Dragon splashes down in Pacific with time-critical experiments – SpaceFlight Insider
By daniellenierenberg
Derek Richardson
July 3rd, 2017
The CRS-11 Dragon capsule re-enters Earths atmosphere. Photo Credit: Jack Fischer / NASA
SpaceXs CRS-11 Dragon capsule splashed down at 8:12 a.m. EDT (12:12 GMT) on July 3, 2017, in the Pacific Ocean just off the coast of Baja California after some 28 days attached to the International Space Station.
After being unberthed using the robotic Canadarm2, the craft was moved to a location some 33 feet (10 meters) below the Destiny laboratory module. It was officially released at 2:41 a.m. EDT (6:41 GMT) on July 3 by Expedition 52 astronauts Jack Fischer and Peggy Whitson of NASA.
The CRS-11 Dragon capsule is positioned for release beneath the ISS. Photo Credit: Jack Fischer / NASA
Dragons been an incredible spacecraft, Fischer said after release. I could even say it was slathered in awesome sauce. This baby has had almost no problems, which is an incredible feat considering its the first reuse of a Dragon vehicle.
The CRS-11 Dragon capsule pressure vessel was the same one used during the CRS-4 mission in 2014.
And the science weve done oh my, the science, Fischer said. Most of the 6,000 pounds [2,700 kilograms] of cargo carried was science, and almost all of the return cargo are precious samples for discoveries we cant wait to see.
Fischer explained that Dragon also brought up various external experiments too, including an external platform for science, a neutron star analyzer and an experimental solar array that was rolled out like a party horn on New Years Eve.
The science on this mission has been non-stop, and we think the scientists will be extremely happy with the volumes of data we gathered for them up here in space in our floating world-class laboratory we call home, Fischer said. For the whole SpaceX team, thank you for building such a great vehicle and for finding us some good weather today to allow us to bring home the science on time. Godspeed and fair winds, Dragon-11.
The spacecraft had originally been planned to splash down on July 2, but due to a forecast of unacceptable sea conditions at the recovery zone, mission managers decided on June 30 to postpone the capsules departure from the station.
Three separate departure burns were performed by the Dragon capsule once the robotic arm released the spacecraft. This gradually pushed the vehicle away from the outpost and outside the 656-foot (200-meter) Keep-Out Sphere (KOS).
Some five hours later, Dragon, using its Draco thrusters, performed a 10-minute de-orbit burn. Minutes after that, its trunk, which is not recoverable, was jettisoned.
Moments after being released by the ISS crew, the CRS-11 Dragon capsule begins its journey back to Earth. Photo Credit: Jack Fischer / NASA
A few minutes before splashing down, the capsule released drogue chutes to slow the capsule a bit and to keep a specific attitude for the three main parachutes to bedeployed. Once that occurred, along with a successful splashdown, it ensured a successful mission for the first re-flight of a commercial spacecraft to and from the ISS.
Now that Dragon is back on Earth and on a recovery ship, it will now be transported to the port of Los Angeles to offload time-sensitive cargo. The most notable include the Fruit Fly Lab-02 experiment, the Systemic Therapy of NELL-1 for osteoporosis study, and the Cardiac Stem Cells experiment.
The Fruit Fly Lab-02 experiment aims to understand the effects of prolonged microgravity exposure on the heart. According to NASA, because flies are small, have a well-known genetic makeup, and age rapidly, thatmakes them good models for heart function studies.
For the Systemic Therapy of NELL-1 for osteoporosis study, a group of rodents were used as models to test a drug that can rebuild bone and block additional bone density loss. It is hoped that this can help reduce bone density loss for astronauts on extended stays in space. Additionally, it can potentially help people with osteoporosis.
According to NASA, in-flight countermeasures, like exercise, can prevent bone density loss from getting worse, but nothing on Earth or in space can restore bone density.
Finally, the Cardiac Stem Cells experiment aims to analyze how microgravity affects stem cells and factors that govern stem cell activity. NASA says the study focuses on cardiac stem cell functions and has numerous biomedical and commercial applications.
The CRS-11 Dragon was launched June 3 from Kennedy Space Centers Launch Complex 39A in Florida. After a two-day rendezvous profile, the capsule was berthed to the Earth-facing port of the Harmony module on June 5.
The next Dragon mission will be CRS-12 on Aug. 10, 2017. It is unclear if this capsule will also be a pre-flown vessel.
Video courtesy of NASA
Tagged: CRS-11 Dragon Expedition 52 International Space Station Lead Stories NASA SpaceX
Derek Richardson has a degree in mass media, with an emphasis in contemporary journalism, from Washburn University in Topeka, Kansas. While at Washburn, he was the managing editor of the student run newspaper, the Washburn Review. He also has a blog about the International Space Station, called Orbital Velocity. He met with members of the SpaceFlight Insider team during the flight of a United Launch Alliance Atlas V 551 rocket with the MUOS-4 satellite. Richardson joined our team shortly thereafter. His passion for space ignited when he watched Space Shuttle Discovery launch into space Oct. 29, 1998. Today, this fervor has accelerated toward orbit and shows no signs of slowing down. After dabbling in math and engineering courses in college, he soon realized his true calling was communicating to others about space. Since joining SpaceFlight Insider in 2015, Richardson has worked to increase the quality of our content, eventually becoming our managing editor.
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July 8th, 2017
Artsakh soldier saves life of cancer patient in Iran – Public Radio of Armenia
By daniellenierenberg
Asbarez The Armenian Bone Marrow Donor Registry (ABMDR) announced that it has facilitated its 30th bone marrow stem cell transplant, thanks to stem cells harvested from a young ABMDR matched donor. The stem cells of the donor, Sergey, who is a 23 year-old army officer serving on the frontline in Artsakh, were utilized to save the life of a cancer patient in Iran.
On July 3, 2017, Sergey became the 30th ABMDR donor to experience the joy of saving the life of someone he had never met, said ABMDR President Dr. Frieda Jordan.
In 2012, Sergey had joined the ranks of ABMDRs donor registry during a recruitment drive at the Vazken Sagsyan Military Institute, in Yerevan. Five years later, he was found to be a perfect match for a patient in Iran who was suffering from leukemia and whose only hope for survival was to receive a bone marrow stem cell transplant from a compatible donor. Sergey turned out to be a perfect match for the patient. He was given a day off to leave the frontline to come to ABMDRs Stem Cell Harvesting Center in Yerevan to donate his stem cells and save a patients life.
Accompanied by his young wife and six-month old son, Sergey was greeted by ABMDR staff at the Stem Cell Harvesting Center. The painless, non-invasive harvesting procedure, performed by Dr. Andranik Mshetsyan, lasted approximately four hours. Also present at the procedure were ABMDR Executive Director Dr. Sevak Avagyan and Medical Director Dr. Mihran Nazaretyan.
At the conclusion of the harvesting, as staff members performed quality-control analyses of the harvested cells and packed them for the special courier who was waiting to transport the precious gift of life to the patient in Iran, Sergey, a hero in the eyes of all, on the frontlines as well as far away from them, joined his young family while someone in Iran was about to get a second chance at life.
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July 7th, 2017
Dragon Splashes Down to Complete Resupply Mission – Space Daily
By daniellenierenberg
SpaceX's Dragon cargo craft splashed down in the Pacific Ocean at 8:12 a.m. EDT, west of Baja California and the recovery process is underway, marking the end of the company's eleventh contracted cargo resupply mission to the International Space Station for NASA.
Expedition 52 astronauts Jack Fischer and Peggy Whitson of NASA released the SpaceX Dragon cargo spacecraft from the International Space Station's robotic arm right on schedule, at 2:41 a.m.
