Thalassemia Patients and Friends
Discussion Forums => Curing Thalassemia. Bone Marrow & Cord Blood Transplant => Topic started by: 7assan on June 03, 2010, 01:36:19 PM
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WASHINGTON - Bone marrow transplants are undergoing a quiet revolution: No longer just for cancer, research is under way to ease the risks so they can target more people with diseases from sickle cell to deadly metabolic disorders.
The old way: High doses of radiation and chemotherapy wipe out a patient's own bone marrow before someone else's is infused to replace it, hopefully before infection strikes.
The new way: Rather than destroying the patient's bone marrow, just tamp it down enough to make space for the donated marrow to squeeze in alongside and a sort of twin immune system takes root. It's what doctors taking a page from mythology call "mixed-cell chimerism" — patient and donor blood and immune cells living together to improve health.
http://www.msnbc.msn.com/id/37068859/ns/health-health_care/
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WASHINGTON (AP) — Bone marrow transplants are undergoing a quiet revolution: No longer just for cancer, research is underway to ease the risks so they can target more people with diseases from sickle cell to deadly metabolic disorders.
The old way: High doses of radiation and chemotherapy wipe out a patient's own bone marrow before someone else's is infused to replace it, hopefully before infection strikes.
The new way: Rather than destroying the patient's bone marrow, just tamp it down enough to make space for the donated marrow to squeeze in alongside and a sort of twin immune system takes root. It's what doctors taking a page from mythology call "mixed-cell chimerism" — patient and donor blood and immune cells living together to improve health.
BONE MARROW: Lawsuit urges payment for donors
To find the best methods for these less intense transplants, different mixes of low-dose radiation and immune-suppressing drugs are under study at hospitals around the country.
But the ultimate quest is to allow transplants even when donors aren't a good genetic match, says Dr. Suzanne Ildstad of the University of Louisville— whose technique involves an experimental tweaking of donated cells to help them grow better.
"It makes it possible for anyone who has a mom or dad willing to donate marrow to them to have a transplant," says Ildstad, who has families with sickle cell and other childhood genetic illnesses lining up to try.
Separately, several hospitals are testing how to combine kidney transplants with bone marrow transplants from the same donor, in hopes that a hybrid immune system lessens the need for lifelong anti-rejection drugs.
"People are watching with eager expectation," says Dr. Lakshmanan Krishnamurti of Children's Hospital of Pittsburgh, who is helping to plan a multi-hospital study of some of the new methods for hard-to-treat adults with sickle cell disease.
Doctors have long known that a traditional bone marrow transplant can cure young children of sickle cell — if they have a well-matched donor. New marrow produces healthy red blood cells to replace the sickle-shaped ones that can't squeeze through small blood vessels, the cause of the disease's pain, infections and life-threatening organ damage.
But only about 17% of children have a suitable donor, usually a healthy sibling. Attempts to transplant adults have failed, their bodies too ravaged from years of the disease. Another hurdle: Certain immune cells in donated marrow sometimes become too aggressive and attack the recipient, called graft-versus-host disease or GVHD.
Enter the new research.
First came a tantalizing success in severely ill adults. Nine of 10 patients who underwent a less intense transplant — using low-dose radiation and two drugs to inhibit problem immune reactions — had their sickle cell apparently eliminated, Dr. John Tisdale and colleagues at the National Institutes of Health reported in December. They developed a hybrid immunity that produces normal red blood cells with no GVHD.
But those people had perfectly matched donor cells provided by healthy siblings. Few patients do.
Back in Louisville, Ildstad gives donated marrow a boost to try to overcome that problem while avoiding GVHD, a risk that worsens with mismatched donors. She removes troublesome immune cells from the donated infusion, leaving concentrated amounts of the blood-producing stem cells patients need plus "facilitating cells" that she discovered seem to help them take root.
In an NIH-funded experiment at Louisville and Duke University, the method so far worked in two children with sickle cell who had well-matched donors and one of four with a half-match.
