I 
115TH CONGRESS 
1ST SESSION H. R. 1255 
To increase research, education, and treatment for cerebral cavernous 
malformations. 
IN THE HOUSE OF REPRESENTATIVES 
FEBRUARY 28, 2017 
Mr. BEN RAY LUJA´N of New Mexico (for himself, Ms. MICHELLE LUJAN 
GRISHAM of New Mexico, and Mr. PEARCE) introduced the following bill; 
which was referred to the Committee on Energy and Commerce 
A BILL 
To increase research, education, and treatment for cerebral 
cavernous malformations. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Cerebral Cavernous 
Malformations Clinical Awareness, Research, and Edu-
cation Act of 2017’’ or the ‘‘CCM–CARE Act’’. 
SEC. 2. FINDINGS. 
Congress finds as follows: 
(1) Cerebral cavernous malformations (referred 
to in this section as ‘‘CCM’’), also known as cav-
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ernous angioma, or cavernoma, is a devastating 
blood vessel disease characterized by vascular lesions 
that develop and grow within the brain and spinal 
cord. 
(2) Detection of CCM lesions is achieved 
through costly and specialized medical imaging tech-
niques, often not accessible or convenient to patients 
who need them. 
(3) While CCM is a common type of vascular 
anomaly, many individuals are not aware they have 
the disease until the onset of serious clinical symp-
toms. CCM is often inherited unknowingly. 
(4) CCM affects an estimated 600,000 people 
in the United States. 
(5) Individuals diagnosed with CCM may expe-
rience neurological deficits, seizure, stroke, or sud-
den death. 
(6) Due to limited research, there is currently 
no treatment for CCM other than brain and spinal 
surgery, and only for certain patients. 
(7) There is also a shortage of trained physi-
cians to provide skilled and timely diagnosis and ap-
propriate treatment for CCM. 
(8) While the hereditary form of CCM may 
occur among any ethnicity, the presence of a muta-
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tion called the ‘‘common Hispanic mutation’’, has 
passed through seventeen or more generations of 
American descendants from the original Spanish set-
tlers of the Southwest in the 1590s. New Mexico has 
the highest population density of CCM in the world; 
Texas, Arizona, and Colorado also have high rates of 
CCM due to the common Hispanic mutation. 
SEC. 3. EXPANSION AND COORDINATION OF ACTIVITIES OF 
NATIONAL INSTITUTES OF HEALTH WITH RE-
SPECT 
TO 
CEREBRAL 
CAVERNOUS 
MAL-
FORMATIONS RESEARCH. 
Part B of title IV of the Public Health Service Act 
(42 U.S.C. 284 et seq.) is amended by adding at the end 
the following: 
‘‘SEC. 409K. CEREBRAL CAVERNOUS MALFORMATIONS RE-
SEARCH ACTIVITIES. 
‘‘(a) EXPANSION
AND COORDINATION
OF ACTIVI-
TIES.—The Director of NIH, in coordination with the di-
rectors of the National Institute of Neurological Disorders 
and 
Stroke, 
the 
National 
Center 
for 
Advancing 
Translational Sciences, the National Heart, Lung, and 
Blood Institute, and other national research institutes, as 
appropriate, for the purpose of conducting research and 
related activities concerning cerebral cavernous malforma-
tions (referred to in this section as ‘CCM’)— 
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‘‘(1) shall strengthen and coordinate efforts of 
the National Institutes of Health; and 
‘‘(2) may award grants and cooperative agree-
ments to public or nonprofit private entities (includ-
ing State health departments, political subdivisions 
of States, universities, and other medical or edu-
cational entities). 
‘‘(b) ACTIVITIES.—The research and related activi-
ties described in subsection (a) shall include the following: 
‘‘(1) CLINICAL, TRANSLATIONAL, AND
BASIC 
RESEARCH.—The Director of NIH shall conduct or 
support, through funding opportunity announce-
ments, grants, or cooperative agreements, basic, clin-
ical, and translational research on CCM, including 
research on— 
‘‘(A) the identification and development of 
biomarkers that fulfill the requirement of the 
Food and Drug Administration for biomarker 
qualification as proper measures of phenotypic 
variation; 
‘‘(B) safety or efficacy for new or 
repurposed currently approved drugs for CCM 
treatment; 
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‘‘(C) research related to improving the 
quality of life for individuals with CCM and 
their families; 
‘‘(D) contributions of genetic variation to 
clinical presentation as targets for therapy; 
‘‘(E) early detection, diagnosis, and treat-
ment of CCM; 
‘‘(F) clinical training programs aimed at 
increasing the number of scientists and clini-
cians who are trained to treat patients and 
carry out the research described in this para-
graph; 
‘‘(G) continued development and expansion 
of novel animal models for preclinical research 
relating to CCM; 
‘‘(H) pre-clinical and clinical research re-
lated to repurposing currently approved drugs 
for treatment of CCM; 
‘‘(I) proteomic, pharmacological, and cell 
biological analysis of CCM molecules; 
‘‘(J) biological mechanisms for lesion gen-
esis, development, and maturation; 
‘‘(K) biological mechanisms for lesion 
bleeding and symptomology; and 
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‘‘(L) novel biomedical and pharmacological 
interventions designed to inhibit new lesion de-
velopment, lesion growth, and lesion bleeding. 
