DKMS, a non-profit organisation that works globally in the area of stem cell donation, along with Bengaluru-based Sankalp India foundation, another non-profit organisation which works in the area of thalassemia management, recently set up a state-of-the-art bone marrow transplant unit in Gujarat's Ahmedabad for children with thalassemia. It is currently functional with 10 specialised BMT beds for children and an outpatient department, too.
India does not have a national registry of thalassemia, but, as per reports, it is estimated that nearly 42 million Indians are thalassemia carriers or have thalassemia minor. Carriers have one faulty gene that causes thalassemia but do not have the disease itself. An estimated 12,000 children in India are born with thalassemia major - an inherited blood disorder characterised by less haemoglobin and fewer red blood cells in the body than normal. Now, with bone marrow transplants possible, and that too free of cost, these kids can dream of leading normal and healthy lives.
India CEO of DKMS, Patrick Paul, spoke to THE WEEK at their recently inaugurated BMT unit in Ahmedabad, in association with city-based Health1 super-speciality hospital on February 8. It is India’s second centre offering crucial BMT services to paediatric thalassemia patients. The two organisations, along with the non-profit Cure2Children, have been running a similar centre in Bengaluru since 2021, where 460 paediatric thalassemia patients have received treatment so far.
He also spoke about the "real need" for stem cell donors in India and the lack of it and why Indians must start making their own decisions. Excerpts:
Q/ Tell us about the need for a BMT facility for thalassemia patients in India
A/ Prenatal screening and genetic counselling are crucial tools to prevent thalassemia because it is a genetic disease. Children born to parents who both have thalassemia minor or major, however, have the disease from birth. It can either be moderate or severe and these children often need regular transfusion of haemoglobin to stay alive. Many patients, especially children, who can immensely benefit from the BMT procedure cannot access it and are doomed to a life of suffering. Our BMT centre will continue to offer the service to all paediatric patients with matching donors, irrespective of their financial situation and affordability. BMT is advised as transplant outcomes tend to be better in young children.
Q/ Do you think it is a challenge to find stem cell donors in India? Why?
A/ I would say it's a big challenge to find donors here in India. While 25 per cent patients suffering from blood cancer or other blood disorders will find a donor in the family, 75 per cent will not. Currently, when a patient is not finding a donor in the family, then there is a chance of 17 per cent only to find a donor outside, that is, an unrelated donor.
Q/ 17 per cent is way too less.
A/ Yes. In Germany, the chance is 70 to 80 per cent. In Germany, we have 80 million population and we have 7 million potential donors in our database. Worldwide it's 12.5 million. But, in India, we have only 1.8 lakh. So, we need many more donors to help many more patients.
Q/ India is also the capital of Thalassemia.
A/ In addition to all the blood cancer patients, India is also the capital of thalassemia. When you want to find donors for Indian patients, you have to go to India, because only Indians with Indian ethnicity are fitting to Indian patients. We have something in our DNA which is called HLA type, and these are markers, and only when these markers are matching, these donors can donate. That's why you need a diversity in blood groups, a person of every blood group should donate. But you have more than 10,000 of HLA markers and they all have to be in certain combination for the transplant to be possible. We call this 8 to 8 or 10 to 10 matches. This is very rare, so you need much more donors to find a fitting donor to a particular patient.
Q/ It is also a cultural thing. Even if there is a match, people won't donate.
A/ We have so far registered more than 1,80,000 donors. So, a lot of donors register by peer pressure or under family's. They are motivated. But, what we see is that then the family says no. And so we are talking to a lot of young people and we see that they depend on so many other people to make decisions for them. But, then they say, oh, no, no, speak to uncle. So, we have also doctors who counsel and try to explain the advantages of becoming a donor. But, nevertheless, the availability of these donors is very low. And I hope that more donors who are actually registering with us are also willing to donate.
Q/ What is the criteria for being a stem cell donor and can someone with diabetes too do it?
A/ I don't want people to simply register, rather, I want them to register and then also donate. Imagine you're a patient. Then your doctor says you need a stem cell transplantation. Then this doctor is looking in the database and he says, yes, there is one donor in Mumbai. He gives this information to the patient. Then I will try to get this donor in Mumbai. But suddenly, the donor says no. This way the patient loses the only hope left in the absence of a related donor. So, stem cell donation is a lifetime commitment. When you are scared of needles, and you can't see blood, then it's not a good choice to be a donor.
We undertake complete diagnostic testing before finalising whether you can donate or not. Someone with diabetes can donate stem cells if the sugars are normal, but not if he or she is on insulin. And the health check is all done for free for the donor. The donor has to incur no cost at all, neither for travel nor for the tests. Plus, we are covering also the salary which the donor may lose because it takes 4-5 days for the process.
Q/ How many donations have happened in India since 2019?
A/ More than 100 for sure, from DKMS. With the increase in the database, last year we registered 79,000 donors. As of now, we have 1,80,000 donors. But, of those, the ones who actually donate are a mere 17 per cent.
Q/ Stem cell donation is maximum for which disease?
A/ It is for blood cancer that we have the most number of unrelated donors. This is also because India has more blood cancer patients than any other disease. With Sankalp Foundation, we have the bone marrow transplantation programme that helps paediatric thalassemia patients.
Q/ You also have your own pathological lab in Kolkata.
A/ Yes. Earlier, all our samples would go to Germany but now we can do the next-gen sequencing at our lab in Kolkata. Our lab helps find HLA match for suitable stem cell donors and recipients.
Q/ And the chemotherapy diagnosis?
A/ In India, you have a lot of hospitals, a lot of physicians, and they have a lot of different protocols on how they use chemotherapy for treatment. Cancer treatment. And sometimes, you overdose chemotherapy or you underdose chemotherapy. When you overdose, it leads to other failures in the body. And when you underdose, you get a relapse.
So, the best way is to refer to Measurable Residual Disease (MRD) as it is the most powerful independent predictor of risk of relapse and long-term survival in adults and children with Acute Lymphoblastic Leukaemia (ALL). One can measure what is the amount of bad cells versus good cells in your body. And when the bad cells are becoming too high, you need a new cycle of chemotherapy. When it's low, you may skip one cycle. For almost all patients with ALL, there is a reliable method to evaluate MRD, which can be done using multi-colour flow cytometry, QPCR - quantitative polymerase chain reaction - to detect specific fusion transcripts or immunoglobulin/T-cell receptor gene rearrangements, and high-throughput next-generation sequencing. Our method is next generation sequencing, which is cheaper than the QPCR and also faster.
