Italy
Posted by rob on May 31, 2006 under Uncategorized | 
Read the First Comment
From xenonb.
Sister’s marrow match offers hope for brother’s cure
Posted by rob on under Uncategorized | Be the First to Comment
By Maureen Walsh/ Correspondent
Wednesday, May 31, 2006
Patrick Doherty, age 16-1/2, just got his driver’s permit. He sold his dirt bike and he’s saving money from his job at Herbie’s Car Wash to someday buy his own car. He goes to concerts, plays frisbee and hangs out at Papa Gino’s with his friends. He loves the Red Sox and the Internet. He is, his mother Donna Doherty said, a typical teenager.
Patrick’s family and friends will celebrate the typical life he’s living these days at the American Cancer Society Relay For Life, June 23-24 at the Weymouth High school track.
At the age of 11, Patrick was diagnosed with chronic myelogenous leukemia and the years since have been “a roller coaster ride,” his mother said, of treatments, relapses, complications and gradual progress towards a more normal life.
”He had come home from school with a fever, and I thought it was just something he caught. The next day he was fine,” she recalled. “For eight days he had on-again, off-again intermittent fevers. One day he had a rash, and then it was gone. The next Sunday, he took himself out of a baseball game. He was white, very pale-looking.”
Within days Patrick had been admitted to Children’s Hospital with a confirmed diagnosis of CML, a cancer of the blood cells that more commonly affects adults.
”They discussed his disease with us. They told us the best case for a cure, not just remission, would be a bone marrow transplant,” said Doherty. She and her husband Gerald were tested, along with their daughter Danielle, then 15, and son Ryan, then age 4. “We found out both our other children were perfect matches.”
Patrick received chemotherapy to wipe out his immune system to prevent rejection, and then bone marrow was harvested from Danielle’s pelvic bones and transplanted to him.
”Typically, it takes 17 days to start seeing the immune system building up again, but for some reason with Patrick it didn’t happen,” said Doherty. “He ended up in the hospital for 60 days. Every day, they’d do the blood work and there were no changes. Finally, it ‘took’ on day 55.”
Patrick joined his family in their apartment at the Ronald McDonald House, where they’d been living during his treatment, and attended the Jimmy Fund clinic for two more weeks. Finally, the Doherty family returned home in early fall under strict precautions to protect Patrick from infections because of his suppressed immune system.
No school. No visitors to the house, except the tutor. No fast food. No visits to public places.
”Everything had to be cooked fresh for him. We washed fruits and vegetables in soap and water. We used anti-bacterial soap. We kept the house spic and span,” his mother said. “He could go to the movies, if no one else was in the theater.”
Patrick was able to go outdoors and the weather stayed warm, allowing for fishing trips and mini-golf and street hockey with his friends. And he was able to join his 6th grade class at South Intermediate School after the February vacation.
”His progress was super-duper, so when he relapsed a year and a half after the transplant, everyone was very surprised, especially with a sibling as his donor,” said Doherty. After discussing a variety of options, Patrick’s parents decided to forgo another bone marrow transplant. Instead Patrick received the medication Gleevac to keep his leukemia in remission, followed a year later by an infusion of lymphocyte cells, with his sister Danielle once again as donor.
”It’s supposed to have a better prognosis,” said Doherty, “but there is a risk of contracting Graft v. Host disease. GVHD fights the leukemia, but it goes after good cells as well.”
Patrick did develop a form of GVHD that attacked the fascia of his muscles, but he responded very well to treatment with prednisone and is slowly being weaned from that, she said.
”He’s okay right now. Every time he goes to the doctor’s, we hold our breath. We went yesterday and he looked good on the regular bloods. The PCR (polymerase chain reaction) test for CML looks down deep into the bone marrow. That’s where it would start, if he had leukemia. In two weeks, we’ll get those results.”
Doherty said people sometimes ask how her family has managed these past five years.
