ST. LOUIS (Ivanhoe Newswire) -- Sickle cell anemia is one of the most common genetic diseases in the United States, affecting 70,000
Americans. The only cure for the disease is a stem cell transplant, but
the perfect donor is hard to find. A new type of transplant is opening
to door to the thousands of children who don't have a donor in their
family
For 14-year-old Albert Pinckney, the best experiences in life are on wheels.
"I like going fast," Pinckney told Ivanhoe.
But having sickle cell anemia forced him to grow up too fast. Pinckney missed all of thefirst grade because of complications with his disease.
"My spleen, gall bladder, tonsils, adenoids -- I've had all those removed," Pinckney said.
Unfortunately, Pinckney's story isn't unusual among kids with severe sickle cell. Thedisease causes abnormally shaped blood cells that have trouble passing through vessels. Patients can get blood transfusions to replace the abnormal cells, but the fix is only temporary.
"We know that we have helped them for a short while, and then they are going to be back again," Shalini Shenoy, M.D., Director of the Bone
Marrow Transplant Unit at St. Louis Children's Hospital in St. Louis,
Mo., told Ivanhoe. The only cure is a marrow or blood stem cell transplant, but until now, this has only
taken place between family members. A new study is allowing patients to receive transplants from unrelated donors.
"The idea is to try to make transplants available to different groups of patients," Dr. Shenoy said.
The study is also trying to ease the long-term effects of the chemotherapy needed before the procedure. Lower doses could prevent organ damage, ovarian failure and sterility.
Although it's been a struggle, Pinckney's disease has made him who he is."It's really affected me and helped me to be a more mentally strong kid than most," Pinckney said. A strong kid whose cure may be around the corner.
The stem cells used in the study come from the National Marrow Donor Program and cord blood registries across the country. Another possible cure for sickle cell disease is gene therapy, which has only been tested in mice.
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FOR MORE INFORMATION, PLEASE CONTACT:
Jackie Ferman-Grothe
St. Louis Children's Hospital (314) 286-0304
| The Future of Sickle Cell Anemia Treatment |
The Sickle Cell Cure Foundation, Inc. (SCCF) is a nonprofit coorporation registered in the State of Oklahoma.
Help For Sickle Cell AnemiaIf you are suffering from sickle cell anemia, what you’re about to read in this report could be the beginning of blessed relief from debilitating pain.
Gene TherapyExcerpt By Janice Billingsley, HealthScoutNews
(HealthScoutNews) -- While a cure remains elusive, victims of sickle cell anemia are living
longer, healthier lives.
cell anemia affects about 80,000 Americans, primarily blacks. Another 2 million Americans have the defective gene that causes the disorder,
making them potential carriers.
Thanks to improvements in diagnosis, treatment and research, half of those with the condition are now more than 50 years old. Until recently, people with sickle
cell rarely survived childhood, according to the National Human
Genome Research Institute.
"I have five adult patients, one more than 60 years old, who watch out for themselves. They are good, cooperative patients who make sure they optimize
their care," says Dr. Kenneth Algazy, a clinical associate
professor of medicine at the University of Pennsylvania School
of Medicine.
May 28th, 2009

No, it wasn’t embryonic stem cell therapy that cured Joseph Davis Jr.’s sickle cell anemia. But from the “mainstream” media’s hype of embryonic stem cell research, you probably guessed it was.
While there is still not a single successful therapy derived from embryonic stem cell (ESC) research, the case of Joseph Davis Jr. is yet another success from a stem cell therapy that, unlike ESC, does not destroy innocent human life.
OneNewsNow ran an article today on the story of little Joseph Davis Jr., son of Joe and Darlene Davis from Texas ,who was born with sickle cell anemia, an autosomal recessive genetic disorder. Because of this disorder, little Joe ran high fevers,
experienced swelling of the hands and feet, and had to have frequent
blood transfusions.
stem cells from baby Isaac to treat Joe Jr.’s sickle cell anemia.
