It's a long road ahead for Madalayna Ducharme after her stem cell transplant Friday.

The seven-month-old baby was diagnosed with malignant infantile osteopetrosis, which stunts growth, impairs vision and hearing and if untreated can be fatal.

The girl's family put out a plea in January to try to find a stem cell donor.

It took a few months, with 1,300 people attending swab events and a match was found.

The family posted on Facebook that The best agreed upon opportunity/match for us after many tests and consultations is our son Henrik.

The 2-year-old Henrik had a bone marrow harvest Friday. The family says he had surgery to remove bone marrow from both hips. Madalyna later received her brother's bone marrow in the form of a blood transfusion.

Doctors told the family the surgery went well.

Henrik has since been discharged and is recovering at the Ronald Mcdonald House.

For Madalayna, it's a matter of wait and see to find out if the marrow going into her bones will work as her own.

It will take between 14 and 27 days before doctors can tell if the transfusion was successful.

In the Facebook post, the family thanked everyone for their thoughts and prayers and asked people to continue to think about the family.

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Baby's stem cell transplant goes well, doctors say - CTV News

By FPJ Bureau|Mar 19, 2017 12:05 am

Mumbai : A Bone Marrow Transplant (BMT) and Birthing Centre was launched by actor Shah Rukh Khan on Friday at Nanavati Super Speciality Hospital (NSSH) at Vile Parle. On the occasion, Khan told the people present to take their medicines on timeand do regular check ups and get rid of ailments.

BMT is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Bone marrow is the soft, fatty tissue inside our bones which produces blood cells. Stem cells are immature cells in the bone marrow that give rise to different blood cells.

Dr Nimish Kulkarni, Associate Consultant of BMT, said, BMT are considered the last-mile treatment solutions for patients with blood and cancer disorders. We specialise in providing transplant services for benign hematological disorders like Thalassemia, Sickle cell disease, Aplastic anemia, bleeding disorders and coagulation disorders. Shah Rukh said, I have been associated with Nanavati Hospital before 25 years ago as a patient. Every year more than 10,000 people are dying of cancer in India, even my parents succumbed to cancer. I am glad now we have a world class treatment.

Dr. Ali has looked after me and my various injuries. He has looked after my sister and wife too. Few people know that when my third son, Abram, was born, he was in a very critical condition. He was rushed to the Paediatric centre here and looked after even before we could meet him. Its a strange cycle that my child was saved in the same hospital which has a ward named after my mother. I am thankful to them, Khan added. Kulkarni added, The specialisation also extends to providing advanced transplant treatments for malignant hematological disorders like acute and chronic leukemia, Hodgkins and Non-Hodgkins Lymphoma, Multiple Myeloma, Myelodysplasia,

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SRK launches BMT, Birthing Centre at Nanavati hospital - Free Press Journal

A non-surgical treatment that uses a patients own bone marrow stem cells to treat chest pain or angina improved both symptoms and the length of time treated patients could be physically active, according to recent research.

Angina is chest pain or discomfort caused when the heart does not get enough oxygen-rich blood due to narrowing or blockages in the arteries leading to the heart.

Most studies that have explored stem-cell therapies for angina required surgery to directly inject stem cells into the heart muscle or the heart blood vessels.

We injected a catalyst molecule that caused bone marrow stem cells to enter the patients blood, then harvested them to re-inject into the patient.

This is not considered a surgical procedure, is easy to implement, and allows for repeated administrations, said Hadyanto Lim, Ph.D., study senior author.

Fifteen patients were first injected with a molecule called granulocyte colony stimulating factor (G-CSF) once a day for four days.

G-CSF stimulates stem cells to migrate into the bloodstream from the bone marrow where they reside. Stem cells have the ability to transform into different types of cells.

On the fourth day, three hours after the last G-CSF injection, blood was drawn and stem cells were separated from the blood.

Stem cells were identified by the presence of a protein called CD34 on the cells surface.

Thirty minutes after the cell separation procedure finished, the collected stem cells were injected back into the patient through an IV.

Four weeks after receiving the treatment, patients experienced significantly fewer angina-related symptoms, and they were able to exercise at a higher intensity and for a longer period of time.

Most patients also reported mild muscle pains in their backs or legs, but the pain could be managed with acetaminophen.

When lifestyle changes and drug therapies do not control chest pains and discomfort, patients are often recommended for surgical procedures.

This includes coronary angioplasty in which a small mesh tube is inserted in the narrow heart artery to open it up and coronary artery bypass grafting in which healthy blood vessels are used to shunt blood around the narrowed heart arteries.

However, 20 percent to 30 percent of patients with severe coronary atherosclerosis are not suitable for these interventions.

The studys limitations are the small number of patients and absence of a control group. Because no control group was used, the placebo effect cannot be ruled out, Lim noted.

Although this treatment is currently used to treat some cancers multiple myeloma and lymphoma it will need more investigation before it can be made available to the general public to treat angina, according to Lim.

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Having chest pain? Your own stem cells may help with the treatment - Knowridge Science Report

I just read a concerning paper about an experimental stem cell treatment for children with autism.

The authors are Himanshu Bansal and colleagues of India. The senior author, Prasad S Koka, is the Editor-in-Chief of the Journal of Stem Cells where the paper appeared, which raises questions about whether the manuscript received a thorough peer review. Koka is actually an author on all five of the research papers published in that issue of the journal. But thats a minor issue compared to the content of the paper.

Bansal et al. describe a procedure in which they extracted fluid from the bone marrow of each child. This fluid (bone marrow aspirate) was treated in the laboratory to purify the stem cells within, and then injected into the childs spinal canal. The whole operation took place under general anaesthesia. 10 autistic children aged 4-12 were treated.

