Histiocytosis - Monoclonal Antibody Development


Team Fundraising Goal: $500,000.00

Total Number of Gifts: 15
Total Value of Gifts: $1,470.00

Recent Donors

Lucus Gamble

Myra O Flynn

Anonymous

Anne Wardlow

Joe & Eileen W

Susan Howe

A&U. Marti and john

Steve Kohler

Anonymous

For Ili

Full Donor List Opens new window.Full Donor List

Histiocytosis - Monoclonal Antibody Development

Goal:
$500,000.00
Achieved:
$1,470.00

0 percent of goal achieved.

Monoclonal Antibody Development for Diagnosis and Treatment of LCH

Langerhans cell histiocytosis (LCH) is a rare disorder affecting children and adults for which the incidence has been estimated to be approximately 5 to 10 patients per million persons(i). LCH is characterized by the clonal proliferation (ii) of phenotypically immature (iii), CD1a positive cells (iv) that are similar to Langerhans cells, which are important in the regulation of the immune system.

Clinical signs and symptoms range from isolated eosinophilic granuloma (v) of bone or limited skin involvement to multi-organ dysfunction and fatal disseminated (vi) disease.

Current approaches to the treatment of patients with LCH include a variety of chemotherapeutic and immunosuppressive agents as well as radiation therapy and sometimes bone marrow transplantation. Despite aggressive treatment, there is a high incidence of recurrent disease and significant morbidity (vii) as well as mortality in patients with disease that is unresponsive to treatment. The identification of mutations in the BRAF1 gene in 50% to 60% of patients has provided another potential approach of treatment that needs careful testing. Also, the development of resistance to targeted therapies is commonly observed, and combination therapies are likely to be important to further test. More effective approaches for imaging disease status as well as novel treatment modalities for patients with LCH are thus still needed and would represent an important advance.

The development of cell surface protein targeted (viii) monoclonal antibodies (MAB) (ix) to treat patients with autoimmune disorders and cancer has resulted in multiple examples of the utility of immunotargeted therapy. Such antibodies represent what the famous Dr. Ehrlich in the early 20th century called ‘magic bullets’ to fight disease. We have thus developed a fully human MAB with high specificity and affinity for a native, external epitope of CD1a that internalizes upon binding to surface CD1a and can also induce significant and selective in vitro and in vivo anti-tumor cytotoxicity. This work has been based on the important findings of work from the United Kingdom using a mouse monoclonal antibody.

While no treatment is likely to be without some possible side effects, monoclonal antibodies have been exceptional agents in terms of the low degree of adverse effects and their ability to be highly selective for the target tumor cells. The figure schematically demonstrates how monoclonal antibodies can selectively eradicate tumor cells as well as identify them in patients with specialized radiology imaging methods.

Our hope is that we can now develop this immunotherapeutic and immunodiagnostic approach for patients with LCH. The need is to produce and test sufficient amounts of the monoclonal antibody under clinical grade conditions so that it can be tested in patients with a feasibility trial. If successful, we would like to develop it as a single ‘one stop’ diagnostic scan that would selectively identify LCH in patients, thus potentially reducing the number of different types of scanning. Such antibody scans could also be used to detect responses of LCH in patients undergoing other types of conventional and new treatments. In addition, we would hope that the monoclonal antibody could also provide benefit as a treatment alone or in combination with other therapies to patients with LCH.

Definitions:

(i) Because of the rare occurrence of LCH, government in the United States has dubbed it an “orphan disease.” This means that they do not provide funds to research orphan diseases, although they do encourage pharmaceutical companies to do research involving orphan diseases through tax incentives.
(ii) clonal proliferation- growth of cells in a genetically identical manner
(iii) phenotypically immature- cells that are visibly immature due to their genetic makeup interacting with the environment
(iv) CD1a positive cells- cells that test positive for the CD1a gene
(v) eosiniphilic granuloma- synonymous with “Histiocytosis X” or “Langerhans Cell Histiocytosis.” A specific type of white blood cell, also part of the immune system, that becomes grainy in form
(vi) disseminated- widespread, distributed
(vi) morbidity- illness
(viii) cell surface protein targeted- The protein on the cell’s surface is the key in this process that unlocks the cell to allow this therapy in. Our hope is that this will deactivate the targeted cells, therefore prohibiting them from reproducing.
(ix) monoclonal antibodies (MAB)- antibodies that come from a single clone.

If you prefer to donate by check, please mail checks to:
Attn: Bonnie Morgan
Phoenix Children's Hospital Foundation
2929 E Camelback Rd, Suite 122
Phoenix, AZ 85016

Thank You!

Guest Book

If you would like, you can add your name and a short message to our Guest Book. Thank you.

