Evidence for the cure of HIV infection by CCR5 – 32 stem cell transplantation

Kristina Allers,*, Gero Hütter, Jörg Hofmann, Christoph Loddenkemper, Kathrin Rieger, Eckhard Thiel and Thomas Schneider

Dec 2: Timothy Ray Brown can personally affirm that stem cells cure HIV disease, according to a December 2 report in the journal BLOOD. Mr. Brown, an HIV-positive American living in Germany, had leukemia and underwent chemotherapy and bone marrow transplantation in Berlin in 2007. His bone marrow match carried a rare gene mutation in the CCR5 receptor protein, rendering the transplanted cells resistant to HIV infection. Twenty months following the bone marrow transplant, the German team reported Mr. Brown’s leukemia appeared cured, and there was no evidence of HIV in his blood even though he had stopped his antiviral medication prior to the bone marrow transplant.

Now, a year later, the German team has re-examined Mr. Brown’s immune system for evidence of latent HIV infected cells, and found none. The uninfected transplanted bone marrow cells have replaced immune cells in all the parts of Mr. Brown’s body examined. This evidence is consistent with a cure of his HIV disease. Blood, Journal of the American Society of Hematology

This promising proof-of-principle success provides additional support for the patient-specific stem cell based therapy described in Bedford’s June Science Highlights.

ARTICLE ABSTRACT: HIV entry into CD4+ cells requires interaction with a cellular receptor, generally either CCR5 or CXCR4. We have previously reported the case of an HIV-infected patient in whom viral replication remained absent despite discontinuation of antiretroviral therapy after transplantation with CCR5{Delta}32/{Delta}32 stem cells. However, it was expected that the long-lived viral reservoir would lead to HIV rebound and disease progression during the process of immune reconstitution. In the present study, we demonstrate successful reconstitution of CD4+ T cells at the systemic level as well as in the gut mucosal immune system following CCR5{Delta}32/{Delta}32 stem cell transplantation, while the patient remains without any sign of HIV infection. This was observed although recovered CD4+ T cells contain a high proportion of activated memory CD4+ T cells, i.e. the preferential targets of HIV, and are susceptible to productive infection with CXCR4-tropic HIV. Furthermore, during the process of immune reconstitution, we found evidence for the replacement of long-lived host tissue cells with donor-derived cells indicating that the size of the viral reservoir has been reduced over time. In conclusion, our results strongly suggest that cure of HIV has been achieved in this patient.



Bedford Research Foundation 2010 Newsletter

Read about all of the progress and the research that has occurred at the Foundation over the course of the past year! Dr. Kiessling outlines her vision for the upcoming year as well. Thank you for your support.

The Testis: a source of stem cells for all men?

Could the newly discovered “testis stem cells” be as versatile as embryonic stem cells? Bedford is launching a major research initiative to find out.

A major barrier to progress in stem cell therapies is that, similar to organ transplantation, the body rejects cells that are not a good match for the patient. Surprisingly, stem cells from testis may offer a solution. In 2008, a German team reported that they successfully derived pluripotent stem cells from adult mouse testis. Because of their unique ability to become any type of cell in the body, pluripotent stem cells hold the promise of cures to many conditions, from diabetes to spinal cord diseases. Then in 2009, two U.S. research teams discovered pluripotent stem cells in human testis.

If pluripotent stem cells can be derived from every human testis, it could launch a new era in stem cell research.

Stem cells derived from human testis could help overcome two of the biggest barriers to stem cell therapies. First, these cells could avoid the controversies of using human eggs and embryos for research. Second, these cells could solve the need for patient-specific stem cells to avoid the rejection that occurs if transferred cells are not a perfect tissue match to the patient’s own cells.

Dr. Martin Dym’s research team at Georgetown University estimates approximately 1 in 100,000 testis stem cells may be pluripotent. For several decades, it has been known that sperm are abundantly produced (billions per week) in the adult male testis for life, and that the sperm arise from “sperm stem cells” termed spermatogonia. However, the new reports indicate that in addition to the spermatogonia, there are pluripotent stem cells in the testis. To follow up these exciting reports, Bedford Research scientists have launched a new research initiative to answer several important questions: (1) Can stem cells be derived from the testis of all men? (2) How stable are the testis stem cells in long term culture? (3) Can they prove useful to treat spinal cord diseases, such as injury, amyotrophic lateral sclerosis, and multiple sclerosis? (4) Can they prove useful to cure HIV disease?


