Skip to Content.
Sympa Menu

homestead - [Homestead] Stem cell cure for bone marrow myelofibrosis, leukemia etc.

homestead AT lists.ibiblio.org

Subject: Homestead mailing list

List archive

Chronological Thread  
  • From: Tvoivozhd <tvoivozd AT infionline.net>
  • To: homestead AT lists.ibiblio.org
  • Subject: [Homestead] Stem cell cure for bone marrow myelofibrosis, leukemia etc.
  • Date: Sun, 29 Aug 2004 15:11:51 -0700

Title: Stem cell cure for bone marrow myelofibrosis, leukemia etc.
Bone marrow cancers are not all that common and previously incurable.  Things are changing with the attention given stem cell treatment. by everyone except idiot Bush.

If I were a few years younger, I'd be laying in a hospital bed right now, undergoing stem cell therapy for myelofibrosis. 


 

BY MERRY SUE BAUM


"For some people, a stem cell transplant is an alternative where there is no other, " says Roger K. Strair, MD, associate professor of medicine at UMDNJ-Robert Wood Johnson Medical School (RWJMS).

One such person was Olga Rabke. While on a trip to her native Italy, Rabke suddenly experienced what felt like a pulled muscle in her groin. When she returned home to Scotch Plains in March, she went to her physician. Except for being slightly anemic, she was given a clean bill of health. He did recommend, however, that she see an orthopedist. She did, and he discovered she was suffering from multiple myeloma, or cancer of the bone marrow that causes tumors to form inside the bones. An x-ray showed that her femur looked like "Swiss cheese."

She started her search for an oncologist. Her neighbor, Rohit Sharma, MD, a student at RWJMS at the time, recommended Strair. She liked him immediately. "He said a few words to me in Italian," she says, "and he took the time to explain everything to me. I felt like I was in very good hands." He recommended an autologous stem cell transplant for his new patient.

According to Strair, who is also director of stem cell transplantation at The Cancer Institute of New Jersey, the procedure, which has been in use for about a decade, has often proven effective in the treatment of multiple myeloma, lymphomas, leukemias, Hodgkin's disease, aplastic anemia, testicular cancer and some congenital immunodeficiency diseases.

Bone marrow transplants were at one time the ultimate treatment for these disorders. And depending on an individual's history and the disease, it is still the preferred therapy.The procedure begins by either taking some of the patient's own marrow and freezing it, or finding a suitable donor.

Next, high doses of chemotherapy and radiation are given to the patient to destroy the cancer-causing cells. While this is perhaps the most grueling part of the treatment, the physician says often it is not nearly as horrific as it's portrayed. "Some people don't get very sick," he says. "And we have drugs that are very effective for controlling nausea."

After the chemotherapy and radiation have been completely metabolized by the body, the preserved or donated marrow is given to the patient via a transfusion. If all goes well, the stem cells in the transplanted bone marrow will give birth to a generation of tumor-free blood cells in about 15 to 25 days.

A stem cell transplant is, in essence, a bone marrow transplant with several distinct differences. Andrew L. Pecora, MD, a UMDNJ-New Jersey Medical School (NJMS) alumnus and director of the Marrow and Stem Cell Transplant Program at Hackensack University Medical Center, explains. "In a bone marrow transplant, the stem cells are in the marrow," he says. "In a stem cell transplant, they are harvested from the circulating bloodstream." Stem cells taken from the bloodstream multiply much faster, so new cells form within only five to 15 days. Since the white cells - the body's defense system - are produced much faster than in a bone marrow transplant, the patient is far less likely to contract a life-threatening infection. Hospital stays, if needed at all, are shorter, and costs are significantly reduced. To stimulate stem cells to move into the bloodstream, a patient is given chemotherapy and a growth hormone for 10 to 14 days, or a growth hormone alone for five days. He or she then goes through an almost painless harvesting procedure known as leukophoresis. It is done on an outpatient basis, in a special stem cell laboratory, and is much like getting a blood transfusion. A needle is inserted into a vein and blood is slowly removed and run through a leukophoresis machine. The stem cells are filtered out, counted, and the remaining blood is pumped back into the patient. The patient next gets high doses of chemotherapy and/or radiation, the stem cells are infused again through a vein and they "home" to the bone marrow and begin making new cells.