I found this pretty shocking. An invasive procedure involving general anaesthesia should only be performed if its medically justified especially in children as young as 4! Bansal et al. provide no scientific explanation for why they thought this treatment was suitable for these patients. They vaguely name immunological and neural dysregulation believed to underlie autism as the target of the cells.

For what its worth, the results showed a slight improvement in autism symptoms after the treatment. However, there was no control group, so placebo effects are likely, and were told that the patients were also given speech therapy, occupational therapy and psychological intervention which might account for the benefits.

So who gave the green light to this project? Well, remarkably, Bansal et al.s paper contains no information about which ethics committee reviewed and approved the study. I dont know about the laws in India, but in the UK or the USA, conducting even the most benign research without the proper ethical approval is serious misconduct. Most journals absolutely wont publish medical research without an ethics statement.

The paper also contains no mention of conflicts of interest another thing that most medical journals require. I believe that financial conflicts of interest are likely to exist in this case because Bansal gives his affiliations as Mother Cell, his own private venture, and RegennMed, who sell various stem cell treatments.

Overall, to say that this paper is ethically questionable is an understatement, and it would have been rejected by any real journal.

This isnt Dr Himanshu Bansals first foray into the amazing world of dodgy stem-cells. He briefly made headlines around the globe last year when he announced his ReAnima project to bring a brain dead woman back to life (with stem cells). Indian authorities eventually blocked his resurrection attempt. Theres some more interesting dirt on Bansal on this forum.

This is also not the worlds first stem cells for autism trial. For example, Duke University launched a $40 million trial in 2014. The treatment in that trial was a blood infusion, so it was pretty non-invasive: no bone marrow, spinal needles, or general anaesthesia. However, critics argue that its pure speculation to think that stem cells would help in autism. Then again, the same could be said about a great many stem cell therapies.

Bansal H, Verma P, Agrawal A, Leon J, Sundell IB, Koka PS. (2016). A Short Study Report on Bone Marrow Aspirate Concentrate Cell Therapy in Ten South Asian Indian Patients with Autism Journal of Stem Cells, 11 (1)

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Unethical Stem Cell Therapy for Autism In India? - Discover Magazine (blog)

Seven-month-old Madalayna Ducharme is receivingthe stem celltransplant she urgently needs to save her life, from a donor found in herWindsor home her two-year-old brother Henrik.

But her mother Tamara cautioned Friday that the hardest days start today as specialistsat Torontos Sick Kids hospital closely monitor for the next several weeks how Madalayna responds to her brothers stem cells, needed to halt the terrible effects of malignant infantile osteopetrosis.

Theone-in-200,000 genetic disorder causes bones toget thicker and more dense. She cant lift her head because its too heavy. One of the most devastatingeffects vision loss caused by thickening bones compressing nerves has already happened since her January diagnosis.

People dont get how difficult this still is, Tamara said during a phone interview Friday from Sick Kids. Once the match was found (last week) everyone was so excited. But then there was eight days of chemo, to kill off Madalaynas own stem cells and make room for those from her brother. The chemo was tough on Madalayna, Tamara said.

And that isnt even the most difficult part. We keep being told its going to get a lot harder before it gets better.

She said doctors will monitor her for signs of rejection for the next 14-27 days in Toronto. Theres a risk of graft versus host disease, which can happen if Henriks donated bone marrow thinks Madalaynas body is foreign and attacks her.

When Tamarawas interviewed on Friday, she and husband Charles were waiting while Henrik was donating his bone marrow a 3-hour procedure called harvesting. Madalayna was on Tamaras lap, waiting for the procedure that involves transfusing the bone marrow into her system, much like a blood transfusion.

The Ducharme family at their home in Windsors Riverside area on Jan. 18, 2017. From left: Henrik, Tamara, Charles, and warrior princess Madalayna. Nick Brancaccio / Windsor Star

Because hes only two, Henrik only understands that hes helping his sister, Tamara said.He was very willing, she said, because he knows she is very sick.

The family went public in January to plead for people to get swabbed tested to see it theyre a match and to register on the international bone marrow registry. About 1,500 volunteered, said Tamara.

Its phenomenal, she said of the response. Its very uplifting, it makes you feel really great that people are behind us. She said social media posts about Madalaynas progressare followed by 7,000-15,000 people.

Shes got a lot of prayers and thoughts behind her, so shes going to be OK.

Tamara said Madalaynas eyes have been affected by the disease, but doctors arent yet 100 per cent sure if shes entirely lost her eyesight a permanent effect from the disease.

Madalayna appeared perfectly healthy when she was born, but at two months, theirfamily doctor noticed herfontanel the soft spot in a babys skull was enlarged. It was the start of a long list of symptoms such as spitting up, seizures, spots on the liver and abnormal findings from blood work, ultrasounds and other tests. The definitive diagnosis came in January.

When the Ducharmes made their public appeal for people to get swabbed, the situation was an emergency due to the possible arrival of terrible complications, including lost eyesight and hearing.

Windsor is such a great community, as soon as you put a call out, people are willing to help, theyre always there in abundance, said Joanne Bedard, whose Katelyn Bendard Bone Marrow Associationorganizedlocal bone marrow registrations in Madalaynas name.

About 1,300 people registered with CanadasOne Matchstem cell and marrow registry, not including other donors who were swabbed at regular Canadian Blood Services blood donor clinics or who submitted their swab sample through the mail.

The fact we had that many people who joined the registry, it just offers so much more hope to the other over 800 Canadians who are waiting for a (stem cell) transplant, said Bedard, whose daughterKatelyn died of leukemia in 2005 when she was three, aftera bone marrow donor could not be found.