A variety of technological and biological studies are returning in Dragon. The Fruit Fly Lab-02 experiment seeks to better understand the effects of prolonged exposure to microgravity on the heart.
Flies are small, with a well-known genetic make-up, and age rapidly, making them good models for heart function studies. This experiment could significantly advance understanding of how spaceflight affects the cardiovascular system and could help develop countermeasures to help astronauts.
Samples from the Systemic Therapy of NELL-1 for osteoporosis will return as part of an investigation using rodents as models to test a new drug that can both rebuild bone and block further bone loss, improving crew health.
When people and animals spend extended periods of time in space, they experience bone density loss, or osteoporosis. In-flight countermeasures, such as exercise, prevent it from getting worse, but there isn't a therapy on Earth or in space that can restore bone density.
The results from this ISS National Laboratory-sponsored investigation is built on previous research also supported by the National Institutes for Health and could lead to new drugs for treating bone density loss in millions of people on Earth.
The Cardiac Stem Cells experiment investigated how microgravity affects stem cells and the factors that govern stem cell activity. The study focuses on understanding cardiac stem cell function, which has numerous biomedical and commercial applications. Scientists will also look to apply new knowledge to the design of new stem cell therapies to treat heart disease on Earth.
The Dragon spacecraft launched June 3 on a SpaceX Falcon 9 rocket from historic Launch Complex 39A at NASA's Kennedy Space Center in Florida, and arrived at the station June 5.
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July 7th, 2017
Stem cell therapies breaking barriers – Guardian (blog)
By daniellenierenberg
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July 6th, 2017
Canadian clinics begin offering stem-cell treatments experts call unproven, possibly unsafe – National Post
By daniellenierenberg
The arthritis in Maureen Munsies ankles was so intense until barely a year ago, she literally had to crawl on hands and knees to get upstairs.
The pain, she recalls now, took my breath away, and played havoc with the avid hikers favourite pastime.
In desperation, Munsie turned to a Toronto-area clinic that provides a treatment many experts consider still experimental, unproven and of questionable safety.
The 63-year-old says the stem cells she received at Regenervate Medical Injection Therapy 18 months ago were transformational, all but eliminating the debilitating soreness and even allowing her to hike Argentinas Patagonia mountains two months ago.
For me its been a life saver, Munsie says. Ive been able to do it all again I dont have any of that pain, at all.
Canadians drawn to the healing promise of stem cells have for years travelled outside the country to such places as Mexico, China or Arizona, taking part in a dubious form of medical tourism.
But Regenervate is one of a handful of clinics in Canada that have begun offering injections of stem cells, satisfying growing demand but raising questions about whether a medical idea with huge potential is ready for routine patient care.
Especially when those patients can pay thousands of dollars for the service.
Clinics in Ontario and Alberta are treating arthritis, joint injuries, disc problems and even skin conditions with stem cells typically taken from patients fat tissue or bone marrow.
The underlying idea is compelling: stem cells can differentiate or transform into many other types of cell, a unique quality that evidence suggests allows them to grow or regenerate tissue damaged by disease or injury.
Researchers including hundreds in Canada alone are examining stem-cell treatments for everything from ailing hearts to severed spinal cords.
With few exceptions, however, the concept is still being studied in the lab or in human trials; virtually none of the treatments have been definitively proven effective by science or approved by regulators like Health Canada.
The fact that Canadian clinics are now offering stem-cell treatments commercially is concerning on a number of levels, not least because of safety issues, says Ubaka Ogbogu, a health law professor at the University of Alberta.
Three U.S. women were blinded after receiving stem-cell injections in their eyes, while other American patients have developed bony masses or tumours at injection sites, Ogbogu said.
Stem cells have to be controlled to act exactly the way you want them to act, and thats why the research takes time, he said. It is simply wrong for these clinics to take a proof of concept and run with it.
Ogbogu says Health Canada must crack down on the burgeoning industry but says the regulator has so far been conspicuous by its inaction.
Other experts say the procedures provided here typically for joint pain are likely relatively safe, but still warn that care must be taken that the stem cells do not develop into the wrong type of tissue, or at the wrong place.
Alberta Health Services convened a workshop on the issue late last year, concluding there is an urgent need to develop a certification system for cell preparation and delivery to avoid spontaneous transformation of (stem cells) into unwanted tissue.
But one of the pioneers of the service in Canada says theres no empirical evidence that such growths can develop, and suggests the treatments only real risk as with an invasive procedure is infection.
Meanwhile, patients at Regenervate have enjoyed impressive outcomes after paying fees from $750 to $3,900, says Dr. Douglas Stoddard, the clinics medical director.
About 80 per cent report less pain, stiffness and weakness within a few months of getting their stem-cell injection, he said.
I believe medical progress is not just limited to the laboratory and randomized double-blind trials, Stoddard said. A lot of progress starts in the clinic, dealing with patients You see something works, you see something has merit, and then its usually the scientists that seem to catch up later.
The Orthopedic Sport Institute in Collingwood, Ont., the Central Alberta Pain and Rehabilitation Institute and Cleveland Clinic in Toronto all advertise similar stem-cell treatments for orthopedic problems.
Edmontons Regen Clinic says it plans to start doing so this fall.
Ottawas Innovo says it also treats a range of back conditions with injections between the vertebrae, and uses stem cells to alleviate nerve damage.
Orthopedic Sport says its doctor focuses on FDA and Health Canada approved stem-cell injection therapy for patient care.
In fact, no treatment of the sort the clinics here provide has ever been authorized.
Health Canada says the vast majority of stem-cell therapies would constitute a drug and therefore need to be authorized after a clinical trial or new drug submission.
A number of stem-cell trials are underway, but only one treatment Prochymal has been approved, said department spokesman Eric Morrissette. Designed to combat graft-versus-host disease where bone marrow transplants for treating cancer essentially attack the patients body its unlike any of the services the stem-cell providers here offer.
But as the U.S. Food and Drug Administration aggressively pursues the hundreds of clinics in America, Health Canada says only that its committed to addressing complaints it receives.
It will take action based on the risk posed to the general public, said Morrissette, who encouraged people to pass on to the department information about possible non-compliant products.
Stoddard said the injections his clinics provide are made up of minimally manipulated tissue from patients own bodies and any attempt to crack down would be regulation for the sake of regulation.
But academic experts remain skeptical about the effectiveness of the treatments.
Scientific evidence suggests the injections may help alleviate joint pain temporarily, but probably just because of anti-inflammatory secretions from the cells not regeneration, said Dr. David Hart, an orthopedic surgery professor at the University of Calgary who headed the Alberta workshop.
Theres a need for understanding whats going on here and theres a need for regulation, he said.
Most of the clinics say they use a centrifuge to concentrate the stem cells after removing them from patients fat tissue or bone marrow. But its unclear if the clinics even know how many cells they are eventually injecting into patients, says Jeff Biernaskie, a stem-cell scientist at the University of Calgary.
Munsie, on the other hand, has no doubts about the value of her own treatment, even with a $3,000 price tag.
The procedure from extraction of fat tissue in her behind to the injection of cells into her ankles took barely over an hour.
Within three months, the retired massage therapist from north of Toronto says she could walk her dogs again. Last week, she was hiking near Banff.
Im a real believer in it, and the possibility of stem cells, says Munsie. I just think Wow, if we can heal with our own body, its pretty amazing.
