Leukemia Research Trials.

Total Therapy for Infants With Acute Lymphoblastic Leukemia (ALL)

Description – This is a phase I/II trial which studies the side effects and best dose of vorinostat and to see how well it works when given together with bortezomib and combination chemotherapy in treating infants with newly diagnosed acute lymphoblastic leukemia. Bortezomib and vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as methotrexate, hydrocortisone, and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing or by stopping them from spreading. The trials main motive is to see if giving more than one drug with bortezomib and vorinostat may be a better treatment for acute lymphoblastic leukemia.

Lead Organization– St. Jude Children’s Research Hospital

Principal Investigator – Tanja A. Gruber

Location

  • California, Los Angeles, Children’s Hospital Los Angeles, Contact: Paul S. Gaynon, Phone: 323-361-2163 Email: pgaynon@chla.usc.edu
  • Orange, Children’s Hospital of Orange County, Contact: Ivan I. Kirov, Phone: 714-997-3000 Email: ikirov@choc.org
  • Palo Alto, Lucile Packard Children’s Hospital Stanford University, Contact: Norman James Lacayo, Phone: 650-497-8953 Email: lacayon@stanford.edu
  • San Diego, Rady Children’s Hospital-San Diego, Contact: Deborah E. Schiff, Phone: 858-966-5934 Email: dschiff@chsd.org
  • Minnesota, Minneapolis, Children’s Hospitals and Clinics of Minnesota – Minneapolis, Contact: Michael Kerr Richards, Phone: 612-813-5193 Email: michael.richards@childrensmn.org
  • North Carolina, Charlotte, Saint Jude Affiliate-Charlotte, Contact: Christine Marie Bolen, Phone: 704-384-1900 Email: cybolen@novanthealth.org
  • Ohio, Cincinnati, Cincinnati Children’s Hospital Medical Center, Contact: Erin Haag Breese, Phone: 513-636-2799 Email: erin.breese@cchmc.org
  • Cleveland, Rainbow Babies and Children’s Hospital, Contact: Yousif (Joe) H. Matloub, Phone: 216-844-5437 Email: yousif.matloub@uhhospitals.org
  • Oregon, Portland, Oregon Health and Science University, Contact: Bill Hoon Chang, Phone: 503-494-1080 Email: trials@ohsu.edu
  • Tennessee, Memphis, St. Jude Children’s Research Hospital, Contact: Tanja A. Gruber, Phone: 901-319-0070 Email: Tanja.Gruber@stjude.org
  • Virginia, Norfolk, Children’s Hospital of The King’s Daughters, Contact: Eric Jeffrey Lowe, Phone: 757-668-7243 Email: eric.lowe@chkd.org

 

Selinexor in Treating Younger Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myeloid Leukemia

Description – This phase I trial studies the side effects and the best dose of selinexor in treating younger patients with acute lymphoblastic leukemia or acute myeloid leukemia that has returned or has become resistant to standard therapies. Selinexor may prevent leukemia cells from growing and may lead to the destruction of leukemia cells.

Lead Organization – Dana-Farber Harvard Cancer Center

Principal Investigator – Andrew Elliott Place

Locations

  • California, San Francisco, UCSF Medical Center-Parnassus, Contact: Elliot Stieglitz Email: elliot.stieglitz@ucsf.edu
  • Colorado, Aurora, Children’s Hospital Colorado, Contact: Lia Gore, Phone: 720-777-6672 Email: Lia.gore@ucdenver.edu
  • Georgia, Atlanta, Children’s Healthcare of Atlanta – Egleston, Contact: Melinda Gordon Pauly Email: melinda.pauly@choa.org
  • Massachusetts, Boston, Boston Children’s Hospital, Contact: Andrew Elliott Place, Phone: 617-632-2313 Email: andrew_place@dfci.harvard.edu
  • Dana-Farber Cancer Institute, Contact: Andrew Elliott Place, Phone: 617-632-2313 Email: andrew_place@dfci.harvard.edu
  • New York, New York, Columbia University / Herbert Irving Cancer Center, Contact: Maria Luisa Sulis Email: mls95@cumc.columbia.edu
  • Pennsylvania, Philadelphia, Children’s Hospital of Philadelphia, Contact: Richard Aplenc Email: raplenc@mail.med.upenn.edu
  • Texas, Houston, Texas Children’s Hospital, Contact: Rachel E. Rau Email: rerau@bcm.edu
  • Washington, Seattle, Seattle Children’s Hospital, Contact: Todd Michael Cooper, Phone: 404-785-1838 Email: Todd.cooper@seattlechildrens.org
  • Wisconsin, Milwaukee, Children’s Hospital of Wisconsin, Contact: Michael James Burke, Phone: 414-955-4198 Email: mmburke@mcw.edu

 

Risk Classification Schemes in Identifying Better Treatment Options for Children and Adolescents with Acute Lymphoblastic Leukemia

 

Description – This randomized phase III trial studies risk classification schemes in identifying better treatment options for children and adolescents with acute lymphoblastic leukemia. Risk factor classification may help identify how strong treatment should be for patients with acute lymphoblastic leukemia.

Lead Organization – Dana-Farber Harvard Cancer Center

Principal Investigator – Lewis Barry Silverman

Locations

  • Massachusetts, Boston, Boston Children’s Hospital, Contact: Lewis Barry Silverman, Phone: 617-632-6191 Email: Lewis_Silverman@dfci.harvard.edu
  • Dana-Farber Cancer Institute, Contact: Lewis Barry Silverman, Phone: 617-632-6191 Email: Lewis_Silverman@dfci.harvard.edu
  • New York, Bronx, Montefiore Medical Center-Weiler Hospital, Contact: Lisa Gennarini, Phone: 718-741-2342 Email: Lfigueir@montefiore.org
  • Buffalo, Roswell Park Cancer Institute, Contact: Kara M. Kelly, Phone: 716-845-2333 Email: Kara.Kelly@RoswellPark.org
  • New York, Columbia University / Herbert Irving Cancer Center, Contact: Maria Luisa Sulis, Phone: 212-305-5808 Email: Mls95@cumc.columbia.edu
  • Rhode Island, Providence, Rhode Island Hospital, Contact: Jennifer J. Greene Welch, Phone: 401-444-5171 Email: jwelch@lifespan.org

Selinexor, Fludarabine Phosphate, and Cytarabine in Treating Younger Patients with Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndromes

Description – This phase I / II trial studies the side effects and best dose of selinexor when given together with fludarabine phosphate and cytarabine in treating younger patients with acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes that did not go into remission after treatment or has come back after treatment. One way cancer cells continue to grow by escaping from mechanisms that normally control human cell growth, such as a type of protein called a tumor suppressor protein. Tumor suppressor proteins normally cause cancer cells to die. Selinexor works by trapping tumor suppressor proteins within the cancer cells, causing them to stop growing or die. Fludarabine phosphate and cytarabine are drugs used in chemotherapy that stop cancer cells from dividing. Giving selinexor with fludarabine phosphate and cytarabine may work better in treating acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes in younger patients.

Lead Organization – St. Jude Children’s Research Hospital

Principal Investigator – Jeffrey E. Rubnitz

Locations

  • Arizona, Phoenix, Phoenix Children’s Hospital, Contact: Jessica Boklan, Phone: 602-667-5669 Email: jboklan@phoenixchildrens.com
  • California, Palo Alto, Lucile Packard Children’s Hospital Stanford University, Contact: Norman James Lacayo, Phone: 650-497-8953 Email: lacayon@stanford.edu
  • Illinois, Chicago, University of Chicago Comprehensive Cancer Center, Contact: Jennifer Lynn McNeer, Phone: 773-702-6808 Email: jmcneer@peds.bsd.uchicago.edu
  • Tennessee, Memphis, St. Jude Children’s Research Hospital, Contact: Jeffrey E. Rubnitz, Phone: 901-216-1878 Email: jeffrey.rubnitz@stjude.org,
  • Texas, Fort Worth, Cook Children’s Medical Center, Contact: Kenneth Matthew Heym, Phone: 682-885-4007 Email: kenneth.heym@cookchildrens.org

 

Transplantation of Ex Vivo Expanded, UCB-derived, Stem & Progenitor Cells vs. Unmanipulated UCB for HM Patients

Description – This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.