‘‘(2) FACILITATION OF RESEARCH RESOURCES; 
CLINICAL TRIAL PREPAREDNESS.— 
‘‘(A) IN GENERAL.—The Director of NIH 
shall award grants and contracts to public or 
nonprofit private entities to fund all or part of 
the cost of planning, establishing, and providing 
basic operating support for a network of CCM 
Clinical Research Centers, including Coordi-
nating and Participating centers regarding re-
search on various forms of CCM. 
‘‘(B) CLINICAL AND RESEARCH COORDINA-
TION CENTERS.— 
‘‘(i) IN
GENERAL.—The Director of 
NIH shall identify and support the devel-
opment of 2 geographically distributed na-
tional clinical and research coordinating 
centers with unique clinical expertise and 
the potential for coordinating multi-site 
clinical drug trials with respect to CCM. 
‘‘(ii) DUTIES.—The coordinating cen-
ters identified under clause (i) shall pro-
vide a model for the participation centers 
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described in paragraph (3), facilitate med-
ical research to develop a cure for CCM, 
and enhance the medical care of individ-
uals with CCM nationwide, including by— 
‘‘(I) maintaining an institutional 
infrastructure capable of hosting clin-
ical trials and facilitating translational 
research projects and collaborations 
for clinical trials; 
‘‘(II) implementing the programs 
dedicated to patient education, patient 
outreach, and awareness developed by 
the Cerebral Cavernous Malformations 
Consortium 
under 
subsection 
(c)(3)(B); 
‘‘(III) developing the capacity to 
establish and maintain communication 
with other major CCM research and 
care institutions internationally for in-
formation sharing and coordination of 
research activities; 
‘‘(IV) demonstrating clinical ex-
pertise in the management of CCM 
and appointing a director and support 
staff, including a trainee and patient 
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representative, for CCM research pro-
gramming; 
‘‘(V) treating a sufficient number 
of eligible patients for participation 
with particular focus on unique sub-
populations, such as patients with the 
common Hispanic mutation, Ash-
kenazi Jewish mutation, or CCM3 
gene mutation carriers; and 
‘‘(VI) maintaining a telehealth 
infrastructure to support and provide 
clinical consultation for remote and 
underserved communities. 
‘‘(3) PARTICIPATION CENTERS.— 
‘‘(A) IN GENERAL.—The Director of NIH 
shall identify and support the development of 
approximately 6 to 10 clinical and research par-
ticipation centers to facilitate medical research 
to develop a cure for CCM and enhance the 
medical care of individuals with CCM, in part-
nership with the coordinating centers under 
paragraph (2) and other national and inter-
national entities, as appropriate. 
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‘‘(B) ELIGIBILITY.—To qualify for selec-
tion as a participation center under subpara-
graph (A), an entity shall— 
‘‘(i) at the time of selection— 
‘‘(I) be affiliated with an estab-
lished research network of the Na-
tional Institutes of Health; and 
‘‘(II) have the potential to par-
ticipate in a multisite clinical drug 
trial with respect to CCM; 
‘‘(ii) demonstrate— 
‘‘(I) an institutional infrastruc-
ture capable of hosting a clinical trial 
site 
and 
facilitating 
translational 
projects and collaborations for clinical 
trials; 
‘‘(II) the capacity to maintain 
communication with other major CCM 
research and care institutions inter-
nationally for information sharing and 
coordination of research activities, es-
pecially through health information 
technology; and 
‘‘(III) clinical expertise in CCM 
disease management or complete the 
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CCM clinical training program under 
subsection (c)(4); and 
‘‘(iii) have a sufficient number of eli-
gible patients with CCM. 
