PKU families ask Premier to invest in Comprehensive Brain Protection Strategy


- Funding solution aims to end three-year refusal to provide access to treatment and care -

VANCOUVER, Feb. 23, 2013 /CNW/ - After more than three years of being denied access to the care and treatment needed to protect the brains of 170 patients in B.C. with phenylketonuria (PKU), the Canadian PKU and Allied Disorders Inc. (CanPKU) is asking Premier Christy Clark to invest in a funding solution that will bring PKU care in the province up to national, and international, standards and best practices. The B.C. government has been screening every newborn baby for PKU since the 1960s,1 to allow treatment to begin immediately after the disease is detected; however, many treatment options aren't accessible to PKU patients who need them.

Today, at the annual B.C. PKU Day, families and patients living with the rare, inherited and brain-threatening metabolic disorder gathered to reconnect with the community and learn about developments in PKU treatment and care from international experts. But the issue top-of-mind for many attending the meeting, including Sparwood, B.C. resident Nicole Pallone, is the government's refusal to fund the basic treatments needed to protect the brains of PKU patients like Pallone's five-year-old daughter, Rosie.

"We are shocked and appalled that the B.C. government continues to put the health of our community at risk by denying patients access to evidence-based, proven treatments that are the standard of care in most developed countries," says Pallone, who is Vice President of CanPKU. "For the past three years, the B.C. Ministry of Health has not only turned its back on PKU patients, but has deliberately and systematically avoided providing basic treatment for PKU patients in this province - the same treatments that other governments right here in Canada provide to their citizens."

Standard of PKU treatment in B.C. stagnant for 50 years

Despite exhaustive efforts by the PKU community, British Columbia is the worst province in Canada for access to PKU treatments. The only public funding that B.C. PKU patients receive is for special low-protein formula,2 which has formed the basis of the complicated and highly-restrictive PKU diet for the past 50 years. 3

Numerous provinces provide access to medical foods for PKU patients - a key treatment necessary to prevent devastating neurocognitive, psychiatric and physical symptoms caused by the disease. However, B.C. only provides the bare minimum of coverage for the medical foods necessary to manage PKU - a nominal $40 monthly stipend, available only to PKU patients on social assistance.2

Three years ago, when Health Canada approved Kuvan (sapropterin) as the first and only drug therapy for this brain-threatening disease,4 CanPKU began calling upon provincial and territorial governments to fund the treatment for the 20 to 56 per cent of patients who respond to it. While there has been positive movement towards funding agreements in some provinces, such as Ontario where Kuvan will be publicly funded as of February 28, 2013, B.C. is the only government to walk away from negotiations with the drug's manufacturer.

However, the B.C. government does fund Kuvan to treat BH4 deficiency (originally called malignant PKU), and as a diagnostic tool to determine whether newborns with high Phe levels have PKU or BH4 deficiency.

"There is no question in my mind that PKU patients who have access to the treatment combination of formula, medical foods and medication have the greatest likelihood of healthy, productive lives, with minimized negative health impacts," says Dr. Barbara Burton, director of the PKU Clinic at the Ann and Robert H. Lurie Children's Hospital in Chicago, who addressed B.C. PKU Day delegates on Saturday. "Many countries around the world have accepted the body of clinical evidence and professional consensus on the sustained health benefits of Kuvan and medical foods, and are making those treatments accessible to patients."

Call for investment in Comprehensive Brain Protection Strategy

Faced with the B.C. government's continued indifference and non-responsiveness to CanPKU's requests for funding, CanPKU has taken a proactive approach by developing a policy paper to demonstrate to government the minimal investment required to make a tangible difference in the lives of PKU patients.

In a policy paper titled, A Comprehensive Brain Protection Strategy for People with PKU: Getting BC from Worst to First, CanPKU has asked for an annual $2.8 million investment by the B.C. government to improve treatment and care for PKU patients. Submitted to the Premier and the Minister of Health on Friday, February 22, the paper is composed of cost-effective, sustainable and realistic solutions to ensure those British Columbians living with PKU have access to all the necessary resources and treatments.