Lead Organization – Gamida Cell

Locations

  • Kansas, Kansas City, University of Kansas Cancer Center
  • Westwood, University of Kansas Hospital-Westwood Cancer Center
  • Minnesota, Minneapolis, University of Minnesota / Masonic Cancer Center
  • North Carolina, Durham, Duke University Medical Center, Contact: Mitchell Eric Horwitz, Phone: 919-668-1045 Email: mitchell.horwitz@duke.edu
  • Ohio, Cleveland, Case Comprehensive Cancer Center

 

Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients with Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant

Description – This phase II trial studies how well sirolimus, cyclosporine and mycophenolate mofetil works in preventing graft-vs-host disease in patients with blood cancer undergoing donor peripheral blood stem cell transplant. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient’s immune system from rejecting the donor’s stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body’s normal cells. Giving total-body irradiation together with sirolimus, cyclosporine, and mycophenolate mofetil before and after transplant may stop this from happening.

Lead Organization – Fred Hutch / University of Washington Cancer Consortium

Principal Investigator – Brenda M. Sandmaier

Locations

  • Colorado, Denver, Presbyterian – Saint Luke’s Medical Center – Health One, Contact: Michael Brian Maris, Phone: 720-754-4800 Email: Michael.Maris@healthonecares.com
  • Washington,
  1. i) Seattle, Fred Hutch / University of Washington Cancer Consortium, Contact: Brenda M. Sandmaier, Phone: 206-667-4961 Email: bsandmai@fredhutch.org
  2. ii) VA Puget Sound Health Care System, Contact: Thomas Reeve Chauncey, Phone: 206-762-1010 Email: chauncey@va.gov

 

Oxidative Phosphorylation Inhibitor IACS-010759 in Treating Patients with Relapsed or Refractory Acute Myeloid Leukemia

Description – This phase I trial studies the side effects and best dose of oxidative phosphorylation inhibitor IACS-010759 in treating patients with acute myeloid leukemia that has come back or does not respond to treatment. Oxidative phosphorylation inhibitor IACS-010759 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Lead Organization – M D Anderson Cancer Center

Principal Investigator – Marina Konopleva

Location– Texas, Houston, M D Anderson Cancer Center, Contact: Marina Konopleva, Phone: 713-794-1628

 

Donor-Derived Tumor-Associated Antigen-Specific T Cells in Treating Participants with Relapsed or Refractory acute myeloid leukemia or myelodysplastic syndrome

Description – This phase I trial studies the best dose and how well donor-derived multi-tumor-associated antigen-specific T cells work in treating participants with acute myeloid leukemia or myelodysplastic syndrome that have come back or does not respond. Tumor-associated antigen-specific T cells are immune system cells that may target cell proteins specific to tumor cells.

Lead Organization – Baylor College of Medicine / Dan L Duncan Comprehensive Cancer Center

Principal Investigator – Premal Lulla

Locations

  • Texas, Houston, Baylor College of Medicine / Dan L Duncan Comprehensive Cancer Center, Contact: Premal Lulla, Phone: 713-441-1450
  • Texas Children’s Hospital, Contact: Premal Lulla, Phone: 713-441-1450

 

 

Tretinoin and Arsenic Trioxide in Treating Patients with Untreated Acute Promyelocytic Leukemia

Description – This phase III trial studies tretinoin and arsenic trioxide in treating patients with newly diagnosed acute promyelocytic leukemia. Standard treatment for acute promyelocytic leukemia involves high doses of a common class of chemotherapy drugs called anthracyclines, which are known to cause long-term side effects, especially to the heart. Tretinoin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Arsenic trioxide may stop the growth of cancer cells by either killing the cells, by stopping them from dividing, or by stopping them from spreading. Completely removing or reducing the amount of anthracycline chemotherapy and giving tretinoin together with arsenic trioxide may be an effective treatment for acute promyelocytic leukemia and may reduce some of the long-term side effects.

Lead Organization – Children’s Oncology Group

Principal Investigator – Matthew A. Kutny

Locations

  • Phoenix, Phoenix Children’s Hospital, Contact: Jessica Boklan, Phone: 602-546-0920
  • California, Los Angeles, Cedars Sinai Medical Center, Contact: Fataneh (Fae) Majlessipour, Phone: 310-423-8965

-Children’s Hospital Los Angeles, Contact: Leo Mascarenhas Email: helpdesk@childrensoncologygroup.org

-Oakland, Children’s Hospital and Research Center at Oakland, Contact: Carla Barbara Golden Email: helpdesk@childrensoncologygroup.org

-Orange, Children’s Hospital of Orange County, Contact: Elyssa M. Rubin Email: helpdesk@childrensoncologygroup.org

– Palo Alto, Lucile Packard Children’s Hospital Stanford University, Contact: Sheri L Spunt Email: helpdesk@childrensoncologygroup.org

-San Diego, Rady Children’s Hospital – San Diego, Contact: William D. Roberts Email: helpdesk@childrensoncologygroup.org

  • Colorado, Denver, Rocky Mountain Hospital for Children-Presbyterian Saint Luke’s Medical Center, Contact: Jennifer Jocelyn Clark Email: helpdesk@childrensoncologygroup.org
  • Florida, Jacksonville, Nemours Children’s Clinic-Jacksonville, Contact: Emi H. Caywood Email: helpdesk@childrensoncologygroup.org,

-Miami, Nicklaus Children’s Hospital, Contact: Enrique Alberto Escalon Email: helpdesk@childrensoncologygroup.org

-Tampa, Saint Joseph’s Hospital / Children’s Hospital-Tampa, Contact: Mark J. Mogul, Phone: 704-384-5369

  • Georgia, Atlanta, Children’s Healthcare of Atlanta – Egleston, Contact: Himalee Shreekant Sabnis Email: helpdesk@childrensoncologygroup.org
  • Illinois, Chicago, Lurie Children’s Hospital-Chicago, Contact: Joanna Lynn Weinstein Email: helpdesk@childrensoncologygroup.org
  • Maryland, Baltimore, Johns Hopkins University / Sidney Kimmel Cancer Center, Contact: Cara Anne Rabik, Phone: 410-955-8804 Email: jhcccro@jhmi.edu
  • Massachusetts, Boston, Dana-Farber Cancer Institute, Contact: Barbara Alsen Degar, Phone: 877-442-3324,
  • Michigan, Ann Arbor, C S Mott Children’s Hospital, Contact: Rajen Mody Email: helpdesk@childrensoncologygroup.org
  • Rochester, Mayo Clinic, Contact: Carola A. S. Arndt, Phone: 855-776-0015
  • New Jersey, Newark, Newark Beth Israel Medical Center, Contact: Peri Kamalakar, Phone: 973-926-7230
  • New York, New York, Columbia University / Herbert Irving Cancer Center, Contact: Alice Lee Phone: 212-305-8615
  • Memorial Sloan Kettering Cancer Center, Contact: Kavitha Ramaswamy Email: helpdesk@childrensoncologygroup.org
  • Ohio, Cincinnati, Cincinnati Children’s Hospital Medical Center, Contact: Maureen Megan, O’Brien Email: helpdesk@childrensoncologygroup.org
  • Oregon, Portland, Legacy Emanuel Children’s Hospital, Contact: Janice Faye Olson, Phone: 503-413-8199
  • Pennsylvania, Philadelphia, Children’s Hospital of Philadelphia,
  • Tennessee, Memphis, St. Jude Children’s Research Hospital, Contact: Jeffrey E. Rubnitz Email: helpdesk@childrensoncologygroup.org
  • Washington, Seattle, Seattle Children’s Hospital, Contact: Douglas S. Hawkins Email: helpdesk@childrensoncologygroup.org

 

 

A Trial of Temsirolimus With Etoposide and Cyclophosphamide in Children With Relapsed Acute Lymphoblastic Leukemia and Non-Hodgkin’s Lymphoma

Description – This is a phase I study of temsirolimus combined with dexamethasone, cyclophosphamide and etoposide in patients with relapsed acute lymphoblastic leukemia, lymphoblastic lymphoma or peripheral T-cell lymphoma.

