on the NHS could help stop breast cancer spreading, research suggests.

Scientists propose that arthritis drugs anakinra, canakinumab and sulfasalazine could be re-purposed to help block cancer reaching bones.

Research teams from the University of Manchester and the University of Sheffield discovered that a protein released by bone marrow, called interleukin 1-beta encouraged breast cancer cells to form secondary tumours once they reach the bone.

Tests on mice found that this molecule can be blocked by drugs already used to treat arthritis.

Read more about cancer treatment:

The study, largely funded by charity Breast Cancer Now and published in the Nature Communications journal, revealed that anakinra was able to prevent breast cancer forming secondary tumours in the bone.

Follow treatment with anakinra, only 14 per cent of mice in the study developed secondary tumours in the bone, compared to 42 per cent of control animals.

According to Breast Cancer Now, around 55,000 women and 370 men are diagnosed with breast cancer each year, making it the UKs most common cancer.About 11,500 women die from the disease each year, almost all from tumours that have spread to other parts of the body.Breast cancer most commonly spreads to the bones, brain, lungs or liver.

Breast cancer stem cells are thought to be responsible for the spread of the disease, with previous research suggesting healthy cells released certain molecules to help the cancer stem cells settle and grow in new locations.

In their new study, researchers grew breast cancer cells using liquid that bone marrow had grown in. They found the cancer cells grew more easily than cells not exposed to these conditions and then identified interleukin 1-beta as the molecule responsible.

It is hoped the findings will lead to trials in women with breast cancer to help prevent cancer spreading to the bone.

Scientists believe more work is needed to understand how Arthritis drugs could halt the spread of breast cancer, study suggestsritis drugs might interact with the immune system and other cancer therapies.

Read more about breast cancer:

Lead author of the study, Dr Rachel Eyre, from the University of Manchester, said: We will now look to see if similar processes are also involved in breast cancer growing in other organs, such as the liver and lungs.

We hope that by continuing this work, we could in future identify those at high risk of their breast cancer spreading, and where possible use drugs already available to prevent this from happening.

Baroness Delyth Morgan, chief executive of Breast Cancer Now, said: These major findings offer another promising step forward in re-purposing existing drugs to prevent the spread of breast cancer.

While more research is needed, its really exciting that these well-tolerated and widely-available arthritis drugs may help prevent secondary breast cancer in the bone.

Continued here:
Arthritis drugs could halt the spread of breast cancer, study suggests - sciencefocus.com

Bone Marrow Stem Cells Comments Off on Arthritis drugs could halt the spread of breast cancer, study suggests – sciencefocus.com | November 20th, 2019

TEL AVIV, Israel, Nov. 19, 2019 /PRNewswire/ --Cellect Biotechnology Ltd. (APOP), a developer of innovative technology which enables the functional selection of stem cells, today reported financial and operating results for the third quarter ended September 30, 2019 and provided a corporate update.

Recent Highlights

"Our clinical and regulatory teams remained focused during the third quarter and the more recent positive developments position us to achieve our goals, both in the U.S. and Israel," commented Dr. Shai Yarkoni, Chief Executive Officer. "In the U.S., the IND approval is a significant achievement and represents our first-ever FDA IND in the U.S., with Washington University School of Medicine. In Israel, our Phase 1/2 clinical study of ApoGraft is progressing slowly and we expect to complete the recruitment around the end of the year."

"With our prudent use of cash during the third quarter and the anticipated cash usage needs over the coming quarters, we continue to believe we have the resources to execute our clinical and regulatory plans for the foreseeable future," said Eyal Leibovitz, Chief Financial Officer.

ThirdQuarter 2019 Financial Results:

*For the convenience of the reader, the amounts above have been translated from NIS into U.S. dollars, at the representative rate of exchange on September 30, 2019 (U.S. $1 = NIS 3.482).

Strategic Review Progress Update

On May 16, 2019, the Company disclosed that it commenced plans to explore strategic alternatives to maximize shareholder value. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, including in other business fields than the Company's in-licensing, or other strategic transaction involving the Company or its assets. The Company continues to evaluate business development opportunities and will keep investors informed as they mature or warrant investor disclosure.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the selection of stem cells from any given tissue, that aims to improve a variety of stem cell-based therapies.

The Company's technology is expected to provide researchers, clinical community and pharma companies with the tools to rapidly isolate stem cells in quantity and quality allowing stem cell-based treatments and procedures in a wide variety of applications in regenerative medicine. The Company's current clinical trial is aimed at bone marrow transplantations in cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Company's history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Company's ability to continue as a going concern; or maintain its current operations; uncertainties involving any strategic transaction the Company may decide to enter into as the result of its current efforts to explore new strategic alternatives; uncertainties of cash flows and inability to meet working capital needs; the Company's ability to obtain regulatory approvals; the Company's ability to obtain favorable pre-clinical and clinical trial results; the Company's technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Company's clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Company's ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Company's industry; unforeseen scientific difficulties may develop with the Company's technology; and the Company's ability to retain or attract key employees whose knowledge is essential to the development of its products. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2018 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov, and in the Company's periodic filings with the SEC.

Story continues

Cellect Biotechnology Ltd

Consolidated Statement of Operation

Convenience

translation

Nine months

ended

Nine months ended

Three months ended

September 30,

September 30,

September 30,

2019

2019

2018

2019

2018

Unaudited

Unaudited

U.S. dollars

NIS

(In thousands, except share and per

share data)

Research and development expenses

2,743

9,551

9,473

2,465

4,125

General and administrative expenses

2,249

7,832

11,001

2,768

3,929

Operating loss

4,992

17,383

20,474

5,233

8,054

Financial expenses (income) due to warrants exercisable into shares

(2,303)

(8,020)

(2,935)

(910)

(1,320)

Other financial expenses (income), net

393

1,369

(1,177)

489

64

Total comprehensive loss

3,082

10,732

16,362

4,812

6,798

Loss per share:

Basic and diluted loss per share

0.015

0.051

0.127

0.021

0.052

Basic and diluted loss per ADS

0.30

1.02

2.54

0.42

1.04

Weighted average number of shares outstanding used to compute basic and diluted loss per share

208,771,303

208,771,303

129,139,278

224,087,799

130,192,799

Cellect Biotechnology Ltd.

Consolidated Balance Sheet Data

ASSETS

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Cellect Biotechnology Reports Third Quarter 2019 Financial and Operating Results - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Cellect Biotechnology Reports Third Quarter 2019 Financial and Operating Results – Yahoo Finance | November 20th, 2019

Bone marrow aspirationand trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

Get More Information at Professional:https://www.trendsmarketresearch.com/report/sample/3374

Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants areconducted in Europe, and it is oneof the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy.

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Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

Full View of Report Description:https://www.trendsmarketresearch.com/report/bone-marrow-processing-systems-market

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Bone Marrow Processing Systems Market With Industry Overview, Supply Chain, Key Players, & Analysis To 2025 - Guru Online News

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing Systems Market With Industry Overview, Supply Chain, Key Players, & Analysis To 2025 – Guru Online News | November 20th, 2019

ARTHRITIS drugs available on the NHS help stop the spread of breast cancer, a study suggests.

They blocked secondary tumours fuelled by a bone marrow protein called interleukin 1-beta, researchers found.

1

In tests, just 14 per cent of mice treated with arthritis drug anakinra developed secondary tumours in the bone, compared with 42 per cent in a control group.

Other arthritis drugs canakinumab and sulfasalazine had similar effects, the Manchester University and Sheffield University researchers found.

Dr Rachel Eyre told journal Nature Communications: We will now look to see if similar processes are also involved in breast cancer growing in organs such as the liver and lungs.

The study was largely funded by charity Breast Cancer Now.

According to Breast Cancer Now, around 55,000 women and 370 men are diagnosed with breast cancer each year, making it the UK's most common cancer.

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About 11,500 women die from the disease each year, almost all from tumours that have spread to other parts of the body.

Breast cancer most commonly spreads to the bones, brain, lungs or liver.

Breast cancer stem cells are thought to be responsible for the spread of the disease, with previous research suggesting healthy cells released certain molecules to help the cancer stem cells settle and grow in new locations.