”You just have to do it. You never stop thinking about it. A couple of times when his bloods were off, it was like a kick in the stomach,” she said. “There are a lot of kids you probably don’t realize have been through this. You never think it’s going to happen to you or your family. At the Jimmy Fund clinic, we’re amazed at the stories.”
Now that Patrick is doing well, Doherty said the family decided it was time to do something to help others, and Weymouth’s first annual Relay For Life seemed like a good idea.
The Doherty/Belcastro team will join with more than 40 others to maintain a constant presence on the track and raise money and awareness for ACS programs of education, advocacy, research and support for patients and families.
Relay For Life is the American Cancer Society’s signature fundraising event. The theme for the Weymouth event is “Wish Upon A Star! Guiding the Way to a Cure.” The family-oriented Relay program also includes games, contests, entertainment, “theme” laps throughout the night and a challenge to teams to design imaginative campsites.
The Relay opens officially with the Survivors’ Ceremonial Lap to honor those living with cancer and their caregivers. A catered reception follows the opening lap. As the sun goes down, the lights will be dimmed as the track is illuminated by thousands of candles in luminaria bags dedicated by donors in memory or in honor of someone with cancer.
More information on the 2006 Relay For Life, including survivor registration, online team registration, donations and dedication of luminaria is available on the website at www.acsevents.org/relay/maweymouth.
Survivorship chair Dianne Rattigan can also be reached by calling her at Crescent Realty, 617-922-9407, or by sending an e-mail to dmrattigan@aol.com. Ficociello can be contacted by e-mail to weymouthrelayforlife@hotmail.com.
Additional information on American Cancer Society programs and events is available on the web at www.cancer.org or by calling 1-800-ACS-2345.
The Community Newspaper Company, publisher of The Weymouth News, is an official sponsor of Relay For Life.
Hanging Garden
Posted by rob on May 30, 2006 under Uncategorized | Be the First to Comment
From finnigh
Survival Benefit with Imatinib Mesylate versus Interferon Alpha-Based Regimens in Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia.
Posted by rob on under Uncategorized | Be the First to Comment
Blood. 2006 May 18;
A survival benefit for imatinib mesylate versus interferon-alpha therapy could not be demonstrated in the randomized study in newly diagnosed Philadelphia chromosome (Ph)-positive chronic phase chronic myelogenous leukemia (CML) due to the high rate of cross over (90%) from interferon-alpha to imatinib within a year of study entry. We compared survival in 279 patients with newly diagnosed CML treated with imatinib at our institution (2000 – 2004) to 650 patients treated with interferon-alpha (1982 – 1997). The complete cytogenetic response rates were 87% with imatinib and 28% with interferon-alpha (p < 0.0001). The estimated 3-year survival rates were 96% with imatinib and 81% with interferon-alpha (p < 0.01). Survival rates with imatinib were significantly better than with interferon-alpha within each of the CML prognostic risks groups. By multivariate analysis, imatinib therapy was identified as an independent favorable prognostic factor, after accounting for the impact of pretreatment factors (hazard ratio 0.04; p < 0.01). By landmark analysis at 12 months, survival within each cytogenetic response category was similar with imatinib or interferon-alpha, suggesting that the survival benefit of imatinib (versus interferon-alpha in newly diagnosed CML) is through improving cytogenetic response.
AMN107 (nilotinib): a novel and selective inhibitor of BCR-ABL.