Lead Organization – Therapeutic Advances in Childhood Leukemia Consortium

Locations

  • California, San Francisco, UCSF Medical Center-Mount Zion
  • Georgia, Atlanta, Children’s Healthcare of Atlanta – Egleston
  • Maryland, Baltimore, Johns Hopkins University / Sidney Kimmel Cancer Center
  • Massachusetts, Boston, Dana-Farber Cancer Institute
  • New York, New York, Columbia University / Herbert Irving Cancer Center
  • Ohio, Cleveland, Case Comprehensive Cancer Center,
  • Oregon, Portland, OHSU Knight Cancer Institute
  • Texas, Dallas, UT Southwestern / Simmons Cancer Center-Dallas, Contact: Marcella West Aguilar, Phone: 214-648-1479 Email: marcella.aguilar@utsouthwestern.edu
  • Houston, Texas Children’s Hospital.

 

 

CD19 / CD22 Chimeric Antigen Receptor T Cells and Chemotherapy in Treating Children or Young Adults with Recurrent or Refractory CD19 Positive B Acute Lymphoblastic Leukemia

Description – This phase I trial studies the best dose and side effects of CD19 / CD22 chimeric antigen receptor (CAR) T cells when given together with chemotherapy, and to see how well they work in treating children or young adults with CD19 positive B acute lymphoblastic leukemia that has come back or does not respond to treatment. A CAR is a genetically-engineered receptor made so that immune cells can attack cancer cells by recognizing and responding to the CD19 / CD22 proteins. These proteins are commonly found in B acute lymphoblastic leukemia. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving CD19 / CD22-CAR T cells and chemotherapy may work better in treating children or young adults with B acute lymphoblastic leukemia.

Lead Organization – Stanford Cancer Institute Palo Alto

Principal Investigator – Crystal L. Mackall

Locations

  • California, Palo Alto, Lucile Packard Children’s Hospital Stanford University, Contact: Crystal L. Mackall, Phone: 650-725-2553 Email: cmackall@stanford.edu
  • Stanford Cancer Institute Palo Alto, Contact: Crystal L. Mackall, Phone: 650-725-2533 Email: cmackall@stanford.edu

 

 

CD5.CAR / 28 T Cells, Cyclophosphamide, and Fludarabine in Treating Participants with Recurrent T-Cell Malignancies Expressing the CD5 Antigen

Description – This phase I trial studies the side effects and best dose of autologous CD5-specific CAR-28 zeta CAR T-cells when given together with cyclophosphamide and fludarabine in treating participants with T-cell cancers expressing the CD5 antigen that has come back. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells including tumor cells. The antibody used in this study is called anti-CD5, which sticks to T-cell leukemia or lymphoma cells because of a substance on the outside of these cells called CD5. The T cells will also contain a substance called CD28 which may help stimulate the T cells and may make them last longer. Drugs used in chemotherapy, such as cyclophosphamide and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving CD5.CAR / 28 T cells with cyclophosphamide and fludarabine may work better in treating participants with T-cell malignancies expressing the CD5 antigen.

Lead Organization – Baylor College of Medicine / Dan L Duncan Comprehensive Cancer Center

Principal Investigator – Rayne Helen Rouce

Location

  • Texas, Houston, Baylor College of Medicine / Dan L Duncan Comprehensive Cancer Center, Contact: Rayne Helen Rouce, ,Phone: 832-824-4716
  • Texas Children’s Hospital, Contact: Rayne Helen Rouce, Phone: 832-824-4716

 

 

CD19-CAR T-cell Immunotherapy in Treating Patients with CD19-Positive Leukemia

Description – This phase I / II clinical trial studies the side effects of CD19-CAR T-cell immunotherapy and how well it works in treating patients with CD19-positive leukemia. Biological therapies, such as CD19-CAR T-cell immunotherapy, use substances made from living organisms that may attack specific tumor cells and stop them from growing or kill them.

Lead Organization – Seattle Children’s Hospital

Principal Investigator – Rebecca Alice Emmons Gardner

Locations –

  • California, Los Angeles, Children’s Hospital Los Angeles, Contact: Michael Allen Pulsipher, Phone: 323-361-2121 Email: mpulsipher@chla.usc.edu
  • Washington, Seattle, Seattle Children’s Hospital, Contact: Rebecca Alice Emmons Gardner, Phone: 206-987-2106 Email: rebecca.gardner@seattlechildrens.org

 

Thiotepa, Fludarabine Phosphate, and Melphalan Hydrochloride in Treating Participants with Blood Cancer Undergoing Donor Stem Cell Transplant

Description – This phase II trial studies how well thiotepa, fludarabine phosphate, and melphalan hydrochloride work in treating participants with blood cancer who are undergoing a donor stem cell transplant. Drugs used in chemotherapy, such as thiotepa, fludarabine phosphate, and melphalan hydrochloride, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Lead Organization – Case Comprehensive Cancer Center

Principal Investigator – Leland L. Metheny

Locations

  • Cleveland, Case Comprehensive Cancer Center, Contact: Leland L. Metheny, Phone: 216-844-0139 Email: Leland.Metheny@uhhospitals.org

 

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients with Blood Cancer

Description – This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells and cancer cells. It may also stop the patient’s immune system from rejecting the donor’s stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells.

Lead Organization – Roswell Park Cancer Institute

Principal Investigator – Sophia Rebecca Balderman

Locations

New York, Buffalo, Roswell Park Cancer Institute, Contact: Sophia Rebecca Balderman, Phone: 716-845-6972 Email: Sophia.Balderman@roswellpark.org

 

 

Autologous IC9-CAR19 T cells in Treating Patients with Recurrent Acute Lymphoblastic Leukemia

Descriptions – This phase I trial studies the side effect of autologous inducible caspase 9 chimeric antigen receptor targeting CD19 antigen (iC9-CAR19) T cells in treating patients with acute lymphoblastic leukemia that has come back. IC9-CAR19 T cells combine antibodies and T cells. Antibodies are proteins that protect the body from disease caused by bacteria or toxic substances and by stopping them from growing. T cells are special infection-fighting blood cells that can kill other cells, including cancer cells or cells that are infected.

Lead Organization – UNC Lineberger Comprehensive Cancer Center

Principal Investigator – Matthew C. Foster

Locations

  • North Carolina, Chapel Hill, UNC Lineberger Comprehensive Cancer Center, Contact: Matthew C. Foster, Phone: 919-843-2447 Email: matthew_foster@med.unc.edu

 

Standard of Care + / – Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients With FLT3-ITD Mutated AML

Description – To determine if the addition of midostaurin to Standard of Care therapy reduces relapse in FLT3-ITD mutated AML patients receiving an allogeneic hematopoietic stem cell transplant.

Lead Organization – Novartis Pharmaceuticals Corporation

Location

  • California, Los Angeles, UCLA / Jonsson Comprehensive Cancer Center, Contact: Gary John Schiller, Phone: 310-825-5513
  • Illinois, Chicago, University of Chicago Comprehensive Cancer Center, Contact: Wendy Stock, Phone: 773-834-2487
  • New York, New York, Memorial Sloan Kettering Cancer Center, Contact: Esperanza Bouza, Papadopoulos, Phone: 212-639-3859
  • Washington, Seattle, Fred Hutch / University of Washington Cancer Consortium, Contact: Bart Lee Scott, Phone: 800-422-6237

 

A Study of the Safety and Effectiveness of Apixaban in Preventing Blood Clots in Children With Leukemia Who Have a Central Venous Catheter and Are Treated With Asparaginase

DescriptionThe purpose of this study is to compare the effect of a blood-thinning drug called Apixaban versus no administration of a blood-thinning drug, in preventing blood clots in children with leukemia or lymphoma. Patients must be receiving chemotherapy, including asparaginase, and have a central line.