When they learned last week that Henrik was a match, doctors were initially hesitant to use him because of concerns his genetic makeup was too close to Madalaynas and could therefore share some of the diseases traits. But they ultimately decided that Henrik was the best option.

Madalayna Ducharme at her family home in Windsors Riverside area on Jan. 18, 2017. The baby warrior princess suffers from the rare disorder of malignant infantile osteopetrosis. Nick Brancaccio / Windsor Star

Friday was Day Zero for Madalayna, and the medical experts will start counting upwards from there as Henriks stem cells start to graft into her system.

Thereare a lot of things that can happen from Day Zero on up until she has accepted this bone marrow, her mother said. So were actually going to be in the roughest days starting today.

A benefit to help the family is planned for April 1 at Parkwood Gospel Temple, 3005 Temple Dr., from 4 p.m. to 8 p.m. Call 226-348-0388 for information about tickets. You can also donate to an account set up at Libro Credit Union. Go to any location and make a deposit to the Warrior Princess account. You can also make an e-transfer towarriorprincesslayna@gmail.com with the password Layna.

bcross@postmedia.com

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Prayers, hopes for Madalayna, as brother donates life-saving stem ... - Windsor Star

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Stem Cell Therapies for Degenerative Disc Disease Clinical Pain Advisor (registration) MSCs derived from bone marrow have been successfully differentiated into cardiopoietic cells and used in treatment of heart failure. Fourth- and fifth-generation techniques use genetically modified MSCs and induced pluripotent stem cells (iPSCs), ...

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3 Women Blinded After Stem Cell Therapy Newser CORRECTS FROM MD ANDERSON HOSPITAL TO MD ANDERSON CANCER CENTER -Senior Clinical Cell Therapy Specialist Megan Raggio prepares stem cells from bone marrow before they are transplanted into sportscaster... (AP Photo/David J. Phillip). These Women Went Blind After A Florida Clinic Injected Fat Cells Into Their EyeballsBuzzFeed News Florida Clinic Blinds Three Patients in Botched 'Clinical Trial'Gizmodo From Hope To Despair: Three Women Blinded By Unproven Stem Cell Therapy [VIDEO]Counsel & Heal The New England Journal of Medicine -The New England Journal of Medicine -The New England Journal of Medicine -Bascom Palmer Eye Institute all 131 news articles »

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3 Women Blinded After Stem Cell Therapy - Newser

I recently donated peripheral blood stem cells (PBSC) to a middle-aged man with myelodysplastic syndrome. This is similar medically to a bone marrow donation (though less painful) and much more involved than a blood donation (which I have done regularly since I was seventeen). I found the whole process fascinating and a testament to the glory of mans mind and modern civilization.

The patient with myelodysplastic syndrome lives in central Europe. His bodys bone marrow was no longer producing healthy functioning blood cellsthat is, red blood cells, white blood cells, and plateletsa deficiency that could have resulted in his bodys loss of ability to fight infections or control bleeding, and possibly leukemia. The cure for his disease involved destroying his defective natural bone marrow and replacing it with someone elsesmine.

Bone marrow compatibility between donor and recipient is more complicated than blood types. He needed a donor whose human leukocyte antigen (HLA) proteins most closely matched his own in order to minimize the chance of graft-versus-host disease. His doctors found my data in the Be the Match Registry, where Im registered as a potential donor, and they judged my HLA proteins to be his best hope.

Eight weeks before the operation would take place, I was notified by phone about the match and the donation process. I was then asked whether I was willing to donate. I said yes (and was given several opportunities later to change my mind). In the following weeks, I provided two sets of blood samples to verify that I was healthy enough to donate and still a good match. I was flown out to the donation facility in Michigan to be examined physically, preview the process, and speak with the doctors and nurses who would collect the donation. My donor representative called me periodically to keep me informed and to verify my continuing consent. She also made the arrangements for collecting the samples, managed my travels, and ensured that my expenses were covered.

Here I am holdingthe final product just prior to its transportation to the recipientin central Europe.

As the donation date drew closer, I received ten shots of Filgrastim, a drug designed to stimulate additional blood stem cell formation, one shot in each arm for five days. This increased my white blood cell count far above normal and forced extra blood stem cells into my bloodstream, thus enabling the technicians to run my blood through an apheresis machine, which separated the phases of blood by density using centripetal acceleration. On the donation day, I sat in a comfy chair with an IV in each arm for four hours as a machine took blood from one arm, separated out the stem cells, and returned the rest of my blood via my other arm. While the process continued throughout the morning, the nurses took a few notes here and there, and, as my arms couldnt bend, fed me lunch (chicken wings from Jets Pizza). Once the machine had collected enough stem cells for the recipient (Im fifteen pounds heavier than he is, so it was easier on my body than it could have been), the IVs were removed, my blood was tested one final time to make sure I was OK to drive home, and I left.

My blood stem cells were then transported by private courier to the patient in central Europe. In preparation for the donation, his entire immune system and blood-producing machinery (bone marrow) had been destroyed using myeloablative chemotherapy in order to eliminate any remaining diseased cells and to suppress any immune response from his body to my replacement tissue. My blood stem cells were injected into his bloodstream by IV and then migrated to his bones to replace his destroyed bone marrow and eventually start producing new red blood cells, white blood cells, and platelets. Essentially, my blood and my immune system are regrowing in his body. With these, he inherits my allergies and infectious disease history, and, if all goes well, my life force for another few decades.

Although the organization through which I donated does not pay for stem cells (because payment is against international registry standards), I was treated well and fully reimbursed for expenses. They paid for flights, a half dozen meals, a private driver at one point, hundreds of miles of my own driving, and my stays at nice hotels.

