First ever progressive MS treatment approved for use by NHS in Scotland – The Scotsman

A leading health charity has welcomed the watershed decision to approve the first ever drug used in the treatment of progressive multiple sclerosis (MS) in Scotland.

Ocrelizumab (Ocrevus) has been given the green light by the Scottish Medicines Consortium (SMC) for treating primary progressive MS where the condition is still early in terms of duration and disability and shows evidence of inflammation.

It becomes the first drug to be made available on the NHS in Scotland for primary progressive MS. There are currently 13 treatments available for people with the relapsing form of the condition.

READ MORE: https://www.scotsman.com/health/groundbreaking-stem-cell-therapy-for-multiple-sclerosis-recommended-for-use-in-scotland-1-5035721This news follows an SMC decision to make ocrelizumab available on NHS Scotland for people with relapsing MS in December 2018 as well as the approval, in May 2019, for its use for people with primary progressive MS in England.

More than 11,000 people in Scotland have MS and around 65 each year are diagnosed with the primary progressive form. Ocrelizumab is the first and only treatment that can slow disability progression in this type of MS, where symptoms gradually worsen over time. It is licensed for early primary progressive MS, which is defined by how long someone has lived with MS symptoms, their level of disability, and MRI scans showing inflammatory activity.

Karine Mather was diagnosed with primary progressive MS six years ago and she, and her wife Sarah, have seen their lives impacted hugely since then. They reflected on what this decision will mean for people in a similar situation in the future.

She said: This is great news for the MS community as people diagnosed with early primary progressive MS in Scotland will now be able to access a treatment for the first time.

Primary Progressive MS has had a massive impact on my life and on the lives of my wife and family.

In the space of five years I went from walking with a slight limp and working full-time to using a powerchair, unable to work and needing round the clock care from my wife who gave up her full-time job.

This medication will slow the progression of MS offering people newly diagnosed a treatment, enabling them to continue working and living a full life."

Sarah said: Since Karine was diagnosed with primary progressive MS we have gone from a working household with two full time incomes to her being unable to work and myself only able to work 10 hours per week due to my caring responsibilities.

Access to ocrelizumab could help people continue to live their lives and help slow progression of the disabilities associated with MS so its wonderful news that others could now benefit from this treatment.

READ MORE: https://www.scotsman.com/health/drugs-trial-offers-new-hope-for-edinburgh-ms-sufferer-1-4855730MS Society Scotland hailed the landmark news and the positive effect it could have for people across the country.

Morna Simpkins, Director for MS Society Scotland said: This is great news and a hugely important development for people diagnosed with primary progressive MS in Scotland.

We want every one of the 11,000 people in Scotland living with MS to have access to the right treatment at the right time and this decision takes us closer than ever to that goal.

Right now, however, there isnt enough evidence to show ocrelizumab can work for everyone, and we know the limited scope of this announcement will be disappointing for those who still dont have any options. Were driving research to find more and better treatments, and calling for drug trials to more fully address the needs of everyone with MS, until the day we are able to stop it in its tracks.

Research has got us to a critical point, and we can see a future where nobody needs to worry about MS getting worse. Our Stop MS appeal is aiming to raise 100 Million over 10 years to make that a reality and build on the treatments, like ocrelizumab, that are available.

Ocrelizumab works by attaching itself to a protein (called CD20) on the surface of a type of white blood cell (called B cells) that attacks the protective coating around the nerves causing inflammation and damage to the nerve fibres. As a result ocrelizumab can block their activity and reduce the symptoms of MS.

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First ever progressive MS treatment approved for use by NHS in Scotland - The Scotsman

Editas Medicine and Sandhill Therapeutics, Inc. Announce Collaboration to Develop Engineered Cell Medicines to Treat Cancer | More News | News…

DetailsCategory: More NewsPublished on Monday, 13 January 2020 17:41Hits: 228

CAMBRIDGE, MA and DALLAS, TX, USA I January 13, 2020 I Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, and Sandhill Therapeutics, Inc., a cellular immuno-oncology company, announced a strategic research collaboration, license, and option agreement to combine their respective genome editing and cell therapy technologies to discover, develop, and manufacture allogeneic engineered natural killer (NK) cells and non-alpha beta T cell medicines for the treatment of cancer.

This collaboration brings together Editas Medicines leading genome editing technology and Sandhills BINATE product process, a novel universal donor technology to extract, isolate, and expand NK cells and non-alpha beta T cells, to develop novel medicines for the treatment of solid tumor cancers.

We are excited to work with Sandhill, combining CRISPR-based genome editing with BINATE cells to accelerate the development of numerous, transformative medicines for people with cancer and improve patient outcomes, said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. We continue to increase our commitment to oncology, and we believe our portfolio of multiple immune system cell types, including T cells, NK cells, and induced pluripotent stem cells (iPSCs), will be effective in making the next generation of allogeneic medicines to fight many common cancers.