Help Us Reach Our Goal

As of November 2010, the Foundation has raised $136,000 towards this project. We need $280,000 to get started, and a total of $550,000 to complete the twoyear study. Please help us meet our goal to pursue this promising research – visit: www.bedfordresearch.org


The 2010 Activated Egg Symposium

Nov 5, 2010: The eighth annual symposium will be co-hosted by Dr. Ann Kiessling and Dr. Gary Stein, University of Massachusetts Medical School. The 2010 keynote will be Dr. Ivar Mendez, Head of Neurosurgery, Dalhousie University, Halifax, Nova Scotia, Canada. Dr. Mendez will speak on the new methods he is developing for delivering stem cells to the brain and the spinal cord. The dinner speaker will be Dr. Ed Wirth, Geron Corp., who will speak on the first FDA approved clinical trial for stem cell therapy. The trial’s focus is acute spinal cord injury. Hamilton Thorne is proud to help sponsor the 2010 Symposium.


New Steps Toward a Stem Cell Cure for HIV: Promising Treatment

March, 2009: A proof of-concept report from German scientists demonstrated that HIV-infected cells could be eliminated from the body using bone-marrow transplantation. At the time of this report, it seemed unlikely that sufficient bone marrow would be available for these types of transplants. But now, developments in stem cell science may have changed that:

First, the possibility of deriving stem cells that are patient-specific, e.g. from testis (see front page) will eliminate the need to use bone marrow cells from bone marrow banks.

Second, the methods for developing bone marrow cells from pluripotent stem cells have greatly advanced in the past two years.

Third, the method for silencing specific genes in human cells (a step necessary to block the HIV receptor) has also greatly advanced in the past two years.

Taken together, it is now theoretically possible for scientists to develop and test this method within 5 to 8 years.

Workshop in Taiwan

April, 2010, BSCRF co-sponsored the International Conference of Stem Cells and Regenerative Medicine for Neurodegenerative Diseases at the Tzu Chi Medical Center in Taiwan.

Research & Therapy in Practice

Foundation scientists and visiting guests took a rare look inside Taiwan’s premiere stem cell institute and shared research and best practices.

Over 20 talks were given by surgeons, basic researchers and neuroscientists. (videos at SpinalCordWorkshop.org) The goal of the meeting, as in past Bedford Spinal Cord Workshops, was to share vital information across disciplines and to discuss the barriers to curing spinal cord injury. Dr. Ed Wirth, Geron Corporation, reported on progress in the first U.S. Food and Drug Administration approved trial for stem cell therapy on acute spinal cord injury. Dr. Wise Young reported on progress in similar trials planned for China and the U.S.

Universal Barriers to Cure

The conference wrapped-up with a Round Table Discussion & Concluding Remarks, moderated by Dr. Ann A. Kiessling, Dr. Wise Young and, Dr. TzuYung Chen. In addition to funding needs, the group described the need for appropriate regulatory guidelines that both protect patients and allow the work  to go forward as quickly as possible in most countries, including India, Japan, China, Canada and the U.S.

Dr. Ann A. Kiessling, Dr. Wise Young and, Dr. TzuYung Chen moderated the lively discussion between surgeons and basic scientists


Foundation Publication

Circadian Rhythms and Cell Division

June, 2010: The second report by Bedford scientists about cell division appeared, as the cover story of the JOURNAL OF ASSISTED REPRODUCTION AND GENETICS. The work builds on the previous report of CLOCK genes that indicate early stem cells may be under the control of circadian rhythms (see report at bedfordresearch.org). These novel findings about controls on cell multiplication will be used in the derivation of testis stem cells to attempt to improve the efficiency and stability of new stem cell lines.


Studies launched to detect genes expressed by men with prostate cancer

Robert Eyre, MD

October, 2010: Dr. Robert Eyre presented recent prostate cancer work by BedfordFoundation Scientists at the New England chapter of the American Urologic Association. Dr. Eyre has studied diseases of the male genitourinary tract with Bedford Research scientists for over 20 years.