Posted by rob on under Uncategorized | Be the First to Comment
Br J Cancer. 2006 May 23;
Chronic myelogenous leukaemia (CML) and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukaemia (ALL) are caused by the BCR-ABL oncogene. Imatinib inhibits the tyrosine kinase activity of the BCR-ABL protein and is an effective, frontline therapy for chronic-phase CML. However, accelerated or blast-crisis phase CML patients and Ph+ ALL patients often relapse due to drug resistance resulting from the emergence of imatinib-resistant point mutations within the BCR-ABL tyrosine kinase domain. This has stimulated the development of new kinase inhibitors that are able to over-ride resistance to imatinib. The novel, selective BCR-ABL inhibitor, AMN107, was designed to fit into the ATP-binding site of the BCR-ABL protein with higher affinity than imatinib. In addition to being more potent than imatinib (IC50<30 nM) against wild-type BCR-ABL, AMN107 is also significantly active against 32/33 imatinib-resistant BCR-ABL mutants. In preclinical studies, AMN107 demonstrated activity in vitro and in vivo against wild-type and imatinib-resistant BCR-ABL-expressing cells. In phase I/II clinical trials, AMN107 has produced haematological and cytogenetic responses in CML patients, who either did not initially respond to imatinib or developed imatinib resistance. Dasatinib (BMS-354825), which inhibits Abl and Src family kinases, is another promising new clinical candidate for CML that has shown good efficacy in CML patients. In this review, the early characterisation and development of AMN107 is discussed, as is the current status of AMN107 in clinical trials for imatinib-resistant CML and Ph+ ALL. Future trends investigating prediction of mechanisms of resistance to AMN107, and how and where AMN107 is expected to fit into the overall picture for treatment of early-phase CML and imatinib-refractory and late-stage disease are discussed.British Journal of Cancer advance online publication, 23 May 2006; doi:10.1038/sj.bjc.6603170 www.bjcancer.com.
Neutrophil Serine Proteases: Future Therapeutic Targets in Patients with Severe Chronic Neutropenia and Leukemia?
Posted by rob on under Uncategorized | Be the First to Comment
Stem Cells. 2006 May 25;
It has recently been discovered that members of the hematopoietic serine protease superfamily, such as cathepsin G, neutrophil elastase, and proteinase 3, may play an important role in myeloid biology by suppressing granulopoiesis. Patients with hematological disorders, such as severe chronic neutropenia and myelogenous leukemia, may have mutations in genes encoding neutrophil serine proteases or alterations in the expression, localization or activity of the proteins. If these findings will stand the test of time, we may have caught a glimpse of the future regarding improved diagnosis, classification, and treatment of these severe blood disorders.
Kinase inhibitors in chronic myelogenous leukemia.
Posted by rob on under Uncategorized | Be the First to Comment
Clin Adv Hematol Oncol. 2006 May ; 4(5): 365-74
The Bcr-Abl tyrosine kinase inhibitor imatinib mesylate launched the era of molecular targeted therapy and constitutes a milestone in oncology history. However, despite impressive cytogenetic response rates achieved with this agent in patients with chronic myelogenous leukemia (CML) in chronic phase, those with advanced-stage CML frequently obtain more modest responses that are in many instances of short duration. Several mechanisms of resistance to imatinib have been described among patients that develop clinical resistance to imatinib. Point mutations in the Bcr-Abl kinase domain that impair the ability of imatinib to inhibit the kinase activity represent the leading cause of resistance. Several approaches are being pursued to overcome these mutations. In addition, many other protein kinases implicated in signaling transduction downstream Bcr-Abl play critical roles in the pathogenesis of CML, thus representing potential therapeutic targets. Multiple compounds are being screened to identify inhibitors of these kinases. This article focuses on the current state of development of new kinase inhibitors for the therapy of CML.
Five years after approval, Gleevec underscores the promise of targeted therapy to provide sustained disease control
Posted by rob on May 12, 2006 under Uncategorized | Be the First to Comment
EDIA RELEASE
Five years after approval, Gleevec underscores the promise of targeted
therapy to provide sustained disease control
• Unprecedented outcomes in Ph+ chronic myeloid leukemia (CML) patients validate
the targeting of a specific cancer cause to create effective and well-tolerated therapies
• First to show reduced annual progression rate with long-term use in newly diagnosed
CML patients – less than 1% progress to more advanced stages of disease in fourth
treatment year
• New data from the largest CML study ever conducted with Gleevec to be presented at
ASCO
• Nearly 15,500 patients in more than 80 countries have received Gleevec through
patient assistance program
East Hanover, May 10, 2006 – Marking an important milestone since its first approval in
May 2001, Gleevec® (imatinib mesylate) tablets,? has become the first oncology drug to be
validated as an effective and generally well-tolerated medicine that targets a specific cause of
a cancer.
