What is Thalassemia?

From Gilgit-Baltistan to London, Dr. Sher Bahadur Anjum proves that a Pakistani doctor can rewrite the story of thalassemia

Thalassemia is a hereditary blood disorder where the body fails to produce enough healthy hemoglobin. Hemoglobin is the protein in red blood cells that carries oxygen throughout the body and gives blood its red color. In thalassemia, defective genes result in reduced or abnormal hemoglobin, causing red blood cells to break down quickly and leading to severe anemia.

Why is it Dangerous?

Patients often need regular blood transfusions every few weeks to survive. Over time, this creates a dangerous condition called iron overload, where excess iron damages the heart, liver, and bones. Managing thalassemia is not just exhausting but life-threatening in the long run.

Current Treatments

So far, most patients depend on lifelong blood transfusions and iron-chelating medicines. In some cases, a bone marrow transplant (BMT) is used, but it is expensive, risky, and requires a perfectly matched donor. This makes it inaccessible for many families, especially in developing countries.

A Breakthrough: Gene Therapy

Recently, a revolutionary success in the UK has brought hope. Two children (aged 11 and 13), both dependent on monthly transfusions, received gene therapy and no longer need blood to survive. They now live healthy, normal lives.

What is Gene Therapy?

Gene therapy directly corrects the faulty DNA in the patient’s blood-forming stem cells. Doctors first remove these cells, repair or replace the defective gene using advanced biotechnology, and then return the corrected cells into the patient’s body. Once inside, the cells start producing healthy hemoglobin, eliminating the need for transfusions.

This treatment is still extremely costly and only available in a few hospitals, but it is a ray of hope for millions worldwide.

A Pakistani Doctor’s Historic Role

Behind this breakthrough is Dr. Sher Bahadur Anjum, a British-Pakistani doctor originally from Gilgit-Baltistan. Working with London’s Newham Hospital and Great Ormond Street Hospital, he successfully treated these children. His achievement shows the world that Pakistani doctors are making groundbreaking contributions on a global level.

The Road Ahead

Thalassemia remains a deadly challenge, but modern science is opening doors once thought impossible. If gene therapy becomes affordable and widely available, it could save millions of lives.

By: Shakir Ullah | Sep 17 2025