The current work is focused on developing improved early detection tests for prostate cancer by taking advantage of technology developed to detect infectious diseases in semen, such as HIV and bacteria. Applying whole genome microarrays, Drs. Kiessling and Eyre have launched studies to detect which genes are over-expressed or under-expressed in semen specimens from men with and without prostate cancer.

The recent work identified 6 genes under-detected and 9 genes overdetected in cancer semen specimens. The research needs to be confirmed in a larger series of study subjects.

Gabbay Award

Dr. Ann Kiessling received the Gabbay award in Biotechnology and Medicine in November 2009 along with Dr. Gianpero Palermo and Dr. Alan Handyside. The award highlights Dr. Kiessling’s nearly 3 decades of research in assisting infertile couples, most recently those living with HIV disease. Learn more at: www.sementesting.org

2010 ISSCR

isscr 2010 meetingJune, 2010: Bedford Research Foundation sponsored a booth at the annual meeting of the International Society of Stem Cell Research in San Francisco. The booth attracted many visitors to its information about parthenote stem cells, testis stem cells, circadian control of the cell cycle, and other Bedford Research programs.


Q & A

With the Director About Stem Cell Research at the Foundation

Q: Has Obama’s Policy Helped Fund Stem Cell Research?

A: President Obama’s executive order to rescind the restrictions on the number of stem cell lines that could be studied with federal funds was valuable for some studies, but as long as the Dickey-Wicker amendment controls federal funds, the development of stem cells from unfertilized eggs, a prime goal of Bedford Research scientists, cannot be federally funded.

Q: Why do patients need their own stem cells?

A: The clinical trials that have provided “proof-of-principle” for cell-based therapies, e.g. transplantation of pancreatic cells for diabetes, have revealed that although some cells function normally, many fail because the transferred cels are rejected as “foreign.” The same is true for transplanted bone marrow in cancer therapies. If the stem cells were the patient’s own (i.e. patient-specific), they would not be rejected.

Q: Why do Bedford scientists work with parthenote and testis stem cells?

A: Parthenote stem cells are derived from unfertilized human eggs, not embryos. Parthenote stem cells behave like embryonic stem cells in the laboratory, multiplying to the trillions needed for therapy, either for the egg donor herself, or for tissue-matched patients. Testis stem cells offer the possibility of deriving stem cells for every man in need, if they can be encouraged to multiply to the trillions possible with parthenote stem cells.

Q: How does Bedford Stem Cell Research Foundation fund its research?

A: By private donations. The Foundation’s licensed clinical laboratory conducts highly specialized fee-for-service tests which cover the costs of the laboratory infrastructure. This allows research activities to proceed with minimal overhead. The lack of dependence on federal dollars also frees the Foundation from the administrative costs of separate accounting practices required by the Dickey-Wicker amendment.

Who is Bedford Research Foundation?

Philanthropy Is The Key To Continued Progress

The average cost of each experiment is $90,000. Because much of our overhead is covered by fee-for-service laboratory tests, 92% of every dollar donated goes directly toward these experiments. This innovative funding model allows Bedford Research scientists greater flexibility to move quickly in promising new research directions.

Continued progress requires meeting our annual funding goal of $450,000 in 2019.

Donate Today!

The History of the Dickey-Wicker Amendment

Ann A. Kiessling, PhD

Federal concern with human embryo research began over 25 years ago with the advent of assisted reproduction technologies, i.e. in vitro fertilization (IVF) or “test tube babies.”

Although the first report of laboratory studies of human fertilization appeared in Science in 1944, (the work was conducted in Brookline, Massachusetts), clinical IVF was successful first in Great Britain in 1978 for couples with infertility. IVF became standard of care in the United States in the early 1980’s. As with all forms of clinical treatment, the medical community looked to basic science research to improve the safety and efficacy of IVF for mothers and babies.

In 1979, an Ethics Advisory Board for the National Institutes of Health issued guidelines for research on early human embryos, but no action was taken. The Federal Policy for the Protection of Human Subjects enacted in 1977 remained in place: 45CFR § 46.204(d), “No application or proposal involving human in vitro fertilization may be funded by the Department or any component thereof until the application or proposal has been reviewed by the Ethical Advisory Board and the Board has rendered advice as to its acceptability from an ethical standpoint.” Since there was no Ethics Advisory Board, federally funded research was not possible.