Lead Organization – Bristol-Myers Squibb

Location:

  • California, Los Angeles, Children’s Hospital Los Angeles, Contact: David Robert Freyer, Phone: 323-361-5973
  • San Diego, Rady Children’s Hospital – San Diego,
  • Georgia, Atlanta, Children’s Healthcare of Atlanta – Egleston, Contact: Mike Aquino Briones, Phone: 404-785-4746
  • Iowa, Iowa City, University of Iowa / Holden Comprehensive Cancer Center,
  • Maryland, Baltimore, Johns Hopkins University / Sidney Kimmel Cancer Center, Contact: Patrick A. Brown, Phone: 410-955-0432
  • Minnesota, Minneapolis, University of Minnesota / Masonic Cancer Center
  • Rochester, Mayo Clinic.
  • New Jersey, New Brunswick, Rutgers Cancer Institute of New Jersey
  • New York, Buffalo, Roswell Park Cancer Institute, Contact: Barbara J. Bambach, Phone: 716-845-4561
  • North Carolina, Chapel Hill, UNC Lineberger Comprehensive Cancer Center
  • Ohio, Cleveland, Case Comprehensive Cancer Center
  • Pennsylvania, Pittsburgh, Children’s Hospital of Pittsburgh of UPMC
  • Tennessee, Nashville, Vanderbilt University / Ingram Cancer Center
  • Texas, Houston, Texas Children’s Hospital
  • San Antonio, University Hospital, Contact: Anne-Marie R. Langevin, Phone: 210-562-9025

 

 Umbilical Cord Blood Transplant, Cyclophosphamide, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients with Hematologic Disease

Description – This phase II trial studies how well giving an umbilical cord blood transplant together with cyclophosphamide, fludarabine phosphate, and total-body irradiation works in treating patients with hematologic disease. Giving chemotherapy, such as cyclophosphamide and fludarabine phosphate, and TBI before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient’s immune system from rejecting the donor’s stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body’s normal cells. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.

Lead Organization -Fred Hutch / University of Washington Cancer Consortium

Principal Investigator – Ann E Dahlberg

Location

Washington, Seattle, Fred Hutch / University of Washington Cancer Consortium, Contact: Ann E Dahlberg, Phone: 206-667-1959 Email: adahlber@fredhutch.org

VA Puget Sound Health Care System, Contact: Thomas Reeve Chauncey, Phone: 206-764-2199 Email: tchaunce@u.washington.edu

 

 

 Study of Crenolanib vs Midostaurin Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed FLT3 Mutated AML

Description – A phase III randomized multi-center study designed to compare the efficacy of crenolanib with that of midostaurin when administered following induction chemotherapy, consolidation chemotherapy and bone marrow transplantation in newly diagnosed AML subjects with FLT3 mutation. About 510 subjects will be randomized in a 1:1 ratio to receive either crenolanib in addition to standard first-line treatment of AML or midostaurin and standard treatment. Potentially eligible subjects will be registered and tested for the presence of FLT3 mutation. Once the FLT3 mutation status is confirmed and additional eligibility is established, a subject will be randomized and enter into the treatment phase.

Lead Organization – Arog Pharmaceuticals, Inc.

Location-

  • Illinois,Chicago, University of Chicago Comprehensive Cancer Center
  • Michigan,Detroit, Wayne State University / Karmanos Cancer Institute
  • New York, New York, Icahn School of Medicine at Mount Sinai

 

 

Hyperthermia in Cancer Treatment

Hyperthermia in Cancer Treatment

Hyperthermia is a type of cancer treatment in which body tissue is exposed to high temperatures (up to 113°F – around 45°C) to damage and kill cancer cells.

Hyperthermia is almost always used with other forms of cancer therapy, such as radiation therapy and chemotherapy.

Several methods of hyperthermia are currently under study, including local, regional, and whole-body hyperthermia.

Many clinical trials (research studies) are being conducted to evaluate the effectiveness of hyperthermia.

What is hyperthermia?

Hyperthermia (also called thermal therapy or thermotherapy) is a type of cancer treatment in which body tissue is exposed to high temperatures (up to 113°F). Research has shown that high temperatures can damage and kill cancer cells, usually with minimal injury to normal tissues (1). By killing cancer cells and damaging proteins and structures within cells (2), hyperthermia may shrink tumors.

Hyperthermia is under study in clinical trials (research studies with people) and is not widely available (see Question 5).

How is hyperthermia used to treat cancer?

Hyperthermia is almost always used with other forms of cancer therapy, such as radiation therapy and chemotherapy (1, 3). Hyperthermia may make some cancer cells more sensitive to radiation or harm other cancer cells that radiation cannot damage. When hyperthermia and radiation therapy are combined, they are often given within an hour of each other. Hyperthermia can also enhance the effects of certain anticancer drugs.

Numerous clinical trials have studied hyperthermia in combination with radiation therapy and/or chemotherapy. These studies have focused on the treatment of many types of cancer, including sarcoma, melanoma, and cancers of the head and neck, brain, lung, esophagus, breast, bladder, rectum, liver, appendix, cervix, and peritoneal lining (mesothelioma) (1, 3–7). Many of these studies, but not all, have shown a significant reduction in tumor size when hyperthermia is combined with other treatments (1, 3, 6, 7). However, not all of these studies have shown increased survival in patients receiving the combined treatments (3, 5, 7).

What are the different methods of hyperthermia?

Several methods of hyperthermia are currently under study, including local, regional, and whole-body hyperthermia (1, 3–9).

In local hyperthermia, heat is applied to a small area, such as a tumor, using various techniques that deliver energy to heat the tumor. Different types of energy may be used to apply heat, including microwave, radiofrequency, and ultrasound. Depending on the tumor location, there are several approaches to local hyperthermia:

External approaches are used to treat tumors that are in or just below the skin. External applicators are positioned around or near the appropriate region, and energy is focused on the tumor to raise its temperature.

Intraluminal or endocavitary methods may be used to treat tumors within or near body cavities, such as the esophagus or rectum. Probes are placed inside the cavity and inserted into the tumor to deliver energy and heat the area directly.

Interstitial techniques are used to treat tumors deep within the body, such as brain tumors. This technique allows the tumor to be heated to higher temperatures than external techniques. Under anesthesia, probes or needles are inserted into the tumor. Imaging techniques, such as ultrasound, may be used to make sure the probe is properly positioned within the tumor. The heat source is then inserted into the probe. Radiofrequency ablation (RFA) is a type of interstitial hyperthermia that uses radio waves to heat and kill cancer cells.

In regional hyperthermia, various approaches may be used to heat large areas of tissue, such as a body cavity, organ, or limb.

Deep tissue approaches may be used to treat cancers within the body, such as cervical or bladder cancer. External applicators are positioned around the body cavity or organ to be treated, and microwave or radiofrequency energy is focused on the area to raise its temperature.

Regional perfusion techniques can be used to treat cancers in the arms and legs, such as melanoma, or cancer in some organs, such as the liver or lung. In this procedure, some of the patient’s blood is removed, heated, and then pumped (perfused) back into the limb or organ. Anticancer drugs are commonly given during this treatment.

Continuous hyperthermic peritoneal perfusion (CHPP) is a technique used to treat cancers within the peritoneal cavity (the space within the abdomen that contains the intestines, stomach, and liver), including primary peritoneal mesothelioma and stomach cancer. During surgery, heated anticancer drugs flow from a warming device through the peritoneal cavity. The peritoneal cavity temperature reaches 106–108°F.

Whole-body hyperthermia is used to treat metastatic cancer that has spread throughout the body. This can be accomplished by several techniques that raise the body temperature to 107–108°F, including the use of thermal chambers (similar to large incubators) or hot water blankets.

