77884 Federal Register / Vol. 75, No. 239 / Tuesday, December 14, 2010 / Notices 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

National Institutes of Health 

Government-Owned Inventions; 

Availability for Licensing 

AGENCY: National Institutes of Health, 

Public Health Service, HHS. 

ACTION: Notice. 

SUMMARY: The inventions listed below 

are owned by an agency of the U.S. 

Government and are available for 

licensing in the U.S. in accordance with 

35 U.S.C. 207 to achieve expeditious 

commercialization of results of 

federally-funded research and 

development. Foreign patent 

applications are filed on selected 

inventions to extend market coverage 

for companies and may also be available 

for licensing. 

ADDRESSES: Licensing information and 

copies of the U.S. patent applications 

listed below may be obtained by writing 

to the indicated licensing contact at the 

Office of Technology Transfer, National 

Institutes of Health, 6011 Executive 

Boulevard, Suite 325, Rockville, 

Maryland 20852–3804; telephone: 301/ 

496–7057; fax: 301/402–0220. A signed 

Confidential Disclosure Agreement will 

be required to receive copies of the 

patent applications. 

Pyruvate Kinase M2 Activators for the 

Treatment of Cancer 

Description of Invention: NIH 

investigators have discovered a series of 

small compounds with the potential to 

treat a variety of cancers as well as 

hemolytic anemia. Contrary to most 

cancer medications, these molecules can 

be non-toxic to normal cells because 

they target a protein specific to the 

metabolic pathways in tumors, thus 

representing a significant clinical 

advantage over less-specific 

chemotherapeutics. 

The invention described here is a 

series of small molecules that activate 

pyruvate kinase (PK) isoform M2. PK– 

M2 is a critical metabolic enzyme that 

is affected in all forms of cancer. 

Inactivation of PK–M2 leads to a 

buildup of metabolic intermediates 

inside the cell. Tumor cells require a 

buildup of metabolic intermediates in 

order to undergo rapid cell growth and 

proliferation. Hence, activation of PK– 

M2 in tumor cells may prevent the 

buildup of metabolic intermediates and 

thereby stall tumor cell proliferation or 

destroy the tumor cells. Further, while 

in normal post-embryonic cells only PK 

isoforms R, L, or M1 are active, in all 

tumors only PK–M2 is active. So, PK– 

M2 activation would affect only tumor 

cells, and small-molecule PK–M2 

activators may not be toxic to healthy 

cells. 

This invention discloses the use of 

two new small molecule 

pharmacophores that can activate PKM2 

through the allosteric site: 3-oxo-3,4- 

dihydro-2H-benzo [b] [1,4] oxazine-7- 

sulfonamides, and 2-oxo-1,2,3,4- 

tetrahydroquinoline-6-sulfonamides. 

Applications: 

• Therapeutic developments for 

various cancers. 

• Diagnostic assays for various 

cancers. 

• Regulation of embryonic stem cell 

proliferation. 

Advantages: 

• Small molecule (series of analogs 

can be derived in search of improved 

performance). 

• Target a select group of cells 

(Cancerous cells). 

Development Status: 

• Pre-clinical; no animal data. 

• In vitro data available. 

Market: 

• Cancer-diagnostics. 

• Cancer-therapeutics. 

• Research tool-proliferation of 

embryonic stem cells and/or cancer 

cells. 

Inventors: Matthew Boxer (NHGRI– 

NCGC); Min Shen (NHGRI–NCGC); 

Doug Auld (NHGRI–NCGC); Craig 

Thomas (NHGRI–NCGC). 

Publications: 

1. Jiang JK et al. (2010) Bioorg Med 

Chem Lett 20:3387–93 [PubMed: 

20451379]. 

2. Boxer MB et al. (2010) J Med Chem. 

53:1048–55 [PubMed: 20017496]. 

Patent Status: U.S. Provisional 

Application No. 61/329,158 filled 29 

April 2010 (HHS Reference No. E–120– 

2010/0–US–01). 

Licensing Status: Available for 

licensing. 

Licensing Contact: Steven H. 

Standley, PhD; 301–435–4074; 

sstand@mail.nih.gov. 

Collaborative Research Opportunity: 

The NIH Chemical Genomics Center 

(NCGC), National Human Genome 

Research Institute, is seeking statements 

of capability or interest from parties 

interested in collaborative research to 

further develop, evaluate, or 

commercialize these pyruvate kinase 

M2 activators. Please contact Dr. 

Matthew Boxer at boxerm@mail.nih.gov 

for more information. 

