Author and Filmmaker's Death Followed Familiar Course

The death this week of author and filmmaker Nora Ephron has brought an outpouring of grief from around the world. Famous for having written films such as When Harry Met Sally, Ephron had been diagnosed with myelodysplastic syndrome (MDS) some time ago and had been receiving treatment.

MDS is actually a large group of similar diseases of the blood and the bone marrow that can be mild or moderate or severe, depending on the individual. It is not uncommon for MDS to develop as a consequence of previous anti-cancer treatment, but oftentimes it develops on its own.

In some instances, as was the case with Ephron, MDS -- which at one time was referred to not as MDS but as pre-leukemia -- can develop into acute myelogenous (or myeloid) leukemia (AML), and at that point the prognosis is typically not good.

AML is an aggressive hematologic cancer that begins in the marrow cells, caused by changes in the cell's DNA.

Treatments for MDS are few and not very effective. The Leukemia & Lymphoma Society is among many groups currently exploring new treatments for this patient population, and they are funding a Phase III trial for rigorsertib (Estybon) developed by Onconova Therapeutics.

Rigorsertib inhibits multiple molecular processes that are crucial in the growth and survival of these cancer cells. Ideally a new MDS treatment would cut down on the need for MDS patients to receive blood transfusions, and ideally the new treatment would make the development to AML less and less likely.

Source: LLS