Sign the Guest Book

Records 1 - 25 of 1,001+

Maria Beatriz Lima
Thu, Jul 27, 2017
Oficialmente, foi confirmada dentro de 24 de abril a 2012 através de fiel buscador, através de lhe blogue oficial Inside Search_.
meu local - curso seo black hat ( %url )

Michaela
Thu, Jul 27, 2017
I have been surfing online more than 4 hours today, yet I never found any interesting article like yours. It is pretty worth enough for me. In my view, if all website owners and bloggers made good content as you did, the net will be much more useful than ever before.
Stop by my web-site; Http://Www.Tw-Nightlife141.Com/Home.Php?Mod=Space&Uid=827883&Do=Profile&From=Space ( %url )

Deloris Polley
Thu, Jul 27, 2017
Thanks a ton for sharing this fantastic website.
My site; funny ( %url )

Nila Vivier
Thu, Jul 27, 2017
Extremely user pleasant website. Immense details offered on few gos to.
Also visit my weblog :: peluang usaha vulkanisir ban ( %url )

Caitlyn Mill
Thu, Jul 27, 2017
Simply just had to say I'm just ecstatic I happened on the webpage!
my weblog; body wash and lotion ( %url )

Terrance Nieves
Thu, Jul 27, 2017
Thanks really valuable. Will certainly share website with my buddies.
My web page; thailand curtain motor ( %url )

Luciana
Thu, Jul 27, 2017
Great looking website. Think you did a great deal of your very own html coding.
my page: tempat hiburan di lombok ( %url )

Cristine Mayers
Thu, Jul 27, 2017
You're an extremely practical internet site; could not make it without ya!
Feel free to surf to my web-site: Buy MIZON US ( %url )

Lenard
Thu, Jul 27, 2017
You have got astonishing info in this article.
Here is my page ... ruang kerja kantor ( %url )

Numbers
Thu, Jul 27, 2017
Sustain the spectacular work !! Lovin' it!
Also visit my web blog ... Watch Jav Online Free ( %url )

Lieselotte Bridgeford
Thu, Jul 27, 2017
I am wholesale jerseys playoffs supplier online, take coupon code here: cheap jerseys limited ( %url )

Latisha Varley
Thu, Jul 27, 2017
Great website! It looks very professional! Maintain the good job!
Also visit my blog post ... 122038 ( %url )

Arnette
Thu, Jul 27, 2017
Extremely informative, looking forward to coming back.
Take a look at my webpage: marijuana for sale ( %url )

Melody Counsel
Thu, Jul 27, 2017
Thank you so much! This a great site!
Also visit my web-site ... Peluang Bisnis ( %url )

Patricia Kilvington
Thu, Jul 27, 2017
Hello there, cool site you have got right now.
Take a look at my web page; Situs Poker Terbesar Di Indonesia ( %url )

Savannah Dick
Thu, Jul 27, 2017
You have awesome stuff at this point.
my web blog: Cara Daftar Taruhan Judi Bola ( %url )

Alba
Thu, Jul 27, 2017
Incredible such a invaluable site.
My page pembersih wajah untuk kulit kering ( %url )

Marianne Cramer
Thu, Jul 27, 2017
Wow cuz this is excellent work! Congrats and keep it up.
Feel free to visit my webpage; rencontre femme mure ( %url )

Wendi
Thu, Jul 27, 2017
Thanks! It a superb internet site!
my web blog - cara mengatasi kulit kering secara alami ( %url )

Mckinley
Thu, Jul 27, 2017
Passion the site-- extremely individual pleasant and lots to see!
my website; Red Bull ( %url )

Lakesha
Thu, Jul 27, 2017
Many thanks for sharing this superb site.
Stop by my webpage; ancor ( %url )

Bette
Thu, Jul 27, 2017
Sustain the remarkable job !! Lovin' it!
Here is my homepage - USA Phoenix Children's Hospital Foundation Online Price ( %url )

Bernie
Thu, Jul 27, 2017
You're an extremely useful site; could not make it without ya!
My site; captcha solvers ( %url )

Vernon McCann
Thu, Jul 27, 2017
Extremely user friendly site. Great information readily available on few clicks on.
my blog - Christian Louboutin Daffodile ( %url )

Jame Dobie
Wed, Jul 26, 2017
Great looking site. Presume you did a bunch of your very own html coding.
My homepage - replica christian louboutin ( %url )

Records 1 - 25 of 1,001+


If you think this page contains objectionable content, please inform the system administrator.

© 2017 PHOENIX CHILDREN'S HOSPITAL - ALL RIGHTS RESERVED - PRIVACY POLICY. WEB SITE DESIGN BY ENVISIONUP

]]