View original post here:
Arthritis drugs available on the NHS help stop the spread of breast cancer to bones - The Sun

Bone Marrow Stem Cells Comments Off on Arthritis drugs available on the NHS help stop the spread of breast cancer to bones – The Sun | November 20th, 2019

TEL AVIV, Israel, Nov. 19, 2019 /PRNewswire/ --Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of stem cells, today reported financial and operating results for the third quarter ended September 30, 2019 and provided a corporate update.

Recent Highlights

"Our clinical and regulatory teams remained focused during the third quarter and the more recent positive developments position us to achieve our goals, both in the U.S. and Israel," commented Dr. Shai Yarkoni, Chief Executive Officer. "In the U.S., the IND approval is a significant achievement and represents our first-ever FDA IND in the U.S., with Washington University School of Medicine. In Israel, our Phase 1/2 clinical study of ApoGraft is progressing slowly and we expect to complete the recruitment around the end of the year."

"With our prudent use of cash during the third quarter and the anticipated cash usage needs over the coming quarters, we continue to believe we have the resources to execute our clinical and regulatory plans for the foreseeable future," said Eyal Leibovitz, Chief Financial Officer.

ThirdQuarter 2019 Financial Results:

*For the convenience of the reader, the amounts above have been translated from NIS into U.S. dollars, at the representative rate of exchange on September 30, 2019 (U.S. $1 = NIS 3.482).

Strategic Review Progress Update

On May 16, 2019, the Company disclosed that it commenced plans to explore strategic alternatives to maximize shareholder value. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, including in other business fields than the Company's in-licensing, or other strategic transaction involving the Company or its assets. The Company continues to evaluate business development opportunities and will keep investors informed as they mature or warrant investor disclosure.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the selection of stem cells from any given tissue, that aims to improve a variety of stem cell-based therapies.

The Company's technology is expected to provide researchers, clinical community and pharma companies with the tools to rapidly isolate stem cells in quantity and quality allowing stem cell-based treatments and procedures in a wide variety of applications in regenerative medicine. The Company's current clinical trial is aimed at bone marrow transplantations in cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Company's history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Company's ability to continue as a going concern; or maintain its current operations; uncertainties involving any strategic transaction the Company may decide to enter into as the result of its current efforts to explore new strategic alternatives; uncertainties of cash flows and inability to meet working capital needs; the Company's ability to obtain regulatory approvals; the Company's ability to obtain favorable pre-clinical and clinical trial results; the Company's technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Company's clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Company's ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Company's industry; unforeseen scientific difficulties may develop with the Company's technology; and the Company's ability to retain or attract key employees whose knowledge is essential to the development of its products. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2018 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov, and in the Company's periodic filings with the SEC.

Cellect Biotechnology Ltd

Consolidated Statement of Operation

Convenience

translation

Nine months

ended

Nine months ended

Three months ended

September 30,

September 30,

September 30,

2019

2019

2018

2019

2018

Unaudited

Unaudited

U.S. dollars

NIS

(In thousands, except share and per

share data)

Research and development expenses

2,743

9,551

9,473

2,465

4,125

General and administrative expenses

2,249

7,832

11,001

2,768

3,929

Operating loss

4,992

17,383

20,474

5,233

8,054

Financial expenses (income) due towarrants exercisable into shares

(2,303)

(8,020)

(2,935)

(910)

(1,320)

Other financial expenses (income), net

393

1,369

(1,177)

489

64

Total comprehensive loss

3,082

10,732

16,362

4,812

6,798

Loss per share:

Basic and diluted loss per share

0.015

0.051

0.127

0.021

0.052

Basic and diluted loss per ADS

0.30

1.02

2.54

0.42

1.04

Weighted average number of sharesoutstanding used to compute basic anddiluted loss per share

208,771,303

208,771,303

129,139,278

224,087,799

130,192,799

Cellect Biotechnology Ltd.

Consolidated Balance Sheet Data

ASSETS

Continue reading here:
Cellect Biotechnology Reports Third Quarter 2019 Financial and Operating Results - BioSpace

Bone Marrow Stem Cells Comments Off on Cellect Biotechnology Reports Third Quarter 2019 Financial and Operating Results – BioSpace | November 20th, 2019

On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. Shed just moved in and hadnt had time to decorate. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket.

Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Doctors thought he had eczema and cradle cap. They said he was allergic to his mothers milk and told her to stop breastfeeding. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the babys bizarre symptoms, which didnt make any sense until physicians, finally, ordered the right blood test.

What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the bubble boy disease. Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). The bubble boy name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta.

Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Childrens Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. I had no idea what would happen to him, his mother recalled.

Approximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. Only about 20 to 50 new cases of the SCID-X1 mutationwhich accounts for about half of all SCID casesappear in the United States each year. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. But fewer than 20 percent of patients have had this option. And Omarion, an only child, was not among them.

As it happened, medical scientists at St. Jude Childrens Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patients own stem cells, which then go on to produce the immune system components needed to fight infection. Simpson enrolled Omarion in the clinical study and Cincinnati Childrens Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months.

St. Jude wasnt the first to try gene therapy for SCID-X1. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.

At St. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldnt have side effects. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. They engineered this vector to include insulators to prevent the vector from disturbing other genes once it integrated into the human genome. The goal was to insert the gene into stem cells that had come from the patients own bone marrow, and those cells would then go on to produce working immune system cells. It was crucial for the viral vector to not deliver the gene to other kinds of cellsand thats what the researchers observed. After gene therapy, for example, brain cells do not have a correct copy of the gene, explained Stephen Gottschalk, who chairs St. Judes Department of Bone Marrow Transplantation and Cellular Therapy.

In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. They went through a two-day, low-dose course of chemotherapy, which made room for the engineered cells to grow. Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system. The remarkably positive results made news headlines after being published this past April in the New England Journal of Medicine. Experimental gene therapy frees bubble boy babies from life of isolation, the journal Nature trumpeted.

So far, the children who participated in that study are thriving, and so are several other babies who received the treatmentincluding Omarion. As a physician and a mom, I couldnt ask for anything better, said Ewelina Mamcarz, lead author of the journal article and first-time mother to a toddler nearly the same age as Omarion. The children in the study are now playing outside and attending day care, reaching milestones just like my daughter, Mamcarz says. Theyre no different. Mamcarz, who is from Poland, came to the United States to train as a pediatric hematologist-oncologist and joined St. Jude six years ago.

Other medical centers are pursuing the treatment. The University of California, San Francisco Benioff Childrens Hospital is currently treating infant patients, and Seattle Childrens Hospital is poised to do the same. Moreover, the National Institutes of Health has seen success in applying the gene therapy to older patients, ages 3 to 37. Those participants had previously received bone marrow transplants from partially matched donors, but theyd been living with complications.

In the highly technical world of medicine today, it takes teamwork to achieve a breakthrough, and as many as 150 peoplephysicians, nurses, regulators, researchers, transplant coordinators and othersplayed a role in this one.

Sorrentino died in November 2018, but hed lived long enough to celebrate the trial results. In the early 90s, we thought gene therapy would revolutionize medicine, but it was kind of too early, said Gottschalk, who began his career in Germany. Now, nearly 30 years later, we understand the technology better, and its really starting to have a great impact. We can now develop very precise medicine, with very limited side effects. Gottschalk, who arrived at St. Jude a month before Sorrentinos diagnosis, now oversees the hospitals SCID-X1 research. Its very, very gratifying to be involved, he said.

For now the SCID-X1 gene therapy remains experimental. But with additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesnt live in a bubble or, for that matter, in a hospital. He can play barefoot in the dirt with other kids, whatever he wants, because his immune system is normal like any other kid, she said. I wish there were better words than thank you.

Original post:
These Scientists May Have Found a Cure for 'Bubble Boy' Disease - Smithsonian.com

Bone Marrow Stem Cells Comments Off on These Scientists May Have Found a Cure for ‘Bubble Boy’ Disease – Smithsonian.com | November 20th, 2019

I could barely think, because all the small blood vessels in my head were getting this thick blood pumping through.

In addition to bad headaches and crippling fatigue, PV sufferers are also prone to blood clots, which can pose a separate health risk.

According to the Leukaemia Foundation, PV is diagnosed in an estimated 250 Australians each year, and is one of three types of blood cancers called MPNs.

Associate Professor Steven Lane says the longer-lasting the version of interferon, the greater the effect it appears to have on PV.

Standard treatment for the condition is old-fashioned bloodletting to help thin the blood, combined with drugs that target the bone marrow, where the cancers stem cells are located.