It is worth noting that the Institute for Justice (IJ) recently sued the U.S. attorney general to legalize bone marrow and stem cell donor compensation.1 As the IJ reports, the Ninth Circuit ruled in our favor, holding that the National Organ Transplant Acts ban on donor compensation does not apply to the most common method for donating marrow. This victory is especially helpful for certain minorities and people with multiracial ancestries who face significantly reduced odds of finding unrelated marrow donors. But direct compensation has been met with strong resistance by the major national and international marrow registry organizations, which also lobbied against IJs efforts in court.2 Currently, compensation for donations is being offered only by smaller organizations.

My motivation for donating cannot be reduced to just one reason, but it certainly was not a sacrifice. My reasons varied in depth and weight, but all were self-interested. I thought the process itself was fascinating. I was able to ask the doctors and nurses unlimited questions and to experience firsthand a medical procedure about which I had no previous knowledge. I enjoyed business-class travel, which, as a college student was a significant treat. Most broadly, I participated in an important aspect of the kind of civil society in which I want to live. I want someone to be willing to donate lifesaving tissue to me or my loved ones, should we need it in the future, and I was happy to donate first. The costs were trivialabout twenty-five hours of volunteered time and some minor discomfort. Overall, the experience was positive and spiritually rewarding.

The option to make a donation of this kind did not even exist a few decades ago. It is a function of many interrelated parts of todays modern, relatively free-market, science-oriented cultures. The establishment and maintenance of an international donor registry requires stable, relatively rights-protecting legal systems that enable long-range and large-scale planning among cooperative strangers. To find matches in a timely manner requires the speed and integrating capacities of computers and the Internet. The medical procedure itself requires the kinds of scientific knowledge and expensive technologies made possible by todays relatively free markets. The ability to pay for such a procedure requires substantial personal wealth, which more people have today than ever before. I am exceedingly grateful to live in our rich, science-oriented, relatively capitalist civilization at the time that I do. And I hope the recipient of my donation is able to enjoy many years more of living and loving life as I do.

Related:

Endnotes

1. Bone MarrowNOTA Challenge, Institute for Justice, http://ij.org/case/bonemarrow/.

2. Coalition Says PBSC Donor Compensation Poses Health Risks to Patients and Donors, Be the Match, February 2, 2012, https://bethematch.org/news/news-releases/coalition-says-pbsc-donor-compensation-poses-health-risks-to-patients-and-donors/.

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My Non-Sacrificial Donation of Stem Cells to Save a Life - The Objective Standard

A retina with macular degeneration. (Image: University of Iowa)

An unproven stem cell therapy conducted by a Florida clinic has blinded three patients in an apparent clinical trial gone horribly wrong. The incident showcases the extent to which unscrupulous clinics will take advantage of desperate patientsand how the lack of government oversight contributes to the problem.

As reported in the New England Journal of Medicine, the clinical trialif it can be called thatinvolved three women between the ages of 72 and 88 who were suffering from macular degeneration, a common progressive disease of the retina that leads to loss of vision. The women, all of whom were experiencing various degrees of vision loss, sought the help of a Florida clinic, which claimed to be testing a stem cell procedure designed to treat macular degeneration. Sometime in 2015, a week after stem cells were injected into their eyes, the women became blind. Two years later, doctors say theres virtually no chance the womens vision will be restored.

The authors of the new report, ophthalmologists Jeffrey Goldberg from Stanford University School of Medicine and Thomas Albini from the University of Miami, said the unfortunate incident serves as a call to awareness for patients, physicians and regulatory agencies of the risks of this kind of minimally regulated, patient-funded research.

Stem cells are undifferentiated cells that havent quite decided what they want to be when they grow up. Under the right conditions, these immature cells can be transformed into virtually any kind of cell found in the body, which is why theyve proven useful in regenerative medicine.

Eventually, scientists hope to be able to use stem cells to regenerate damaged tissue and organsand possibly even repair the effects of macular degenerationbut were not there yet. The only truly effective clinical application of stem cells to date has been in bone marrow transplants, in which stem cells extracted from a donors bone marrow are used to produce a fresh blood system for patients suffering from blood disorders such as leukemia. A recent study showed that there are nearly 600 clinics peddling unproven stem-cell procedures in the United States for a wide range of conditions, including arthritis, autism, cerebral palsy, stroke, muscular dystrophy, and cancer.

As noted in the NEJM report, two of the three patients learned about the stem cell trial for macular degeneration on ClinicalTrials.gov, a registry run by the US National Library of Medicine. The listings on this site arent fully scrutinized for scientific efficacy. The patients were reportedly under the assumption that they were participating in a bonafide clinical trial, but the consent form and other materials made no mention of a trial. Tellingly, each patient had to pay $5,000 for the procedure. This is highly unorthodox for a clinical trial, and it should have been cause for alarm. Im not aware of any legitimate research, at least in ophthalmology, that is patient-funded, Albini said in a statement.

The NEJM study didnt identify the Florida clinic responsible, but (conveniently) the authors provided the name of the trial: Study to assess the safety and effects of cells injected intravitreal in dry macular. A quick Google search calls the trial up, along with the name of the company responsible: Bioheart Inc., otherwise known as US Stem Cell. As the ClinicalTrials.gov page indicates, the study has been withdrawn prior to enrollment. According to Goldberg and Albini, the company is no longer performing the procedure, but it is still seeing patients.

The trial itself was a joke, lacking in all the components of a properly designed test. It wasnt based on prior laboratory experiments, no control group was assigned, no data was collected, and no plans were made for follow-ups.