Nitisinone for Treatment of 

Oculocutaneous/Ocular Albinism and 

for Increasing Pigmentation 

Description of Invention: Albinism 

(also called achromia, achromasia, or 

achromatosis) is a congenital disorder 

characterized by the complete or partial 

absence of pigment in the skin, hair and 

eyes due to absence or defect in any one 

of a number of proteins involved in the 

production of melanin. Certain forms of 

albinism are known to be due to 

mutations in tyrosine metabolism. In 

oculocutaneous albinism (OCA), 

pigment is lacking in the eyes, skin and 

hair. In ocular albinism, only the eyes 

lack pigment. Patients with albinism 

experience varying degrees of vision 

loss associated with foveal hypoplasia, 

nystagmus, photophobia and/or glare 

sensitivity, refractive errors, and 

abnormal decussation of ganglion cell 

axons at the optic chiasm. Current 

treatment options for vision problems 

caused by albinism are limited to 

correction of refractive errors and 

amblyopia, low vision aids, and (in 

some cases) extraocular muscle surgery. 

Nitisinone (NTBC) is an FDAapproved drug used in the treatment of 

tyrosinemia, type 1. The drug blocks the 

normal degradation pathway of tyrosine 

thus allowing greater circulating plasma 

levels of tyrosine. NIH investigators 

have identified that administration of 

NTBC to subjects (e.g., mice or humans) 

with certain forms of albinism, can 

result in increased circulating tyrosine 

levels, an increase in tyrosinase activity, 

and, subsequently, increased 

pigmentation. 

This technology provides methods for 

increasing tyrosine plasma 

concentrations in patients suffering 

from oculocutaneous albinism or ocular 

albinism by administering a 

pharmaceutically acceptable 

composition of NTBC. Specifically, this 

technology can be useful in treating 

patients with type OCA1a albinism, 

who possess no measurable tyrosinase 

activity, or type OCA1b albinism, who 

exhibit greatly diminished tyrosinase 

activity. 

Applications for this technology 

include treatment of impaired vision in 

patients suffering from oculocutaneous 

albinism, or ocular albinism, and as a 

treatment for increasing pigmentation in 

the eyes, hair and/or skin of patients. 

Inventors: Brian P. Brooks (NEI), 

David R. Adams (NHGRI), William A. 

Gahl (NHGRI). 

Patent Status: U.S. Provisional 

Application No. 61/308,771 filed 26 Feb 

2010 (HHS Reference No. E–113–2010/ 

0–US–01). 

Licensing Status: Available for 

licensing. 

Licensing Contact: Suryanarayana 

(Sury) Vepa, PhD, J.D.; 301–435–5020; 

vepas@mail.nih.gov. 

Collaborative Research Opportunity: 

The National Eye Institute, Ophthalmic 


srobinson on DSKHWCL6B1PROD with NOTICES
Federal Register / Vol. 75, No. 239 / Tuesday, December 14, 2010 / Notices 77885 

Genetics and Visual Function Branch, is 

seeking statements of capability or 

interest from parties interested in 

collaborative research to further 

develop, evaluate, or commercialize the 

use of nitisinone (NTBC) for 

oculocutaneous albinism or as a 

treatment for increasing pigmentation in 

the eyes, hair and/or skin of patients. 

Please contact Alan Hubbs, PhD at 301– 

594–4263 or hubbsa@mail.nih.gov for 

more information. 

Modulators of Survival Motor Neuron 

Production 

Description of Invention: This 

technology discloses compounds that 

modulate the amount of Survival Motor 

Neuron protein (SMN). Low levels of 

SMN protein are associated with Spinal 

Muscular Atrophy (SMA), which 

constitutes a group of inherited diseases 

that cause progressive muscle 

degeneration leading to death. 

Consequently, therapeutic inventions 

have focused on increasing SMN protein 

levels. This invention discloses novel 

arylthiazolyl piperidines which are 

shown to be modulators of SMN 

production. This invention also 

discloses methods of treating SMA by 

administering SMN protein modulators. 

Applications: Therapeutic 

developments for Spinal Muscular 

Atrophy. 

Advantages: Small molecule (series of 

analogs can be derived in search of 

improved performance). 

Development Status: 

• Pre-clinical; no animal data. 

• In vitro data available. 

Market: Muscular dystrophy. 

Inventors: Juan Jose Marugan 

(NHGRI–NCGC); Wei Zheng (NHGRI– 

NCGC); Noel Southall (NHGRI–NCGC); 

Jingbo Xiao (NHGRI–NCGC); Steve Titus 

(NHGRI–NCGC); Elliot Androphy 

(University of Massachusetts Medical 

School); Jonathan Cherry (University of 

Massachusetts Medical School). 

Patent Status: U.S. Provisional 

Application No. 61/323,963 filed 14 

April 2010 (HHS Reference No. E–109– 

2010/0–US–01). 

Licensing Status: Available for 

licensing. 

Licensing Contact: Steven H. 

Standley, PhD; 301–435–4074; 

sstand@mail.nih.gov. 