The team at Editas Medicine has one of the most innovative technology platforms, and we look forward to combining our technologies to create new medicines for the treatment of cancer. Together, we are dedicated to transforming cellular immuno-oncology and developing new therapies, said Annemarie Moseley, M.D., Ph.D., Chief Executive Officer, Sandhill Therapeutics, Inc.

Under the terms of the agreement, Editas Medicine obtains an exclusive license to Sandhills technology to research, develop and commercialize immuno-oncology engineered cell medicines for solid tumors originating within a given area of the body and an option to expand such license to two additional areas. In return, Sandhill will receive an upfront payment, development and sales-based milestone payments, and royalties on sales of resulting Editas products.

RBC Capital Markets acted as exclusive financial advisor to Sandhill for the transaction.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About Sandhill Therapeutics, Inc.Sandhill Therapeutics is a privately held, development stage cellular immunotherapy company dedicated to improving the lives of children and adults with cancer. Sandhills BINATE leverages dual innate cell synergy, resulting in a highly activated, readily available, universal off-the-shelf treatment for both solid tumors and blood cancers. Sandhills activated innate cell immunotherapy is generated by a cost-effective, feeder-free campaign manufacturing process. For more information, visitwww.sandhilltx.com.

SOURCE: Editas Medicine

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Editas Medicine and Sandhill Therapeutics, Inc. Announce Collaboration to Develop Engineered Cell Medicines to Treat Cancer | More News | News...

Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers – Yahoo…

NEW YORK, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, today announced preliminary net revenues for 2019, as well as outlined key BPDCN market successes and upcoming commercial and clinical milestones.

Unaudited preliminary 2019 results include:

The above financial information is based on preliminary unaudited information, is subject to adjustment, and does not present all information necessary for an understanding of the Companys full-year and fourth quarter financial results for 2019. Stemline expects to report complete audited 2019 financial results on or before March 16, 2020.

Robert Francomano, Chief Commercial Officer of Stemline, stated, We are very pleased with the solid uptake seen in the first year of the ELZONRIS launch, as we continue to successfully create, penetrate and grow a new market in BPDCN. Given the orphan nature and unique features of this disease, we believe patient starts were subject to significant quarterly variance a phenomena that will likely continue throughout 2020. We are actively implementing a host of tactics to expand and further penetrate this emerging market.

Ivan Bergstein, CEO of Stemline, commented, 2019 was a transformational year for Stemline as we launched ELZONRIS, the first and only CD123 targeted agent and first agent ever approved for patients with BPDCN. We continue to pursue growth opportunities not only in BPDCN but also in a number of malignancies where targeting CD123 could provide therapeutic benefit. We look forward to data readouts in CMML, MF, and AML, including in patient subsets with high CD123, later this year and on into next year. Given our continued commercial and clinical progress, we look forward to a productive 2020 and beyond.

Corporate Highlights and Key Commercial and Clinical Milestones

BPDCN

Chronic Myelomonocytic Leukemia (CMML)

Myelofibrosis (MF)

Acute Myeloid Leukemia (AML)

About ELZONRISELZONRIS(tagraxofusp), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit http://www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at http://www.bpdcninfo.com.

About CD123CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkins lymphoma (HL), and certain Non-Hodgkins lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

About Stemline Therapeutics Stemline Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics. ELZONRIS(tagraxofusp), a targeted therapy directed to CD123, is FDA-approved and commercially available in the U.S. for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML). Additional pipeline candidates include: felezonexor (SL-801) (XPO1 inhibitor; Phase 1 in advanced solid tumor patients ongoing), SL-1001 (novel RET kinase inhibitor, IND-enabling studies ongoing), SL-701 (immunotherapeutic; Phase 2 in glioblastoma patients completed), and SL-901 (novel kinase inhibitor; prior abbreviated European Phase 1, IND-enabling studies ongoing). For more information, please visit the companys website at http://www.stemline.com.