With this modest investment, CanPKU proposes that B.C.'s medical food subsidy be brought up to standards comparable to Alberta, Saskatchewan, Quebec and Ontario. It would also allow for the government to provide dedicated funding for proven, innovative therapies, like Kuvan. Additionally, the investment would enable a full-time psychologist, PKU Clinic Coordinator, nurse and a part-time dietitian to be added to the PKU/metabolic clinic team at B.C. Children's Hospital.

"We want to show that we're still willing to cooperate with government to ensure protection for patients from the devastating health impacts associated with PKU, while respecting budgetary constraints," says John Adams, President and CEO of CanPKU, whose adult son has PKU. "There is nothing unreasonable about our request - in fact, we think it's a no brainer. This investment would represent about 0.1 per cent of the $2.4 billion in new health care funding over the next three years that was just announced in B.C."

If approved, funding for the Comprehensive Brain Protection Strategy would be phased in over a three-year period to allow the government time to more easily absorb the costs. CanPKU hopes that the 170 British Columbians living with PKU would then finally have access to all the available resources and treatments that will improve their health outcomes and quality of life.

Adams adds that the longer funding is delayed, the longer the government is needlessly putting the brains of PKU patients - from infants to adults - at risk.  "The time is now for the Premier to make good on her stated commitment to 'Families First' by working with us to bring treatment for PKU patients in this province from worst to first."

About PKU
PKU (phenylketonuria) is a rare inherited, brain-threatening metabolic disorder, observed when the body is unable to process phenylalanine ("Phe"), an essential amino acid found in dietary protein.  The resulting accumulation of Phe in the blood is toxic to the brain, and if left untreated, symptoms can range from mild cognitive impairment to severe mental retardation.5,6 Approximately 1 in 12,000 to 15,000 infants in Canada is born with PKU.7

About Canadian PKU and Allied Disorders Inc.
Canadian PKU and Allied Disorders Inc. is a non‐profit association of volunteers, dedicated to providing accurate news, information and support to families and professionals dealing with PKU and similar, rare, inherited metabolic disorders. Our mission is to improve the lives of people with PKU and allied disorders and the lives of their families. By allied disorders we mean other rare, inherited metabolic disorders also detected by newborn screening. For more information, visit and our new comprehensive resource for patients and families, PKU and the Brain.

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1 Canada lags on newborn screening. Canadian Medical Association. Available at Accessed on January 18, 2012.
2 Canadian PKU and Allied Disorders Inc. Coverage for PKU Diet Formulas & Medical Foods - May 2012. Accessed on January 29, 2013. Available at:
3 Singh RH, Quirk ME. Using change in plasma phenylalanine concentrations and ability to liberalize diet to classify responsiveness to tetrahydrobiopterin therapy in patients..., Mol. Genet. Metab. (2011), doi:10.1016/j.ymgme.2011.09.009
4 Kuvan [product monograph]. Toronto, ON: BioMarin Pharmaceutical (Canada) Inc.; 2010.
5 Leuret O, Barth M, Kuster A et al. Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuria. J Inherit Metab Dis, 2012; DOI 10.1007/s10545-012-9464-3.
6 Martynyuk A et al., Epilepsy in Phenylketonuria: A Complex Dependence on Serum Phenylalanine Levels. Epilepsia. 2007, 48(6):1143-50
7 Arnold GL. Phenylketonuria. 2009;

Video with caption: "Families appeal for access to treatments for Canadian PKU patients". Video available at:

Image with caption: "Poster - Premier Clark: To Harm or Protect? (CNW Group/Canadian PKU and Allied Disorders Inc.)". Image available at:

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SOURCE: Canadian PKU and Allied Disorders Inc.

For further information:

Beth Daniher
Cohn & Wolfe
416-924-5700 ext. 4070 (office)
416-417-8093 (mobile)

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