From the OneNewsNow article:CitizenLink ran an interview with the Davis family last year in which Darlene said of 8-year-old Joe:“[Cells from] Isaac’s umbilical cord blood, adult stem cells, [were] going to save my son’s life, and that adult stem cell got my son cured today. He is healed,” Darlene adds. “What I’m talking about is no more sickle cell, no more running
That was back in 2002 when Isaac was born and Joe Jr. was treated using Isaac’s umbilical cord stem cells, and the Joe Jr. is still doing fine.
to the hospital, no more fevers, no more blood transfusion. He’s a
healthy child today.”
He’s doing great and he’s loving life. When he was little, the doctors told me he would have to take antibiotics every day for the rest of his life. Guess what? He’s not taking anything. And I thank God for that.
The total number of successful therapies using adult stem cells, umbilical cord stem cells and other non-destructive sources is between 70 and 80 now. Therapies include
treatments for meningitis-related limb damage, brain injury, stroke, retina regeneration, heart tissue regeneration, angina, diabetes, bone cancer, nerve regeneration, cerebral palsy, cartilage regeneration, Parkinsons, kidney damage, liver cancer, lupus, multiple sclerosis, and leukemia.
Last year I had the pleasure of meeting Carol Franz while I was in Washington D.C. Carol is an adult stem cell therapy patient and survivor of multiple myeloma cancer, a blood cancer. When I met her, she was energetically hopping into a 10th-floor
elevator with her luggage in tow, and she was wearing a neon shirt
which declared she had received adult stem cell therapy. You can read
Carol’s story at her website and see before-and-after pictures of her.
Unfortunately, despite the dozens of successful treatments dereived from benign sources of stem cells, some people insist on destroying innocent human life to harvest stem cells from human embryos. Earlier this year President Barack Obama authorized
taxpayer funds for embryonic stem cell research, using our tax dollars
in connection with the destruction of innocent human life.
To some, it doesn’t seem to matter that one line of research is already yielding tremendous results while the other has yet to produce a single useful treatment. It doesn’t seem to matter to some that ESC faces practical
hurdles such as tissue rejection (like that seen in organ transplants)
and tumor generation in the recipient, such as those afflicting the
poor Israeli boy treated with fetal stem cells. It seems irrelevant to some that an
innocent human life is destroyed in the harvesting of embryonic stem
cells.
Regardless of all this, they insist that ESC research go forward, and they insist you the taxpayer pay for it–even as some in the “mainstream” media are starting to admit that ESC research just isn’t necessary.
One has to marvel at the irrationality of today’s death culture.stem cell research may finally put sickle cell anemia on the run.

Sickle cell anemia, the first genetic disorder recognized by medicine, is still not well understood.
Identified in 1910 by James B. Harrick in Chicago, the disease affects close to 100,000 people in the United States, mostly African American, and millions worldwide. The consequences for patients are devastating.According to Sophie Lanzkron, director of the Sickle Cell Center for Adults at Johns Hopkins University, 30 percent of sickle cell patients experience pain 90 percent of days. "This is a tough disease; they have intermittent episodes of excruciating pain — they can’t go to college or have careers. Patients spend their lives just trying to manage the pain.”
With no objective measure of the disease, Lanzkron said patients are frequently stigmatized when they seek medical attention — being mistaken for addicts in search of narcotics — and treated with disdain by emergency-room personnel unfamiliar with the disorder.The physiological consequences of sickle cell anemia include severe damage to organ systems, particularly to the kidneys and lungs, and can reduce life expectancy for those with sickle cell to the mid-40s. Complications include life-threatening infections and stroke — even in young children.
Children who suffer intermittent attacks miss school with predictable consequences for their lives and for their futures. Meanwhile, their caregivers must miss work to tend to them. Added to this are the health-care costs. Sickle cell patients, Lanzkron said, are often “understandably depressed."“There are so many unmet needs in this patient population,” says Lanzkron, “and I see people come in with horrible complications.” But researchers are targeting the disease with new multidisciplinary approaches with promising results. Combining a novel chemotherapy protocol with a proven bone marrow transplant technique, Robert Brodsky, director of the hematology division at Johns Hopkins University Medicine, announced the cure [see correction below] of an adult sickle cell anemia patient — Pamela Newton of Capitol Heights, Md. And researchers with the National Institutes of Health have developed innovative techniques spanning the spectrum from chemotherapy to irradiation to move step-by-step towards a cure for sickle cell and related disorders.