Dr. Joseph Leventhal of Northwestern University gave an Ildstad-treated stem cell infusion to a handful of kidney transplant recipients who developed hybrid immune systems that seem to be holding nearly a year later. The first three treated are using one anti-rejection drug instead of the usual cocktail, and one soon will attempt full weaning.
"We're doing this in patients where it could have potentially the biggest impact," those with unrelated donors, says Leventhal, who anticipates giving one patient a month the dual transplant as the study continues at Northwestern Memorial Hospital.
The attraction to families: "You don't die from the new way," is how Bob Evanosky of Aurora, Ill., puts it.
His three sons have a devastating metabolic disease called metachromatic leukodystrophy, or MLD. Last summer, son John got an experimental outpatient transplant at Duke — a far cry from the months his brother Jack had to spend in intensive care after a well-matched transplant the old-fashioned way.
Dad was John's donor even though he's only a half-match — and the new cells are making the enzyme his body had lacked, too late to reverse the brain damage that has paralyzed the 8-year-old but perhaps able to ease some complications, says Evanosky, who plans to donate to his John's twin Christopher this fall.
Copyright 2010 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.
http://www.usatoday.com/news/health/2010-05-10-bone-marrow_N.htm
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Bone marrow is in flat bones like the breast bone, hip bone, skull, ribs, and shoulder blades and at the ends of the femur (upper leg bone) and humerus (upper arm bone). It contains stem cells that produce red and white blood cells, blood vessels and several other types of cells.
The standard bone marrow transplant requires that a person’s bone marrow be completely wiped out before the donor’s marrow is put in. This period of time when the person has no immune system can be deadly if they get an infection. But bone marrow transplants cure children of sickle cell anemia and are used to treat cancer patients and those with metabolic disorders.
What doctors are doing is partially destroying the recipient’s bone marrow and adding the donor’s so the patient develops a hybrid immune system, a combination of theirs and the recipient’s. Doctors refer to it as “mixed-cell chimerism.”
There are two problems that doctor hope this new technique will overcome. One is finding a suitable match. Only 17 percent of kids with sickle cell anemia have a match, most likely a healthy sibling. Adults with sickle cell anemia do not respond well to bone marrow transplants. The second is that immune cells from the donor can end up attacking the recipient, a phenomenon called “graft-versus-host disease,” of GVHD.
Several studies have already demonstrated success with this technique. In one study with two children, one with a well-matched donor and the other with a half-match received bone marrow that had the cells implicated in GVHD removed before receiving the transplant. Both are doing well.
A study was done in which 9 adults with sickle cell anemia who were severely ill had low dose radiation and two immunosuppressant drugs before receiving bone marrow no longer suffer from the disease and did not experience GVHD. They did developed a hybrid immune system.
This technique may also be useful for those receiving organ transplants. A study in which four kidney transplant recipients who have hybrid immune systems through this technique are only taking one immunosuppressant drug rather than the cocktail that is normally prescribed.
Hospitals involved in this research are using different combinations of low-dose radiation and immunosuppressant drugs to determine how to achieve the best results
http://indyposted.com/22043/new-bone-marrow-transplant-technique-allows-for-less-than-perfect-match/
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Thanks for sharing Baloch.
Zaini.
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it,s ok any time
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You know me all, I haven't read it all. Simple hasn't any time left to do it.
Won't those two systems start fighting with eachother
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Enter the new research. First came a tantalizing success in severely ill adults. Nine of 10 patients who underwent a less-intense transplant had their sickle cell apparently eliminated. They developed a hybrid immunity that produces normal red blood cells. Those people had perfectly matched donor cells provided by healthy siblings, but few patients do.
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I have heard of this before. About scd and mini-transplants
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The linke in the up is from health news web site wich is the date 15 may 2010 i think so
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The new way: Rather than destroying the patient's bone marrow, just tamp it down enough to make space for the donated marrow to squeeze in alongside and a sort of twin immune system takes root. It's what doctors taking a page from mythology call "mixed-cell chimerism" — patient and donor blood and immune cells living together to improve health.
