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+

Mon, Mar 27, 2017
Wow cuz this is great work! Congrats and keep it up.
Feel free to visit my website; wi_cej info ( %url )

Mon, Mar 27, 2017
Maintain the remarkable work !! Lovin' it!

Mon, Mar 27, 2017
Great looking site. Think you did a bunch of your own html coding.
Also visit my web site :: máy làm mát b_ng h_i n__c ( %url )

Mon, Mar 27, 2017
Wow because this is very helpful work! Congrats and keep it up.wholesale mlb jerseys ( %url )

Mon, Mar 27, 2017
Very revealing looking onward to coming back again.
my web site :: agen bola terpercaya indonesia ( %url )

Mon, Mar 27, 2017
Ensure that to at all times backup your registry previously trying to repair any troubles.
Here is my page :: malware protection ( %url )

Mon, Mar 27, 2017
Avira Free Antivirus protects you from viruses, Trojans, worms, spyware, adware, and numerous different kinds of malware, making it a fully practical antimalware device and one of the best free antivirus applications.
Feel free to visit my weblog: registry cleaner ( %url )

Mon, Mar 27, 2017
Just simply had to express I am just ecstatic that i came on your internet page!wholesale nfl jerseys ( %url )

Kirby Marino
Mon, Mar 27, 2017
Fantastic Web page, Continue the useful job. Thanks.
Take a look at my web-site; 7 Judi Bola Terbaik Indonesia ( %url )

Mon, Mar 27, 2017
Keep up the awesome work !! Lovin' it!
Feel free to visit my web blog :: Bandar Bola Terpercaya Indonesia ( %url )

Jeannie Royer
Mon, Mar 27, 2017
Hi there! I could have sworn I've been to this site before but after checking through some of the post I realized it's new to me. Nonetheless, I'm definitely delighted I found it and I'll be bookmarking and checking back frequently!
Here is my page Adidas zx FLUX ( %url )

Mon, Mar 27, 2017
Great looking website. Assume you did a bunch of your own coding.
My site :: domino ( %url )

Edison Oconnor
Mon, Mar 27, 2017
Hello there! I know this is kind of off topic but I was wondering if you knew where I could locate a captcha plugin for my comment form? I'm using the same blog platform as yours and I'm having difficulty finding one? Thanks a lot!
Have a look at my blog post see more ( %url )

Mitchell Greenup
Mon, Mar 27, 2017
El reloj digital hombre amazon ( %url ) Nabu es una herramienta de altas capacidades, rebosante de tecnología de vanguardia.

Remona Vandorn
Mon, Mar 27, 2017
Great website! It looks extremely professional! Sustain the good work!
Have a look at my site :: Kumpulan Agen Judi Bola Terpercaya ( %url )

Jillian Talley
Mon, Mar 27, 2017
The posts is amazingly useful.
My web-site; pieczatki ( %url )

Mon, Mar 27, 2017
Anadrol is an oral steroid that is still being produced today.
Feel free to surf to my site: get steroids ( %url )

Mon, Mar 27, 2017
Extremely interesting look onward to returning.
Look into my web page - Domain Hosting ( %url )

Albertina Speed
Sun, Mar 26, 2017
Thanks intended for furnishing these types of wonderful write-up.
cheap jerseys from china ( %url )

Kam Blundell
Sun, Mar 26, 2017
Great looking website. Think you did a great deal of your very own coding.wholesale jerseys ( %url )

Angelica Enyeart
Sun, Mar 26, 2017
Sustain the incredible work !! Lovin' it!
my blog post; avakin life hack avacoins ( %url )

Ashly Ehret
Sun, Mar 26, 2017
Un enorme fallo es arruinar el presente, recordando el pasado que ya no tiene futuro y en consecuencia no volvera, pero si tu lo revives solo servirá para arruinar tu presente, esta es la pero linda frases dibujos de amor para dibujar a lapiz paso a paso ( %url ) vida para que aprendas.

Sun, Mar 26, 2017
Every firm needs to have seo services miami ( %url ) solution pricing available for you and also other possible customers to information exactly how they work-- and also if they do not, it's an excellent sign that you need to locate Search Engine Optimization bundles prices elsewhere.

Sun, Mar 26, 2017
Most of the time, you don't think about calling an IT repair provider till something goes unsuitable with your laptop or desktop pc - and that is okay.
my web blog; malware cleaner ( %url )

Kassandra Born
Sun, Mar 26, 2017
Good day, good site you've got presently.
Feel free to visit my weblog: Bandar Bola Online Indonesia ( %url )

Records 1 - 25 of 1,001+

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