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July 4th, 2017
Skin Stem Cells Used to Generate New Brain Cells – AANS …
By daniellenierenberg
| Newsline
UCI-led study to advance understanding of the role of micoglia in Alzheimers disease
Using human skin cells, University of California, Irvine neurobiologists and their colleagues have created a method to generate one of the principle cell types of the brain called microglia, which play a key role in preserving the function of neural networks and responding to injury and disease. The finding marks an important step in the use of induced pluripotent stem (iPS) cells for targeted approaches to better understand and potentially treat neurological diseases such as Alzheimers. These iPS cells are derived from existing adult skin cells and show increasing utility as a promising approach for studying human disease and developing new therapies. Skin cells were donated from patients at the UCI Alzheimers Disease Research Center. The study, led by Edsel Abud, Wayne Poon and Mathew Blurton Jones of UCI, used a genetic process to reprogram these cells into a pluripotent state capable of developing into any type of cell or tissue of the body.
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July 2nd, 2017
Paul from Weymouth to climb up Snowdon to help fight cancer – Dorset Echo
By daniellenierenberg
Oneyoung Weymouth man is climbing Mount Snowdon on his 21st birthday to help in the fight against cancer.
Paul Cashman-Roberts, 20, is hiking up the mountain on Friday, September 8, to raise money for the Anthony Nolan charity, in support of his aunt and his cousin.
His cousin, Avril Bailey, was diagnosed with breast cancer and had to have one of her breasts removed, while his aunt, Terina Cashman-Stimpson, still suffers from several forms of cancer one of which is blood cancer.
Anthony Nolan works to deliver lifesaving stem cell and bone marrow transplants to those with blood cancer or other blood disorders and conducts vital research into making these transplants more effective.
Paul expressed admiration for both his cousin and his aunt and said he wanted to do something in support of their struggle.
He said: I thought I would do something to raise money for experts who are going to do some fantastic research in the field. I did the Duke of Edinburgh while at Wey Valley School and I love walking my dog all the time.
Paul said that he specifically chose Mount Snowdon to walk up partly because it was more accessible than Ben Nevis.
When he announced he was going to be doing the climb up Snowdon last Thursday Paul received an amazing response from people.
He said: Within the first 24 hours we managed to raise 120.
Initially it was hoped the climb would raise up to 1,000.
Paul added: However, I have been in contact with the guys at Ayya nightclub in Weymouth and we are going to be holding a mini-music festival there to raise even more money.
The young hiker is hoping this will help him to go beyond the 1,000 mark. Both Pauls family and the people at Anthony Nolan have been extremely supportive of him and he is looking forward to his climb up the tallest mountain in England and Wales.
He said: I could not be more thrilled. Really, the end result is about how much we can raise and about saving peoples lives that is really what its all about.
To donate search for Pauls name at http://www.justgiving.com
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June 30th, 2017
Detroit Lions’ Paul Worrilow has beaten odds, inspires others to help – Detroit Free Press
By daniellenierenberg
Free Press Lions beat writer Dave Birkett answers your Twitter questions in a video mailbag June 26, 2017, before summer vacation.
Detroit Lions linebacker Paul Worrilow with daughters Juliet, left, and Rowan, right.(Photo: Paul Worrilow)
Paul Worrilow has a great back story.
The veteran linebacker, who signed with the Lions in March, was undrafted out of Delaware.
He made the Atlanta Falcons, against all odds.
Cracked the starting lineup, against all odds.
Became the teams leading tackler, against all odds.
Stuck around for four seasons, against all odds.
But theres more.
Its a story about being selfless and thinking about others. Its a story that should be repeated, if only to inspire others to follow his lead.
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And it started with a simple cheek swab.
Its so simple, Worrilow said.
When Worrilow was a sophomore at Delaware, he joined the Be The Match Foundation Registry, hoping to become a bone marrow donor.
Four months later, he was matched with a 23-year-old woman with leukemia.
Worrilow donated peripheral blood stem cells to the woman, although he doesnt know what happened to her. He doesnt know her name. He never has met her.
Its so simple, Worrilow said. They do a cheek swab. You get put in the database. If you match somebody, there are two ways to do it. You can donate actual bone marrow or do it like I did, peripheral blood stem cells.
Lions linebacker Paul Worrilow takes part in OTAs on Wednesday, May 24, 2017 at the Allen Park practice facility.(Photo: Kirthmon F. Dozier, Detroit Free Press)
About one in 40 registry members will be called for additional testing.
About one in 300 will be selected as the best possible donor for a patient.
And about one in 430 on the registry go on to donate bone marrow or peripheral blood stem cells.
Odds are, you never will be asked to donate.
But you just might give somebody hope.
Its so simple, Worrilow said. Its not painful. Its a small part of your time, to have a great impact, a tremendous impact on another person and their family. Its a no-brainer. You can have a great impact at such a small cost to yourself.
Worrilow is on a mission to let people know about it: The cool part about it is being able to share it (with people) who are ignorant to the process and their ability to help other people. Encouraging people. People who just dont know what Be The Match Foundation is. Or how you can help people with blood cancers, or how you can join.
Worrilow signed with the Lions during the off-season.
This team is tremendous, Worrilow said. The family-oriented vibe here. From the head coach, from the ownership on down, you can feel it. Its a family vibe. They look after you, care for you.
In June, during minicamp, Worrilow played weak-side linebacker. But he can also play in the middle.
Its going good, he said. There is a lot of competition. The team is great that way, pushing each other. Any action I can get on the field is exciting. When youre winning games, and everyones in here practicing hard, its just awesome.
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Worrilow has a track record for good tackling, but he has been criticized for his inability to cover.
If (the criticism) is there, its probably there for a reason, he said. Criticism, if it doesnt come from a bad place, it is probably warranted. And thats something I have to improve on. Thats with all parts of my game. I dont feel like I have played my best football yet.
Worrilow said he is adjusting to the Lions defense, making defensive calls.
Compared to the other places Ive played, there is a bigger volume of calls, he said. Thats something I like. Both linebackers do it. One guy has the indicator, but if you are not out there talking, you arent going to be out there.
And now, as he takes time off before training camp, he feels confident about himself and this team.
I dont feel like I could be in a better place, life-wise, work-wise, everything, he said. Its an encouraging place to come in and work every day. The linebacker group is awesome. Its young. Its competitive. Thats what you want.
So, this guy keeps breaking the odds.
Sticking in the NFL. And trying to use that platform to help others.
Trying to raise awareness.
Trying to encourage people to make a difference.
Trying to break the odds.
Help us, Detroit Lions: You are Detroit sports fans' only hope right now
For new Detroit Lions LB Paul Worrilow, dad duties come first
Contact Jeff Seidel: jseidel@freepress.com. Follow him on Twitter @seideljeff. To read his recent columns, go to freep.com/sports/jeff-seidel/.
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June 29th, 2017
First in Human Administration of UCART123 in Cellectis’ AML Phase I Clinical Trial at Weill Cornell Medicine … – Markets Insider
By daniellenierenberg
Regulatory News:
Cellectis (Alternext: ALCLS; Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T-cells (UCART), announced today the first administration in the Phase I clinical study in Acute Myeloid Leukemia (AML) for its investigational product UCART123, one of the Companys wholly-controlled TALEN gene-edited product candidates. This marks the first allogeneic, "off-the-shelf gene-edited CAR T-cell product candidate targeting CD123 to be investigated in clinical trials.
This clinical research in AML is led by Principal Investigator Dr. Gail J. Roboz, Professor of Medicine at Weill Cornell Medicine and Director of the Clinical and Translational Leukemia Programs at Weill Cornell Medicine and NewYork-Presbyterian Hospital.