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Yes, mixed chimerism does work, as previously noted in the thread at http://www.thalassemiapatientsandfriends.com/index.php?topic=2704.msg25654#msg25654
Dr Krishnmaurti has been a leader in this work of finding a gentler way to do BMT. We are seeing this same difference in approach between the two different gene therapy groups and this is one of the reasons I much prefer Dr Sadelain's approach.
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Andy,
This is all very exciting. We were lucky to hear some of these developments in New York.
Have you heard anything more about Dr. Sadelaine's research and the clinical trials?
Thanks Andy,
Sharmin
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I recently received this message from Pat Girondi.
Dear Andy,
I am sorry that i have not communicated lately.
I also want to apologize for the lateness in the project.
Michel, Isabelle and team will submit the IND by June 30th. We should be in patients in September.
Give my love to all of rocco's brothers and sisters. [Rocco is Pat's son. Rocco has thal major].
I am presently in India looking ahead. India has at least 200,000 of our brothers and sisters and certain experts estimate the population over 500,000.
I know that all of us live for this dream. A dream that would not have been possible without Michel Sadelain, Isabelle Riviere. Chris Ballas, Norb Weich, Jason Feldman, Sam Salman, kostas Antypas, Joe Feldman, Enrico Colucci, Vito Cirrottola, Giuseppe Schmidt, Paritosh Landahni, Teddy Jimbob Beechcroft, Georgey Hanley, Adrian Herzog, Aurelio Maggio, Angela Iacono, Renzo Galanello, Renzo Arbore, Onofrio Pepe, Mauro Bove, Franco Locatelli, Ron Capano, John Sion, Jonathon Ascoff, Alessandro Contessa, Billy boy Mc Cluskey, Mark Step, the Bucellatos, Derek Holmes, Derek Persons, Angela Chen, Sharon Carsten, Greg Swan, Charles Columbus, Susan Agnelli (late), Steve Petrosky John Walton (late) Mike Ahearn, Hsuan Hargest...
Frankly, I could never finish thanking all of the many people and there are no guarantees that the trials will go well. The drug Thalagen looks great in the sick cells treated thus far.
As you know we have done the the stem cell mobilization in patients already thanks to the support of Dr George Stam.
Please continue to send your love.
I'll end on a funny note.
Recently we were looking around for some cake. I told Jason to check the Orphan Dream acct. The Orphan Dream acct. Gets the money for the music.
Recently, Focaccia Blues the second most popular film from italy used our two songs 'Flim Flam Man' and 'It's Your Time'. The film is now at Cannes and won the Golden Globe Award.
Anyway, we needed cake for the final payment of the vector. Jason checked the OD account to find 8,000.
Wow!!! The bad taste in music by so many is wonderful.
This year at Gateway the Capano family and their fantastic friends hold a golf outing for the Cooleys' Intl (not to be confused with the foundation). To date the Cooly's Intl have invested 100,s of thousands for us.
Last year at the even Ron, Flo, Gina and gang dished over 200k.
This year I'm going to tell them that I'll do a performance... They may just send a check without telling me the date.
Excuse me for all of the people that I did not mention. The list would be 10 times longer.
We now have 3 pharma companies looking at the project and as always we need help. My favorite is Sigma Tau. Write gregg Lapointe and thank him for their past support and tell them how much you appreciate them considering to help us further.
gregg.lapointe@sigmatau.com
Sigma T is our kind of people.
Ok my brother Andy. Get the message out!
September!!!
All we need is a little gene expression of the beta globin gene and they'll be knocking the doors down to help us...