Collaborative Research Opportunity: 

The NIH Chemical Genomics Center 

(NCGC), National Human Genome 

Research Institute, is seeking statements 

of capability or interest from parties 

interested in collaborative research to 

further develop, evaluate, or 

commercialize these SMN modulator 

compounds. Please contact Dr. Juan 

Marugan at maruganj@mail.nih.gov for 

more information. 

Use of Sterculic Acid To Treat 

Choroidal Neovascularization 

Description of Invention: Sterculic 

acid is a naturally occurring 

cyclopropene acid present in kapok 

seed oil, cottonseed oil, and in the seeds 

of the Sterculia foetida tree. Sterculic 

acid has been reported to be a nonspecific inhibitor of stearoyl-Co 

desaturase (SCD), which has been 

implicated in several disease states, 

including cardiovascular disease, 

obesity, non-insulin-dependent diabetes 

mellitus, skin disease, hypertension, 

neurological diseases, immune 

disorders and cancer (Ntambi JM, J. 

Lipid Res., 1999, 40(9):1549–1558). NIH 

investigators have recently discovered 

that sterculic acid inhibits the 

neovascularization of the chick 

chorioallantonic membrane 

demonstrating that this compound 

exhibits a potent anti-angiogenic 

activity. Further, the NIH investigators 

have shown that sterculic acid inhibits 

the formation of choroidal 

neovascularization in the retina of laser 

treated rats. These results suggest that 

sterculic acid possesses anti-angiogenic 

effect likely through regulating genes 

involved in the angiogenic process. 

The present invention is directed to 

methods of using sterculic acid for the 

treatment of inflammation, in particular, 

7-ketocholesterol mediated 

inflammation, 7-ketocholesterol 

cytotoxicity, or unregulated 

angiogenesis. Diseases mediated by 7- 

ketocholesterol-induced inflammation 

and 7-ketocholesterol cytotoxicity 

include atherosclerosis age-related 

macular degeneration, and Alzheimer’s 

disease. Diseases mediated by 

unregulated angiogenesis include 

certain cancers and age-related macular 

degeneration. Also disclosed are 

methods of treating atherosclerosis or 

Alzheimer’s disease using sterculic acid. 

Applications: Therapeutics for 

inflammation, in particular, 

atherosclerosis, age-related macular 

degeneration, and Alzheimer’s disease 

Development Status: Early stage in 

vitro and animal model data. 

Inventors: Ignacio R. Rodriguez et al. 

(NEI). 

Patent Status: U.S. Provisional 

Application No. 61/358,485 filed 25 Jun 

2010 (HHS Reference No. E–092–2010/ 

0–US–01). 

Licensing Status: Available for 

licensing. 

Licensing Contact: Suryanarayana 

Vepa, PhD, J.D.; 301–435–5020; 

vepas@mail.nih.gov. 

Collaborative Research Opportunity: 

The National Eye Institute (NEI), 

Laboratory of Retinal Cell and 

Molecular Biology, is seeking statements 

of capability or interest from parties 

interested in collaborative research to 

further develop, evaluate, or 

commercialize sterculic acid, and its 

derivatives for the treatment of diseases 

related to angiogenesis or mediated by 

7-ketocholesterol-induced 

inflammation. Please contact David L. 

Whitmer, Technology Development 

Coordinator, NEI, at 301–496–4876 or 

whitmerd@mail.nih.gov for more 

information. 

Dated: December 8, 2010. 

Richard U. Rodriguez, 

Director, Division of Technology Development 

and Transfer, Office of Technology Transfer, 

National Institutes of Health. 

[FR Doc. 2010–31320 Filed 12–13–10; 8:45 am] 

BILLING CODE 4140–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

National Institutes of Health 

Government-Owned Inventions; 

Availability for Licensing 

AGENCY: National Institutes of Health, 

Public Health Service, HHS. 

ACTION: Notice. 

SUMMARY: The inventions listed below 

are owned by an agency of the U.S. 

Government and are available for 

licensing in the U.S. in accordance with 

35 U.S.C. 207 to achieve expeditious 

commercialization of results of 

federally-funded research and 

development. Foreign patent 

applications are filed on selected 

inventions to extend market coverage 

for companies and may also be available 

for licensing. 

ADDRESSES: Licensing information and 

copies of the U.S. patent applications 

listed below may be obtained by writing 

to the indicated licensing contact at the 

Office of Technology Transfer, National 

Institutes of Health, 6011 Executive 

Boulevard, Suite 325, Rockville, 

Maryland 20852–3804; telephone: 301/ 

496–7057; fax: 301/402–0220. A signed 

Confidential Disclosure Agreement will 

be required to receive copies of the 

patent applications. 

Software System for Quantitative 

Assessment of Vasculature in Three 

Dimensional Images 

Description of Invention: 

This invention offered for licensing 

and further development is a software 

system that provides the capability of 


srobinson on DSKHWCL6B1PROD with NOTICES