II 

113TH CONGRESS 

1ST SESSION S. 1223 

To amend the Public Health Service Act to expand and intensify programs 

of the National Institutes of Health and the Centers for Disease Control 

and Prevention with respect to translational research and related activities concerning cavernous angioma, and for other purposes. 

IN THE SENATE OF THE UNITED STATES 

JUNE 26, 2013 

Mr. UDALL of New Mexico (for himself and Mr. HEINRICH) introduced the 

following bill; which was read twice and referred to the Committee on 

Health, Education, Labor, and Pensions 

A BILL 

To amend the Public Health Service Act to expand and 

intensify programs of the National Institutes of Health 

and the Centers for Disease Control and Prevention with 

respect to translational research and related activities 

concerning cavernous angioma, and for other purposes. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Cavernous Angioma 

5 Research Resource Act of 2013’’. 

6 SEC. 2. FINDINGS. 

7 Congress makes the following findings: 


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1 (1) Cavernous angioma, also termed ‘‘cerebral 

2 cavernous malformations’’ or ‘‘CCM’’, affects an es3 timated 1,500,000 people in the United States. 

4 (2) Cavernous angioma is a devastating blood 

5 vessel disease that is characterized by the presence 

6 of vascular lesions that develop and grow within the 

7 brain and spinal cord. 

8 (3) Detection of cavernous angioma lesions is 

9 achieved through costly and specialized medical im10 aging techniques. These techniques are often not 

11 readily available where patients live, and require se12 dation for children and disabled adults. 

13 (4) Cavernous angioma is a common type of 

14 vascular anomaly, but individuals may not be aware 

15 that they have the disease until the onset of serious 

16 clinical symptoms. In the genetic forms, they may 

17 not be aware that it may be passed on to their chil18 dren. 

19 (5) Individuals diagnosed with cavernous 

20 angioma may experience neurological deficits, sei21 zure, stroke, or sudden death. 

22 (6) Due to limited research with respect to cav23 ernous angioma, there is no treatment regimen for 

24 the disease other than brain and spinal surgery. 


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1 (7) Some individuals with cavernous angioma 

2 are not candidates for brain surgery. No alternative 

3 treatment option is available for such individuals. 

4 (8) There is a shortage of physicians who are 

5 familiar with cavernous angioma and affected indi6 viduals may find it difficult to receive timely diag7 nosis and appropriate care. 

8 (9) Due to the presence of a specific disease9 causing mutation, termed the ‘‘common Hispanic 

10 mutation’’ that has passed through as many as 17 

11 generations of Americans descended from the origi12 nal Spanish settlers of the Southwest in the 1590s, 

13 New Mexico has the highest population density of 

14 cavernous angioma in the world. Cavernous angioma 

15 affects thousands of individuals in New Mexico and 

16 with ancestry in New Mexico. 

17 (10) Other States with high rates of cavernous 

18 angioma due to the common Hispanic Mutation in19 clude Texas, Arizona, and Colorado. 

20 (11) To address the public health threat posed 

21 by cavernous angioma in New Mexico and through22 out the United States, there is a need to identify in23 stitutions capable of running clinical trial for this 

24 debilitating brain disorder. 


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1 SEC. 3. CAVERNOUS ANGIOMA RESEARCH ACTIVITIES. 

2 Part B of title IV of the Public Health Service Act 

3 (42 U.S.C. 284 et seq.) is amended by adding at the end 

4 the following: 

5 ‘‘SEC. 409K. CAVERNOUS ANGIOMA RESEARCH ACTIVITIES. 

6 ‘‘(a) EXPANSION, INTENSIFICATION, AND COORDINA7 TION OF ACTIVITIES.—The Director of NIH, acting 

8 through the director of the National Institute of Neuro9 logical Disorders and Stroke, shall expand and intensify 

10 programs of the National Institutes of Health or may 

11 award grants and cooperative agreements to public or non12 profit private entities (including State health departments, 

13 political subdivisions of States, universities, and other edu14 cational entities) for research and related activities con15 cerning cavernous angioma. 

16 ‘‘(b) ACTIVITIES.—In expanding and intensifying 

17 programs under subsection (a), the Director of NIH may 

18 carry out the following: 

19 ‘‘(1) BASIC, TRANSLATIONAL, AND CLINICAL

20 RESEARCH.—Conduct or financially support basic, 

21 clinical, and translational research on cavernous 

22 angioma, including research on the following: 

23 ‘‘(A) Proteomic, pharmacological, and cell 

24 biological analysis of the cerebral cavernous 

25 malformations (referred to in this section as the 

26 ‘CCM’) molecules. 


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1 ‘‘(B) Continued development and expan2 sion of novel animal models for cavernous 

3 angioma preclinical research. 

4 ‘‘(C) Early detection, diagnosis, and treat5 ment of cavernous angioma. 

6 ‘‘(D) Biological mechanisms for lesion gen7 esis, development, and maturation. 

