Tag: bone marrow

Bioethics News

The situation of the clinical use of stem cells

In January 2017, an article was published in JAMA (The Promise of Palliative Care – Translating Clinical Trials to Clinical Care) which, in our opinion, summarises the situation of the clinical use of stem cells extremely well.

It begins by referring to the possible sources of stem cells: embryonic, adult and induced, the latter known as iPS cells.

Stem cells clinical use

It continues by saying that stem cells have many clinical applications, especially stem cells from bone marrow; these are used particularly in haematological diseases, but also in bone fractures, retinal diseases, spinal cord injuries, Parkinson’s and Huntington’s disease, and in myocardial infarction, although many of these treatments are still in the experimental phase.

While stem cells might sooner or later help to treat many patients, they can also have negative side effects if not used correctly, as these cells can emigrate to other parts of the body and create problems, as well as causing tumors.

Finally, the authors refer to the proliferation of clinics offering stem cell treatments, warning that many of these do not meet the necessary quality requirements, since they are not supervised by the health control agencies of the different countries and, as such, they offer the possibility of remarkable cures that are not yet medically proven. Cosmetic treatments and treatments for arthritis and autism are particularly sought after.

See our Special Report about this matter.

La entrada The situation of the clinical use of stem cells aparece primero en Bioethics Observatory.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

First cell gene therapy to treat “Bubble boy” disease has been legalised in Europe

Last July, an article was published in medical journal Blood, which described the success of a cell gene therapy modality to treat the immunodeficiency caused by adenosine deaminase deficiency – “Bubble boy” disease. This is a disease that alters purine metabolism, leading to an accumulation of metabolites that are toxic for lymphocyte development. As a result, the immune defences of these patients are much weakened, so they often suffer serious infections. It can also be associated with cognitive and hearing deficits.

Now, a group of researchers from Ospedale San Raffaele, Milan (Italy) and the Telethon Foundation, in collaboration with pharmaceutical giant GSK, have developed their product “Strimvelis”, which is the first gene therapy to use genetically modified stem cells. It is also the first product approved by the European Commission to treat the aforementioned immunodeficiency.

The medication is generated from a gene modification of the patient’s own stem cells. The defect that causes the disease is corrected in the cells, which are then injected into the patient via intravenous infusion so that they can henceforth colonise their bone marrow.

As they are genetically modified cells from the patient himself, and not from a third party, the risk of graft-versus-host disease is drastically reduced, which is clearly an unquestionable medical advantage.

Authorisation was obtained from the European Commission after assessing Strimvelis in 18 children, in whom a 3-year survival rate of 100% was found. This opens a promising path for application of this medication in patients with adenosine deaminase deficiency.

From an ethical point of view, there is no difficulty in the use of Strimvelis, provided that the necessary requisites for implementing a new cell therapy are met since the genetic modification is performed in the patient’s own adult stem cells.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Equipoise and Caution Regarding “Ethical” Stem-Cell Therapy

You may have seen one of the many news reports this week about an “adult” stem cell treatment gone bad.  In it, doctors, not working in regulated industry or in the bounds of a clinical trial, injected stem cells derived from a person’s fatty tissue into the eyeballs of three people in an attempt to treat a vision-destroying condition called macular degeneration—and all three lost what remaining vision they had.  T

In a separate experience, two people received “reprogrammed” stem cells, also called “induced pleuripotent stem cells” or “iPSCs,” for the same condition, and they did not appear harmed, and may have been showing early signs of improvement, one year later.  In this second case, the cells are described as having undergone rigorous quality testing before being injected into the patients’ eyeballs.

The two cases are described in this week’s New England Journal of Medicine.  The links above are to those reports; subscription may be required to read them.  NEJM makes some of its editorials and perspective pieces generally available, other articles not.  Two such articles, one from FDA and one from a physician at Boston Childrens’ Hospital, address these cases.  In both opinion articles, the authors argue that careful regulation and quality control of stem cell studies is critical to their ethical testing.

To say this position is hard to argue with is a gross understatement.  I will confess that on this blog, in the past, I have wondered whether some use of “adult” or “somatic” stem cells is over-regulated, as, for example, when such cells are obtained from a woman’s fatty tissue, and reinjected into her in an attempt at breast reconstruction after surgery. 

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

The Value of Oversight in a Century of Promise & ‘Cures’

This post, by CEC member Paul McLean, originally appeared on WBUR’s CommonHealth blog.
The blood-thinner heparin is not a 21st-century cure. It was discovered 100 years ago by a scientist looking for something else entirely, and is one of the oldest drugs still in regular use.
After my daughter was diagnosed with a potentially fatal blood disorder, heparin played a key daily role in her treatment. We’d wash our hands meticulously, lay out gloves and antiseptic wipes, saline flushes for the access lines to her fragile immune system, and finally the sealed heparin syringe.
For many months, we went through boxes of heparin and never questioned its safety. Never had reason to.
But in 2008, after my daughter was officially declared cured and we’d used heparin for the last time, contamination in the supply from China killed 19 Americans and harmed many others. Writing that sentence still gives me the chills. My daughter survived thanks to medicine, but it also could have killed her.
So you can understand why, as the 21st Century Cures Act sailed to passage, I experienced both excitement and dread.
That heparin contamination was in part due to lax oversight of the drug supply chain. It reminds me why I do not want to see the work of the Food and Drug Administration compromised by overeager drug companies taking advantage of the hopes of desperate patients, and taking shortcuts on safety.
The “giant piñata” of a bill, as science blogger Derek Lowe aptly described the 21st Century Cures Act, is destined to explode in unexpected ways.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