During the procedure, the patients had some of their fat cells (i.e. adipose tissue) removed, along with a standard blood withdrawal. The fat tissues were then processed with an enzyme to draw out stem cells. Once plasma was isolated from the blood and added to the stem cells, the mixture was injected into both eyes of each patientyes, both eyes. Again, another serious clinical no-no; normally, only one eye would be injected for an experimental procedure like this in the event that something should go wrong. The entire procedure lasted less than an hour.

A week later, all three women were blind. As noted in the NEJM report, the blindness was accompanied by detached retinas and hemorrhaging.

The patients severe visual loss after the injection was associated with ocular hypertension, hemorrhagic retinopathy, vitreous hemorrhage, combined traction and rhegmatogenous retinal detachment, and lens dislocation. After one year, the patients visual acuity ranged from 20/200 to no light perception.

Goldberg and Albini say the preparation of the stem cells was likely shoddy, and the injections may have been contaminated. Once in the eye, the stem cells could have changed into myofibroblasts, a type of cell associated with scarring.

The Florida clinic, it would appear, was appealing to the desperation of their patients, while taking advantage of a regulatory loophole. As the authors write in their report:

Adipose tissuederived stem cells have been increasingly used by stem-cell clinics because of the relative ease of obtaining and preparing these cells. Many of the clinics that provide these stem-cell therapies have done so under the auspices of patient-funded, institutional review boardapproved research, and the research is listed on ClinicalTrials.gov without an investigational new drug filing with the FDA.

At the time, the procedure was not subject to FDA approval because the cells werent transferred between patients, and because the cells were considered minimally processed. The FDA has since revised its requirements, and it now needs approval for these types of procedures. In addition to updating its regulations, the FDA is also clamping down on stem cell clinics.

Thats obviously a good thing, but its a little too late for the women involved. This incident shows what happens when regulations and oversight are weak, and how shady companies will take risks with their patients health. Certainly food for thought as Trump and his cronies start to recreate the FDA in their own image.

Update: We reached out to US Stem Cell Clinic for comment and they responded with this statement:

Founded in 1999, U.S Stem Cell, Inc. has been committed to the research and development of effective cell technologies to treat patients with a variety of diseases and injuries. Since 2001, our clinics have successfully conducted more than 7,000 stem cell procedures with less than 0.01% adverse reactions reported. We are unable to comment further on specific cases due to patient confidentiality or legal confidentiality obligations. Neither US Stem Cell nor US Stem Cell Clinic currently treats eye patients.

[New England Journal of Medicine]

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Florida Clinic Blinds Three Patients in Botched 'Clinical Trial' - Gizmodo

Runner's World

Stem Cell Therapy Runner's World This is why researchers and physicians think this therapy may help joint injuries caused by worn-out cartilage; in cell cultures, stem cells can grow new cartilage, and if this can happen in a joint, it may prevent the need for a joint replacement ... Nutrients Boost Stem Cell FunctionProHealth all 19 news articles »

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Stem Cell Therapy - Runner's World

Adam Krief, with his wife, Lia, had a rare form of blood cancer that proved to be fatal. (Facebook)

(JTA) Adam Krief, a Jewish cancer patient whose search for a bone marrow donor captured the attention of social media and celebrities including Kim Kardashian, Mayim Bialik and Jason Biggs, has died.

Krief, a father of three from Los Angeles, died Tuesday, a family friend confirmed to JTA. He was 32.

Krief was diagnosed with primary myelofibrosis, a rare form of blood cancer that is likely fatal if a stem celltransplant matchis notfound.To find anHLA, or gene complex matchfor Krief something more difficultto track downthan a blood type match drives were held around the world, including in North America,Israel, France and Mexico.

Kardashian posted about Krief on Facebook in September, saying he was a friend of a friend.

A bone-marrow donor was found last December seven matches were found, in fact, through the donor drives organized for him.

This is what cloud 9 looks like Im so grateful to let you all know that a donorhas been found, Krief wrote at the time, sharing a video with two of his children.

The Hope 4 Adam Facebook page on March 8 called for a Worldwide Unity Shabbat for March 11 and March 18 for the recovery of Krief, asking followers to Help us bring about a miracle.

On Monday, the Eretz Kabbalah Facebook page of the Los Angeles-based Eretz Cultural Center posted a call for followers to recite Tehillim, or psalms, on behalf of Krief.

After a long search for a bone-marrow match to save his life, he finally received one. However, after some complications, he is said to only have a few hours to live, the post said.

Krief is survived by his wife, Lia, and his young children.

RELATED:

Jewish cancer patient finds bone marrow transplant following worldwide search, Kim Kardashians pitch

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Adam Krief, Jewish father of 3 whose bone marrow search inspired celebrities, dies - Jewish Telegraphic Agency

"The impact of Alzheimer's disease is vast, far exceeding the medical community's current ability to treat it," said Joshua M. Hare, M.D., Longeveron's Co-Founder and Chief Science Officer. "Regenerative medicine and cell-based therapies offer a promising new approach to close this gap and address the urgent need for effective therapies to combat the condition."

An important component in the progression of Alzheimer's disease is neuroinflammation. Longeveron was recently awarded a $1 million Part the Cloud Challenge on Neuroinflammation grant from the Alzheimer's Association to help support this research.

"Adult stem cells are very potent anti-inflammatories. The characteristic amyloid plaques found in the brains of Alzheimer's disease patients produce inflammation, and stem cells can reduce inflammation," explained Bernard S. Baumel, M.D., Principal Investigator for the trial. "Alzheimer's also impairs the brain's ability to adequately produce new brain cells in the memory area known as the hippocampus. Stem cells can stimulate the brain to produce these new cells needed to form memory. We believe that an infusion of LMSCs may improve the condition or at least halt the progression of the disease."

