A recent report revealed that cancer has outrun cardiovascular diseases and emerged to be the topmost killer globally. If there’s anything that can serve as a ray of hope, it is the Bone Marrow Transplant (BMT) – at least for some types of cancer. Arabian Gazette had a tete-tete with Dr. Gaurav Gaurav Kharya, a renowned Bone Marrow Transplant specialist Apollo Hospital, New Delhi, to find out the benefits, cost and advancements in BMT.
Here are the excerpts…
How is Bone Marrow Transplant (BMT) helping cure disease and what are the latest trends?
Basically, BMT is bone marrow transplant. It is also called hematopoietic stem cell transplant and some people also call it stem cell transplant. These are basically interchangeable terminologies. Bone marrow transplant, or hematopoietic stem cell transplant, firstly can cure any disease where the bone marrow is not working properly. So, the disease might be affecting the red blood cells. The disease might be affecting the white blood cell population. The disease might be affecting the platelets. Or it might be affecting even the immune system.
So, if you look at the diseases which can be cured by bone marrow transplant, hemoglobin disorders like thalassemia are a bigger chunk here; sickle cell disease; then aplastic anemia; then inherited [bone] marrow failure syndrome, which is bigger spectrum [that] includes all the diseases [where] the bone marrow is not working properly. Then, it can also be used to treat leukemia and other blood cancers. So, this is what bone marrow transplant can treat.
A lot of awareness is growing nowadays. A lot of advancements have also happened. When it comes to bone marrow transplant, obviously, we need a donor, a healthy donor, who can donate a stem cell, just like kidney or bone marrow transplant.With the techniques that we had earlier, only a sibling, a brother or sister, who is a complete genetically identical, was constituted the ideal good donor with good results. But the biggest problem with that was that almost 75 percent of patients who needed transplant were not able to get a transplant because the genetic match donor was not available.
But in the last decade or two, we’ve made significant progress in doing the donated transplants like half-matched transplant. In Half-matched transplant, the genetic matching is not complete, it is less than 90 percent. So, like I have a small family. It’s me, my brother, my parents, we are four. Suppose I need a transplant and my brother is not a match, then, we can explore my parents also? So, we will be having three potential half-matched donors. The outcomes of half-matched transplants also have improved significantly, and, for majority of diseases, close to 90 percent are solved.
In short, any child or any patient who needs a transplant nowadays can undergo the procedure with very good outcomes in centers with experience in treating all these diseases.
Just to clarify on that, is stem cell therapy and bone marrow transplant similar?
Yeah, this is a valid question. People often confuse these two things. Stem cell therapy and stem cell transplant, these are two different things. Stem cell transplant, which we are talking about today, is an established modality to treat various diseases. It is a standard of care. But when we talk about stem cell therapy, stem cell therapy is experimental. It is the use of cells, which are not specific to form blood components. Those cells are used to treat various diseases like cerebral palsy, or cardiomyopathy, or diabetes, or other various conditions. This is experimental modality. It is not a standard of care. It should not be done outside the experimental setting.
Recently, you have performed BMT on a one-month-old baby. Can you share the details?
Yeah. This little girl who was the first born to parents who are in consanguineous marriage – wedding within the family.This girl, she was the first born and was doing well initially. Then after one month and a half, the parents noticed that she’s getting pale. Her color was not pink. So, they took her to the doctor and it was figured out that her hemoglobin was very low, her white cell count was very low, her platelets were very low. Also, they also noticed that her eyes, she was not able to fix the gaze. On further evaluation, she was diagnosed as a case of Osteopetrosis. This disease have various mutations, not all can be cured by BMT. But there are certain mutations, if intervened early through bone marrow transplant, we can cure the disease. Fortunately, she had a mutation which was borderline and could be treated
She was presented to us at three months of age. By that time, the damage was not quite a lot, so we did a transplant. This was the first born so there was no match donor available. So, we screened both parents and then we chose [one] parent as the donor, the half-matched donor. We did the transplant using the mother as the donor. [We used] a different technique called (T cell depleted) transplant.