Associate Professor Steven Lane, who is the head of QIMR Berghofers Cancer Program and a Royal Brisbane and Womens Hospital clinical haematologist, said the current most effective drug on the market in Australia was a form of interferon, a protein-based anti-cancer drug.

However, the form used most widely had to be taken up to three times a week, which, given the drug will often have to be used for the rest of the patients life, was not ideal.

A long-acting version of the drug has now been approved for use in Australia that only needs to be given once a week we call it the long-acting version of the drug, Dr Lane said.

Theres actually an even longer-acting version of the drug, an ultra-long-acting version, which has been approved in Europe but not Australia, and wed like to see that approved here.

Thats because Dr Lanes recent research into PVs response to drug therapy found that, in mice, the longer-acting the version of interferon, the better the effect on reducing the cancer symptoms.

People need to remain on this drug for a long time, and it actually doesnt usually start having an effect for six months, he said.

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And as you increase the duration of action of the drug, not only is it more convenient for the people who need to inject themselves with it, it appears to be more effective as well.

Ms Chapman switched to the long-acting version of interferon around a year ago, and said she finally felt a sudden improvement in her condition just three months ago, just ahead of her 50th birthday in December, and her daughters 21st.

When youve been living with this for 12 or 13 years, you sometimes forget what its like to feel normal, she said.

I would love to get access to the ultra-long-acting version it would be wonderful to have even fewer injections, and better control over the disease as well.

The QIMR research has been published in the journal Leukemia.

Our journalists abide by a set of reporting guidelines when writing about medical research. If you would like to read them click here.

Stuart Layt covers health, science and technology for the Brisbane Times. He was formerly the Queensland political reporter for AAP.

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Call for better drug to be approved for sufferers of rare blood cancer - The Age

Bone Marrow Stem Cells Comments Off on Call for better drug to be approved for sufferers of rare blood cancer – The Age | November 20th, 2019

Autologous stem cell and non-stem cell based therapiesinvolve an individuals cell to be cultured and then re-introduced to the donors body. These therapies do not use foreign organism cells and are therefore free from HLA incompatibility, disease transmission, and immune reactions.Increasing demand for the new therapies in the field of regenerative medicine is directly facilitating the growth of autologous stem cell and non-stem cell based therapies market. Furthermore, since the risk to transplantation surgeries is significantly reduced in these therapies, they are increasingly being preferred for treatment of bone marrow diseases, aplastic anemia, multiple myeloma, non-Hodgkins lymphoma, Hodgkins lymphoma, Parkinsons disease, thalassemia, and diabetes.

Moreover, rising incidents of cancer, diabetes and cardiovascular diseases along with growing geriatric population is another factor attributed for its high growth. However, side-effects of autologous stem cell and non-stem cell based therapies such as nausea, infection, hair loss, vomiting, diarrhea, etc. are expected to affect the market to an extent. High cost is another factor that can act as challenge to autologous stem cell and non-stem cell based therapies market. In spite of this, less risk post transplantation surgeries and favorable tax reimbursement policies are anticipated to reduce the impact of these limitation during the forecast period.Autologous stem cell and non-stem cell based therapies market can be segmented on the basis of application, end-user, and region.

In terms of application, the autologous stem cell and non-stem cell based therapies market can be segmented into blood pressure (BP) monitoring devices, intracranial pressure (ICP) monitoring devices, and pulmonary pressure monitoring devices.

In terms of end-user, the market can be segmented into ambulatory surgical center and hospitals. By region, the market can be segmented into North America, Europe, Asia Pacific, Middle East and Africa and South America. Amongst all, Asia Pacific is anticipated to be the most attractive market owing to favorable reimbursement policies in the region.The players operating in autologous stem cell and non-stem cell based therapies market are limited. They are consistently involved in research and development activities for product development to keep up with the growing competition, thereby aiding the growth of autologous stem cell and non-stem cell based therapies market across the world.

The major players operating in autologous stem cell and non-stem cell based therapies market are Regennex, Antria(Cro), Bioheart, Orgenesis Inc., Virxys corporation , Dendreon Corporation, Tigenix, Georgia Health Sciences University, Neostem Inc, Genesis Biopharma, Brainstorm Cell Therapeutics, Tengion Inc., Fibrocell Science Inc., Opexa Therapeutics Inc, Regeneus Ltd, and Cytori Inc., among others.

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Autologous Stem Cell And Non Stem Cell Based Therapies Market Opportunity Analysis and Industry Forecast up to 2026 - Guru Online News

Bone Marrow Stem Cells Comments Off on Autologous Stem Cell And Non Stem Cell Based Therapies Market Opportunity Analysis and Industry Forecast up to 2026 – Guru Online News | November 20th, 2019

Adipose Derived Stem Cell Therapy Market Report 2018-2026includes a comprehensive analysis of the present Market. The report starts with the basic Adipose Derived Stem Cell Therapy industry overview and then goes into each and every detail.

Adipose Derived Stem Cell Therapy Market Report contains in depth information major manufacturers, opportunities, challenges, and industry trends and their impact on the market forecast. Adipose Derived Stem Cell Therapy also provides data about the company and its operations. This report also provides information on the Pricing Strategy, Brand Strategy, Target Client, Distributors/Traders List offered by the company.

Description:

Adipose derived stem cells (ADSCs) are stem cells derived from adipocytes, and can differentiate into variety of cell types. ADSCs have multipotency similar to bone marrow mesenchymal stem cells, thus ADSCs substitute for bone marrow as a source of stem cells. Numerous manual and automatic stem cell separation procedures are adopted in order to separate adipose stem cells (ASCs) from adipose tissue. Flow cytometry can also be used to isolate ADSCs from other stem cells within a cell solution.

Get Request Sample Copy of Research Report @ https://www.coherentmarketinsights.com/insight/request-sample/2357

Important Features that are under offer & key highlights of the report:

1) What all regional segmentation covered? Can the specific country of interest be added?Currently, the research report gives special attention and focus on the following regions:North America (U.S., Canada, Mexico), Europe (Germany, U.K., France, Italy, Russia, Spain etc), South America (Brazil, Argentina etc) & Middle East & Africa (Saudi Arabia, South Africa etc)** One country of specific interest can be included at no added cost. For inclusion of more regional segment quote may vary.

2) What all companies are currently profiled in the report?The report Contain the Major Key Players currently profiled in this market.** List of companies mentioned may vary in the final report subject to Name Change / Merger etc.

3) Can we add or profiled new company as per our need?Yes, we can add or profile new company as per client need in the report. Final confirmation to be provided by the research team depending upon the difficulty of the survey.** Data availability will be confirmed by research in case of a privately held company. Up to 3 players can be added at no added cost.

4) Can the inclusion of additional Segmentation / Market breakdown is possible?Yes, the inclusion of additional segmentation / Market breakdown is possible to subject to data availability and difficulty of the survey. However, a detailed requirement needs to be shared with our research before giving final confirmation to the client.** Depending upon the requirement the deliverable time and quote will vary.

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Keyplayers :

Adipose Derived Stem Cell Therapy Market competition by top manufacturers/players, with Adipose Derived Stem Cell Therapy sales volume, Price (USD/Unit), Revenue (Million USD) and Market Share for each manufacturer/player; the top players including: BioRestorative Therapies, Inc., Celltex Therapeutics Corporation, Antria, Inc., Cytori Therapeutics Inc., Intrexon Corporation, Mesoblast Ltd., iXCells Biotechnologies, Pluristem Therapeutics, Inc., Thermo Fisher Scientific, Inc., Tissue Genesis, Inc., Cyagen US Inc., Celprogen, Inc., and Lonza Group, among others.

Adipose Derived Stem Cell Therapy Market Dynamics in the world mainly, the worldwide 2018-2026 Adipose Derived Stem Cell Therapy Market is analyzed across major global regions. CMI also provides customized specific regional and country-level reports for the following areas:

Region Segmentation:

North America (USA, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Columbia etc.)Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Key questions answered in the report:

1. What will the market growth rate of Adipose Derived Stem Cell Therapy market in 20262. What are the key factors driving the global Adipose Derived Stem Cell Therapy market3. Who are the key manufacturers in Adipose Derived Stem Cell Therapy market space?4. What are the market opportunities, market risk and market overview of the Adipose Derived Stem Cell Therapy market?5. What are sales, revenue, and price analysis by types and applications of Adipose Derived Stem Cell Therapy market?6. What are sales, revenue, and price analysis by regions of Adipose Derived Stem Cell Therapy industry?

