The Aga Khan University’s (AKU) Centre for Regenerative Medicine and Stem Cell Research has won a Rs 14.5 million (US$ 90,324) grant to develop new, targeted therapies for a common form of blood cancer in adults, Philadelphia chromosome positive (Ph+) leukaemia.
The chromosomal defect known as Philadelphia chromosome (Ph) is a characteristic of two important types of blood cancer: chronic myeloid leukaemia (CML) and Ph positive acute lymphoblastic leukaemia (Ph+ ALL). The Ph chromosome is formed when chromosome 9 and 22 break and exchange portions, which leads to creation of a new fusion gene called BCR-ABL. This fusion causes certain blood cells to become cancerous.
It is the main cause of the common, CML, in adults and accounts for 20 percent of (Ph+ ALL) cases globally.
Generally, Ph+ ALL and advanced forms of CML are difficult to cure and survival rates are poor. AKU researchers are looking to develop a more effective treatment by engineering stem cells to deliver a protein with therapeutic properties, the Helix 2 peptide, directly to the leukaemia cells.
“We aim to combine approaches in molecular medicine and stem cell therapy to create a more precise form of treatment that has the potential to be more convenient for the patient since it will only require a single administration,” says Dr Afsar Mian, the principal investigator of the study.
“We are anticipating that once introduced, the stem cells will grow and multiply in the body and continuously release the therapeutic peptide to target the cancer cells, inhibit their growth and ultimately cure the disease.”
Existing medicines for Ph+ leukaemia are currently proving to be less effective due to growing drug resistance and the problem of side effects. Also, they require life-long use by the patient and do not lead to complete eradication of the disease.
This innovative use of stem cells will also generate evidence on the use of peptides to provide targeted treatment in other types of cancers. In the future, Dr Mian and his team seek to combine their expertise in cancer research as well as drug design and delivery to see if such approaches can be used to treat other diseases.
The grant was awarded by the Higher Education Commission of Pakistan’s flagship National Research Programme for Universities that seeks to support high-level and promising scientific research projects that demonstrate strategic relevance and impact to local industry and society.
This article was adapted from a story published on the Aga Khan University’s website.