Story continues

Forward-Looking StatementsSome of the statements included in this press release may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The factors that could cause our actual results to differ materially include: the risk that our actual revenue for the fourth quarter and year endedDecember 31, 2019may differ materially from our estimated results for these periods as a result of the completion of year-end closing procedures or the audit of our financial statements; the success of our U.S. launch and commercialization; the success of our MAA submission to the EMA and potential launch in Europe; the success and timing of our clinical trials and preclinical studies for our product and product candidates, including ELZONRIS in additional indications and our other pipeline candidates, including site initiation, institutional review board approval, scientific review committee approval, patient accrual, safety, tolerability and efficacy data observed, and input from regulatory authorities including the risk that the FDA, EMA, or other ex-U.S. national drug authority ultimately does not agree with our data, find our data supportive of approval, or approve any of our product candidates; the possibility that results of clinical trials are not predictive of safety and efficacy results of our product candidates in broader patient populations or of our products if approved; our plans to develop and commercialize our product candidates, including, but not limited to delays in arranging satisfactory manufacturing capabilities and establishing commercial infrastructure for ELZONRIS; product efficacy or safety concerns resulting in product recalls or regulatory action; the risk that estimates regarding the number of patients with the diseases that our product and product candidates may treat are inaccurate; inadequate market penetration of our products; our products not gaining acceptance among patients (and providers or third party payors) for certain indications (due to cost or otherwise); the risk that third party payors (including governmental agencies) will not reimburse for the use of ELZONRIS at acceptable rates or at all; the companys ability to produce, maintain or increase sales of ELZONRIS; the companys ability to develop and/or commercialize ELZONRIS; the adequacy of our pharmacovigilance and drug safety reporting processes; our available cash and investments; our ability to obtain and maintain intellectual property protection for our product and product candidates; delays, interruptions, or failures in the manufacture and supply of our product and product candidates; the performance of third-party businesses, including, but not limited to, manufacturers, clinical research organizations, clinical trial sponsors and clinical trial investigators; and other risk factors identified from time to time in our reports filed with the SEC. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not intend to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

Contact: Investor RelationsStemline Therapeutics, Inc.750 Lexington AvenueEleventh FloorNew York, NY 10022Tel: 646-502-2307Email: investorrelations@stemline.com

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Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers - Yahoo...

The potential use of CRISPR to treat disease is gaining momentum – BioNews

13 January 2020

Promising results from clinical trials give hope for using CRISPR/Cas9 genome editing to treat various heritable diseases and cancer in humans.

It has been seven years since the discovery that the CRISPR/Cas9 defence system, used by microbes to destroy viruses, could be re-engineered to edit the human genome. Since then researchers have carried out an array of experiments to explore potential applications.

Biophysist Dr He Jiankui sparked global controversy concerning the ethics of genome editing when he used CRISPR to genetically modify embryos, resulting in the birth of the first genome-edited babies (see BioNews 977).

Yet researchers worldwide have at the same time been investigating the use of CRISPR for non-heritable changes, modifying the genes in non-embryonic cells to treat a wide range of diseases.

'There's been a lot of appropriate caution in applying this to treating people, but I think we're starting to see some of the results of that work,' said Dr Edward Stadtmauer, a haematologist at the University of Pennsylvania, Philadelphia.

Over a dozen new clinical trials testing CRISPRtherapy on diseases such as cancer, HIV and sickle cell anaemia were listed on the clinicaltrials.gov database last year. One trial in its early stages used CRISPR to treat sickle cell anaemia and beta-thalassaemia, both genetic blood disorders that result in the production of an abnormal form of the oxygen-carrying protein, haemoglobin.

Two patients with these disorders were treated by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, using CRISPR to inactivate a gene that switches off the production of an alternative form of haemoglobin. Preliminary results of the study suggest that this therapy improved some of the symptoms but the participants will need to be followed for a longer period to be sure.

Results from two other trials, one in which genome-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancer, were less successful. In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University, Beijing, China and a lead author of the work.

Other researchers are trying to move beyond editing cells in vitro. In July 2019 a clinical trial was launched to treat Leber congenital amaurosis 10 (LCA10), a rare genetic disease that causes blindness. The trial, launched by two pharmaceutical companies, Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin, Ireland, will be the first trial that uses CRISPR to edit cells inside of the body. The researchers are testing AGN-151587 (EDIT-101), which is a novel CRISPR treatment delivered via adeno-associated virus (AAV) directly to the eye's light-sensing photoreceptor cells to remove the mutation that causes LCA10.

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The potential use of CRISPR to treat disease is gaining momentum - BioNews

At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease – The New York Times

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

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At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease - The New York Times

At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens – Boston.com

BOSTON Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.

The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.

It was a critical moment in medical science.

For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the diseases horrifying hallmark: episodes of agony that begin in babyhood.

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts said the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African American, often poor or of modest means.

The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.

This is the story of two quests for a sickle cell cure one by the Obando family and one by a determined scientist at Boston Childrens Hospital, Dr. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s.

Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee Obando. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood.

Scientists have been experimenting with gene therapy for two decades, with mixed success. And it will be years before they know if this new procedure is effective in the long term. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.

Four weeks after the infusion of stem cells, Helen was strong enough to be discharged. At home, in Lawrence, Massachusetts, on a sofa with her mother by her side, she put a hand over her eyes and started to sob. She and her family wondered: Would it work? Was her suffering really over?

A Familys Nightmare

Sheila Cintron, 35, and Byron Obando, 40, met when she was in the eighth grade and he was a high school senior. They fell in love. Haylee, their first child, was born in 2001, when Cintron was 17.