Further in the report, the Adipose Derived Stem Cell Therapy market is examined for Sales, Revenue, Price and Gross Margin. These points are analyzed for companies, types, and regions. In continuation with this data, the sale price is for various types, applications and region is also included. The Adipose Derived Stem Cell Therapy industry consumption for major regions is given. Additionally, type wise and application wise figures are also provided in this report.

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In this study, the years considered to estimate the market size of 2018-2026 Adipose Derived Stem Cell Therapy Market are as follows:History Year: 2015-2017Base Year: 2017Estimated Year: 2018Forecast Year 2018 to 2026

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Adipose Derived Stem Cell Therapy Market New Business Opportunities and Investment Research Report - Bishop's Cleeve Bulletin

Bone Marrow Stem Cells Comments Off on Adipose Derived Stem Cell Therapy Market New Business Opportunities and Investment Research Report – Bishop’s Cleeve Bulletin | November 20th, 2019

Just a few months after Health Canada began cracking down on private clinics offering unapproved stem cell therapies, at least one U.S. clinic has moved in to fill the vacuum with direct marketing to Canadian consumers.

The clinic from Burlington, Vermont, even offers shuttle buses to transport people from Ottawa to the clinic four hours away for treatment it suggests will end joint pain, among other things. Lunch and dinner are free, but each injection costs $6,880. Two for $10,880.

The treatments, using umbilical cord-derived mesenchymal stem cells, are not approved in either Canada or the United States. Health Canada warns that Canadians who travel abroad for stem cell treatments may put themselves at risk.

While stem cells, which were discovered at the University of Toronto in 1961 by James Till and Ernest McCulloch, promise to revolutionize many treatments and could offer breakthroughs for diseases, almost all are still considered experimental and have yet to be proven safe or effective. Clinical trials on numerous potential stem cell therapies are under way, including in Ottawa.

While research progresses, private stem cell clinics have popped up around the world making promises for treatments not yet proven safe or effective.

A 2018 study by Leigh Turner of the University of Minnesota Center for Bioethics found 43 clinics offering stem cell treatments in Canada and 750 in the U.S. Earlier this year, Health Canada sent Canadian clinics, including some in Ottawa, cease-and-desist letters.

Clinics in Vermont, near the Canadian border, appear to have ramped up marketing to Canadians since then. One clinic has been holding back-to-back seminars. Another says it stopped marketing in Canada after receiving a warning from Health Canada.

There have been cases of harm as a result of treatments, including two women who had permanent damage to their sight after stem cells were injected into their eyes at a Florida clinic. Other patients have been infected with unsterilized equipment and others have developed tumours at the site of stem cell injections.

A common harm, critics say, is exploitation.

Dr. Michael Rudnicki is director of the regenerative medicine program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute, says of stem cell therapy claims: If it sounds too good to be true, it probably is too good to be true.jpg

Health officials say the clinics are misusing the promise of stem cell therapy to exploit vulnerable patients.

These patients are in pain and they are suffering and they are looking for help and they are being exploited, said Dr. Michael Rudnicki, director of the regenerative medicine program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute.

If it sounds too good to be true, it probably is too good to be true.

At a recent seminar at a west-end Ottawa hotel meeting room, Roseanna Ammendolea of the Vermont Center for Regenerative Medicine told a packed room that her clinic and others like it had successfully treated people for pain related to arthritis, neuropathy and other ailments that affected joints using mesenchymal stem cells from umbilical cords. The stem cells, she claimed, are both effective and safe, saying there had been no issues with cell rejection.

We will not give injections if we feel that this injection will not be beneficial to our patients. This is why we are so successful.

Participants, including some who walked with canes and others who talked about being in pain and having mobility issues, were shown videos of people described as Canadian clients who claimed the treatments worked. One man said it was probably the best money I have spent in my life as far as my health. Another said she would do it again in a heartbeat and was able to do things she hadnt been able to do earlier.

They were also shown a slide showing long wait times for hip and knee replacements in Ontario, We are not a priority, she said. Where does that leave us? Participants werent told exactly how the stem cells were supposed to work, but claimed they had successfully improved pain and mobility issues in clients.

What the seminar goers werent told is that, even in the U.S., the treatment is not covered by health insurance because it remains unproven.

The U.S. Federal Drug Administration has issued a warning to consumers not to use cell therapies that are unapproved or unproven.

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

The only stem-cell-based products that are FDA-approved for use in the United States are blood-forming stem cells derived from cord blood for limited use in patients with disorders affecting the body system that is involved in the production of blood. Bone marrow is also used for these treatments, but is generally not regulated by the FDA for that use.

Health Canada has granted market authorization for a stem cell therapy to treat graft-versus-host disease and two cell-based gene therapies to treat certain cancers. Most cell therapies are still experimental.

I totally understand the skepticism of it, Doug Argento, who works at the Vermont Center for Regenerative Medicine, said in a telephone interview, but the fact is that things that are approved now and medically paid for were seen as renegade 20 or 30 years ago.

The treatment employs technology developed by Neil Riordan, founder, chairman and chief science officer of the Stem Cell Institute in Panama, using human umbilical cord tissue-derived mesenchymal stem cells. There are 41 such clinics across the U.S. Riordan also played a role in the development of a nutritional product called Stem-Kine, which producers claim without scientific backing increases the number of stem cells circulating in a persons body.

The stem cells injected in the clinic, Argento said, are from umbilical cord tissue as a result of caesarean births to reduce risk of infection.

Rudnicki, of The Ottawa Hospital Research Institute, says there is no evidence that these sorts of cells are regenerative at all. It would not pass muster in Canada.

The public has to understand that there are people out to remove them from their money.

Rudnicki says he regularly receives inquiries from people desperate to get stem cell treatments. He says he tries to connect them with clinical trials that they might be able to participate in.

Rudnicki noted there were multiple clinical trials in Canada, including treatments of autoimmune diseases, trials involving treatment for Type 1 diabetes and others.

But the use of these inappropriate cell types for treating arthritis and joints and so on is certainly not approved by Health Canada and would not be allowed in Canada under the regulations.

There is some evidence that injections of some stem cell products might have a temporary positive impact on inflammation, he said, but it will not be regenerative and will not restore function to joints. They are being sold a bill of goods.

Leigh Turner of the University of Minnesota Center for Bioethics, meanwhile, says the explosion in clinics offering unproven stem cell therapies in the U.S. is a marketplace that traffics in misrepresentation. It is easy to see how people are taken advantage of and scammed.

It is also difficult to find out about physical harms being done to patients.

There are no safety studies. We dont have good data. But we do know there have been some serious harms.

Stem cell therapies have the potential to become standard treatment in some areas, but they are not there yet, Turner said.

Businesses are tapping into genuine human suffering, desperation and also hope.

Turner also noted there was an excellent chance that the vials of liquid being injected into patients did not actually contain stem cells.

Dr. Jonathan Fenton of another stem cell clinic in Burlington, the Vermont Regenerative Medicine, said he had complained about the new clinic, the Vermont Center for Regenerative Medicine, which has a similar name and employs hard-sell tactics, he said.

His clinic takes bone marrow from patients hips and injects it. The procedure is done the same day. He says he regularly sees Canadian patients for bone marrow aspiration therapy and platelet-rich plasma treatments, using their own blood. The treatments, he says, speed healing and are allowed in the U.S. The use of bone marrow aspiration is neither proven nor allowed in Canada.

Fenton, who is secretary-treasurer of the American Academy of Orthopedic Medicine, acknowledged many people offering stem cell treatments are not doing it to the highest ethical standards.

He has filed complaints with state officials over clinics selling unsafe or fraudulent treatments. I have asked the state and federal judiciary to close down this clinic for committing fraud.

He said his platelet and bone marrow treatments were covered by a major Vermont health insurer because they saw the cost of benefits were going down and patients were requiring fewer surgeries.

He said he was told by Health Canada that he could not market in Canada. Representatives of the Vermont Center for Regenerative Medicine, meanwhile, said they had discussions with Health Canada about what they could and could not say when marketing in Canada before holding seminars in Ottawa and Halifax.