The clinical trial will investigate the safety and efficacy of UCART123 in patients with AML. AML is a devastating clonal hematopoietic stem cell neoplasm which is characterized by uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure. In the U.S., there are an estimated 19,950 new AML cases per year, with 10,430 estimated deaths per year. While complete response rates can be as high as 80 percent in younger patients who undergo initial induction cytotoxic chemotherapy, the majority of AML patients relapse and die from the disease. AML patients with high-risk genetic features have an especially urgent unmet medical need, as their outcomes are dismal with all existing treatment modalities, including allogeneic stem cell transplantation.
"After being granted rapid approval from Regulatory Authorities and Institutional Review Boards to initiate UCART123 studies, the enrollment and treatment of the first patient represents a major milestone for Cellectis, and we are eager to hit the ground running with the recruitment of our first patient for our second UCART123 Phase I study in BPDCN soon, said Dr. Loan Hoang-Sayag, Cellectis Chief Medical Officer. "This first program targeting CD123 will be a paradigm shift for our Company, as it will provide a wealth of valuable additional knowledge and data to drive our gene-edited allogeneic CAR T-cell platform.
"We are excited to be enrolling our first patient with UCART123 and are hopeful that this novel immunotherapy modality will prove to be a significant and effective weapon against AML, said Dr. Roboz.
The clinical trial is part of a strategic translational research alliance that was formed between Cellectis and Weill Cornell Medicine in 2015. Dr. Monica Guzman, an associate professor of pharmacology in medicine at Weill Cornell Medicine, is co-principal investigator whose work focuses on preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.
About Cellectis
Cellectis is a clinical-stage biopharmaceutical company focused on developing a new generation of cancer immunotherapies based on gene-edited T-cells (UCART). By capitalizing on its 17 years of expertise in gene editing built on its flagship TALEN technology and pioneering electroporation system PulseAgile Cellectis uses the power of the immune system to target and eradicate cancer cells.
Using its life-science-focused, pioneering genome engineering technologies, Cellectis goal is to create innovative products in multiple fields and with various target markets.
Cellectis is listed on the Nasdaq market (ticker: CLLS) and on the NYSE Alternext market (ticker: ALCLS). To find out more about us, visit our website: http://www.cellectis.com
Talking about gene editing? We do it. TALEN is a registered trademark owned by the Cellectis Group.
Disclaimer
This press release contains "forward-looking statements that are based on our managements current expectations and assumptions and on information currently available to management. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. The risks and uncertainties include, but are not limited to, the risk that the preliminary results from our product candidates will not continue or be repeated, the risk of not maintaining regulatory approval to pursue UCART123 clinical trials, the risk of not obtaining regulatory approvals to commence clinical studies on UCART123 in other countries or on other UCART product candidates, the risk that any one or more of our product candidates will not be successfully developed and commercialized. Further information on the risks factors that may affect company business and financial performance, is included in filings Cellectis makes with the Security Exchange Commission from time to time and its financial reports. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170627006309/en/
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June 28th, 2017
After two stem cell transplants and several rounds of chemo, ‘now he’s just like a normal 2-year-old’ – GoDanRiver.com
By daniellenierenberg
When Shannon DeAndrea saw a knot on her 18-month-old sons head last July, she thought he had just fallen.
But more popped up and wouldnt go away. He also began feeling sick.
I finally decided he needed to see a pediatrician, said DeAndrea, who lives in Blairs.
She was told he had ear infections and her son, Nathan, was put on rounds of antibiotics. The knots were normal, she was told.
Another medical provider said he looked anemic. Blood work revealed his hemoglobin was dangerously low.
We ended up in the ER, DeAndrea said. They couldnt figure out why he was anemic.
Shannon and Nathan were sent to Roanoke, where he was diagnosed with a stage 4 neuroblastoma on Aug. 23. He had a tumor in his abdomen that spread to his bone marrow. He had spots on his skull, ribs and spine.
Neuroblastomas are cancers that begin in early nerve cells of the sympathetic nervous system, according to the American Cancer Society.
Since his diagnosis, her son now 2 has had several rounds of chemotherapy and two stem cell transplants and is doing well.
Now hes just like a normal 2-year-old, DeAndrea said. Hes running around with his sister. Hes eating well.
Dr. William Clark is associate professor of medicine and attending physician at Virginia Commonwealth University Massey Cancer Center Stem Cell Transplantation Program. Clark said the procedure is used for conditions including multiple myeloma, lymphoma, sickle cell anemia and leukemia.
Stem cell transplants are used to replace bone marrow that has been destroyed by cancer or destroyed by the chemo and/or radiation used to treat the cancer, according to the American Cancer Society.
High doses of chemo (sometimes along with radiation), work better than standard doses to kill cancer cells. However, high doses can also kill the stem cells and cause the bone marrow to stop making blood cells, which are needed for life. The transplanted stem cells replace the bodys stem cells after the bone marrow and its stem cells have been destroyed by treatment, according to the American Cancer Society.
Two types of stem cell transplants include autologous, which uses stem cells from the patients own body, and allogeneic using stem cells from another person, Clark said.
For leukemia patients, most of the time, we give them stem cells from someone else, Clark said. Chemotherapy helps lower the leukemia disease burden, but the new immune system provided by the new stem cells can fight against the cancer cells and get rid of them, he said.
Virginia Commonwealth Universitys cancer center performs an average of about 160-195 stem cell transplants per year, Clark said. Slightly more than half are autologous procedures, and the rest are allogeneic, he said.
Whitt Clement, former delegate who represented the Danville area in the General Assembly, underwent a stem cell transplant for acute myeloid leukemia in September 2015.
The most important aspect for patients is being self-aware and their own best advocates, Clement said.
My experience was that the patient has to ask a lot of questions throughout the process, he said.
He suspected something was wrong when he noticed his platelet count declining over seven years. He went to a hematologist and had a bone marrow biopsy that revealed his condition.
If I had not taken the initiative myself and gone to see a hematologist, matters would have progressed to the point where I would have been symptomatic, Clement said.
Finding the perfect match in a donor is also important, Clement said. Fortunately, he had a sibling who met all the criteria and donated stem cells.
A person can get great matches from unrelated donors, but its preferable for a donor to be a sibling, said Clement, partner at Hunton & Williams law firm in Richmond.
Your body has an easier time tolerating the new stem cells, he said.
Clement served in the Virginia House of Delegates from 1988-2002, and as Virginias secretary of transportation from 2002-2005 under Gov. Mark Warner.
For someone with multiple myeloma, the transplant does not cure the disease but delays the time it returns by up to seven and a half years, Clark said.
Lymphoma, leukemia and sickle cell anemia can be cured with the procedure, Clark said. Lymphoma can be cured in about 50 to 80 percent of cases, depending on the lymphoma, Clark said.
The first 30 days after the transplant are the most critical, Clement said. During that time, different organs can have varying reactions to the new cells. It can affect the kidneys, liver, gastrointestinal tract, skin, and cause other side effects.
The idea is that the closer the match, the less likely youll have those adverse reactions, he said.
The process includes being put on an immunosuppressant to prevent the immune system from attacking the new cells, Clement said.
He credits the quality of his recovery to asking lots of questions and being his own advocate tape recording conversations with medical providers, coming in with written questions.
Ive been able to recover better because of that, he said.
Its a long journey and so a person confronted with the transplant situation has got to prepare himself for a long journey that requires a lot of questions along the way, Clement said.
There are about 20 million potential stem cell/bone marrow donors in the BeTheMatch Registry in the United States, Clark said.
Stem cell transplants began in the late 50s/early 60s with the first successful procedure done in an identical twin, Clark said. However, stem cell transplants were limited until medicines that prevent rejections became available.
The number of procedures increased in the 1980s, Clark said.
Danville resident Susan Mathena, cancer patient navigator at Danville Regional Medical Center, became a donor about 20 years ago because she wanted to help people. Mathena has also been an organ donor since she got her drivers license.