Gleevec was first approved in May 2001 by the US Food and Drug Administration (FDA) in
an unprecedented 11 weeks – the fastest FDA review period of any cancer drug at that time –
as a treatment for patients with advanced stage Philadelphia chromosome-positive (Ph+)
chronic myeloid leukemia (CML).
Recent data marking 4.5 years of use showed that more than 90% of patients taking Gleevec
continued to survive and were free from progressing to advanced disease. A five-year update
from the IRIS study (International Randomized Interferon versus STI571), the largest clinical
trial to date for newly diagnosed adult patients with Ph+ CML, will be presented at the
American Society of Clinical Oncology meeting on June 3.
Before Gleevec was available, about 50% of patients progressed to the more advanced stages
of Ph+ CML after only three to five years, and survival was generally short for those patients.
Traditional, less-targeted treatments were associated with significant toxicities that often
limited the ability of patients to stay on therapy long-term.
“Gleevec has an unprecedented record of efficacy and safety for the treatment of patients
suffering from chronic myeloid leukemia, allowing many to resume their daily lives” said Dr.
Daniel Vasella, Chairman and CEO of Novartis. “After more than a decade of research and
clinical development as well as over 200,000 patient years of clinical treatment, we are
moving forward with confidence in the principles of rational drug design pioneered with
? Known as Glivec® (imatinib) outside the U.S.
1/5 2/5 Gleevec. The success of Gleevec gives us confidence in the search for novel medicines that offer improved treatment outcomes.” No other drug for this disease has ever established such a proven and durable track record of efficacy and safety. Interim 54-month data from the IRIS study showed that approximately 93% of newly diagnosed patients with Ph+ CML treated with Gleevec had not progressed to the more advanced and terminal stages. “What Gleevec tells us is that a precise understanding of what drives the growth of a particular cancer allows us to target those abnormalities specifically and develop an effective, durable and well-tolerated treatment,” said Brian Druker, MD, JELD-WEN Chair of Leukemia Research at the Oregon Health and Science University Cancer Institute, Howard Hughes Medical Investigator and lead investigator of the key Gleevec clinical trials. “After five years, we know with certainty that going after the root cause of a cancer, and shutting it down, not only makes sense – it works.” Gleevec is the first targeted therapy for patients with Ph+ CML since it has been proven to inhibit Bcr-Abl, the definitive cause of the disease. Following the rapid US approval on May 10, 2001, Gleevec was approved later that year in the European Union and subsequently in other countries worldwide. Gleevec has also been approved in the meantime for all phases of Ph+ CML. After initial approval, Novartis led the industry in creating the Gleevec International Patient Assistance Program (GIPAP). Nearly 15,500 patients in more than 80 countries have received Gleevec under GIPAP and in the United States under the US Patient Assistance Program. These programs provide Gleevec, in accordance with the drug’s specific approved use in countries, at no cost to qualified patients who are properly diagnosed, not insured, not reimbursed and have no other financial resources. Novartis has continued to investigate Gleevec for use in treating patients with other types of cancer. In 2002, Gleevec was approved worldwide for the treatment of patients with unresectable and/or metastatic Kit (CD117)-positive gastrointestinal stromal tumors (GISTs). In 2005, Gleevec was also submitted in the US and EU as a treatment for the solid tumor dermatofibrosarcoma protuberans and certain forms of myeloproliferative disorders as well as for the treatment of adult patients with Ph+ acute lymphoblastic leukemia (ALL). This year, Novartis submitted additional applications for the use of Gleevec in treating two rare hematologic malignancies: hypereosinophilic syndrome and systemic mastocytosis. All five of these diseases are considered rare but may be life threatening and often have no approved treatments.