We have looked at the information provided and have not identified any immediate non-compliance with advertising regulations pertaining to Canadian health products, a Health Canada spokesperson said, adding that the agency was continuing to assess.

Back at a west-end Ottawa hotel, some participants in the seminar, including a retired pharmacist, said they were considering getting the treatments. But its expensive.

Another participant said he was skeptical. They seemed very sketchy when I went online.

epayne@postmedia.com

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U.S. stem cell clinic offering unapproved therapies brings direct-to-consumer marketing to Ottawa - Ottawa Citizen

Bone Marrow Stem Cells Comments Off on U.S. stem cell clinic offering unapproved therapies brings direct-to-consumer marketing to Ottawa – Ottawa Citizen | November 18th, 2019

In my view, few investment sectors are as frustrating as the pharmaceutical industry. One moment, you could be riding high on bullish momentum. The next, you could be staring at unfathomable losses. For stakeholders of Constellation Pharmaceuticals (NASDAQ:CNST), though, theyre enjoying the positive end of this dynamic. Year to date, CNST stock is up a blistering 846%.

Source: Shutterstock

Most of these bonkers gains came within the last two months. Since the beginning of October, Constellation Pharmaceuticals stock has jumped nearly 400%. And in this month alone, CNST is up over 68%. Seemingly, this company has no downside, inspiring others to jump aboard this extreme momentum name. Should you follow suit?

Unlike other speculative gambles, a fundamental case exists for the massive skyrocketing of CNST stock. Among the underlying companys therapies is an experimental drug called CPI-0610, a treatment for myelofibrosis. According to pharma giant Celgenes (NASDAQ:CELG) website, myelofibrosis is a rare blood cancer. Only 5,000 people in the U.S. are diagnosed with the illness each year.

Further, myelofibrosis starts in the stem cells of the bone marrow, leading to the production of faulty blood cells. Prior efforts in treating this illness have not produced substantive results. However, Constellations CPI-0610 has performed exceptionally well in a phase II study; hence, the massive surge in Constellation Pharmaceuticals stock.

In fact, all four patients that participated in the study responded positively to the drug. Because of the positive data that came from the clinical trial, Constellation will expand the study to include more patients. This, of course, suggests supreme confidence in the CPI-0610 therapy, and that could ultimately represent a paradigm-shifting breakthrough.

Still, I think you should consider the long road ahead before jumping aboard CNST stock.

By their very nature, rare diseases are difficult to address. And among this class of debilitating conditions, myelofibrosis is particularly nasty. According to Dr. Ruben Mesa, myelofibrosis is a variable disease. This means that medical doctors must apply custom-tailored treatments for different patients.

Thus, while Constellation may have won the initial round in its Phase II study, the real work is coming ahead. With many more test subjects, the chances that CPI-0610 could be considered ineffective or even adverse jump significantly.

In other words, the enthusiasm were seeing now with CNST stock could quickly go the other way.

Theres also the little matter of the economics and politics of addressing myelofibrosis. As you might imagine, combating rare diseases without financial incentives wouldnt make much economic sense. But the Orphan Drug Act, passed in 1983, encouraged pharmaceuticals to address rare diseases through various incentive programs.

Unfortunately, like anything involving government action, good intentions gave way to hellish results. Pharmaceuticals gamed the system the Orphan Drug Act created, pocketing massive profits for rare-disease therapies. Since the patients had no recourse in this monopolized environment, they (and their insurance companies) foot the bill.

Underlining the current bullish thesis for CNST stock is the idea that Constellation will become the only viable myelofibrosis player. Celgene is trying but is coming up short. Essentially, Constellation can charge what they want for their drug if theyre successful.

But even if they are successful and thats a huge if the political environment for price-gouging pharmaceuticals is extremely unfavorable.

No matter how great a scientific achievement Constellation has made, diving into Constellation Pharmaceuticals stock seems risky. With shares gaining 400% in the past month and a half, most of the good news is surely baked in.

Of course, we could hear even better results once the company expands its myelofibrosis study. But that too is a risky perspective.

For those who are not familiar with the pharmaceutical industry, I highly recommend reading Dr. Mario Beauregards book Brain Wars. Among the many topics that Dr. Beauregard covers, a central motif is the mysteries of the mind. Compelling evidence indicates that our mental state can generate healing.

But a flipside to this concept is that an alarming number of pharmaceuticals fail the placebo test; that is, many if not most drugs are no more effective than patients belief in their efficacy.

Soon, well see how good CPI-0610 really is. For those that cant handle extreme price swings, you should stay away from Constellation Pharmaceuticals stock.

As of this writing, Josh Enomoto did not hold a position in any of the aforementioned securities.

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CNST Stock Is Particularly Risky After Its Recent Run - Investorplace.com

Bone Marrow Stem Cells Comments Off on CNST Stock Is Particularly Risky After Its Recent Run – Investorplace.com | November 18th, 2019

Independent data from the PRESTIGIO Registry Study Group in Italy shows leronlimab inhibits multi-drug resistant HIV-1 viruses in Heavily Treatment-Experienced (HTE) patients

VANCOUVER, Washington, Nov. 18, 2019 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that an abstract from the PRESTIGIO Registry Study Group showing that leronlimab was effective at inhibiting multi-drug resistant HIV-1 isolates from HTE patients was accepted for presentation at CROI. The HTE HIV-1-infected patients harbored documented 4-class resistances and were enrolled in the Italian PRESTIGIO Registry Study Group. The authors conclude that, in HTE patients with multi-drug resistance to HIV-1, all CCR5 tropic strains were fully susceptible to PRO 140.

PRESTIGIO is an Italian registry (http://www.registroprestigio.com) involving 40 clinical centers throughout Italy, coordinated by the IRCCS San Raffaele Hospital (Milan). The registry is performing medical research in a study involving HTE HIV-1-infected patients with a documented 4-class drug resistance (multi-drug resistance MDR), including novel treatment approaches.

This completely independent research study on clinical isolates from patients with documented 4-class drug resistances demonstrates that leronlimab may play a key role in patients with very limited therapeutic options, stated CytoDyn President and CEO, Nader Pourhassan, Ph.D. This is further support for our belief that leronlimab is positioned to change the treatment paradigm in HIV therapy, Dr. Pourhassan concluded.

This data, co-authored by Stefano Rusconi, M.D., et al., University of Milan, on behalf of the PRESTIGIO Registry Study Group, will be presented at the March 8-11, 2020 CROI meeting at the Hynes Convention Center, Boston, MA.

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) has granted a "Fast Track" designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with highly active antiretroviral therapy (HAART) for HIV-infected patients, and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including non-alcoholic steatohepatitis (NASH). Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays a vital role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. CytoDyn is conducting additional research with leronlimab in the setting of oncology and NASH with plans to conduct further clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD. Blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted "orphan drug" designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a crucial role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication, which, if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients. Some patients on leronlimab monotherapy have viral suppression for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and has received clearance to initiate a clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties, and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements but, their absence does not mean that a statement is not forward-looking. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSInvestors: Nader Pourhassan, Ph.D.President & CEOnpourhassan@cytodyn.com

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CytoDyn Announces Acceptance of Leronlimab (PRO 140) Data for Presentation at the Conference on Retroviruses and Opportunistic Infections (CROI) in...

Bone Marrow Stem Cells Comments Off on CytoDyn Announces Acceptance of Leronlimab (PRO 140) Data for Presentation at the Conference on Retroviruses and Opportunistic Infections (CROI) in… | November 18th, 2019

Illustration by Adam De Souza

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A few days after my stem cell transplant this year, a young cleaner entered my hospital room to disinfect and swab. Broad faced and friendly, she saw me lying in bed reading a book.

Do you like reading, she asked? Well, I have the book for you. It is called Fifty Shades of Grey. Its porno!

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That last part was whispered behind a cupped hand, as she grinned and then giggled. For good measure, she also recommended the teen vampire series Twilight.

Once shed left I laughed out loud in a way I hadnt done for days, weeks in fact. When you have cancer, these moments are golden.

Over the last year I have spent months in hospitals, being infused with chemotherapy that laid me low and then undergoing a risky transplant of stem cells from a heroic unknown donor. During this long period of remission and recovery, I have valued every opportunity to smile, to breathe and to feel hope. Much of this sense of being fully alive has come from the kindness of others.