The effectiveness of hyperthermia treatment is related to the temperature achieved during the treatment, as well as the length of treatment and cell and tissue characteristics (1, 2). To ensure that the desired temperature is reached, but not exceeded, the temperature of the tumor and surrounding tissue is monitored throughout hyperthermia treatment (3, 5, 7). Using local anesthesia, the doctor inserts small needles or tubes with tiny thermometers into the treatment area to monitor the temperature. Imaging techniques, such as CT (computed tomography), may be used to make sure the probes are properly positioned (5).

Does hyperthermia have any complications or side effects?

Most normal tissues are not damaged during hyperthermia if the temperature remains under 111°F. However, due to regional differences in tissue characteristics, higher temperatures may occur in various spots. This can result in burns, blisters, discomfort, or pain (1, 5, 7). Perfusion techniques can cause tissue swelling, blood clots, bleeding, and other damage to the normal tissues in the perfused area; however, most of these side effects are temporary. Whole-body hyperthermia can cause more serious side effects, including cardiac and vascular disorders, but these effects are uncommon (1, 3, 7). Diarrhea, nausea, and vomiting are commonly observed after whole-body hyperthermia (7).

What does the future hold for hyperthermia?

A number of challenges must be overcome before hyperthermia can be considered a standard treatment for cancer (1, 3, 6, 7). Many clinical trials are being conducted to evaluate the effectiveness of hyperthermia. Some trials continue to research hyperthermia in combination with other therapies for the treatment of different cancers. Other studies focus on improving hyperthermia techniques.

To learn more about clinical trials, call the National Cancer Institute’s (NCI) Cancer Information Service at the telephone number listed below or visit NCI’s Clinical Trials Home Page.

Here is the link to the complete article.

 

NeuroBlastoma (9 months old boy)

This testimonial is about a Neuroblastoma first diagnosed at the age of 9 months old. Today this boy is 4.5 years old and fought successfully 2 relapses.
This is an amazing testimonial from the relapse master, Muli’s mom.

In this interview, MyChildCancer’s founder, Oded Grinstein, is interviewing Myriam Safrai from Israel about the treatment and challenges her family went through with her son’s Neuroblastoma cancer. Her son, Muli, was diagnosed with Neuroblastoma when he was 9 months old. After early signs were misdiagnosed for a couple of months by the family’s pediatrician, he was diagnosed with a stage 4 NB. On the time this interview was made Muli was 4.5 years old, with no evidence of cancer however learning from past experience his parents were just about to start yet another clinical trial in an effort to prevent a third(!) relapse.

Watch the amazing story of Muli. 4.5 years old including 4 years of treatment, 3 different types of treatment 3 different locations on the globe, 2 relapses and 1 amazing and forth-thinking mother.

This interview is part of MyChildCancer’s mission to extract hidden life-saving information and make it easily accessible to other parents all over the world. Because knowledge saves lives.

Text version:

 

–>Please, state your boy’s name, age, and type of cancer.

Muli, his real name is Shmuel, was diagnosed when he was one year and four months. Now he’s four years and six months. He was diagnosed with NeuroBlastoma high risk, with MYC amplified.

Our story in a Nutshell:

We did all the front-line therapy in Israel, which is the SIOPEN (SIOP-Europe) protocol, after he had the first relapse. He gets two more cycles of chemotherapy, TVD (Topotecan, Vincristine, Doxorubicin) X 2 and TVC X 2 cycles.

He had a huge fungal infection, so we stopped all the treatment, and it was recommended that we do a big trip and enjoy the time that we have left. We moved to Hershey in PA (Pennsylvania, U.S.A) for a medical trial. It was a vaccine trial, with Decitabine. We were in Hershey for six months, and he was still clear after the trial.

I was flying back and forth to Michigan, for the DFMO trial of Dr. (Giselle) Sholler, for nine cycles. Then, he relapsed again, and he had two cycles of MIBG in San Francisco at UCSF Medical Center without any chemotherapy or any other drug, just MIBG, and he was NED (No Evidence of Disease) after the first cycle. We did the second round as a consolidation.

Now, we are in Memorial Sloan Kettering and the purpose is to do the antibodies – one course at least, and after that to do the vaccine trial with Dr. (Brian) Kushner.

–> Could you please tell us about the first signs, and the diagnosis process?

Muli had Anemia. They tried to treat it with Serotonin but it didn’t work. He also had a little bit of hypotonia in the legs and we took him to the pediatrician who said everything was fine. One night, it was difficult for Muli to sleep so I took him to the emergency center because I had a bad feeling. I was feeling something in my stomach, and I am a doctor, I thought he had an infection. When we did the ultrasound, they saw a huge mass in his abdomen. Afterward they did an x-ray and everything began. We did an MIBG and we saw many spots metastasized everywhere in the bone. And so, we started the protocol.

–> You mentioned that he had a pre-condition – Anemia. What were the first signs that you can now relate to the cancer? How long between these first signs and the actual cancer diagnosis?

I think the anemia was the first thing. He was also FTT (Failure To Thrive) so he was not growing so well. I have been to the doctor many times, and I told him everything that he was eating and he said that everything was fine.

So, I think the failure to treat the FTT was the first sign, there was also the anemia. And he also had the hypotonia in the leg, which today we know was caused by the tumor which was compressing his core. These were the three signs that we had. And it’s not that we didn’t notice this signs however it took months to diagnose. We went to doctors but they failed to diagnose. I have been to the doctor just 3 days before I had to take him to the E.R., and he said everything was fine.

–> How old was Muli then?

One year and four months when he was diagnosed and started treatment. Maybe six or nine months when the first signs were noticed. I can’t be sure because it was a growing process.

–> So once you took him to the E.R. and they did the scans, how did they identify the cancer, and how did they identify the type of cancer?

They made an ultrasound first of all and they reviewed the mass. The differential diagnosis for a mass in the abdominal at this age was to find what it is. So they did the biopsy, for two purposes: they needed tissue before the final diagnosis, and they needed also to decide if it’s a high risk or an intermediate risk, since before the age of one year and six months it matters if there is MYC amplification or not. Muli had MYC amplification.

–> You have mentioned that you are a doctor. Most of the people who will see this are not doctors. Please explain about the disease, the different subtypes – you mentioned MYC: is it rare?

In Neuroblastoma there are five stages: 1, 2, 3, 4, and 4S. If a child is diagnosed before the age of one year old, despite if he has metastasis, he would usually have a very good prognosis, and it would be stage 4S.

After that age, the child is usually stage 3 if it a local disease or stage 4 if the patient has a metastatic disease. Muli had metastasis, so he was stage 4.

After that doctors need to decide if he’s high risk or intermediate risk. There are few things that will help to decide if the child is high risk, which is a less good prognosis, or intermediate risk. Within stage 3 and stage 4, and you need to decide if it’s high risk or not. The age is a factor: if more than one year and six months, it’s high risk.
Now within the high risk, you have an equivalent of “very high” risk, and doctors need to look for the factors that will make the prognosis more difficult. One of the most important factors is the MYC mutation: The MYC mutation is a mutation in a specific chromosome. When doctors see in the gene of the cancer cell that amplification exist, and if they see that there is more than ten copies of the gene MYC, it’s “very” high risk.

–> So MYC is a part of a gene?
Yes. And you see the genome and you see that there’s an amplification of the genome of this part of the mutation. Muli had so many that it was very clear.

–> But Muli was under the age of 18 months?

Yes. It was the only good thing, but if you take into consideration all the factors and see what is most important in the staging, the thing that has the most accurate prognosis is the MYC. If you have MYC, it’s not good.

–> So Muli was a year and four months when he was diagnosed, and he had metastasis, so it was stage 4, and he had MYC amplified?

Yes: Neuroblastoma, stage 4, high risk, MYC amplified.

–> Tell us about the SIOPEN (SIOP-European) protocol.