About Gleevec Tablets
Gleevec (imatinib mesylate) tablets are indicated for the treatment of newly diagnosed adult
patients with Philadelphia chromosome–positive (Ph+) chronic myeloid leukemia (CML) in
chronic phase. Follow-up is limited. Gleevec tablets are also indicated for the treatment of
patients with Ph+ CML in blast crisis, in accelerated phase, or in chronic phase after failure
of interferon-alpha (IFN-?) therapy. Gleevec tablets are also indicated for the treatment of
pediatric patients with Ph+ chronic phase CML whose disease has recurred after stem cell
transplant or who are resistant to IFN-? therapy. There are no controlled trials in pediatric
patients demonstrating a clinical benefit, such as improvement in disease-related symptoms
or increased survival. Gleevec tablets are also indicated for the treatment of patients with KIT
(CD117)-positive unresectable and/or metastatic malignant gastrointestinal stromal tumors.
The effectiveness of Gleevec is based on objective response rate. There are no controlled trials
demonstrating a clinical benefit, such as improvement in disease-related symptoms or
increased survival.
3/5
Important Safety Information*
Severe (NCI Grades 3/4) neutropenia (3%–48%), anemia (<1%–42%), thrombocytopenia
(<1%–33%), hemorrhage (1%–19%), fluid retention (eg, pleural effusion, pulmonary edema,
and ascites <1%–8%) and superficial edema (1%–6%), musculoskeletal pain (1%–9%), and
hepatotoxicity (3%–8%) were reported among Gleevec® recipients. Patients should be
weighed and monitored regularly for signs and symptoms of edema, which can be serious or
life-threatening. There have also been reports, including fatalities, of cardiac tamponade,
cerebral edema, increased intracranial pressure, papilledema, and gastrointestinal
perforation.
Bullous dermatologic reactions (eg, erythema multiforme and Stevens-Johnson syndrome)
have also been reported. In some cases, the reaction recurred upon rechallenge. Several
foreign postmarketing cases note a resolution or improvement of bullous reaction following
dose reduction with or without supportive care.
Dose adjustments may be necessary due to hepatotoxicity, other nonhematologic adverse
events, or hematologic adverse events. Therapy with Gleevec was discontinued for adverse
events in 3% to 5% of patients.
Patients with severe hepatic impairment should be treated at a starting dose of 300mg/day
and should be closely monitored.
Gleevec is metabolized by the CYP3A4 isoenzyme and is an inhibitor of CYP3A4, CYP2D6,
and CYP2C9. Dosage of Gleevec tablets should increase by at least 50% and clinical
response should be carefully monitored in patients receiving Gleevec tablets with a potent
CYP3A4 inducer such as rifampin or phenytoin. Examples of commonly used drugs that may
significantly interact with Gleevec include acetaminophen, warfarin, erythromycin, and
phenytoin. Please see enclosed full prescribing information for other potential drug
interactions.
For daily dosing of 800mg and above, dosing should be accomplished using the 400mg
tablets to reduce exposure to iron.
Use of Gleevec tablets is contraindicated in patients with hypersensitivity to imatinib or to
any other component of Gleevec tablets.
Women of childbearing potential should be advised to avoid becoming pregnant while taking
Gleevec tablets.
Because of the potential for serious adverse reactions in nursing infants, women should be
advised to avoid breast-feeding while taking Gleevec tablets.
* Gleevec® (imatinib mesylate) tablets prescribing information. East Hanover, NJ: Novartis
Pharmaceuticals Corporation; 2005.
4/5
Common Side Effects of Gleevec Tablets*
The majority of the approximately 1700 adult patients who received Gleevec in clinical
studies experienced adverse events at some time, but most were mild to moderate in severity.