Prior research shows that adult MSCs target and reduce inflammation, promote tissue repair and improve brain function in mouse models of Alzheimer's disease. Longeveron's trial is the first U.S. clinical study of exogenously administered mesenchymal stem cells derived from the bone marrow of healthy adult donors for treating Alzheimer's disease.

To learn about participating in the clinical trial, visit: https://clinicaltrials.gov/ct2/show/NCT02600130

About Longeveron

Longeveron is a regenerative medicine therapy company founded in 2014. Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company's research focus areas include Alzheimer's disease, Aging Frailty and the Metabolic Syndrome. Longeveron produces LMSCs in its own state-of-the-art cGMP cell processing facility. http://www.longeveron.com

Contact: Suzanne Liv Page spage@longeveron.com 305.342.9590

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/longeveron-achieves-milestone-in-groundbreaking-stem-cell-trial-for-alzheimers-disease-300424206.html

SOURCE Longeveron

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Longeveron Achieves Milestone in Groundbreaking Stem Cell Trial for Alzheimer's Disease - PR Newswire (press release)

Adam Krief, a Jewish cancer patient whose search for a bone marrow donor captured the attention of social media and celebrities including Kim Kardashian, Mayim Bialik and Jason Biggs, has died.

Krief, a father of three from Los Angeles, died Tuesday, a family friend confirmed to JTA. He was 31.

Krief was diagnosed with primary myelofibrosis, a rare form of blood cancer that is likely fatal if a stem cell transplant match is not found. To find an HLA, or gene complex match for Krief something more difficult to track down than a blood type match drives were held around the world, including in North America, Israel, France and Mexico.

Kardashian posted about Krief on Facebook in September, saying he was a friend of a friend.

A bone-marrow donor was found last December seven matches were found, in fact, through the donor drives organized for him.

This is what cloud 9 looks like Im so grateful to let you all know that a donor has been found, Krief wrote at the time, sharing a video with two of his children.

The Hope 4 Adam Facebook page on March 8 called for a Worldwide Unity Shabbat for March 11 and March 18 for the recovery of Krief, asking followers to Help us bring about a miracle.

On Monday, the Eretz Kabbalah Facebook page of the Los Angeles-based Eretz Cultural Center posted a call for followers to recite Tehillim, or psalms, on behalf of Krief.

After a long search for a bone-marrow match to save his life, he finally received one. However, after some complications, he is said to only have a few hours to live, the post said.

Krief is survived by his wife, Lia, and his young children.

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American Jewish father of 3 whose bone marrow search inspired celebrities dies - Jerusalem Post Israel News

A longtime product developer is bringing a peptide ingredient to the US market that has been researched for a unique property promoting the growth of bone marrow stem cells.

Called DH Stemogen, the product is the brainchild of Dr Marvin Heuer MD who has a history of product development with sports nutrition company MuscleTech. Dr. Heuer has a background in clinical research, having spent many years in drug development at Glaxo Smith Kline. He also runs a contract research firm, Heuer M.D. Research Inc. and is the CEO of omega-3 supplement manufacturer Blue Ocean Nutrascience.

The new product, called DH Stemogen is based on a Cyclo-{L-ALA-L-GLU(TRP-OH) peptide that was developed by a Russian biochemist.

Its a peptide that is a mimic of a naturally occurring thymic peptide,Dr. Heuer told NutraIngredients-USA. Heuer was promoting the launch of the product at the recent Expo West trade show in Anaheim, CA. At Heuer M.D. Research, as a company we are out looking for novel ingredientsto bring out, hopefully in the nutraceutical area.

We got interested in Prof. Vlad Deigins peptide research, Dr. Heuer said (Deigin is associated with the Institute of Bioorganic Chemistry at the Russian Academy of Sciences in Moscow.)We looked at this particular compound that he was launching as an ingredient in Russia about a year ago.

The peptide in DH Stemogen targets a particular type of stem cell hematopoietic cells (HSC). Stem cells in general are the building blocks of our bodies. These cells are able to transform themselves into almost any type of cell. There are various sources of stem cells in an adult body. One of the most important of them comprises the bone marrow, where the HSCs are produced. HSCs transform into all the main cell types in our blood, including red blood cells and white blood cells. Dr. Heuer said there is some evidence that those cells are able to reconstruct other body tissues by transforming into the specific tissue type cell such as liver, nervous tissue, kidney and skin.

These properties would seem to make Stemogen a natural for a healthy aging product positioning, Dr. Heuer said. But Deigins research, trending as it does over into disease endpoints, is a little problematical when it comes to supporting US-style structure function claims, he admitted. Other countries dont make the same hard and fast distinctions between dietary ingredients meant for supplement applications and active pharmaceutical agents meant for drugs, he said.

We are going to be very cautious about making structure/function claims,Dr. Heuer said. The product at the moment saysSupport your immune system and Support healthy levels of stem cells in your blood.

We are about to begin a whole profile of research in the U.S. and Canada, he added.

Dr. Heuer said one thing thats unique about the ingredient (and something that he says Deigin has patented) is a structural twist that improves the peptides stability. The criticism of some other novel peptides has been that interesting as their properties might be, once they hit the stomachs gastric fluid they blow apart into their constituent amino groups and all those novel properties are lost.

He has a patent on the way he makes this with a hex ring on the end that protects it in the GI tract and allows it to be absorbed,he said.

Bringing a synthetic analogue of a naturally occurring peptide to market as a dietary ingredient would seem to pose significant regulatory challenges. Dr. Heuer said hes confident there is a way through that thicket. The plan is to start first with a GRAS filing, and Dr. Heuer said he believes that the peptide would fall under the amino acid category in the DHSEA definitions of what constitutes a dietary ingredient.