The SIOPEN protocol has five parts. (1) First of all, eight rounds of chemo (2) Afterward a surgery. (3) Then you have a self-bone marrow transplant. (4) After that you get radiation. (5) The fifth and last step is immunotherapy.
For us, it was not too bad. Muli went through the whole protocol very fine. He was clear of all the metastasis after the eight rounds of chemo and before the surgery.
He had the surgery and he recovered very well. After only one week we were at home. After that he had a bone marrow transplant that was very fine. Twenty-six days we were in the isolation room but it’s very short. It was hard, but it was fine. Then he had radiation. It was fine – he was just tired. For the radiation he needed anesthesia every day because he was small. And lastly we had immunotherapy.
For that last part we had two possibilities for immunotherapy; CH18.14 with or without I02. We get it with I02, and it was very hard. Muli had a huge reaction the first night and he almost died. He had a capillary leak, which means that fluids are not staying in the blood vessels and are leaking into the small tissue. The blood pressure dropped to 30 or 50 and he stopped giving urine. We stopped everything. They took us to give him dopamine for the blood pressure, and then we decided instead of giving him the infusion of the antibodies in eight hours, as it should be in the protocol, we gave in about twelve hours. It was also hard and we have done the treatment in the ICU. We finished the first cycle in the ICU, and after that we moved to a different hospital that was more accurate for this kind of treatment and we did it, as I said, in twelve hours. It was hard. It was a hard treatment.
It was very painful. Muli all the time had some complications that were manageable. He had once a Pericardial effusion (fluid around the heart), which was hard. He had pneumonia once. Each time something else, but it was very manageable. for example, his weight was fluctuating: he was keeping so much of the fluid, he gained like 3 kilos in one week. I needed to buy him new clothes towards the end of the treatment, because all the clothes were too small. After that he would just urinate a lot and one week after we got back the Muli that we knew, the small one. We did everything we could. He had the Acutan as well, which makes the skin very dry, and we finished the protocol. We were very hopeful and very happy.
In the last scan, before we were scheduled to move to the follow-up part, we saw a relapse: one relapse in the Humerus bone. He relapsed.

–> The protocol that you were treated under, is it an Israeli protocol or a European protocol?

It’s a European protocol. We had few randomizations, no one immunization. Back then you had two options of drug / chemotherapy before the transplant. We received the CEM, but it was not the best. The BuMel is the best, and not everybody is getting the BuMel.

–> Where were you treated in Israel?

Jerusalem Hadassah Hospital.

–> And then the second hospital?

Schneider Children Hospital. In Hadassah Muli was the first kid to get the immunotherapy.

–> So, the immunotherapy is an experimental treatment?

Everything is experimental. Every protocol has some randomization so it’s all experimental because you have the immunotherapy with or without the IO2. You have few randomizations to the protocol.

–> How many children do you know in Israel that got the same treatment as Muli?

Well it depends if we are referring to all exactly the same or just the same SIOPEN protocol. Muli was I think one of the last SIOPEN chemotherapy before the transplant. Today everybody is getting the BuMel, but I know many children are getting the SIOPEN treatment.

–> Who is the best doctor you could recommend parents in Israel to visit?

We have been in two hospitals in Israel: Hadassah and then Schneider, and then we’ve returned to Hadassah.
I think the doctor who knows the most about NB is the head of the Children Oncology department in Schneider. Dr. Itzhak Yaniv. That being said, we decided to go back for treatment to Hadassah in Jerusalem since it was easier for Muli. the protocol was the same, and one can always take a second opinion. We got a second opinion with Schneider at the beginning, but I don’t think there’s much different between these two hospitals.
The treatment in Hadassah was much smoother for us. The day care was much more convenient for us, and the hospitalization as well.

–> How long was the treatment in Israel, from the beginning and until the relapse?

One year and one month.

–> After one year and one month of treatment in Israel, there was the relapse. Muli was, by then two years and five months old. What’s next?

We saw the relapse and I wanted to do the MIBG therapy because I read about it a lot.
There was a misunderstanding, and we didn’t do the MIBG therapy in Israel because it’s not a treatment we could do in Israel, and we didn’t really know how to get it. They gave him chemotherapy – two TVC and two TVD, plus local radiation, and Muli was clear at the end of the treatment, so Muli was not approved for the MIBG therapy.
When you are clear, there’s a paradox, because everybody knows that you shouldn’t treat a child with no evidence of the disease (NED), but it doesn’t mean that for 100% there is no disease; it just means that you can’t see the disease, so it’s like you have minimal disease and it’s better to treat when you have minimal disease. Everything is more effective. The problem is that the clinical trial, most of the clinical trials are usually open for childrenwith signs of disease, so there are very few treatments that you can have at this stage.
For Muli we knew that the prognosis was very bad. Muli was high risk with one relapse. In all the papers, the five-year survivor rate after one relapse of Neuroblastoma practically zero. I wanted him to get the treatment, so we went to get the treatment that was open for us – which was the vaccine therapy, in Hershey hospital in Pennsylvania U.S.

–> After the first cycle ended and they saw the relapse, what were the considerations, and how did you make the decision to go to Hershey, from all the options? Where did you apply, and what was the process?

Well there was not so much applying, because Muli already got antibodies. We had two options: a treatment in Sloan-Kettering (NY) and the one in Hershey (PA).
The CH14.18 that he already got during the treatment was an anti GD2 (antibody treatment). The GD2 is something on the cell of the Neuroblastoma, and the antibodies were an anti GD2. The Sloan-Kettering antibodies were also anti GD2, so we went for a different approach, in Hershey.
There weren’t so many options because Muli was clear of diseased, and for the research purpose it is always better if you have an active disease with evident signs, so you can measure everything.

We were recommended by our doctors to stop everything and enjoy the time we have left, so we didn’t have a lot of choices, but we decided to continue the fight and we moved to Hershey.

–> Was there any other place in the world that you checked?

We checked, yes, we checked a lot of others, but there was no trial for children without evidence of disease.

–> All the other places were accepting only kids with evidence?

Yes. You may have more options when you have evidence, but these clinical trial are closing and opening all the time. I wanted to try a vaccine trial for him, but it was closed.
It’s a matter of timing. Timing plays a big role in the options you get to choose from.

–> For better or worse, the only option you could find was in Hershey.

Yes.

–> Did you check also other countries, other than the U.S?

Yes. We checked in Europe for MIBG therapy, but we already got the European protocol, so what they recommended was chemo. Muli had the fungus infection during his treatment, from which he almost died. He had three surgeries in four days to treat it, and a lot of treatment after that, and we could not give him any more chemo, so it also closed some options for us. Chemo is not curable for Neuroblastoma high-risk relapse.

–> Is the Israeli protocol equivalent to the European protocol?

Yes.

–> And this is why it made less sense for you to go to Europe?

Yes but also because in Europe there are not so many clinical trials like in America. He could not get any more chemo at that time and the European protocol is all about chemotherapy.

–> Tell us about the second phase in Hershey: after you got there, how did you get along, how did you make the connection? Tell us also about the treatment itself.

The bureaucracy was very fast. Hershey was not used to having international patients; I think Muli was one of the first. We got a paper saying “for the Israeli patient”. It was funny — there’s no international department in the hospital or anything.
It was very, very fast, and they were very, very nice and helpful, and when we decided and they checked everything and say that we can only after we were there with Muli. For ten days, me and my husband, we made a peripheral blood, like before the bone marrow transplant, they harvest peripheral cells. We take the cells and after we go back to Israel and for three weeks and they prepare the cells for the vaccine. After we move, we have a daughter, we move together, we lived in the Ronald McDonald house for five and a half months, and Muli gets the treatment.
It’s four courses of treatment.
There is one week of chemotherapy, low-dose Decitabine chemotherapy, two injections. In weeks number 2 through 4 it’s only blood tests. Everything was outpatient, unless he had a fever or neutropenia or something, but it’s not lowering the count so much.
It was for courses like this, and we had one delay because the count was too low to begin the Decitabine.
It was fine. It was an easy treatment – only the shot were not so nice.

–> So it was five months?

Yes. Roughly between the age of three and three and a half, approximately, because he had chemotherapy in Israel. After they finished and after they saw the relapse, they gave Chemotherapy — two TVD and two TVC. Plus local radiation.

–> Local radiation. That was after the relapse?

Yes. Then, he was clear, and then we got to the Decitabine treatment.

