IJN and Pfizer Shines the Spotlight on a Rare but Deadly Heart Disease


On May 8 this year, the National Heart Institute (IJN) and Pfizer Malaysia came together to promote greater awareness of transthyretin amyloid cardiomyopathy (often shortened to ATTR-CM), a rare, life-threatening disease.

An overview of ATTR-CM. Click on the image for a larger, clearer version. Image courtesy of Pfizer and IJN.
  • ATTR-CM is caused by unstable transthyretin proteins that misfold and aggregate into amyloid fibrils that build up in the heart and other parts of the body.
  • This build-up of amyloid then causes the heart muscle to stiffen over time, eventually leading to heart failure.
  • Treatment is available, but detection of this disease tends to occur only at advanced stages due to lack of awareness of the disease.

“Diagnosis of ATTR-CM was often delayed or underdiagnosed due to the lack of optimal diagnostic modalities,” says Dato’ Dr Azmee Mohd Ghazi, a consultant cardiologist.


According to Dato’ Dr Azmee:

  • ATTR-CM can lead to progressive heart failure, arrhythmias, and conduction system diseases (diseases that affect the ability of the heart to transmit electrical signals that keep the heart beating), all of which can result in sudden cardiac death due to fatal arrhythmias or complete heart block.
  • Once diagnosed, the average life expectancy for people with ATTR-CM is about 2 to 3.5 years, depending on the sub-type, if left untreated.

“Given how similar ATTR-CM symptoms are to other diseases’ symptoms, getting an accurate diagnosis from the onset can be an uphill battle,” Dato’ Dr Azmee. “However, medical advancements ensure that there are multiple channels to diagnose ATTR-CM, including nuclear scintigraphy and echocardiography.”

He adds that studies have also found that a multidisciplinary approach, involving multiple health departments, works best to facilitate earlier diagnosis and management of the disease.

Symptoms of ATTR-CM, courtesy of Pfizer and IJN. Click on the image for a larger, clearer version.

Symptoms of ATTR-CM are not specific, and they are often similar to symptoms of other conditions such as an abnormal heart rhythm, muscle weakness, and bladder dysfunction.

Nonetheless, one should be alert for these symptoms and consult a doctor for confirmation, as early detection will improve the odds for a better treatment outcome.

Could Stem Cell Therapy Be the Future of Medicine in Malaysia?


Stem cells are a unique type of cell in our body. This is because they are the only type of cells that can develop, under the right triggers and condition, into a variety of more specialized cells, such as blood cells, muscle cells, bone cells, and more.

  • Researchers can generate a variety of human cells from stem cells in the laboratory. These cells are then used to test new medications for possible side effects.
  • Research is also conducted on generating healthy cells that can replace those in our body that are dying or damaged due to age or disease. Such use of stem cells is called regenerative medicine.
  • Hematopoietic stem cells (usually abbreviated as HSCs), which are found in cord blood, bone marrow, and peripheral blood
  • Mesenchymal stem cells (MSCs), which as found in the gel-like substance (Wharton’s jelly) of the umbilical cord, bone marrow, fat tissue, and tooth pulp

Judging from the versatility of stem cells when used to produce of a variety of healthy human cells, regenerative medicine has obvious benefits for people whose tissues are damaged or dying due to diseases as well as people in need of tissue transplant.

Just some of the cells that can be generated from stem cells in the laboratory. Click the image above for a larger version.
Promising for treating heart diseases

Dr Lee Tjen Jhung, a cardiologist affiliated with the National Heart Institute (IJN), says, “While heart diseases are usually treated with medication, angioplasty and by-pass surgery, cell-based regenerative therapy is gaining popularity in the cardiovascular field due to the ability of mesenchymal stem cells to provide new blood in areas affected by restricted blood flow as well as promote the formation of new blood vessels.”

An answer to ageing-related health issues?

Dato’ Dr Rajbans Singh, the President of the Malaysian Wellness Society, points out that stem cells can play a crucial role in treating aging-related health problems.

He elaborates: “At birth, the human body has approximately 80 million active stem cells However, by age 40, less than 25 million active stem cells are functional. Ageing leads to a massive loss of stem cells in the body, affecting the ability of organs and tissues to repair and regenerate as the body gradually ages and deteriorates.”

Here’s a general, simplified overview of how the whole procedure works
  1. Stem cells are grown in a laboratory, and then manipulated to grow and specialize into a more specific type of cells (heart muscle cells, blood cells, etc)
  2. These cells are then implanted into the person that can benefit from this procedure, for example a person with heart disease could be implanted with heart muscle cells, injected into their heart
  3. These transplanted cells will then play a role in repairing and/or replacing the affected damaged tissue or organ of that person.

In reality, the procedure is not so straight forward, as there are many potential complications to take into account.

Nonetheless, the promise of regenerative medicine is too good to resist, hence researchers are continuously looking into creating and fine-tuning various procedures that can hopefully one day reduce or even replace our reliance on medications!


There is considerable ethical debate on the use of stem cells that are sourced from fertilized egg cells or embryos that are donated for research.

These days, many centres that offer stem cell therapy claim to source their stem cells ethically—from the umbilical cords of young and healthy women after they have delivered their babies.

In Malaysia, the sourcing and processing of stem cells are governed by the Current Good Manufacturing Practices or cGMP, issued by the National Pharmaceutical Regulatory Agency (NPRA). You can view it here (link opens in a new tab).