Certainly there is a precedent of complex peptides being sold on the market, he said.

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Peptide aimed at stem cell genesis debuts on supplement market - NutraIngredients-usa.com

March 13, 2017 6:01 PM By Stephanie Stahl

PHILADELPHIA (CBS) Doctors at the University of Pennsylvania School of Veterinary Medicine are conducting a study to see if stem cell therapy will ease the pain of arthritis and the results of their research could benefit human patients as well.

Its Zoeys last check up,walking on a special mat called a forceplate to measure how much weight she puts on each leg.

It was just a year ago that putting weight on her front legs was painful.The 2-year-old Golden Retriever was diagnosed with elbow dysplasia, a condition that created arthritis in both elbows.

It is the most common cause of chronic pain in dogs, saidDr. Kimberly Agnello at Penn Vet.

Zoeys owner, Christine Brown, says she was a bundle of energy when she first got Zoey.

She was so sweet, said Brown. She was your typical energetic puppy.

But soon Brown knew her dog was hurting.

After coming back from a walk and taking a nap, she would get up and limp, said Brown. With her being a puppy it was devastating.

Zoey was enrolled in aPenn Vet trial to determine the benefits of stem cell therapy as a treatment to ease arthritic pain.

They are randomized into three groups, whether they receive an interarticular joint injection of hyaluronic acid or they geteither stem cells derived from their bone marrow or stem cells derived from fat, saidAgnello.

The stems cells from the dogs bone marrow are injected back into the elbow joint. Doctors hope it will relieve the arthritic pain.

We also remove a little fragment of bone that can be causing some more pain, saidAgnello.

The research isnt just about arthritis in dogs but humans as well.

The goals of this study are to look for different treatments to not only help our canine patientsbut also to help human patients with arthritis, saidAgnello.

For now results are promising.

Oh my gosh, she is not limping, she runs and jumps, and has a great time, said Brown.

The trial is ongoing so there is no hard data yet to show final results if stem cells are effective for treating arthritis, but Dr.Agnello says there are many dogs in the study and almost all of them have improved during the year-long research.

Stephanie Stahl, CBS 3 and The CW Philly 57s Emmy Award-winning health reporter, is featured daily on Eyewitness News. As one of the television industrys most respected medical reporters, Stephanie has been recognized by community and he...

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Veterinary Doctors Conduct Study Looking To Ease Arthritis Pain - CBS Philly

TiGenix announces positiveone-year results forits phase I/II trial of donor-derived cardiac stem cell therapy in acute myocardial infarction (AMI).

The Belgian biotech TiGenixis developing allogeneic stem cell therapies. Now the companyhasannouncedthat its cardiac stem cell therapyAlloCSC-01 reached its primary endpoints in aphase I/IItrial.

In 2015, the companyacquired Coretherapixin a292M deal for its allogeneic cardiac stem cell pipeline, which is being developed for the treatment of AMI.The first-in-human trial was designed to test the safety and feasibility of an intracoronary infusion of donor-derivedexpanded cardiac stem cells (AlloCSCs)in patients with AMI and left ventricular dysfunction.

AlloCSC-01consists of adult allogeneic cardiac stem cells isolated from the heartof donors and expanded in vitro. In vivo studies suggest that these cellshave cardio-reparative potential by activating regenerative pathways and promoting the formation of new hearttissue.

Thecurrent phase II study demonstrated thesafety of these allogeneic stem cells. Initial results also revealed a larger reduction of infarct size in a subgroup of patients.

Myocardial infarction caused by blockade of coronary arteries

TiGenix is well known forChondroCellect, which was the first cell therapyto reach the European market for the repair of knee cartilage.After the companyrecently withdrew its market authorization for this product, due to a lack of reimbursement, the biotech is focusing on another stem cell therapy, Cx601, in addition to AlloCSC-01. Under development for Crohns disease, Cx601 is currently awaitingEMA approval and is in phase III trials in the US.

For a late-stage clinical company, TiGenix has a low market cap of191M. Even so, the company seems to be doing well these days with the progress of Cx601 and AlloCSC-01.

If AlloCSC-01 obtains market approval, it could treat the more than 1.9 millionpeople affected by AMI, a major cause of heart failure. So far, most treatments are palliative or restore myocardial function by angioplasty and insertion of a stent to support the vascular lumen.

Stem cell therapy of the heart is definitely not a new topic, but many trials have been conducted using the patients own stem cells derived from the bone marrow. A recent meta-analysisof such trials has suggested that these therapies are safe, but do not enhance cardiac function. TiGenixs approach using allogeneic heart-derived stem cells may offer a new and promisingopportunity in thefield.

Images via shutterstock.com / Liya Graphics andVeronika Zakharova

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New Cardiac Stem Cell Therapy passes Phase I/II Trials - Labiotech.eu (blog)

On March 8-9 in Boston, stem cell medicine biotechnology start-up Asymmetrex led attendees at the 6th Annual Clinical Trials Supply New England 2017 conference in discussions about the need for quality controls for the supply of tissue stem cells used for treatments in either FDA-approved clinical trials or unregulated private stem cell clinics. Though these two stem cell treatment settings are often contrasted regarding their safety and effectiveness, Asymmetrex stressed that patient care and research progress is compromised in both because of the lack of essential quality control tests for the number and quality of transplanted tissue stem cells.