Throughout the 1980’s, public debate about conducting research on early human embryos took place in Great Britain. Many were in favor, many were opposed. The debate ultimately led to the formation of a regulatory body to oversee research on human fertilization. That regulatory body remains active today, which is why embryonic stem cell research was first possible in England.

In 1993, former President Bill Clinton initiated the National Institutes of Health Revitalization Act(Pub. L. No. 103-43), section 121(c) which simply eliminated 45CFR § 46.204(d), paving the way for Federal funding of grant applications to study human fertilization without the need for additional review by an Ethical Advisory Board.

When this possibility became known to the U.S. Congress in 1996, Representatives Jay Dickey and Roger Wicker authored a rider for the budget of the National Institutes of Health: Balanced Budget Downpayment Act, I, Public Law No 104-99, § 128, 110 Stat. 26, 34 “…none of the funds appropriated shall be used to support any activity involving: 1) the creation of a human embryo or embryos for research purposes; or 2) research in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death greater than that allowed for research on fetuses in utero under 45 C.F.R 46.208(a)(2) and section498(b) of the Public Health Service Act (42 USC 289g(b).” Further, “For purposes of this section, the term ‘human embryo or embryos’ includes any organism, not protected as a human subject under 45 CFR 46 as of the date of the enactment of this ACT, that is derived by fertilization, parthenogenesis, cloning, or any other means from one or more human gametes or human diploid cells.”

The Dickey-Wicker amendment to the budget of the National Institutes of Health has been renewed each year since 1996. Both men are currently active in the U.S. congress. Jay Dickey is a Congressman from Arkansas and Roger Wicker is a Senator from Mississippi. Neither President Bush’s nor President Obama’s Executive Order changed this Federal moratorium. (see August 24, 2010: Preliminary Injunction)

Given the wide-spread support among U. S. citizens for human embryonic stem cell research, it seems highly likely that broad public debate could convince congress that the will of the people is to allow taxpayer dollars to conduct research on the use of embryonic stem cells for regenerative medicine.

Until that time, private and state funding seems imperative to move this promising field forward. Increasing tax benefits for philanthropists who donate to stem cell research would help bridge the current gap in funding between the number of U.S. scientists capable and eager to conduct the necessary research, and the funding currently available.

For additional information, see the Connecticut Law Review, Vol 36, #4, 2004 that contains 8 essays on “What is an Embryo?” and the Rejoinder, Connecticut Law Review, Vol 37, #1, 2004.



Preliminary Injunction against Federal Funds for Stem Cell Research: What does it mean?

Ann A. Kiessling, PhD

Judge Lamberth’s Preliminary Injunction against Federal Funds for Stem Cell Research: What does it mean?

On August 23, 2010, U. S. District Court Judge Royce Lamberth issued a preliminary injunction against the use of federal funds for human embryonic stem cell research. If upheld, this injunction would reverse the executive orders of both former President George W. Bush, issued August 9, 2001, and President Barack Obama, issued March 9, 2009. Because of a long-standing prohibition on federal funding for research on human eggs and embryos (see “The History of the Dickey-Wicker amendment for a description of the Amendment“), Mr. Bush was the first U. S. president to release federal funds for research on human embryonic stem cell lines. His executive order restricted funds to research on those cell lines created prior to his order, in order to ensure that no tax payer dollars were used to create new cell lines by the destruction of human embryos. President Obama’s 2009 order eliminated Mr. Bush’s restrictions on eligible stem cell lines, thus allowing federal funds to study “to the extent permitted by law” all stem cells derived according to strict research guidelines issued by the National Institutes of Health (NIH).

Therefore, before Judge Lamberth’s preliminary injunction, according to new NIH guidelines, federal funds could be used to study any embryonic stem cells derived from human embryos, as long as the embryos had been donated for the research under strict guidelines, but tax payer dollars could still NOT be used to derive new embryonic stem cells. A surprising restriction was the prohibition against federal funds to both study and derive stem cell lines from unfertilized eggs. Termed parthenote stem cells, the stem cells derived from unfertilized eggs also promise to be useful for regenerative medicine, and do not carry the ethical concerns related to the destruction of embryos. (See the State of the Stem Cell)

The impact of Judge Lamberth’s preliminary injunction on on-going federally funded human embryonic stem cell research is not clear, nor has it been tallied how many scientists will be stalled in their efforts. The NIH indicates budgeting approximately $130 million annually for embryonic stem cell research, a small fraction of it’s $30 billion dollar annual budget. California’s Institute for Regenerative Medicine has a larger budget for stem cell research. Other states have set up programs to support stem cell research and bridge the gap in federal funding, including Connecticut, Illinois, Maryland, Massachusetts, New Jersey, New York and Ohio, but funding is only stable in California. Now, more than ever before, private philanthropic support is essential to the U.S. effort in embryonic stem cell research.