‘‘(C) DURATION OF SUPPORT.—The Direc-
tor of NIH may provide support for participa-
tion centers under this section for a period not 
to exceed 5 years. The Director of NIH may ex-
tend the period of support for a center for one 
or more additional periods, not to exceed an ad-
ditional 5 years, if the operations of such center 
have been reviewed by an appropriate technical 
and scientific peer review group established by 
the Director of NIH and if such group has rec-
ommended to the Director that such period 
should be extended. 
‘‘(c) CEREBRAL CAVERNOUS MALFORMATIONS CON-
SORTIUM.— 
‘‘(1) IN GENERAL.—The Director of NIH shall 
convene a Cerebral Cavernous Malformations Re-
search Consortium (referred to in this section as the 
‘consortium’). 
‘‘(2) MEMBERSHIP.—The consortium— 
‘‘(A) shall include representatives of— 
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‘‘(i) the coordinating centers selected 
under subsection (b)(2); and 
‘‘(ii) at least 1 national CCM patient 
advocacy organization, which may be an 
entity that receives a grant or contract 
under subsection (b)(2)(A); and 
‘‘(B) may include representatives of the 
National Institutes of Health or the Food and 
Drug Administration, in an advisory or ex offi-
cio role. 
‘‘(3) RESPONSIBILITIES.—Through a consensus 
based decisionmaking model, the consortium shall 
divide assignments and be responsible for— 
‘‘(A) developing and implementing training 
programs for clinicians and scientists in accord-
ance with paragraph (4); 
‘‘(B) developing patient education, out-
reach, and awareness programs and materials, 
which may be tailored for specific regional 
needs at coordinating centers, including— 
‘‘(i) a regional multimedia public 
awareness campaign; 
‘‘(ii) patient education materials for 
distribution by regional physician and sur-
geon offices; 
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‘‘(iii) an education program for ele-
mentary and secondary school nurses to fa-
cilitate early detection and diagnosis of 
CCM in areas in which there is a high den-
sity of cases of CCM; 
‘‘(iv) regular regional patient and 
family-oriented 
educational 
conferences; 
and 
‘‘(v) nationally relevant electronic 
health teaching and communication tools 
and a network of professional capacity and 
patient and family support; and 
‘‘(C) preparing a biannual report to Con-
gress, in accordance with paragraph (5). 
‘‘(4) TRAINING PROGRAM FOR CLINICIANS AND 
SCIENTISTS.— 
‘‘(A) IN
GENERAL.—The consortium, in 
cooperation with the coordinating centers, shall 
establish or expand a physician training pro-
gram, including information and education on 
advances in the diagnosis and treatment of 
CCM, and training and continuing education 
through programs for scientists, physicians, 
medical students, and other health professionals 
and care coordinators who provide care for pa-
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tients with CCM, telehealth, and research rel-
evant to CCM, for the purpose of supporting 
the development of new participation centers 
through educational programming to gain the 
expertise needed to become clinical and research 
participation centers with the potential to par-
ticipate in clinical drug trials. 
‘‘(B) STIPENDS.—The Director of NIH 
may provide stipends for health professionals 
who are enrolled in the training programs de-
scribed in subparagraph (A). 
‘‘(C) ELIGIBILITY.—To be eligible to par-
ticipate in the training program, an individual 
shall be affiliated with an entity that is in an 
existing clinical research network of the Na-
tional Institutes of Health. 
‘‘(5) REPORT TO CONGRESS.—The Director of 
NIH, on behalf of the consortium, shall biennially 
submit to the Committee on Health, Education, 
Labor, and Pensions of the Senate and the Com-
mittee on Energy and Commerce of the House of 
Representatives a report that describes the research, 
education, and other activities on CCM conducted or 
supported through the Department of Health and 
Human Services. Each such report shall include— 
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‘‘(A) a research plan; 
‘‘(B) provisions specifying the amounts ex-
pended by the Department of Health and 
Human Services with respect to various forms 
of CCM, including those affected by the com-
mon Hispanic Mutation, Ashkenazi Jewish mu-
tation, CCM3 gene mutations, and other famil-
ial and sporadic forms of cerebral cavernous 
malformation; and 
‘‘(C) 
recommendations 
for 
particular 
projects or types of projects that the national 
research institutes or other entities in the field 
of research should conduct on inherited or non- 
inherited forms of CCM.’’. 
SEC. 4. CENTERS FOR DISEASE CONTROL AND PREVEN-
TION CEREBRAL CAVERNOUS MALFORMA-
TIONS SURVEILLANCE AND RESEARCH PRO-
GRAMS. 
