Bone Marrow Transplant News and Research

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Researchers to test use of ‘viral specific’ T-cells to target toxic viruses in children

Michael Pulsipher, MD, of the Children's Center for Cancer and Blood Diseases at Children's Hospital Los Angeles, along with Michael Keller, MD from Children's National Health System in Washington, DC, have been awarded $4.8 million by the California Institute for Regenerative Medicine to study the use of a new T-cell therapy to help fight active viral infections in children with severe immune deficiencies.

7 Nov 2017

Conference to focus on preventing and reversing consequences of sickle cell disease

Leading experts in the field of sickle cell disease (SCD) research will convene in Washington, D.C., for the Physiological and Pathophysiological Consequences of Sickle Cell Disease conference (November 6–8). The conference, organized by the American Physiological Society, will explore SCD- the world's most prevalent single-gene mutation disease-; and new research on preventing and reversing its deadly consequences.

1 Nov 2017

Cascade of costs could push new gene therapy above $1 million per patient

Outrage over the high cost of cancer care has focused on skyrocketing drug prices, including the $475,000 price tag for the country's first gene therapy, Novartis' Kymriah, a leukemia treatment approved in August.

17 Oct 2017

Study to determine lowest dose of chemotherapy needed for babies with SCID

Michael Pulsipher, MD, of the Children's Center for Cancer and Blood Diseases at Children's Hospital Los Angeles, along with co-principal investigator, Sung-Yun Pai, MD, of Boston Children's Hospital, has been awarded nearly $9 million from the National Institute of Allergy and Infectious Diseases of the NIH to study a new treatment approach for babies born with severe combined immunodeficiency (SCID) which prevents the immune system from functioning normally.

6 Oct 2017

Moms of children with rare genetic illness push for wider newborn screening

Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler had a rare brain disorder.

5 Oct 2017

Study explores periodontal status of patients with Chédiak-Higashi syndrome

The International and American Associations for Dental Research have published an article titled "Periodontitis in Chédiak-Higashi Syndrome: An Altered Immunoinflammatory Response" in the JDR Clinical & Translational Research.

31 Aug 2017

UChicago Medicine working with Novartis to offer breakthrough gene therapy for pediatric ALL patients

The University of Chicago Medicine is one of a limited number of U.S. sites working to offer a breakthrough gene therapy for pediatric acute lymphoblastic leukemia (ALL), which was just approved by the U.S. Food and Drug Administration.

31 Aug 2017

Targeting bone marrow protein could be effective way to improve stem cell transplants

Bone marrow contains hematopoetic stem cells, the precursors to every blood cell type. These cells spring into action following bone marrow transplants, bone marrow injury and during systemic infection, creating new blood cells, including immune cells, in a process known as hematopoiesis.

28 Aug 2017

UC San Diego researchers receive $18.2 million grant for B cell cancer clinical trial

The Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine today unanimously approved an $18.29 million grant to University of California San Diego School of Medicine researchers to fund a phase Ib/IIa clinical trial of a novel combination drug therapy for B-cell cancers.

26 Aug 2017

Our body contains more diverse microbes than previously known, new survey suggests

A new survey of DNA fragments circulating in human blood suggests our bodies contain vastly more diverse microbes than anyone previously understood.

23 Aug 2017

‘Breakthrough’ leukemia drug also portends ‘quantum leap’ in cost

When doctors talk about a new leukemia drug from Novartis, they ooze enthusiasm, using words like "breakthrough," "revolutionary" and "a watershed moment."

23 Aug 2017

FDA approves fixed dose combination chemotherapy for subtypes of AML that have poor survival outcomes

The U.S. Food and Drug Administration (FDA) yesterday approved Vyxeos - a fixed dose combination chemotherapy for the treatment of adults with two types of acute myeloid leukemia (AML). The types of AML that would be treated with Vyxeos include newly diagnosed therapy-related AML (t-AML) and AML with myelodysplasia-related changes (AML-MRC). Vyxeos is a fixed-combination of the chemotherapy drugs daunorubicin and cytarabine.

3 Aug 2017

FDA gives its nod to new drug Imbruvica (Ibrutinib) for chronic Graft versus Host Disease

The U.S. Food and Drug Administration (FDA) yesterday in a press release informed about the approval of Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) who have previously failed with one or more treatment. This is the first drug that the FDA has given its approval to in this disease condition.

2 Aug 2017

$New drug Idhifa for patients with refractory acute myeloid leukemia (AML) gets FDA nod$

New drug Idhifa for patients with refractory acute myeloid leukemia (AML) gets FDA nod

The U.S. Food and Drug Administration (FDA) in a statement yesterday reported its approval for a new drug, Idhifa (enasidenib) for the treatment of acute myeloid leukemia (AML) in adults that has either relapsed or is refractory to standard chemotherapy.

2 Aug 2017

Latinos left out of clinical trials … and possible cures

Two decades ago, Luis Antonio Cabrera received devastating news: He likely had only three months to live.

19 Jul 2017

Seattle Children’s opens first clinical trial to study IBD and GVHD in pediatric patients

In an effort to find new strategies to personalize treatment for pediatric patients, Seattle Children's has opened the first clinical trial applying next-generation T-cell receptor sequencing and single-cell gene expression analysis to better understand how the immune system drives both inflammatory bowel disease in pediatric autoimmunity patients and graft-versus host disease in pediatric bone marrow transplant patients.

From 10x Genomics, Inc. 23 May 2017

Hematologist-oncologist to lead bone marrow transplantation program for treating blood-borne cancers

Hematologist-oncologist Ahmad Samer Al-Homsi MD, MBA, will lead a new bone marrow transplantation program at NYU Langone's Perlmutter Cancer Center for treating blood-borne cancers, including leukemia, lymphoma and multiple myeloma, and potentially utilize transplantation as an adjunct to immunotherapy for solid tumors.

10 Apr 2017