There are three types of marrow and stem cell transplants: autologous, in which the patient's own marrow or stem cells are transplanted; syngeneic, in which marrow or stem cells from an identical twin are used; and allogeneic, in which marrow or stem cells from another person with similar blood and tissue types are used.

"Allogeneic is much more complicated," Pecora says. "Patients undergo prolonged therapy with immunosuppressant drugs to avoid rejection of the new bone marrow. The drugs lower the white cell count making the patient very susceptible to infections. They must take antibiotics and be monitored very closely."

But the upside, he says, is that the new marrow or stem cells are highly unlikely to be contaminated with tumor cells, so the chances of recurrence are lowered. Also, the patient now has a different immune system that may see the tumor cells as foreign and kill them.

An autologous transplant is much safer. "Everyone survives," he says, "but there is always the chance that a few tumor cells got into the harvested marrow or stem cells, and the disease will return. Syngeneic is probably ideal: there is next to no chance of rejection and no chance of contamination. However, most people don't have an identical twin."

In Rabke's case, she first received chemotherapy to mobilize the stem cells into the bloodstream. Throughout the chemotherapy, she continued teaching Italian, as she had for years, at area book stores and adult high schools. "I'm very strong," she says. "Sometimes students would come to my house if I was too tired, but I was never really sick."

After having a chest catheter inserted as an outpatient at Robert Wood Johnson University Hospital in New Brunswick, Rabke underwent three sessions, about six hours each, of leukophoresis. Next came two days of high doses of chemotherapy. She and her husband stayed in a hotel nearby while she received the treatments. One day later, November 1, her own stem cells were infused back into her body through the chest catheter. "It was a big day for me," she says. "I finally made it to the end of the road."

Rabke says she lost her hair, which was probably the most traumatic part of the treatment. And it took about three months for her appetite to return, but once it did, she was her old self. "Dr. Strair said I didn't need vitamins, I should just eat well. I'm Italian. I love to cook. So I ate well."

BENEFITS OF UMBILICAL CORD BLOOD

Patients with certain blood or bone marrow diseases (such as leukemia or congenital immunodeficiency diseases) can be cured by replacing their diseased blood-forming cells with normal blood stem cells from healthy donors. This procedure is called stem cell transplantation. After chemotherapy and/or radiation to reduce or eliminate their diseased blood cells, patients receive transfusions of the donors' blood or marrow stem cells, which begin producing new blood cells within a few weeks.

The immune cells transplanted among the donor cells can benefit the patient by fighting against the patients' diseased cells. In some cases, however, the patient's body can be attacked by the donor's transplanted immune cells because the patient's and donor's immune systems recognize each other as 'foreign.' Such an attack, called graft vs. host disease or GVHD, can lead to serious disability or death, even after the original disease has been eradicated. In order to reduce the risk of severe GVHD, the donors' cells must be closely matched to the patients' immune system markers (termed HLA typing). If the patient has healthy siblings, the chance of a complete match is approximately 1 in 4 for each sibling. Cells from incompletely HLA-matched siblings are likely to cause clinically significant GVHD. Cells from unrelated donors, even if matched, are more likely to cause clinically significant GVHD due to immune system mismatches that are not identified in HLA typing. Unfortunately, it is often difficult to find even unrelated matches for individuals of ethnic minorities (i.e., non-Caucasians in the United States) among the traditional bone marrow (stem cell) registries.

It has long been recognized that blood from umbilical cords of newborn babies contains higher numbers of stem cells than adult blood. Cord blood is collected after delivery of the placenta, without any risk or discomfort to mother or baby. Since the late 1980s, several hundred stem cell transplants for both children and adults with either malignant or non-malignant diseases have been performed using unrelated cord blood when no matched siblings were available. In general, the results have been better in children with non-malignant diseases; however, many adults with malignant blood diseases have had very long disease-free survivals.