Fortunately, it worked well and it’s almost nine months post-transplant. Her bone marrow looks normal. Her vision also is slightly getting better. Her neurological improvement has also been significant.
So, there is no age limit for BMT?
There isn’t any age limit. The earliest we’ve done is around two or three months of age and the oldest depends on the condition of the patient.
What are the signs and symptoms of cancer?
It varies from each patient.Some patients who are presented with cancer are presented with fever or color becomes pale. They might have bleeding manifestations here or there. For other diseases like aplastic anemia, they can also present almost similar kind of phenomena. Or some diseases like immunodeficiency diseases, the kids are born and then soon after their birth, within a month, they start having infections, because they do not have immunity. They were born with immunodeficiency. The presented symptoms can vary, depends upon the condition of the patient.
For parents, any problem extending beyond a certain time like five days should not be ignored and it should be brought to medical attention and proper medical evaluation should be done.
Do you see increasing rate of prevalence of cancer among kids and, what according to you are the main reasons for it?
I am seeing an increasing trend in cancers, not only in pediatrics, but overall in others as well in this part of the world. The reason being multifactorial. First, the awareness is going on. Several patients initially were not diagnosed. They used to die before they reached the medical facility without a proper diagnosis. Nowadays, majority of diagnosis, people, patients are diagnosed.
Secondly, because of the exposure to several chemicals, fertilizers, among other things. Because they are getting exposed to [them], the chances of getting cancer is much more. So, because of all these factors, we feel that the chances or the incidence of cancer is going [up] in this part of the world.
Parents who are healthy are often confused like in kids, they don’t have any such life style issues like smoking or chewing tobacco, then why do they get cancer. The incidence of each cancer is different.
What is the cost for a typical BMT?
The cost for a typical BMT is usually around USD 30,000 to USD 35, 000. This includes the stay in the hospital and the fee transplant. When we talk about the half-matched transplant, the cost goes up by around 15 to 20 percent because we have to do it in a different way. More planning is required. For several indications, we immediately start planning almost a month or two months earlier. So, it’s around 15-20 percent more for a half-matched transplant.
If you talk about unrelated donor transplant, the cost of unrelated donor transplant depends a lot on from where we are getting the unrelated donor. So, if the unrelated donor, we get it from the German Registry, it will cost around 8,500 euros. But if we get the donor from MDPT or the UK Registry, the cost can be around USD 45,000 or 30,000 – 35,000 pounds. So, in cases of unrelated donor transplant, apart from the initial cost of the transplant, which is around USD 35,000, the additional cost depends on registry.
Do you see any particular pattern of diseases or prevalence in the UAE which BMT might be of help?
No offense meant but in this community because of marriages within the family, there are many health conditions prevailing. Immunodeficiency is very, very common, and [for] majority of immunodeficiency, transplant is the only solution.They must be treated as a medical emergency. We can’t wait a year or two to do the transplant. As soon as the diagnosis is made, if the transplant is done within three months, then the outcome is excellent. If we delay, then the outcome goes down. So, primary immunodeficiencies, metabolic disorders, hemoglobin disorders like thalassemia or sickle cell disease can be treated with BMT.
So, my message is this only that pediatric ailments where bone marrow transplant can be offered as curative modality, the success rate is improving significantly. For parents, they should not have this apprehension that if we don’t have a proper donor, a sibling donor, who is genetically matched, to go ahead with the transplant. But the progress we have made in the past decades, the outcomes are excellent. The key thing in deciding the outcome is how soon the patient is presented to the facility. If the patient is presented at early stages of the disease, then obviously the outcomes are much better compared to the same child, same patient at the late stages of the disease. So, these are the messages that I want to convey to the parents, to the general public. Consult a doctor as soon as you see a problem.