Boston, MA (PRWEB) March 14, 2017

At the 6th Annual Clinical Trials Supply New England 2017 conference, held in Boston from March 8-9, James Sherley, M.D., Ph.D., director of Asymmetrex, led discussions that evaluated the quality of U.S. supplies of stem cells used in clinical trials compared to private stem cell clinics. Private stem cell clinics have been criticized for not employing research standards that are necessary to establish the therapeutic effectiveness of treatments with statistical confidence. In part because of this difference in practice, they are also often accused of making unproven claims about the effectiveness of their therapies.

Sherley presented comparisons of key operational elements to argue that, given good intent in both settings, the two different settings of stem cell treatments had both distinct and shared shortcomings. He noted, however, that the most significant shortcoming, which stem cell clinical trials and private stem cell clinics share, was perennially overlooked.

Based on the number of reported stem cell clinical trials and private stem cell clinics, Sherley estimated that close to a quarter-million patients in the U.S. now receive stem cell treatments each year. Though many of these occur within FDA-approved clinical trials, their number is dwarfed nearly 10 times by the number of treatments that occur in private stem cell clinics. It shocked the audience of clinical trial suppliers to learn that there was no stem cell quality control test performed for any of these many treatments.

Even for approved stem cell medicine treatments like bone marrow transplantation and umbilical cord blood transplant, there is no stem cell-specific quality control test available. Counts of total cells are made, but these do not adequately predict stem cell number or function. Biomarkers designated for tissue stem cells are also expressed by stem cells' more abundant non-stem cell products. So, the biomarkers lack sufficient specificity to be used to count and monitor tissue stem cell function.

Without a quality control test for tissue stem cell number, stem cell treatments in all settings proceed without knowing the dose of treating tissue stem cells. This previously unavoidable therapeutic blind spot creates an instant treatment risk. It also precludes effective analyses to optimize treatment procedures, to compare different treatments, or to relate treatment outcomes to tissue stem cell dose. Without knowing stem cell dose, the interpretation of any stem cell treatment in terms of stem cells as the responsible agents is compromised.

In this context, Sherley announced briefly to attendees that Asymmetrex's new AlphaSTEM Test for counting adult tissue stem cells and providing data on their viability and tissue cell renewal function represented the needed first quality control test for tissue stem cell treatments, whether in clinical trials, in private stem cell clinics, or approved therapies. In particular, he indicated that both stem cell treatment patients and progress in stem cell medicine would benefit from existing clinical trial supply companies developing into future private stem cell clinic supply companies to insure the quality of stem cell treatment preparations. Sherley said that, of course, their partnership with Asymmetrex to implement its new stem cell-specific quality control test was an all around best solution for accelerating progress in stem cell transplantation medicine.

About Asymmetrex

Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. Asymmetrex's founder and director, James L. Sherley, M.D., Ph.D. is an internationally recognized expert on the unique properties of adult tissue stem cells. The company's patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing induced pluripotent stem cells for disease research purposes. Currently, Asymmetrex's focus is employing its technological advantages to develop and market facile methods for monitoring adult stem cell number and function in stem cell transplantation treatments and in pre-clinical assays for drug safety.

For the original version on PRWeb visit: http://www.prweb.com/releases/2017/03/prweb14146903.htm

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At 6th Annual Clinical Trial Supply New England 2017 Conference in Boston Asymmetrex Introduces A First Specific ... - Benzinga

Ottawa Sun

'Butterfly Boy' steels himself for second stem-cell transplant | Ottawa ... Ottawa Sun Bracing for his second stem-cell transplant in seven months, Jonathan Pitre knows all too well the mountain in front of him, its hardships and precipices. and more »

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'Butterfly Boy' steels himself for second stem-cell transplant | Ottawa ... - Ottawa Sun

SPOKANE, Wash. - A simple cheek swab and a few minutes of your time could save the life of someone in need of a bone marrow transplant.

In 2015, 34-year-old Danielle Vaughan had a stem cell transplant to save her life. For nearly a decade she suffered from mysterious symptoms and illnesses.

"So I had these spin and brain lesions that were very scary," said Danielle Vaughan of Spokane. "I had seizures. I was seeing doctors at Stanford, University of Washington and in Spokane, trying to figure out what was going on."

Eventually, doctors diagnosed Vaughan with Common Variable Immune Deficiency. Her sister, Dina Medin, also had the disorder and underwent a bone marrow transplant a few years ago. The sisters are two of six people in the world with CVID who have had transplants.

"All other treatment options had failed. There was nothing else, that was the only option," said Vaughan.

In September of 2015, Vaughan traveled to Seattle to prepare for the transplant. Vaughan's medical team turned to Be The Match, a national bone marrow donor registry to find her a match. Vaughan said 44 people came back as a perfect match. Her donor was a 27 year old man from the United States. She'd love to meet him one day.

"I would like to say thank you for really giving me a second chance at life. But mostly giving me the opportunity to watch my kids grow," said Vaughan.

Vaughan is now encouraging others to join Be The Match.

"You don't know who you could be helping and you are going to save that person's life," said Vaughan.

The Dairy Queen in Post Falls at 3560 E Seltice Way is hosting a Be The Match donor sign up on Thursday, March 16th from 1:45 p.m.- 4:00 p.m. To register all you need to do is fill out paperwork and swab the inside of your cheek with a special Q-tip.

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Bone marrow recipient encouraging others to "Be the Match" - KXLY Spokane

Globalnews.ca

Stem cell swabbing for cancer-stricken Edmonton boy so successful it ran out of kits Globalnews.ca A Thursday evening stem cell swab event aimed at finding a match for an eight-year-old Edmonton boy with leukemia was so successful, it ran out of kits. Brady Mishio has acute myeloid leukemia, a cancer of the blood, and has undergone three rounds of ...

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Stem cell swabbing for cancer-stricken Edmonton boy so successful it ran out of kits - Globalnews.ca