Pat g
Sent from my BlackBerry® wireless handheld
I loved that last note about the funds. We do make a difference by purchasing Pat's music. Go to http://www.cdbaby.com/Search/cGF0IGdpcm9uZGk%3d/0 to buy his CDS or download his music. All proceeds go to help fund gene therapy. Tell your friends. It's a cheap and easy way to help out. The only obstacle to success is money. Let's do everything we can to spread the word. For Pat. For my new Facebook friend, his son Rocco. For all of you. Let's get this done.
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Thanx for the update but the new way is allready don for 10 adults . so that mine adults can do bmt and the chanc going up day by day
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dear andy mixed chimerism is befor bmt or after bmt and bmt done with out High doses of radiation and chemotherapy......?
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Mixed chimerism is after a BMT or cord blood transplant. It is the combination of the DNA of both the host and the donor.
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thnks for update
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But In New way Rather than destroying the patient's bone marrow, just tamp it down enough to make space for the donated marrow that mine you dont need chemotrepy befor bmt you just make space for marrow that what i understand from the new article . can u explain more and read first article
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Possible to explain to me more .............?
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I was told today exactly the same at the heart of our Dutch stem cell transplant centre.
I have heard no radiation is needed for us kind of sct' patients. Do you got more questions?
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Hi baloch
I get the feeling that you are somehow confused about the techniques or protocols concerning transplants in general.
Generally speaking in transplants, you need to inject the patient with normal and matched stem cells. The sources of these stem cells vary.They can be taken from the bone marow of the donor and eventually this is called Bone marrow transplant. Or you can have them from the umblical cord of a baby (these kind of cells are characterised by not being so resistant to the host(patient) and therefore lowering GVHD, but the disadvantage is that they are quiet few in quantity as the cord blood does not have a lot of stem cells compared to other sources.) In this case the transplant is called cord blood transpalnt. The other source of stem cells could be directly coming from the donor blood stream through injecting the donor by certain drugs that increase the quantity of stem cells in the blood stream so they can be collected. In this case the transplant is called Stem cell transplant.
So the above explains all the possible sources in obtaining stem cells. But these stem cells have to match with the patients HLA type. So if the stem cells of the donor and the patient (host) match exactly, we call this 100% HLA matched. But still some transplants succeed without having 100% match . So there is a range for this match, but the closest the match the more sucesseful is the transplant because you reduce the risk that GVHS occurs ( this means when the stem cells start to attack the patient instead of curing)
How these stem cells are injected, which drugs used before and after transplant, kind and quantity of chemotherapy before transpalnt...... all these are called protocol and this is what differniate a center from another and this what makes the variation of success rate from one center to another. Every center or hospital has a certain technique or protocol to follow.
The article you posted was highlightening a certain techique or a different protocol which is the usage of certain drugs that lowers the immune system only instead of using chemotherapy. The advantage of this is that the side effects are gentler than those of the chemotharapy. Dr Krishnmaurti is the pioneer in using this technique. At the same time more transpalnts were done using chemotherapy than this technique and the advantage of this is that you have a big number of operations done in the world by this way and this allows you to have statistics in order to weigh your pros and cons
Results of ANY transplant:
1-Success..the marrow of the patient is 100% made from the donor cells which is called 100% engrafment (the patient is cured and can have some side effect that lie in a range)
2- Mixed chimerism (this happenes when the marrow of the patient and the donor co-exist peacefully togather but the part of the donor is able to produce ''good'' blood allowing the patient to be independant of transfusions. Thsi mixed chimerism is supposed to continue through all the life of the patient in order to consider him/her cured
3- Rejection (the patient rejects the donor cells and he/she is back to thal)
4- Failure (it is the same as rejection but could be accompanied with severe GVHD that could be fatal in some patients.)
The bad thing about transplant in general is that you don't choose or know which result you are going to end. Of courese the doctor will tell the percentage of sucess or failure according to each case, but there is no a concrete gurantee
There are a lot of variable that play a role in increasing the success rate
1- Patient's health from all aspects ( ferritin, hepatits, antibodies, liver, heart, kidney functions.....)