The most frequently reported adverse events were superficial edema (58%–81%), nausea
(47%–74%), diarrhea (39%–70%), muscle cramps (28%–62%), vomiting (21%–58%), rash
(36%–53%), fatigue (30%–53%), musculoskeletal pain (30%–49%), and abdominal pain
(30%–40%)**
Supportive care may help management of most mild-to-moderate adverse events so that prescribed dose can be maintained whenever possible. Gleevec tablets should be taken with food and a large glass of water to minimize gastrointestinal (GI) irritation. Gleevec tablets should not be taken with grapefruit juice. The foregoing release contains forward-looking statements that can be identified by terminology such as “unprecedented outcomes”, “promise”, “durable”, “long-term use”, “survival” or similar expressions, or by express or implied discussions regarding potential new indications for Gleevec or potential future sales of Gleevec, or regarding the long-term impact of a patient’s use of Gleevec. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results with Gleevec to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Gleevec will be approved for any additional indications in any market. Nor can there be any guarantee regarding potential future sales of Gleevec. Neither can there be any guarantee regarding the long-term impact of a patient’s use of Gleevec. In particular, management’s expectations regarding commercialization of Gleevec could be affected by, among other things, additional analysis of Gleevec clinical data; new clinical data; unexpected clinical trial results; unexpected regulatory actions or delays or government regulation generally; the company’s ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry, and general public pricing pressures; and other risks and factors referred to in the Company’s current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing this information as of this date and does not undertake any obligation to update any forward-looking statements contained in this document as a result of new information, future events or otherwise.
About Novartis
Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets
leading innovative prescription drugs used to treat a number of diseases and conditions,
including central nervous system disorders, organ transplantation, cardiovascular diseases,
dermatological diseases, respiratory disorders, cancer and arthritis. The company’s mission is
to improve people’s lives by pioneering novel healthcare solutions.
Located in East Hanover, New Jersey, Novartis Pharmaceuticals Corporation is an affiliate
of Novartis AG (NYSE: NVS) – a world leader in pharmaceuticals and consumer health. In
2005, the Group’s businesses achieved sales of USD 32.2 billion and pro forma net income of
* Gleevec® (imatinib mesylate) tablets prescribing information. East Hanover, NJ: Novartis
Pharmaceuticals Corporation; 2005.
**Numbers indicate the range of percentages in 5 studies among adult patients with Phpositive
CML and KIT-positive GIST. For more detailed study information, please see the full
prescribing information for Gleevec.
5/5 USD 6.1 billion. The Group invested approximately USD 4.8 billion in R&D. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 91,000 people and operate in over 140 countries around the world. For further information please consult http://www.novartis.com. # # #
Contacts
John Gilardi
Novartis Global Media Relations Tel +41 61 324 3018 Mobile +41 79 596 1408 John.Gilardi@novartis.com
Geoff Cook
Novartis Oncology Tel +1 862 778 2675 (direct) Geoffrey.Cook@novartis.com
A Painless Donation, an Enduring Lifeline
Posted by rob on May 3, 2006 under Uncategorized | Be the First to Comment
Martin Mark, 33, who came to the United States from Uganda 11 years ago to get an education, is now working on a master’s degree in computer systems and international business at New Mexico State University. He is also battling a disease, chronic myelogenous leukemia. Though it is now controlled by drugs, the only possible cure is a transplant of cells that would enable bone marrow to form new blood cells.
But the chances that a black person from Africa can find a suitable match for his tissue type are, as he put it, “quite slim,” especially in this country, where only 23 percent of those who have signed up as volunteer donors represent minority groups.
But even Caucasians can sometimes have a hard time finding a well-matched donor of these life-giving cells. Ten years ago Stephen Sprague of New York had the same disease as Mr. Mark but at a time when there were no drugs to keep him going.
He had no relative who could help, and there was no match for him among the millions of potential donors in the National Marrow Donor Registry. On the verge of death, he was saved by a transplant of umbilical cord cells from a newborn girl, whose cell type proved to be a perfect match for him.
An Emerging Procedure
“Somewhere on the streets of New York there’s an 8-year-old girl who has absolutely no idea what she’s done for me,” said Mr. Sprague, 58, all of whose blood cells are now female. Mr. Sprague, who works for the New York Blood Center’s National Cord Blood Program, expects a bright future for cord blood transplants even though so many logistical and technical obstacles remain before it can be widely used.