The transplant had made me feel very sick and there was a point at which I was terrified of dying. I asked the hospital staff for a spiritual adviser and the next day a Buddhist monk came to visit me. I didnt expect this, but his calm face and compassionate manner brought me peace. He read me poems for meditation, encouraged deep breathing, and assured me that all emotions in illness are human expressions of identity and not to be judged or feared. His gentleness was echoed two days later, when a nurse with the loveliest face I had ever seen knelt down next to my bed, held my hand, and reassured me I would be okay.

Day by day, my son, his girlfriend, and my husband encouraged and supported me, too, even when I could barely hold up my head or speak without tears. My 21-year-old son sat with me through many painful procedures, setting his phone to play Bachs Brandenburg Concertos, squeezing my hand, looking into my face, loving me and giving me strength I didnt think I had.

I was diagnosed with acute myeloid leukemia in February 2019; before that fateful month I was a modern German historian teaching university students on the Weimar Republic, Nazism and the Holocaust. There were days when I had wept and raged with my students over the historical accounts of Nazi inhumanity, barbarity and chilling callousness inflicted upon innocent civilians, especially the Jews. I have often questioned whether human nature is fundamentally selfish, violent and nasty. Right now, in this world of hateful populism and climate devastation, I ask these questions even more. But since I became sick, the kindness, indeed the goodness, of other people has been a constant companion to me. I have been overwhelmed by the extraordinary outpouring of support and concern from so many. Compassion, care, affection, hope all have been expressed to me by family, friends, students and colleagues. Blood drives were organized in my name, and students asked me if they could be tested as a possible bone-marrow donor. My sister (who hates medical procedures) underwent several tests to see if she could be a sibling transplant. One colleague even offered me the umbilical blood he had saved from his three children. (Ultimately the hospital found a donor from an international registry.)

Friends and family kept in touch or visited despite the long drives to the two hospitals where I received treatment. Two of my girlfriends texted me every day, sending love, inspiration and photos of flowers. From other well-wishers I received quilts and artwork and shawls, books and lotion and lip balm. I read notes and e-mails that told me I was not alone, that love surrounded me and would lift me up. Prayers were said for me in Protestant, Catholic, Unitarian, Muslim and Jewish places of worship. Students sent me good luck charms, including a chemo bear (it worked! I went into remission). Money was donated in a go-fund-me campaign to help with the costs of travel and accommodation to cancer centres. Strangers (friends of friends) offered their homes at the times when we couldnt find accommodation. Delicious meals were dropped off at my home or brought to the hospitals: lentil soups, macaroni and cheese, banana bread and smoothies, all preventing me from having to imbibe those horrible meal-replacement drinks or the cafeteria food. Cancer patients came to see me and shared their experiences and wisdom. A quietly stoic man in his 40s with Stage 4 colorectal cancer expressed hope in the advances in cancer treatment; another inspirational friend with breast cancer revealed she had undergone over 100 chemo treatments and still managed to propel her bike in the annual Ride to Conquer Cancer. Other leukemia patients in my wards became friends and sources of enormous support. My sister-in-law, a liver transplant survivor, understood my physical and emotional pain and talked me through several hard times. On the stranger than fiction level, old boyfriends and ex-friends reappeared, expressing their love and sending me cards or messages that brought tears to my eyes. At the same time high-school and university pals from my ancient past got in touch and told me to hang in there!

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I got through the worst days because of the superb doctors and nurses, the donor who gave her or his stem cells, and our excellent health-care system. But I also made it this far because I did not feel alone. I was constantly reminded that I am loved and that I have so much to live for. In the arduous world of my cancer treatment, the face of compassion has appeared so many times and in such beautiful ways that I now place much more faith in the goodness of human nature because I have seen that many of us will care for each other, especially in hard times.

I may not decide to read Fifty Shades of Grey, but I love that this young woman wanted to suggest something to make me forget the cancer and feel better. And, really, because of her and the support that surrounded me, I did.

Carolyn Kay lives in Peterborough, Ont.

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Im grateful for the kindness of strangers in my cancer recovery - The Globe and Mail

Bone Marrow Stem Cells Comments Off on Im grateful for the kindness of strangers in my cancer recovery – The Globe and Mail | November 15th, 2019

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A 25-year-old Redding, Connecticut woman meets the Arizona man who was battling deadly Acute Lymphoblastic Leukemia (ALL) until she saved his life by donating her bone marrow.

Jennie Bunce joined Gift of Life Marrow Registry through a sorority swab drive at North Carolinas High Point University in 2016. "I can remember being like 13 or 14 years old during some school bucket list project. On there was save a life and I got to cross it off so thats pretty cool."

Her life-saving match-- 33-year-old father of six from Mesa, Mark Roser. Roser learned he had ALL after breaking a hip and feeling increasingly weak in 2018.

He needed a bone marrow transplant to survive. He says, "When they discovered it, 94% of my blood cells basically contaminated, so I was really at the final deadline."

Gift of Life Marrow Registry matched the Jennie to Mark with months.

The pair met for the first time at Boca Oyster Bar in Bridgeport in October. Mark says, " I feel great. Im much more positive between work and family. My priorities have completely changed. Time with the kids, time with my wife, just being there for them instead of working so much... I treasure every moment with them now."

According to the gift of Life marrow registry website: "Blood cancer is an umbrella term for cancers that affect the blood, bone marrow and lymphatic system. In most blood cancers, normal blood cell development is interrupted by uncontrolled growth of abnormal blood cells. The abnormal blood cells can prevent blood from fighting off infection or preventing uncontrolled bleeding.

Unfortunately, blood cancer can strike any one of us at any time. Approximately every three minutes, a child or adult in the United States is diagnosed with a type of blood cancer. Thats 360 people a day, 130,000 people a year.

There are three main types of blood cancers: Leukemia, cancer that is found in your blood and bone marrow; Lymphoma, blood cancer that affects the lymphatic system; and Myeloma, blood cancer that specifically targets your plasma cells.

For many, there is hope of a cure through a bone marrow or peripheral blood stem cell transplant. Today, transplantation, of healthy stem cells donated by related and unrelated volunteers, offers hope to many patients suffering from these sometimes deadly diseases.

Advances in transplantation have made this procedure a reality for thousands who are alive today because a stranger gave them the Gift of Life!."

check out: https://www.giftoflife.org to learn more and even register for a swab kit and become a donor yourself.

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Redding woman donates bone marrow, saves life of a father - FOX61 Hartford

Bone Marrow Stem Cells Comments Off on Redding woman donates bone marrow, saves life of a father – FOX61 Hartford | November 15th, 2019

SHANGHAI and SUZHOU, China, Nov. 15, 2019 /PRNewswire/ --Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell therapy company, today announced five presentations to be delivered at the upcoming American Society of Hematology (ASH) Annual Meeting in Orlando, Florida, held from December 7-10.

The presentations centre on Gracell's breakthrough FasTCARtechnology, and other two platform technology in four product categories used in the treatment of hematological malignancies, each with well-defined objectives, including:

The four product candidates are currently being studied in ongoing phase I clinical trials conducted by Gracell, Hebei Yanda Lu Daopei Hospital, and Xinqiao Hospital of AMU, and six other hospitals nationwide in China.

"These clinical studies demonstrated Gracell's product development strategy and strong capabilities to bring multiple novel therapies through clinical investigations," said Dr. William CAO, CEO of Gracell. "These invaluable data provides guidance for and enhance our confidence in pipeline selection."