–> Tell us a bit about the treatment in Hershey. Was it biological? How did it work etc.

There’s two parts. There’s the Decitabine, the chemotherapy, with a very low dose, which just felt like fine-tuning of the white cells and red cells to increase the immunity response. They took some cells from Muli and they worked with them in the labs, and we injected them back into the body. The body does not fight these cells because it’s cells from the body, and it showed in the lab that the cells fight against Neuroblastoma.
For us the treatment was good and NED – almost six months after, he was continuing to stay clear. For us, it was very good; we were very happy. It was hard to think that he will still be clear. It was good.
He was at the age of three and a half when he finished.

–> The tests, what are the tests they did at the end?

We did MIBG in the middle and at the end, and we saw there was no relapse.

In the MIBG Test, as oppose to the MIBG treatment, they inject something radioactive. The cells are absorbing it – it’s radioactivity plus iodine, and the Neuroblastoma cells absorb iodine. After that the child is lying in the machine and a radioactive sensor scans the body and you can see if there are any spots.

–> It’s like PET-CT?

It’s exactly like PET-CT, but PET-CT is with glucose and the Neuroblastoma cell is avid to iodine. So MIBG test is like PET-CT, but they switch the glucose with iodine.

–> Did you have insurance for this clinical trial?

NO. We paid out of pocket in Hershey Medical Center, and I think it’s important for people to know the costs, out of pocket. The Decitabine treatment was almost $60,000.

–> That’s for the six months in Hershey?

Only the medical treatment, yes – not including flying back, hotel, food, car etc. Only the medical treatment. All the other flights in the U.S, the visits, the meetings with the doctor, we had to pay for each of the meetings.

–> Why was the Hershey treatment not covered by your insurance in Israel? Everyone in Israel has medical insurance from the state, and I assume that your treatment in the Israeli hospitals was all covered. Why wasn’t the Hershey treatment covered by the insurance?

Because it was a medical trial, and the only thing in Neuroblastoma that we knew that the medical insurance in Israel would cover only a surgery in the case it cannot be done in Israel, or MIBG treatment. At that stage, Muli was not a candidate for the MIBG treatment because he was without any evidence of disease.

–> After five months in Hershey, the MIBG test showed no evidence of cancer. What’s next?

After the treatment in Hershey, before we came back to Israel, I wanted Muli to be in some of the computers in the States because of bureaucracy. During the first relapse, the time it took us to get any answers was too long and I didn’t want to spend any more time, if something should happen again.
We got a second opinion meeting at CHOP (Children Hospital Of Philadelphia) with Dr. John Maris. He had a medical appointment there, so he was in the computer already.
Also, I sent many e-mails to the head of five of the most important doctors for Neuroblastoma who I knew about.
We also went to UCSF Medical Center in San Francisco. We flew all the way back there because the head of the entire COG (Children’s Oncology Group) was there.
It was a thing that was very important for us to do, to try to skip all the bureaucracy, in case there’s going to be another relapse because in Israel where we live, if there’s a relapse, you speak to your doctor and the doctor speaks to another doctor and it takes time which we don’t have. So now, if the child is already in the computer, you can speak directly to your doctor which is overseas.

–> Amazing; genius! For one visit to the doctor, you’re already in the hospital’s system. You now know the doctor, and from now on you have a direct channel of communication. Wow!

Yes, it’s a non-writing contract. Your child is a patient of him, so you can be in contact with him.
It took almost two months to have the appointment in CHOP, and one month to the appointment at UCSF. It’s time that you don’t have when your child relapses. It’s completely different to do it when you have time. Also, I sent a few emails and I had some calls with other doctor. I knew that there’s going to be a treatment which was about to open with Dr. Sholler for maintenance treatment. I was looking for a magical drug that will keep Muli clear, because Muli is without any evidence of disease.
So I was at the medical conference in Texas, and I spoke with Dr. (Giselle) Sholler from Michigan, Grand Rapids. Helen DeVos Hospital. We saw that the trial was not yet open but I was in touch with her, and ten days after I came back to Israel I flew with Muli to Michigan to be with seven kids to be enrolled in a new treatment of maintenance for Neuroblastoma, the DFMO trial in Grand Rapids, Michigan.

–> Thanks to your visit to the conference in Texas..

Yes, I was there because I knew they were going to speak about the DFMO trial. It’s the reason I went to the conference, and to meet with her and to know her. I wrote her an email before.

–> You established contact, and then once you finished the tour in the U.S, you went back to Israel and ten days afterward you came back to Michigan for this trial.

Yes.

–> Tell us, please, about the Michigan chapter.

The drug is called DFMO and is for maintenance. The purpose is to prevent any relapse. It’s a drug that you take twice a day orally.
One course of treatment is one month, and every three months you have an MIBG scan, a CT scan, and any other special tests you may need.
The periodic tests (blood etc.) you can do in Israel however you need to be in Michigan to get the drug because they cannot ship the drugs overseas. They need to see you, check on the kids, and after that you can fly back with the medical drugs. This treatment was very easy on Muli. We didn’t see any side effect.
After nine months he relapsed, so we stopped the treatment. Usually, the protocol for this treatment is two years.

–> Where was the relapse?

There were two sites of relapse: one in the first rib: a bony relapse with a little mass around it, and one in the leg. Only bones. It metastasized in the bone.

–> Who manages the DFMO?

Dr. Giselle Sholler in Van Handel Hospital in Grand Rapids, Michigan.

–> Were you happy with the treatment in Michigan?

Yes, very happy.

–> What happened after the relapse? I assume the trial in Michigan had to stop?

Yes. Before the relapse we were always thinking and discussion what we will do in case of a relapse, because we knew that when the relapse happens you don’t have time. So we already knew and the doctor knew as well that in case of a relapse we want to do an MIBG therapy. I’ve been in touch with CHOP, where my child was already in the system, and with UCSF. The treatment in both hospitals is similar so we wanted to go to CHOP because it’s much closer; Philadelphia to Israel than San Francisco, but the bureaucracy there was too long. So eventually, after one month we began the treatment in UCSF (University of California, San Francisco).
In CHOP the bureaucracy took too long. They asked for many things and it was just.. We didn’t have the time to spend, so we flew to UCSF. It was a bureaucracy decision between the hospitals because the treatment would have been the same.

–> How long have you been in Israel before you went to UCSF?

One month. He got a little dose of some chemo just to prevent it from spreading anywhere else. We also needed to harvest stem cells since in order to make the MIBG therapy, you need to have stem cells.
If you want to make it a full dose of MIBG therapy, you need to have bags of stem cells, so we harvested some stem cells. We’d already harvested in the past, but after MIBG therapy, you cannot harvest any more stem cells, so we harvested another bag of stem cells. And then we flew to San Francisco to do the MIBG therapy.

–> Tell us a bit about the MIBG therapy please.

The MIBG therapy is like the MIBG scan, but it’s a much higher dose of radioactivity, so it just will burn the disease. It will go to the disease and burn it. The child is radioactive after the infusion so you cannot be with him for about one week. He’s in a special room and there’s a shield, a special led shield. If you need to help him with urinating or changing diapers or anything else you can go to the other side of the shield for a short period of time but normally you would stay outside the room. It was not an easy week for Muli, but thanks to an iPad that we got him it was fine. If you have a child that doesn’t know how to use an iPad and you are about to do an MIBG therapy, teach him how to use an iPad! Because we wrapped it a lot and he was just playing with the iPad for hours. It was very good.

–> So, the MIBG treatment is basically trying to burn the cancer cells by identify them with the iodine and then burn them with the radiation?

Yes. They have the iodine plus radiation. They inject them together. It’s going everywhere in the body, but it only stays in the tumor.
It’s basically a radioactive treatment. The tough part about MIBG treatment is that the kid is radioactive and you cannot get next to him. He was for almost a week alone in a room. Parents can go back and forth for short periods of time. They don’t do the treatment if you don’t have two caregivers. We took three caregivers: I, my husband, and my mother-in-law. When you are three caregivers you can win more minutes inside, because everyone has an amount of minutes. The hardest part is only for the first three days, after which you can basically go back and forth. It’s not so radioactive;
All in all the treatment was fine. It’s not a painful treatment.

