Multiple myeloma

Multiple myeloma arises when plasma cells, white blood cells that help fight infections, begin to develop abnormally. Those cells crowd out the healthy cells in your bone marrow and may also form tumors. Over time, they can cause a wide range of symptoms, from calcium build-up and kidney problems to anemia, bone lesions and fractures.

What is Multiple Myeloma?

Multiple myeloma is a cancer of plasma cells, a type of white blood cell found in the bone marrow. Healthy plasma cells create antibodies in response to infections and are an important part of the immune system. When plasma cells become cancerous, they can crowd out normal white blood cells, red blood cells and platelets, all of which are made in the bone marrow. The disease is called “multiple” because it often affects several areas of the body, and “myeloma” because it affects the bone marrow.

Multiple myeloma can begin with no symptoms, a condition called “smoldering” myeloma, and if symptoms arise, that is considered active myeloma.

Bausch Health Companies (BHC)

Formerly known as Valeant Pharmaceuticals, Bausch Health is a global specialty pharmaceutical, consumer health, and medical device company with a focus on branded products for the dermatology, gastrointestinal, and ophthalmology markets. The company reports revenue in five segments: Bausch+Lomb (45% of revenue), Salix Pharmaceuticals (24%), international pharma (14%), ortho dermatology (7%), and diversified products (10%). Bausch is the market leader in consumer visioncare in India and China and the fourth-largest visioncare company by sales in the United States.

 

Gene therapy

There are two main types of gene therapy, a method of shuttling genetic instructions into the body for a one-time, long-lasting disease treatment.

One method is an in vivo process, in which a therapeutic gene is introduced into the patient’s body. The other is the ex vivo variety, in which patients’ stem cells are harvested, equipped with a healthy version of a gene, and then infused back into the body. Everything remains experimental, but in vivo therapy is currently the more common approach, pursued by companies such as Spark Therapeutics (NASDAQ: ONCE), BioMarin Pharmaceutical (NASDAQ: BMRN), and UniQure (NASDAQ: QURE).

Less common is the ex vivo procedure used by companies such as Bluebird Bio (NASDAQ: BLUE), U.K.-based startup Orchard Therapeutics, and Boston-area startup Avrobio. Through this process, a virus from the lentivirus family that has been genetically modified to be harmless carries healthy genes into stem cells harvested from the patient. The stem cells are then grown in a culture and infused back into the patient, where they are supposed to give rise to more cells with the correct gene.