I see patients all the time that need stem cell transplants, Mathena said. We always need a source of bone marrow donation.
Though she will age out of the stem cell donor list soon, she could still be contacted if she is the only match for someone in need, she said.
Clark will speak next month on stem cell/bone marrow transplants at Ballou Recreation Center at an event held by the Cancer Research and Resource Center of Southern Virginia in Danville.
Thousands of patients with blood cancers like leukemia or other diseases like sickle cell anemia need a bone marrow/stem cell transplant to survive, including some of our own community members, said Kate Stokely Powell, coordinator at the center.
Clarks presentation offers an opportunity in Southside for people battling illness, medical students and professionals and the public to learn from an expert in the field of stem cell transplants, Powell said.
Doctors, hospitals and families affected by a blood cancer disease have done a great job of building a massive database of blood types for potential donor matches, Clement said.
For DeAndrea and her son, Nathan, the first transplant included four or five days of chemo. The new stem cells following the chemo that killed off his old stem cells from the transplant were like a rescue, she said.
Its wiping you out and then giving you your cells back to restart your immune system, DeAndrea said.
A second round of heavy chemo was to try to kill what was left of the cancer and replenish cells, she said.
It was rough, it was a nightmare, DeAndrea said. It was by far the worst phase of his treatment, but I believe, in the long run, its worth it.
She said the procedures should increase Nathans chances for survival and prevent a relapse.
Nathan just finished radiation Tuesday and will go in for a biopsy of his bone marrow this week, DeAndrea said.
Well find out next week where we stand as far as the cancer goes, she said.
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After two stem cell transplants and several rounds of chemo, 'now he's just like a normal 2-year-old' - GoDanRiver.com
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June 25th, 2017
Jonathan Pitre still ailing as doctors search for answers – Ottawa Citizen
By daniellenierenberg
Photo of Jonathan Pitre and his mother, Tina Boileau, taken in Minnesota. Tina Boileau / -
Doctors in a Minnesota hospital continue to search for answers to a mysterious infection that has left Jonathan Pitre feverish, nauseated and short of breath.
Pitre, 17, of Russell, has been in the University of Minnesota Masonic Childrens Hospital for the past two weeks, suffering from an array of complications more two months after his stem cell transplant. Doctors are also trying to adjust his medications to better deal with his increased pain levels.
Hes having a tough run, said his mother, Tina Boileau, and I really dont know when it will get better.
The teenager suffers from a severe form of epidermolysis bullosa (EB), a painful and progressive skin disease that has left deep, open wounds on his body.
Last week, Pitres face and neck became swollen in response to what doctors believed was some kind of viral infection. That swelling has been brought under control, but a battery of tests has yet to reveal the source of the infection, which continues to cause problems.
Pitres breathing is laboured and hes running a high-grade fever of about 104 F (40 C); he has also developed bleeding and painful sores in his mouth.
We still have no idea what were dealing with, said Boileau. Its frustrating because Im at the point where it would be nice to see that all that Jonathan has gone through has been worth it.
Doctors are monitoring Pitre for graft-versus-host-disease (GVHD), but all of his tests have so far been inconclusive.Anyone who receives stem cells from another person is at risk of developing GVHD, a condition in which the donors white blood cells turn on the patients own tissues and attack them as foreign. It can range from mild to life-threatening.
About one-third of the almost 50 EB patients who have had a stem cell transplant at the Masonic Childrens Hospital have experienced the condition.
Pitre checked back into hospital earlier this month just three days after being released following a stem cell transplant that had successfully taken root in his bone marrow. Bone marrow stem cells produce most of the bodys blood cells, and are responsible for arming its immune system.
Pitre has been in Minnesota since mid-February to undergo the transplant, his second. The first ended in disappointment on Thanksgiving Day last year.
Tests show Pitres latest transplant remains fully engrafted, and there are signs that it has started to improve the condition of his skin.
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Jonathan Pitre still ailing as doctors search for answers - Ottawa Citizen
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June 24th, 2017
Aging-related mutations in blood cells represent major new risk factor for cardiovascular disease – Medical Xpress
By daniellenierenberg
June 22, 2017 Credit : Susanna M. Hamilton, Broad Communications
Scientists at the Broad Institute of MIT and Harvard have found that a set of genetic mutations in blood cells that arises during aging may be a major new risk factor for cardiovascular disease. In contrast to inherited genetic predispositions and traditional lifestyle risk factors, such as smoking or an unhealthy diet, the new mutations are "somatic mutations" that originate in stem cells in the bone marrow as people age.
Because the mutations are relatively common in older people (over 10% of people over the age of 70 harbor at least one of these mutations), potential future efforts to screen for the mutations in blood cells, identify people at increased risk for coronary heart disease, and reduce risk in those individuals through lifestyle changes or therapeutic interventions could have a significant clinical impact, according to the researchers.
"There is more work to be done, but these results demonstrate that pre-malignant mutations in blood cells are a major cause of cardiovascular disease that in the future may be treatable either with standard therapies or new therapeutic strategies based on these findings," said Benjamin Ebert, a co-senior author of the new study, an institute member at the Broad, a professor of medicine at Harvard Medical School, and a hematologist at Brigham and Women's Hospital.
Featured in the New England Journal of Medicine, the work also contributes to the broader understanding of pathogenesis in coronary heart disease by supporting the hypothesis that inflammation, in addition to elevated cholesterol levels, plays an important role in this illness and potentially other diseases of aging.
"A key finding from this study is that somatic mutations are actually modulating risk for a common disease, something we haven't seen other than in cancer," said first author Siddhartha Jaiswal, a pathologist at Massachusetts General Hospital and researcher in the Ebert lab. "It opens up interesting questions about other diseases of aging in which acquired mutations, in addition to lifestyle and inherited factors, could modulate disease risk."
Previous research led by Ebert and Jaiswal revealed that some somatic mutations that are able to confer a selective advantage to blood stem cells become much more frequent with aging. They named this condition "clonal hematopoiesis of indeterminate potential," (CHIP), and found that it increases the risk of developing a blood cancer more than 10-fold and it appeared to increase mortality from heart attacks or stroke. In the new study, the researchers analyzed data from four case-control studies on more than 8,000 people and found that having one of the CHIP-related mutations nearly doubled the risk for coronary heart disease, with the mutations conferring an even greater risk in people who have previously had a heart attack before age 50.
While the human genetics data showed a strong association between CHIP and coronary heart disease, the team hoped to uncover the underlying biology. Using a mouse model prone to developing atherosclerosis, the scientists showed that loss of one of the CHIP-mutated genes, Tet2, in bone marrow cells leads to larger atherosclerotic plaques in blood vessels, evidence that this mutation can accelerate atherosclerosis in mice.
Atherosclerosis is believed to be a disease of chronic inflammation that can arise in response to excess cholesterol in the vessel wall. To examine this on a cellular level the team turned to the macrophage, an immune cell found in atherosclerotic plaques that can develop from CHIP stem cells and carry the same mutations. Because Tet2 and other CHIP-related mutations are known to be so-called "epigenetic regulators" that can alter the activity of other genes, the team examined gene expression levels in the Tet2-mutated macrophages from mice. They found that the mutated cells appear to be "hyper-inflammatory" with increased expression of inflammatory molecules that contribute to atherosclerosis. In support of this finding, humans with TET2 mutations also had higher levels of one of these molecules, IL-8, in their blood.
The work demonstrates that CHIP associates with coronary heart disease in humans, that mutation of the CHIP-related gene Tet2 causes atherosclerosis in mice, and that an inflammatory mechanism likely underlies the process. More work is needed to show whether other genes that are mutated in CHIP also lead to increased inflammation. The team is also exploring whether interventions such as cholesterol lowering therapy or anti-inflammatory drugs might have benefit in people with CHIP.