–> How many cycles of MIBG treatment?

You can do two MIBG cycles. After the first one we have done we saw that Muli was NED, however we decided to do another one. We weren’t sure what to do, but every doctor that we asked told us that if the disease responds now for MIBG, it’s not sure that it will have a similar influence in the future however on the flip side it may be good to ‘keep’ the second round as a backup measure in case another relapse happens.
We were certain that there were cells that we didn’t see, and decided it would be better to give him another cycle and try to kill it. So we did another cycle.

To summarize the UCSF chapter: we have been for one month in San Francisco. Starting with the bone marrow biopsy, CT scan and MIBG test for the purpose to get into the trial. When Muli was in, we did the trial. It was one week in the room. Then we waited for two weeks to get the stem cells back in America. We chose to stay there to get the stem cells in San Francisco because in Israel, nobody gets MIBG therapy and we didn’t want to fly with a child overseas if there was any allergic reaction, any side effects – we wouldn’t have known what to do with him. After they gave him the stem cells back, we flew back to Israel where we did blood count twice a week.
In Israel he gets platelets and whatever else is needed. After another a month and a half we flew back to UCSF and we made an MIBG scan (Not therapy) and we saw that there is no disease left, we decided then that we will do another cycle of MIBG therapy to ‘consolidate’. Again, we stayed one month, and we flew back to Israel where he gets blood count, platelets and whatever he needs.

And now we came to Memorial Sloan Kettering Cancer Center (MSKCC). We made the scan on purpose to get him into another study, and we saw that Muli is still NED 🙂

–> How old was Muli when you finished the second MIBG?

Four years and four months.

–> The MIBG treatment; who was running it and what can you tell other parents about this treatment?

Dr. Katherine Matthay was our doctor. A very, very good doctor, very nice. We stayed there in front of the hospital in the family house. It was very nice because it was just in front of the hospital so it was easy for us to go back and forth.

–> Tell us a bit about the Memorial Sloan-Kettering chapter.

We just began, so we don’t know a lot, but we hope to do the mouse vaccine or mouse antibody, one course, and see if we can treat the HAMA (Human Anti-Mouse Antibodies).
They treat the antibody against the antibodies, so then if you have HAMA you cannot continue the treatment but you can lower the HAMA. We will see; we don’t exactly know. The purpose is to make some antibodies, whatever we can, and then to do the vaccine trial.

–> What is HAMA?

HAMA stands for Human Antibody Against Mouse Antibodies.
It’s the antibody that your body is doing against the antibody that we’re trying to give you.

–> They’re going to take something and put it in mice?

They already have the antibodies and they give it to you, but if your body is making antibodies against the antibodies that they try, you cannot treat the disease.
He will get on course, and then we will try to manage it. And if no we will go straight to the vaccine. If you get any other antibodies before, you will get antibodies before and after.

–> At Memorial Sloan Kettering, they accept patients without any evidence of disease?

Yes, after relapse. After a relapse, you can get this treatment.

Lessons learned

–> Looking back, in retrospection, if you could go back in time, to which point in your amazing journey would you go, and what would you have done differently?

I would have harvested more stem cells in the beginning, before any treatment, because it’s like gold down the road and it’s very different to harvest cells after transplants. I would definitely try to harvest more cells in advance. I think that’s the only thing that I would have done differently. That’s the most important thing.

–> May a doctor have any objection to doing it?

No. In Israel, we harvest much more cells.

–> Didn’t they harvest the maximum possible?

No. They harvested just for the transplant, but now they know that it’s important for the future. If I could go back, I’d ask them to harvest much more.

–> What would you advise other parent who are now starting to deal with Neuroblastoma in a very young child?

I think it’s important to know exactly what your child is getting, and to check what works and what is available even if it’s not your field.
You need to know what medicines and procedures should come and check that it’s coming. It’s important because there are mistakes in the hospital all the time.
Also, it’s important to know what is going to be your next step, hoping you don’t really need to use it but to know, because when a big mess happens like a relapse you don’t really have the time to think. I think it’s important to know who the best doctor is. If you can be in contact with them, it’s important.

–> For a parent who is just now starting the Neuroblastoma journey, please mention a couple of names and hospitals that you recommend he should get in touch with.

In CHOP (Children’s Hospital of Philadelphia), Dr. John Maris and Dr. Yael Mossé.
In UCSF (University of California, San Francisco), Dr. Katherine Matthay and Dr. Steven DuBois.
In Hellen Devos hospital (Grand Rapids, Michigan), Dr. Giselle Sholler.
In Chicago, Dr. Susan Cohn.
In Boston, Dr. Lisa Diller.
In Israel: Dr. Iris Fried in Hadassah and Dr. Shifra Ash and Dr. Itzhak Yaniv in Schneider.
There are others which I don’t know.

–> Should a parent get in touch with all of them or only some of them?

It depends. The treatment you can have in CHOP, in Boston, in San Francisco and in Chicago is under the same protocol. So, it’s not important to know everybody, However sometimes it made the difference for us with the bureaucracy and the time frame you can get your child into one of the trials.

–> Tell us a bit about the side effects of the treatment in Muli.

Muli has very low side effects, no real problems. Despite all the chemo that he gets, it’s completely fine. The only thing is, his blood count needs to recover after a huge treatment. That’s the only side effect. For example, he needs to get platelets. But it’s a matter of time and it will pass. Everything that we can have in my pocket and an able to give to him I don’t really care. It will pass.

–> How about growing, the radiation effect on his internal organs?

Nothing, nothing, nothing, nothing. We are lucky.

–> How many surgeries did he undergo?

One big one for the removal of a tumor. He had a smaller one for the removal of the fungus. He had port insertion. Three surgeries. He also had biopsies, but only one big surgery.

–> Did you ever use alternative medicine?

Yes. Anthroposophy medicine, in the beginning, and also for the side effect we used reflexology for the appetite and the nausea.

–> Anything you can recommend? Did you see any evidence?

I think the reflexology was helping for the appetite.

–> How many kids do you know that had a similar journey to you?

Nobody.

–> How many children with Neuroblastoma do you know?

In Israel about Fifteen or so. Here in the U.S I know many others.

–> Could you please mention websites and sources of information that a parent could use?

NIH (National Institute of Health): For the clinical trial, there’s a list of all the clinical trials. I think it’s an important site. That’s our main source of information for clinical trials.

–> Do you know of any communities or Facebook groups that you can recommend to parents to check out?

There’s a Facebook group for Neuroblastoma, and there’s also a list in the INRC, a very good list of emails. If somebody is sending an email it goes to all the people. You respond and you can see. It’s very good. It’s www.BlastNB.com and they have all types of cancer, not only Neuroblastoma. You can choose what kind you want to be in touch with. This can enable a parent to get in touch with another parent of the same type of cancer.

–> How did it affect the family?

It’s hard. It’s very hard. It’s not what we planned. We’re not making the career that we wanted. We are not with our friends and family. The life is completely different.

–> And you have another baby?

Yes, we have two children.

–> How old is the younger baby?

One year and four months.

–> So he was born into..

Yes. When I flew back when we were in Hershey, I did the delivery and came back.

–> Thank you very much for sharing.

Thank you!

 

Search and stay up to date

Using GoldenFeeds innovative search engine we enable our parents to stay updated with information about their child’s specific type of cancer.

Here is how it works:

  1. Parents can submit specific terms to be searched by GoldenFeeds search engine. 
  2. After a few days you will receive an Excel file with the first 150 results from the web (same as if you would search Google)
  3. Go over the list and highlight only the sources which you find most trustable and relevant to you.
  4. Send us back the filtered list and GoldenFeeds will now search, every week, just the selected websites which you highlighted as most important.
  5. Once a week we will upload the search results (for your selected list of websites) here on this page. By an easy comparison you will be able to track right away any new pages on the web about the type of cancer you are dealing with.

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