Given the wide-spread support among U. S. citizens for human embryonic stem cell research, it seems highly likely that broad public debate could convince congress that the will of the people is to allow taxpayer dollars to conduct research on the use of embryonic stem cells for regenerative medicine.

Until that time, private and state funding seems imperative to move this promising field forward. Increasing tax benefits for philanthropists who donate to stem cell research would help bridge the current gap in funding between the number of U.S. scientists capable and eager to conduct the necessary research, and the funding currently available.


2010 ISSCR: a remarkable lack of new clinical trials for stem cell therapy announced

isscr 2010 meetingThis past week (June 16-19) in San Francisco, Bedford Research Foundation had a booth at the ISSCR (International Society for Stem Cell Research) 8th annual conference.

We joined over 3,500 scientists, students and advisers attending the meeting from around the world. The conference boasted more than 200 talks, and some eye opening research from scientists such as Fred Gage, Salk Institute and George Daley, Children’s Hospital, Boston.

However, there was a remarkable lack of new clinical trials for stem cell therapy being announced, and no reports about recent discoveries of the importance of Circadian Rhythms in cell development.

And although several talks focused on the importance of “niche environment” to cell differentiation (the process of transforming stem cells into brain cells, skin cells, heart cells, etc.) none focused on the importance of “equivalence groups” in the early stages of development.

“Equivalence groups” are groups of cells that elect to work together to develop a specific tissue (e.g. heart or lung), and are able to communicate about the complex sequence of steps involved. Cells in an “equivalence group” will not opt to move to the next step of development, until the previous step has been completed successfully.

We hope that at the ISSCR 2011 we’ll see more talks featuring studies about how these groups communicate, as well as analysis of the sequences they are programmed to complete.



Curing HIV Disease With Stem Cell Therapy

Science Highlights by Ann A. Kiessling, PhD

What is HIV disease?

Human Immunodeficiency Virus (HIV) infects specific types of cells in the immune system. Like most viruses, in order for HIV to infect a cell, the virus must bind to a specific protein, termed a receptor, on the cell’s surface. There are many different types of cells in our immune system, and each plays a specific role in fighting infections, both bacterial and viral. Our bodies produce billions of new immune cells every day from stem cell reservoirs in bone marrow.

Patient Specific Stem Cells

HIV has a complex life cycle that includes becoming part of the genetic information of the host cell so the cell is infected for life. Infection can be dormant, with no new virus produced, or active, with new virus produced continuously

HIV infects immune cells that have a protein termed CD4 on their surface. Some HIV-infected CD4 cells die, but others remain in the body, prepared to fight another infection at a later date. When the HIV infected person encounters a new infection, such as the flu, or an infected injury, the HIV-infected CD4 cell responds like a reliable member of the immune system. It becomes activated, multiplies, and as a side effect, produces new HIV particles before it dies. The new HIV particles then infect new CD4 cells, setting up a repeat of the cycle. Because billions of new immune cells are made every day, it generally takes several years for an HIV infected individual to lose enough CD4 cells to have a negative impact on his/her ability to fight other infections. Once the number of CD4 cells is depleted to the point that the individual can no longer effectively fight new infections, their HIV disease has advanced to a new condition termed Acquired Immunodeficiency Syndrome (AIDS).

Virus receptor: the protein on the surface of a cell that allows the virus to bind to, and then enter, the cell to infect it

Is there a cure for HIV infection?

No. It is currently treated with drugs that block specific steps in the life cycle of HIV infection in the CD4 cells, but because some CD4 cells live for decades, and are not killed by the HIV drugs, the potential for them to activate, multiply, and give rise to new virus particles persists for decades. The long life of immune cells is important for disease memory, i.e. it is the reason adults don’t get childhood diseases, such as chicken pox, and the reason that vaccination is effective against diseases, such as polio, for many decades.

