PANKAJ MALHOTRA, MDChandigarh, India

• Associate Professor, Department of Internal Medicine Pgimer, Chandigarh, India

• Dr. Pankaj Malhotra received his medical degree from Punjab University and his training in internal medicine at PGIMER, Chandigarh, India. He has several invited and peer reviewed publications. Dr. Malhotra was awarded the CML Fellowship by International CML Foundation (iCMLF) to Knight Cancer Centre, Oregon Science and Health University, Portland, USA. Areas of Interest include leukemia, stem cell transplant and myeloma.

Novel treatmentsPankaj Malhotra, MD, FACP

Professor of Clinical Hematology

Dept. of Internal Medicine, PGIMER, Chandigarh, India

Normal production of globinchains

Hallmark of b thal

Produce b chains:1. Stem celltransplantation2. Gene therapy3. antisense molecules to silence mRNA splicing4. Induced pluripotent stem cells

Increase the g chain production:1. Drugs2. Gene therapy

Decrease a chain production

Gene therapy

1. Selection of virus2. Technique

1. How long will it work

2. Long term side

effects

1. Selection of virus2. Technique

1. Selection of virus2. Technique

1. How long will it work

2. Long term side

effects

iPSC

Caution!

Silence Hb F suppression

Increase the g chain production

1. Drugs2. Gene therapy

Silence Hb F suppression

Drugs for increasing Hb F

Other drugs for increasing Hb F

Conclusions on novel (not so) treatments in thalassemia

b globin production

• Gene therapy

• Antisense molecules to alter RNA splicing

• iPS cells

Upregulation of gamma globin production

• Drugs (HbF production)

• Upregulation of genes