2- HLA match ( the more the match the better the results and less side effects)
3- Expierence of the transplant center
4- When all the above points are present, the age would be a minor variable. But if the above points are not well taken of and the patient is some how ''old'' , then at this point, age matters
Manal
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baloch
You said in another post that your brother will do a BMT in 2 months, where are you going to do it??
Did the doctor suggested keeping his sperms in a bank (since he is an adult) in order to be used later on when he decides to have children ???
manal
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thnx manal for repaly and my bro did not have other poblem ferritin, hepatits, antibodies, liver, heart, kidney functions all is negtiv bm mateced 100% and doctor suggested me to donet my marrow for my bro to do bmt im jordan or egypt or indai that alll
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The old way: High doses of radiation and chemotherapy wipe out a patient's own bone marrow before someone else's is infused to replace it, hopefully before infection strikes.
The new way: Rather than destroying the patient's bone marrow, just tamp it down enough to make space for the donated marrow to squeeze in alongside and a sort of twin immune system takes root. It's what doctors taking a page from mythology call "mixed-cell chimerism" — patient and donor blood and immune cells living together to improve health.
Until the issue is not understandable for me
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By using chemotherapy, the patient bone marrow is completly dead, so that the donor cells will be replaced instead of it.
In the second technique, no chemotherapy is given but only drugs that reduce the immunity of the patient. Since no chemotherapy is used therefore the patient bone marrow is still there and not destroyed and therefore whn the new marrow cells of the donor start to produce blood, they co-exist togather with the old marrow. that is two say mixed chimerism which means that in the body of the patients there are two marrows: the donor marrow and the patient marrow
This is not 100% guanteed to happen because at some time the old marrow overlaps the donor marrow and the transplant is rejected.
manal
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that mixed-cell chimerism is befor dmt . Surveyors understood, but these Updating existing in Egypt. What do you suggest the right brotherly settled with bone marrow or what
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I really do not understand all the question
But to the part that i understood, in Egypt and to my knowledge we follow the protocol of using chemotherapy before the transplant in order to completly get rid of the muted or the ''bad'' marrow of the patient.
Mixed chimerism is the result of the transplant and NOT a procedure done
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my bro did not have other poblem ferritin, hepatits, antibodies, liver, heart, kidney functions all is negtiv bm mateced 100% and doctor suggested me to donet my marrow for my bro to do bmt in jordan or egypt or indai that alll . wht u will say wher sould i will don it .... wht u will say wait for gene therpy or do bmt
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Having a 100% matched BM with a good patient's health increases the sucess rate of the transplant.
About whether to choose gene therapy or BMT, ....gene therapy is a procedure that is under trails and it is not availabel right now for public and it will take years to be there. Patients who are going through gene therapy this year (as mentioned in other posts) are voulnteers that are willing to try this cure on their ownselves. So everything concerning gene therapy is trails.
BMT is a cure that is available right now world wide and it has been done with known percentages and results
The decision of doing a BMT or not has to be taken y the patient after weight the benefit and the risk resulting from this transplant. To understand the risk, the patient should sit and talk with the doctor to evaluate the patient's case in particular with regards to all the variables we discussed above.
As for where to do it, there are a lot of expierenced centers worldwide, the most well known are in Italy... Of course there are other countries like USA, France, Saudi Arabia, India ......, but Italy has the highest success rate of BMT in thalassemia
Concerning the countries you mentioned, you have to directly corresponds with centers specialised in BMT in order to discuss such procedures.
As for Egypt, we do BMT but the majority is for cancer patients compared to thal. Best doctors here for tansplant are Dr. Hossam Kamel and Dr. Alaa el Hadad
manal
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Hi Manal
Thanx for the update and mybe after 2 months we will go for bmt and we will loook mybe that will be in egypt or india . There can be damages in the future we want to work with bone marrow as soon as possible.
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Was there made any decision in which country your brother will have his bmt?