Immune system: the collection of cells that respond to and eliminate infection and foreign cell invaders

Can stem cells cure HIV disease?

Over 50 years ago, treatments for some diseases of the immune system were developed, and are the original stem cell therapies. The treatments involve destroying all the diseased immune cells, such as leukemias, with radiation treatment and cancer drugs. (6,7,8). Once the diseased immune system is destroyed, it is replaced by transplanting new immune cells from the bone marrow of a healthy donor.

Bone marrow transplant: the transfer of healthy bone marrow stem cells from a donor to a recipient whose own immune system has been destroyed

This has now become a routine treatment for many cancers and diseases of the blood(1). Early in the HIV pandemic, it was recognized that bone marrow transplants might cure HIV disease. But obstacles have stood in the way of this therapeutic approach:

First, all of the HIV-infected CD4 cells in the recipient must be destroyed before the transplant. If not, the donor bone marrow cells will become infected with HIV, and the transplant will have been for naught. Since not all CD4 cells everywhere in the body are destroyed by the radiation and drugs, infection of transplanted bone marrow was observed (2). Since bone marrow is limited in supply, the medical community was reluctant to “waste” valuable bone marrow to infection by HIV.

Second, the transplanted bone marrow must be a perfect match to the recipient’s cells, or the new immune system will attack them as “foreign,” leading to a life threatening condition known as “graft versus host disease” (see: Patient Specific Stem Cells). Since few matches are perfect, bone marrow recipients are usually treated with immune suppressing drugs. Since immune suppression of HIV infected persons leads to AIDS, this possibility further limited enthusiasm for bone marrow transplant treatment for HIV disease, and restricted it to those individuals who also developed a cancer for which bone marrow transplant was needed.

Importantly, proof-of-concept for the efficacy of bone marrow transplant for HIV disease was reported in 2009 in the New England Journal of Medicine(3). A team of German physicians treating an HIV-infected man with a cancer, lymphoma, by bone marrow transplant, was able to use a bone marrow match from an individual who was naturally resistant to HIV infection. Unlike earlier reports, the new bone marrow cells did not become infected with HIV.

What is natural resistance to HIV infection?

Studies of persons routinely exposed to HIV, but who did not become infected, revealed that in addition to cells having the CD4 protein, efficient infection also needs one of two additional receptor proteins, termed CXCR4 and CCR5. CXCR4 is a protein expressed on the surface of many cells, not just CD4 cells, but CCR5 is less commonly expressed. Individuals genetically lacking CCR5 appear normal and demonstrate remarkable resistance to HIV infection. The bone marrow donor for the German patient was genetically lacking the CCR5 protein.

How can stem cells provide therapy for HIV disease?

The proof-of-concept report from Germany supports the value of bone marrow transplant for HIV disease. New developments in stem cell science open new avenues to solve the main barriers to this therapeutic approach.

First, the possibility of deriving patient-specific stem cells (see: Patient-specific stem cells) will eliminate wasting valuable bone marrow.

Second, the laboratory methods for developing bone marrow stem cells from patient-specific stem cells have greatly advanced in the past two years (4), thus eliminating the need for a good tissue match from a bone marrow bank.

Third, the laboratory methods for silencing genes in stem cells has also greatly advanced in the past two years(5).

Taken together, it is now possible to derive patient-specific stem cells from HIV-infected individuals, differentiate them into bone marrow stem cells, and knock-out the CCR5 protein, rendering them resistant to HIV infection. This source of cells would then be available for transplant into the HIV infected individual, who may or may not have to prepare by going through radiation and drug treatment for complete ablation of all HIV-infected cells. Because the new cells will not be susceptible to HIV infection, it may be possible that over time, they would simply replace the individual’s HIV infected cells.

What is the timeline to develop patient-specific, CCR5 negative, bone marrow stem cells for HIV treatment?

The science of patient-specific stem cells is moving rapidly. By mid-2011, the best sources could be at hand. Within the same time frame, the most efficient laboratory methods for developing stem cells into bone marrow stem cells will also be identified. Hence, 2012 is a realistic time frame for the development of reliable methods to derive patient-specific bone marrow stem cells.