Inflammation is also thought to modulate several other diseases of aging besides cardiovascular disease, such as autoimmune disorders and neurodegenerative disease. Because CHIP also increases in frequency with age, somatic mutations that alter inflammatory processes could influence several diseases of aging, though more work is needed to test this possibility.
"By combining genetic analysis on large cohorts with disease model and gene expression studies, we've been able to confirm the earlier hints of CHIP's surprising role in cardiovascular disease," said co-senior author Sekar Kathiresan, director of the Broad's Cardiovascular Disease Initiative, associate professor of medicine at Harvard Medical School, and director of the Center for Genomic Medicine at Massachusetts General Hospital. "Beyond the mutations that you inherit from your parents, this work reveals a new genetic mechanism for atherosclerosismutations in blood stem cells that arise with aging."
Explore further: A role for mutated blood cells in heart disease?
More information: Siddhartha Jaiswal et al. Clonal Hematopoiesis and Risk of Atherosclerotic Cardiovascular Disease, New England Journal of Medicine (2017). DOI: 10.1056/NEJMoa1701719
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June 22nd, 2017
He broke ground in stem-cell research. Now he’s running for Congress. – Washington Post
By daniellenierenberg
The small pack of scientists running for political office has grown by one.
Stem-cell researcher Hans Keirstead, 50, announced last week that he will try to unseat Californias Rep. Dana Rohrabacher (R). Keirstead, a Democrat with a PhD in neuroscience from the University of British Columbia, was a professor at the University of California at Irvinebefore launching and selling several biotech companies.
Rohrabacher, who represents the 48th District in Southern California, has been in Congress since 1988. Democrats there see 2018 asa vulnerable year for the incumbent. Although Republicans outnumber Democrats in thedistrict, Hillary Clinton swung it in the 2016 election. And Rohrabacher has come under scrutiny for his support of acloser relationship with Russia. In May, the chair of Orange County Democrats toldThe Washington Post that challengers were coming out the woodwork to oppose him. Five candidatesbesides Keirstead have declared they are running for the seat.
Keirstead emerged from academic and entrepreneurial fields. Hepioneered a technique to purify stem cells You cant go putting toenails into the spinal cord, he said and applied this method to spinal-cord injuries and diseases such ascancer and amyotrophic lateral sclerosis, or ALS. In 2014,he sold a stem-cell company in a deal reportedly worth more than $100 million. (He will not fundhis own campaign, he told the Los Angeles Times.) Keirstead has thesupportof314 Action, a nonprofit group that encourages scientists to seek public office.
The Post spoke by phone with the first-time candidate. The following is lightly edited for space and clarity.
TWP: Your opponent, who is a member of the House Science Committee, told Science magazine in 2012 that he loved science. How would you compare your approaches to science?
Keirstead:Im delighted that Dana Rohrabacher loves science. Thats fabulous. But Im also very convinced that he doesnt understand science. Theres a real big difference. If you love science, thats one thing. If you dont understand it, you cant effect change, and you make wrong decisions.
Dana Rohrabacher does not understand global warming. He actually attributed it to the flatulence of dinosaurs, in a serious manner, a while back. [Rohrabacher hassaid this wasa joketo make fun of scientists who study cow methane.]
His inaction and lack of understanding has tremendous detriment on the scientific community. Likewise is the funding to health care and how to fix the health-care system that [former president Barack] Obama put in place. That was not a perfect system by any means; its got problems.But it has also bettered our system. It needs to be worked with in order to further better our system.
TWP: Has your career in stem-cell research influenced your politics?
Keirstead:I was front and center in the national and international debate on stem cells. I was the first scientist in the world to have developed a treatment for spinal-cord injury using stem cells. The dramatic nature of the recovery we saw in rodents, going from paralyzed to walking, drew a great deal of attention and really put me at the center of this issue as it was just coming to light in the public forums.
I did a lot of advising of senators and congressmen all throughout those years and periodically since that time. . . . I was one of the key scientific advisers to Proposition 71 that turned into the $3 billion California Institute of Regenerative Medicine, a not-for-profit that distributes $300 million every year for regenerative medicine in a broad sense.
That was a very good example of how medical breakthroughs and discoveries and advancement are not at odds with economic development. You do not have to cut medical budgets to stimulate the economy. Any scientist and medical doctor will tell you: Give me some time, and I will generate a treatment. And most of the time they are right. What happens with that treatment is small companies are born, people stop dying, quality of life improves.
I see what the governments doing right now as very much opposite that. Frankly, when I look at the deficits of Congress, I see why. When I look at who is in the administration, the types of individuals that we have in Congress, I see very hard-working people doing what they feel is a terrific job. But there is just not the broad and deep field experience in the medical and health-care sectors.
TWP: Was it this perceived deficit that motivated you to run for Congress?
Keirstead:First and foremost, I see it as a continuation of my lifelong pursuits of trying to help people.
I see Congress as a larger stage to effect positive change. If I could have some positive influence in Congress, I could aid [those] that are trying to do good in the world but are having difficulty.
Let me give you an example: Im now expanding into brain cancer. Im running a Phase 2clinical trial with my team.I will not be able to do that if these policy changes of Trumps are instituted and a small company like mine is faced with double user fees. Its not in the budget. I cant ask an investor for another half of a million dollars for an administrative fee.
I see the administration putting insurmountable challenges in front of small businesses. Im about generating treatments to help people, putting medicines in peoples homes. And Im looking to the future and seeing that tap shut off.
Read more:
As scientists erupt in protest, a volcanologist runs for Congress
This group wants to fight anti-science rhetoric by getting scientists to run for office
Tens of thousands marched for science. Now what?
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He broke ground in stem-cell research. Now he's running for Congress. - Washington Post
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June 21st, 2017
Awesome Dawson: The legacy an 8-year-old boy who battled leukemia leaves behind – ABC10
By daniellenierenberg
Frances Wang, KXTV 8:33 PM. PDT June 17, 2017
Dawson Deschaine passed away on June 10th after a 2-and-a-half year battle with leukemia. He was only 8-years-old.
While most kids try to earn gold stars for their work, Dawson Deschainefelt a little pride every time he earned a bead.
"The Beads of Courage program is these beads that [represent] every poke, every hospital stay, every bone marrow biopsy, every chemotherapy," said Breanna Deschaine, Dawson's mother.
Dawson was diagnosed with leukemia in January 2015 at just 6-years-old. The battle would last two-and-a-half years. It was all Dawson ever knew.
"He mostly knew nurses, doctors, family," said Jason Deschaine, Dawson's father. "He had a lot more adult conversations than kid ones."
Breanna said he was an old soul. He even drank a cup of coffee every morning (decaf, of course).
"They accidentally sent him coffee [instead of hot chocolate] to his room one day," said Breanna.
Dawson's mom said he was an old soul. After getting coffee instead of hot chocolate, that's what he drank every morning (decaf, of course). pic.twitter.com/eYx7Ed57XX
Dawson battled leukemia for 2-and-a half years. Smiled through it all. Chemo, stem cell transplanted, bone marrow biopsies... pic.twitter.com/NN5NoFSFLI
Now you know an infectious smile like Dawson's comes with some pretty funny stories.
Last August, Nevada County made Dawson an honorary firefighter. They called it 'Dawson Day' with a ceremony and all.
Sweet Dawson passed away this Sat. after battling leukemia. He was only 8 years old. Last yr, he became an honorary Nevada Co. firefighter. pic.twitter.com/RkVOY0WGhD
Dawson was given a badge, his own turnouts, and boots. He even got to respond to an injured biker.
