ASH News Daily 2017 - Issue 3 - B-46

ASH News Daily

Page B-46

Monday, December 11, 2017

Drs. Luigi Naldini and Marina Cavazzana
to Present the 2017 Ernest Beutler Lecture Today

Naldini and Cavazzana will
his afternoon, Luigi
present the topic, "Turning
Naldini, MD, PhD,
Foes Into Friends: Exploiting
of the San Raffaele
HIV to Generate Efficient
Telethon Institute for Gene
and Safe Vectors to Cure InTherapy in Milan, and Maherited and Acquired Blood
rina Cavazzana, MD, PhD,
Diseases."
of Paris Descartes UniverDr. Naldini is Director of
sity, will be awarded the
the San Raffaele Telethon
Ernest Beutler Lecture and
Institute for Gene Therapy
Prize in Basic Science and Marina Cavazzana, MD, PhD Luigi Naldini, MD, PhD
in Milan, and is Professor of
Clinical Science, respectively. This award is named for the for more than 50 years. Each year Cell and Tissue Biology and Gene
late Dr. Ernest Beutler, past presi- this two-part lecture recognizes ma- and Cell Therapy at the Vita Salute
dent of ASH and physician-scientist jor translational advances, and Drs. San Raffaele University School of

MCL-1, a BCL-2 family member, is an

in MCL-1-dependent AML1-3

To learn more about MCL-1, visit
us at the ASH Annual Meeting
at Booth #503

A matter of cell life
and cell death

Learn more at www.toleropharma.com

Tolero Pharmaceuticals, Inc. is a leader in developing novel therapeutics to inhibit biological drivers of
hematologic and oncologic diseases by targeting pathways for gene transcription and drug resistance.

References: 1. Glaser SP, Lee EF, Trounson E, et al. Anti-apoptotic Mcl-1 is essential for the development and sustained growth of acute myeloid leukemia. Genes Dev. 2012;26(2):120-125.
2. Xiang Z, Luo H, Payton JE, et al. Mcl1 haploinsufficiency protects mice from Myc-induced acute myeloid leukemia. J Clin Invest. 2010;120(6):2109-2118. 3. Perciavalle RM, Opferman JT. Delving deeper:
MCL-1's contributions to normal and cancer biology. Trends Cell Biol. 2013;23(1):22-29.
Tolero Pharmaceuticals is a registered trademark of Tolero Pharmaceuticals, Inc. in the US. ©2017 Boston Biomedical, Inc. All rights reserved. PM-ALV-0002 10/2017

20299_M06_ALV_MCL1_ASHNewsDaily_IslandAd.indd 1

11/1/17 11:31 AM

Medicine in Milan. "It was a great
pleasure to hear I'd been selected,"
he said. "I am honored by the recognition of my effort, and that of a large
team of people. This award recognizes the entire field of gene therapy,
which has long been plagued with
skepticism as to whether it could really provide benefit to patients."
While recognizing that great challenges remain, Dr. Naldini is proud
of the work he and his collaborators
have accomplished, especially the
success with the first clinical testing
of an HIV-derived vector in metachromatic leukodystrophy - a rare
and deadly disease in children. This
was the first use of hematopoietic
stem cell (HSC) gene therapy using
the new vector, and as he explains,
"I found patients, I would dare to
say, cured of the disease as long as
they could be treated before symptom onset."
Dr. Cavazzana is a Professor of
Hematology at Paris Descartes University, Head of the Department
of Biotherapy at Hospital NeckerEnfants Malades, and Chief of Research of the Laboratory of Human
Lympho-hematopoiesis at the Institut Imagine in Paris. Today, she will
review the current stage of clinical
application and benefit of HSC gene
transfer by lentiviral vectors.
After years of hard work and
dedication, Dr. Cavazzana takes a
great deal of pride in the work she
has done with colleagues, especially
her research with Professor Philippe
Leboulch around the treatment of
patients with sickle cell disease. "For
me it represents a great breakthrough
toward a wider use of gene therapy
to treat patients with sickle cell disease because it is a worldwide problem, and I am optimistic that we can
use this approach, given its ongoing
progress, to treat many patients."
Like Dr. Naldini, Dr. Cavazzana
acknowledges that challenges remain in this area of research, but she
describes an optimistic view of the
future. "My idea is to develop an automatic process using the appropriate machines with a standard program that can be used everywhere,
in any hospital around the world, to
perform autologous bone marrow
transplantations." She advises those
considering setting out on the journey of hematology to "go ahead. Be
strong and creative, and you will be
successful."
Join your colleagues this afternoon at 1:30 p.m. in Hall C2-C3 of
the Georgia World Congress Center.
If you miss today's presentation, be
sure to pick up ASH News Daily's
Tuesday issue for complete coverage.

Table of Contents for the Digital Edition of ASH News Daily 2017 - Issue 3