It is a common belief that bone marrow transplants can successfully treat cancers like leukemia and lymphoma – but not many people know that they are also surprisingly used to treat various other conditions, a few of which also include genetic and autoimmune diseases, apart from rare metabolic disorders.

According to experts, the technology can also be used in cases of severe aplastic anemia or bone marrow failure.

Bone marrow transplant, or BMT, also known as hematopoietic stem cell transplantation, is becoming a potentially life-saving therapy for many different non-cancerous conditions, many of which are often not recogn, in India and around the world.

“What makes HSCT effective beyond cancer is the central role hematopoietic stem and progenitor cells play in blood formation, immune function, and cellular metabolism. In inherited immune deficiencies, replacing a faulty immune system with a healthy one can restore resistance to infections,” Dr. Rasmi Palassery, Consultant, Pediatric Oncology, Ramaiah Institute of Oncosciences, Ramaiah Memorial Hospital, told Times Now.

According to Dr. Palassery, in storage and metabolic disorders, where toxic substances accumulate in cells due to enzyme deficiencies, donor-derived cells deliver the missing enzyme systemically. Even in some mitochondrial and genetic disorders, transplanting healthy stem cells can mitigate disease progression by introducing normal metabolic or structural cell components. “This broad therapeutic potential explains why HSCT is being used to treat a growing number of non-malignant diseases worldwide,” she added.

A few of the surprising diseases, other than cancer, treated using bone marrow transplants include:

Thalassemia major, or β-thalassemia, is an autosomal recessive disorder characterised by absent or severely deficient synthesis of β-globin chains of hemoglobin resulting in severe anemia that requires lifelong blood transfusions. It is one of the most common indications for BMT in Indian children. “Data from Indian transplant centers demonstrate that about 35 per cent of pediatric allogeneic transplants have been performed for thalassemia,” said Dr. Palassery.

The growing success rates of haploidentical HSCT have expanded access to patients lacking matched donors, significantly improving outcomes in India. However, despite this progress, a substantial gap remains between the number of procedures performed and the estimated 100,000 patients eligible for transplant in India. This underscores a significant growth potential and the urgent need for expanded transplant access.

Aplastic anemia is a condition where the bone marrow does not produce sufficient blood cells and is another major non-cancerous indication for BMT in India. Scientific studies suggest that inherited causes of aplastic anemia, such as Fanconi anemia and other inherited bone marrow failure syndromes, may be more prevalent in Asian populations, including India, than in Western countries.

Doctors say early diagnosis of these inherited forms is essential because they often require specialized management and have distinct genetic counselling implications. Recent data from various transplant centres in India demonstrate that survival outcomes for aplastic anemia patients in India are now comparable to global standards, reflecting advances in diagnosis, supportive care, and transplant expertise.

Sickle cell disease is prevalent in parts of central and western India, where the condition causes recurring pain crises, infections, and progressive organ damage. BMT is currently the only curative treatment for this inherited disorder. Increasingly, Indian hospitals are offering BMT to children and young adults, allowing many of them to return to a normal and pain-free life.

In addition to BMT, gene therapy for sickle cell disease has recently shown promising results, having cleared phase 3 clinical trials internationally. Although gene therapy is not yet widely available in India, there are ongoing clinical trials exploring its safety and effectiveness. This emerging treatment could potentially offer a less invasive curative option in the future.

Children born with severe primary immunodeficiency disorders such as Wiskott-Aldrich syndrome or severe combined immunodeficiency (SCID) face lifelong struggles with life-threatening infections. BMT can restore effective immune function. Several Indian centers have documented BMT for these rare disorders, resulting in improved quality of life and survival.

Recent evidence from India and centers worldwide indicates that BMT can reprogram the immune system in treatment-resistant autoimmune diseases such as multiple sclerosis, lupus, and systemic sclerosis. In these cases, autologous hematopoietic stem cell transplantation (auto-HSCT), which uses the patient’s stem cells, is typically performed.

The process involves harvesting the patient’s stem cells, followed by high-dose chemotherapy or immunosuppressive treatment to eliminate the malfunctioning immune cells. The harvested stem cells are then reinfused to "reset" the immune system, promoting the development of a new, healthier immune profile that no longer attacks the body’s tissues. While still considered experimental in certain scenarios, early results are promising.