"He kept telling the EMTs how to wrap the leg!" said Breanna.
And his firefighter card, he never took for granted.
"He was getting ready to go to clinic one day," said Jason. "He comes back running in the house saying 'I need my ID Card!'...'No no, I need it. Just in case mom gets pulled over, [I can say police officer,] I am with the fire department.
It's reminiscing and laughing about stories like this that keep his family strong and smiling even when it hurts.
"People always ask how we're doing. As you can tell we smile. Dawson would never let us cry in the room," said Breanna.
Dawson's community made sure his last months in this world was full of adventure. Sadly, he got too sick for his Make-A-Wish: to go to Hawaii and swim with dolphins.
Dawson had plenty of adventures his last 6 months. Sadly, he never got to swim with dolphins in Hawaii... it was his dream . pic.twitter.com/UQeAqIwzW3
On June 10th at 6:05 AM, with his parents and his sister by his side, Dawson passed away.
Even up until his very last moment, he gave his family a thumbs up.
"He couldn't talk very much. Just the thumbs up that he was still good. He was Awesome Dawson," said Breanna, through tears. "It was his signature. No matter what...I told him 'It's OK Dawson. You fought the hardest battle and you won.'"
"You're not supposed to cry!" said Melody, Dawson's grandmother. "He's watching you."
And if Dawson was watching, what would they say?
"I would say thank you, for the opportunity...you pulled our community together and made our family so strong," said Melody. "We all love him very, very much. And miss him."
Breanna said she would read to him again from his favorite book 'Love You Forever.'
"His favorite saying from his favorite book: 'I love you forever. I like you for always. As long as I'm living, my baby you'll be," said Breanna.
Dawson's family hopes to continue his legacy by bringing the Beads of Courage program that got him through his darkest days into more hospitals.
Donations can be made on the Beads of Couragewebsite, under Dawson's name.
And until they see Dawson again, the family says they'll live by this motto: 'Don't cry because it's over. Smile because it happened.'
That is what Dawson would've wanted.
2017 KXTV-TV
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Awesome Dawson: The legacy an 8-year-old boy who battled leukemia leaves behind - ABC10
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June 18th, 2017
Station Crew Researches Mold, Rodents and Stem Cells as Cargo Ship Chases Station – Space Fellowship
By daniellenierenberg
Russias Progress 67 (67P) cargo craft is orbiting Earth and on its way to the International Space Station Friday morning carrying over three tons of food, fuel and supplies. Meanwhile, the three member Expedition 52 crew researched a variety of space science on Thursday while preparing for the arrival of the 67P.
Commander Fyodor Yurchikhin and Flight Engineer Jack Fischer will monitor the automated docking of the 67P to the Zvezda service module Friday at 7:42 a.m. EDT. NASA TV will broadcast live the resupply ships approach and rendezvous beginning at 7 a.m. The 67Ps docking will mark four spaceships attached to the space station.
Fischer spent the morning photographing mold and bacteria samples on petri dishes as part of six student-led biology experiments that are taking place inside a NanoRacks module. In the afternoon, he removed protein crystal samples from a science freezer, let them thaw and observed the samples using a specialized microscope.
Flight Engineer Peggy Whitson tended to rodents Thursday morning cleaning their habitat facilities and restocking their food. In the afternoon, she moved to human research swapping out samples for the Cardiac Stem Cells study that is exploring why living in space may accelerate the aging process.
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June 16th, 2017
Researchers Discover Body’s Stem Cell Army Hits a Wall When … – Newswise (press release)
By daniellenierenberg
Newswise You might think stem cells only exist inside a fetus, but your adult body has a stockpile of stem cells, armed and ready to respond. These remarkable cells can develop into any other type of cell, like muscle or bone or nerve cells.
Researchers know heart attacks and strokes summon these cells. They flock to your heart or brain from all over your body to help you stay alive.
But, scientists did not realize other injuries, like a torn ACL of the knee, could command the army of stem cells to deploy.
Kevin Baker, Ph.D., Beaumont director of Orthopedic Research, conducted a study with Beaumont orthopedic surgeon Kyle Anderson, M.D., and others that revealed ACL tears send a signal to stem cells throughout our body.
After an ACL tear, Dr. Baker and his colleagues found a six-fold increase in stem cells circulating around the knee, similar to the bodys response to a major, life-threatening event like a stroke or heart attack.
However, when the stem cells arrive to help regenerate and repair the injured ligament, they get stuck. They cant get through the thick membrane that surrounds the knee joint.
We think this discovery will help us to understand how the body responds to an ACL injury, and also how post-traumatic osteoarthritis develops after a joint injury, Dr. Anderson said.
Post-traumatic osteoarthritis is a form of arthritis that develops after a knee injury. Its a common injury that affects veterans, athletes and anyone who puts stress and strain on their knees. But, until now, little was known about how the body attempts to heal these injuries.
As we age, the number of stem cells in our body declines. This could explain why your knee joint doesnt heal as well after a trauma when you are older, Dr. Baker said.
Osteoarthritis affects more than 30 million adults in the United States, according to the Centers for Disease Control and Prevention, and many of these cases occur after trauma to a joint. Its also a leading cause of disability.
The next step of our research will be finding methods to get the stem cells inside the joint. If the stem cells can get through the membrane around the knee, they could help speed up the healing process and perhaps delay or prevent arthritis, Dr. Baker added.
The study, funded in part by the American Orthopedic Society of Sports Medicine, is entitled, Acute mobilization and migration of bone marrow-derived stem cells following anterior cruciate ligament rupture. The authors believe it is the first study of its kind to reveal the bodys systemic stem cell response to an ACL injury.
Dr. Baker and Dr. Andersons research will appear in an upcoming edition of the journal Osteoarthritis and Cartilage. Other members of the research team are Perry Altman, M.D., Beaumont orthopaedic surgery resident, as well as Asheesh Bedi, M.D., and Tristan Maerz, Ph.D., of the University of Michigan.
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June 16th, 2017
Domainex, Imperial College London Extend Cardiac Therapy Collaboration – Genetic Engineering & Biotechnology News
By daniellenierenberg
Domainex will expand its two-year-old collaboration with Imperial College London to discover new therapies that reduce heart muscle damage during heart attacks, the partners said today.
Domainex and Imperial aim to discover a treatment that inhibits the enzyme MAP4K4, which is linked to cell death following heart attacks. Since the collaboration was launched in 2015, the partners said, they have discovered novel, potent, and selective MAP4K4 inhibitors using human cardiac muscle grown from human induced pluripotent stem cells (iPSCs).
The inhibitors have shown promise in protecting these cells against oxidative stress, a trigger for cell death during heart attacks, Domainex and Imperial said.
As a result of the progress, Imperial College London said, its Professor Michael Schneider, Ph.D., has secured a follow-on award of 4.5 million (nearly $5.8 million) from the Wellcome Trusts Seeding Drug Discovery initiative to continue the research.
From its Medicines Research Centre near Cambridge, U.K., Domainex said, its researchers will continue to provide integrated drug discovery servicesincluding further biochemical, cellular and biophysical assay screening, and structure-guided medicinal chemistry coupled with drug metabolism, safety, and pharmacokinetic assessment of promising candidates.
Domainex and Imperial said they aim to advance potential treatments into preclinical development and ultimately to clinical evaluation.
"We have already identified a number of very exciting, novel inhibitors through structure-based drug design," Domainex CSO Trevor Perrior said in a statement. The innovative cardiac muscle assay developed by the team here at Domainex working in partnership with Imperial College London, is enabling early testing on human cardiac muscle cells, which will make cardiac drug discovery more efficient and effective in identifying efficacious candidate drugs.