Most people, and even many doctors, are unaware of the tremendous strides that have been made in the field of bone marrow transplants. They also don’t realize that today it can be much easier to be a donor. No longer is it necessary to be anesthetized and have bone marrow extracted from your hips. Now blood stem cells can be removed from your blood with minimal risk to the donor. And when mothers-to-be choose to donate umbilical cord blood at a hospital equipped to process it, there is no risk at all to the donor.
Dr. Jason Aaron Sokol, then a medical intern, had no idea when he chose his profession that the first time he would try to save a life would have nothing to do with his medical training. Dr. Sokol signed up to be a marrow donor at his synagogue in New York. Nine months later he was told he was a match for a young man dying of leukemia.
Given a choice between donating marrow or peripheral blood stem cells, Dr. Sokol chose the latter. For five days he took a drug, Neupogen, that stimulates the formation of new blood cells, pushing millions of stem cells into his bloodstream. Then he sat for about six hours hooked up to a machine that extracted the stem cells from his blood and returned all the rest of his blood to his body.
Although the recipient of his cells died within a year, the transplant gave the man nine good months and, Dr. Sokol said without hesitation, he’d donate his cells again if asked.
When I signed up to be a bone marrow donor in the late 1980’s, there was only one way to donate. Under anesthesia in a hospital, a large needle would be inserted into both hip bones to extract about a quart of marrow, which the body replaces. Anesthesia presents the greatest risk, followed by possible infection and unavoidable soreness at the extraction sites, as if you’d fallen hard on the ice.
Still, having witnessed a child I loved die of leukemia, I thought it was a small price to pay for the reward of possibly saving someone’s life.
I am now an emeritus donor; over 60, I’m considered too old to donate to a nonrelative. But I’m not too old to call for more volunteers, especially those from minority ethnic and racial groups who are vastly underrepresented as potential donors.
Of more than six million donor volunteers in the National Registry, 1.4 million are now from ethnic groups: more than 465,000 blacks, nearly 400,000 Hispanics, 398,000 Asians and more than 73,000 American Indians. But the chances can be one in a million of finding a good patient-donor match.
Each year some 35,000 children and adults with life-threatening illnesses could benefit from marrow, stem cell or cord blood transplants. Many receive transplants from related donors, some are considered unsuitable for transplants and others die waiting for a transplant.
Today a majority of marrow transplants involve peripheral blood stem cells. Last year the national registry helped bring about transplants from unrelated donors to more than 2,800 recipients: 1,700 peripheral blood stem cells, 821 bone marrow and 324 cord blood transplants.
Matching donor and recipient is especially crucial when doctors transplant cells that produce blood cells because among them are cells that attack foreign invaders. If the match is less than perfect, the donated cells can attack the recipient as foreign — a problem called graft-versus-host, or G.V.H., disease. The closer the match, the less problem with G.V.H.
Finding the Right Match
Matching is done by analyzing the tissue types, or H.L.A. (for human leukocyte antigen) profiles, of donor and recipient. When you sign up to be a potential donor, your profile type is determined from a two-tablespoon sample of your blood. The result is filed in the donor registry. If someone in need of marrow very closely matches your type, the registry will contact you to determine if you are still willing and medically fit to be a donor.
With donations of both bone marrow and peripheral blood stem cells, very close matching is crucial. But with cord blood transplants, less than perfect matches seem to do very well, making it more likely that a patient will find a donor. “The big advantage of cord blood is you don’t have to find a person who agrees to donate,” Mr. Sprague noted. “The cells are frozen and ready to go.”
A major stumbling block is that, while cord blood is often adequate to treat a child, too few cells are derived from an umbilical cord to help an adult. Scientists are experimenting with ways to enrich cord blood samples so that they can be used in adults. One of those methods helped Mr. Sprague. Another approach is to use two well-matched cord blood samples for one patient.
A second problem: too few hospitals are equipped to process cord blood.
Still, Mr. Sprague urges mothers-to-be to look into the possibility of donating her baby’s cord blood. Others interested in becoming donors can contact the donor program at 1-800-627-7692 or marrow.org.