8 ‘‘(E) Biological mechanisms for lesion 

9 bleeding and symptomology. 

10 ‘‘(F) Novel biomedical and pharmacological 

11 interventions designed to prohibit new lesion de12 velopment, lesion growth, and lesion bleeding. 

13 ‘‘(G) Contributions of genetic variation to 

14 clinical presentation as targets for therapy. 

15 ‘‘(H) Identification and development of 

16 biomarkers to measure phenotypic variation. 

17 ‘‘(I) Research related to improving the 

18 quality of life for individuals with cavernous 

19 angioma and their families. 

20 ‘‘(J) Clinical training programs aimed at 

21 increasing the number of scientists and clini22 cians who are trained to treat patients and 

23 carry out these research directions. 

24 ‘‘(2) FACILITATION OF RESEARCH RESOURCES; 

25 CLINICAL TRIAL PREPAREDNESS.— 


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1 ‘‘(A) COORDINATION.—Identify and sup2 port the development of a clinical and research 

3 coordinating center with the potential of coordi4 nating a multi-site clinical drug trial for cav5 ernous angioma. Such coordinating center shall 

6 provide a model for additional trial sites, facili7 tate medical research to develop a cure for cav8 ernous angioma, and enhance the medical care 

9 of individuals with cavernous angioma nation10 wide. Such coordinating center shall— 

11 ‘‘(i) have an institutional infrastruc12 ture that is capable of hosting a clinical 

13 trial site and facilitating translational 

14 projects and collaborations for clinical 

15 trials; 

16 ‘‘(ii) have the capacity to maintain 

17 programs dedicated to patient education, 

18 patient outreach, and awareness, includ19 ing— 

20 ‘‘(I) launching a national multi21 media public awareness campaign; 

22 ‘‘(II) creating and distributing 

23 patient education materials for dis24 tribution by national physician and 

25 surgeon offices; 


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1 ‘‘(III) establishing an education 

2 program for elementary and sec3 ondary school nurses to facilitate early 

4 detection and diagnosis of cavernous 

5 angioma in areas of high cavernous 

6 angioma population density; 

7 ‘‘(IV) coordinating regular pa8 tient and family-oriented educational 

9 conferences; and 

10 ‘‘(V) developing nationally rel11 evant electronic health teaching and 

12 communication tools and a network of 

13 professional capacity and patient and 

14 family support; 

15 ‘‘(iii) have the capacity to establish 

16 and maintain communication with other 

17 major cavernous angioma research and 

18 care institutions internationally for infor19 mation sharing and coordination of re20 search activities; 

21 ‘‘(iv) have demonstrated clinical ex22 pertise in cavernous angioma management; 

23 ‘‘(v) have a sufficient number of eligi24 ble patients for participation with par25 ticular focus on unique subpopulations inVerDate Mar 15 2010 00:16 Jul 02, 2013 Jkt 029200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S1223.IS S1223

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1 cluding Common Hispanic Mutation and 

2 CCM3 gene mutation carriers; and 

3 ‘‘(vi) have a telehealth infrastructure 

4 to support and to provide clinical consulta5 tion for remote and underserved commu6 nities. 

7 ‘‘(B) PARTICIPATION.—Identify and sup8 port the development of clinical and research 

9 participation centers with the potential to par10 ticipate in a multi-site clinical drug trial for 

11 cavernous angioma. Such participation centers 

12 may facilitate medical research to develop a 

13 cure for cavernous angioma and enhance the 

14 medical care of individuals with cavernous 

15 angioma in partnership with the coordinating 

16 center under subparagraph (A) and other na17 tional and international centers. Such participa18 tion centers shall— 

19 ‘‘(i) have an institutional infrastruc20 ture capable of hosting a clinical trial site 

21 and facilitating translational projects and 

22 collaborations for clinical trials; 

23 ‘‘(ii) have the capacity to maintain 

24 communication with other major cavernous 

25 angioma research and care institutions 


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1 internationally for information sharing and 

2 coordination of research activities; 

3 ‘‘(iii) have demonstrated clinical ex4 pertise in cavernous angioma management; 

5 and 

6 ‘‘(iv) have a sufficient numbers of eli7 gible patients for participation with par8 ticular focus on unique subpopulations in9 cluding Common Hispanic Mutation and 

10 CCM3 gene mutation carriers as these 

11 unique populations may provide insight to 

12 other genetic and non-genetic forms of the 

13 illness. 

14 ‘‘(c) TRAINING PROGRAM FOR CLINICIANS AND SCI15 ENTISTS.— 

16 ‘‘(1) IN GENERAL.—Eligible coordinating and 

17 participation centers under this section shall estab18 lish or expand training programs for medical and al19 lied health clinicians and scientists in clinical prac20 tice and research relevant to cavernous angioma. 