More than meets the eye
Normal red blood cells are disk-shaped and flexible, explained Lanzkron,
allowing them to squeeze through tiny capillaries to bring oxygen and
nutrients into every part of the body.
vessels and capillaries. That’s where the pain starts. “But we know it’s more complicated than that,” she said. The sickle cells also have
a reduced oxygen capacity and shortened life expectancy compared to
normal blood cells, and they seem to have a negative impact on the
blood vessels themselves, “It affects the whole environment.”
Lanzkron elaborated: “People carrying the trait can still get malaria, but they are less likely to die from it.” But, she said, “There is no harm in having the trait,” which is carried by a recessive gene.
However, when two parents have the trait, their offspring each have a one-in-four chance of having sickle-cell disease. Although mostly associated with blacks in the United States, the disorder is also seen among Latinos, Asians and others with Mediterranean or African ancestry.Aside from the not-always-effective pain medications, only two approved treatments exist for sickle cell, Jordan said — the chemotherapy drug hydroxyurea to ameliorate crises and bone marrow transplant.
An Elusive and Risky Cure
Bone marrow transplants have been recognized as a cure for sickle cell for
more than 20 years, but they have always required a perfect tissue
match between the donor and the recipient. Only siblings can meet that
requirement, and even then, the odds are one in four of a perfect
match. However, because sickle cell is hereditary, closely matched
siblings also have a high probability of sharing the disorder. Lanzkron describes a lifesaving operation: “We give the patient agents that knock out the bone marrow; then it’s like getting a transfusion. We harvest a bit of marrow from the donor and infuse it into the patient.
Although the procedure has been used to cure the disease in about 200 patients — all of them children — bone marrow transplant is so
problematic in adults that it is rarely attempted except in
life-threatening circumstances.
“It is very difficult for adults to embark on a transplant course because, by that time, they have experienced so many transfusions that finding a match in itself is a challenge,” Jordan explained. Furthermore, in adulthood, mounting complications and decades of organ damage may render patients too fragile to withstand the chemotherapy required to make it work.
Over the past year, important advances have been made both in widening the pool of potential marrow donors and in lowering the risks of bone marrow transplant.The intensive chemotherapy regimen, employed up until the 1990s to make way for bone marrow transplants, destroys the patient’s native bone marrow so that a graft can take root. But if the graft fails, the patient, with his own marrow obliterated, can no longer produce new blood cells and must be re-transplanted immediately in order to survive.
But this scenario is turning around dramatically thanks to Brodsky's innovative technique developed at Johns Hopkins. Brodsky and his team, who reported the first adult cure in May of last year, took a new approach by utilizing a less toxic chemotherapy regime than customarily used for bone marrow transplants.It employs just enough chemotherapy to prevent the patient's immune system from rejecting the donated stem cells while preserving as much of the patient's bone marrow as possible. Three days after the bone marrow transplant, doctors dose the patient with the drug cyclophosphamide to kill off the donor’s lymphocytes (white blood cells that attack foreign cells) before they can begin an attack upon the body of their new host. This combination therapy gives the recipient patient's new stem cells a chance to
establish themselves in the bone marrow and begin producing healthy
blood cells while a compatible — and home-grown — immune system
develops.
Most significantly, Brodsky said the procedure allows for transplants not only between fully matched siblings, but also between half-matched pairs, expanding the potential donor pool to parents and other relatives.
Immunosupressive Advances for Sickle Cell and Related Disorders
Jonathan Powell, a National Institutes of Health research fellow, has been collaborating with Dr. John Tisdale at NIH on a different approach, which shows promise not only for sickle cell, but also for thalassemia, Diamond-Blackfan anemia and other severe congenital blood disorders.
Following Tisdale and Powell’s protocol, the donor receives specialized chemotherapy for five days with injections of G-CSF, an agent that stimulates white blood cell formation, to prepare the marrow for transplantation. The cells, once harvested, are frozen for five months before being infused into the patient. Patients undergo
their own course of immune suppressing chemotherapy, and are given a
single dose of full-body radiation to retard the immune system’s
response to the donated stem cells.