Researchers Develop New Stem Cell Technique for Bone Repair

February 9, 2017

(UPI) – Researchers from Ulsan National Institute of Science and Technology in South Korea, or UNIST, have developed a method of repairing bone using a mix of stem cells and a carbon material with photocatalytic properties. Prior studies have used human bone marrow-derived mesenchymal stem cells, or hBMSCs, to treat fractures because of their potential to regenerate bone in patients who have lost large areas of bone from disease or trauma.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

First Stem Cell Study Could Lead to Development of Therapy to Reduce Inflammation Caused by CF

February 1, 2017

(News-Medical) – A 39-year-old man with cystic fibrosis (CF) made history by becoming the first person to receive human adult stem cells in a new research study that researchers hope will someday lead to the development of a therapy to reduce the inflammation and infection caused by CF. The pioneering subject in the study is Bob Held from Alliance, Ohio, who on Jan. 26 received an infusion of cells called allogeneic human mesenchymal stem cells (hMSC), adult stem cells collected from the bone marrow of healthy volunteers. Mr. Held was diagnosed with CF when he was 16 months old.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

New Stem Cell Method Could Lead to Powerful Treatments for Skeletal System Injuries

January 25, 2017

(News-Medical) – A recent study, affiliated with UNIST has developed a new method of repairing injured bone using stem cells from human bone marrow and a carbon material with photocatalytic properties, which could lead to powerful treatments for skeletal system injuries, such as fractures or periodontal disease. This research has been jointly conducted by Professor Youngkyo Seo of Life Sciences and Dr. Jitendra N. Tiwari of Chemistry in collaboration with Professor Kwang S. Kim of Natural Science, Professor Pann-Ghill Suh of Life Sciences, and seven other researchers from UNIST.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Find and Replace: DNA Editing Tool Shows Gene Therapy Promise

Caption: This image represents an infection-fighting cell called a neutrophil. In this artist’s rendering,  the cell’s DNA is being “edited” to help restore its ability to fight bacterial invaders.
Credit: NIAID, NIH

For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly.

While the research is just getting under way, progress is already being made for a rare inherited immunodeficiency called chronic granulomatous disease (CGD). As published recently in Science Translational Medicine, a team of NIH researchers has shown with the help of the latest CRISPR/Cas9 gene-editing tools, they can correct a mutation in human blood-forming adult stem cells that triggers a common form of CGD. What’s more, they can do it without introducing any new and potentially disease-causing errors to the surrounding DNA sequence [1].

When those edited human cells were transplanted into mice, the cells correctly took up residence in the bone marrow and began producing fully functional white blood cells. The corrected cells persisted in the animal’s bone marrow and bloodstream for up to five months, providing proof of principle that this lifelong genetic condition and others like it could one day be cured without the risks and limitations of our current treatments.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

She aborted her child to save her brother’s life. A paradigmatic case of medical and ethical manipulation

pdfCan bone marrow really not be obtained from a pregnant woman? Because if it can, there would be no medical reason to have to resort to abortion.

Yang Li’s brother urgently needed a bone marrow transplant to treat his disease. Yang Li, who was 12 weeks pregnant, was an immunologically compatible match, but doctors warned her that donating bone marrow for the transplant could endanger her pregnancy, leaving her with a difficult choice:  continue her pregnancy and the transplant would not be performed, or abort to perform it. According to British tabloid The Daily Mail , Li decided to abort her baby to be able to donate her bone marrow to her brother. Doctors will perform the procedure when the 24 year old recovers from her abortion.

The case has opened a medical and ethical debate in the media and social networks. Some see Yang Li as a heroine, while others believe that she should not have had an abortion.

The question that we ask and that must be answered is: Can bone marrow really not be obtained from a pregnant woman? Because if it can, there would be no medical reason to have to resort to abortion.

Clinical approach

To answer this question, we at the Bioethics Observatory asked a specialist, Javier de la Rubia, head of the haematology department at La Fe University Hospital in Valencia (Spain) and professor of haematology at UCV to assess the case. His clinical response was as follows:

Haematopoietic stem cells for allogeneic transplant can be obtained in different ways:

  1. If bone marrow is used, the procedure is carried out in theatre under general anaesthesia by repeated puncture-aspiration in both posterior-superior iliac crests, and usually lasts about 90 minutes.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

She aborted her child to save her brother’s life. A paradigmatic case of medical and ethical manipulation

pdfCan bone marrow really not be obtained from a pregnant woman? Because if it can, there would be no medical reason to have to resort to abortion.

Yang Li’s brother urgently needed a bone marrow transplant to treat his disease. Yang Li, who was 12 weeks pregnant, was an immunologically compatible match, but doctors warned her that donating bone marrow for the transplant could endanger her pregnancy, leaving her with a difficult choice:  continue her pregnancy and the transplant would not be performed, or abort to perform it. According to British tabloid The Daily Mail , Li decided to abort her baby to be able to donate her bone marrow to her brother. Doctors will perform the procedure when the 24 year old recovers from her abortion.

The case has opened a medical and ethical debate in the media and social networks. Some see Yang Li as a heroine, while others believe that she should not have had an abortion.

The question that we ask and that must be answered is: Can bone marrow really not be obtained from a pregnant woman? Because if it can, there would be no medical reason to have to resort to abortion.

Clinical approach

To answer this question, we at the Bioethics Observatory asked a specialist, Javier de la Rubia, head of the haematology department at La Fe University Hospital in Valencia (Spain) and professor of haematology at UCV to assess the case. His clinical response was as follows:

Haematopoietic stem cells for allogeneic transplant can be obtained in different ways:

  1. If bone marrow is used, the procedure is carried out in theatre under general anaesthesia by repeated puncture-aspiration in both posterior-superior iliac crests, and usually lasts about 90 minutes.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.