21 ‘‘(2) RESEARCH RESOURCES.—In carrying out 

22 this subsection, the Director of NIH may— 

23 ‘‘(A) use information collected by the Na24 tional Institutes of Health pursuant to other 


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1 provisions of law or prior to the date of the en2 actment of this section; 

3 ‘‘(B) take into consideration the avail4 ability of other research resources; 

5 ‘‘(C) encourage the use of research re6 sources for research on, and development of, 

7 therapies and treatments for individuals with 

8 cavernous angioma; and 

9 ‘‘(D) encourage the inclusion of individuals 

10 with cavernous angioma in clinical trials con11 ducted or supported by the National Institutes 

12 of Health. 

13 ‘‘(3) CAVERNOUS ANGIOMA CONSORTIUM.—The 

14 Director of NIH may provide for the participation of 

15 agencies of the National Institutes of Health in a 

16 consortium to facilitate the exchange of information 

17 and to make the research effort on cavernous 

18 angioma more efficient and effective by ensuring 

19 consistent communication, minimizing duplication of 

20 effort, and integrating the varied perspectives of 

21 partner agencies, organizations, and individuals. 

22 Such consortium shall include at least one national 

23 cavernous angioma patient advocacy organization 

24 and may be the same consortium receiving a grant 

25 or contract under subsection (b)(2)(A).’’. 


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1 SEC. 4. CENTERS FOR DISEASE CONTROL AND PREVEN2 TION CAVERNOUS ANGIOMA SURVEILLANCE 

3 AND RESEARCH PROGRAMS. 

4 Part B of title III of the Public Health Service Act 

5 (42 U.S.C. 243 et seq.) is amended by inserting after sec6 tion 317T the following: 

7 ‘‘SEC. 317U. CAVERNOUS ANGIOMA SURVEILLANCE AND RE8 SEARCH PROGRAMS. 

9 ‘‘(a) IN GENERAL.—The Secretary, acting through 

10 the Director of the Centers for Disease Control and Pre11 vention, may award grants and cooperative agreements to 

12 public or nonprofit private entities (including State health 

13 departments, political subdivisions of States, universities, 

14 and other educational entities) for the collection, analysis, 

15 and reporting of data on cavernous angioma. In making 

16 such awards, the Secretary may provide direct technical 

17 assistance, including personnel support, in lieu of cash. 

18 ‘‘(b) NATIONAL CAVERNOUS ANGIOMA EPIDEMI19 OLOGY PROGRAM.— 

20 ‘‘(1) GRANTS.—The Secretary, acting through 

21 the Director of the Centers for Disease Control and 

22 Prevention, may award grants to public or nonprofit 

23 private entities (including State health departments, 

24 political subdivisions of States, universities, and 

25 other educational entities) for the purpose of car26 rying out epidemiological activities regarding cavVerDate Mar 15 2010 00:16 Jul 02, 2013 Jkt 029200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\S1223.IS S1223

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1 ernous angioma, including collecting and analyzing 

2 information on the number, incidence, correlates, 

3 and symptoms of cases and the clinical utility (in4 cluding costs and benefits) of specific practice pat5 terns. In making such awards, the Secretary may 

6 provide direct technical assistance, including per7 sonnel support, in lieu of cash. 

8 ‘‘(2) NATIONAL SURVEILLANCE PROGRAM.—In 

9 carrying out subsection (a), the Secretary shall— 

10 ‘‘(A) provide for a national surveillance 

11 program; and 

12 ‘‘(B) where possible, ensure that the sur13 veillance program is coordinated with the data 

14 and sample collection activities of the National 

15 Institutes of Health under section 409K.’’. 

16 SEC. 5. FOOD AND DRUG ADMINISTRATION CAVERNOUS 

17 ANGIOMA CLINICAL TRIAL PREPAREDNESS 

18 AND SUPPORT PROGRAM. 

19 (a) INVESTIGATIONAL NEW DRUG APPLICATION.— 

20 The Commissioner of Food and Drugs shall work with 

21 clinical centers, investigators, and advocates to support 

22 appropriate investigational new drug application under 

23 section 505(i) of the Federal Food, Drug, and Cosmetic 

24 Act in an effort to hasten the pace of clinical trials for 

25 cavernous angioma. 


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1 (b) ORPHAN PRODUCT DEVELOPMENT.—Where ap2 plicable in rare subpopulations of cavernous angioma re3 quiring unique pharmacological intervention, including 

4 those with the Common Hispanic Mutation or CCM3 gene 

5 mutations, the Commissioner of Food and Drugs shall 

6 support appropriate requests for designations of drugs as 

7 orphan drugs under section 526 of the Federal Food, 

8 Drug, and Cosmetic Act. 

9 SEC. 6. REPORT TO CONGRESS. 

10 Not later than January 1, 2015, and each January 

11 1 thereafter, the Secretary of Health and Human Services 

12 shall prepare and submit to the appropriate committees 

13 of the Congress a report concerning the implementation 

14 of this Act and the amendments made by this Act. 

Æ 


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