According to Powell, 10 of the 11 people with sickle cell anemia who have undergone this procedure have taken well to the grafts. And in both studies, failure is not necessarily of dire consequence.
Javier Bolaños-Meade, assistant professor of oncology at Johns Hopkins, says with the reduced chemotherapy approach used by both NIH and Hopkins, “the worst case scenario is that the transplant doesn’t take — then we’re back to square one and the person ends up still having sickle cell,” but their bone marrow recovers and they can survive to try an alternative therapy.
Powell said NIH’s results have been promising “not in numbers, but in the positive response the patients have had. It shows that we’re on the right track.
“There are no major technical hurdles left. Bone marrow transplant is a way to cure sickle cell — unequivocally.”
In January 2009, NIH’s bone marrow transplant study was expanded to include not only fully matched adults, but also children and half-matched donor/recipient pairs, and recruitment for these new participants is currently under way; likewise for an ongoing study at Hopkins.Powell says he is exploring collaboration between the NIH study and Brodsky’s research at Hopkins that would draw upon the best of both worlds. “We may end up combining ideas.”
Bolaños-Meade, who led the Hopkins research team, is also confident and says sickle cell shows some of the best results for transplant therapy for any blood disorder. “Given that this is a common condition, if there’s a way to correct the problem, we believe we can have a profound impact.”
*Correction: In an earlier version, we said that Johns Hopkins researcher Robert Brodsky and his colleagues developed "the first-ever cure of an adult sickle cell anemia patient." Dr. Brodsky says that Pamela Newton was cured, but her case is not the first ever adult cure for sickle cell — other adult patients have successfully received bone marrow transplants and been cured. Theapproach being developed at Johns Hopkins potentially allows for broader application of bone marrow transplants to cure sickle cell.
Brodsky notes that this new technique, used for the first time on Ms.
Newton, was probably the first successful haploidentical bone marrow
transplant for sickle cell — he's unaware of others, but he's not 100
percent certain that there aren't more out there.
abroad to receive repair (adult) stem cell therapy (something I am desperately trying to change!), but in this case, the patient and his
family moved to the United States seeking (and finding) a successful
repair stem cell treatment for his sickle cell anemia:
Edna Chang-Vega and her husband left behind good jobs, a comfortable home
and extended family in Panama in the hope of obtaining better treatment for Isaac, who suffered from debilitating sickle cell anemia.
Worried about Isaac’s bouts with pneumonia and frequent
hospitalizations, the family moved in with a cousin in Antioch. They
figured someone in the United States could help ease Isaac’s symptoms
Their sacrifice would succeed beyond anything they could have imagined.
Isaac’s story highlights the international draw of Children’s Hospital Oakland. Italso shows what Dr. Bertram Lubin, president of the hospital’s research institute, hopes to accomplish through its new “global health initiative.”
“What we do here really has impact throughout the world,” Lubin said. “We’re one of a few programs in the United States that’s demonstrated that you can cure children with sickle cell anemia with either a bone-marrow or cord-blood transplantation.”Click here to read the whole uplifting article
4/12/2009 Research and Treatment for Sickle Cell Anemia![]() Sickle cell anemia is a disease in which the body makes sickle-shaped, or “C”-shaped, blood cells. This abnormality makes it difficult for the blood to travel through the blood vessels, causing
chronic pain and many other health issues. Fortunately, researchers Dr. Smith-Whitley begins by talking about the overwhelming number of presentations at the meeting, and what this new research means for
patients and families of those affected by sickle cell disease. The In addition to reporting on the various research studies conducted around the country, Dr. Smith-Whitley goes into detail about genetic
testing for sickle cell, the need for families to be aware of disease Guests: Kim Smith-Whitley, M.D. , Pediatric Hematologist/Oncologist, Children's Hospital of Philadelphia ![]() How to participate Email questions to: questions@patientpower.info For more information on upcoming shows and events please visit the Patient Power® calendar. |
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