Part B of title III of the Public Health Service Act 
(42 U.S.C. 243 et seq.) is amended by inserting after sec-
tion 317T the following: 
‘‘SEC. 317U. CEREBRAL CAVERNOUS MALFORMATIONS SUR-
VEILLANCE AND RESEARCH PROGRAMS. 
‘‘(a) IN GENERAL.—The Secretary, acting through 
the Director of the Centers for Disease Control and Pre-
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vention, may award grants in such sums as may be nec-
essary and cooperative agreements to public or nonprofit 
private entities (including State health departments, polit-
ical subdivisions of States, universities, and other medical 
or educational entities) for the collection, analysis, and re-
porting of data on cerebral cavernous malformations (re-
ferred to in this section as ‘CCM’). 
‘‘(b) NATIONAL CEREBRAL CAVERNOUS MALFORMA-
TIONS EPIDEMIOLOGY PROGRAM.—The Secretary shall 
award grants and cooperative agreements, including tech-
nical assistance, to public or nonprofit private entities 
for— 
‘‘(1) the collection, analysis, and reporting of 
data on CCM; and 
‘‘(2) epidemiological activities, including col-
lecting and analyzing information on the number, in-
cidence, correlates, and symptoms of cases and the 
clinical utility of specific practice patterns. 
‘‘(c) NATIONAL
SURVEILLANCE
PROGRAM.—The 
Secretary shall— 
‘‘(1) provide for a national surveillance program 
for the purpose of carrying out epidemiological ac-
tivities regarding CCM, including collecting and ana-
lyzing information on the number, incidence, cor-
relates, and symptoms of cases of CCM and the clin-
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ical utility (including costs and benefits) of specific 
practice patterns; and 
‘‘(2) wherever possible, ensure that the surveil-
lance program is coordinated with the data and sam-
ple collection activities of the National Institutes of 
Health under section 409K. 
‘‘(d) TECHNICAL ASSISTANCE.—In making awards 
under this section, the Secretary may provide direct tech-
nical assistance, including personnel support. 
‘‘(e) COORDINATION WITH CLINICAL CENTERS.— 
The Secretary shall ensure that epidemiological informa-
tion is made available to clinical centers as supported by 
the Director of the National Institutes of Health under 
section 409K. 
‘‘(f) AUTHORIZATION OF APPROPRIATIONS.—There 
are authorized to be appropriated such sums as may be 
necessary to carry out this section.’’. 
SEC. 5. FOOD AND DRUG ADMINISTRATION CEREBRAL CAV-
ERNOUS MALFORMATIONS CLINICAL TRIAL 
PREPAREDNESS AND SUPPORT PROGRAM. 
(a) BIOMARKER QUALIFICATION PROGRAM.—The 
Secretary of Health and Human Services, acting through 
the Commissioner of Food and Drugs, shall coordinate 
with clinical centers, investigators, and advocates to sup-
port the qualification of appropriate surrogate biomarkers 
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in an effort to hasten the pace of clinical trials for cerebral 
cavernous malformation. 
(b) CLINICAL OUTCOME ASSESSMENT QUALIFICA-
TION.—The Secretary of Health and Human Services, act-
ing through the Commissioner of Food and Drugs, shall 
coordinate with clinical centers, investigators, and advo-
cates to support qualification of newly developed patient 
reported outcome measures for qualify of life as a clinical 
outcome in an effort to hasten the pace of clinical trials 
for cerebral cavernous malformation. 
(c) INVESTIGATIONAL NEW DRUG APPLICATION.— 
The Secretary of Health and Human Services, acting 
through the Commissioner of Food and Drugs, shall co-
ordinate with clinical centers, investigators, and advocates 
to support appropriate investigational new drug applica-
tions under section 505(i) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355(i)) in an effort to hasten 
the pace of clinical trials for cerebral cavernous malforma-
tion. 
(d) ADAPTIVE TRIAL DESIGN AND EXPEDITED RE-
VIEW PATHWAYS.—The Secretary of Health and Human 
Services, acting through the Commissioner of Food and 
Drugs, shall coordinate with clinical centers, investigators, 
and advocates to support appropriate adaptive trial de-
signs for rare disease research and expedited review mech-
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anisms for including Fast Track, Breakthrough Therapy 
Designation, Priority and/or Accelerated Review, where 
appropriate, in an effort to hasten the pace of clinical 
trials for cerebral cavernous malformation. 
Æ 
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