Laboratory methods to knock-out the CCR5 protein may also take 2 to 3 years. Several approaches are currently under study(5).

Once the CCR5 negative, patient-specific bone marrow stem cells are at hand, possibly by 2013, they must be studied for safety and efficacy. This may be the longest phase of the work since it will be necessary to prove long-term survival and lack of negative side effects in an animal model. A conservative estimate for this phase is 3 to 5 years.

Hence, if funding is available, it will be known within 5 to 8 years if patient-specific, CCR5-negative, bone marrow stem cells are a useful tool in the fight against HIV disease.

Will the cost be too high?

Until the efficiencies with which patient-specific, CCR5-negative, bone marrow stem cells can be derived are known, it will not be possible to predict overall costs per treatment.

However, given the current cost of $25,000 to $50,000 per year per patient for monitoring and treating HIV disease in the U.S., it is highly likely that stem cell therapy may be substantially less costly.

Bedford Research scientists will begin the patient-specific Testis Stem Cell Project in 2010, as soon as funding is available.


  1. Kiessling AA and Anderson SC 2007 Human Embryonic Stem Cells, Jones and Bartlett plublishers
  2. Krishnan A,Zaia J, and Forman SJ 2003. Should HIV-positive patients with lymphoma be offered stem cell transplants? Bone Marrow Transplantation 32: 741-748
  3. Hutter G, Nowak D, Mosner M, Ganepola S, Mubig A, Allers K, Schneider T, Hofmann J, Kucherer C, Blau O, Blau I, Hofmann W, Thiel E 2009. New England Journal of Medicine 360: 693-698.
  4. Goodrich A, Ersek A, Varain N, Groza D, Cenariu M, Thain D, Almeida-Porada G, Porada C, Zanjani E 2010. In vivo generation of b-cell-like cells from CD34+ cells differentiated from human embryonic stem cells. Experimental Hematology 38: 516-525.
  5. Shimizu S, Hong P, Arumugam B, Pokomo Ll, Boyer J, Koizumi N, Kittipongdaja P, Chen A, Bristol G, Ballic Z, Zack J, Yang O, Chen I, Lee B, An D 2010. A highly efficient short hairpin RNA potently down-regulates CCR5 expression in systemic lymphoid organs in the hu-BLT mouse model. Blood 115: 1534-1544.

Day Two of the Int’l Conference: Reports from the Front Lines of Stem Cell Therapy Around the World

Day Two: April 23, 2010

stem cell conference international

The conference speakers, organizers and some attendees.

Our second day of the International Conference of Stem Cells and Regenerative Medicine for Neurodegenerative Diseasesbegan with a talk by Dr. Wise Young, of Rutgers University (USA), entitled, “Lithium Effects on Blood and Brain Stem Cells,” in it he summarized the clinical trial design for using umbilical cord blood stem cell therapies for spinal cord injury in China and the US.

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Day One: Int’l Conference of Stem Cells and Regenerative Medicine for Neurodegenerative Diseases

April 22, 2010

Dr. Kiessling and Dr. Shyr

Dr. Kiessling and Dr. Shyr

The first day of the International Conference of Stem Cells and Regenerative Medicine for Neurodegenerative Diseases began with a full auditorium in the beautiful and immaculate Tzu Chi Hospital conference center.

The conference was opened by Dr. Ann Kiessling and Dr. Ming-Hwang Shyr (Superintendent of Tzu Chi General Hospital) together they emphasized the importance of international cooperation in stem cell research. Dr. Kiessling said, “International scientific collaboration is absolutely fundamental to moving stem cell treatments forward as fast as possible.”

Dr. Kiessling’s talk, What is a Pluripotent Cell? And Is Pluripotency Important to Neuronal Differentiation? highlighted her recent research into the importance of circadian rhythms in stem cell biology. Discovering the impact of light and dark cycles for developing cells may be key to our understanding of developing stem cell lines.

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Early Human Embryos Make “Mistakes” as a Matter of Survival, Could Be Key to Understanding Stem Cell



Early human embryos may be naturally prone to making mistakes in chromosome allocation to new cells, according to a report by Bedford Stem Cell Research Foundation scientists. Their new findings indicate rapid increases in total genetic information may be more important to embryo survival than accurate allocation of genetic information to each new cell.

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