Oral presentations:

A Feasibility and Safety Study of a New CD19-Directed Fast CAR-T Therapy for Refractory and Relapsed B cell Acute Lymphoblastic LeukemiaAbstract #825Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Therapeutics StrategiesPresenter: Peihua Lu, M.D., Hebei Yanda Lu Daopei HospitalLocation: Orange County Convention Center, Tangerine 1 (WF1), Level 2Time: 5:00 pm, Monday, December 9, 2019https://ash.confex.com/ash/2019/webprogram/Paper121751.html

Anti-CD19/CD22 Dual CAR-T Therapy for Refractory and Relapsed B-Cell Acute Lymphoblastic LeukemiaAbstract #284Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Novel TherapiesPresenter: Peihua Lu, M.D., Hebei Yanda Lu Daopei HospitalLocation: Orange County Convention Center, W224, Level 2Time: 4:15pm, Saturday, December 7, 2019https://ash.confex.com/ash/2019/webprogram/Paper126429.html

Poster presentations:

CD19-Directed Fast CART Therapy for Relapsed/Refractory Acute Lymphoblastic Leukemia: From Bench to BedsideAbstract #1340Session Name: 614. Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster IPresenter: Cheng Zhang, M.D., Xinqiao Hospital of AMULocation: Orange County Convention Center, Hall B, Level 25:30-7:30 pm, Saturday, December 7, 2019https://ash.confex.com/ash/2019/webprogram/Paper128006.html

A Bcma and CD19 Bispecific CAR-T for Relapsed and Refractory Multiple MyelomaAbstract # 3147Session Name: 653. Myeloma: Therapy, excluding Transplantation: Poster IIPresenter: Hua Zhang, PhD., Gracell Biotechnology Ltd., Shanghai, China, Shanghai, ChinaLocation: Orange County Convention Center, Hall B, Level 26:00 PM-8:00 pm, Sunday, December 8, 2019https://ash.confex.com/ash/2019/webprogram/Paper131056.html

Role of Donor-Derived CD19.CAR-T Cells in Treating Patients That Relapsed after Allogeneic Hematopoietic Stem Cell TransplantationAbstract #4561Session Name: 723. Clinical Allogeneic and Autologous Transplantation: Late Complications and Approaches to Disease Recurrence: Poster IIIPresenter: Cheng Zhang, M.D., Xinqiao Hospital of AMULocation: Orange County Convention Center, Hall B, Level 26:00-8:00 pm, Monday, December 9, 2019https://ash.confex.com/ash/2019/webprogram/Paper128262.html

About FasT CAR-19

FasT CAR-19, or GC007F, is an investigational CD19-targeted CAR-T cell therapy for adolescent and adult patients with refractory or relapsed B-ALL, as well as aggressive non-Hodgkin lymphoma. Thanks to Gracell's patented FasTCAR technology, the bioprocessing time for GC007F has been significantly reduced from two weeks to 24 hours with substantially lower cost. The improved CAR-T cell fitness resulted in superior proliferation capabilities, potency, and extensive bone marrow migration making GC007F a potential best-in-class therapy for refractory or relapsed B-ALL.

About Dual CAR-19-22

Dual CAR-19-22, or GC022, is an investigational CAR-T cell therapy redirected to target CD19 and CD22, in treating patients with CD19+, or/and CD22+ relapsed/refractory B-ALL. A low toxicity with dose-dependent high CR rate including patients who previously treated with CD19 CAR-T cells were observed.

About Dual CAR-BCMA-19

Dual CAR-BCMA-19, or GC012, is an investigational CAR-T cell therapy redirected to target BCMA and CD19, in treating patients with BCMA+, or/and CD19+ relapsed/refractory multiple myeloma. Previous research shows CD19 could express on the myeloma progenitor cells, while BCMA is a well validated target for MM.

About Donor CAR-19

Donor CAR-19, or GC007G, is an investigational CD19 targeted CAR-T cell therapy manufactured in use of donor's lymphocytes. The objective of this study is to further investigate and better understand the safety and efficacy of donor derived CAR-T cells in treatment of relapsed and refractory B-ALL patients.

About B-ALL

B-ALL is a sub-type of acute lymphoblastic leukemia, although rare, is one of the most common forms of cancer in children between the ages of two and five and adults over the age of 50[1]. In 2015, ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide[2]. It is also the most common cause of cancer and death from cancer among children. ALL is typically treated initially with chemotherapy aimed at bringing about remission. This is then followed by further chemotherapy carried out over several years.

About MM

Myeloma begins when a plasma cell becomes abnormal. The abnormal cell divides to make copies of itself. These abnormal plasma cells are called myeloma cells. In time, myeloma cells collect in the bone marrow. They may damage the solid part of the bone. When myeloma cells collect in several of your bones, the disease is called "multiple myeloma." This disease may also harm other tissues and organs, such as the kidneys. Myeloma cells make antibodies called M proteins and other proteins. These proteins can collect in the blood, urine, and organs[3].

About Gracell

Gracell Biotechnologies Co., Ltd. ("Gracell") is a clinical-stage biopharma company, committed to developing highly reliable and affordable cell gene therapies for cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T, such as high production costs, lengthy manufacturing process, lack of off-the-shelf products, and inefficacy against solid tumors. Led by a group of world-class scientists, Gracell is advancing FasTCAR, TruUCAR (off-the-shelf CAR), Dual CAR and Enhanced CAR-T cell therapies for leukemia, lymphoma, myeloma, and solid tumors.

CONTACT:

Linc HE Associate Director of Business Developmentsunwei.he@gracellbio.com

Dr. William Cao Founder, Chairman and CEOwilliam.cao@gracellbio.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/gracell-announces-five-presentations-at-the-annual-meeting-of-american-society-of-hematology-ash-300958982.html

SOURCE Gracell

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Gracell Announces Five Presentations at the Annual Meeting of American Society of Hematology (ASH) - BioSpace

Bone Marrow Stem Cells Comments Off on Gracell Announces Five Presentations at the Annual Meeting of American Society of Hematology (ASH) – BioSpace | November 15th, 2019

A 6-year-old cocker spaniel in California that was recently diagnosed with cancer is slated to receive stem cells from her mother living 4,000 miles away in the United Kingdom.

Coco the cocker spaniel gave birth to a litter of puppies six years ago. One of those puppies, Millie, was adopted by Serena and Andrew Lodge, who now live in San Francisco. They may live across the world from each other, but the mother and daughter will soon be reunited for the rare treatment, reported South West News Service, or SWNS, a British news agency.

CHEAPER MEDICATION FOR DOGS WITH SEPARATION ANXIETY NOW APPROVED, FDA SAYS

Coco, left, and daughter, Millie. (SWNS)

The transplant will occur at the North Carolina State Veterinary Hospital in Raleigh. The facility isreportedly one of only a few animal hospitals in the world to offer the treatment, which involves taking healthy stem cells from Cocos bone marrow and injecting them intoMillies.

"Serena and Andrew started chemo on Millie three months ago but they've been told the only chance they'll have of curing her is if they find a positive donor so she can have a transplant, said Cocos owner, Robert Alcock, 52. He arrived with Coco in North Carolina on Wednesday.

Millie while in an animal hospital. (SWNS)

"They contacted us, and we sent some blood samples for testing, along with samples from one of Coco's other pups, he added. They both came back positive but because Coco is Millie's mother the vet said she would be a better match."

"Coco will go into hospital on Sunday for the procedure and then the cells will be donated on Monday, he continued, noting the Lodges have paid for everything.

Robert Alcock and his cocker spaniel, Coco. (SWNS)

BLACK LAB GIVES BIRTH TO 13 PUPPIES, SHOCKS OWNERS: 'THEY WERE JUST FLYING OUT'

Coco is expected to make a full recovery following the procedure. However, there is only a 50 percent chance Millie will be cured even if the treatment is successful, according to SWNS.

Stem cell therapy for pets is costly, typically setting an owner back between $2,000 and $3,000, according to Pet WebMD.

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Cocker spaniel with cancer to receive stem cells from mother living 4,000 miles away - Fox News

Bone Marrow Stem Cells Comments Off on Cocker spaniel with cancer to receive stem cells from mother living 4,000 miles away – Fox News | November 14th, 2019

Bone marrow is soft, spongy tissue within some bones, including those in the hips and thighs. People with certain blood-related conditions benefit from a transplant that replaces damaged cells with healthy cells, possibly from a donor.

Bone marrow transplants can be lifesaving for people with conditions such as lymphoma or leukemia, or when intensive cancer treatment has damaged blood cells.

This type of transplant can be an intensive procedure, and recovery can take a long time.

Here, we provide an overview of bone marrow transplants, including their uses, risks, and recovery.

Bone marrow contains stem cells. In healthy people, stem cells in bone marrow help create:

If a medical condition such as one that damages the blood or immune system prevents the body from creating healthy blood cells, a person may need a bone marrow transplant.

A person with any of the following conditions may be a candidate for a bone marrow transplant:

There are three types of bone marrow transplant, based on where the healthy bone marrow cells come from.

In many cases, the donor is a close family member, such as a sibling or parent. The medical name for this is an allogenic transplant.

Transplants are more likely to be effective if the donated stem cells have a similar genetic makeup to the person's own stem cells.