When a newborn screening test showed that Haylee had the disease, her father asked, Whats sickle cell?

They soon found out.

As the family gathered for her first birthday party, Haylee started screaming inconsolably. They rushed her to the hospital. It was the first of many pain crises.

Doctors warned the parents that if they had another baby, the odds were 1 in 4 that the child would have sickle cell, too. But they decided to take the chance.

Less than two years later, Helen was born. As bad as Haylees disease was, Helens was much worse. When she was 9 months old, a severe blockage of blood flow in her pelvis destroyed bone. At age 2, her spleen, which helps fight bacterial infections, became dangerously enlarged because of blocked blood flow. Doctors surgically removed the organ.

After Helen was born, her parents decided not to have any more children. But four years later, Cintron discovered she was pregnant again.

But they were lucky. Their third child, Ryan Obando, did not inherit the sickle cell mutation.

As Ryan grew up, Helens health worsened. When he was 9, Helens doctors suggested a drastic solution: If Ryan was a match for her, he might be able to cure her by giving her some of his bone marrow, though there would also be major risks for her, including death from severe infections or serious damage to organs if his immune system attacked her body.

As it turned out, Ryan matched not Helen but Haylee.

The transplant succeeded, but her parents asked themselves how they could stand by while one daughter was cured and the sicker one continued to suffer.

There was only one way to get a sibling donor for Helen: have another baby. In 2017, the couple embarked on another grueling medical journey.

Obando had a vasectomy, so doctors had to surgically extract his sperm from his testicles. Cintron had 75 eggs removed from her ovaries and fertilized with her husbands sperm. The result was more than 30 embryos.

Not a single embryo was both free of the sickle cell gene and a match for Helen.

So the family decided to move to Mesa, Arizona, from Lawrence, where the cold, which set off pain crises, kept Helen indoors all winter. The family had already sold their house when they heard that doctors at Boston Childrens were working on sickle cell gene therapy.

Cintron approached Dr. Erica Esrick, a principal investigator for the trial. But the trial wasnt yet open to children.

Figuring Out the Science

Nothing had prepared Orkin for the suffering he witnessed in his 30s as a medical resident in the pediatric hematology ward at Boston Childrens. It was the 1970s, and the beds were filled with children who had sickle cell crying in pain.

Orkin knew there was a solution to the puzzle of sickle cell, at least in theory: Fetuses make hemoglobin the oxygen-carrying molecules in blood cells with a different gene. Blood cells filled with fetal hemoglobin do not sickle. But the fetal gene is turned off after a baby is born, and an adult hemoglobin gene takes over. If the adult gene is mutated, red cells sickle.

Researchers had to figure out how to switch hemoglobin production to the fetal form. No one knew how to do that.

Orkin needed ideas. Supported by the National Institutes of Health and Howard Hughes Medical Institute, he kept looking.

The breakthrough came in 2008. The cost of gene sequencing was plummeting, and scientists were finding millions of genetic signposts on human DNA, allowing them to home in on small genetic differences among individuals. Researchers started doing large-scale DNA scans of populations, looking for tiny but significant changes in genes. They asked: Was there a molecular switch that flipped cells from making fetal to adult hemoglobin? And if there was, could the switch be flipped back?

They found a promising lead: an unprepossessing gene called BCL11A.

In a lab experiment, researchers blocked this gene and discovered that the blood cells in petri dishes started making fetal instead of adult hemoglobin.

Next they tried blocking the gene in mice genetically engineered to have human hemoglobin and sickle cell disease. Again, it worked.

Patients came next, in the gene therapy trial at Boston Childrens that began in 2018.

The trial run by Dr. David Williams, an expert in the biology of blood-forming stem cells at Boston Childrens, and Esrick has a straightforward goal: Were going to reeducate the blood cells and make them think they are still in the fetus, Williams said.

Doctors gave adult patients a drug that loosened stem cells immature cells that can turn into red blood cells from the bone marrow, their normal home, so they floated free in the bloodstream. Then they extracted those stem cells from whole blood drawn from the patient.

The researchers used a disabled genetically engineered AIDS virus to carry information into the stem cells, flipping on the fetal hemoglobin gene and turning off the adult gene. Then they infused the treated stem cells into patients veins. From there, the treated cells migrated into the patients bone marrow, where they began making healthy blood cells.

With the success in adults, the Food and Drug Administration said Boston Childrens could move on to teenagers.

When her mother told her about the gene therapy trial, Helen was frightened. But the more she thought about it, the more she was ready to take the risk.

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared.

Helen was scheduled for her six-month checkup Dec. 16. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved, even with blood transfusions. She had no signs of sickle cell disease.

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At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens - Boston.com