Cord blood cells are immunologically immature and seem to better tolerate the patients' tissues, resulting in lower frequency and severity of GVHD than adults' stem cells. Incompletely matched cord blood cells are being used more frequently for transplantation in unrelated patients. GVHD appears to be relatively less problematic with cord blood transplantation than adult stem cell transplantation.

As a result of the encouraging results with cord blood transplants, several organizations in the United States, Europe, Japan and other regions have initiated programs to bank cord blood from large numbers of newborns. It is hoped that a large inventory of banked cord blood units from widely diverse ethnic backgrounds and HLA types will provide grafts for those individuals for whom the traditional bone marrow registries cannot find donors.

The legislature of the State of New Jersey has provided funding to develop a public umbilical cord blood bank for the citizens of the region, called the New Jersey Cord Blood Resource, to be housed at the Coriell Institute for Medical Research in Camden. At least two additional medical centers in the northern and southern parts of New Jersey will be recruited to participate in the program.

Richard D. Huhn, MD
Coriell Institute for Medical Research

So far Rabke is doing fine. She's still teaching and is active in the theater, one of her loves.A veteran actress - she's performed in Italy and off-Broadway - she recently did a 10-minute monologue on her life at the Miranda Theater in New York City. She didn't talk about her illness, she says, but she did tell the audience, "Whatever pasta and wine won't cure, there is no cure for."

While stem cell transplants aren't new, what is new is their possible use as a treatment and perhaps cure in diseases other than cancer. Currently NJMS, Hackensack University Medical Center and Thomas Jefferson University in Philadelphia are collaborating on a project to evaluate the effectiveness of stem cell transplants on multiple sclerosis - better known as MS - and lupus erythematosus. Stuart D. Cook, MD, president of UMDNJ, and professor of neurosciences at NJMS, is one of the neurologists involved in the project.

Pecora explains that in these two diseases, T cells, which are part of the white cell defense team, go awry. In MS they attack a person's own nervous system, and in lupus they destroy connective tissue in vital organs like the kidneys and heart.

By doing an autologous stem cell transplant, Pecora says, the adult, rogue T cells are destroyed and new immature T cells are born. "It's like re-booting a computer," he says. "These new T cells haven't been programmed to attack the nervous system and connective tissue, so it's possible that the disease can be cured." Stem cell transplants are being done around the country for the two diseases, and many have been very successful.

Another disease that may respond to the treatment is breast cancer. The news media has portrayed it as ineffective, Pecora says, but the jury is still out. "We need a few more years for the data to mature before we'll know," he explains. "But there are strong indications that when appropriately applied, it does help in certain types of breast cancer."

He recently published a paper in the Journal of the American Medical Association that delineates in which cases stem cell transplantation is helpful and which ones it's not. He reported that 43 percent of young women were still alive with no breast cancer after three years if they were in a group that had metastatic disease, had not previously been treated with chemotherapy, responded to chemotherapy for the disease, and the disease was limited in the number of sites.

While stem cell transplants are helping more people than ever before, some people simply can't be cured. Strair says that the patients themselves, however, are an inspiration. "In spite of all the difficulties and the uncertainty these patients face, they all have a dignity that comes shining through," he says. "It's people like Mrs. Rabke who keep you going."

Both Pecora and Strair are optimistic about the future of the therapy. Stem cells are now being grown in the laboratory, and umbilical cord blood has been discovered as a ready source. In fact, Pecora had two patients in the final stages of chronic myelogenous leukemia who have survived now for one year and two years, after a cord blood stem cell transplant. "The number of stem cells in the cord blood was too low, so we grew more in our lab," he says.

Strair believes that in the not-too-distant future, cell engineering will be another method of curing these diseases. "We hope to be able to take a few cells from a person, manipulate them or add a gene, then give them back and the body will cure itself."

In the meantime, the physicians continue to research better methods of marrow and stem cell transplantation. And with any luck at all, someday they both may be out of business.


Winter Table of Contents
HealthState Home

The magazine of the University of Medicine and Dentistry of New Jersey

 

 




  • [Homestead] Stem cell cure for bone marrow myelofibrosis, leukemia etc., Tvoivozhd, 08/29/2004

Archive powered by MHonArc 2.6.24.

Top of Page