If a close family member is not available, the doctor will search a registry of donors to find the closest match. While an exact match is best, advances in transplant procedures are making it possible to use donors who are not an exact match.

In a procedure called an autologous transplant, the doctor will take healthy blood stem cells from the person being treated and replace these cells later, after removing any damaged cells in the sample.

In an umbilical cord transplant, also called a cord transplant, doctors use immature stem cells from the umbilical cord following a baby's birth. Unlike cells from an adult donor, the cells from an umbilical cord do not need to be as close a genetic match.

Before a bone marrow transplant, the doctor will run tests to determine the best type of procedure. They will then locate an appropriate donor, if necessary.

If they can use the person's own cells, they will collect the cells in advance and store them safely in a freezer until the transplant.

The person will then undergo other treatment, which may involve chemotherapy, radiation, or a combination of the two.

These procedures typically destroy bone marrow cells as well as cancer cells. Chemotherapy and radiation also suppress the immune system, helping to prevent it from rejecting a bone marrow transplant.

While preparing for the transplant, the person may need to stay in the hospital for 12 weeks. During this time, a healthcare professional will insert a small tube into one of the person's larger veins.

Through the tube, the person will receive medication that destroys any abnormal stem cells and weakens the immune system to prevent it from rejecting the healthy transplanted cells.

Before entering the hospital, it is a good idea to arrange:

A bone marrow transplant is not surgery. It is similar to a blood transfusion.

If a donor is involved, they will provide the stem cells well in advance of the procedure. If the transplant involves the person's own cells, the healthcare facility will keep the cells in storage.

The transplant typically takes place in several sessions over several days. Staggering the introduction of cells in this way gives them the best chance of integrating with the body.

The healthcare team may also use the tube to introduce liquids such as blood, nutrients, and medications to help fight infection or encourage the growth of bone marrow. The combination depends on the body's response to treatment.

The procedure will temporarily compromise the person's immune system, making them very susceptible to infection. Most hospitals have a dedicated, isolated space for people undergoing bone marrow transplants to help reduce their risk of infection.

After the last session, the doctor will continue to check the blood each day to determine how well the transplant has worked. They will test whether new cells are beginning to grow in bone marrow.

If a person's white blood cell count starts to rise, it indicates that the body is starting to create its own blood, indicating that the transplant has been successful.

The amount of time that it takes for the body to recover depends on:

Many other factors can affect recovery, including:

Some people are able to leave the hospital soon after the transplant, while others need to stay for several weeks or months.

The medical team will continue to monitor the person's recovery for up to 1 year. Some people find that effects of the transplant remain for life.

A bone marrow transplant is a major medical procedure. There is a high risk of complications during and after it.

The likelihood of developing complications depends on various factors, including:

Below are some of the more common complications that people who receive bone marrow transplants experience:

Some people die as a result of complications from bone marrow transplants.

A person who receives a bone marrow transplant may also experience reactions that can follow any medical procedure, including:

The body's response to a bone marrow transplant varies greatly from person to person. Factors such as age, overall health, and the reason for the transplant can all affect a person's long term outlook.

If a person receives a bone marrow transplant to treat cancer, their outlook depends, in part, on how far the cancer has spread. Cancer that has spread far from its origin, for example, responds less well to treatment.

According to the National Marrow Donor Program, the 1-year survival rate among people who have received transplants from unrelated donors increased from 42% to 60% over about the past 5 years.

A bone marrow transplant is a major medical procedure that requires preparation. This involves determining the best type of transplant, finding a donor, if necessary, and preparing for a lengthy hospital stay.

The time that it takes for the body to recover from a transplant varies, depending on factors such as a person's age and overall health and the reason for the transplant.

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Bone marrow transplant: What it is, uses, risks, and recovery - Medical News Today

Bone Marrow Stem Cells Comments Off on Bone marrow transplant: What it is, uses, risks, and recovery – Medical News Today | November 14th, 2019

ONE man and his dog are travelling to North Carolina this week, to provide a puppy with a pioneering stem cell transplant that could save her life.

Robert Alcock and his cocker spaniel, Coco, are making the journey so Coco, 7, can donate her stem cells to one of her own puppies, Millie, 6, who has cancer.

The experimental procedure is not yet available in the UK and can only be performed at one US hospital, the NC State Veterinary Hospital.

It involves using stem cells from the bone marrow of one dog and injecting them into the other.

Even if the operation is a success, there is only a 50 percent chance that Millie will be cured.

Millie was taken to the USA when her owners, Serena and Andrew Lodge, emigrated for work. After moving across the pond, Millie contracted cancer.

Millie the dog last week and (inset) before she became ill

Mr Alcock, who lives in Darwen, said the only way to help her is the transplant.

The 52-year-old catering manager said: Serena and Andrew started chemo on Millie three months ago but theyve been told the only chance theyll have of curing her is if they find a positive donor so she can have a transplant.

They contacted us, and we sent some blood samples for testing, along with samples from one of Cocos other pups.

They both came back positive but because Coco is Millies mother the vet said she would be a better match.

Mr and Mrs Lodge then asked Mr Alcock if he would fly to the USA with Coco so she could help save Millies life.

On Wednesday, Mr Alcock made the journey to North Carolina, to the only animal hospital in the States that can perform that kind of transplant on dogs.

Mr Alcock added: The Lodges have paid for everything, and I didnt like to ask how much the operation is costing but I think it will be in the thousands.

We will be in America for about a week.

Coco will go into hospital on Sunday for the procedure and then the cells will be donated on Monday.

Coco is expected to make a full recovery from the operation, but there is only a 50 per cent chance that Millie could be cured once the transplant has been completed.

Robert and Coco

Mr Alcock added: If it was a human then the chances of survival would be really good.

But this is a pioneering procedure, they havent done very many of these transplants before, so well have to wait and see what happens.

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Cocker Spaniel Coco goes to US to try and save her puppy's life - Lancaster and Morecambe Citizen

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NEW YORK, Nov. 14, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today that the Company has received a $495,330 grant from the National Multiple Sclerosis Society, through its Fast Forward program, to advance BrainStorms Phase 2 open-label, multicenter clinical trial of repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive Multiple Sclerosis (NCT03799718).

Chaim Lebovits, President and CEO of BrainStorm stated, We are very pleased to receive this generous grant from the National MS Society. Currently, we are conducting our Phase 2 study in three leading US medical centers: The Keck School of Medicine of USC, The Stanford School of Medicine, and Cleveland Clinic. This research funding will help advance our investigational therapy NurOwn as a potential unmet need for patients with progressive MS. MS continues to devastate the lives of patients and their families and we thank the National MS Society for helping us advance our innovative research program.

Currently, progressive MS treatment options are limited and NurOwn is a promising new autologous cellular treatment modality that has the potential to directly address MS disease pathways, said Ralph Kern MD MHSc, COO and CMO of BrainStorm. He added, This funding from the National MS Society will help us explore key neuroinflammation and neural repair biomarkers in progressive MS to confirm NurOwns unique mechanism of action and guide the design of future clinical trials to address this important unmet patient need.

Leveraging resources in this Phase 2 clinical study of a cell-based therapy for progressive MS exemplifies our work to accelerate research to improve clinical care for people living with MS. said Mark Allegretta, PhD, Vice President of Research at the National MS Society. Were pleased to work with BrainStorm to test a broad panel of biomarkers of neuroinflammation and repair as correlates of the effect of treatment with NurOwn.

About Multiple SclerosisMultiple sclerosis is an unpredictable, often disabling disease of the central nervous system. There is currently no cure for MS. Symptoms vary from person to person and range from numbness and tingling, to mobility challenges, blindness and paralysis. An estimated 1 million people live with MS in the United States. Most people are diagnosed between the ages of 20 and 50 and it affects women three times more than men.

About The National Multiple Sclerosis Society:The National MS Society, founded in 1946, funds cutting-edge research, drives change through advocacy, and provides programs and services to help people affected by MS live their best lives. Connect to learn more and get involved: nationalMSsociety.org, Facebook, Twitter, Instagram, YouTube or 1-800-344-4867.

About NurOwnNurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic 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. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

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 NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com.

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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.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

Link:
BrainStorm Cell Therapeutics Announces Research Grant Award From the National Multiple Sclerosis Society - Yahoo Finance

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics Announces Research Grant Award From the National Multiple Sclerosis Society – Yahoo Finance | November 14th, 2019