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June 14th, 2017
Regenerative Medicine Can Help Make America Great – Morning Consult
By daniellenierenberg
When President Donald Trump urged the biopharmaceutical industry to reduce the price of new medicines and to increase its manufacturing in the United States, many took it as a threat.
We believe its a call to action. Americas ingenuity in biomedical research is unsurpassed. However, our country is losing out to other nations in the fastest growing biotechnology sector, called regenerative medicine: harnessing the capacity of our cells to repair and restore health and sustain well-being.
Second place is not an option. The regenerative medicine market is growing about 21 percent a year and is expected to be worth over $350 billion by 2050. Today, the U.S. regenerative medicine sector is generating $3.6 billion in revenues and has produced 14,000 jobs. By 2050, the industry could create nearly a million new jobs nationwide.
Regenerative medicine will also reduce the cost of disease. Such therapies will replace drugs, devices, and surgery, saving lives, increasing productivity, and reducing the cost of care. This transformation will add trillions in value to our economy.
Finally, regenerative medicine will also make America more secure. Our nation still lacks the ability to quickly and cheaply mass produce vaccines, antidotes, and cell therapies to counter pandemics and bioterrorism. Our fighting forces need reliable sources of these countermeasures and deserve immediate access to treatments that give them back their lives. We shouldnt outsource the safety and well-being of our nation and our Armed Forces to other countries.
To regain leadership in regenerative medicine, U.S. firms dont need government loans, tax credits or massive de-regulation. Instead, it needs the opportunity to invest in reducing the time and cost of manufacturing cellular therapies. To the extent that regenerative medicine is curative it must be made available at vaccine like prices. At present, only a handful of people can afford such treatments.
China and Japan are now in forefront of reducing the cost of producing stem cells, tissue, and other products with restorative biological properties. As a result, they are attracting more capital and forming more new companies than the U.S.
In 2014 Japan became the first country in the world to adopt an expedited approval system specifically for regenerative medical products and to allow outsourced cell culturing. Two products were approved under the new system within a year of its adoption.
By contrast, the Food and Drug Administration regulates any use of manufactured stem cells as equally risky without regard to prior use, health benefit, or therapeutic potential. Indeed, many of the most common stem cell therapies including bone marrow transplants and blood transfusions would require 10 years of FDA review if they were brought to market today.
The problem isnt over-regulation. Its outdated regulation. Safety checks and benchmarks for cell manufacturing should be based on real world evidence of past applications. Regulation should focus on the specific potential side effects for each specific potential use. In this regard, we agree with incoming FDA Commissioner Scott Gottlieb, who has noted, Expediting the development of these novel and transformative technologies like gene- and cell-based therapies doesnt necessarily mean lowering the standard for approval, as I believe other countries have done. But it does mean having a framework thats crafted to deal with the unique hypothetical risks that these products pose.
In fact, the United States has the best regenerative medicine manufacturing technology in the world. But it is literally sitting unused in warehouses.
For example, under the Accelerated Manufacture of Pharmaceuticals program, private companies partnered with the Defense Advanced Research Projects Agency to develop mobile cell and tissue manufacturing plants that can be set up almost anywhere. The facilities can produce cells and tissues at a fraction of the current cost. These mobile factories make real-time production of vaccines and biologics for potential bioterrorist threats and pandemics possible. They are also low-cost, high-tech platforms for experimental evaluation of any type of regenerative medicine.
AMPs are operating in Indonesia, Singapore, China, and Japan where cell products including vaccines are being mass produced. Not a single AMP is being used in the United States because of outdated regulations.
To remove this regulatory obstacle, the Trump administration should establish a separate regenerative medicine pathway. This pathway, which could be developed by DARPA, FDA, and the Centers for Disease Control and Prevention, would develop regulatory standards for the safe manufacturing and testing of development of regenerative products to treat battlefield related traumas such as traumatic brain injury, life-threatening limb damage, and drug-resistant pathogens.
The focus on the conditions and circumstances unique to war or counter-terrorism is both appropriate and strategic. After World War II, Franklin Roosevelt directed that the scientific and entrepreneurial talents used to achieve ramp up war-time production of penicillin and blood plasma be used in the days of peace ahead for the improvement of the national health, the creation of new enterprises bringing new jobs, and the betterment of the national standard of living.
What was created exceeded that vision. The cooperative efforts to achieve mass production of penicillin and blood plasma inspired and supported the creation of industries that employ millions of people today.
Similarly,developing an affordable source of cell therapies to heal our fighting forces and protect the homeland will yield a wide array of affordable technologies and cures that will produce, in FDRs words, a fuller and more fruitful employment and a fuller and more fruitful life. Simply put, by making the manufacture of regenerative medicine affordable can help make America great.
Robert Hariri is CEO of Celularity. Robert Goldberg is vice president of Center for Medicine in the Public Interest.
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Regenerative Medicine Can Help Make America Great - Morning Consult
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June 12th, 2017
Potential Baldness Pathway Uncovered while Studying Rare Skin Disease – Genetic Engineering & Biotechnology News
By daniellenierenberg
The researchers constructed mouse models for WNT10A-associated HED by deleting the Wnt10a gene. The mutant mice displayed the same symptoms as HED patients with severe loss-of-function mutations in the WNT10A gene. Long-term absence of WNT10A leads to miniaturization of hair follicle structures and enlargement of the associated sebaceous glands, a phenomenon that is also observed in male pattern baldness.
We showed "that -catenin pathway activity and adult epithelial progenitor proliferation are reduced in the absence of WNT10A, and identify Wnt-active self-renewing stem cells in affected tissues including hair follicles, sebaceous glands, taste buds, nails, and sweat ducts, the authors wrote. Human and mouse WNT10A mutant palmoplantar and tongue epithelia also display specific differentiation defects that are mimicked by the loss of the transcription factor KLF4. We found that -catenin interacts directly with region-specific LEF/TCF [lymphoid enhancer factor/T-cell factor] factors, and with KLF4 in differentiating, but not proliferating, cells to promote expression of specialized keratins required for normal tissue structure and integrity.
Interestingly, the UPenn team also discovered that cracking and scaling of palm and foot sole skin in WNT10A patients is due to decreased expression of a structural protein called keratin 9, which is specifically expressed in these regions of skin and contributes to its mechanical integrity.
"Our studies took us back and forth between human patients and our mouse model," said Dr. Millar. "Our goal was to find what happened to cellular components affected by the WNT10A mutation to make better treatments."
Dr. Millar and her colleagues showed that decreased proliferation and keratin 9 expression in the absence of WNT10A resulted from the failure of signaling through a well-characterized pathway that stabilizes -catenin, allowing it to enter the cell nucleus and activate gene transcription. These findings indicate that small-molecule drugs that activate the -catenin pathway downstream of WNT10A could potentially be used to treat hair thinning and palm and sole skin defects in WNT10A patients. These agents may also be useful for preventing hair loss in a subgroup of people with male-pattern baldness.
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Potential Baldness Pathway Uncovered while Studying Rare Skin Disease - Genetic Engineering & Biotechnology News
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June 9th, 2017
Want to save a life? Cuban American searches for bone marrow donor – Miami Herald
By daniellenierenberg
Miami Herald | Want to save a life? Cuban American searches for bone marrow donor Miami Herald According to Gift of Life, a nonprofit, Boca Raton-based bone marrow and blood stem cell registry, 55 percent of Hispanic cancer patients and 75 percent of multiracial patients are never matched, some dying while waiting to get a transplant. The data ... |
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Want to save a life? Cuban American searches for bone marrow donor - Miami Herald
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June 3rd, 2017