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


Anne Wardlow

Joe & Eileen W

Susan Howe

A&U. Marti and john

Steve Kohler


For Ili

Full Donor List Opens new window.Full Donor List

Histiocytosis - Monoclonal Antibody Development


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.


(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+

Wed, Dec 07, 2016
Wow, lovely site. Thnx ...
Feel free to surf to my webpage: devenir riche sur internet ( %url )

Wed, Dec 07, 2016
Howdy! Quick question that's entirely off topic. Do you know how to make your site mobile friendly? My weblog looks weird when browsing from my apple iphone. I'm trying to find a template or plugin that might be able to correct this problem. If you have any recommendations, please share. Many thanks!
my website: click here ( %url )

Wed, Dec 07, 2016
You're a really practical internet site; couldn't make it without ya!
Also visit my blog post :: San Juan Capistrano California Dating ( %url )

Wed, Dec 07, 2016
A good travel agent will be sure to have the right peace of mind with the proper travel insurance to keep your family safe and guarded.
my page; travel agency houston ( %url )

Wed, Dec 07, 2016
Wow, beautiful site. Thnx ...
My blog post :: gamefly free trial ( %url )

Hiram Lindley
Wed, Dec 07, 2016
Thank you so much for sharing this excellent web-site.
My page: gamefly free trial ( %url )

Julius Herrmann
Wed, Dec 07, 2016
Amazing internet site you have got going here.
Have a look at my weblog ... assholes worldwide ( %url )

Ernie Campos
Wed, Dec 07, 2016
Hello, Neat post. There is a problem with your site in web explorer, would check this? IE still is the market leader and a good portion of other people will pass over your excellent writing because of this problem.
Here is my web page visit ( %url )

Wed, Dec 07, 2016
Thanks, this site is really helpful.
My web page - gamefly free Trial ( %url )

Wed, Dec 07, 2016
You've gotten one of the better internet sites.
my blog; best forex signal provider ( %url )

Brayden Gamble
Wed, Dec 07, 2016
You have got possibly the best internet sites.
Review my homepage ... gamefly free trial ( %url )

Wed, Dec 07, 2016
You're an extremely beneficial site; couldn't make it without ya!
Visit my blog; Laguna Hills California Online Dating ( %url )

Wed, Dec 07, 2016
I love the details on your website. thnx.
Take a look at my blog post; remix remix ( %url )

Wed, Dec 07, 2016
Here is my web page dibujos de flores a color para tatuajes ( %url )

Loreen Gaudet
Wed, Dec 07, 2016
You've gotten incredible info right.
Here is my web blog: gamefly free trial ( %url )

Houston Salinas
Wed, Dec 07, 2016
I like this site so much, bookmarked.
Here is my page: NitroNos X And TestoRip X ( %url )

Wed, Dec 07, 2016
Incredible, such a beneficial webpage.
Also visit my homepage ... assholes worldwide ( %url )

Tia Rees
Wed, Dec 07, 2016
A webinar is a lecture presented either reside or through podcast or video that presents prospects with data that can assist solve a issue or supply business secrets.
Also visit my web page: youtube marketing internship ( %url )

Wed, Dec 07, 2016
I like this post, enjoyed this one regards for putting up.
Feel free to visit my page - Yeezy Boost 350 ( %url )

Dotty Stewart
Wed, Dec 07, 2016
I treasure the knowledge on your site. Thanks for your time.
My blog post - best seller books 2016 ny times ( %url )

Shirleen Lavarack
Wed, Dec 07, 2016
Wow, stunning portal. Thnx ...
my blog post: Forex Signal Machine ( %url )

Felix Boettcher
Wed, Dec 07, 2016
Wonderful blog! I found it while surfing around on Yahoo News. Do you have any tips on how to get listed in Yahoo News? I've been trying for a while but I never seem to get there! Thanks
Here is my web blog: web hosting service ( %url )

Marcela Dunaway
Wed, Dec 07, 2016
By opening Siri up to developers in ios 10.1.1 jailbreak ( %url ) 10, Apple is ensuring that users will benefit from an even better experience.

Shasta Jacoby
Wed, Dec 07, 2016
You have got amazing stuff on this site.
my page ... assholes the community ( %url )

Wed, Dec 07, 2016
Nice web-site you've gotten right here.
My web blog: gamefly Free Trial ( %url )

Records 1 - 25 of 1,001+

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